Healthcare Utilization and Health Related Quality of Life in Persons with Von Willebrand Disease

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 3-4
Author(s):  
Jonathan C. Roberts ◽  
Roshni Kulkarni ◽  
Peter A. Kouides ◽  
Robert F. Sidonio ◽  
Shannon L Carpenter ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Healthcare Utilization ◽  
Research Funding ◽  
Von Willebrand Disease ◽  
Advisory Committees ◽  
Related Quality ◽  
Joint Health ◽  
Health Related ◽  
The Impact

Background: von Willebrand disease (VWD) is the most common inherited bleeding disorder. Questions remain regarding the impact of VWD related bleeding phenotype on healthcare utilization, joint health, and health-related quality of life (HRQoL) in the US. Objective: Our study investigated the impact of VWD bleeding phenotype on healthcare utilization, joint health, and HRQoL in a geographically diverse cohort of individuals with VWD who obtain care at seven US Hemophilia Treatment Centers (HTCs). Methods: Hematology Utilization Group Studies (HUGS) prospectively examined the cost and burden of illness in persons with VWD. The current study enrolled individuals age ≥12 with VWD Type 1 (VWF:Ag/RCo: ≤30%), low VWF (VWF:Ag/RCo: 30-50%), Type 2, and 3. Participants completed a standardized interview to collect sociodemographic and clinical data, self-reported healthcare utilization and bleeding in last 6 months, self-reported pain, joint health and HRQoL measured by EQ-5D-3L. Clinical chart reviews abstracted information about VWD type and treatment. Association of bleeding and VWD type with healthcare utilization, joint health, and HRQoL were assessed using Chi-square or Fisher exact tests for categorical variables and Student T-tests or one-way ANOVA for continuous variables. P value ≥0.05 indicates not statistically significant (NS). Results: We analyzed 100 participants with complete baseline information. Mean age was 31.7 (SD=18.6) years, 67.0% were adults ≥18 years, 80.0% were female, 67.7% had Type 1/low VWF, and 3.0% had Type 3 VWD. Mean age at VWD diagnosis was 13.6 (SD=13.0) years. Persons with low VWF were diagnosed and received VWD treatment at an older age (mean 19.2, SD=11.8; 19.4, SD=11.9 years for diagnosis and treatment respectively) than those with Type 1 (13.7, SD=12.7; 15.6, SD=13.7), or Types 2&3 (9.4, SD=12.9; 13.3, SD=16.6), p=0.03 and p=NS. As compared to individuals without bleeding in the previous 6 months, those reported bleeding had significantly higher rate of medical procedures related to treating bleeding events (42.5% vs. 13.3%, p=0.001), and overnight hospitalization (20.0% vs. 3.3%, p<0.01). The individuals with recent bleeding also reported higher proportion of emergency room visits (25.0% vs. 11.9%, p=NS), physician visits (55.0% vs. 38.3%, p=NS), joint pain (48.7% vs. 40.0%, p=NS), and joint range of motion limitation (35.9% vs. 20.0%, p=NS) than those without bleeding, although the differences were not statistically significant. Those with recent bleeding reported more problems in EQ-5D mobility (33.3% vs. 15.0%, p=0.03) and usual activities (41.0% vs. 26.7%, p=NS) than those without recent bleeding. EQ VAS (71.6 vs. 75.2, p=NS) and EQ index scores (0.79 vs. 0.83, p=NS) were not significantly different between persons with and without recent bleeding. Those with Types 2&3 VWD were more likely to have hospitalization (25.0% vs. 5.0%, 2.1%, p<0.01), bleeding (50.0% vs. 35.0%, 36.2%, p=NS), joint pain (53.1% vs. 30.0%, 41.3%, p=NS), joint range of motion limitation (40.6% vs. 20.0%, 19.6%, p=NS) than those with low VWF or Type 1 VWD. Individuals with low VWF (50.0%) reported higher rate of anxiety or depression per EQ-5D than Type 1 (34.8%) or Type 2&3 (40.6%), p=NS. Covariate-adjusted EQ-5D VAS or index score were lower among persons with low VWF (64.7±8.2 for VAS, 0.76±0.07 for EQ-index) than those with Type 1 (77.3±7.6, 0.84±0.06) or Types 2&3 (78.7±7.4, 0.84±0.66), p values=NS. Conclusions: Our study demonstrates that persons with VWD who obtain care at US HTCs experience significant illness burden regardless of severity. Self-reported recent bleeding as expected was associated with increased healthcare utilization and negative impact on joint health and HRQoL. Bleeding phenotype was significantly associated with healthcare utilization differences. Delayed diagnosis and treatment for persons with low VWF may impact their HRQoL, and if confirmed in a larger sample size would underscore the fact that low VWF is not necessarily a mild disorder compared to other VWD subtypes. Disclosures Roberts: Octapharma: Consultancy, Speakers Bureau; Sanofi: Consultancy, Speakers Bureau; Novo Nordisk: Consultancy, Speakers Bureau; Pfizer: Consultancy; Takeda: Consultancy, Research Funding, Speakers Bureau; uniQure: Consultancy. Kulkarni:Bioverativ/Sanofi, BPL, Genentech, Kedrion, Novo Nordisk, Octapharma, Pfizer, Takeda, Catalyst Bioscience Bayer: Membership on an entity's Board of Directors or advisory committees; Sanofi/ Bioverativ, Bayer, Biomarin, Shire/Takeda, Novo Nordisk, Freeline: Other: clinical trial research grants . Sidonio:Octapharma, Grifols, Takeda and Genentech: Research Funding; Bayer, Bioverativ/Sanofi, Novo Nordisk, Takeda, Uniqure, Biomarin, Octapharma, Catalyst, Grifols, Sigilon, Tremeau, Genentech/Roche: Consultancy. Carpenter:Kedrion: Honoraria; Novo Nordisk: Honoraria; Genentech, Inc.: Honoraria; American Thrombosis and Hemostasis Network: Membership on an entity's Board of Directors or advisory committees; CSL Behring: Research Funding; Shire: Research Funding; Hemostasis & Thrombosis Research Society: Membership on an entity's Board of Directors or advisory committees; American Academy of Pediatrics: Other: PREP Heme/Onc editorial board. Konkle:Sigilon: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding; BioMarin: Consultancy; Sanofi: Consultancy, Research Funding; Takeda: Research Funding; Uniquire: Research Funding; CSL Behring: Consultancy; Roche: Consultancy; Baxalta: Research Funding; Spark: Consultancy, Research Funding. Wu:Baxalta US Inc., Bannockburn, IL (a Takeda Company), CSL Behring L.L.C., and Octapharma USA, Inc.: Research Funding. Curtis:USC Hemophilia Utilization Group Study (HUGS): Consultancy; Bayer: Consultancy; Novo Nordisk: Consultancy; Patient Reported Outcomes, Burdens and Experiences: Consultancy. Carrasco:Baxalta US Inc., Bannockburn, IL (a Takeda Company), CSL Behring L.L.C., and Octapharma USA, Inc.: Research Funding. Tran:Novo Nordisk: Consultancy; Bioverativ: Consultancy; Takeda: Consultancy; Bayer: Consultancy. Nichol:Global Blood Therapeutics: Research Funding; Octapharma: Research Funding; CSL Behring: Research Funding; Pfizer: Research Funding; Baxalta US Inc., Bannockburn, IL (a Takeda Company): Research Funding; Genentech Inc.: Research Funding.

scholarly journals Health Related Quality of Life and Fatigue Improve on Second Line Treatments in Pediatric Immune Thrombocytopenia (ITP)

Blood ◽  
2017 ◽  
Vol 130 (Suppl_1) ◽  
pp. 752-752
Author(s):  
Rachael F. Grace ◽  
Robert J Klaassen ◽  
Rukhmi Bhat ◽  
Michelle Neier ◽  
Melissa J. Rose ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Research Funding ◽  
Line Treatment ◽  
Second Line ◽  
Advisory Committees ◽  
Related Quality ◽  
Health Related ◽  
Effect Of Treatment ◽  
The Impact ◽  
Parent Proxy

