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 Validation of the Qualms Questionnaire to Assess Health-Related Quality of Life in European and Israeli Patients with Myelodysplastic Syndromes: Results from the MDS-Right Project

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 1982-1982
Author(s):  
Fabio Efficace ◽  
Karin A Koinig ◽  
Francesco Cottone ◽  
David Bowen ◽  
Moshe Mittelman ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Board Of Directors ◽  
Research Funding ◽  
Discriminant Validity ◽  
Project Manager ◽  
Advisory Committees ◽  
Horizon 2020 ◽  
Related Quality ◽  
Health Related

Abstract Background The assessment of health-related quality of life (HRQoL) in patients with myelodysplastic syndromes (MDS) is critical to organize more robustly patient-centered care. Therefore, availability of well-validated disease-specific HRQoL measures in this area is important. Objectives We investigate the validity of the Quality of Life in Myelodysplasia Scale (QUALMS), to assess HRQoL in MDS patients reported to the European MDS (EUMDS) Registry. A prior validation effort has been reported (Abel G, et al. Haematologica, 2016 June; 781-88), but this was largely a North American cohort and subscales suggested by the original principal components analysis had not been validated. Patients and Methods The QUALMS questionnaire was included in the EUMDS Registry (de Swart L, et al. Br J Haematol. 2015 Aug;170:372-83), a large international initiative including several European countries and Israel. We used proportions, median and interquartile range (IQR) to summarize patients' characteristics at baseline. We also assessed internal consistency and test-retest reliability by Cronbach's alpha (threshold for acceptability Cronbach's alpha ≥0.70). Confirmatory factor analysis (CFA) was used to examine the model fit for the underlying scale structure. The model performance was evaluated by Comparative Fit Index (CFI), the Tucker-Lewis Index (TLI), and the Root Mean Squared Error of Approximation (RMSEA), where acceptable values were &gt;0.90 for CFI and TLI and RMSEA&lt;0.08. We assessed concurrent validity calculating the Spearman's rank correlation between scales from QUALMS and EQ-5D-3L questionnaire. Discriminant validity was assessed using the Wilcoxon-Mann-Whitney test to compare the scores between patient subgroups, defined as low vs. intermediate/high MDS comorbidity index (MDS-CI), lower vs higher (&lt; 90 vs. ≥ 90) Karnofsky performance status (KPS), anemic vs. non-anemic patients as defined by gender-specific thresholds from the World Health Organization, males vs. females and having received or not red blood cell (RBC) transfusions previously. Responsiveness to change of QUALMS scales was evaluated by Wilcoxon signed-rank test to assess changes in the scores from baseline in patients who reported an improvement in Hb-levels (≥1.5 g/dL) and in those who reported a clinically meaningful deterioration in the Visual Analogue Scale (VAS) of the EQ-5D-3L. Results Overall, 270 MDS patients completed the QUALMS questionnaire, from 17 centers across four countries: Austria (N = 61), Israel (N = 67), The Netherlands (N = 37) and United Kingdom (N = 105). The majority of patients were male (67.4%) with a median age at diagnosis of 74 years (IQR =68.0-80.0) and a low MDS-CI score (n=158). CFA indicated acceptable model fit, respectively CFI=0.93, TLI=0.93 and RMSEA=0.07. For all analyses, the scales QUALMS-physical burden (P), QUALMS-emotional burden (E) and the total score showed satisfactory results, while the scale QUALMS-benefit finding (BF) showed worse results. Internal consistency and test-retest reliability were acceptable for the total score (TS) and all scales but BF (see figure). Pairwise correlations between QUALMS-P, QUALMS-E, TS and all the scales from EQ-5D-3L (including VAS) showed satisfactory concurrent validity (range from -0.67 to 0.60). Only the QUALMS-BF did not correlate with any of the EQ-5D-3L scales (range from -0.07 with EQ-VAS to 0.14 with EQ-5D Anxiety/ Depression). As per discriminant validity, all known-group comparisons were statistically significant and consistent with the underlying clinical construct for all scales apart for the QUALMS-BF. For example, the QUALMS-P mean scores were 75.8 vs 54.6 (p&lt; 0.001) for patients with a higher vs lower KPS, while the corresponding QUALMS-BF mean scores were 49.4 vs 50.9 (p=0.829). The same profile resulted for responsiveness to change, where all scales reflected significant changes in Hb levels and in EQ-VAS score in the expected direction, while QUALMS-BF did not. For example, those patients who experienced a clinically meaningful deterioration in the VAS score also had also a worsening in QUALMS-P (Δ=-6, p=0.003), QUALMS-E (Δ=-1.8, p=0.075) and TS (Δ=-3, p=0.003). Conclusions Current findings provide novel evidence-based data that support the validity of the QUALMS for its use in European and Israeli patients with MDS. Implementation of the QUALMS in MDS research may further raise PRO standards in this area. Figure 1 Figure 1. Disclosures Efficace: Takeda: Consultancy; Janssen: Consultancy; Abbvie: Consultancy, Other: Grants (to Institution); Amgen: Consultancy, Other: Grants (to Institution). Mittelman: Janssen · Roche · Novartis · Takeda · Medison / Amgen · Neopharm / Celgene / BMS · Abbvie · Gilead: Research Funding; Novartis · Takeda · Fibrogen · Celgene / BMS · Onconova · Geron: Other: Clinical Trial Support; Onconova · Novartis · Takeda · Silence: Membership on an entity's Board of Directors or advisory committees; MDS HUB: Consultancy; Celgene / BMS · Novartis: Speakers Bureau. Van Marrewijk: Novartis Pharmacy B.V. Oncology Europe: Other: project manager of the EUMDS Registry, is funded from the EUMDS (educational grants from Novartis Pharmacy B.V. Oncology Europe); Amgen Limited: Other: project manager of the EUMDS Registry, is funded from the EUMDS (educational grants from Amgen Limited); Celgene International: Other: project manager of the EUMDS Registry, is funded from the EUMDS (educational grants from Celgene International); Janssen Pharmaceutical: Other: project manager of the EUMDS Registry, is funded from the EUMDS (educational grants from Janssen Pharmaceutical); Takeda Pharmaceuticals International: Other: project manager of the EUMDS Registry, is funded from the EUMDS (educational grants from Takeda Pharmaceuticals International); EU's Horizon 2020 program: Other: MDS-RIGHT (grant from EU's Horizon 2020 program) project budgets. de Witte: Takeda: Research Funding; Novartis: Research Funding; Janssen: Research Funding; Celgene: Research Funding; Amgen: Research Funding. Stauder: Celgene/BMS: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Travel support; Novartis: Honoraria, 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 The Relationship between Minimal Residual Disease and Patient Reported Outcomes in Relapsed/Refractory Multiple Myeloma

