scholarly journals Content Validation of the Sickle Cell Pain Diary-Self Report

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 2277-2277
Author(s):  
Miranda Bailey ◽  
Patricia Stebbins ◽  
Denise D'Alessio ◽  
Kimberly Raymond ◽  
Michelle K White
Keyword(s):  
Sickle Cell ◽  
Research Funding ◽  
Qualitative Interviews ◽  
Self Report ◽  
Content Validation ◽  
Expert Review ◽  
Patient Interviews ◽  
The Us ◽  
The Impact ◽  
Response Choices

Abstract Background: Sickle Cell Disease (SCD) is a genetic, progressive, vascular disease that affects approximately 100,000 children and adults in the US. Vaso-occlusive crises (VOCs), also referred to as pain crises, are a primary complication of SCD. VOCs are thought to be caused by several factors, including inflamed, damaged vasculature and increased cellular adherence to the endothelium and other cells, resulting in occlusion of the microvascular system. VOCs have been associated with increased morbidity and mortality. Healthcare visit data partially reflect VOC and SCD outcomes and provide an understanding of healthcare resource utilization (HCRU). However, evaluating only HCRU data misses the breadth and depth of the patient experience and burden of SCD as it fails to capture daily variations of pain and other important quality of life (QoL) concepts. Additionally, patients who avoid seeking care outside the home are not represented in HCRU data. The Sickle Cell Pain Diary- Self Report (SCPD-S) was developed as a daily patient-reported outcome (PRO) measure primarily intended to capture the frequency and severity of SCD-related pain during and outside of a VOC and secondarily to examine the impact of the pain on other patient-relevant QoL concepts. The objective of this study was to investigate the content validity of the SCPD-S. Methods: A draft diary was developed from the literature. In line with FDA guidance for PRO development, the content validation of the SCPD-S included four consecutive steps: (1) literature review to identify important concepts to measure and mapping of concepts to draft diary; (2) expert review to improve wording and layout; (3) round 1 (N=13) of qualitative interviews with patients; and (4) round 2 (N=6) of qualitative interviews with patients. Revisions made to the diary after each step were tested in the following step. In total, 18 in-depth, hybrid concept elicitation (CE) and cognitive debriefing (CD) interviews were conducted in English in person (n=13) and by phone (n=5) with SCD patients in the US aged ≥12 years. A semi-structured guide was used to explore concepts relevant to patients' experience with VOCs and to test the draft diary. The CD portion of the interviews required patients to state their thoughts while reading the diary aloud, after which interviewers probed on areas that seemed confusing and asked questions specific to the diary instructions, recall period, items, and response choices. All interviews were recorded, transcribed, coded and analyzed. Results: All four steps of this content validation study resulted in changes to the SCPD-S. The literature review, expert review, and round 1 patient interviews resulted in expansion of the SCPD-S from 8 to 19 items covering concepts highlighted by patients as important. For example, impact of VOCs on school/work, activities of daily living, social and recreational activities, sleep, and emotional wellbeing were added. Five items from the original diary were significantly revised, as were all response choices. Round 2 interviews confirmed the comprehensiveness of the revised diary, comprehensibility of the wording, and appropriateness of the recall period and response choices. Final changes to the diary included adding skip logic to ensure transition to an electronic format. Saturation analyses revealed that no additional interviews were needed. Conclusions: This study provided evidence that supports the content validity of the SCPD-S, a self-report SCD daily diary focused on capturing daily variations of pain in SCD. Evidence gathered during patient interviews indicated that the SCPD-S is a valuable, fit for purpose measure of VOC-related pain frequency and severity as well as the impact of this pain on other QoL concepts including fatigue and emotional health. In addition, the diary captures the QoL of patients who may not seek care outside the home providing a more holistic view of the overall impact of VOCs and SCD. The numerous changes to the SCPD-S as a result of the study findings highlight the importance of the content validation process in establishing a PRO measure. Future work on the SCPD-S includes development of a scoring algorithm and user's manual, and conducting psychometric validation. Use of the SCPD-S is anticipated in future clinical trials enrolling patients who experience VOCs as a way to help capture the QoL impacts of new interventions designed to reduce the frequency and intensity of VOCs. Disclosures Bailey: Novartis: Employment. Stebbins:Optum: Employment; Novartis: Research Funding. D'Alessio:Novartis: Employment. Raymond:Novartis: Research Funding; Optum: Employment. White:Akcea: Research Funding; Optum: Employment.

scholarly journals Content validation of a self-report daily diary in patients with sickle cell disease

2021 ◽  
Vol 5 (1) ◽  
Author(s):  
Michelle K. White ◽  
Cory Saucier ◽  
Miranda Bailey ◽  
Denise D’Alessio ◽  
April Foster ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Sickle Cell ◽  
Self Report ◽  
Content Validation ◽  
Cell Disease ◽  
Concept Elicitation ◽  
Related Quality ◽  
Health Related ◽  
The Impact

