GM-CSF clinical trials: pediatric aplastic anemia and Fanconi's anemia.

Two clinical trials were undertaken to evaluate the effect of human recombinant granulocyte-macrophage colony stimulating factor (GM-CSF) in pancytopenic pediatric patients with aplastic anemia and Fanconi's anemia. In the aplastic anemia trial, 9 out of 12 patients had some improvement when treated with GM-CSF. In the Fanconi's anemia trial, 6 of 7 patients showed some improvement when treated with GM-CSF. For both groups, improvement in white blood cell count and absolute neutrophil count were the most common response. Side effects observed during these studies were fever, rash, urticaria, and flu-like symptoms. Nursing care of both groups focused on the effects of pancytopenia, as well as the potential adverse effects of GM-CSF. Patient education focused on teaching drug preparation and storage, subcutaneous injection, and potential side effects.