Journal
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
Volume 23, Issue 10, Pages 1669-1677Publisher
ELSEVIER SCIENCE INC
DOI: 10.1016/j.bbmt.2017.06.002
Keywords
Bone marrow failure; Diamond Blackfan Anemia; Shwachman-Diamond; Syndrome; Reduced-toxicity conditioning in nonmalignant diseases
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Funding
- National Heart, Lung, and Blood Institute, National Institutes of Health (Bethesda, MD) [P01 HL122173, P01 HL036444, K23 HL085288]
- Medac, GmbH (Hamburg, Germany)
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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/m(2)), fludarabine (150 mg/m(2)), +/- 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. (C) 2017 American Society for Blood and Marrow Transplantation.
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