Long-Term Functional Outcomes following Hematopoietic Stem Cell Transplant for Early Infantile Krabbe Disease.

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) can retard the progression of early infantile Krabbe disease (EIKD). Superior outcomes are achieved if HSCT is performed prior to the onset of symptoms, however little information is available about the long-term outcomes in surviving patients. We now describe functional outcomes in pre-symptomatic infants who underwent HSCT for EIKD at ≤2 months of life. Records of the 19 patients who underwent HSCT for EIKD at ≤2 months of age from 1996-2010 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 19-61). Median follow-up of the cohort was 12.6 years. Overall survival at 5 and 10 years post-transplant was 84.2% (95% CI:58.7-94.6%) and 78.6% (95% CI:52.5-91.4%), respectively. More favorable outcomes were seen in patients who underwent HSCT at <30 days of age, particularly in domains of mobility (p=0.01), communication (p=0.02), and feeding (p=0.008). 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.

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Citation

Published Version (Please cite this version)

10.1016/j.bbmt.2018.06.020

Publication Info

Allewelt, Heather, Mahsa Taskindoust, Jesse Troy, Kristin Page, Susan Wood, Suhag Parikh, Vinod K Prasad, Joanne Kurtzberg, et al. (2018). Long-Term Functional Outcomes following Hematopoietic Stem Cell Transplant for Early Infantile Krabbe Disease. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 63. pp. S97–S97. 10.1016/j.bbmt.2018.06.020 Retrieved from https://hdl.handle.net/10161/17210.

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Scholars@Duke

Troy

Jesse David Troy

Associate Professor of Biostatistics & Bioinformatics

I am the Associate Director of Graduate Studies for the Master of Biostatistics program. I am also an instructor in the Clinical Research Training Program. I work on research studies in cancer therapeutics and palliative care at the Duke Cancer Institute.

Page

Kristin Marie Page

Assistant Professor of Pediatrics

Stem cell transplantation and/or cellular therapies can be used to treat a variety of pediatric diseases including malignancies such as leukemia, lymphoma and myelodysplastic syndrome in addition to certain non-malignant conditions (such as immune deficiencies, inherited metabolic diseases, hemoglobinopathies, and bone marrow failure syndromes). As the Director of the Pediatric Transplant and Cellular Therapy Survivorship Clinic, my goal is optimize the care of survivors of pediatric stem cell transplantation, cellular therapies and chemotherapy. To further support this goal, my research interests include improving patient-reported health related quality of life, access to survivorship care, and further understanding the pathophysiology of certain late effects with the ultimate goal of identifying treatments.

Parikh

Suhag H. Parikh

Associate Professor of Pediatrics

Stem cell transplantation for a variety of disorders - ranging from malignant diseases such as leukemia, lymphoma and myelodysplastic syndrome to nonmalignant diseases such as sickle cell disease, thalassemias, aplastic anemia, histiocytosis and leukodystrophies. My clinical research interest is stem cell transplantation for children with primary immune deficiency disorders and hemoglobinopathies such as sickle cell anemia,thalassemia and other non-malignant disorders. In addition,I am interested in developing startegies, such as reduced intensity conditioning, to make transplant safer.


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