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End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and 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.

SUBMITTER: Farrell AT

PROVIDER: S-EPMC6963237 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature

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

Farrell Ann T AT Panepinto Julie J Carroll C Patrick CP Darbari Deepika S DS Desai Ankit A AA King Allison A AA Adams Robert J RJ Barber Tabitha D TD Brandow Amanda M AM DeBaun Michael R MR Donahue Manus J MJ Gupta Kalpna K Hankins Jane S JS Kameka Michelle M Kirkham Fenella J FJ Luksenburg Harvey H Miller Shirley S Oneal Patricia Ann PA Rees David C DC Setse Rosanna R Sheehan Vivien A VA Strouse John J Stucky Cheryl L CL Werner Ellen M EM Wood John C JC Zempsky William T WT

Blood advances 20191201 23

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 ...[more]

PMID: 31809538

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Project description:Pediatric patients with sickle cell disease (SCD) experience a range of medical complications that result in significant morbidity and mortality. Recent advances in prophylactic and curative treatment approaches have highlighted the need for sensitive and clinically-meaningful trial endpoints. The detrimental effects of cognitive and psychological difficulties on social and economic mobility are well described. Although numerous reviews have assessed cognitive outcomes in other rare genetic disorders, SCD has not received the same focus. This review describes the cognitive (i.e., executive function and processing speed) and psychological domains (i.e., depression and anxiety) that are consistently associated with SCD pathology and, therefore, may be of particular interest as clinical trial endpoints. We then discuss corresponding well-validated and reliable cognitive tests and patient-reported outcomes (PROs) that may be appropriate for clinical trials given their robust psychometric properties, ease of administration, and previous use in the SCD population. Further, we provide a discussion of potential pitfalls and considerations to guide endpoint selection. In line with the move toward patient-centered medicine, we identify specific tests (e.g., NIH Toolbox Cognition Module, Wechsler Cancellation Test) and psychological PROs (e.g., PROMIS depression and anxiety scales) that are sensitive to SCD morbidity and have the potential to capture changes that are clinically meaningful in the context of patients' day to day lives. In particularly vulnerable cognitive domains, such as executive function, we highlight the advantages of composite over single-test scores within the context of trials. We also identify general (i.e., practice effects, disease heterogeneity) and SCD-specific considerations (i.e., genotype, treatment course, and disease course, including degree of neurologic, pain, and sleep morbidity) for trial measures. Executive function composites hold particular promise as trial endpoints that are clinically meaningful, amenable to change, relatively easy to collect, and can be incorporated into the routine care of patients with SCD in various settings and countries.

Dataset Information

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

Publications

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

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