CYTOKINE TREATMENT OF INHERITED BONE-MARROW FAILURE SYNDROMES

The inherited bone marrow failure states are rare hematologic disorder s which result in peripheral blood cytopenias. To date, various treatm ent modalities have resulted in partial and usually short-lived respon ses. The recent large scale production of recombinant human hematopoie tic stimulating factors (cytokines) has offered a new possible treatme nt modality. Recent phase I-II studies indicate a significant but unpr edictable proportion of these patients will respond to cytokines with an improvement in blood counts. Multilineage responses in pancytopenic patients are rare. Duration of response, effect on natural history an d potential adverse effects of long-term treatment have yet to be dete rmined.