ArticleThe patient2021
Patient- and Caregiver-Reported Burden of Transfusion-Dependent β-Thalassemia Measured Using a Digital Application.
Article in The patient, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers, 1 of them a synthesis that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
22 citing papers in PubMed, 1 synthesis or guideline pooled it, 49 citations in OpenAlex.
- Health-related quality of life in hemoglobinopathies: A systematic review from a global perspective.Frontiers in pediatrics · 2022Pooled it
- Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel.Blood advances · 2025Trial
- Humanistic and economic burden among caregivers of adults and children with transfusion dependent β-thalassemia: a mixed-methods study.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2026Article
- Disease Burden in Transfusion-Dependent β-Thalassemia (TDT) Patients and Caregivers in Italy: Results of a Cross-Sectional Descriptive Online Survey.Drugs - real world outcomes · 2026Article
- Clinical Trial Landscape of Gene-Edited Autologous Hematopoietic Stem Cells for Hemoglobinopathies and Immunodeficiencies.International journal of molecular sciences · 2026Review
- Health-related quality of life and economic impacts in adults with transfusion-dependent β-thalassemia: findings from a prospective longitudinal real-world study.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2025Article
- [Delayed physical growth and related factors in pediatric patients with transfusion-dependent thalassemia].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Article
- Gene therapy in transfusion-dependent non-β0/β0 genotype β-thalassemia: first real-world experience of beti-cel.Blood advances · 2025Article
- Optical coherence tomography findings in beta-thalassemia major: a systematic review and Meta-analysis.International journal of ophthalmology · 2025Article
- Appropriateness of the EQ-5D-5L in capturing health-related quality of life in individuals with transfusion-dependent β-thalassemia: a mixed methods study.Health and quality of life outcomes · 2024Article
- Health-Related Quality-of-Life Impacts Associated with Transfusion-Dependent β-Thalassemia in the USA and UK: A Qualitative Assessment.The patient · 2024Article
- Polypharmacy and medication regimen complexity in transfusion-dependent thalassaemia patients: a cross- sectional study.International journal of clinical pharmacy · 2024Observational
- Systematic literature review of the indirect costs and humanistic burden of β-thalassemia.Therapeutic advances in hematology · 2024Review
- Economic burden of adult patients with β-thalassaemia major in mainland China.Orphanet journal of rare diseases · 2023Article
- Article
- Understanding the Association between Red Blood Cell Transfusion Utilization and Humanistic and Economic Burden in Patients with β-Thalassemia from the Patients' Perspective.Journal of clinical medicine · 2023Article
- Chronic Fatigue Syndrome in Patients with Deteriorated Iron Metabolism.Diagnostics (Basel, Switzerland) · 2022Review
- Predictors of health state utility values using SF-6D for Chinese adult patients with β-thalassemia major.Frontiers in public health · 2022Article
- Quality of Life and Burden of Disease in Italian Patients with Transfusion-Dependent Beta-Thalassemia.Journal of clinical medicine · 2021Article
- Concerns of Thalassemia Patients, Carriers, and their Caregivers in Malaysia: Text Mining Information Shared on Social Media.Healthcare informatics research · 2021Article
Corrections and comments
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Authors and funding
6 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
objectiveTransfusion-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.
methodsWe 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).
resultsEighty-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).
conclusionsThe patient- and caregiver-reported burden of TDT is high, influenced by disease-management time, fatigue, pain, and quality-of-life impairment.
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