Evidence map›Paper›PMID 40246805›Full record

ArticleQuality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation2025

Health-related quality of life and economic impacts in adults with transfusion-dependent β-thalassemia: findings from a prospective longitudinal real-world study.

Jennifer Drahos, Adriana Boateng-Kuffour, Melanie Calvert, Laurice Levine, Neelam Dongha, Nanxin Li, Zahra Pakbaz, Farrukh T Shah, Nick Ainsworth, Antony P Martin

Abstract read
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In one paragraph

Article in Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

The trial behind it

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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed.

  1. Article
  2. Review
  3. 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 · 2026
    Article
  4. Thalassaemia.Nature reviews. Disease primers · 2026
    Review
  5. Article
  6. Article
  7. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors.

Jennifer DrahosVertex Pharmaceuticals Incorporated, Boston, MA, USA. Jennifer_Drahos@vrtx.com.ORCID http://orcid.org/0000-0002-8454-9863
Adriana Boateng-KuffourVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Melanie CalvertCentre for Patient-Reported Outcomes Research, University of Birmingham, Birmingham, UK.ORCID http://orcid.org/0000-0002-1856-837X
Laurice LevineIndependent Consultant, CA, USA.
Neelam DonghaIndependent Consultant, London, UK.
Nanxin LiVertex Pharmaceuticals Incorporated, Boston, MA, USA.
Zahra PakbazDivision of Hematology Oncology, University of California Irvine School of Medicine, Orange, CA, USA.
Farrukh T ShahNHS Blood and Transplant, London, UK.
Nick AinsworthQC Medica, Liverpool, UK.ORCID http://orcid.org/0000-0003-4640-6666
Antony P MartinQC Medica, Liverpool, UK.ORCID http://orcid.org/0000-0003-4383-6038

Funding

Vertex Pharmaceuticals Vertex Pharmaceuticals
6 · The paper itself

Abstract

purposeTo characterize the health-related quality of life (HRQoL) and economic impacts of transfusion-dependent β-thalassemia (TDT).

methodsA prospective longitudinal real-world study was conducted in adults with TDT in the US and Europe (France, Germany, Italy, UK) over 6 months. Eligibility criteria included receiving ≥ 8 blood transfusions/year in each of the 2 years before enrollment. Study outcomes assessed at months 0, 3, and 6, included demographics, clinical characteristics, patient-reported outcome measures (EQ-VAS, FACT-G, FACIT-F, 11-point Numerical Rating Scale (NRS) of pain, TranQoL, and WPAI), and responses to bespoke questions on financial burden/distress.

resultsOverall, 155 adults with TDT participated (mean age [standard deviation (SD)]: 38.5 [10.8] years; 65.8% female). Most participants resided in the US (53.5%) or UK (22.6%). EQ-VAS, FACT-G, and FACIT-F mean (SD) scores at month 0 were lower among participants than the US general population (67.1 [20.4] versus 80.4 [15.6], 70.1 [19.0] versus 77.0 [16.1], and 27.9 [13.7] versus 43.6 [9.4], respectively; all p < 0.001). Mean (SD) TranQoL total score was 53.9 (18.5) and overall level of pain was "moderate" (NRS mean [SD]: 3.5 [2.8]). Part- or full-time employed participants missed a mean of 5.9 h of work in the preceding 7 days with out-of-pocket expenses being a moderate-to-major burden for 55% of participants. The findings were consistent over time.

conclusionsParticipants reported substantial humanistic and economic burdens associated with living with TDT. Our findings highlight the need for alternative treatment options that contribute to improvements in HRQoL and reduce economic burden in this patient population.

Indexed as

beta-ThalassemiaBlood TransfusionCost of IllnessQuality of LifeAdultEuropeFemaleHumansLongitudinal StudiesMaleMiddle AgedPatient Reported Outcome MeasuresProspective StudiesSurveys and QuestionnairesUnited StatesEconomic analysisHealth-related quality of lifeHumanistic impactPatient-reported outcomesTransfusion-dependent β-thalassemia

Identifiers

PMID40246805
PMCPMC12182489

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.