USE OF RECOMBINANT GRANULOCYTE-COLONY-STIMULATING FACTOR IN FANCONIS-ANEMIA

Background and Objective. Granulocyte colony-stimulating factor (G-CSF ) has been shown to improve the neutropenic status of patients with bo ne marrow failure. The side effects in prolonged treatment still need to be determined. Design and Methods. We have studied the efficacy and the long-term side effects of G-CSF in four patients with Fanconi's a nemia and severe neutropenia. Results. Three patients responded with a n increase in their absolute neutrophil count; neither improvement in platelet count and hemoglobin concentration nor effect on transfusion requirements was seen. CFU-GM and BN-E were undetectable before, durin g and after treatment. Responders showed an important reduction in num ber and severity of infections, with a marked improvement of clinical status. The fourth patient developed acute myeloid leukemia after 4 we eks of G-CSF treatment. During maintenance, one patient was treated wi th G-CSF for 18 months, until she received bone marrow transplantation , without presenting side effects. In the second responding patient G- CSF treatment was stopped because of appearance of immature cells in p eripheral blood and myeloid blasts in bone marrow. The third respondin g patient presented immature peripheral myeloid cells during the third year of G-CSF treatment: disappearance of immature cells was observed after G-CSF reduction. In two cases FISH analysis revealed monosomy 7 after G-CSF treatment. interpretation and Conclusions. G-CSF use resu lts in an improvement of clinical status, but long term administration may cause adverse experiences and requires a close hematological moni toring. (C)1998, Ferrata Storti Foundation.