scholarly journals Evaluation of Longitudinal Pain Study in Sickle Cell Disease (ELIPSIS) by Electronic Patient-Reported Outcomes, Actigraphy, and Biomarkers

Blood ◽  
2020 ◽  
Author(s):  
Debra Pittman ◽  
Patrick C Hines ◽  
David Roger Beidler ◽  
Denis Rybin ◽  
Andrew L Frelinger ◽  
...  
Keyword(s):  
Health Care ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Health Care Utilization ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Care Utilization ◽  
Cell Disease ◽  
Patient Reported ◽  
At Home

Clinical trials in sickle cell disease (SCD) often focus on health care utilization for painful vaso-occlusive crises (VOC). However, no objective, quantifiable pain biomarkers exist, pain is not specific to VOCs, health care utilization varies between patients, unreported at-home VOCs likely contribute to long-term outcomes, and patient-reported outcomes are seldom considered. This non-interventional, longitudinal, 6-month study aimed to develop tools to identify VOCs in SCD patients with or without health care utilization. Participants wore an actigraph device, tracking sleep and activity. SCD patients used an electronic patient-reported outcome (ePRO) tool collecting pain, medication, fatigue, and daily function. Patients self-reported when they experienced VOC pain (VOC day). Biomarkers were collected every 3 weeks (non-VOC). Self-reported VOCs triggered at-home or in-hospital blood collection. The study enrolled 37 participants with SCD; 35 completed the study. Participants reported 114 VOC events and 346 VOC days, of which 62.3% and 78.3%, respectively, were self-treated at home. The ePRO and actigraphy captured endpoints of pain, functionality, fatigue, activity, and sleep; each was significantly altered on VOC days compared with non-VOC days. Biomarkers collected at home or in hospital on VOC days were significantly altered compared with non-VOC baseline values, including leukocyte-platelet aggregates, microfluidic-based blood cell adhesion, interleukin-6, C-reactive protein, interleukin-10, tumor necrosis factor-alpha, and thrombin-antithrombin. ELIPSIS: demonstrates the feasibility of accurately monitoring out-of-hospital pain, using patient-reported VOC days as potential endpoints for clinical trials in SCD; showed changes in biomarkers and activity measured by actigraphy that may enable improved identification and assessment of VOCs.

Quality Metrics and Health Care Utilization for Adult Patients with Sickle Cell Disease

2019 ◽  
Vol 111 (1) ◽  
pp. 54-61 ◽  
Author(s):  
Monica Ter-Minassian ◽  
Sophie Lanzkron ◽  
Alphonse Derus ◽  
Elizabeth Brown ◽  
Michael A. Horberg
Keyword(s):  
Health Care ◽  
Sickle Cell Disease ◽  
Health Care Utilization ◽  
Sickle Cell ◽  
Quality Metrics ◽  
Adult Patients ◽  
Care Utilization ◽  
Cell Disease

scholarly journals End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain

2019 ◽  
Vol 3 (23) ◽  
pp. 3982-4001 ◽  
Author(s):  
Ann T. Farrell ◽  
Julie Panepinto ◽  
C. Patrick Carroll ◽  
Deepika S. Darbari ◽  
Ankit A. Desai ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Cell Disease ◽  
End Points ◽  
Additional Clinical Trial ◽  
Patient Reported ◽  
The Brain

Abstract To address the global burden of sickle cell disease (SCD) and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to: patient-reported outcomes (PROs), pain (non-PROs), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the PROs, pain, and brain panels, as well as relevant findings and recommendations from the biomarkers panel. The panels identify end points, where there were supporting data, to use in clinical trials of SCD. In addition, the panels discuss where further research is needed to support the development and validation of additional clinical trial end points.