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 3273-3273
Author(s):  
Hervé Avet-Loiseau ◽  
Jianming He ◽  
Katharine S. Gries ◽  
Huiling Pei ◽  
Sourish Saha ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Board Of Directors ◽  
Health Related Quality ◽  
Advisory Committees ◽  
Related Quality ◽  
Eortc Qlq C30 ◽  
Health Related ◽  
Index Value ◽  
Eortc Qlq

Abstract Objective With the introduction of novel treatments for multiple myeloma, patients are now achieving deeper and sustainable clinical responses. Recent studies have demonstrated that achieving Minimal Residual Disease (MRD) negativity leads to better progression-free survival and overall survival outcomes (Lahuerta JJ, et al. J Clin Oncol 2017. 35[25]:2900-10; Munshi NC, et al. JAMA Oncol 2016. 3[1]:28-35; Landgren O, et al. Bone Marrow Transplant 2016. 51[12]:1565-1568). However, the relationship between MRD status and patient reported outcomes (PRO) has not been reported. The objective of this analysis is to evaluate whether PRO endpoints change by MRD status using data from two randomized clinical trials of daratumumab containing treatment regimens, POLLUX (Dimopoulos MA, et al. N Engl J Med 2016. 375[14]:1319-1331) and CASTOR (Palumbo A, et al. N Engl J Med 2016. 375[8]:754-766), for patients with relapsed or refractory multiple myeloma. Methods MRD status was assessed in POLLUX at the time of suspected CR, and at 3 and 6 months post-suspected CR for responders. Similarly, in CASTOR, MRD status was assessed for patients at the time of suspected CR and at 6 months and 12 months after first dose. MRD was assessed via next generation sequencing using the clonoSEQ® assay V2.0 (Adaptive Biotechnologies, Seattle, WA) at sensitivities of 0.001%. The PRO instruments (EORTC-QLQ-C30 and EQ-5D-5L) were collected in both POLLUX and CASTOR study prior to treatment, during the treatment phase, and post-progression. EQ-5D-5L assessed general health status and included an index value and visual analog scale (VAS) score. EORTC QLQ C30 assessed health related quality of life and included five functional scales (physical, role, emotional, social and cognitive), three symptom scales (fatigue, nausea & vomiting and pain) and a global health status (GHS) scale as well as six single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). Summary statistics (mean, standard deviation, median, min and max) by MRD status (baseline, prior to MRD negativity, MRD negativity prior to progression) were reported and for subjects who did not achieve MRD negativity (baseline, post baseline prior to progression) on a pooled sample of subjects from the two clinical trials. To interpret a meaningful change, a 5-point threshold was defined based on the EORTC guidelines for assessing quality of life in clinical trials. Results Overall 137 subjects in both CASTOR and POLLUX achieved MRD negativity and had PRO data available for analysis. At baseline, GHS, EQ-5D-5L VAS and index value were 62.1, 66.7, and 0.72 respectively (GHS and VAS scores closer to 100, and index value closer to 1.0 represent better health state). Mean values increased to 67.2, 70.9, and 0.75 after achieving MRD negativity. Pain scale (symptom scores closer to 0 represent less symptoms) reduced from 30.4 to 23.5 and fatigue was similar (33.8 at baseline to 31.2) when patients achieved MRD negativity. However, when we compared the five functional scales prior to and post MRD negativity, no evident differences were identified. The mean change from baseline to post-MRD-negativity in the EORTC QLQ-C30 GHS and Pain scores exceeded a 5-point threshold, reflecting a meaningful change in subject's health-related quality of life. A total of 893 subjects in the pooled data set did not achieve MRD negativity and had PRO data available for analysis (EQ-5D-5L data were not available for 3 subjects). Baseline values for these MRD positive subjects were 60.0, 65.3, and 0.71 and the mean post-baseline (pre-progression) values remained similar at 61.1, 66.0, and 0.71 for GHS, VAS, and the index value, respectively. Pain reduced from mean 33.3 to 29.4 and fatigue was similar, changing from 36.2 to 37.6. Conclusion To our knowledge, this is the first analysis exploring the relationship between MRD status and PRO endpoints. Results from this analysis demonstrate that patients who achieve MRD negativity status show a trend in better health-related quality of life, with meaningful improvement in EORTC QLQ-C30 GHS and pain scores. These preliminary findings indicate that overall health-related quality of life and symptom domains of EORTC-QLQ-C30 and EQ-5D-5L might be sensitive to changes in MRD status, with changes in GHS and Pain exceeding meaningful threshold for subjects. Disclosures Avet-Loiseau: Amgen: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Sanofi: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Takeda: Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: 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; Abbvie: Membership on an entity's Board of Directors or advisory committees. He:Janssen global services: Employment. Gries:Janssen Research & Development, LLC: Employment. Pei:Janssen Research & Development, LLC: Employment. Saha:Janssen Research & Development, LLC: Employment. Chiu:Janssen Research & Development, LLC: Employment. Cote:Janssen Research & Development, LLC: Employment. Lam:Janssen Global Services, LLC: 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.

