A road map for uniform, comprehensive long-term follow-up after curative therapy for sickle cell disease.

ABSTRACT:

Abstract

An increasing number of allogeneic transplant and autologous gene modification transplant therapies seek to eradicate sickle hemoglobin and the consequent hemolysis, vasculopathy, functional compromise, morbidity, and mortality. Because these modalities are used in parallel, it is important to be able to define the spectrum and stability of correction, long-term effects, and the pros and cons of each modality. A comparison between interventions that will be sought by providers and patients undergoing intervention requires uniform assessments that evaluate disease- and intervention-related effects for informed decision-making. This expert summary outlines a pathway to functional evaluations with timing recommendations, provides broad management guidelines, and touches upon

SUBMITTER: Shenoy S 

PROVIDER: S-EPMC12432986 | biostudies-literature | 2025 Jun

REPOSITORIES: biostudies-literature