ArticleMediterranean journal of hematology and infectious diseases2017
Treosulfan-Based Conditioning Regimen in Sibling and Alternative Donor Hematopoietic Stem Cell Transplantation for Children with Sickle Cell Disease.
Article in Mediterranean journal of hematology and infectious diseases, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 1 of them a synthesis that pooled it.
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Who cites it
14 citing papers in PubMed, 1 synthesis or guideline pooled it, 35 citations in OpenAlex.
- Effect of allogeneic hematopoietic stem cell transplantation on sickle cell disease-related organ complications: A systematic review and meta-analysis.American journal of hematology · 2024Pooled it
- Hematopoietic Stem Cell Transplantation in Severe Pediatric Sickle Cell Disease: Outcome and long-term complications, Saudi experience at King Faisal Specialist Hospital, Riyadh, Saudi Arabia.Mediterranean journal of hematology and infectious diseases · 2025Article
- Clinical Outcomes Among Patients With Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia Treated With Allogeneic Hematopoietic Stem Cell Transplantation: A Systematic Literature Review.Journal of blood medicine · 2025Review
- Late effects of hemopoietic stem cell transplant for sickle cell disease: monitoring and management.Expert review of hematology · 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
- Cerebral hemodynamic changes after haploidentical hematopoietic stem cell transplant in adults with sickle cell disease.Blood advances · 2024Article
- Across the Myeloablative Spectrum: Hematopoietic Cell Transplant Conditioning Regimens for Pediatric Patients with Sickle Cell Disease.Journal of clinical medicine · 2022Review
- Fertility preservation for pediatric patients with hemoglobinopathies: Multidisciplinary counseling needed to optimize outcomes.Frontiers in endocrinology · 2022Review
- Stem cell therapies for autoimmune hepatitis.Stem cell research & therapy · 2021Review
- Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies.Bone marrow transplantation · 2020Article
- Choice of Donor Source and Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Sickle Cell Disease.Journal of clinical medicine · 2019Review
- HLA-haploidentical hematopoietic stem cell transplantation in pediatric patients with hemoglobinopathies: current practice and new approaches.Bone marrow transplantation · 2019Review
- Haploidentical stem cell transplantation cures autoimmune hepatitis and cerebrovascular disease in a patient with sickle cell disease.Bone marrow transplantation · 2018Article
- Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: A Multidimentional Review.Cell transplantationReview
Corrections and comments
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Authors and funding
20 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
objectivesLack of suitable donors and regimen related toxicity are major barriers for hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD). The aim of the study is the assessment of efficacy and toxicity of Treosulfan-based conditioning regimen for SCD also when alternative donors such as mismatched unrelated donor and haploidentical donor are employed.
methodsWe report our single-center experience: 11 patients with SCD received HSCT with a Treosulfan/Thiotepa/Fludarabine/Anti-thymoglobulin conditioning regimen between 2010 and 2015. The donor was a matched sibling donor (n= 7), a haploidentical parent (n= 2), a matched unrelated donor (n= 1) or a mismatched unrelated donor (n=1). The haploidentical and mismatched unrelated donor grafts were manipulated by removing TCRαβ and CD19 positive cells.
resultsAll patients survived the procedure and achieved stable engraftment. Stable mixed chimerism was observed in 5/11 patients. Grade III-IV regimen related toxicity was limited to mucositis and no grade III-IV graft-versus-host disease (GvHD) occurred. No SCD manifestation was observed post transplant and cerebral vasculopathy improved in 3/5 evaluable patients. Organ function evaluation showed no pulmonary, cardiac or renal toxicity but gonadal failure occurred in 1/4 evaluable patients.
conclusionOur data suggest that Treosulfan is associated with low toxicity and may be employed also for unrelated and haploidentical donor HSCT.
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