Abstract Background: The impact of second line therapies on health related quality of life (HRQoL) and fatigue in pediatric patients with ITP is not well studied. Objective: To describe the impact of second line therapies on HRQoL and fatigue in North American pediatric patients with ITP. Methods: A longitudinal observational cohort of 120 children with ITP starting second line treatments was enrolled from 2013-2015 at 21 ICON centers. Enrollment requirements included age 1-17y and starting a second line treatment (not IVIG, corticosteroids or anti-D immunoglobulin) as monotherapy. HRQoL (Kids ITP Tool - KIT) and fatigue (Hockenberry Fatigue Scale - FS) surveys were completed prior to starting treatment (baseline) and 1 and 12 months after starting treatment by patient/caregiver. KIT is scored from 0 (worst) to 100 (best), and the FS scores were re-scaled so that 0 is no fatigue and 100 is highest fatigue. At the same time points as the patient/caregiver surveys, physicians assessed the perceived effect of treatment on patient HRQoL using a 7-point scale. ANOVA was used to compare the baseline means of the treatment groups. This study specifically compared change from baseline to 1 month in the KIT and FS using paired t-tests within each treatment group. The 12 month timepoint was not used in the paired analysis of individual treatments due to attrition between 1 and 12 months. Results: The median age at enrollment was 11.3 y (1.2-17.8), and 16% (19/120) had newly diagnosed ITP, 31% (37/120) had persistent ITP, and 53% (64/120) had chronic ITP. The median number of prior treatments was 3 (range: 1-9). Fifty-eight (48%) patients had received at least one prior second line treatment. Treatments selected for second line treatment included: rituximab (n=43), romiplostim (n=31), eltrombopag (n=20), oral immunosuppressants (n=19), splenectomy (n=4), and dapsone (n=3). The child and parent proxy KIT scores significantly improved on rituximab (p<0.001 for both), oral immunosuppressants (p=0.02, p=0.001), and eltrombopag (p=0.01 for both). Child KIT scores also significantly improved on romiplostim (p=0.003); however, there was no significant change in the parent proxy score (p=0.29). The parent impact KIT scores significantly improved from baseline to 1 month on all treatments (p<0.001), although the scores were not significantly different between treatment types (p=0.67). Child, parent proxy, and parent impact KIT scores significantly increased between 1 month and 12 months in paired analysis combining treatments (p<0.001). As previously described, at enrollment, physicians reported that ITP had impacted the patients' HRQoL severely in 15%, significantly in 45%, moderately in 38%, and almost not at all in 3%. Physicians reported that HRQoL improved in 68% (range: 64-75%) of patients while on treatment from baseline to 1 month with no significant difference by treatment group (p=0.46). The physician's assessment of the patient's baseline HRQoL significantly correlated with the child and parent proxy KIT report (p<0.0001); however, after 1 month of treatment, the physician's assessment no longer correlated with the child (p=0.26) or parent proxy KIT report (p=0.11). At enrollment, the median FS-Child score (n=54) was 18.5 (range 0-85), the median FS-Adolescent score (n=42) was 20.2 (0-73), and the median FS-Parent (n= 100) score was 35 (7-81). One month FS-Child improved for those who were treated with rituximab (p=0.03); there was no significant change in fatigue on the other treatments. One month FS-Parent significantly improved for those treated with rituximab (p=0.015) and eltrombopag (p=0.009). Conclusions: In this pediatric cohort, all second line treatments appear to significantly improve HRQoL in ITP. Rituximab had the greatest impact in decreasing fatigue at one month. Physician assessment of patient HRQoL did not correlate well with patient assessment after treatment was started, suggesting there may be challenges in ascertaining the effect of treatment on HRQoL. Future analysis of ICON1 will consider the impact of treatment on HRQoL and fatigue while also accounting for the treatment effect on bleeding and platelet count. Disclosures Grace: Agios: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding. Klaassen: Amgen: Consultancy; Hoffman-La Roche LTD: Consultancy; Octapharma: Honoraria; Baxalta: Honoraria; Biogen Canada LTD: Consultancy; Agios Pharmaceuticals: Consultancy. Despotovic: Sanofi: Consultancy; Schell Cooley LLP: Other: Expert witness. Bussel: Protalex: Membership on an entity's Board of Directors or advisory committees, Research Funding; Momenta: Membership on an entity's Board of Directors or advisory committees; Rigel: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding; Amgen: Membership on an entity's Board of Directors or advisory committees, Research Funding. Rothman: Pfizer: Consultancy; Agios Pharmaceuticals: Honoraria. Haley: Genentech: Honoraria; Baxalta: Honoraria; CSL Behring: Honoraria. Neufeld: Octapharma: Consultancy, Honoraria; Genentech: Consultancy, Membership on an entity's Board of Directors or advisory committees; Novartis: Membership on an entity's Board of Directors or advisory committees. Lambert: Educational Concepts in Medicine: Honoraria; Novartis: Honoraria; AstraZeneca: Research Funding.

Variation in Health-Related Quality of Life by Age Among Patients with Chronic Lymphocytic Leukemia

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 2085-2085
Author(s):  
Chris L. Pashos ◽  
Christopher R Flowers ◽  
Mark Weiss ◽  
Nicole Lamanna ◽  
Charles M Farber ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Research Funding ◽  
Age Groups ◽  
Lymphocytic Leukemia ◽  
Age Group ◽  
Employment Equity ◽  
Advisory Committees ◽  
Related Quality ◽  
Health Related ◽  
Equity Ownership

Abstract Abstract 2085 Introduction. Although advanced patient age is commonly used as a factor in selecting therapy for patients with chronic lymphocytic leukemia (CLL), based on presumed associations with functional status, limited data exist regarding the relationships between age and physical, emotional, social, and functional well being. We examined the relationships between age and these domains of health-related quality of life (HRQOL) for CLL patients treated in US community practices. Methods. Baseline data were collected as part of Connect CLL®, a prospective observational registry initiated in March 2010 involving centers in the US. Data on patient demographics and clinical characteristics were provided by clinicians. HRQOL was self-reported by patients in the clinic at enrollment. Patients completed 3 psychometrically validated instruments: the Brief Fatigue Inventory (BFI), EQ-5D, and Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu). Standard analyses were conducted of each instrument given clinical characteristics at that time. Reported mean BFI, EQ-5D and FACT-Leu scores were analyzed by age group (<65, 65–74, >74). Statistical significance of score differences among sub-cohorts was ascertained by ANOVA using SAS 9.1. Results. Baseline HRQOL data were reported by 604 patients, enrolled from 161 centers. Patients were predominantly male (62%) and white (90%) with mean age at 69.9 (standard deviation [SD] 11.2) yrs. HRQOL scores by age group are presented: There were no significant differences between the age groups in fatigue as measured by the BFI, or differences in overall HRQOL as measured by the EQ-5D Visual Analogue Scale (VAS) or the FACT-G. Anxiety/depression and self care are EQ-5D domains that also did not vary by age. Although mobility was most impaired in the oldest age group compared to the two younger groups, usual activities and pain/discomfort were worse in both the younger and older cohorts compared to those 65–74 years of age. FACT-Leu results indicated that the social/family domain scores did not vary by age, but that physical, emotional, and functional domains did vary statistically with the oldest typically doing better than the 65–74 year olds, but not necessarily better than those <65. Conclusions. Initial results from the Connect CLL® Registry indicate that HRQOL does not worsen monotonically with older age. In this cohort, both the youngest and oldest age groups had worse HRQOL in certain domains, presenting an inverted v-shaped relationship. Future analyses should be conducted on: (1) how HRQOL may be affected over time with changes in disease; and, (2) how HRQOL may be influenced by alternative therapies. Results reported here should serve as a useful baseline reference. Disclosures: Pashos: Celgene: Membership on an entity's Board of Directors or advisory committees. Flowers:Genentech/Roche (unpaid): Consultancy; Celgene: Consultancy; Millennium/Takeda: Research Funding; Wyeth: Research Funding; Novartis: Research Funding. Weiss:Celgene: Membership on an entity's Board of Directors or advisory committees. Lamanna:Celgene: Membership on an entity's Board of Directors or advisory committees. Farber:Celgene: Membership on an entity's Board of Directors or advisory committees. Kipps:Igenica: Equity Ownership, Membership on an entity's Board of Directors or advisory committees; Celgene: Consultancy, Research Funding; Abbot Industries: Research Funding; Pharmacyclics: Membership on an entity's Board of Directors or advisory committees; Genentech: Research Funding; GSK: Research Funding; Gilead Sciences: Consultancy, Research Funding; Amgen: Research Funding. Lerner:Celgene: Membership on an entity's Board of Directors or advisory committees. Kay:Celgene: Membership on an entity's Board of Directors or advisory committees. Sharman:Celgene: Membership on an entity's Board of Directors or advisory committees. Grinblatt:Celgene: Membership on an entity's Board of Directors or advisory committees. Flinn:Celgene: Membership on an entity's Board of Directors or advisory committees. Kozloff:Celgene: Membership on an entity's Board of Directors or advisory committees. Swern:Celgene Corporation: Employment, Equity Ownership. Kahn:Celgene Corporation: Employment, Equity Ownership. Street:Celgene: Employment, Equity Ownership. Sullivan:Celgene: Employment, Equity Ownership. Keating:Celgene: Membership on an entity's Board of Directors or advisory committees.

