Trial reportBlood2016
A trial of unrelated donor marrow transplantation for children with severe sickle cell disease.
Trial report in Blood, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 91 papers, 3 of them syntheses that pooled 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.
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.
Unrelated Donor Reduced Intensity Bone Marrow Transplant for Children With Severe Sickle Cell Disease (BMT CTN #0601)
Closely Matched Unrelated Donor Peripheral Blood Stem Cell Transplantation With TCRαβ+ T Cell and B Cell Depletion For Patients With Sickle Cell Disease and Thalassemia Major
Who cites it
91 citing papers in PubMed, 3 syntheses or guidelines pooled it, 207 citations in OpenAlex.
- American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation.Blood advances · 2021Guideline
- Hematopoietic stem cell transplantation for people with sickle cell disease.The Cochrane database of systematic reviews · 2020Pooled it
- Analysis of Risk Factors Associated With Poor Outcome in Posterior Reversible Encephalopathy Syndrome After Treatment in Children: Systematic Review and Meta-Analysis.Frontiers in neurology · 2020Pooled it
- Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial.Nature medicine · 2025Trial
- Hematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease.Blood advances · 2025Trial
- Haploidentical Bone Marrow Transplantation for Sickle Cell Disease.NEJM evidence · 2025Trial
- Significant improvement of child physical and emotional functioning after familial haploidentical stem cell transplant.Bone marrow transplantation · 2022Trial
- Post-Transcriptional Genetic Silencing ofThe New England journal of medicine · 2021Trial
- Familial Haploidentical Stem Cell Transplant in Children and Adolescents With High-Risk Sickle Cell Disease: A Phase 2 Clinical Trial.JAMA pediatrics · 2020Trial
- Functional and Radiologic Assessment of the Brain after Reduced-Intensity Unrelated Donor Transplantation for Severe Sickle Cell Disease: Blood and Marrow Transplant Clinical Trials Network Study 0601.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2019Trial
- Effect of increased dose of total body irradiation on graft failure associated with HLA-haploidentical transplantation in patients with severe haemoglobinopathies: a prospective clinical trial.The Lancet. Haematology · 2019Trial
- Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study.American journal of hematology · 2019Trial
- Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2018Trial
- Haploidentical transplantation in sickle cell disease: toward donor availability for all patients.Haematologica · 2026Article
- Hematopoietic Cell Transplantation for Sickle Cell Disease.Children (Basel, Switzerland) · 2026Review
- Exploring Affordable Curative Therapy for Sickle Cell Disease in Africa: A Comprehensive Overview.American journal of hematology · 2026Review
- Curative Approach to the Treatment of Beta-Thalassemia and Sickle Cell Disease with Hematopoietic Stem Cell Transplantation.Journal of clinical medicine · 2026Review
- Article
- Abatacept for acute graft versus host disease prophylaxis in children with sickle cell disease patients undergoing HLA identical sibling hematopoietic stem cell transplantation.Bone marrow transplantation · 2025Article
- A road map for uniform, comprehensive long-term follow-up after curative therapy for sickle cell disease.Blood advances · 2025Review
31 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
29 authors at 20 institutions in 1 country.
Funding
Abstract
Children with sickle cell disease experience organ damage, impaired quality of life, and premature mortality. Allogeneic bone marrow transplant from an HLA-matched sibling can halt disease progression but is limited by donor availability. A Blood and Marrow Transplant Clinical Trials Network (BMT CTN) phase 2 trial conducted from 2008 to 2014 enrolled 30 children aged 4 to 19 years; 29 were eligible for evaluation. The primary objective was 1-year event-free survival (EFS) after HLA allele-matched (at HLA-A, -B, -C, and -DRB1 loci) unrelated donor transplant. The conditioning regimen included alemtuzumab, fludarabine, and melphalan. Graft-versus-host disease (GVHD) prophylaxis included calcineurin inhibitor, short-course methotrexate, and methylprednisolone. Transplant indications included stroke (n = 12), transcranial Doppler velocity >200 cm/s (n = 2), ≥3 vaso-occlusive pain crises per year (n = 12), or ≥2 acute chest syndrome episodes (n = 4) in the 2 years preceding enrollment. Median follow-up was 26 months (range, 12-62 months); graft rejection was 10%. The 1- and 2-year EFS rates were 76% and 69%, respectively. The corresponding rates for overall survival were 86% and 79%. The day 100 incidence rate of grade II-IV acute GVHD was 28%, and the 1-year incidence rate of chronic GVHD was 62%; 38% classified as extensive. There were 7 GVHD-related deaths. A 34% incidence of posterior reversible encephalopathy syndrome was noted in the first 6 months. Although the 1-year EFS met the prespecified target of ≥75%, this regimen cannot be considered sufficiently safe for widespread adoption without modifications to achieve more effective GVHD prophylaxis. The BMT CTN #0601 trial was registered at www.clinicaltrials.gov as #NCT00745420.
Indexed as
Identifiers
What OpenQuestion holds
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.