Farrell AT, Panepinto J, Carroll CP, Darbari DS, Desai AA, King AA, Adams RJ, Barber TD, Brandow AM, DeBaun MR, Donahue MJ, Gupta K, Hankins JS, Kameka M, Kirkham FJ, Luksenburg H, Miller S, Oneal PA, Rees DC, Setse R, Sheehan VA, Strouse J, Stucky CL, Werner EM, Wood JC, Zempsky WT. End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain. Blood advances. 2019 Dec 10;3(3). 3982-4001. PMID: 31809538 [PubMed] PMCID: PMC6963237
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.