scholarly journals Health-Related Quality of Life in a Biologic Assignment Trial of Reduced Intensity Hematopoietic Cell Transplantation Based on Donor Availability in Patients Aged 50-75 with Advanced Myelodysplastic Syndrome

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 421-421
Author(s):  
Rachel Cusatis ◽  
Michael Martens ◽  
Ryotaro Nakamura ◽  
Corey Cutler ◽  
Wael Saber ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Research Funding ◽  
Health Related Quality ◽  
Advisory Committees ◽  
No Donor ◽  
Free Survival ◽  
Related Quality ◽  
Health Related

Abstract Introduction The Blood and Marrow Transplant Clinical Trials Network study (BMT CTN 1102, NCT02016781) was a multicenter, biologic assignment trial in older adults aged 50-75 with higher risk de novo MDS (IPSS Intermediate-2 or High) who were candidates for reduced-intensity conditioning (RIC) allogeneic HCT. The trial compared outcomes of those with a suitable HLA-identical sibling or unrelated donor (Donor arm) to those where no donor was identified (No Donor arm) within a search window of 90 days. The trial reported a survival benefit for patients in the Donor arm compared to the No Donor arm [Nakamura et al, JCO 2021, in press]. Here, we compare the health-related quality of life (QOL) for patients between the two arms through 36 months after enrollment. We also describe the predictors and trajectories of QOL. Methods English and Spanish-speaking subjects were invited to complete patient-reported outcome (PRO) measures, including the Functional Assessment of Cancer Therapy - General (FACT-G), the SF-36 yielding a Physical Component Score (PCS) and Mental Component Score (MCS), and the EQ-5D, at enrollment and every 6 months until 24 months, then 36 months after enrollment. Validation studies indicate a clinically meaningful difference of 5 points for FACT-G, PCS, and MCS and 2 points for associated subscales. To account for the missingness of assessments, including those missing due to death, we compared each score between arms using an inverse probability weighted - independent estimating equation (IPW-IEE) model, which models the scores in surviving patients while using IPW to account for baseline variables and follow-up outcomes that impact the likelihood of missingness. Since 4 scores were evaluated, a Bonferroni corrected significance level of 1.25% = 5% / 4 was used. Cox regression models were used to evaluate the impact of QOL measures on overall and leukemia free survival. The IPW-IEE models adjust for baseline score and follow-up assessment time as well as age, race/ethnicity, performance status, IPSS score, duration of disease, and response to prior hypomethylating therapy; the Cox models adjusted for the 6 latter variables and treatment arm. Trajectories of QOL are shown by plotting mean +/- standard error by group over time. Results Between January 2014 and November 2018, 384 subjects (median age 66.7 years, range: 50.1-75.3) were enrolled at 34 centers and biologically assigned to Donor (n=261) or No Donor arm (n=123) by 90 days from enrollment. For the Donor arm the median duration from registration to HCT was 3.9 months (range 0.3-20.7). Completion rates were generally high at 65-78% of eligible survivors at each timepoint. At enrollment, 204 subjects (78.2%) in the Donor arm and 85 subjects (69.1%) in the No Donor arm completed at least one QOL form, with 4 patients unable to complete due to language (non-English or Spanish speaking). While there were some small differences at 18 months favoring the Donor arm, no clinically significant differences in PRO scores or subscales were seen between the arms at any timepoint (Figure 1) or in the scores over time. In general, similar trajectories for the Donor arm were seen for each PRO, with most decreasing or stable from baseline to 6 months and improving thereafter. Compared to published averages of U.S cancer populations, FACT-G means in both arms were higher beginning at 18 months. Baseline and 6-month PRO scores were the strongest predictors of later PRO scores despite adjusting for patient demographic and clinical factors. Overall survival was predicted by baseline FACT-G &lt;70 (HR=1.61, p&lt;.01) and PCS scores &lt;40 (HR=1.82, p&lt;0.001), while leukemia-free survival was predicted by baseline FACT-G &lt;70 (HR=1.61, p&lt;0.01) and PCS &lt;40 (HR=1.80, p&lt;0.002). No associations of PROs with AML transformation, relapse, or acute or chronic GVHD were found. Non-compliance with biologic assignment was less likely in Donor arm compared to No Donor, but baseline QOL was not a confounding factor. Conclusion In older adults with MDS, the survival advantage associated with Donor availability and HCT does not come at the cost of worse QOL in comparison to the No Donor arm. Baseline PRO scores were the strongest independent predictors of subsequent QOL outcomes and survival, even after controlling for clinical and patient-level factors. These results should reassure older patients and clinicians who prefer curative approaches to MDS. Figure 1 Figure 1. Disclosures Cutler: Mallinckrodt: Consultancy; Editas: Consultancy; CareDx: Consultancy; Kadmon: Consultancy; Pfizer: Consultancy; Incyte: Consultancy; Cimeio: Consultancy; Deciphera: Consultancy; Omeros: Consultancy; Syndax: Consultancy; Mesoblast: Consultancy; Jazz: Consultancy. Saber: Govt. COI: Other. Lee: Takeda: Research Funding; Syndax: Research Funding; Pfizer: Research Funding; Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding; Kadmon: Research Funding; National Marrow Donor Program: Membership on an entity's Board of Directors or advisory committees; Janssen: Other; Incyte: Research Funding; AstraZeneca: Research Funding; Amgen: Research Funding. Shaw: mallinkrodt: Other: payments; Orca bio: Consultancy. Horowitz: Magenta: Consultancy, Research Funding; Astellas: Research Funding; Jazz Pharmaceuticals: Research Funding; GlaxoSmithKline: Research Funding; Genentech: Research Funding; CSL Behring: Research Funding; Gamida Cell: Research Funding; Allovir: Consultancy; Daiicho Sankyo: Research Funding; Amgen: Research Funding; bluebird bio: Research Funding; Chimerix: Research Funding; Actinium: Research Funding; Medac: Research Funding; Kiadis: Research Funding; Xenikos: Research Funding; Vor Biopharma: Research Funding; Bristol-Myers Squibb: Research Funding; Regeneron: Research Funding; Pharmacyclics: Research Funding; Pfizer, Inc: Research Funding; Orca Biosystems: Research Funding; Omeros: Research Funding; Novartis: Research Funding; Miltenyi Biotech: Research Funding; Mesoblast: Research Funding; Kite/Gilead: Research Funding; Janssen: Research Funding; Sanofi: Research Funding; Seattle Genetics: Research Funding; Shire: Research Funding; Sobi: Research Funding; Stemcyte: Research Funding; Takeda: Research Funding; Tscan: Research Funding; Vertex: Research Funding.

scholarly journals Clinical Study to Investigate the Efficacy and Safety of Wilate during Prophylaxis in Previously Treated Patients with Von Willebrand Disease (VWD)