Abstract Background Sickle Cell Disease (SCD) is a genetic progressive vascular disease that impacts patients overall health and quality of life. Sickle-cell pain crises (SCPCs) are a hallmark clinical presentation of SCD and have been associated with increased morbidity and mortality. The Sickle Cell Pain Diary- Self Report (SCPD-S) was developed as a daily patient-reported outcome (PRO) measure primarily intended to capture the frequency and severity of SCD-related pain during and outside of a SCPC. The SCPD-S also examines the impact of the pain associated with an SCPC on other health-related quality of life concepts. The objective of this study was to investigate the content validity of the SCPD-S. Methods The content validation testing included 18 in-depth hybrid concept elicitation and cognitive debriefing interviews conducted with SCD patients in the US aged 12 years and older. Interviewers used a semi-structured interview guide and a think-aloud approach for the cognitive debriefing portion. All interviews were recorded, transcribed, coded and analyzed. Results Eighteen interviews across two rounds were conducted. Round 1 hybrid interviews (n = 12) resulted in the expansion of the SCPD-S from 13 to 19 items. Items on the impact of an SCPC on social and recreational activities, sleep, and emotional well-being were added. Five items were significantly revised, as were three response choice sets. Round 2 hybrid interviews (n = 6) confirmed the comprehensiveness of the revised diary, understandability of the wording, and appropriateness of the recall period and response sets. Saturation analyses specific to concept elicitation revealed that no additional interviews were needed. Conclusions This study provided evidence to support the content validity of the SCPD-S, a self-report daily diary. Data gathered during patient interviews indicated that the SCPD-S is a fit for purpose measure of SCD and SCPC-related pain frequency and severity and the impact of this pain on other health-related quality of life concepts including fatigue and emotional health. The numerous changes to the SCPD-S as a result of the study findings highlight the importance of the content validation process when developing a PRO measure.

scholarly journals COVID Symptoms and COVID Anxiety in Sickle Cell Disease

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 16-17
Author(s):  
Wally R Smith ◽  
Benjamin Jaworowski ◽  
Shirley Johnson ◽  
Thokozeni Lipato ◽  
Daniel M Sop
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Research Funding ◽  
Telephone Survey ◽  
Cell Disease ◽  
Weighted Mean ◽  
The Us ◽  
Associated Symptoms ◽  
At Home

Background Even before the US upswing of the current COVID pandemic, the number of sickle cell disease (SCD) patients coming to hospitals and EDs appeared to fall drastically. This happened despite SCD patients having often been heavy utilizers of the ED and hospital for their iconic vaso-occlusive crises (VOC). Though ambulatory SCD clinics quick converted largely to telehealth in order to comply with stay-at-home orders designed to suppress person-to-person transmission, some SCD patients appeared to avoid care, delay care, or refuse doctors' invitations for care. Presumably patients did so out of COVID fears, but this has not been confirmed in the literature. Further, whether these patients had COVID symptoms but stayed at home has not been studied. As part of quality improvement (QI) to conduct COVID surveillance in an adult sickle cell program, we sought to explain and predict SCD health care utilization patterns we were observing, as well as to determine urgent physical and mental health needs of patients who appeared to be avoiding care. Methods Fifteen staff in the Adult Sickle Cell Medical Home at Virginia Commonwealth University, a large urban academic medical center, conducted a telephone survey ("wellness check"was used when we talked to patients) of all known adults with SCD over 19 days in 2020. A staff member confirmed the patient had SCD, asked permission to proceed, then asked about symptoms consistent with COVID-19. At the end of the telephone survey, respondents wer invited to complete an email survey of sickle cell and COVID-19 utilization attitudes (19-33 items, depending on the response pattern, either drawn from the National Health Interview Survey, from the Adult Sickle Cell Quality of Life Measurement quality of care survey, or drafted by the authors), the Sickle Cell Stress Survey-Adult (SCSS-A, a 10-item previously validated survey), and anxiety and depression (PHQ9 of the PRIME-MD). Results Of 622 adults approached by phone call, 353 responded to the following yes/no screening questions regarding the prior 14 days: fever over 100 F 0/353 (0.00%); cough 3/353(0.01%); difficulty breathing 0/353(0.00%); unexplained shortness of breath 2/353(0.01%); sore throat 2/353 (0.01%); unexplained muscle soreness 2/353(0.01%);contact with anyone who tested positive for COVID-19 2/353(0.01%); testing for COVID 19 6/353(0.02%). For QI purposes, we set a threshold of three or more COVID-associated symptoms or the presence of fever as criteria requiring intense telephone or in-person staff monitoring for the following week. Only three patients met criteria. A total of 219/353 had email surveys sent. Of 63 patients (28.8%) who returned email surveys by June 10, 2020, 35.9% had already managed a "pain attack" at home 4 or more times in the prior 12 months, and 45.5% of these said their bad ER experiences were very or somewhat important in that decision. In the prior 14 days, although 30/64 reported a crisis for at least one day, only 4/64 had visited the Emergency Department for pain. On a 0-10 scale, 21/61 patients endorsed "0" for worry that they would be COVID-infected by going for medical care (weighted mean 3.9), but 18/59 endorsed "10" for worry they were more at risk of COVID because of SCD (weighted mean 6.31), and 22/60 endorsed "10" for worry they would fare worse than others if COVID infected (weighted mean 6.97). Many patients forwent "needed" care (16/62) or delayed "needed" care by at least a day (36/61). Eleven patients met criteria for moderately severe to severe depression on the PHQ-9, and 28/63 somewhat or strongly agreed with the statement "death is always on the back of my mind" on the SCSS-A. Conclusions In adolescents and adults with SCD, many were already reticent to come to the ED for pain, but a significant portion reported delays or avoidance of needed care during the early stages of the US COVID pandemic, and few reported using the ED despite over half reporting at least one crisis day in 14. Patients nonetheless reported very few COVID-associated symptoms. Fears of COVID infection/susceptibility may limit visits for needed sickle cell care among adults. Acknowledgements: Mica Ferlis RN, FNP, Caitlin McManus, RN, FNP, Emily Sushko, RN, FNP, Justin West, RN, Kate Osborne, RN, Stefani Vaughan-Sams, Marla Brannon, BS, Nakeiya Williams, BS Disclosures Smith: GlycoMimetics, Inc.: Consultancy; Emmaeus Pharmaceuticals, Inc.: Consultancy; Novartis, Inc.: Consultancy, Other: Investigator, Research Funding; Global Blood Therapeutics, Inc.: Consultancy, Research Funding; Shire, Inc.: Other: Investigator, Research Funding; NHLBI: Research Funding; Patient-Centered Outcomes Research Institute: Other: Investigator, Research Funding; Health Resources and Services Administration: Other: Investigator, Research Funding; Incyte: Other: Investigator; Pfizer: Consultancy; Ironwood: Consultancy; Novo Nordisk: Consultancy; Imara: Research Funding; Shire: Research Funding.

