Correction of autoimmune lymphoproliferative syndrome by bone marrow transplantation

B. J. Sleight, V. S. Prasad, C. DeLaat, P. Steele, E. Ballard, R. J. Arceci, C. L. Sidman

Research output: Contribution to journalArticle

Abstract

This report describes a child with a severe phenotype of autoimmune lymphoproliferative syndrome (ALPS) who developed progressive disease requiring stem cell transplantation. This severe form of ALPS was associated with a novel Fas gene splice site mutation that resulted in functional deletion of exons 8 and 9. While this child shared many clinical features with previously described ALPS cases, including massive lymphadenopathy and circulating αβ+ CD3+CD4-CD8- T cells, his disease progressed despite immunosuppressive therapy to a clinically aggressive oligoclonal lymphoproliferation which resembled a diffuse large cell non-Hodgkin's lymphoma. After partial remission was achieved with cytotoxic therapy the patient underwent BMT from an unrelated donor. This is the first reported case of ALPS in which BMT was successfully attempted for correction of a Fas deficiency.

Original languageEnglish (US)
Pages (from-to)375-380
Number of pages6
JournalBone marrow transplantation
Volume22
Issue number4
DOIs
StatePublished - Jan 1 1998

Keywords

  • Autoimmune lymphoproliferative syndrome
  • Fas gene defects
  • Lymphoma
  • Lymphoproliferative disorder

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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