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4931-4931
Author(s):  
Robert F. Sidonio ◽  
Bruce A. Schwartz
Keyword(s):  
Board Of Directors ◽  
Research Funding ◽  
Prophylactic Treatment ◽  
Von Willebrand Disease ◽  
Advisory Committees ◽  
Previously Treated Patients ◽  
Previously Treated ◽  
Type 3 ◽  
Von Willebrand

Background: Inherited von Willebrand disease (VWD) is the most common inherited hemorrhagic disorder, with an estimated prevalence of 1 in every 100 individuals. Type 1 and type 3 (the most severe form) are characterized by a quantitative deficiency of von Willebrand factor (VWF) and type 2 arises from a qualitative deficiency of VWF. Treatment of VWD depends on the type and severity of the disease. Severe bleeding is reported in patients with all subtypes, leading to progressive joint disease as well as diminished quality of life (QoL). VWF/factor VIII (FVIII) concentrates have become the mainstay of VWD treatment for these patients with severe disease or for those patients in whom other treatments (e.g., desmopressin) are ineffective or contraindicated but this is broadly applicable only for on demand treatment. Aims: The primary objective of this study is to determine the efficacy of VWF/FVIIII concentrate in the prophylactic treatment of previously treated patients with type 3, type 2 (except 2N), or severe type 1 VWD. Secondary objectives of this study will be to collect data to 1) Assess the VWF:Ac and VWF:Ag incremental IVR of VWF/FVIIII concentrate over time and, 2) Assess the safety and tolerability of VWF/FVIIII concentrate in this indication. The study will also examine, the efficacy of VWF/FVIIII concentratein the treatment of breakthrough bleeding episodes (BEs), and in surgical prophylaxis, as well as the QoL during prophylaxis with VWF/FVIIII concentrate. Methods: The study is planned to enroll 28 patients aged ≥6 years and with VWD type 1, 2A, 2B, 2M, or 3. Eligible patients must be receiving on-demand treatment with a VWF-containing product, with at least 1, and an average of ≥2, documented spontaneous BEs per month in the preceding 6 months requiring treatment with a VWF-containing product. This will be assessed as part of a run-in observational study to collect the bleeding profile prior to the start of prophylaxis. From the beginning of the study, patients will receive prophylactic treatment with VWF/FVIIII concentrate for 12 months and record all BEs in a patient diary. Based on these data, the frequency of BEs and the annualized bleeding rate (ABR) under prophylactic treatment will be calculated. Treatment efficacy of BEs will be assessed by the patient (together with the investigator in case of on-site treatment) using a 4-point scale (excellent, good, moderate, none) In patients that undergo surgeries, efficacy of VWF/FVIIII concentratewill be assessed at the end of surgery by the surgeon and at the end of the postoperative period by the haematologist. In both cases, predefined assessment criteria will be used. In addition, an overall assessment of efficacy will be made at the end of the postoperative period by the investigator. Results: Data will be monitored on an ongoing basis and the study is expected to end Q2 2021. Conclusions: Prophylactic treatment in other congenital bleeding disorders is widely accepted as the standard of care to prevent bleeding and preserve QoL in patients but to date, this form of treatment in VWD is not well characterized. This study will provide data on the efficacy of prophylactic treatment in reducing the rate of bleeding and on the impact of prophylaxis on the QoL in VWD patients. Disclosures Sidonio: Genetech: Membership on an entity's Board of Directors or advisory committees, Research Funding; Takeda-Shire: Membership on an entity's Board of Directors or advisory committees, Research Funding; Biomarin: Membership on an entity's Board of Directors or advisory committees; Octapharma: Membership on an entity's Board of Directors or advisory committees, Research Funding; Bioverativ: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novo Nordisk: Membership on an entity's Board of Directors or advisory committees; Grifols: Membership on an entity's Board of Directors or advisory committees, Research Funding; Kedrion: Research Funding; Uniqure: Membership on an entity's Board of Directors or advisory committees. Schwartz:Octapharma: Employment.

scholarly journals Longitudinal Changes of Impairments in Health-Related Quality of Life in Lower-Risk MDS Patients: A European Leukemianet Study

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 3097-3097
Author(s):  
Reinhard Stauder ◽  
Ge Yu ◽  
Karin A Koinig ◽  
Dominic Culligan ◽  
Pierre Fenaux ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Research Funding ◽  
Self Care ◽  
Initial Diagnosis ◽  
Health Related Quality ◽  
Advisory Committees ◽  
Related Quality ◽  
Health Related ◽  
Anxiety Depression

Abstract Introduction Patient perception and perspective have become relevant in individualised therapy planning in MDS. Thus, integration of patient-reported outcomes (PRO), including health-related quality of life (HRQoL), in studies and in clinical practice is essential. We demonstrated recently pronounced impairments in HRQoL in patients with MDS (Stauder et al., Leukemia 2018). However, longitudinal data on aspects of HRQoL in MDS are rare. Objectives 1. To describe longitudinal changes of HRQoL in MDS and 2. To compare the time course in different patient subgroups to define patients at high risk of deterioration of HRQoL. Methods The EUMDS Registry is a prospective, non-interventional longitudinal study, enrolling newly diagnosed patients with IPSS low or intermediate-1 MDS from 143 haematology centres in 17 European countries and Israel. HRQOL was assessed by EQ-5D questionnaire. Patients were selected, if they fully completed the EQ-5D score (five dimensions and VAS) at baseline and at 6 and 12 months, resulting in a total of 743 subjects at 86 centres in 15 countries. Differences in response for the five EQ-5D dimensions between patients subgroups were evaluated using chi-squareMcNemar tests. For EQ-VAS, the mean score with standard deviation was calculated. Wilcoxon's signed ranks tests were conducted to identify changes over time between HRQOL values at baseline and at 6, 12 months. Results Moderate/severe impairments at initial diagnosis were observed in the dimensions mobility (39.4%), self-care (10.2%), usual activities (32.6%), pain discomfort (46.6%) and anxiety/depression (36.7%). As compared with baseline a pronounced increase in moderate/severe problems was observed at 12 months in the dimensions self-care (10.2% (baseline) vs 15.5% (12 mo); p=0.003) and in usual activities (32.6% vs 40.6%; p=0.001). In contrast, self-reported mobility, pain and anxiety/depression did not change significantly. Decrease in VAS over time revealed a trend toward significance (75 vs. 70; p=0.056). More pronounced impairment of HRQOL was most significantly observed in patients of advanced age: 60-75 yrs in self-care 8.3% vs 14.3% (p=0.016) and in usual activities 28% vs 35.7% (p=0.032); in persons 75+ years in self-care 14.2% vs 19.8% (p=0.058) and in usual activities 48.7% vs 50.3% (p=0.003). Similarly, VAS significantly decreased in the latter group from 70 to 66 (p=0.038). Increases in impairments were most prominent in male patients in self-care (9.7 vs 16.7%; p=0.002) and in usual activities (29.4 vs 38.7%; p=0.003), whereas in women HRQoL at the different time points was not significantly different. Pronounced decreases in HRQoL aspects was observed in anemic patients (Hb-levels <10g/dl) at initial diagnosis: mobility 47.4 vs 55.2% (p=0.06), self-care 13.5 vs 21.1% (p=0.015), usual activities 41.3 vs 52.7% (p=0.006), pain/discomfort 43.3 vs 55.6% (p=0.058) and VAS (70 vs 68; p=0.051). A reduction in HRQoL was observed even in patients with a Hb-level ≥10 g/dl in the dimension self-care (7.9 vs 12.1%; p=0.042). Pronounced decreases in problems in HRQoL were observed in anemic patients (Hb<10 g/dL) who received transfusions in self-care (18.1% vs 25.6%; p=0.029), in usual activities (43.6% vs 61.5%; p=0.038), and in VAS (67.3 vs 61.9; p=0.003). Transfusion dependent patients were at high risk to develop impairments in VAS (70 vs 64; p=0.006) and in most dimensions of EQ-5D (mobility 50.2 vs 62%, p=0.017), self-care (8.8 vs 11.5%, p=0.017), usual activities (41.6 vs 62.9%, p=0.01) and pain/discomfort (48.3 vs 58.5%, p=0.038). Conclusions Low-risk MDS patients report relevant restrictions in distinct dimensions at initial diagnosis. A relevant drop in HRQoL is observed at 12 months particularly in self-care and in usual activities. Patients of advanced age, males and those with initial low Hb-levels most frequently reported declines in HRQoL. Transfusion-need represents a relevant predictor of deterioration of HRQoL. These analyses form the basis to identify vulnerable patients and to tailor individualized interventions and treatment approaches. Analyses are planned to unravel the role of intervention therapies on observed changes in HRQoL. Disclosures Stauder: Teva: Research Funding; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees; Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees. Fenaux:Celgene: Honoraria, Research Funding; Jazz: Honoraria, Research Funding; Roche: Honoraria; Otsuka: Honoraria, Research Funding; Janssen: Honoraria, Research Funding. Garelius:novartis: Honoraria. Efficace:Incyte: Consultancy; Amgen: Consultancy; TEVA: Consultancy; Bristol Meyers Squibb: Consultancy; Orsenix: Consultancy; Seattle Genetics: Consultancy; Lundbeck: Research Funding; TEVA: Research Funding; AMGEN: Research Funding. de Witte:Amgen: Consultancy, Research Funding; Celgene: Honoraria, Research Funding; Novartis: Research Funding.