Right-to-Left Shunts Are Unexpectedly Common in Adults with HbSS

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3659-3659 ◽  
Author(s):  
Bryan Christopher Hambley ◽  
Rania Abdul Rahman ◽  
Mary Ann O'Riordan ◽  
Nathan Langer ◽  
Seth Rotz ◽  
...  
Keyword(s):  
Sickle Cell ◽  
Research Funding ◽  
Medical Center ◽  
Adult Population ◽  
Laboratory Data ◽  
Acute Chest Syndrome ◽  
Compound Heterozygous ◽  
Continuous Variables ◽  
Cardiopulmonary Complications ◽  
The Impact

Abstract INTRODUCTION: Patients with homozygous sickle cell anemia (SCA) have frequent cardiopulmonary complications. The clinical prevalence and consequences of intracardiac or intrapulmonary right-to-left shunts in SCA are unknown. Here we report a retrospective examination of this complication, and clinical and laboratory correlates at clinical baseline, in an adult population with SCA. These shunts may be of particular relevance, due to the susceptibility of these patients to thrombosis and the role that desaturated hemoglobin plays in the underlying pathophysiology of this disease. METHODS: This single-institution study included 153 patients with homozygous HbSS, who are followed at Case-UH Medical Center, Cleveland. Clinical and laboratory data were gathered on patients who underwent an echocardiogram with bubble contrast. Echocardiograms were reviewed to confirm the presence, characteristics, and degree of a right-to-left shunt. Immediate (intracardiac) or delayed (intrapulmonary) shunts were identified; the latter were quantitated as <5, 5-15, or >15 bubbles visualized in the left heart. Continuous variables were described, using mean ± standard deviation (SD) and nominal variables were described as N (%). The relationship between clinical characteristics and echo results was determined using linear regression. All analyses were undertaken using SAS v9.4 The SAS Institute, Cary, NC. n.b. Compound heterozygous sickle cell disease patients showed this complication rarely, and were not examined systematically. RESULTS: 82/153 (53.6%) of studied SCA patients were female. Mean (SD) age in years was 32.2 (11.8). 90 (58.8%) patients had an echo with bubble study. In our population, 27 (17.6%) patients had an intracardiac shunt, 41 (26.8%) had an intrapulmonary shunt, and 22 (14.4%) had no shunt present; 63 (41.2%) patients did not have a bubble study. Mean (SD) LDH was 523.3 (318.1) for those with intracardiac shunts and 570.5 (205.4) for those with intrapulmonary shunts. Mean (SD) LDH for all shunt patients combined was 551.3 (255.9). Mean LDH (SD) for patients either with no shunt or no contrast echocardiogram was 415.5 (193.0). In a combined analysis, patients with shunts (N=68) showed a statistically higher baseline mean LDH when compared to a mean LDH of patients with either a negative bubble study or no bubble study (N=85), (551.3 vs 415.5, p<0.001). No difference was seen in prevalence of clinical outcomes of acute chest syndrome or stroke between groups. Laboratory markers of disease activity, such as absolute reticulocyte count or baseline hemoglobin were not different in shunt versus non-shunt patients. 8 patients had <5 bubbles in the left atrium or ventricle, 7 had 5-15 and 25 >15 bubbles. There was no evidence that this had an effect at clinical baseline, but the impact of such shunts during crises or multi-organ failure has not yet been assessed. A small number of individuals with shunts showed significant oxygen desaturation with exercise, but these were not statistically distinct from other groups. CONCLUSIONS: 68 patients with HbSS, or almost half of the adult SCA population, had right-to-left shunting on echocardiograms, either intracardiac or intrapulmonary, when evaluated at clinical baseline. These patients had significantly higher LDH than did patients with no bubble study or a negative bubble study, when evaluated in a combined post hoc analysis. The pathophysiology of these shunts is not known, but these have the potential to delay reoxygenation of sickle hemoglobin at clinical baseline and to facilitate right-to-left passage of fat or thrombotic emboli during clinical exacerbation. We are currently evaluating the potential impact of these shunts in patients with HbSS prospectively, both at baseline and during clinical exacerbation. Disclosures Schilz: Genetech: Speakers Bureau; Bayer: Consultancy, Speakers Bureau; Gilead: Consultancy, Speakers Bureau; Actelion: Consultancy, Speakers Bureau; United Therapeutics: Consultancy, Research Funding; Merck: Research Funding; Arena: Research Funding; Eiger: Research Funding.

scholarly journals Relationship of Hydroxyurea Adherence to Depression and Fatigue Among Children and Adolescents with Sickle Cell Disease: A Longitudinal Cohort Study

