Gene therapy in sickle cell disease: Attitudes and informational needs of patients and caregivers.

ABSTRACT:

Background

Sickle cell disease (SCD) is an inherited blood disorder that results in serious morbidity and early mortality. Novel therapies for SCD, most notably genetic therapies (GTs) and HLA-mismatched donor hematopoietic cell transplantation, are in clinical trials. While potentially curative, these interventions are some of the most intensive treatments for SCD and are associated with serious and life-altering side effects, which may manifest several years after treatment. Little is known about knowledge, beliefs, and attitudes of individuals with SCD, or their caregivers, toward existing and these emerging therapies.

Methods

Patients with SCD at least 13 years of age (n = 66) and caregivers (n = 38) were surveyed about knowledge, attitudes, and beliefs surrounding treat

SUBMITTER: Sharma A 

PROVIDER: S-EPMC10187715 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature