Successful treatment of Wolman disease by unrelated umbilical cord blood transplantation |
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| Authors: | Jerry Stein Ben Zion Garty Yael Dror Eyal Fenig Marsha Zeigler Isaac Yaniv |
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| Institution: | (1) Bone Marrow Transplantation Unit, Department of Pediatric Hematology/Oncology, Schneider Children’s Medical Center of Israel, Petach Tikva, Israel;(2) Department of Pediatrics B, Schneider Children’s Medical Center of Israel, Petach Tikva, 49202, Israel;(3) Department of Clinical Nutrition, Schneider Children’s Medical Center of Israel, Petach Tikva, Israel;(4) Department of Oncology Rabin Medical Center Petach Tikva with Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel;(5) Department of Human Genetics, Hadassah University Hospital, Jerusalem, Israel |
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| Abstract: | Wolman disease is a rapidly fatal lysosomal storage disease caused by the complete absence of lysosomal acid lipase activity.
We report the cure of an infant with Wolman disease following transplantation of unrelated HLA-mismatched umbilical cord blood-derived
stem cells. Umbilical cord blood was chosen as the stem-cell source because of its immediate availability and reduced tendency
to cause graft-versus-host disease. The transplantation resulted in restoration of normal acid lipase levels before the onset
of permanent end-organ damage. Four years after transplantation, the patient is thriving and has normal levels of acid lipase
in peripheral blood cells. To our knowledge, this is the first report of a successful unrelated cord blood transplant in a
patient with Wolman disease. Umbilical cord stem cells transplantation can restore acid lipase levels in Wolman disease, and
if performed early, can cure the disease. |
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| Keywords: | Wolman disease Stem cell transplant |
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