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3104-3104
Author(s):  
Sherif M. Badawy ◽  
Kathryn King ◽  
Olivia E. Atherton ◽  
Daniel Mroczek ◽  
Alexis A. Thompson ◽  
...  
Keyword(s):  
Cohort Study ◽  
Sickle Cell Disease ◽  
Children And Adolescents ◽  
Sickle Cell ◽  
Research Funding ◽  
Self Report ◽  
Cell Disease ◽  
Laboratory Markers ◽  
Relationship Of ◽  
Over Time

Abstract Background: Sickle cell disease (SCD) is the most common genetic disorder in the United States, seen in 100,000 Americans. SCD complications include pain episodes, chronic anemia and long-term end organ damage, leading to significant impairment in health-related quality of life (HRQOL) across the lifespan. Hydroxyurea (HU) reduces morbidity and mortality, improves HRQOL and lowers healthcare utilization, yet adherence remains suboptimal. Limited evidence from cross-sectional studies demonstrates an association between lower HU adherence and worse HRQOL scores. Objective: To assess the longitudinal relationship of HU adherence to HRQOL domains, including fatigue and depression. We hypothesized that higher HU adherence over time would be associated with improvement in HRQOL domain scores, especially depression and fatigue. Methods: In this longitudinal cohort study (NCT04675645), patients were enrolled from the comprehensive sickle cell clinic at Lurie Children's Hospital of Chicago. Patients were eligible if they were ³8 years old, had SCD (any genotype), and on HU with a stable dose for ³2 months. Study assessments included PROMIS ® measures for depression and fatigue, self-report of adherence using visual analogue scale (VAS), and patient demographics. Assessments were completed at baseline and every 3 months with a total of 5 visits (0, 3, 6, 9 and 12 months). Laboratory markers of adherence collected from chart review, including fetal hemoglobin (HbF%) and mean corpuscular volume (MCV). We conducted bivariate correlations among demographic variables, adherence markers and HRQOL scores as well as among adherence variables (VAS, HbF, MCV) at each visit. We conducted different multilevel models (MLMs), fixed and random effects, to understand the extent to which between- and within-person variation in adherence was associated with HRQOL scores over the 12-month period. We report unstandardized betas (B) and 95% Confidence Intervals (CI) from the MLMs. Results: Twenty-three patients have been enrolled (96% HbSS, 65% females, 100% Black, median age 15 [range 9-22] years old). At baseline, participants had a median Hb level of 9.5 (IQR 8.3-10.3 g/dl) with a HbF of 16.4% (IQR 13.1-28.7%) and MCV of 106.5 fl (IQR 91.6-113.9 fl). Participants' MCV levels significantly correlated with HbF% and VAS at visit 1 (r=0.58, P &lt;0.01; r=0.6, P &lt;0.01), visit 2 (r=0.66, P &lt;0.01; r=0.63, P &lt;0.01), visit 4 (r=0.76, P &lt;0.01; r=0.72, P &lt;0.01) and visit 5 (r=0.71, P &lt;0.01; r=0.59, P &lt;0.05), respectively. Participants' VAS adherence levels significantly increased from visit 1 to visit 5 (median 72 [IQR 60-92] vs. 88 [IQR 75-95], P=0.04, respectively) along with significant improvement in their fatigue scores (median 52.8 [IQR 35.1-70.5] vs. 30.8 [IQR 13.2-48.4], P=0.001, respectively). Variation in fatigue and depression scores across the study period was due to between-person differences (38% and 71%, respectively) or within-person fluctuations (62% and 29%, respectively). Using fixed and random effect MLMs, between-person differences in HU adherence over 12 months using VAS and HbF% were significantly related to participants' reported depression (B -0.43, 95% CI -0.69 to -0.17, P &lt;0.01; B -0.58, 95% CI -1 to -0.15, P &lt;0.05, respectively) (Figure 1) and fatigue scores (B -0.42, 95% CI -0.68 to -0.16, P &lt;0.01; B -0.43, 95% CI -0.78. to -0.06, P &lt;0.05, respectively) (Figure 2). In contrast, we found no statistically significant effects of within-person variation in adherence, using VAS and HbF, on participants' reported fatigue and depression scores over 12 months, which could be due our small sample size. Conclusions: Children and adolescents who were more adherent to HU across the entire study period were less likely to experience fatigue and depression, compared to those who were less adherent. Participants' self-report and laboratory markers of adherence were significantly correlated across study visits. Within-person fluctuations in adherence were not associated with changes in fatigue and depression scores across the study period. Future multi-institutional studies with a larger sample size are needed to better understand the within-person effects of variation in HU adherence on HRQOL scores over time. Behavioral interventions, such as mHealth apps, that are focused on improving HU adherence among children and adolescents with SCD has the potential to improve HRQOL and other important health outcomes. Figure 1 Figure 1. Disclosures Badawy: Bluebird Bio Inc: Consultancy; Vertex Pharmaceuticals Inc: Consultancy; Sanofi Genzyme: Consultancy. Thompson: Biomarin: Research Funding; Baxalta: Research Funding; bluebird bio, Inc.: Consultancy, Research Funding; Celgene/BMS: Consultancy, Research Funding; CRISPR Therapeutics: Research Funding; Vertex: Research Funding; Editas: Research Funding; Graphite Bio: Research Funding; Novartis: Research Funding; Agios: Consultancy; Beam: Consultancy; Global Blood Therapeutics: Current equity holder in publicly-traded company. Cella: FACIT: Membership on an entity's Board of Directors or advisory committees.

scholarly journals Risk of Venous Thrombosis in Patients with Sickle Cell Trait after Orthopedic Surgery