Occupational History For Adults With Sickle Cell Disease Compared With Healthy Siblings

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 2244-2244 ◽  
Author(s):  
Modupe Idowu ◽  
Omotayo Fawibe ◽  
Paul Rowan ◽  
Harinder S. Juneja ◽  
Deborah Brown
Keyword(s):  
Health Care ◽  
Sickle Cell Disease ◽  
Health Care Utilization ◽  
Sickle Cell ◽  
Negative Impact ◽  
Care Utilization ◽  
Job Retention ◽  
Cell Disease ◽  
Healthy Siblings ◽  
Health Related

Abstract Introduction Sickle cell disease (SCD) has a negative impact on academic and job functioning. Recurrent acute vaso-occlusive crises and organ dysfunction related to SCD can limit employment options and interfere with job retention. Potential associates of unemployment and poor job performance in adults with SCD include health-related (pain frequency and intensity, health-care utilization) and psychosocial (support system, coping mechanism). Moreover, poor academic achievement may increase the rates of unemployment in this patient group. The goal of this study is to compare patients’ rates of unemployment and disability with those of their unaffected siblings. Patients and Methods Twenty adult SCD patients (16 hemoglobin SS and 4 hemoglobin SC ; age: median = 29, range 20-56 years; sex: 8 males, 12 females) completed questionnaires relating to occupational experiences. Questionnaires were administered during routine clinic visits. Questions on the questionnaires relate to demographics, chronic pain scale, health-care utilization, use of hydroxyurea, employment status, length of employment, annual income, job satisfaction and use of tobacco, alcohol, or illicit drugs. These questionnaires also included questions relating to patients unaffected siblings who are within 5 years of age difference. We also performed paper and electronic chart review to obtain each patient’s health related data including number of days in the hospital for acute illnesses and for routine clinic appointments in the previous year, number of pain medications, other chronic medical conditions and SCD-related laboratory and tests results. Fourteen out of twenty patients (70%) were on hydroxyurea therapy and 14/20 (70%) had 3 or more hospitalizations in the previous one year. Patients on chronic transfusion therapy and those with other disabling conditions unrelated to SCD were excluded. Results Four out of twenty SCD patients (20%) are currently employed as compared to 15/20 (75%) of their healthy siblings (p < .001, all tests reported are Fisher’s Exact Test). Two of the SCD patients have been employed for more than 2 years as compared to 11 of their siblings (p = .006). Fourteen of SCD patients are on disability, while this is true for only one of the siblings (p < .001). Four of the twenty SCD patients are college graduates compared with 6 of the 20 unaffected siblings (p = .36). It is interesting to note that 12 out of 14 (86%) SCD patients who are currently on disability have previously had a job. Five of the fourteen (36%) SCD patients on disability reported that they missed more than 3 days per week of work due to their SCD. Of the unemployed patients, fourteen out of 16 (88%) wished they had a job. Conclusions This study highlights the negative impact of sickle cell disease on adult patients’ employment rates and job retention. Even though 16/20 (80%) of the SCD patients have previously had a job, only 20% are currently employed. Extension of this study will focus on development of specific interventions that may improve accommodation of SCD patients by encouraging collaboration between patients, families, medical providers, and employers. We believe that this is necessary in order to provide the most favorable environment for these patients to have better job functioning and it will also result in less economic burden on the society. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Outpatient Pain Predicts Subsequent One-Year Acute Health Care Utilization Among Adults With Sickle Cell Disease

2014 ◽  
Vol 48 (1) ◽  
pp. 65-74 ◽  
Author(s):  
Miriam O. Ezenwa ◽  
Robert E. Molokie ◽  
Zaijie Jim Wang ◽  
Yingwei Yao ◽  
Marie L. Suarez ◽  
...  
Keyword(s):  
Health Care ◽  
Sickle Cell Disease ◽  
Health Care Utilization ◽  
Sickle Cell ◽  
Care Utilization ◽  
Cell Disease ◽  
Acute Health Care ◽  
One Year

scholarly journals Opioid Prescription Filling Trends Among Children with Sickle Cell Disease After the Release of State-Issued Guidelines on Pain Management

Pain Medicine ◽  
2020 ◽  
Vol 21 (10) ◽  
pp. 2583-2592
Author(s):  
Susan E Creary ◽  
Deena J Chisolm ◽  
Sharon K Wrona ◽  
Jennifer N Cooper
Keyword(s):  
Health Care ◽  
Sickle Cell Disease ◽  
Health Care Utilization ◽  
Sickle Cell ◽  
Care Utilization ◽  
Cell Disease ◽  
Filling Rate ◽  
Opioid Prescription ◽  
Opioid Prescribing ◽  
Acute Health Care

