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Allogeneic Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for Treatment of Marrow Failure Disorders.
Burroughs, Lauri M; Shimamura, Akiko; Talano, Julie-An; Domm, Jennifer A; Baker, Kelsey K; Delaney, Colleen; Frangoul, Haydar; Margolis, David A; Baker, K Scott; Nemecek, Eneida R; Geddis, Amy E; Sandmaier, Brenda M; Deeg, H Joachim; Storb, Rainer; Woolfrey, Ann E.
Afiliação
  • Burroughs LM; Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington; Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington; Department of Medicine, University of Washington School of Medicine, Seattle, Washington; Hematology-Oncology, Seattle
  • Shimamura A; Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington; Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington; Department of Medicine, University of Washington School of Medicine, Seattle, Washington; Hematology-Oncology, Seattle
  • Talano JA; Division of Hematology/Oncology/Blood and Marrow Transplant, Department of Pediatrics, Medical College of Wisconsin and Children's Hospital of Wisconsin, Milwaukee, Wisconsin.
  • Domm JA; Pediatric Hematology Oncology, Vanderbilt University, Nashville, Tennessee.
  • Baker KK; Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.
  • Delaney C; Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington; Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington; Department of Medicine, University of Washington School of Medicine, Seattle, Washington; Hematology-Oncology, Seattle
  • Frangoul H; Pediatric Hematology Oncology, Vanderbilt University, Nashville, Tennessee.
  • Margolis DA; Division of Hematology/Oncology/Blood and Marrow Transplant, Department of Pediatrics, Medical College of Wisconsin and Children's Hospital of Wisconsin, Milwaukee, Wisconsin.
  • Baker KS; Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington; Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington; Department of Medicine, University of Washington School of Medicine, Seattle, Washington; Hematology-Oncology, Seattle
  • Nemecek ER; Pediatric Blood and Marrow Transplant Program, Doernbecher Children's Hospital, Oregon Health and Sciences University, Portland, Oregon.
  • Geddis AE; Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington; Department of Medicine, University of Washington School of Medicine, Seattle, Washington; Hematology-Oncology, Seattle Children's Hospital, Seattle, Washington.
  • Sandmaier BM; Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington; Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington; Department of Medicine, University of Washington School of Medicine, Seattle, Washington.
  • Deeg HJ; Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington; Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington; Department of Medicine, University of Washington School of Medicine, Seattle, Washington.
  • Storb R; Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington; Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington; Department of Medicine, University of Washington School of Medicine, Seattle, Washington.
  • Woolfrey AE; Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington; Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington; Department of Medicine, University of Washington School of Medicine, Seattle, Washington; Hematology-Oncology, Seattle
Biol Blood Marrow Transplant ; 23(10): 1669-1677, 2017 Oct.
Article em En | MEDLINE | ID: mdl-28602958
Hematopoietic cell transplantation (HCT) is effective in the treatment of inherited marrow failure disorders and other nonmalignant diseases. Conventional myeloablative conditioning regimens have been associated with high transplant-related mortality, particularly in patients with comorbid conditions. Here we report on 14 patients with marrow failure disorders (Shwachman-Diamond syndrome, n = 3; Diamond Blackfan anemia, n = 4; GATA2 deficiency, n = 2; paroxysmal nocturnal hemoglobinuria, n = 4; and an undefined marrow failure disorder, n = 1) who underwent HCT on a prospective, phase II, multicenter clinical trial. Patients were given HLA-matched related (n = 2) or unrelated (n = 12) grafts after conditioning with treosulfan (42 g/m2), fludarabine (150 mg/m2), ± thymoglobulin (n = 11; 6 mg/kg). All patients engrafted. At a median follow-up of 3 years, 13 patients are alive with complete correction of their underlying disease. These results indicate that the combination of treosulfan, fludarabine, and thymoglobulin is effective at establishing donor engraftment with a low toxicity profile and excellent disease-free survival in patients with marrow failure disorders.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doenças da Medula Óssea / Bussulfano / Transplante de Células-Tronco Hematopoéticas / Condicionamento Pré-Transplante Tipo de estudo: Clinical_trials Limite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Ano de publicação: 2017 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doenças da Medula Óssea / Bussulfano / Transplante de Células-Tronco Hematopoéticas / Condicionamento Pré-Transplante Tipo de estudo: Clinical_trials Limite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Ano de publicação: 2017 Tipo de documento: Article