GM-CSF in the treatment of Fanconi's anaemia

Br J Haematol. 1994 Aug;87(4):871-2. doi: 10.1111/j.1365-2141.1994.tb06756.x.

Abstract

We have used recombinant human (rh) GM-CSF in two 12-year-old Fanconi's aplastic anaemia patients. They had not received any previous therapy except blood transfusions. Each patient was given three 21 d courses of rh-GM-CSF, the first two at a dose of 3.5 micrograms/kg/d and the third at 7 micrograms/kg/d s.c. There were significant increases in WBC and absolute neutrophil counts after the first week of rh-GM-CSF which lasted as long as the treatment was continued. Following the cessation of treatment, WBC and ANC dropped rapidly. We conclude that rh-GM-CSF can be used in FAA, especially in severely neutropenic cases.

Publication types

  • Case Reports

MeSH terms

  • Child
  • Fanconi Anemia / blood
  • Fanconi Anemia / therapy*
  • Female
  • Granulocyte-Macrophage Colony-Stimulating Factor / therapeutic use*
  • Hemoglobins
  • Humans
  • Leukocyte Count
  • Male
  • Neutrophils
  • Recombinant Proteins / therapeutic use

Substances

  • Hemoglobins
  • Recombinant Proteins
  • Granulocyte-Macrophage Colony-Stimulating Factor