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Early Engraftment and Immune Kinetics Following Allogeneic Transplant Using a Novel Reduced-Toxicity Transplant Strategy in Children/Adolescents with High-Risk Transfusion-Dependent Thalassemia: Early Results of the ThalFAbS Trial.
Transplant Cell Ther
December 2024
Hematology/Oncology, SickKids Hospital and the University of Toronto, Toronto, Ontario, Canada.
Allogeneic hematopoietic stem cell transplantation is challenging for patients with transfusion-dependent thalassemia who have experienced iron overload and received chronic transfusion support. A transplantation strategy including a reduced-intensity preparative regimen and tailored immunosuppression to support donor engraftment and prevent graft-versus-host disease (GVHD) was developed for this population. The combination of a pretransplantation immunosuppression phase with reduced dosing of fludarabine/prednisone, a treosulfan-based preparative regimen with reduced cyclophosphamide dosing, and introduction of a calcineurin/methotrexate-free GVHD prophylaxis/engraftment supporting regimen with abatacept/sirolimus/antithymocyte globulin was tested.
View Article and Find Full Text PDFMelphalan Dose in Combination With Fludarabine Affects Gastrointestinal Toxicity and Graft-Versus-Host Disease After Allogeneic Transplantation in Acute Myeloid Leukemia and Myelodysplastic Syndromes.
Transplant Cell Ther
November 2024
Department of Blood and Marrow Transplantation - Cellular Immunotherapy, Moffitt Cancer Center, Tampa, Florida. Electronic address:
Fludarabine (Flu) and melphalan (Mel) reduced-intensity conditioning is frequently used for allogenic hematopoietic cell transplant (allo-HCT) in patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). However, there is limited evidence on the impact of Mel dosing on toxicities and clinical outcomes of allo-HCT. We retrospectively compared 8/8 HLA-matched donor allo-HCT outcomes of 345 patients with AML or MDS receiving total Mel dose of 100 mg/m (Mel-100, n = 62) versus 140 mg/m (Mel-140, n = 283) in combination with Flu.
View Article and Find Full Text PDF[Current trends in the treatment of aplastic anemia].
Rinsho Ketsueki
July 2024
Faculty of Medicine, Department of Medical Sciences, Hematology, University of Tsukuba.
Aplastic anemia is a syndrome characterized by reduced hematopoietic stem cells, bone marrow hypoplasia, and pancytopenia, and is often considered a T-cell-mediated autoimmune disease. It is predominantly treated with hematopoietic stem cell transplantation and immunosuppressive therapy with anti-human thymocyte immunoglobulin (ATG) and cyclosporine. Only rabbit ATG was previously available in Japan, but equine ATG was recently approved for use in 2023.
View Article and Find Full Text PDFAllogeneic stem cell transplantation for inherited metabolic disorders: 35 years' experience at a single institution.
Int J Hematol
September 2024
Department of Pediatrics, Tokai University School of Medicine, 143 Shimokasuya, Isehara, Kanagawa, 259 1193, Japan.
Hematopoietic stem cell transplants for inherited metabolic disorders performed at Tokai University Hospital between June 5, 1986, and May 28, 2021, were analyzed and compared between the period before 2007 and the period from 2007 onward based on availability of medical resources. Transplants were performed for 38 patients with mucopolysaccharidosis, 33 with adrenoleukodystrophy, and 16 with another disorder. Before 2007, oral busulfan-based regimens were mainly used.
View Article and Find Full Text PDFSynthesis and cytotoxicity of novel 6,8,9-trisubstituted purine analogs against liver cancer cells.
Bioorg Med Chem Lett
July 2024
Department of Pharmaceutical Chemistry, Faculty of Pharmacy, Ankara University, 06560, Ankara, Turkey. Electronic address:
A series of novel 6-(substituted phenyl piperazine)-8-(4-substituted phenyl)-9-cyclopentyl purines, 10-51, were synthesized by a four-step synthesis, achieving an overall yield of about 43 %. The reaction conditions were effectively optimized, and the final products were obtained with high purity and yield in all synthesis steps. The synthesized nucleobases were evaluated for their in vitro cytotoxic activities on selected human cancer cell lines (HUH7 (liver), HCT116 (colon), and MCF7 (breast)) using the Sulforhodamine B (SRB) assay.
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