Addition of High-Dose Cytarabine to Fludarabine-Based Conditioning for Hematopoietic Stem Cell Transplantation for Treating Fanconi Anemia Patients with Advanced Myeloid Malignancy: A Single-Center Experience and Literature Review

Biol Blood Marrow Transplant. 2016 Sep;22(9):1725-1728. doi: 10.1016/j.bbmt.2016.05.021. Epub 2016 May 28.

Abstract

The complication of Fanconi anemia (FA) with acute leukemia is rare and challenging to treat because of high relapse rates, despite the improved outcome of hematopoietic stem cell transplantation with fludarabine-based conditioning for treating FA patients with hematological abnormalities. We added high-dose cytarabine to fludarabine-based conditioning to promote an enhanced antitumor effect and successfully subjected 4 patients with FA, including 3 with acute leukemia, to hematopoietic stem cell transplantation. All patients remain alive without treatment-related mortality or evidence of disease. Adding high-dose cytarabine to fludarabine-based conditioning may be tolerable and effective for treating FA patients with acute leukemia.

Keywords: Acute leukemia; Fanconi anemia; Fludarabine-based conditioning; Hematopoietic stem cell transplantation; High-dose cytarabine.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Antimetabolites, Antineoplastic / therapeutic use
  • Child
  • Cytarabine / therapeutic use*
  • Fanconi Anemia / complications*
  • Fanconi Anemia / therapy*
  • Female
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Leukemia, Myeloid, Acute / etiology
  • Leukemia, Myeloid, Acute / therapy
  • Male
  • Myeloablative Agonists / therapeutic use
  • Transplantation Conditioning / methods*
  • Treatment Outcome
  • Unrelated Donors
  • Vidarabine / analogs & derivatives
  • Vidarabine / therapeutic use

Substances

  • Antimetabolites, Antineoplastic
  • Myeloablative Agonists
  • Cytarabine
  • Vidarabine
  • fludarabine