Allogeneic Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for Treatment of Marrow Failure Disorders.
Submitted by Eneida R Nemecek on
Title | Allogeneic Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for Treatment of Marrow Failure Disorders. |
Publication Type | Journal Article |
Year of Publication | 2017 |
Authors | Burroughs, LM, Shimamura, A, Talano, J-A, Domm, JA, Baker, KK, Delaney, C, Frangoul, H, Margolis, DA, K Baker, S, Nemecek, ER, Geddis, AE, Sandmaier, BM, H Deeg, J, Storb, R, Woolfrey, AE |
Journal | Biol Blood Marrow Transplant |
Volume | 23 |
Issue | 10 |
Pagination | 1669-1677 |
Date Published | 2017 Oct |
ISSN | 1523-6536 |
Keywords | Adolescent, Antilymphocyte Serum, Bone Marrow Diseases, Busulfan, Child, Child, Preschool, Disease-Free Survival, Female, Graft Survival, Hematopoietic Stem Cell Transplantation, Humans, Infant, Male, Transplantation Conditioning, Transplantation, Homologous, Treatment Outcome, Vidarabine, Young Adult |
Abstract | 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), fludarabine (150 mg/m), ± 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. |
DOI | 10.1016/j.bbmt.2017.06.002 |
Alternate Journal | Biol. Blood Marrow Transplant. |
PubMed ID | 28602958 |
PubMed Central ID | PMC5605451 |
Grant List | K23 HL085288 / HL / NHLBI NIH HHS / United States P01 HL036444 / HL / NHLBI NIH HHS / United States P01 HL122173 / HL / NHLBI NIH HHS / United States |