Association of Race with Health-Related Quality of Life (HRQOL) Among Multiple Myeloma Patients

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 5070-5070 ◽  
Author(s):  
Chris L. Pashos ◽  
Brian GM Durie ◽  
Robert Rifkin ◽  
Howard Terebelo ◽  
Cristina Gasparetto ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Board Of Directors ◽  
Employment Equity ◽  
Advisory Committees ◽  
Patient Race ◽  
The Us ◽  
Related Quality ◽  
Health Related ◽  
Equity Ownership

Abstract Abstract 5070 Introduction. Studies in the United States (US) have identified variation in incidence and survival of multiple myeloma (MM) patients of different races, and noted that MM is the most common hematologic cancer among African Americans. This analysis was conducted to evaluate whether the health-related quality of life (HRQOL) of patients in the US as they initiate treatment with active, symptomatic MM varies by race. Methods. Data were collected in Connect MM®, a prospective observational registry begun in September 2009 involving centers in the US. Data on patient demographics and clinical characteristics were provided by clinicians. HRQOL was reported by patients in the clinic at enrollment, within two months of diagnosis. Patients completed 3 psychometrically validated instruments: the Brief Pain Inventory (BPI), EQ-5D, and Functional Assessment of Cancer Therapy-Multiple Myeloma (FACT-MM). Standard analyses were conducted of each instrument. Reported mean BPI, EQ-5D and FACT-MM scores were analyzed by patient race. Statistical significance of score differences was ascertained by ANOVA using SAS 9.1 in two ways: (1) among three different racial categories (White, Black and Other), and (2) between Blacks and non-Blacks. Hispanic/Latino patients and Asian patients comprise most of the Other cohort. Results. HRQOL data at baseline were reported by 916 pts, enrolled from 189 centers. Of these patients, 82% were White, 12% were Black, and 5% were of another race (Other). Among evaluable patients, the different race groups did not differ statistically by ECOG status or multiple myeloma stage (measured by either the International Staging System or the Durie and Salmon system). The three racial groups differed by age, with the mean (SD) age of Blacks (63 (11)) and Others (63 (13)) being less than that of Whites (68 (11), p<0.0001). Gender was trending, but not statistically significantly different, between races, with males comprising 58% of Whites, 52% of Blacks, and 46% of Others (p=0.1803). HRQOL scores by race are presented here: Overall FACT-MM results indicate that baseline HRQOL does not vary between races, as the main summary scores do not differ statistically, although the FACT-MM Total scores of Blacks and Whites are trending higher than those of Others. Only the emotional and social/family domain scores of the FACT-MM vary statistically, as Blacks show a worse mean score in the social/family domain and a better score in the emotional domain. BPI data (on a scale of 0 [no pain] to 10 [worst pain] do not show a statistically significant difference between races, and the same is true of the five EQ-5D domains (measured on a scale of 1 [no problem] to 2 [some problems] to 3 [incapacity]. Conclusions. Initial results from the Connect MM® Registry indicate that HRQOL at baseline prior to initiation of treatment does not vary by patient race. As such, these results may serve as a baseline reference for future analyses. In particular, as patients proceed through therapy, analyses should be conducted of patients by race, among other factors, to determine whether it may be associated with subsequent clinical outcomes and HRQOL over time. Disclosures: Pashos: Celgene: Membership on an entity's Board of Directors or advisory committees. Durie:Celgene: Membership on an entity's Board of Directors or advisory committees. Rifkin:Celgene: Membership on an entity's Board of Directors or advisory committees. Terebelo:Celgene: Membership on an entity's Board of Directors or advisory committees. Gasparetto:Celgene: Membership on an entity's Board of Directors or advisory committees. Mehta:Celgene: Membership on an entity's Board of Directors or advisory committees. Toomey:Celgene: Membership on an entity's Board of Directors or advisory committees. Abonour:Celgene: Membership on an entity's Board of Directors or advisory committees. Fonseca:Celgene: Membership on an entity's Board of Directors or advisory committees. Narang:Celgene: Membership on an entity's Board of Directors or advisory committees. Shah:Celgene: Membership on an entity's Board of Directors or advisory committees. Swern:Celgene Corporation: Employment, Equity Ownership. Sullivan:Celgene: Employment, Equity Ownership. Street:Celgene: Employment, Equity Ownership. Khan:Celgene: Employment, Equity Ownership.

