期刊
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
卷 24, 期 8, 页码 1651-1656出版社
ELSEVIER SCIENCE INC
DOI: 10.1016/j.bbmt.2018.04.025
关键词
Stem cell transplantation; Conditioning regimen; Myelodysplastic syndromes; Acute myeloid leukemia
资金
- Johnny Crisstopher Children's Charitable Foundation St. Baldrick's Consortium Grant
- Medac GmbH
- National Cancer Institute (NCI) [5U24CA076518]
- National Heart, Lung and Blood Institute (NHLBI)
- National Institute of Allergy and Infectious Diseases
- NHLBI [4U10HL069294]
- NCI [4U10HL069294]
- Health Resources and Services Administration (HRSA/DHHS) [HHSH250201200016C]
- Office of Naval Research [N00014-17-1-2388, N0014-17-1-2850]
- Actinium Pharmaceuticals, Inc.
- Amgen, Inc.
- Amneal Biosciences
- Angiocrine Bioscience, Inc.
- Astellas Pharma US
- Atara Biotherapeutics, Inc.
- Be the Match Foundation
- bluebird bio, Inc.
- Bristol Myers Squibb Oncology
- Celgene Corporation
- Cerus Corporation
- Chimerix, Inc.
- Fred Hutchinson Cancer Research Center
- Gamida Cell Ltd.
- Gilead Sciences, Inc.
- HistoGenetics, Inc.
- Immucor
- Incyte Corporation
- Janssen Scientific Affairs, LLC
- Jazz Pharmaceuticals, Inc.
- Juno Therapeutics
- Karyopharm Therapeutics, Inc.
- Kite Pharma, Inc.
- Medac, GmbH
- Medlmmune
- Medical College of Wisconsin
- Mediware
- Merck Co, Inc.
- Mesoblast
- MesoScale Diagnostics, Inc.
- Millennium
- Takeda Oncology Co.
- Miltenyi Biotec, Inc.
- National Marrow Donor Program
- Neovii Biotech NA, Inc.
- Novartis Pharmaceuticals Corporation
- Otsuka Pharmaceutical Co, Ltd. - Japan
- PCORI
- Pfizer, Inc
- Pharmacyclics, LLC
- PIRCHE AG
- Sanofi Genzyme
- Seattle Genetics
- Shire
- Spectrum Pharmaceuticals, Inc.
- St. Baldrick's Foundation
- Sunesis Pharmaceuticals, Inc.
- Swedish Orphan Biovitrum, Inc.
- Takeda Oncology
- Telomere Diagnostics, Inc.
- University of Minnesota
This multicenter study evaluated a treosulfan-based regimen in children and young adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplant (HCT). Forty patients with median age 11 years (range, 1 to 19) underwent allogeneic HCT for AML in first (n = 18), second (n =11), and third or greater remission (n = 3) or MDS (n = 8) using bone marrow (n = 25), peripheral blood stem cells (n = 5), or cord blood (n = 9). The regimen consisted of body surface area (BSA)-based treosulfan 10 g/m(2)/day (BSA <= .5 m(2)), 12 g/m(2)/day (BSA > .5 to 1.0 m(2)), or 14 g/m(2)/day (BSA > 1.0 m(2)) on days -6 to -4; fludarabine 30 mg/m(2)/day on days -6 to -2; and a single fraction of 200 cGy total body irradiation on day-1. Graft-versus-host disease (GVHD) prophylaxis included tacrolimus and methotrexate for marrow and peripheral blood stem cell and cyclosporine/mycophenolate mofetil for cord blood. One-year overall survival, disease-free survival, and nonrelapse mortality were 80%, 73%, and 3%, respectively. One-year relapse was 38% for AML and 13% for MDS. No serious organ toxicities were observed. All 37 assessable patients engrafted. Cumulative incidences of grades II to IV acute GVHD and chronic GVHD were 22% and 40%, respectively. BSA-based treosulfan dosing resulted in predictable area under the curve and maximum concentration, which is required for dosing without measuring individual pharmacokinetic parameters. Observed differences in pharmacokinetics did not impact disease control or regimen toxicity. This BSA-based treosulfan regimen resulted in excellent engraftment and disease-free survival and minimal toxicity and transplant-related mortality (3%) in children and young adults with AML and MDS. (C) 2018 American Society for Blood and Marrow Transplantation.
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