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 417-417
Author(s):  
Jahnavi Gollamudi ◽  
Sadeer Al-Kindi ◽  
Petra Martin ◽  
Jane Little ◽  
Lalitha V. Nayak
Keyword(s):  
Logistic Regression ◽  
Sickle Cell ◽  
Orthopedic Surgery ◽  
Research Funding ◽  
Sickle Cell Trait ◽  
Knee Surgery ◽  
Orthopedic Procedures ◽  
Major Orthopedic Surgery ◽  
The Us ◽  
Hip And Knee Surgery

Abstract Introduction Sickle cell trait (SCT) is considered to confer a hypercoagulable state. Historically, venous thromboembolism (VTE, deep vein thrombosis and PE) rates for untreated patients after major orthopedic surgery (hip or knee replacement or hip fracture surgery) have been close to 4.3%, however with the introduction of post-op anticoagulation, the rates have been as low as 1.15%. Although guidelines exist regarding anticoagulation for up to 35 days after major orthopedic surgery, there are no specific recommendations for patients with SCT. The purpose of this retrospective study is to examine the rates of VTE after major orthopedic surgery in a cohort of patients with SCT. We hypothesize that rates of VTE would be higher in patients with SCT and the risk of VTE would persist beyond 35 days. Methods A commercial database (Explorys Inc, Cleveland, OH, USA), an aggregate of electronic health record data from 26 major integrated US healthcare systems representing a sixth of the US population, was queried for data, using Systematized Nomenclature of Medicine (SNOMED) clinical terms or codes. Cases were defined as patients with SCT who underwent major knee or hip surgery. Since a majority of the US population with SCT are African American (AA) patients, controls were defined as AA patients without SCT undergoing major orthopedic surgery. For the primary end point of VTE, only adult patients (≥18 years) were selected. Those with previous history of VTE, thrombophilia, malignant disease, antiphospholipid antibody syndrome and other hemoglobinopathies such as sickle cell disease were excluded. 30 and 90-day rates of VTE were recorded for both groups. Logistic regression models were used to adjust of confounding variables (defined a priori as age > 65 or< 65, smoking, gender and presence or absence of body mass index > 30). Of note, SCT is likely under-estimated due to incomplete diagnosis. Rates or proportions were compared using Chi-squared testusing Medcalc software (2018). Logistic regression analysis was done using Statistical Package for Social Sciences (SPSS, version 21, IBM Corp, Armonk, NY). P< 0.05 was considered statistically significant. Results A total of 1360 major orthopedic surgeries in patients with SCT and 74040 surgeries in non-SCT patients were identified. 30 and 90-day VTE for SCT patients undergoing major orthopedic surgery was 9.7% each. 30 and 90 day VTE for non-SCT patients undergoing major hip and knee surgery were 5.9 % and 6.4 % respectively. The difference in 30-day and 90-day VTE rates between the SCT and non-SCT group was statistically significant (30 day VTE difference 3.1%, 95% CI 1.6650-4.7569, p < 0.001; 90 day VTE difference=3.6%; 95% CI 2.1658-5.2562, p= <0.001). The rates of anticoagulant dispensation (oral Xa inhibitors, enoxaparin or warfarin) after surgery were 56% and 46% in SCT and non-SCT group respectively (difference = 10%, 95% CI 7.32-12.64, p <0.001). Despite the higher proportion of patients prescribed for anticoagulants in the SCT population, there was still a higher 30 and 90-day VTE rate in that group. Compliance to anticoagulation and mortality from VTE could not be assessed in this study. Logistic regression of risk factors associated with risk of VTE revealed age over 65 years of age, female gender, active smoking status, obesity (BMI >30), and presence of sickle cell trait were all significantly associated with increased risk of both 30 and 90 day VTE post major orthopedic surgery. Please see Table 1 and 2 for further details. Conclusion Our study represents real life data outside of a clinical trial. We found that patients with SCT who underwent major hip and knee surgery had an increased 30 and 90-day VTE rates compared to non-SCT patients undergoing the same procedures. Overall, this cohort of AA patients had VTE rates higher than that were described in literature. Of note, AA patients overall are at a higher risk of VTE than are their Caucasian counterparts. The results from the study seem to suggest a role for extended prophylaxis in people with SCT who are undergoing orthopedic procedures, and warrants further study. Disclosures Little: Doris Duke Charitable Foundations: Research Funding; NHLBI: Research Funding; PCORI: Research Funding; Hemex: Patents & Royalties: Patent, no honoraria.

scholarly journals Implementation and Preliminary Effectiveness of mHealth Apps for Improving Sickle Cell Disease Care during COVID-19: A Mixed-Methods Evaluation

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3038-3038
Author(s):  
Sherif M. Badawy ◽  
Lisa DiMartino ◽  
Donald Brambilla ◽  
Ana Baumann ◽  
Ebony Burns ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Research Funding ◽  
Health Inequities ◽  
Cell Disease ◽  
Structured Interviews ◽  
Research Staff ◽  
Blood Disorder ◽  
The Impact ◽  
Mhealth Apps