Abstract Objective To assess the impact of Ohio’s 2012, 2013, and 2016 opioid prescribing guidelines on opioid and nonsteroidal anti-inflammatory drug (NSAID) prescription filling and health care utilization for pain among children with sickle cell disease (SCD). Design Quasi-experimental retrospective cohort study. Setting Ohio Medicaid claims data from August 2011 to August 2016. Subjects Medicaid beneficiaries under age 19 years with SCD. Methods Interrupted time series analyses comparing population-level rates of opioids and NSAID prescriptions filled, standardized amounts of opioids dispensed, and acute health care utilization for pain before and after release of each guideline. Results In our cohort of 1,505 children with SCD, there was a temporary but significant decrease in the opioid filling rate (–2.96 prescriptions per 100 children, P = 0.01) and in the amount of opioids dispensed (–31.39 milligram morphine equivalents per filled prescription, P &lt; 0.001) after the 2013 guideline but a temporary but significant increase in the opioid filling rate (7.44 prescriptions per 100 children, P &lt; 0.001) and in the amount of opioids dispensed (72.73 mg morphine equivalents per filled prescription, P &lt; 0.001) after the 2016 guideline. The NSAID filling rate did not significantly change after any of the guidelines. Acute health care utilization rates for pain after the 2016 guideline were similar to those before the 2013 guideline (rate ratio = 1.04, P = 0.63). Conclusions Our results suggest that Ohio’s 2013 and 2016 guidelines were associated with significant but nonsustained changes in opioid prescription filling among children with SCD. Additional studies are needed to confirm that opioid guidelines have a sustained impact on excessive opioid prescribing, filling, and misuse.

scholarly journals Clinical trial considerations in sickle cell disease: patient-reported outcomes, data elements, and the stakeholder engagement framework

Hematology ◽  
2021 ◽  
Vol 2021 (1) ◽  
pp. 196-205
Author(s):  
Sherif M. Badawy
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Stakeholder Engagement ◽  
Patient Reported Outcomes ◽  
Outcomes Assessment ◽  
Cell Disease ◽  
Patient Centered ◽  
Patient Reported

Abstract Patients with sickle cell disease (SCD) have significant impairment in their quality of life across the life span as a consequence of serious disease burden with several SCD-related complications. A number of disease-modifying therapies are currently available, yet long-term clinical benefits in real-world settings remain unclear. Over the past few years, a number of important initiatives have been launched to optimize clinical trials in SCD in different ways, including: (1) established panels through a partnership between the American Society of Hematology (ASH) and the US Food and Drug Administration; (2) the ASH Research Collaborative SCD Clinical Trials Network; (3) the PhenX Toolkit (consensus measures for Phenotypes and eXposures) in SCD; and (4) the Cure Sickle Cell Initiative, led by the National Heart, Lung, and Blood Institute. Electronic patient-reported outcomes assessment is highly recommended, and patient-reported outcomes (PROs) should be evaluated in all SCD trials and reported using Standard Protocol Items Recommendations for Interventional Trials guidelines. Patient-centered outcomes research (PCOR) approaches and meaningful stakeholder engagement throughout the process have the potential to optimize the execution and success of clinical trials in SCD with considerable financial value. This article reviews several clinical trial considerations in SCD related to study design and outcomes assessment as informed by recent initiatives as well as patient-centered research approaches and stakeholder engagement. A proposed hematology stakeholder-engagement framework for clinical trials is also discussed.

Pain, Coping and Health Care Utilization in Younger and Older Adults with Sickle Cell Disease

2010 ◽  
Vol 15 (1) ◽  
pp. 131-137 ◽  
Author(s):  
Kathryn A. Sanders ◽  
Susan M. Labott ◽  
Robert Molokie ◽  
Sarah R. Shelby ◽  
Joseph Desimone
Keyword(s):  
Older Adults ◽  
Health Care ◽  
Sickle Cell Disease ◽  
Health Care Utilization ◽  
Sickle Cell ◽  
Care Utilization ◽  
Pain Coping ◽  
Cell Disease

scholarly journals Sickle-Cell Disease Co-Management, Health Care Utilization, and Hydroxyurea Use

2020 ◽  
Vol 33 (1) ◽  
pp. 91-105 ◽  
Author(s):  
Nancy Crego ◽  
Christian Douglas ◽  
Emily Bonnabeau ◽  
Marian Earls ◽  
Kern Eason ◽  
...  
Keyword(s):  
Health Care ◽  
Sickle Cell Disease ◽  
Health Care Utilization ◽  
Sickle Cell ◽  
Care Utilization ◽  
Cell Disease
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