Trial reportBone marrow transplantation2005
Hematopoietic stem cell transplantation for multiply transfused patients with sickle cell disease and thalassemia after low-dose total body irradiation, fludarabine, and rabbit anti-thymocyte globulin.
Trial report in Bone marrow transplantation, 2005. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00029393 (Induction of Stable Chimerism for Sickle Cell Anemia), which is not on this map. Cited by 47 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.
Induction of Stable Chimerism for Sickle Cell Anemia
Who cites it
47 citing papers in PubMed, 3 syntheses or guidelines pooled it, 120 citations in OpenAlex.
- Hematopoietic stem cell transplantation for people with sickle cell disease.The Cochrane database of systematic reviews · 2020Pooled it
- 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 for people with sickle cell disease.The Cochrane database of systematic reviews · 2016Pooled it
- A trial of unrelated donor marrow transplantation for children with severe sickle cell disease.Blood · 2016Trial
- Immune parameter analysis of children with sickle cell disease on hydroxycarbamide or chronic transfusion therapy.British journal of haematology · 2015Trial
- 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
- Comparing two different doses of anti-thymocyte globulin in allogeneic hematopoietic stem cell transplantation for transfusion-dependent thalassemia: an open-label, randomized clinical trial.Therapeutic advances in hematology · 2026Article
- Successful Second Hematopoietic Stem Cell Transplantation Using Total Body Irradiation-Based Conditioning for Children With Transfusion-Dependent Beta-Thalassemia.Journal of hematology · 2025Article
- Across the Myeloablative Spectrum: Hematopoietic Cell Transplant Conditioning Regimens for Pediatric Patients with Sickle Cell Disease.Journal of clinical medicine · 2022Review
- Transcranial Doppler sonography and the effect of haematopoietic stem cell transplantation in sickle cell disease.Neurological research and practice · 2022Article
- Development and characterization of a preclinical total marrow irradiation conditioning-based bone marrow transplant model for sickle cell disease.Frontiers in oncology · 2022Article
- Hematopoietic cell transplantation for sickle cell disease: updates and future directions.Hematology. American Society of Hematology. Education Program · 2021Article
- Long--Term Free Survival of Two Class III β-Thalassemic Patients after Non-Myeloablative Stem Cell Transplantation.International journal of hematology-oncology and stem cell research · 2020Article
- Hematopoietic Cell Transplantation for Sickle Cell Disease.Frontiers in pediatrics · 2020Review
- Review
- Stem cell transplantation in sickle cell disease: therapeutic potential and challenges faced.Expert review of hematology · 2018Review
- Curative therapies: Allogeneic hematopoietic cell transplantation from matched related donors using myeloablative, reduced intensity, and nonmyeloablative conditioning in sickle cell disease.Seminars in hematology · 2018Review
- Toward dual hematopoietic stem-cell transplantation and solid-organ transplantation for sickle-cell disease.Blood advances · 2018Review
- Allogeneic bone marrow transplant in the absence of cytoreductive conditioning rescues mice with β-thalassemia major.Blood advances · 2017Article
- Immunohaematological complications in patients with sickle cell disease after haemopoietic progenitor cell transplantation: a prospective, single-centre, observational study.The Lancet. Haematology · 2017Observational
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 (N = 3) and thalassemia (N = 1) with high-risk features received hematopoietic stem cell transplantations (HCT) to induce stable (full or partial) donor engraftment. Patients were 9-30 years of age. Fludarabine, rabbit anti-thymocyte globulin (ATG), and 200 cGy total body irradiation were administered pre-transplant. Patients received bone marrow (N = 3) or peripheral blood stem cells (N = 1) from HLA-identical siblings, followed by mycophenolate mofetil and cyclosporine for post-grafting immunosuppression. Significant lymphopenia, but only moderate neutropenia and thrombocytopenia developed post transplant. No grade IV nonhematological toxicities or acute graft-versus-host disease (GVHD) were observed. At 3 months after transplantation, three of four patients had evidence of donor myeloid chimerism (range, 15-100%). However, after post transplant immunosuppression was discontinued, graft rejection occurred in all but one patient. This patient is now doing well 27 months post transplant with full donor engraftment. One patient died after a second transplant, and another patient experienced a stroke as her graft was being rejected. These results suggest that stable donor engraftment after nonmyeloablative HCT is difficult to achieve among immunocompetent patients with hemoglobinopathies and that new approaches will need to be developed before wider application of this transplantation method for hemoglobinopathies.
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.