Bone marrow transplantation from matched siblings in patients with fanconi anemia utilizing low-dose cyclophosphamide, thoracoabdominal radiation and antithymocyte globulin

Bone Marrow Transplant. 2001 Jan;27(2):139-43. doi: 10.1038/sj.bmt.1702754.

Abstract

Nineteen patients with Fanconi anemia (FA) and bone marrow failure underwent bone marrow transplantation (BMT) from matched siblings. Median age at BMT was 8.7 years. Conditioning consisted of low-dose cyclophosphamide (CY 5 mg/kg x 4 days) and thoracoabdominal irradiation (TAI 400 cGy). Graft-versus-host disease (GVHD) prophylaxis was cyclosporin A (CsA) in 13 patients and CsA plus methotrexate in 6 patients. Antithymocyte globulin (ATG) was added in the pretransplant as well as the post-transplant period. All patients received high-dose acyclovir from day 2 pre-BMT to day 28 post BMT, and intravenous immunoglobulins (IVIG), 500 mg/kg weekly from day 7 pre-BMT to day 90 post BMT. No fungal prophylaxis was given. All patients engrafted, (median, 14 days for an absolute neutrophil count > or =0.5 x 10(9)/l; median, 37 days for platelet count > or =20 x 10(9)/l). Fourteen (74%) patients are alive with sustained engraftment and are transfusion independent. Three (16.6%) patients developed acute GVHD; none developed chronic GVHD. Five (26%) patients developed invasive fungal infections, and two (10%) developed fatal CMV disease. We believe the addition of ATG may have contributed to the increased incidence of severe life-threatening fungal and viral infections in our series.

MeSH terms

  • Antilymphocyte Serum / administration & dosage*
  • Bone Marrow Transplantation*
  • Child
  • Child, Preschool
  • Cyclophosphamide / administration & dosage*
  • Fanconi Anemia / therapy*
  • Female
  • Hemibody Irradiation
  • Histocompatibility Testing
  • Humans
  • Immunosuppressive Agents / administration & dosage*
  • Infant
  • Male
  • Transplantation, Homologous

Substances

  • Antilymphocyte Serum
  • Immunosuppressive Agents
  • Cyclophosphamide