scholarly journals Continuous Mobile Wearable Bio-Monitoring of Newly Diagnosed Multiple Myeloma Patients Undergoing Initial Chemotherapy

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4751-4751
Author(s):  
Elizabet Tavitian ◽  
Donna Mastey ◽  
Meghan Salcedo ◽  
Andrew Zarski ◽  
Aisara Chansakul ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Multiple Myeloma ◽  
Board Of Directors ◽  
Research Funding ◽  
Baseline Period ◽  
Newly Diagnosed ◽  
Advisory Committees ◽  
Activity Measurements ◽  
Bio Monitoring

Abstract Introduction The current standard to assess chemotherapy tolerability relies on patient self-reporting. However, as the sole mechanism of managing symptom burden, this may be inconsistent and fraught with bias. Mobile wearable health devices have the ability to monitor and aggregate objective activity and sleep data over long periods of time, but have not been systematically used in the oncology clinic. The aim of the study was to assess whether the use of mobile wearable technology establishes patterns of "sleep" and "wake" states in newly diagnosed Multiple Myeloma (NDMM) patients receiving therapy, and whether these patterns differ over time. Methods Patients presenting to the myeloma clinic at Memorial Sloan Kettering Cancer Center (MSKCC) with a new diagnosis of Multiple Myeloma and smart phone or tablet (iOS or Android) compatible with the Garmin Vivofit device were offered to participate in a mobile wearable bio-monitoring study. All eligible participants were required to receive primary chemotherapy treatment at a MSKCC facility. Treatment was determined by physician. NDMM patients were assigned to one of two cohorts (20 in each; Cohort A - patients <65 years; Cohort B - patients ≥ 65 years). Patients were given Garmin Vivofit devices and asked to download a Garmin Vivofit application and Medidata electronic patient reported outcome (ePRO) application on their phone or tablet. Patients were bio-monitored for physical activity and sleep during baseline period (1-7 days prior to chemotherapy initiation) and continuously up to 6 cycles of chemotherapy. Additionally, patients completed mobile ePRO questionnaires [(EORTC - QLQC30 and MY20) and brief pain inventory scales (BPI)] using the Medidata application at baseline and after each induction cycle. Activity, sleep data, and completed ePRO questionnaire data were automatically synced or transferred to Medidata Rave database through Medidata Sensorlink technology. In this abstract, we report initial results on prospective collection of activity measurements. Additional data from the health-related quality of life questionnaires and clinical outcomes will be presented at later date. Results Between February 2017-March 2018, 37 patients (19 males and 18 females) enrolled onto the study, with 20 in cohort A and 17 in cohort B. The mean age was 55 years (range 41-64) for cohort A and 72 years (range 65-82) for cohort B. Treatment regimens included Carfilzomib/Revlimid/Dexamethasone 14(38%), Velcade/Revlimid/Dexamethasone 10(27%), Daratumumab/Carfilzomib Revlimid/Dexamethasone, 7(19%), Cyclophosphamide/Velcade/Dexamethasone 3(8%), Revlimid/Dexamethasone 2(5%), and Velcade/Revlimid/Dexamethasone-Lite 1(3%). Twenty-four patients have completed the trial, and 7 remain active. Six patients came off-study due to the following reasons: lost devices (n=4), intolerable rash during cycle 3 (n=1), and incompletion of baseline activity (n=1). Three patients were excluded for incomplete data sets with no baseline data collection at the time of analysis. Fifteen patients were available for data review including 10 in cohort A and 5 in cohort B. Mean activity for cohort A was 6,437 steps/24 hr period (1,002 - 12,754) versus for cohort B was 3,218.37 steps/24 hr period (387 - 6,155) (p <0.05). In comparing pre- and post-therapy, overall mean activity for cohort A increased from 5,995 to 6,513 steps/24 hr, 8.6% increase (p=0.78), and for cohort B mean activity increased from 2,249 to 3,420 steps/24 hr, a 52% increase (p=0.2140). We assessed short term effects therapy initiation had on activity for NDMM patients by comparing percent changes in activity (steps/24 hrs) from baseline period to cycle 1 period. We found 3 patients had a >100% increase, 1 patient had 50-100% increase, and 11 patients had within +/- 50% change in activity from baseline. Conclusion Electronic mobile wearable device monitoring in symptomatic NDMM patients may be a useful tool to assess a patient's overall wellness and health as they are receiving chemotherapy. For three patients, we were able to capture a dramatic increase in activity after initiation of treatment. Overall activity in the elderly NDMM patients is decreased compared to younger patients. Mobile wearable monitoring may be an even more useful strategy for tracking elderly and unfit patients that are more prone to side effects, where the balance of response versus quality of life is paramount. Figure. Figure. Disclosures Mailankody: Physician Education Resource: Honoraria; Janssen: Research Funding; Takeda: Research Funding; Juno: Research Funding. Hassoun:Oncopeptides AB: Research Funding. Lesokhin:Squibb: Consultancy, Honoraria; Serametrix, inc.: Patents & Royalties: Royalties; Janssen: Research Funding; Bristol-Myers Squibb: Consultancy, Honoraria, Research Funding; Genentech: Research Funding; Takeda: Consultancy, Honoraria. Smith:Celgene: Consultancy, Patents & Royalties: CAR T cell therapies for MM, Research Funding. Shah:Amgen: Research Funding; Janssen: Research Funding. Landgren:Takeda: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Consultancy, Research Funding; Pfizer: Consultancy; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Amgen: Consultancy, Research Funding; Merck: Membership on an entity's Board of Directors or advisory committees; Karyopharm: Consultancy. Korde:Amgen: Research Funding.

