ArticleBone marrow transplantation2018
Allogenic peripheral stem cell transplantation from HLA-matched related donors for adult sickle cell disease: remarkable outcomes from a single-center trial.
Article in Bone marrow transplantation, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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Who cites it
11 citing papers in PubMed, 32 citations in OpenAlex.
- Review
- HLA-identical related hematopoietic stem cell transplantation in severe sickle cell disease: age is not a barrier to successful outcome.Bone marrow transplantation · 2022Article
- Article
- Cardiac pathophysiology in sickle cell disease.Journal of thrombosis and thrombolysis · 2021Review
- Excellent outcomes of allogeneic transplantation from peripheral blood of HLA-matched related donors for adult sickle cell disease with ATLG and posttransplant cyclophosphamide-containing regimen: an update work.Bone marrow transplantation · 2020Article
- Hematopoietic Cell Transplantation for Sickle Cell Disease.Frontiers in pediatrics · 2020Review
- Review
- Optimal disease management and health monitoring in adults with sickle cell disease.Hematology. American Society of Hematology. Education Program · 2019Review
- Allogeneic Hematopoietic Stem Cell Transplantation for Adults with Sickle Cell Disease.Journal of clinical medicine · 2019Review
- 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
- Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: A Multidimentional Review.Cell transplantationReview
Corrections and comments
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Authors and funding
12 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Adult patients with sickle cell disease (SCD) are highly susceptible to stem cell transplant complications, including drug toxicity, graft versus host disease (GVHD), and graft rejection due to SCD-related tissue damage, endothelial activation, and inflammation. The scarcity of compatible stem cells for transplantation further limits treatment options, with only 43 cases of adult allogeneic peripheral blood stem cell transplantation (allo-PSCT) from human leukocyte antigen (HLA)-identical sibling donors reported in the international registry for the period 1986-2013. Herein we report remarkable outcomes in a cohort of adult SCD patients who underwent allo-PSCT using a fludarabine (Flu), busulfan (Bu), and anti-T-cell lymphocyte globulin (ATG)-based conditioning regimen in combination with very low dose total body irradiation (TBI), followed by post-transplant cyclophosphamide (Cy) and sirolimus as GVHD prophylaxis. We performed a single-center, retrospective study consisting of 20 consecutive patients (mean age 33.4 years) who underwent allo-PSCT from HLA-matched related donors with a conditioning regimen of Flu 150/Bu 3.2/Cy 29/ATG 30 (Fresenius)/TBI 200 between September 2013 and September 2017. Data were validated by an independent data audit group of the affiliated JACIE-accredited transplantation center. All patients experienced a sustained donor cell engraftment. Full donor chimerism (total cell) occurred within 180 days in all patients. Mean duration of follow-up was 13.8 months (range: 0.3-50 months), with 12 (60%) patients completing 12 months. No non-relapse mortality or graft rejection occurred. Successful treatment was achieved without the presence of graft loss, grade III-IV acute GVHD, extensive chronic GVHD, or other major complications. Allo-PSCT in combination with Flu 150/Bu 3.2/Cy 29/ATG 30(Fresenius)/TBI 200- Cy/Sirolimus therapy yielded encouraging outcomes with no mortality and low incidence of GVHD. Further controlled studies will be necessary to compare transplant protocols and long-term outcomes.
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