Abstract Background: Sickle cell disease (SCD) is a chronic blood disorder, which disproportionately impacts Black individuals. Hydroxyurea therapy prevents SCD-related complications; yet it is underutilized, which contributes to further health inequities. Mobile health (mHealth) apps for patients and providers have the potential to improve hydroxyurea adherence. Our objective was to use the RE-AIM framework to evaluate the impact of the COVID-19 pandemic on implementation and effectiveness of patient and provider hydroxyurea (HU) mHealth apps (InCharge Health app and HU Toolbox app, respectively) in two large academic medical centers. Methods: Patient-level data (n=46) were collected between 11/2019-7/2020. Adherence was measured by calculating Percentage of Days Covered (PDC) from prescription records over 24 weeks before implementing the mHealth apps and during 12 and 24 weeks of implementation. As a response to the COVID-19 pandemic and to reduce virus spread, both sites temporarily suspended non-emergent clinical activities. During implementation, Site A clinics shut down for approximately 2 months (3/2020-5/2020) and Site B clinics for 7 months (3/2020-10/2020). To better understand contextual factors associated with mHealth implementation, we purposively sampled participants according to app use level (high vs low) and conducted semi-structured interviews with adult SCD patients, providers (physicians, nurse practitioners, and physician assistants), administrators, and research staff between 6/2020 and 3/2021. Results: A total of 46 patients participated and contributed to the PDC hydroxyurea adherence data. Mean change in PDC, adjusted for baseline PDC, was greater at Site A than at Site B (12-weeks: difference = 16.59%, p=.01, Figure 1A; 24-weeks: difference = 15.08%, p=.01, Figure 1B). Eleven patients (mean age 26.2 years old, 64% males, 100% Black, 73% HbSS, 45% low app users) and 11 providers (mean age 36.7 years old, 73% females, 36% Black, 54.5% physicians, average 8 years in practice, 36% low app users) completed the semi-structured interviews across the 2 sites. Site B was more affected during the COVID-19 pandemic where patients had difficulty obtaining hydroxyurea and other challenges related to reaching their providers and clinic setting for non-urgent or emergent reasons. In addition, participants with lower baseline hydroxyurea adherence level, as measured by PDC, had a more remarkable improvement in their PDC values at 12 weeks (Figure 1C) and 24 weeks (Figure 1D) Additional qualitative data focused on the implementation process were collected from 3 administrators, and 4 research staff. Among patients, both high and low app users reported the pandemic was a barrier to getting needed care (e.g., difficulty getting to hospital/clinic) and obtaining hydroxyurea; this was particularly a concern among low app users at Site B. Among providers, all but 2 high app users reported the pandemic did not impact app use, but nearly all low users perceived the pandemic to be a barrier to using the provider app because fewer patients came to clinic for maintenance SCD visits during the COVID-19 pandemic. Low users also reported the pandemic would negatively impact continued use of the app. Administrators and research staff also said reduced in-person clinic visits were a barrier to app implementation. Conclusions: mHealth apps are promising tools for improving hydroxyurea adherence among adolescents and adults with SCD. Contextual data show that some patients who experienced challenges accessing healthcare during COVID-19 pandemic have also experienced challenges navigating mHealth implementation. A focus on removing barriers to mobile apps use during care disruptions will likely improve patient and provider mHealth apps implementation, and ultimately, reduce health inequities for this vulnerable population. Our findings suggest that strategies such as including an mHealth facilitator has the potential to help addressing some of these implementation challenges. Note: Sherif Badawy and Lisa DiMartino are co-first authors with equal contribution Figure 1 Figure 1. Disclosures Badawy: Sanofi Genzyme: Consultancy; Bluebird Bio Inc: Consultancy; Vertex Pharmaceuticals Inc: Consultancy. Shah: Alexion: Speakers Bureau; Bluebird Bio: Consultancy; CSL Behring: Consultancy; Emmaus: Consultancy; GBT: Consultancy, Research Funding, Speakers Bureau; GLG: Consultancy; Guidepoint Global: Consultancy; Novartis: Research Funding, Speakers Bureau. Hankins: Bluebird Bio: Consultancy; UpToDate: Consultancy; Vindico Medical Education: Consultancy; Global Blood Therapeutics: Consultancy.

The Impact of the Child with Thalassemia On the Family: Parental Assessment by Child Health Questionnaire.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 1371-1371
Author(s):  
Robert Yamashita ◽  
Amy Sobota ◽  
Felicia Trachtenberg ◽  
Yan Xu ◽  
Zahra Pakbaz ◽  
...  
Keyword(s):  
Research Funding ◽  
Bodily Pain ◽  
Well Being ◽  
Research Network ◽  
Health Questionnaire ◽  
Parental Time ◽  
Significant Difference ◽  
The Family ◽  
The Us ◽  
The Impact

Abstract Abstract 1371 Poster Board I-393 The study examines the quality of life (QOL) of pediatric thalassemia patients and their families enrolled in the Thalassemia Longitudinal Cohort (TLC) study of the NHLBI-sponsored Thalassemia Clinical Research Network, which comprises 17 centers in the US, Canada, and London, UK. The study evaluates 99 baseline responses to the Children's Health Questionnaire (CHQ) PF28, a self-administered survey filled out by the parent or guardian of patients age 5 y and older. The CHQ utilizes 12 scales that can be grouped into parental assessments of the child's physical well-being; mental, emotional and behavioral health; and the familial context. We previously showed that compared to the US population, the thalassemia population is significantly different on 7 of the 12 scales. Here we evaluate these data by patient gender, race, age, and chelator type. The mean age of the population was 9.7 y (range 5.0-13.8 y), with 48% male, and 65% non-white. While parents assessed males to have a lower QOL than females, the only significant difference was in their assessment of the child's emotional role/behavior (school or friends). When comparing white to non-white (predominantly Asian) patients, with the exception of bodily pain, non-white thalassemia patients reported poorer PF28 scores. However, only the reported impact on parental time and emotion are significant. These variances appear to mirror the US population. Figure 1 shows PF28 summary scores by age in thalassemia compared to the general US population. Parents evaluate their child with thalassemia with lower physical health than US norms (p<0.0001), with an apparent decline in scores in adolescents (though p=0.10 for age effect). In contrast, psychosocial scores are close to US norms (p=0.81). Finally, when CHQ PF28 assessments are compared by chelator type (subcutaneous deferoxamine vs. oral deferasirox), parents report that children receiving deferoxamine have generally lower QOL than the rating for those receiving Deferasirox. However, the only significant differences are with perceived physical function, impact on family activities, and the overall physical summary scale. The CHQ PF28 data provides important insight into the impact the child with thalassemia has on the family. Although psychosocial QOL is similar, children, and especially adolescents, with thalassemia have lower physical QOL, especially those on chelation with Deferoxamine. Because PF28 takes the parents' point of view, it can't be determined from these data alone whether the reported difference between parenteral and oral chelator would hold true for direct patient assessments. The gender of the child also appears to affect parental expectation of the child's QOL. These data validate the observational evidence that a child with thalassemia has a significant impact on the family. Figure 1 TLC CHQ PF28 Summary Scales compared to US norms Figure 1. TLC CHQ PF28 Summary Scales compared to US norms Disclosures: Odame: Novartis: Consultancy, Speakers Bureau. Thompson: Novartis: Research Funding. Neufeld: Novartis: Research Funding.