Health Related Quality of Life Results from the Open-Label, Randomized, Phase III Endeavor Trial Evaluating Carfilzomib and Dexamethasone Versus Bortezomib and Dexamethasone in Patients with Relapsed or Refractory Multiple Myeloma

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3309-3309 ◽  
Author(s):  
Heinz Ludwig ◽  
Philippe Moreau ◽  
Meletios A Dimopoulos ◽  
Maria-Victoria Mateos ◽  
Martin F Kaiser ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Multiple Myeloma ◽  
Board Of Directors ◽  
Research Funding ◽  
Sensitivity Analyses ◽  
Life Questionnaire ◽  
Advisory Committees ◽  
Related Quality ◽  
Health Related

Abstract Introduction: The randomized, phase 3 study ENDEAVOR (NCT01568866; N=929) demonstrated a clinically meaningful and statistically significant improvement in progression-free survival for patients with relapsed or refractory multiple myeloma who were treated with carfilzomib and dexamethasone (Kd) versus bortezomib and dexamethasone (Vd; median, 18.7 vs 9.4 months; hazard ratio: 0.53; 95% confidence interval [CI]: 0.44-0.65; P<0.0001) (Dimopoulos et al. Lancet Oncol. 2016;17:27−38). Patient-reported outcomes (PROs) were included as exploratory endpoints in the ENDEAVOR study. Here, we present results of a prespecified analysis of health-related quality of life (HR-QoL) in the ENDEAVOR trial. Methods: HR-QoL was assessed by 3 validated instruments: the European Organization of Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30-item questionnaire (QLQ-C30), the EORTC Quality of Life Questionnaire-multiple myeloma specific 20-item module (QLQ-MY20), and the Functional Assessment of Cancer Therapy/Gynecologic Oncology Group-Neurotoxicity (FACT/GOG-Ntx) "Additional Concerns" neurotoxicity subscale. These instruments were assessed prior to treatment administration on day 1 of cycle 1, and then every 28 days until disease progression, withdrawal of consent, or commencement of other nonstudy anticancer treatment. Due to differing treatment cycle lengths, the PRO assessments coincided across groups every 12 weeks. The primary PRO hypothesis was superiority of Kd over Vd for the Global Health Status/Quality of Life (GHS/QoL) scale of the QLQ-C30. Seven further subscales were prespecified from the QLQ-C30 (fatigue, nausea/vomiting, pain, physical functioning, role functioning) and the QLQ-MY20 (disease symptoms, side effects of treatment). PRO subscales were compared between groups using a restricted maximum likelihood-based mixed model for repeated measures (MMRM). Two sensitivity analyses were performed for the GHS/QoL scale to evaluate the robustness of the MMRM to missing data. Clinical interpretation for the EORTC QLQ-C30 subscales was guided by pre-specifying minimum important differences (MIDs) based on evidence-based guidelines (5 points for the GHS/QoL scale). For the QLQ-MY20 subscales, the standard error of measurement was used as a proxy for the MID. The proportion of patients who had improved (≥5 points) from baseline on the GHS scale was summarized at each coinciding time point. Results: Baseline completion of the QLQ-C30 questionnaire was similar between groups (Kd, 87.7%; Vd, 84.3%). Compliance was high when calculated for all patients expected to provide a questionnaire at each time point (ie, alive and on-study), ranging from 73.1% to 93.9%. Median duration on study treatment was 40 weeks and 27 weeks for Kd and Vd patients, respectively. Using the MMRM model, Kd was associated with statistically significantly higher GHS/QoL scores compared with Vd (p<0.0001). However, the overall treatment difference point estimate of 3.5 (95% CI, 2.0-5.1) did not reach the pre-defined MID. When including the treatment by time interaction (p=0.28) to estimate the treatment difference at timepoints where HR-QoL assessments coincided with day 1 of a cycle, the point estimates increased over time, with the differences at week 60 and 72 reaching clinical significance (5.4 and 5.8, respectively) (Figure). Results from the two sensitivity analyses confirmed findings from the MMRM analysis. Statistically significant benefits were observed in favor of the Kd group for fatigue (P=0.04), pain (P=0.02), side effects (P<0.0001) and NTx subscales (p=0.0002), although these differences did not meet MID thresholds. The proportion of patients reaching a ≥5 point improvement in the GHS scale was numerically higher in the Kd group up to week 48, although the difference between the groups did not reach statistical significance. Conclusions: This analysis of PROs in the ENDEAVOR study demonstrated that Kd was statistically superior to Vd on the QLQ-C30 GHS/QoL scale, with clinically meaningful differences observed at later timepoints but not on average overall. For patients remaining on longer term treatment, the clinical benefits of Kd compared with Vd were associated with better GHS/QOL. Although not meeting MID thresholds, statistically significant benefits were also observed in favor of the Kd group for other aspects of HR-QoL. Disclosures Ludwig: Janssen: Speakers Bureau; BMS: Speakers Bureau; Takeda: Research Funding, Speakers Bureau; Amgen: Research Funding, Speakers Bureau. Moreau:Amgen: Honoraria; Celgene: Honoraria; Takeda: Honoraria; Janssen: Honoraria; BMS: Honoraria; Novartis: Honoraria. Dimopoulos:Amgen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Genesis: Consultancy, Honoraria; Celgene: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees. Mateos:Janssen: Honoraria, Membership on an entity's Board of Directors or advisory committees; Celgene: Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees; Takeda: Honoraria, Membership on an entity's Board of Directors or advisory committees; BMS: Honoraria, Membership on an entity's Board of Directors or advisory committees; Glicomimetics: Honoraria, Membership on an entity's Board of Directors or advisory committees. Kaiser:BMS: Consultancy, Other: Travel Support; Takeda: Consultancy, Other: Travel Support; Amgen: Consultancy, Honoraria; Janssen: Consultancy, Honoraria; Celgene: Consultancy, Honoraria, Research Funding; Chugai: Consultancy. Feng:Amgen: Employment, Equity Ownership. Cocks:Amgen: Consultancy; Celgene: Consultancy; BMS: Consultancy; Endomag: Consultancy. Buchanan:Amgen: Employment, Equity Ownership. Weisel:BMS: Consultancy, Honoraria; Novartis: Honoraria; Takeda: Consultancy, Honoraria; Amgen: Consultancy, Honoraria; Onyx: Consultancy; Janssen: Consultancy, Honoraria, Research Funding; Celgene: Consultancy, Honoraria, Research Funding.

scholarly journals Symptoms, Health-Related Quality of Life, and Tolerability of Loncastuximab Tesirine in Patients with Relapsed or Refractory Diffuse Large B Cell Lymphoma

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 3-4
Author(s):  
Alexander I. Spira ◽  
Lei Chen ◽  
Xiaolei Zhou ◽  
Ari Gnanasakthy ◽  
Luqiang Wang ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Research Funding ◽  
B Cell Lymphoma ◽  
Patient Reported Outcome ◽  
Publicly Traded ◽  
Advisory Committees ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related ◽  
Night Sweats