scholarly journals Impact of Elotuzumab Plus Pomalidomide and Dexamethasone on Health-Related Quality of Life in Patients with Relapsed/Refractory Multiple Myeloma Enrolled in the ELOQUENT-3 Study

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3480-3480 ◽  
Author(s):  
Katja Weisel ◽  
Agne Paner ◽  
Monika Engelhardt ◽  
Fiona Taylor ◽  
Kim Cocks ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Multiple Myeloma ◽  
Symptom Severity ◽  
Research Funding ◽  
Longitudinal Analyses ◽  
Completion Rates ◽  
Health Related Quality ◽  
Related Quality ◽  
Health Related

Introduction: Novel triplet therapies (tx) for relapsed/refractory multiple myeloma (RRMM) have improved outcomes and extended survival. However, the use of multi-agent tx over an extended period increases the tx-related symptom burden and impacts health-related quality of life (HRQoL). Therefore, highly effective tx that preserve HRQoL are needed. The randomized phase 2 ELOQUENT-3 study (NCT02654132) demonstrated that addition of elotuzumab (elo) to pomalidomide and dexamethasone (EPd) resulted in a 46% reduction in the risk of progression/death vs Pd, without affecting HRQoL, in patients (pts) with RRMM for whom lenalidomide (len) and a proteasome inhibitor (PI) had failed (Dimopoulos et al. N Engl J Med 2018; Weisel et al. ASH 2018). Here, we present pt-reported outcomes (PROs) with EPd vs Pd after extended follow-up (FU) of ELOQUENT-3. Methods: PROs were an exploratory endpoint, assessed using the 3-level EuroQoL 5 Dimensions Questionnaire (EQ-5D-3L) and the MD Anderson Symptom Inventory MM module (MDASI-MM). The EQ-5D-3L includes a global health visual analog scale (VAS) and utility index (UI); the MDASI-MM measures total symptom severity (13 core items plus 7 MM items) and symptom interference (6 items). EQ-5D-3L UI and VAS scores range from −0.59 to 1 and 0 to 100, respectively, with minimally important differences (MIDs) of 0.08 and 7. MDASI-MM scores range from 0 to 10; MIDs were based on the standard error of the mean for subscales. Higher scores indicate better health for EQ-5D-3L, but more severe symptoms for MDASI-MM. PRO data were collected at baseline (BL), at the start of every 28-d tx cycle, at the end of tx, and during FU. All randomized pts with BL and ≥1 post-BL assessment were included in the PRO analysis. Completion rates and changes from BL scores were evaluated descriptively; completion rates from the 'expected population' did not include pts who had died or discontinued. Longitudinal analyses of change from BL used mixed effects models. First deterioration/improvement was defined as the first change from BL that was ≥responder definition threshold. Results: Of 117 randomized pts, 106 (EPd n=55; Pd n=51) had BL and ≥1 post-BL assessment and were included in PRO analyses (database lock, Nov 2018; minimum FU, 18.3 mo). BL characteristics of the PRO population were generally balanced between arms and representative of the entire study population. PRO completion rates from the expected population were ≥79% and ≥96% for the MDASI-MM and EQ-5D-3L, respectively, for all on-tx timepoints. Although completion rates between arms were similar throughout, between-tx HRQoL analysis was not feasible after Cycle 13 due to low Pd pt numbers. Mean BL scores were similar between arms: EPd vs Pd MDASI-MM total symptom severity, 1.5 vs 1.6; symptom interference, 2.5 vs 2.3; EQ-5D-3L UI, 0.70 vs 0.68; VAS, 65.6 vs 69.2. In the EQ-5D-3L UI, neither tx arm had a clinically meaningful deterioration (CMD); in the VAS, there was a CMD in the Pd arm only. In MDASI-MM total symptom severity, there was a CMD in both arms (Figure). In MDASI-MM symptom interference, there was a CMD in both arms at some timepoints (Figure). However, Pd sample sizes were small for the MDASI-MM (n≤15). Longitudinal analyses demonstrated no clinically meaningful differences between arms for EQ-5D-3L UI and VAS or MDASI-MM total symptom severity and symptom interference or the items of pain, fatigue, or bone aches. There were no statistically significant differences in time to deterioration between arms for EQ-5D-3L UI or VAS. However, there was a trend towards a reduction in the risk of deterioration in the EQ-5D-3L VAS for pts receiving EPd vs Pd (HR 0.70; 95% CI 0.43-1.14; p=0.110). Median time to deterioration was generally similar between arms across the MDASI-MM subscales. Hospitalizations were similar between EPd (32 pts [53%]) and Pd arms (31 pts [54%]). Mean duration of hospitalization was 9.9 d with EPd and 12.9 d with Pd. Conclusions: HRQoL was similar between pts who received EPd and Pd in ELOQUENT-3, demonstrating the addition of elo to Pd did not impair HRQoL. These pt-reported findings complement extended FU data that demonstrated EPd gave clinically meaningful improvements in survival without increasing toxicity, further supporting the use of EPd in pts with RRMM after failure of len and a PI. Further PRO analysis in a larger study is warranted. Study support: BMS. Writing support: Adam Gill, Caudex, funded by BMS. Disclosures Weisel: Sanofi: Consultancy, Honoraria, Research Funding; Janssen: Consultancy, Honoraria, Research Funding; Adaptive Biotech: Consultancy; Celgene: Consultancy, Honoraria, Research Funding; Bristol-Myers Squibb: Consultancy, Honoraria; Amgen: Consultancy, Honoraria, Research Funding; Takeda: Consultancy, Honoraria; Juno: Consultancy. Paner:Rush University Medical Center: Employment; Dova: Consultancy, Honoraria; Takeda: Consultancy, Honoraria; Cellectar: Consultancy, Honoraria; Cellectar: Consultancy, Honoraria; Janssen: Consultancy, Honoraria; Abbvie: Consultancy, Honoraria; Amgen: Consultancy, Honoraria; Celgene: Consultancy, Honoraria; Oncopeptides: Consultancy, Honoraria. 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. Cocks:Amgen: Consultancy; BMS: Consultancy; Adelphi Values: Employment; Celgene Corporation: Consultancy; Endomag Ltd.: Consultancy. Popa-McKiver:Bristol-Myers Squibb: Employment. Chen:Bristol-Myers Squibb: Employment. Cavo:Janssen, Celgene: Other: Travel Accommodations; Celgene, Janssen, Amgen, BMS, Abbvie, Takeda: Honoraria; Janssen, Celgene: Speakers Bureau; Janssen, Celgene, Amgen, Abbvie: Consultancy, Membership on an entity's Board of Directors or advisory committees.

