Patient- and Caregiver-Reported Burden of Transfusion-Dependent β-Thalassemia Measured Using a Digital Application.

ABSTRACT:

Background and objective

Transfusion-dependent β-thalassemia (TDT) is a rare genetic disease characterized by a deficiency of functional β-globin, ultimately leading to lifelong dependence on blood transfusions. There is little patient- and caregiver-reported data with which to understand the holistic and societal impact of TDT. The objective of this study was to evaluate the patient- and caregiver-reported disease-management, symptom, and quality-of-life burden of TDT.

Methods

We conducted a prospective, observational, real-world study of adults with TDT and caregivers of adolescents with TDT, in Italy, the UK, and the USA. Over 90 days, participants used a smartphone application to respond to surveys about their or their dependent's TDT, including bespoke background and di

SUBMITTER: Paramore C 

PROVIDER: S-EPMC7884594 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature