Long-Term Functional Outcomes after Hematopoietic Stem Cell Transplant for Early Infantile Krabbe Disease. Biol Blood Marrow Transplant 2018 Nov;24(11):2233-2238
Date
06/23/2018Pubmed ID
29933067DOI
10.1016/j.bbmt.2018.06.020Scopus ID
2-s2.0-85051028331 (requires institutional sign-in at Scopus site) 47 CitationsAbstract
Allogeneic hematopoietic stem cell transplantation (HSCT) can retard the progression of early infantile Krabbe disease (EIKD). Superior outcomes are achieved if HSCT is performed before the onset of symptoms; however, little information is available about the long-term outcomes in surviving patients. We now describe functional outcomes in presymptomatic infants who underwent HSCT for EIKD at ≤ 2 months of age. Records of the 19 patients who underwent HSCT for EIKD at ≤ 2 months of age from 1996 to 2010 were reviewed. Long-term functional outcomes were compared between those transplanted at < 30 days and ≥ 30 days of life. Median age at transplant was 27 days (range, 19 to 61). Median follow-up of the cohort was 12.6 years. Overall survival at 5 and 10 years post-transplant was 84.2% (95% confidence interval, 58.7% to 94.6%) and 78.6% (95% confidence interval, 52.5% to 91.4%), respectively. More favorable outcomes were seen in patients who underwent HSCT at < 30 days of age, particularly in domains of mobility (P = .01), communication (P = .02), and feeding (P = .008). Improved functional outcomes were observed when HSCT was performed in the first month of life, defining a critical period for intervention. These results support the implementation of newborn screening to enable rapid diagnosis and early treatment of infants with EIKD.
Author List
Allewelt H, Taskindoust M, Troy J, Page K, Wood S, Parikh S, Prasad VK, 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
Cord Blood Stem Cell TransplantationFemale
Hematopoietic Stem Cell Transplantation
Humans
Infant, Newborn
Leukodystrophy, Globoid Cell
Male
Survival Analysis
Transplantation Conditioning
Treatment Outcome