scholarly journals Health-Related Quality-of-Life Results From the Open-Label, Randomized, Phase III ASPIRE Trial Evaluating Carfilzomib, Lenalidomide, and Dexamethasone Versus Lenalidomide and Dexamethasone in Patients With Relapsed Multiple Myeloma

2016 ◽  
Vol 34 (32) ◽  
pp. 3921-3930 ◽  
Author(s):  
A. Keith Stewart ◽  
Meletios A. Dimopoulos ◽  
Tamás Masszi ◽  
Ivan Špička ◽  
Albert Oriol ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Multiple Myeloma ◽  
Minimal Important Difference ◽  
Phase Iii ◽  
Life Questionnaire ◽  
Health Related Quality ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related

Purpose To determine the effects of carfilzomib, lenalidomide, and dexamethasone (KRd) versus lenalidomide and dexamethasone (Rd) on health-related quality of life (HR-QoL) in the Carfilzomib, Lenalidomide, and Dexamethasone Versus Lenalidomide and Dexamethasone for the Treatment of Patients With Relapsed Multiple Myeloma (ASPIRE) trial. Methods Patients with relapsed multiple myeloma were randomly assigned to receive KRd or Rd. The European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire C30 and myeloma-specific module were administered at baseline; day 1 of cycles 3, 6, 12, and 18; and after treatment. The Global Health Status/Quality of Life (GHS/QoL) scale and seven subscales (fatigue, nausea and vomiting, pain, physical functioning, role functioning, disease symptoms, and adverse effects of treatment) were compared between groups using a mixed model for repeated measures. The percentages of responders with ≥ 5- or 15-point GHS/QoL improvement at each cycle were compared between groups. Results Baseline questionnaire compliance was excellent (94.1% of randomly assigned patients). KRd patients had higher GHS/QoL scores versus Rd patients over 18 treatment cycles (two-sided P < .001). The minimal important difference was met at cycle 12 (5.6 points) and approached at cycle 18 (4.8 points). There was no difference between groups for the other prespecified subscales from ASPIRE. A higher proportion of KRd patients met the GHS/QoL responder definition (≥ 5-point improvement) with statistical differences at cycle 12 (KRd v Rd patients, 25.5% v 17.4%, respectively) and 18 (KRd v Rd patients, 24.2% v 12.9%, respectively). Conclusion KRd improves GHS/QoL without negatively affecting patient-reported symptoms when compared with Rd. These data further support the benefit of KRd in patients with relapsed multiple myeloma.

scholarly journals Health Related Quality of Life and Fatigue in Patients with Pyruvate Kinase Deficiency

