Treating sickle cell anemia: A new era dawns

摘要

A single mutation in the p-hemoglobin gene allows deoxyHbS to polymerize and initiate the complex pathophysiology of sickle cell disease, a hemolytic anemia with chronic complications punctuated by acute events (Figure I). This commentary encompasses certain assumptions: opinions on how to use new therapeutics are many-data are limited; drugs preventing HbS polymerization are the most efficacious and should be started early; curative cell-based genetic therapy might soon be approved; untoward effects of each new modality will be acceptable; treatment algorithms proposed today will be modified tomorrow as new information accrues; only high-resourced countries can afford the regimens proposed.