A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study of Mitapivat in Patients with Sickle Cell Disease: RISE UP Phase 2 Results

Modupe Idowu, Lucas Otieno, Bogdan Dumitriu, Clarisse LC Lobo,Swee Lay Thein,Biree Andemariam,Obiageli E Nnodu,Adlette Inati,Alexander K Glaros,Sara Teresinha Olalla Saad,Pablo Bartolucci,Raffaella Colombatti,Ali T Taher,Miguel R Abboud,Abdulafeez Oluyadi,Varsha Iyer,Ophelia Yin, Susan Morris, Amber M Yates, Hui Shao

Blood（2023）

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摘要

Background: Sickle cell disease (SCD) is characterized by the presence of hemoglobin S (HbS), a structural variant caused by a mutation in the HBB gene. Deoxygenated HbS molecules polymerize into “fibers”, causing red blood cells (RBCs) to sickle and hemolyze. Sickled RBCs have a shortened lifespan and impede blood flow to tissues, causing painful vaso-occlusive crises. Recurrent microvascular damage and chronic hemolytic anemia result in progressive multi-organ damage.

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