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4807-4807 ◽  
Author(s):  
Eduard J. Van Beers ◽  
Kevin H. M. Kuo ◽  
D. Holmes Morton ◽  
Wilma Barcellini ◽  
Stefan W. Eber ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Pyruvate Kinase ◽  
Research Funding ◽  
Healthy Population ◽  
Missense Mutations ◽  
Advisory Committees ◽  
Related Quality ◽  
Health Related ◽  
Euroqol 5D

Abstract Background: Pyruvate Kinase (PK) deficiency is the most common enzyme defect of the glycolytic pathway causing hereditary non-spherocytic hemolytic anemia. Patients have a broad phenotypic spectrum, ranging from mild anemia to transfusion dependence, and there is wide variation in transfusion practices and decisions about splenectomy. No prior studies have reported on the use of validated health related quality of life (HRQoL) measures in this population. Aim: To describe patient reported outcomes including general HRQoL and fatigue in adults with PK deficiency and the correlation with clinical and laboratory features. Methods: Patients were enrolled on the PK Deficiency Natural History Study, a prospective 30 site international study. All patients had molecularly confirmed PK deficiency. Adults (n=132), ages ≥18 years, completed the EuroQol-5D (high index equivalent to better QoL), PROMIS Fatigue Short Form 7a (high scores equivalent to higher fatigue), and the Functional Assessment of Cancer Therapy-Anemia (FACT-An, high scores equivalent to less fatigue) surveys at enrollment and annually. Timing of administration was convenience based. Survey data were analyzed according to proprietary scoring guidelines. Tests of association were performed using Fisher's exact test (categorical) and Wilcoxon rank sum test (continuous). Regular transfusions were defined as ≥6 transfusions in 12 months. Genotypes were grouped as two missense mutations (M/M), one missense/one non-missense (M/NM), or two non-missense mutations (NM/NM)); non-missense included deletions or other drastic variants. The minimal important difference (MID) for the FACT-An has been reported as 7 points (Cella et al, J Pain Symptom Manage 2002). P-values <0.05 were considered statistically significant. Results: At enrollment, 128 adults completed the FACT-An with a median total score of 156 (IQR 122-190). Patients receiving regular transfusions reported significantly lower FACT-An scores than those who were not regularly transfused (median 129 vs 156, p=0.004) with significantly lower scores for physical, emotional, and functional well-being and anemia sub-scores (Table). This difference also surpassed the MID. However, non- regularly transfused patients with hemoglobin (Hb) <8 g/dl did not report significantly different scores than those with Hb≥ 8 g/dl (p=0.75). There were also no significant differences in FACT-An scores by splenectomy status or age. The FACT-An score differences were greater than the MID for patients with iron overload (ferritin >1000 ng/dL or chelation), higher number of lifetime transfusions, and two missense mutations. Females reported significantly lower scores than males (median 143 vs. 160, p=0.006) with significantly lower anemia sub-scores (p=0.0009). FACT-An surveys completed at the one year follow-up time point validated these findings. EuroQol-5D scores (n=124) at enrollment were similar to the healthy population (median PK deficiency index score 0.88; healthy population index mean 0.88, Shaw et al, Medical Care 2005). No significant differences were found by Hb level, splenectomy status, transfusion status, or genotype group. The median PROMIS fatigue T score (n=66) was 52.1 (IQR 40.5-63.7). Similar to the FACT-An survey data, PROMIS fatigue scores were significantly worse in patients who were regularly transfused (67.0 vs 52.4, p=0.02). PROMIS fatigue scores were also significantly worse in patients ≥40 years old (p=0.05) and females (p=0.01). Conclusions: Using the FACT-An and PROMIS Fatigue measures, patients with PK deficiency who are regularly transfused report significantly more fatigue and worse HRQoL compared with those who are not transfused. Important differences were also seen by iron status and mutation group using the FACT-An. Patients report similar fatigue levels regardless of Hb level, which suggests that symptoms, rather than Hb value alone, should be factored into clinical decision making. In contrast to anemia related HRQoL scores, overall HRQoL scores using validated generic measures in patients with PK deficiency show no differences compared with the healthy population, suggesting that disease-specific measures may better detect the effects of PK deficiency on HRQoL. Disclosures Van Beers: Agios Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding. Glader:Agios Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding. Chonat:Agios Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees. Despotovic:AmGen: Research Funding; Novartis: Research Funding; Sanofi: Consultancy. Kwiatkowski:bluebird bio: Consultancy, Honoraria, Research Funding; Agios Pharmaceuticals: Consultancy, Research Funding; Novartis: Research Funding; Apopharma: Research Funding; Terumo: Research Funding. Thompson:La Jolla Pharmaceutical: Research Funding; Baxalta/Shire: Research Funding; Novartis: Research Funding; Celgene: Research Funding; bluebird bio: Consultancy, Research Funding; Biomarin: Research Funding; Amgen: Research Funding. Newburger:TransCytos LLC: Consultancy; Janssen Research & Development, LLC: Consultancy, Honoraria; X4 Pharmaceutics: Consultancy, Honoraria. Ravindranath:AGIOS: Other: Site Investigator for Pyruvate Kinase Deficiency. Sheth:Terumo Corporation: Research Funding; Novartis: Research Funding; La Jolla Pharmaceutical Company: Research Funding; Celgene Corporation: Consultancy, Research Funding; Bluebird Bio: Consultancy. Grace:Agios Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees; Agios Pharmaceuticals: Research Funding; Agios Pharmaceuticals: Consultancy.

