Trial reportBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2007
HLA-matched related donor hematopoietic cell transplantation in 43 patients with Fanconi anemia conditioned with 60 mg/kg of cyclophosphamide.
Trial report in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
What it found
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The trial behind it
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Who cites it
18 citing papers in PubMed, 73 citations in OpenAlex.
- Trial
- Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2013Trial
- Treatment of Fanconi anemia patients using fludarabine and low-dose TBI, followed by unrelated donor hematopoietic cell transplantation.Bone marrow transplantation · 2011Trial
- Medulloblastoma in children with Fanconi anemia: Association with FA-D1/FA-N, SHH type and poor survival independent of treatment strategies.Neuro-oncology · 2024Article
- Fanconi anemia phenotypic and transplant outcomes' associations in Iranian patients.Health science reports · 2023Article
- Two decades of stem cell transplantation in patients with Fanconi anemia: Analysis of factors affecting transplant outcomes.Clinical transplantation · 2023Article
- Kidney complications in 107 Fanconi anemia patients submitted to hematopoietic cell transplantation.European journal of pediatrics · 2022Article
- Special pre- and posttransplant considerations in inherited bone marrow failure and hematopoietic malignancy predisposition syndromes.Hematology. American Society of Hematology. Education Program · 2020Review
- Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia.Bone marrow transplantation · 2017Article
- Hematopoietic cell transplantation in Fanconi anemia: current evidence, challenges and recommendations.Expert review of hematology · 2017Review
- Transplantation for bone marrow failure: current issues.Hematology. American Society of Hematology. Education Program · 2016Review
- Second Allogeneic Hematopoietic Cell Transplantation for Patients with Fanconi Anemia and Bone Marrow Failure.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2015Article
- Hematopoietic stem cell transplantation for primary immunodeficiencies.Hematology/oncology clinics of North America · 2014Review
- Cyclophosphamide-based in vivo T-cell depletion for HLA-haploidentical transplantation in Fanconi anemia.Pediatric hematology and oncology · 2012Article
- Fanconi anemia - learning from children.Pediatric reports · 2011Article
- Diagnosis of Fanconi anemia in patients with bone marrow failure.Haematologica · 2009Article
- HLA-matched sibling hematopoietic stem cell transplantation for fanconi anemia: comparison of irradiation and nonirradiation containing conditioning regimens.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2008Article
- Fanconi Anemia patient with AML1 gene amplification and monosomy 7 in pre-transplant myelodysplasia (MDS) relapsing 7 years after successful allo-SCT.Bone marrow transplantation · 2008Article
Corrections and comments
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Authors and funding
12 authors at 3 institutions in 2 countries.
Funding
Abstract
Cells from Fanconi anemia (FA) patients are hypersensitive to alkylating agents and radiation traditionally used as conditioning regimens for marrow cell transplantation, and patients experience serious toxicities. To reduce toxicities, we used progressively lower doses of cyclophosphamide (CY) for conditioning. Here, we report the results in 43 FA patients who received marrow transplantation from HLA-matched related donors (37 siblings and 6 other relatives). Conditioning consisted of 15 mg CY/kg/day for 4 days along with Mesna. Methotrexate and cyclosporine were given for graft-versus-host disease (GVHD) prophylaxis. Forty patients (93%) are alive with a median follow-up of 3.7 (range 0.6 to 7.9) years. One patient with primary graft failure was successfully retransplanted. Three of 4 patients with late graft failures were retransplanted, and 2 of those are alive; 1 died before a second marrow graft. Twelve patients including 3 with rejection had cytogenetic abnormalities in their marrow cells before transplantation. Acute grade II-III and chronic GVHD (aGVHD, cGVHD) were seen in 17% and 28.5% of patients, respectively. These results confirm and extend our previous observations that conditioning with 60 mg CY/kg allows for sustained engraftment of HLA-matched related marrow grafts in most FA patients and is associated with low toxicity, low incidences of aGVHD and cGVHD, and excellent long-term survival.
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