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End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings

2019; Elsevier BV; Volume: 3; Issue: 23 Linguagem: Inglês

10.1182/bloodadvances.2019000883

2473-9537

Ann T. Farrell, Julie A. Panepinto, Ankit A. Desai, Adetola A. Kassim, Jeffrey D. Lebensburger, Mark C. Walters, Daniel E. Bauer, Rae Blaylark, Donna DiMichele, Mark T. Gladwin, Nancy Green, Kathryn L. Hassell, Gregory J. Kato, Elizabeth S. Klings, Donald B. Kohn, Lakshmanan Krishnamurti, Jane A. Little, Julie Makani, Punam Malik, Patrick T. McGann, Caterina P. Minniti, Claudia R. Morris, Isaac Odame, Patricia O’Neal, Rosanna Setse, Poornima Sharma, Shalini Shenoy,

Myeloproliferative Neoplasms: Diagnosis and Treatment

To address the global burden of sickle cell disease 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 outcome, pain (non-patient-reported outcomes), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the end-organ considerations, measurement of cure, and low-resource settings panels as well as relevant findings and recommendations from the biomarkers panel.