scholarly journals Pilot Trial of the My Hematology Oncology Patient Experience (MyHOPE™) for Multiple Myeloma (MM) Digital Solution in Patients with MM

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 3-4
Author(s):  
Noopur S. Raje ◽  
Omar Nadeem ◽  
Joseph Mikhael ◽  
Catherine Ludwig ◽  
Amit Agarwal ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Board Of Directors ◽  
Pilot Trial ◽  
Advisory Committees ◽  
Digital Platforms ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related ◽  
The Usa

Background: There is growing evidence to suggest that continuous monitoring and a proper use of patient-reported outcomes (PROs) in the clinical setting may improve patient care by facilitating doctor-patient communication, promoting individualized supportive care, and increasing patient satisfaction (Velikova, J Clin Oncol. 2004;22(4):714-24; Yang, Support Care Cancer. 2018 Jan;26(1):41-60). This may be of particular importance for patients with MM, a condition characterized by considerable heterogeneity in PROs at different timepoints within a patient's disease journey (TA King et al, Semin Oncol Nurs. 2017;33(3):299-315;R Abonour et al, Ann Hematol. 2018;97(12):2425-36). There is also a growing body of evidence to support that digital platforms in healthcare can have an impact on patients' lives and improve the patient care experience across diverse medical settings. Web-based platforms that provide education and allow patients to track data have demonstrated improvement across a range of PROs measuring empowerment, self-efficacy, and mastery when compared against standard of care in a variety of disease states (MR Fu et al, Internet Interv. 2016;5:56-64; G Bouma et al, Support Care Cancer. 2017;25(7):2075-2083). This may be particularly relevant in light of the current coronavirus disease 2019 (COVID-19) global pandemic. Patient-reported outcome data (e.g., symptoms) that can be readily shared with healthcare professionals (HCPs) can support clinical decision making and impact patient outcomes (E Basch et al, JAMA. 2017 Jul 11;318(2):197-8). Data collected through wearables (such as emoji scales and activity) have been shown to be correlated with traditional, validated PRO measures in patients with cancer (CA Thompson et al, Blood. 2017;130(Suppl 1):2179). Digital medication tracking has led to better medication adherence, increased prescription refills, and better clinical outcomes. Centralized disease management digital platforms have shown potential to reduce patients' risk of complications (S Kumar et al, Abstract for the 77th American Diabetes Association. 2017). Despite this body of evidence supporting the potential impact of the use of digital platforms, there is little evidence specifically in MM. The MyHOPE™ for MM Solution is a validated investigational digital technology platform designed to provide patients with a comprehensive set of tools and resources to support the patient throughout their overall experience with MM and to collect biometrics and self-reported data such as symptom tracking, medication adherence, and health-related quality of life (HRQoL) with the ability to share this data with the patient's care team. MyHOPE™ for MM is the first prospective study evaluating the impact of a digital intervention for patients with hematological malignancies. Study Design and Methods: This is a multi-center, randomized, pilot trial of the MyHOPE™ for MM Solution. Approximately 126 adult patients (≥ 18 years of age) with a diagnosis of MM and who reside in the USA will be recruited from approximately 30 study sites within the USA, reflecting both community and academic centers. Patients will be stratified according to disease status (newly-diagnosed multiple myeloma transplant-eligible or ineligible, newly-diagnosed multiple myeloma in patients undergoing their first autologous stem cell transplant, or relapsed and/or refractory) and will be randomized in a 2:1 manner to either the Patient App + HCP Portal (Cohort 1) or Patient App Alone (Cohort 2). Primary objectives include feasibility of the platform and patient empowerment and self-efficacy. Other objectives include user satisfaction with the platform, health-related quality of life, and clinical outcomes. Enrolled patients will receive MM-treament regimen according to their physician's care plan. The study is in start-up and recruitment is expected through 2021. Disclosures Raje: Celgene: Consultancy. Nadeem:Amgen: Membership on an entity's Board of Directors or advisory committees; Adaptive: Membership on an entity's Board of Directors or advisory committees; Sanofi: Consultancy, Membership on an entity's Board of Directors or advisory committees; Takeda: Consultancy, Membership on an entity's Board of Directors or advisory committees, Other: TRAVEL, ACCOMMODATIONS, EXPENSES; Celgene: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: TRAVEL, ACCOMMODATIONS, EXPENSES; Janssen: Consultancy, Honoraria, Other: TRAVEL, ACCOMMODATIONS, EXPENSES. Mikhael:Amgen, Celgene, GSK, Janssen, Karyopharm, Sanofi, Takeda: Honoraria. Ludwig:Bristol Myers Squibb: Consultancy. Agarwal:Bristol-Myers Squibb Company: Current Employment, Current equity holder in publicly-traded company. Inumerable:Bristol Myers Squibb: Current Employment. Ong:Bristol Myers Squibb: Current Employment. Jagannath:BMS, Janssen, Karyopharm, Legend Biotech, Sanofi, Takeda: Consultancy.

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