Introduction In the ongoing single-arm, open-label phase 2 ADCT-402-201 study (LOTIS 2, NCT03589469), loncastuximab tesirine has shown substantial antitumor activity with a manageable toxicity profile in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who had failed ≥ 2 prior therapies, including activity in patients with high-risk disease characteristics. The overall response rate was 48.3% (based on positron emission tomography-computed tomography [PET-CT] assessed by independent review according to Lugano response criteria), and the median duration of response was 10.25 months. This analysis examined symptoms, health-related quality of life (HRQoL), and tolerability using validated patient-reported outcome instruments. Methods Enrolled patients aged ≥18 years received loncastuximab tesirine as an intravenous infusion on day 1 of each 3-week treatment cycle. Responders were defined as patients with a best overall response of complete or partial response. Patient-reported symptoms and HRQoL were measured using the Functional Assessment of Cancer Treatment-Lymphoma (FACT-Lym) and EQ-5D 5 Levels (EQ-5D-5L) instruments at baseline, day 1 of each cycle while patients were treated, and at the end-of-treatment visit. Descriptive statistics for change from baseline scores and percentages of patients with improved, stable, or worsened symptoms were summarized by visit and clinical response. Analysis was conducted using data collected from study initiation (1 August 2018) through 6 April 2020. Results The 145 patients enrolled in this study had a median age of 66 years (range, 23-94). Patients were heavily pretreated, with a median of 3 (range, 2-7) prior systemic therapies. A baseline patient-reported outcome score and at least one post-baseline score were available for 130 patients. Completion rates among patients treated at each visit were ≥ 92% for EQ-5D-5L and ≥ 88% for FACT-Lym up to cycle 9, day 1 (24 weeks). After cycle 9, fewer than 20 patients had patient-reported outcome scores available for analysis. For symptoms assessed in the FACT-Lym lymphoma subscale, pain in certain parts of the body, lumps/swelling, trouble sleeping at night, and fatigue were the most frequently reported symptoms at baseline (34%-59% reported "somewhat" to "very much"). Most patients (≥ 80%) reported "not at all" or "a little bit" at baseline for fever, night sweats, losing weight, itching, and loss of appetite. During the course of treatment, higher percentages of patients reported improvement than worsening for pain and lumps/swelling for the majority of visits. Fever, night sweats, and losing weight did not change for most patients. Itching was the only symptom for which more patients experienced worsening than improvement. For other symptoms, similar percentages of patients reported improvement and worsening. The mean change from baseline in EQ-5D Visual Analog Scale (VAS) score showed a trend of improvement in overall health over time (see figure). The mean VAS change reached the minimally important difference of 7 points at cycle 8, day 1. This improvement was associated with clinical response. When patients were asked how much they were bothered by side effects of treatment, most patients (&gt; 60%) reported "not at all" or "a little bit" for all visits throughout the treatment. Conclusions Results of this analysis suggest that patients who responded to treatment with loncastuximab tesirine generally had stable or improved symptoms and overall HRQoL. The treatment was well tolerated by patients. These findings further support the clinical use of loncastuximab tesirine for the treatment of relapsed or refractory DLBCL. Disclosures Spira: ADCT:Research Funding;Janssen:Consultancy;Incyte:Consultancy;BMS:Consultancy;Merck:Consultancy;Novartis:Consultancy;Takeda:Consultancy;Cardiff Oncology:Research Funding.Chen:ADCT:Current Employment, Current equity holder in publicly-traded company.Zhou:ADCT:Research Funding.Gnanasakthy:ADCT:Research Funding.Wang:ADC Therapeutics America, inc:Current Employment, Current equity holder in publicly-traded company.Ungar:ADC Therapeutics:Current Employment, Current equity holder in publicly-traded company.Curiel:ADCT:Current Employment, Current equity holder in publicly-traded company.Radford:GlaxoSmithKline:Current equity holder in publicly-traded company, Other: Spouse;AstraZeneca:Current equity holder in publicly-traded company, Other: Spouse;Takeda:Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau;Pfizer:Research Funding;ADCT:Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding;BMS:Consultancy, Honoraria, Speakers Bureau;Novartis:Consultancy, Honoraria;Seattle Genetics:Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau.Kahl:AstraZeneca Pharmaceuticals LP:Consultancy, Membership on an entity's Board of Directors or advisory committees;Celgene Corporation:Consultancy;Genentech:Consultancy;Pharmacyclics LLC:Consultancy;Roche Laboratories Inc:Consultancy;BeiGene:Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding;Janssen:Consultancy, Membership on an entity's Board of Directors or advisory committees;Acerta:Consultancy, Research Funding;ADC Therapeutics:Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding;AbbVie:Consultancy.

scholarly journals Depression and Anxiety in Persons with Von Willebrand Disease

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4052-4052
Author(s):  
Jonathan C. Roberts ◽  
Roshni Kulkarni ◽  
Peter A. Kouides ◽  
Robert F. Sidonio ◽  
Shannon L Carpenter ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Research Funding ◽  
Clinical Characteristics ◽  
Important Variable ◽  
Von Willebrand Disease ◽  
Depression And Anxiety ◽  
Advisory Committees ◽  
Type 3 ◽  
Von Willebrand

