scholarly journals Hematopoietic Stem Cell Transplantation for Pediatric Patients with Hereditary Bone Marrow Failure Syndromes Without Total Body Irradiation

2012 ◽  
Vol 18 (2) ◽  
pp. S232
Author(s):  
A.A. Hamidieh ◽  
M. Behfar ◽  
A. Hamdi ◽  
M.R. Ostadali ◽  
M. Jalili ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Total Body Irradiation ◽  
Pediatric Patients ◽  
Bone Marrow Failure ◽  
Hematopoietic Stem ◽  
Bone Marrow Failure Syndromes ◽  
Total Body

HSCT FOR ACQUIRED BONE MARROW FAILURE SYNDROMES

JBMTCT ◽  
2021 ◽  
Vol 4 (1) ◽  
pp. 245-250
Author(s):  
Sociedade Brasileira de TMO SBTMO ◽  
Elias Hallack Atta ◽  
Luiz Guilherme Darrigo Junior ◽  
Carmem Bonfim ◽  
Phillip Scheinberg
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Marrow Transplantation ◽  
Bone Marrow Failure ◽  
Hematopoietic Stem ◽  
Blood And Marrow Transplantation ◽  
Brazilian Society ◽  
Bone Marrow Failure Syndromes

THE BRAZILIAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION (SBTMO) PRESENTS THE BRAZILIAN GUIDELINES ON HEMATOPOIETIC STEM CELL TRANSPLANTATION

Allogeneic Hematopoietic Stem Cell Transplantation Using An Intravenous Busulfan Based Myeloablative Conditioning without Total Body Irradiation for Pediatric Patients with Acute Lymphoblastic Leukemia

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 3547-3547
Author(s):  
Eugene Goussetis ◽  
Anna Paisiou ◽  
Ioulia Peristeri ◽  
Vassiliki Kitra ◽  
George Vessalas ◽  
...  
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Graft Versus Host Disease ◽  
Total Body Irradiation ◽  
Pediatric Patients ◽  
Hematopoietic Stem ◽  
Graft Versus Host ◽  
Total Body

Abstract Abstract 3547 Allogeneic hematopoietic stem cell transplantation (allo-HSCT) for acute lymphoblastic leukaemia (ALL) has been most commonly performed using a myeloablative, total body irradiation (TBI)-based preparative regimen; however, there are emerging concerns about long-term sequelae of TBI in pediatric patients. The availability of intravenous (i.v.) busulfan (Bu) prompted us to evaluate the effectiveness of i.v. Bu-based preparative regimens on pediatric patients who underwent allo-SCT for ALL. We retrospectively evaluated the outcomes of 31 children with ALL transplanted with i.v. Bu-based preparative regimens at our institution between January 2005 and May 2010. Twenty-one patients were in first complete remission (CR1), 8 in CR2, 1 in CR3, and 1 in advanced disease. The donors were HLA-matched siblings (n=9), matched unrelated (n=20), or 1-antigen mismatched related donors (n=2). The median age of the patients was 4.8 years (range, 2 months-16.7 years). Twenty patients received bone marrow, 8 peripheral blood stem cells, and 3 cord blood. Busulfan was administered as a 2 h IV infusion every 6 h over 4 days (16 administrations). Five dose levels were defined on body weight as follows: 1.0 mg/kg for <9 kg; 1.2 mg/kg for 9 to <16 kg; 1.1 mg/kg for 16–23 kg; 0.95 mg/kg for >23-34 kg; 0.80 mg/kg for >34 kg. Busulfan administration was followed by Cyclophosphamide and VP-16 in 21 patients, Cyclophosphamide and Thiotepa in 6, Cyclophosphamide and Melphalan in 2 and Cyclophosphamide and Fludarabine in 2 patients. Graft versus host disease (GVHD) prophylaxis consisted of Cyclosporine A and Methotrexate in all patients transplanted with blood or marrow stem cells, while Methotrexate was omitted in those patients who underwent cord blood allo-HSCT. Anti-thymocyte globulin was added in patients transplanted from an unrelated donor. All patients but one achieved sustained engraftment. Median time to ANC>500, and platelets>20.000 was 19 days (range14-29), and 21.5 days (range 12–44) respectively. One patient died on day 10 and another patient relapsed on day 22; both were considered invaluable for engraftment. There were 6 cases of mild veno-occlusive disease, and 5 cases of hemorrhagic cystitis. Grade II-IV acute graft-versus-host disease (GVHD) and chronic GVHD (cGVHD) occurred in 15/31 and 5/31 patients, respectively. At median follow-up of 27 months, 23 patients are alive and diseases free. Four patients died of relapse and 4 died of transplant-related mortality (TRM). The overall survival (OS) rate, relapse rate, and TRM rate were 67 %, 24%, and 10%, respectively. These results are comparable to those reported with TBI-based preparative regimens and suggest that it is time to re-evaluate the use of TBI in ALL patients. Disclosures: No relevant conflicts of interest to declare.

scholarly journals RECENT ADVANCES IN HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR INHERITED BONE MARROW FAILURE SYNDROMES

JBMTCT ◽  
2020 ◽  
Vol 2 (1) ◽  
pp. 69-76
Author(s):  
Luiz Guilherme Darrigo Junior ◽  
Carmem Bomfim
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Bone Marrow Failure ◽  
Hematopoietic Stem ◽  
Bone Marrow Failure Syndromes ◽  
Risk Of Cancer

The inherited bone marrow failure syndromes (IBMFS) are a heterogeneous group of genetic disorders characterized by the inadequate production of at least one of the hematopoietic lineages, leading to the development of both isolated cytopenia (anemia, neutropenia, or thrombocytopenia) or pancytopenia. Different biological mechanisms justify the pathophysiological changes found in the IBMFS, emphasizing the repair pathways in Fanconi anemia (FA), maintenance of telomeres in congenital dyskeratosis, and ribosome biogenesis in Shwachman Diamond syndrome (SSD) and Blackfan Diamond anemia. These disorders are generally associated with the presence of congenital malformations and an increased risk of cancer, mainly hematological, gynecological, and head and neck neoplasms. Although the diagnosis occurs typically in childhood, adult patients, mostly below 40 years of age with signs and symptoms suggestive of IBMFS, should be investigated. Currently, hematopoietic stem cell transplantation (HSCT) is the only curative option for hematological complications related to IBMFS.  It is essential to highlight that these patients must be monitored throughout their lives to prevent or detect early treatable neoplasia.

scholarly journals HSCT FOR INHERITED BONE MARROW FAILURE SYNDROMES

JBMTCT ◽  
2021 ◽  
Vol 4 (1) ◽  
pp. 39-43
Author(s):  
Sociedade Brasileira de TMO SBTMO ◽  
Luiz Guilherme Darrigo Junior ◽  
Phillip Scheinberg ◽  
Elias Hallack Atta ◽  
Carmem Bonfim
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Marrow Transplantation ◽  
Bone Marrow Failure ◽  
Hematopoietic Stem ◽  
Blood And Marrow Transplantation ◽  
Brazilian Society ◽  
Bone Marrow Failure Syndromes

THE BRAZILIAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION (SBTMO) PRESENTS THE BRAZILIAN GUIDELINES ON HEMATOPOIETIC STEM CELL TRANSPLANTATION

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