Allogeneic hematopoietic cell transplantation for inborn metabolic diseases

Research output: Contribution to journalArticle

8 Scopus citations

Abstract

Clinical experience for more than two decades has shown that allogeneic HCT may benefit some but not all patients with inherited metabolic diseases. The HCT procedure is most effective in pre-symptomatic patients and those with indolent forms of storage diseases but is ineffective in those with overt neurological symptoms or aggressive neonatal or infantile forms. HCT alone does not correct skeletal dysplasia in MPSs and may not prevent development or progression of the peripheral neuropathy in sphingolipidoses and ALD. Decisions regarding HCT in patients with storage diseases should be made by investigators knowledgeable about these diseases, with judicious use of laboratory and clinical resources necessary to reach the best therapeutic decision for the individual patient.

Original languageEnglish (US)
Pages (from-to)S16-S19
JournalAnnals of Hematology
Volume81
Issue numberSUPPL. 2
StatePublished - Dec 1 2002

ASJC Scopus subject areas

  • Hematology

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