HUMAN GRANULOCYTE-COLONY-STIMULATING FACTOR (RHUG-CSF) FOR TREATMENT OF NEUTROPENIA IN SHWACHMAN SYNDROME

We report a boy affected by Shwachman syndrome (SS) who presented seve re neutropenia and frequent suppurative infections, which we treated s uccessfully with granulocyte colony-stimulating factor (rHuG-CSF). Dai ly injections of 7.5 mu g/kg/day significantly increased the absolute neutrophil count and he was free from infections. In order to avoid th e risk of side effects and to improve the child's quality of life, we intermittently administered lower doses of rHuG-CSF. A weekly dose of 2 mu g/kg/day was able to maintain the absolute neutrophil count high enough (0.58 - 1.2 x 10(9)/L) to prevent suppurative infections. Durin g the follow-up period (2.5 years) we tried suspending rHuG-CSF twice, but the absolute neutrophil count (0.18 - 0.31 x 10(9)/L) fell signif icantly and suppurative infections reoccurred (otitis, perianal absces s). rHuG-CSF may be a useful therapeutic agent in patients with sympto matic neutropenia in SS.