Successful allogeneic stem cell transplant for leukocyte adhesion deficiency using an adjusted busulfan-containing regimen

Pediatr Transplant. 2007 Jun;11(4):453-5. doi: 10.1111/j.1399-3046.2007.00723.x.

Abstract

LAD is a rare and fatal congenital disorder in which there is a defect of the leukocyte adhesion molecule (CD18) on neutrophils. Severe LAD results in frequent and potentially fatal infections. Although allogeneic HSCT is the only curative treatment for severe LAD, strategies for HSCT remain to be established since a high engraftment failure rate and severe persistent infection are still seen. We report a four-month-old boy with LAD who was successfully treated with allogeneic HSCT from an HLA-complete matched sibling following a conditioning regimen of pharmacokinetically adjusted individual busulfan doses.

Publication types

  • Case Reports

MeSH terms

  • Busulfan / therapeutic use*
  • Follow-Up Studies
  • Graft Rejection / prevention & control
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Leukocyte-Adhesion Deficiency Syndrome / surgery*
  • Male
  • Transplantation Conditioning / methods*
  • Transplantation, Homologous

Substances

  • Immunosuppressive Agents
  • Busulfan