Correction of neutropenia following treatment with granulocyte colony-stimulating factor results in a decreased frequency of infections in Shwachman's syndrome.

We report the clinical efficacy of granulocyte colony-stimulating factor (G-CSF) in a 17-year-old woman with Shwachman's syndrome who was repeatedly admitted with severe infections and neutropenia. Bone marrow culture showed no increase in granulocyte colony-forming units upon stimulation with granulocyte-macrophage colony-stimulating factor (GM-CSF). The patient was treated with G-CSF 300 micrograms 3 times a week subcutaneously. A complete and sustained response of the neutropenia was obtained without side-effects during 21 months of treatment. No infectious episodes were encountered after starting G-CSF. This observation demonstrates the beneficial effect of G-CSF in treating the severe neutropenia in patients with Shwachman's syndrome.