Symptoms of Multiple Myeloma: Results of Hybrid Concept Elicitation/Cognitive Debriefing Interviews

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 5973-5973 ◽  
Author(s):  
Peter C. Trask ◽  
Bhumi Trivedi ◽  
Andrew Palsgrove ◽  
William Benton Jones ◽  
Colleen McHorney
Keyword(s):  
Multiple Myeloma ◽  
Research Funding ◽  
Plasma Cells ◽  
Qualitative Interviews ◽  
Cognitive Debriefing ◽  
Symptom Scale ◽  
Sleep Difficulty ◽  
Novel Treatments ◽  
Third Line ◽  
The Impact

Abstract Aims: Multiple myeloma (MM) is a malignant plasma cell disease that is characterized by clonal proliferation of plasma cells in the bone marrow and the production of excessive amounts of a monoclonal immunoglobulin (usually of the IgG or IgA type or free urinary light chain [paraprotein, M protein, or M-component]). Hallmark symptoms of MM include bone disease, which contributes to pain, and anemia, which manifests itself as tiredness and fatigue. Recently, we developed a new MM Symptom Scale based on a systematic literature review and previously conducted qualitative interviews with MM patients. The instrument presents a set of symptoms and evaluates their frequency and severity over the last seven days. In this study, we conducted one-on-one interviews with third-line plus relapsed or refractory (RR) MM patients to: (1) elicit and confirm key disease symptoms that patients describe in response to MM using open-ended questions; and (2) assess the clarity, understanding, and content validity of the newly-developed draft MM Symptom Scale. Methods: Third-line plus RR MM patients were recruited for one-on-one interviews in San Diego, CA and Fort Lauderdale, FL. All participants had to be adult, symptomatic RR MM patients who had previously been treated with an immunomodulary drug and a proteasome inhibitor. They also had to have evidence of disease progression or be currently on treatment to qualify for the study. The first half of the interview focused on open-ended questions regarding important symptoms that patients had experienced. In the second half of the interview, the MM Symptom Scale was introduced, the patient completed the scale in a think-aloud cognitive-debriefing interview, and each question was evaluated for clarity and understanding. Results: A total of nine patients participated in the hybrid interviews of whom four were female and five were male. The average age of the participants was 61.6 years. The average severity of MM symptoms across the nine participants was 6.2 on a scale of 1-10. The average report of the worst level of pain experienced in the last week was 5.2 on a scale of 1-10. The average report on the worst level of tiredness experienced in the last week was 6.2 on a scale of 1-10. The most frequently-reported symptoms were pain (specifically back pain) and tiredness (100% of participants). Other commonly-reported symptoms include swelling of the extremities, itching, feeling depressed, and diarrhea. During the cognitive-debriefing portion of the interview, most participants did not find difficulty in understanding any of the items. Some symptoms, however, were deemed not particularly relevant for the nine MM patients. For example, all of the nine MM patients believed that the symptoms “loss of bladder control” and “loss of bowel control” did not apply to them. These and other symptoms will be evaluated more closely for potential exclusion from the MM Symptom Scale. Some symptoms – such as neuropathy, sleep difficulty, constipation, and weight gain − were endorsed by at least three patients and will be considered for inclusion in the MM Symptom Scale. Conclusions: Health-related quality of life (HRQoL) in MM patients is characterized largely by the burden of pain and tiredness in addition to a few other key symptoms. These can be reliably and validly quantified using HRQoL instruments. The MM Symptom Scale is currently being refined to provide a standardized way through which clinician investigators can assess the impact of novel treatments on patients’ HRQoL. Disclosures Trask: Sanofi: Employment. Trivedi:Sanofi: Research Funding. Palsgrove:Sanofi: Research Funding. Jones:Sanofi: Employment. McHorney:Sanofi: Research Funding.

scholarly journals Impact of Oral Arginine Therapy on Global Arginine Bioavailability in Nigerian Children with Sickle Cell Anemia and Vaso-Occlusive Pain

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 22-23
Author(s):  
Richard Onalo ◽  
Peter Cooper ◽  
Barend C. Vorster ◽  
Marli Dercksen ◽  
Antoinette Cilliers ◽  
...  
Keyword(s):  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Opioid Use ◽  
Nigerian Children ◽  
Pain Scores ◽  
The Us ◽  
Plasma Arginine ◽  
The Impact ◽  
Arginine Concentration ◽  
Arginine Supplementation