Abstract Background: Depression and anxiety are associated with poor health-related quality of life (HRQoL), lower functioning and decreased treatment adherence. In 2019, 7% adults in the US had moderate/severe symptoms of depression, while &lt;5% had anxiety. Impacts of depression and anxiety in persons with von Willebrand disease (VWD) are unclear and less studied. Objective: We assessed sociodemographic and clinical characteristics associated with depression and anxiety in a geographically diverse cohort of individuals with VWD obtaining care at seven US Hemophilia Treatment Centers (HTCs). Methods: The study enrolled and collected data on individuals age ≥12 with VWD Type 1 (VWF:Ag/RCo: ≤30%), low VWF(VWF:Ag/RCo: 30-50%), Type 2, and type 3 between September 2018-June 2021. Participants completed a survey at enrollment to collect sociodemographic and clinical characteristics, self-reported pain, joint problems and HRQoL measured by the EQ-5D-3L. A quarterly survey administered one year post-enrollment collected similar data. The patient health questionnaire (PHQ-8) and the generalized anxiety disorder (GAD-7) were administered with the last follow-up survey after August 2019. Chart reviews abstracted VWD type information. The association of sociodemographic and clinical characteristics with depression or anxiety was assessed using Chi-square tests for categorical variables, as well as logistic regression models with stepwise selection. Results: We analyzed data from 77 participants who completed both baseline and last follow-up surveys. Mean age was 34.2 (standard deviation (SD)=18.8) years, 74.0% were adults ≥18 years, 79.2% were female, 60.8% had Type 1/low VWF, and 3.9% had Type 3 VWD. Mean age at VWD diagnosis was 13.9 (SD=13.2) years. Overall reported depression rate was 63.4%, and 58.3% for anxiety (values ≥10 on either PHQ-8 or GAD-7). Proportion of those with depression (75% vs. 62%) or anxiety (58% vs. 58%) prior to and during the COVID-19 pandemic were not significantly different. Persons with low VWF had higher rates of depression (86.7%) or anxiety (69.2%) as compared to those with type 1 VWD (55.3% for depression, 52.8% for anxiety) or types 2 and 3 (62.5%, 60.9%, p=0.10, not significant (NS) for depression and p=0.56, NS for anxiety, respectively). Females reported a higher rate of anxiety (61.4%) than males (46.7%, p=0.30, NS). When compared to individuals who rated their general health as the same or better than 3-months ago, those who rated their health as worse had significantly higher rates of depression (92.3% vs. 57.8%, p=0.02) and anxiety (83.3% vs. 53.3%, p=0.05). Participants with chronic pain reported a significantly higher depression rate (81.6% vs. 36.8%, p=0.0003). Those who reported having joint problems also reported depression at a significantly higher rate (82.4% vs. 48.8%, p=0.002) or anxiety (74.1% vs. 46.3%, p=0.02) than those without joint problems. Logistic regression analyses demonstrated that among adults or parents of pediatric patients, being single or not with a partner was the most important variable associated with depression (odds ratio (OR)=7.0, confidence interval (CI): 1.7-29.0), followed by having joint problems (OR=6.3, CI=2.0-20.1). The most important variable associated with anxiety was being a youth aged 12-18 years old (OR=6.7, CI=1.6-26.9), followed by being single or not with a partner (OR=10.8, CI=2.5-47.5), or having worse health compared to 3-months prior (OR=12.3, CI=1.3-116.2). Mean covariates adjusted EQ index scores were lower among persons with depression (0.75±standard error (SE) 0.03 vs. 0.83±0.04, p=0.06 NS) or anxiety (0.75±0.03 vs. 0.82±0.04, p=0.7 NS) than among those without depression or anxiety. As compared to individuals without depression or anxiety, mean covariates adjusted EQ VAS was significantly lower in persons with depression (68.7±3.1 vs. 77.6±4.2, p=0.03), but not among those with anxiety (69.3±3.7 vs. 71.3±4.3, p=0.66 NS). Conclusions: Our study revealed higher rates of major depression and anxiety in this VWD sample than the general US population. Depression had a significant negative impact on HRQoL. Mental health screening is imperative for persons with VWD, especially those with low VWF, chronic pain or joint problems. Special attention should be paid to women and youth. This study underscores the need for a multidisciplinary approach in the comprehensive care of patients seen at HTCs. Disclosures Roberts: Genentech, Novo Nordisk, Octapharma, Pfizer, Sanofi, Takeda, uniQure: Consultancy; Takeda; Speakers Bureau: Novo Nordisk, Octapharma, Sanofi, Takeda.: Research Funding. Kulkarni: Genentech: Honoraria, Membership on an entity's Board of Directors or advisory committees; CSL Behring: Honoraria, Membership on an entity's Board of Directors or advisory committees; Shire/Takeda: Honoraria, Membership on an entity's Board of Directors or advisory committees; Novo Nordisk: Honoraria, Membership on an entity's Board of Directors or advisory committees; Bayer: Honoraria, Membership on an entity's Board of Directors or advisory committees; Sanofi Genzyme: Honoraria, Membership on an entity's Board of Directors or advisory committees; Pfizer: Honoraria, Membership on an entity's Board of Directors or advisory committees; Octapharma: Honoraria, Membership on an entity's Board of Directors or advisory committees. Sidonio: Bayer: Consultancy; Catalyst: Consultancy; Genentech: Consultancy, Research Funding; Novo Nordisk: Consultancy; Guardian Therapeutics: Consultancy; Octapharma: Consultancy, Research Funding; Biomarin: Consultancy; Pfizer: Consultancy; Takeda: Consultancy, Research Funding. Carpenter: Genentech: Honoraria; Novo Nordisk: Honoraria; Kedrion Pharmaceuticals: Honoraria; Hemophilia and Thrombosis Research Society: Membership on an entity's Board of Directors or advisory committees. Konkle: Pfizer, Sangamo, Sanofi, Sigilon, Spark, Takeda and Uniqure: Research Funding; BioMarin, Pfizer and Sigilon: Consultancy. Wu: Baxalta US Inc., Bannockburn, IL (a Takeda Company), CSL Behring L.L.C., Octapharma USA, Inc., Genentech Inc.: Research Funding. Curtis: Pfizer, Bayer, and Novo Nordisk: Consultancy; University of Southern California: Consultancy. Nichol: Pfizer, Genentech Inc., Baxalta US Inc., Bannockburn, IL (a Takeda Company), Octapharma, CSL Behring, Global Blood Therapeutics, and Novo Nordisk: Research Funding.

scholarly journals Clustering of Bleeding Symptoms in Patients Previously Diagnosed As Type 3 Von Willebrand Disease: Results from a Large Cohort of Type 3 Von Willebrand Disease (the 3Winters-Ips Project)

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 2465-2465
Author(s):  
Alberto Tosetto ◽  
Zahra Badiee ◽  
Mohammad-Reza Baghaipour ◽  
Luciano Baronciani ◽  
Javier Battle ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Research Funding ◽  
Advisory Board ◽  
Von Willebrand Disease ◽  
Severe Bleeding ◽  
Advisory Committees ◽  
Bleeding Score ◽  
Type 3 ◽  
Von Willebrand

Abstract Patients with type 3 von Willebrand Disease (VWD) usually have markedly reduced FVIII/VWF levels and very severe bleeding manifestations but, because of their rarity, their bleeding phenotype is poorly described. We aimed at evaluating the distribution of bleeding symptoms in patients with type 3 VWD, comparing them with previously available data from a cohort of type 1 patients, and describing site-specific clustering of bleeding symptoms in these patients. We analyzed clinical data from the type 3 Von Willebrand International RegistrieS Inhibitor Prospective Study (3WINTERS-IPS),a no-profit, investigators initiated, multicenter, European-Iranian observational, retrospective and prospective study on patients with diagnosis of type 3 VWD. Aims of the 3WINTERS-IPS is 3-fold: a) to identify the main phenotypic and molecular characteristics of a large cohort of VWD patients; b) to evaluate the risk factors responsible for the severe bleeding phenotype; c) to assess the efficacy and safety of the treatment with VWF concentrates with or without FVIII including the risk of anti-VWF antibodies. Retrospective information on bleeding symptoms at presentation was collected using the MCMDM-1 VWD bleeding questionnaire, and bleeding severity summarized as bleeding score. Individual bleeding symptoms were considered as relevant when having a score >1 (hence requiring medical attention). Data was compared with that retrieved from the MCMDM-1 VWD study database on patients affected by type 1 VWD (index cases and affected family members). The study enrolled a total of 260 patients, of which we analysed 243 patients with available bleeding score at recruitment. The median age at study inclusion was 29 years (interquantile range, 26.5 years); 140 were females (53.8%). There were 108 patients of Iranian descent, while the remaining of patients were from Europe. The median number of bleeding symptoms was 5, and the median bleeding score was 15 (interquantile range, 13). Only 7/243 patients (2.8%) had a single bleeding symptom. Epistaxis was the most frequent relevant symptom, being present in 195 patients (80.2%), followed by menorrhagia in 99 females (70.7%). Males had a higher frequency of hemarthroses and hematomas than females (53.4% vs 42.1% and 40.8% vs 27.1%, respectively). When comparing the clinical presentation of type 3 vs. type 1 VWD, clearly increased bleeding scores were evident for all age-classes and even in paediatric cases. The association between symptoms having a relative frequency >20% is presented in the circle diagram, showing that some symptoms appeared to cluster with others in a variable degree (e.g., menorrhagia with epistaxis, hemarthrosis or oral cavity bleeding; post-extraction bleeding again with epistaxis, hemarthrosis or oral cavity bleeding; surgical bleeding or gastrointestinal bleeding with epistaxis alone). These findings confirm the severity of type 3 VWD and extend the knowledge of symptoms distribution in the widest available cohort of type 3 VWD patients. Disclosures Tosetto: Stago, Novo-Nordisk, BMS: Speakers Bureau; Werfen: Other: Member of Advisory Board, Speakers Bureau. Berntorp:Octapharma: Consultancy; CSL Behring: Consultancy; Shire: Consultancy, Other: honoraria for lecturing . Eikenboom:CSL: Research Funding. Mazzucconi:Baxalta-Shire: Consultancy, Speakers Bureau; Bayer: Consultancy, Speakers Bureau; Novartis,: Consultancy, Speakers Bureau; Amgen: Consultancy, Speakers Bureau; Novo Nordisk: Consultancy, Speakers Bureau; CSL Behring: Consultancy, Speakers Bureau. Oldenburg:Pfizer: Honoraria, Membership on an entity's Board of Directors or advisory committees; Biogen Idec: Honoraria, Membership on an entity's Board of Directors or advisory committees; Chugai: Honoraria, Membership on an entity's Board of Directors or advisory committees; Grifols: Honoraria, Membership on an entity's Board of Directors or advisory committees; Roche: Honoraria, Membership on an entity's Board of Directors or advisory committees; Shire: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Octapharma: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Novo Nordisk: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; CSL Behring: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Biotest: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Bayer: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Swedish Orphan Biovitrum: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Peyvandi:Kedrion: Consultancy; Ablynx: Other: Member of Advisory Board, Speakers Bureau; Shire: Speakers Bureau; Roche: Speakers Bureau; Shire: Speakers Bureau; Kedrion: Consultancy; Kedrion: Consultancy; Ablynx: Other: Member of Advisory Board, Speakers Bureau; Grifols: Speakers Bureau; Roche: Speakers Bureau; Octapharma US: Honoraria; Octapharma US: Honoraria; Sobi: Speakers Bureau; Ablynx: Other: Member of Advisory Board, Speakers Bureau; Kedrion: Consultancy; Shire: Speakers Bureau; Roche: Speakers Bureau; Roche: Speakers Bureau; Novo Nordisk: Speakers Bureau; Shire: Speakers Bureau; Ablynx: Other: Member of Advisory Board, Speakers Bureau; Grifols: Speakers Bureau; Shire: Speakers Bureau; Ablynx: Other: Member of Advisory Board, Speakers Bureau; Grifols: Speakers Bureau; Novo Nordisk: Speakers Bureau; Octapharma US: Honoraria; Octapharma US: Honoraria; Sobi: Speakers Bureau; Grifols: Speakers Bureau; Grifols: Speakers Bureau; Kedrion: Consultancy; Sobi: Speakers Bureau; Roche: Speakers Bureau; Novo Nordisk: Speakers Bureau; Novo Nordisk: Speakers Bureau; Novo Nordisk: Speakers Bureau; Sobi: Speakers Bureau; Octapharma US: Honoraria; Sobi: Speakers Bureau. Schneppenheim:SHIRE: Consultancy; CSL Behring: Consultancy. Tiede:Alnylam, Bayer, Biogen Idec, Biotest, Bristol-Myers-Squibb, Boehringer Ingelheim, CSL Behring, Leo Pharma, Novo Nordisk, Octapharma, Pfizer, Roche, Shire, and SOBI: Consultancy; Alnylam, Bayer, Biogen Idec, Biotest, Bristol-Myers-Squibb, Boehringer Ingelheim, CSL Behring, Leo Pharma, Novo Nordisk, Octapharma, Pfizer, Roche, Shire, and SOBI: Honoraria; Alnylam, Bayer, Biogen Idec, Biotest, Bristol-Myers-Squibb, Boehringer Ingelheim, CSL Behring, Leo Pharma, Novo Nordisk, Octapharma, Pfizer, Roche, Shire, and SOBI: Research Funding.

