@article{a98e97f5c7754b509939667e75760efe,
title = "A reanalysis of pain crises data from the pivotal L-glutamine in sickle cell disease trial",
abstract = "The pivotal Endari trial in sickle cell disease showed a reduction in pain crises events. This reanalysis of the L-glutamine phase 3 trial using annual rates of pain crises, consistent with other SCD studies, supported the statistically significant outcomes of the original analysis. The observed 45% difference in the VOC rate is comparable to what was reported in other sickle cell therapeutics used to reduce the incidence of pain. The results presented in this communication are informative for clinicians evaluating treatment effects across available SCD therapeutic options based on studies that utilized VOC as the primary endpoint.",
keywords = "Annual crisis rate, Endari, L-glutamine, Pain crises, Sickle cell disease",
author = "Zaidi, {Ahmar U.} and Jeremie Estepp and Nirmish Shah and Salam Alkindi and Hatoon Ezzat and Hung Lam and Minniti, {Caterina P.}",
note = "Funding Information: JHE receives research support from Pfizer, Global Blood Therapeutics, Forma Therapeutics, and Eli Lilly and Company and serves as a consultant for Daiichi Sankyo, Forma Therapeutics, Esperion, Emmaus Medical, Inc. and Global Blood Therapeutics. CM is a consultant for Novartis, Global Blood Therapeutics, Roche, Forma Therapeutics, bluebird bio (end point adjudicator), Emmaus Medical, Inc. CSL Behring (end point adjudicator), Chiesi Farmaceutici, and Novo Nordisk. NS receives research support from Novartis and Global Blood Therpeutics; is consultant for Global Blood Therapeutics, Novartis, Emmaus Medical, Inc. and bluebird bio; and is a speaker for Global Blood Therapeutics, Novartis, and Alexion. SAK receives Honoria from Novartis, Emmaus Medical, Inc. and AstraZeneca, participated in clinical trials with Novartis and Global Blood Therapeutics, and serves as a consultant for Novartis, Global Blood Therapeutics, Emmaus Medical, Inc, and Biologix. HL is an employee of MMS, a CRO consultant to Emmaus Medical, Inc. AUZ is serves as a consultant for Global Blood Therapeutics, Novartis, Emmaus Medical, Inc. Cyclerion, bluebird bio, Chiesi Farmaceutici, Novo Nordisk; receives research support from Emmaus Medical, Inc. Global Blood Therapeutics and Novartis; and is on the speaker bureau for Global Blood Therapeutics. Publisher Copyright: {\textcopyright} 2021 The Authors",
year = "2021",
month = nov,
doi = "10.1016/j.cct.2021.106546",
language = "English",
volume = "110",
journal = "Contemporary Clinical Trials",
issn = "1551-7144",
publisher = "Elsevier Inc.",
}