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

Clark Paramore, Laurice Levine, Emma Bagshaw (), Chengyu Ouyang, Amber Kudlac and Mark Larkin
Additional contact information
Clark Paramore: bluebird bio
Laurice Levine: Self-Employed Medical Consultant
Emma Bagshaw: Vitaccess Ltd
Chengyu Ouyang: Vitaccess Ltd
Amber Kudlac: Vitaccess Ltd
Mark Larkin: Vitaccess Ltd

The Patient: Patient-Centered Outcomes Research, 2021, vol. 14, issue 2, No 4, 197-208

Abstract: 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 disease-management surveys, the Brief Fatigue Inventory (BFI), the Transfusion-dependent Quality of life questionnaire (TranQol), and the Brief Pain Inventory Short Form (BPI-SF). Results Eighty-five individuals participated. Mean BFI and TranQol scores on enrollment were 5.0 (0–10 scale; 10 = worst symptoms) and 51 (0–100 scale; 100 = best quality of life), respectively. Mean transfusion frequency was every 3.2 weeks. Mean time spent on TDT management was 592 min on transfusion days and 91 min on non-transfusion days (11 h per week). Mean BFI and BPI-SF “worst fatigue” and “worst pain” scores were higher in the 5 days pre-transfusion than in the 5 days post-transfusion (fatigue 5.05 vs 4.29; pain 4.33 vs 3.85; 0–10 scale; 10 = worst symptoms). Conclusions The patient- and caregiver-reported burden of TDT is high, influenced by disease-management time, fatigue, pain, and quality-of-life impairment.

Date: 2021
References: View complete reference list from CitEc
Citations:

Downloads: (external link)
http://link.springer.com/10.1007/s40271-020-00473-0 Abstract (text/html)
Access to the full text of the articles in this series is restricted.

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:spr:patien:v:14:y:2021:i:2:d:10.1007_s40271-020-00473-0

Ordering information: This journal article can be ordered from
http://www.springer.com/economics/journal/40271

DOI: 10.1007/s40271-020-00473-0

Access Statistics for this article

The Patient: Patient-Centered Outcomes Research is currently edited by Christopher I. Carswell

More articles in The Patient: Patient-Centered Outcomes Research from Springer, International Academy of Health Preference Research
Bibliographic data for series maintained by Sonal Shukla () and Springer Nature Abstracting and Indexing ().