scholarly journals Health-Related Quality-of-Life Outcomes in Patients with Classical Hodgkin Lymphoma: A Prospective, Observational Study in US Oncology Practices

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2192-2192
Author(s):  
Jakub Svoboda ◽  
Philippe Armand ◽  
Fiona Taylor ◽  
Xiaowu Sun ◽  
Alejandro Moreno-Koehler ◽  
...  
Keyword(s):  
Observational Study ◽  
Board Of Directors ◽  
Research Funding ◽  
Advisory Committees ◽  
Meaningful Improvement ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related ◽  
Treatment Naïve ◽  
Multi Agent

Introduction: Classical Hodgkin lymphoma (cHL) is highly curable with front-line multi-agent chemotherapy with or without radiotherapy. The treatment landscape is rapidly evolving, with a continued focus on optimizing available therapies in order to improve outcomes and minimize toxicity. Randomized controlled trials provide valuable insights into new treatments and outcomes; however, real-world data describing treatment effectiveness, safety, and health-related quality of life (HRQoL) are limited. This analysis was conducted to understand patient-reported outcomes (PROs) in patients who were treated for newly diagnosed cHL across a range of academic and community settings. Methods: Study CA209655 is an ongoing, prospective, observational study (NCT02856646) at ~80 US oncology practices with a target enrollment of 500 patients and a planned follow-up of ≤ 5 years. Eligible patients are ≥ 18 years old with histologically confirmed cHL and are treatment naive or within ± 2 weeks of beginning any line of therapy at time of enrollment. HRQoL is a secondary endpoint and evaluated using the Functional Assessment of Cancer Therapy-Lymphoma (FACT-Lym) questionnaire. The FACT-Lym combines a generic cancer-related core consisting of the FACT-General (FACT-G; 27 items) that assesses physical (PWB; 7 items), social/family (SWB; 7 items), emotional (EWB; 6 items), and functional well-being (FWB; 7 items), and a 15-item disease-specific lymphoma subscale (LymS) that assesses lymphoma-specific symptoms. PROs were assessed using questionnaires at baseline (BL), every 3-6 months for ≤ 2 years, and annually thereafter. All patients with BL and ≥ 1 post-BL assessment were included in the PRO analysis. Completion rates and changes from BL scores were evaluated descriptively and analyzed based on published estimates for clinically important differences and individual responder definitions (Hlubocky et al. Lymphoma 2013; Carter et al. Blood 2008; Yost and Eton. Eval Health Prof 2005). High scores indicate better health/functioning for all scales/subscales. Results: Of 278 enrolled patients at data cutoff (Feb 2019), 226 (81%) were treatment naive; for those, the most common index therapy was chemotherapy (n = 210; 93%). The most common index chemotherapies were ABVD (doxorubicin, bleomycin, vinblastine, dacarbazine; n = 187, 83%) and AAVD (adriamycin, brentuximab, vinblastine, dacarbazine; n = 10, 5%). Only patients receiving index chemotherapy were included in the full analysis set. In all, 89% (87/210) of patients had BL and ≥ 1 post-BL assessment and were included in the PRO analysis. PRO completion rate at BL was 97% (204/210) and remained > 70% up to 24 months for the expected population. Analysis of PROs was not feasible after 24 months owing to small patient numbers (≤ 10); therefore, PRO analyses were focused on the first 18 months. At BL, mean (SD) scores were: FACT-Lym total, 122 (24); FACT-G, 82 (15); PWB, 21 (6); SWB, 25 (3); EWB, 18 (4); FWB, 18 (6); and LymS, 40 (11). At 3 months there was a deterioration in mean scores from baseline in FACT-G (−3.8) and PWB (−3.0) (Figure A). Clinically meaningful improvement of mean scores was observed starting at 6 months in LymS (+4.4) and at 9 months in FACT-G (+7.1), PWB (+3.1), FWB (+2.8), and FACT-Lym total (+15.6). There were no clinically meaningful improvements in SWB and EWB. Based on individual responder definitions, ≥ 50% of patients had improvements in PWB (Figure B) and FACT-G from 12 months, and FACT-Lym total from 9 months. Conclusions: Initial data from this observational study suggest most patients receive multi-agent cytotoxic chemotherapy as first-line treatment of cHL. Patient-reported PWB initially declined with chemotherapy, but improved with time, and patients had a clinically meaningful improvement in HRQoL by 9 months. The timing of this improvement may be due to discontinuation of chemotherapy due to disease control. Future analyses will evaluate the relationship between improvements in HRQoL and other factors including response, use of immunotherapies versus chemotherapy, and the timing of treatment discontinuation. Study support: Bristol-Myers Squibb. Disclosures Svoboda: Seattle Genetics: Consultancy, Research Funding; BMS: Consultancy, Research Funding; Merck: Research Funding; Incyte: Research Funding; Pharmacyclics: Consultancy, Research Funding; AstraZeneca: Consultancy; Celgene: Research Funding; Kyowa: Consultancy; Kite: Consultancy. Armand:Roche: Research Funding; Merck: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Bristol-Myers Squibb: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Adaptive: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Affimed: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Otsuka: Research Funding; Sigma Tau: Research Funding; Infinity: Consultancy; Genentech: Research Funding; Pfizer: Consultancy; ADC Therapeutics: Consultancy; Tensha: Research Funding. Taylor:Adelphi Values: Employment, Other: I am an employee of Adelphi Values, a consulting firm who has received payment from Bristol-Myers Squibb for statistical data analysis in Bristol-Myers Squibb's trials. Sun:Adelphi Values: Employment. Gajavelli:Bristol-Myers Squibb: Employment. Peterson:Bristol-Myers Squibb: Employment, Equity Ownership. Chen:Bristol-Myers Squibb: Employment.

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