Reduced toxicity and prompt engraftment after minimal conditioning of a patient with fanconi anemia undergoing hematopoietic stem cell transplantation from an HLA-matched unrelated donor

Peter Kurre, Michael Pulsipher, Ann Woolfrey, Michael Maris, Brenda Sandmaier, Hans Peter Kiem, Rainier Storb

Research output: Contribution to journalArticle

12 Scopus citations

Abstract

Given the profound sensitivity of patients with Fanconi anemia to conventional conditioning regimens before hematopoietic stem cell transplantation (HSCT), we developed a minimally toxic regimen consisting of 2 Gy total body irradiation, 90 mg/m2 fludarabine, and postgrafting immunosuppression with cyclosporine and mycophenolate to treat FA patients undergoing HSCT from HLA-matched unrelated donors. With over 10 months follow-up, our first patient has complete and sustained engraftment. Graft-versus-host disease was limited to mild skin and liver and moderate gut manifestations. We conclude that the approach is well tolerated and ideally suited to reduce regimen-related toxicities while achieving sustained engraftment.

Original languageEnglish (US)
Pages (from-to)581-583
Number of pages3
JournalJournal of Pediatric Hematology/Oncology
Volume25
Issue number7
DOIs
StatePublished - Jul 1 2003

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Keywords

  • Fanconi anemia
  • Minimal toxicity conditioning regimen
  • Stem cell transplantation

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

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