SynthesisBlood advances2021
A systematic review of quality of life in sickle cell disease and thalassemia after stem cell transplant or gene therapy.
Synthesis in Blood advances, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers, 1 of them a synthesis that pooled it.
What it found
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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.
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.
Who cites it
35 citing papers in PubMed, 1 synthesis or guideline pooled it, 72 citations in OpenAlex.
- Adherence to Iron Chelation Therapy among Adults with Thalassemia: A Systematic Review.Hemoglobin · 2022Pooled it
- Incremental eligibility criteria for the BMT CTN 1507 haploidentical trial for children with sickle cell disease.Blood advances · 2024Trial
- Significant improvement of child physical and emotional functioning after familial haploidentical stem cell transplant.Bone marrow transplantation · 2022Trial
- Informed shared decision-making or rigid eligibility and exclusion criteria for gene therapy for sickle cell disease?Blood advances · 2026Review
- Experiences of family building counseling and perceptions of reproductive technology among adults with sickle cell disease.PloS one · 2026Article
- Article
- Emergency care for children with sickle cell disease: a focus on pain and fever.Expert review of hematology · 2025Review
- Gut microbiota and metabolites drive chronic sickle cell disease pain in mice.Cell host & microbe · 2025Article
- A road map for uniform, comprehensive long-term follow-up after curative therapy for sickle cell disease.Blood advances · 2025Review
- Article
- The transplant debate: defining innovation for sickle cell.Blood advances · 2025Article
- Cardiac injury caused by iron overload in thalassemia.Frontiers in pediatrics · 2025Review
- Longitudinal outcomes of chronically transfused adults with sickle cell disease and a history of childhood stroke.Transfusion · 2024Article
- Knowledge, attitude and acceptance regarding bone marrow transplantation in caregivers of beta-thalassemia major patients.Journal of community genetics · 2024Article
- Assessing Psychosocial Risk and Resilience to Support Readiness for Gene Therapy in Sickle Cell Disease: A Consensus Statement.JAMA network open · 2024Article
- Longitudinal neurocognitive effects of nonmyeloablative hematopoietic stem cell transplant among older adolescents and adults with sickle cell disease: A description and comparison with sibling donors.Neuropsychological rehabilitation · 2024Article
- Health-related quality of life with standard and curative therapies in thalassemia: A narrative literature review.Annals of the New York Academy of Sciences · 2024Review
- Health-Related Quality-of-Life Profile of Pediatric Patients with β Thalassemia after Hematopoietic Stem Cell Transplantation.Journal of clinical medicine · 2023Article
- Sense of coherence or self-efficacy as predictors of health-related quality of life in sickle cell disease patients.Annals of hematology · 2023Article
- Multimodal phenotyping and correlates of pain following hematopoietic cell transplant in children with sickle cell disease.Pediatric blood & cancer · 2023Observational
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 2 institutions in 1 country.
Funding
Abstract
Patients with sickle cell disease (SCD) and thalassemia experience several complications across their lifespan that lead to impairment in different health-related quality of life (HRQOL) domains. There is increasing interest in curative therapies for patients with SCD and thalassemia, including hematopoietic stem cell transplant (HSCT) and gene therapy; however, the effect of these therapies on various HRQOL domains remains unclear. Our objective was to systematically evaluate the most recent evidence for the effect of HSCT and gene therapy on HRQOL in patients with SCD and thalassemia. A systematic search of medical literature databases was conducted. A total of 16 studies (thalassemia, n = 9; SCD, n = 6; both, n = 1) involving 517 participants met inclusion criteria (thalassemia, n = 416; SCD, n = 101). HSCT was associated with a small to large positive effects in most HRQOL domains (Cohen's d; mean = 0.47; median = 0.37; range, 0.27-2.05). In thalassemia, HSCT was frequently associated with large positive effects in physical and emotional HRQOL domains (median d = 0.79 and d = 0.57, respectively). In SCD, HSCT was associated with large positive effects in all HRQOL domains. Emerging data suggest improvement in HRQOL outcomes across different domains following gene therapy in thalassemia and SCD. The quality of evidence was moderate in 13 studies (81%). HSCT has a positive impact on several HRQOL domains in patients with SCD and thalassemia; however, more longitudinal studies are warranted to assess the sustainability of these effects. Reporting HRQOL outcomes from ongoing gene therapy or gene-editing trials in SCD and thalassemia is key to better understand the benefits of such therapies.
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Registered trials
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.