ArticleBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2011
Umbilical cord blood transplantation for children with thalassemia and sickle cell disease.
Article in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04523376 (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), which is not on this map. Cited by 68 papers, 3 of them syntheses 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.
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
68 citing papers in PubMed, 3 syntheses or guidelines pooled it, 208 citations in OpenAlex.
- American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation.Blood advances · 2021Guideline
- Hematopoietic stem cell transplantation for adult sickle cell disease in the era of universal donor availibility.Bone marrow transplantation · 2018Pooled it
- Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel.Haematologica · 2014Guideline
- Haploidentical Bone Marrow Transplantation for Sickle Cell Disease.NEJM evidence · 2025Trial
- Allogeneic stem cell transplantation with omidubicel in sickle cell disease.Blood advances · 2021Trial
- Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders.Blood advances · 2020Trial
- 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
- Changes in the incidence, patterns and outcomes of graft failure following hematopoietic stem cell transplantation for Hurler syndrome.Bone marrow transplantation · 2017Trial
- A trial of unrelated donor marrow transplantation for children with severe sickle cell disease.Blood · 2016Trial
- A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2014Trial
- Long-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2013Trial
- Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN).Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2012Trial
- Unrelated cord blood as a source of stem cells: cryopreservation damage can be alleviated by restoring mitochondrial function.Blood science (Baltimore, Md.) · 2025Article
- Article
- Beta Thalassemia in Children: Established Approaches, Old Issues, New Non-Curative Therapies, and Perspectives on Healing.Journal of clinical medicine · 2024Review
- The effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease: Systematic review and meta-analysis.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2024Review
- Review
- Thalassemia-free and graft-versus-host-free survival: outcomes of hematopoietic stem cell transplantation for thalassemia major, Turkish experience.Bone marrow transplantation · 2022Article
- Hurdles to the Adoption of Gene Therapy as a Curative Option for Transfusion-Dependent Thalassemia.Stem cells translational medicine · 2022Article
- Hematopoietic Cell Transplantation for Inborn Errors of Immunity Other than Severe Combined Immunodeficiency in Japan: Retrospective Analysis for 1985-2016.Journal of clinical immunology · 2022Article
8 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
20 authors at 11 institutions in 5 countries.
Funding
Abstract
We examined the efficacy of unrelated cord blood (CB) transplantation in children with thalassemia (n = 35) and sickle cell disease (n = 16), using data reported to 3 registries. Donor-recipient pairs were matched at HLA-A and -B (antigen level) and DRB1 (allele level) in 7 or HLA mismatched at 1 (n = 18), 2 (n = 25), or 3 loci (n = 1). Transplant conditioning was myeloablative (n = 39) or reduced intensity (n = 12). Neutrophil recovery with donor chimerism was documented in 24 patients; 11 patients developed grade II-IV acute graft-versus-host disease (aGVHD) and 10 patients, chronic GVHD (cGVHD). Overall survival (OS) and disease-free survival (DFS) were 62% and 21% for thalassemia and 94% and 50% for sickle cell disease (SCD), respectively. In multivariate analysis, engraftment rate (hazard ratio [HR] 2.2, P = .05) and DFS (HR 0.4, P = .01) were higher with cell dose >5 × 10(7)/kg. The 2-year probability of DFS was 45% in patients who received grafts with cell dose >5 × 10(7)/kg and 13% with lower cell dose. Primary graft failure was the predominant cause of treatment failure occurring in 20 patients with thalassemia and 7 patients with SCD. Primary graft failure was fatal in 5 patients with thalassemia. These results suggest that only CB units containing an expected infused cell dose >5 × 10(7)/kg should be considered for transplantation for hemoglobinopathy.
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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.