Use of Mycophenolate Mofetil for Chronic, Refractory Immune Cytopenias in Children With Autoimmune Lymphoproliferative Syndrome

Br J Haematol. 2005 May;129(4):534-8. doi: 10.1111/j.1365-2141.2005.05496.x.

Abstract

Autoimmune lymphoproliferative syndrome (ALPS) is a disorder of apoptosis associated most often with heritable FAS mutations leading to lymphadenopathy, hypersplenism and chronic refractory autoimmune cytopenias. Mycophenolate mofetil (MMF) was used to treat cytopenias in 13 ALPS patients aged 9 months to 17 years from a cohort of 118 children (aged < 18 years) and 82 adults. Twelve responded for a median follow-up of 49 weeks (range 38-240 weeks), defined by maintenance of adequate blood counts and reduction in dosage or cessation of other immunosuppressive agents. This preliminary experience suggests that MMF may spare steroid usage in patients with ALPS-associated cytopenias.

Publication types

  • Clinical Trial
  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Anemia, Hemolytic, Autoimmune / drug therapy
  • Anemia, Hemolytic, Autoimmune / immunology
  • Autoimmune Diseases / drug therapy*
  • Autoimmune Diseases / immunology
  • Child
  • Child, Preschool
  • Follow-Up Studies
  • Humans
  • Immunosuppressive Agents / adverse effects
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Leukocyte Count
  • Lymphoproliferative Disorders / drug therapy*
  • Lymphoproliferative Disorders / immunology
  • Male
  • Mycophenolic Acid / adverse effects
  • Mycophenolic Acid / analogs & derivatives*
  • Mycophenolic Acid / therapeutic use*
  • Neutropenia / drug therapy
  • Neutropenia / immunology
  • Pain / chemically induced
  • Prodrugs
  • Thrombocytopenia / drug therapy
  • Thrombocytopenia / immunology

Substances

  • Immunosuppressive Agents
  • Prodrugs
  • Mycophenolic Acid