Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice Guidelines from the Hunter's Hope Leukodystrophy Care Network.
Abstract
The leukodystrophies are a heterogeneous group of inherited diseases characterized
by progressive demyelination of the central nervous system leading to devastating
neurologic symptoms and premature death. Hematopoietic stem cell transplantation (HSCT)
has been successfully used to treat certain leukodystrophies, including adrenoleukodystrophy,
globoid leukodystrophy (Krabbe disease), and metachromatic leukodystrophy, over the
past 30 years. To date, these complex patients have primarily been transplanted at
a limited number of pediatric centers. As the number of cases identified through pregnancy
and newborn screening is increasing, additional centers will be required to treat
these children. Hunter's Hope created the Leukodystrophy Care Network in part to create
and standardize high-quality clinical practice guidelines to guide the care of affected
patients. In this report the clinical guidelines for the care of pediatric patients
with leukodystrophies undergoing treatment with HSCT are presented. The initial transplant
evaluation, determination of patient eligibility, donor selection, conditioning, supportive
care, and post-transplant follow-up are discussed. Throughout these guidelines the
need for early detection and treatment and the role of the partnership between families
and multidisciplinary providers are emphasized.
Type
Journal articleSubject
HumansLeukodystrophy, Globoid Cell
Leukodystrophy, Metachromatic
Hematopoietic Stem Cell Transplantation
Infant, Newborn
Practice Guidelines as Topic
Allografts
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https://hdl.handle.net/10161/24569Published Version (Please cite this version)
10.1016/j.bbmt.2019.09.003Publication Info
Page, Kristin M; Stenger, Elizabeth O; Connelly, James A; Shyr, David; West, Tara;
Wood, Susan; ... Kurtzberg, Joanne (2019). Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice
Guidelines from the Hunter's Hope Leukodystrophy Care Network. Biology of blood and marrow transplantation : journal of the American Society for
Blood and Marrow Transplantation, 25(12). pp. e363-e374. 10.1016/j.bbmt.2019.09.003. Retrieved from https://hdl.handle.net/10161/24569.This is constructed from limited available data and may be imprecise. To cite this
article, please review & use the official citation provided by the journal.
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Show full item recordScholars@Duke
Laura Elizabeth Case
Associate Professor in Orthopaedic Surgery
Laura E Case, PT, DPT, MS, PCS, C/NDT is a board-certified clinical specialist in
pediatric physical therapy. She has dedicated her career to teaching, research in
childhood-onset neuromusculoskeletal disorders, and to the lifelong treatment of people
with childhood-onset neurological and neuromuscular disorders such as cerebral palsy,
traumatic brain injury, Duchenne muscular dystrophy, spinal muscular atrophy, Pompe
disease, myelodysplasia, juvenile rheumatoid arthriti
Joanne Kurtzberg
Jerome S. Harris Distinguished Professor of Pediatrics
Dr. Kurtzberg is an internationally renowned expert in pediatric hematology/oncology,
pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation,
and novel applications of cord blood and birthing tissues in the emerging fields of
cellular therapies and regenerative medicine. Dr. Kurtzberg serves as the Director
of the Marcus Center for Cellular Cures (MC3), Director of the Pediatric Transplant
and Cellular Therapy Program, Director of the Carolina
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 t
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