ArticleJournal of clinical medicine2021
Fludarabine-Cyclophosphamide-Based Conditioning with Antithymocyte Globulin Serotherapy Is Associated with Durable Engraftment and Manageable Infections in Children with Severe Aplastic Anemia.
Article in Journal of clinical medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
What it found
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Who cites it
5 citing papers in PubMed, 9 citations in OpenAlex.
- Hemorrhagic cystitis in pediatric severe aplastic anemia undergoing haploidentical hematopoietic stem cell transplantation: incidence, risk factors and outcomes.BMC pediatrics · 2025Article
- The state of the art in the treatment of severe aplastic anemia: immunotherapy and hematopoietic cell transplantation in children and adults.Frontiers in immunology · 2024Review
- Current status and perspectives of hematopoietic cell transplantation in patients with paroxysmal nocturnal hemoglobinuria.Frontiers in immunology · 2024Review
- Shifting Paradigms: The Case of Autologous Reconstitution after an Upfront Matched Unrelated Hematopoietic Cell Transplantation for Severe Acquired Aplastic Anemia in a Child.Medicina (Kaunas, Lithuania) · 2023Review
- Short- and long-term outcome of allogeneic stem cell transplantation in infants: A single-center experience over 20 years.Frontiers in pediatrics · 2022Article
Corrections and comments
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Authors and funding
16 authors at 1 institution in 1 country.
Funding
Abstract
Severe aplastic anemia (SAA) is a bone marrow failure syndrome that can be treated with hematopoietic cell transplantation (HCT) or immunosuppressive (IS) therapy. A retrospective cohort of 56 children with SAA undergoing transplantation with fludarabine-cyclophosphamide-ATG-based conditioning (FluCyATG) was analyzed. The endpoints were overall survival (OS), event-free survival (EFS), cumulative incidence (CI) of graft versus host disease (GVHD) and CI of viral replication. Engraftment was achieved in 53/56 patients, and four patients died (two due to fungal infection, and two of neuroinfection). The median time to neutrophil engraftment was 14 days and to platelet engraftment was 16 days, and median donor chimerism was above 98%. The overall incidence of acute GVHD was 41.5%, and that of grade III-IV acute GVHD was 14.3%. Chronic GVHD was diagnosed in 14.2% of children. The probability of 2-year GVHD-free survival was 76.1%. In the univariate analysis, a higher dose of cyclophosphamide and previous IS therapy were significant risk factors for worse overall survival. Episodes of viral replication occurred in 33/56 (58.9%) patients, but did not influence OS. The main advantages of FluCyATG include early engraftment with a very high level of donor chimerism, high overall survival and a low risk of viral replication after HCT.
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Registered trials
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