Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning. Biol Blood Marrow Transplant 2016 Sep;22(9):1627-1635
Date
06/07/2016Pubmed ID
27264632DOI
10.1016/j.bbmt.2016.05.024Scopus ID
2-s2.0-84978920140 (requires institutional sign-in at Scopus site) 34 CitationsAbstract
Infants and young children who undergo allogeneic cord blood transplantation (CBT) are at increased risk for late effects because of exposure of developing organs to chemotherapy and radiation therapy typically used in transplant conditioning regimens. Busulfan (Bu)-based myeloablative regimens were developed to eliminate radiation exposure in these young children with the hope that late effects would be minimized. We now describe the late effects in 102 consecutive patients surviving a minimum of 5 years (median follow-up, 12.9 years) post-CBT. Patients were conditioned with high-dose chemotherapy using Bu-containing regimens. No patient received total body irradiation. The median age at transplant was 1 year (range, .1 to 2). Diagnoses included inherited metabolic diseases (59.8%), leukemia (17.6%), congenital immune deficiency (20.2%), bone marrow failure/myelodysplastic syndrome (3.9%), and hemoglobinopathy (2%). Among patients surviving 5 years, the overall survival rate at 10 years post-CBT was 93% (95% CI, 84.9 to 96.8). Virtually all patients (98%) experienced at least 1 significant late effect. Most (83.3%) experienced 2 or more late effects, and more than half of the patients (64.7%) experienced 3 or more late effects. The most commonly observed late effects included dental problems (92.2%), short stature (55.9%), cognitive deficits (53.6%), pulmonary dysfunction (18.6%), and abnormal pubertal development (27.9%). This is the first report of late effects of Bu-based conditioning in a cohort of very young patients at the time of transplant. These results will inform clinical care guidelines for long-term follow-up and add to the growing information regarding outcomes of hematopoietic stem cell transplantation.
Author List
Allewelt H, El-Khorazaty J, Mendizabal A, Taskindoust M, Martin PL, Prasad V, Page K, Sanders J, Kurtzberg JAuthor
Kristin Page MD, MHS, MEd Associate Professor in the Pediatrics department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
BusulfanChemically-Induced Disorders
Child, Preschool
Cord Blood Stem Cell Transplantation
Drug-Related Side Effects and Adverse Reactions
Follow-Up Studies
Humans
Infant
Long Term Adverse Effects
Myeloablative Agonists
Survival Analysis
Transplantation Conditioning