Clinical trial considerations in sickle cell disease: patient-reported outcomes, data elements, and the stakeholder engagement framework.
Hematology Am Soc Hematol Educ Program
; 2021(1): 196-205, 2021 12 10.
Article
in English
| MEDLINE | ID: covidwho-1666617
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.
Full text:
Available
Collection:
International databases
Database:
MEDLINE
Main subject:
Anemia, Sickle Cell
Type of study:
Experimental Studies
/
Prognostic study
/
Randomized controlled trials
Limits:
Humans
Country/Region as subject:
North America
Language:
English
Journal:
Hematology Am Soc Hematol Educ Program
Journal subject:
Hematology
Year:
2021
Document Type:
Article
Affiliation country:
Hematology.2021000252
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