European Journal of Pediatrics

, Volume 166, Issue 7, pp 663–666

Successful treatment of Wolman disease by unrelated umbilical cord blood transplantation

  • Jerry Stein
  • Ben Zion Garty
  • Yael Dror
  • Eyal Fenig
  • Marsha Zeigler
  • Isaac Yaniv
Original Paper

DOI: 10.1007/s00431-006-0298-6

Cite this article as:
Stein, J., Zion Garty, B., Dror, Y. et al. Eur J Pediatr (2007) 166: 663. doi:10.1007/s00431-006-0298-6

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.

Keywords

Wolman diseaseStem cell transplant

Abbreviations

WD

wolman disease

BMT

bone marrow transplant

UCB

umbilical cord blood

GVHD

graft versus host disease

LDL

low-density lipoproteins

MRI

magnetic resonance imaging

Copyright information

© Springer-Verlag 2006

Authors and Affiliations

  • Jerry Stein
    • 1
  • Ben Zion Garty
    • 2
  • Yael Dror
    • 3
  • Eyal Fenig
    • 4
  • Marsha Zeigler
    • 5
  • Isaac Yaniv
    • 1
  1. 1.Bone Marrow Transplantation Unit, Department of Pediatric Hematology/OncologySchneider Children’s Medical Center of IsraelPetach TikvaIsrael
  2. 2.Department of Pediatrics BSchneider Children’s Medical Center of IsraelPetach TikvaIsrael
  3. 3.Department of Clinical NutritionSchneider Children’s Medical Center of IsraelPetach TikvaIsrael
  4. 4.Department of Oncology Rabin Medical Center Petach Tikva with Sackler Faculty of MedicineTel Aviv UniversityTel AvivIsrael
  5. 5.Department of Human GeneticsHadassah University HospitalJerusalemIsrael