Introduction: Severe vaso-occlusive pain episodes (VOE) are a major cause of morbidityand mortality in sickle cell anemia (SCA). Low arginine bioavailability is associated with pain severity and predicts need for pediatric hospitalization (Morris et. al, 2000). Arginine supplementation has opioid-sparing effects and was found to significantly decrease pain scores in children hospitalized with SCA-VOE compared to placebo in phase-2 randomized placebo-controlled trials (RCT) performed in the United States (US, Morris et. al, 2013) and Nigeria (Onalo et al, ASH 2019), while also significantly decreased time-to-crisis-resolution and length of hospital stay (LOS) in Nigerian children (Onalo et al, ASH 2019). Its mechanism-of-action and impact on arginine bioavailability is unclear. Objectives: To determine the impact of oral arginine supplementation on arginine bioavailability and total opioid use in children with SCA-VOE. Methods: A double-blind RCT of oral L-arginine (100 mg/kg/dose every 8 hours until discharge; up to 15 doses maximum) was performed in children with SCA hospitalized with severe VOE defined as a Numerical Pain Scale Score (PS) of at least 7 on a scale of 0-10, at one of two hospitals in Abuja, Nigeria (clinical outcomes previously reported, Onalo et al ASH 2019). Plasma arginine concentration and the global arginine bioavailability ratio (GABR, defined as arginine/[ornithine+citrulline]) was measured with high performance liquid chromatography tandem mass spectrometry before supplementation and at day 5 or discharge, whichever came first. Indices of arginine bioavailability were calculated and compared between the study groups. Demographics, clinical characteristics and total opioid use (mg/kg of morphine equivalents) were obtained. The impact of oral arginine supplementation on arginine bioavailability, and correlation of changes in arginine bioavailability with total opioid use was assessed. Results: Sixty-eight children with SCA were recruited, aged 5-17years (mean: 10.6±0.4 years), and 85% were male; 35 children were randomized into the arginine arm and 33 into the placebo arm. Baseline characteristics were similar between arms. Clinical outcomes of significantly lower total analgesic use, lower pain scores, decreased time-to-crisis resolution and shorter LOS in the arginine group vs. placebo were previously reported (Onalo et al, ASH 2019). Oral arginine supplementation increases plasma arginine levels by 125% [95% CI, 61-187%] in the arginine as against 29% [1-58%] in the placebo group (Table 1), p=0.007. GABR was higher after supplementation in patients treated with arginine: 59% [20-98%] vs. -2% [-27-22%] in the placebo group (p=0.009). Patients with the lowest arginine level at presentation experienced the greatest increase in plasma arginine concentration, particularly patients with acute chest syndrome (ACS). Percent increase in GABR inversely correlated with total opioid used (mg/kg; r=-0.35;p=0.02, figure 1). Conclusion: Arginine deficiency plays a role in acute pain requiring hospitalization in Nigerian children with SCA, similar to what has been reported in the US. Plasma arginine levels significantly increased with arginine supplementation, and improved global arginine bioavailability was inversely associated with total opioids used in VOE management. Lowest arginine levels were found in children with ACS, as previously reported in the US (Morris et al, 2000), a phenomenon that warrants further investigation. Low arginine bioavailability in children with SCA-VOE is improved by oral arginine supplementation. Funded by Tertiary Education Trust Fund (to RO) and in part by NIH/NCCIH K24AT009893 (to CRM); Pan African Clinical Trial Registry number PACTR 201611001864290). Disclosures No relevant conflicts of interest to declare. OffLabel Disclosure: Oral L-arginine for treatment of sickle cell anemia; it is a nutritional supplement

scholarly journals Comparison of Diabetic Macular Edema Treatment Patterns and Outcomes by European and US Retina-treating Ophthalmologists

2014 ◽  
Vol 07 (01) ◽  
pp. 63 ◽  
Author(s):  
Eric G John ◽  
James F Harris ◽  
◽  
Keyword(s):  
Macular Edema ◽  
Diabetic Macular Edema ◽  
Treatment Patterns ◽  
Self Report ◽  
Patient Treatment ◽  
Current Therapy ◽  
Visual Outcomes ◽  
Significant Difference ◽  
The Us ◽  
The Impact

AIM:To uncover and evaluate diabetic macular edema (DME) patient treatment patterns in the US and the largest EU countries.METHODS:Quantitative analyses of self-report data from retina-treating ophthalmologists via 30-minute Internet-based surveys. Data were collected across the US (one study) and EU (two studies— EU-1 and EU-2) between November 2011 and January 2013. Visual outcomes and rates of treatment success or failure were evaluated.RESULTS:Respondents were surveyed across the US (n=71) and EU (EU-1: n=180; EU-2: n=106). Nearly one-third of treated patients (US 38.6 %; EU-1 37.8 %; EU-2 30.7 %) reached a plateau in vision improvement. Geographical comparison showed no significant difference in responses between the US and the EU (EU-1, EU-2) (p>0.05).CONCLUSIONS:These data help to quantify the size of the chronic DME patient population in the US and EU, which is not achieving an acceptable functional outcome with current therapy. The paper provides solid empirical evidence regarding similarities in DME patient treatment and outcomes in the US and EU. The global and reproducible similarities in terms of physicians’ perceptions of the threshold of DME treatment burden and the visual outcomes when treatment benefit is limited are compelling. This is especially interesting within the present context where information regarding the effect of current treatments is limited. Diabetes is growing around the world at epidemic proportions, and these data provide insight into the impact DME may have on healthcare systems.

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