Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial.

doi:10.1016/S2352-3026(21)00059-4

Article

Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial.

The Lancet. Haematology - 1 May 2021

Howard Jo, Ataga Kenneth I, Brown Robert C, Achebe Maureen, Nduba Videlis, El-Beshlawy Amal, Hassab Hoda, Agodoa Irene, Tonda Margaret, Gray Sarah, Lehrer-Graiwer Joshua, Vichinsky Elliott

Abstract excerpt

BACKGROUND: For decades, patients with sickle cell disease have had only a limited number of therapies available. In 2019, voxelotor (1500 mg), an oral once-daily sickle haemoglobin polymerisation inhibitor, was approved in the USA for the treatment of sickle cell disease in patients aged 12 years and older on the basis of HOPE trial data. To further describe the applicability of voxelotor as a treatment for this...

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Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial.

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