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Title: Successful treatment of Diamond-Blackfan anemia with interleukin 3.
Authors: Gillio AP,  Faulkner LB,  Alter BP,  Reilly L,  Klafter R,  Heller G,  Young DC,  Lipton JM,  Moore MA,  O'Reilly RJ
Journal: Stem Cells
Date: 1993 Jul
Branches: CGB
PubMed ID: 7691318
PMC ID: not available
Abstract: This report describes the response of 18 Diamond-Blackfan anemia (DBA) patients to recombinant human interleukin 3 (rhIL-3). rhIL-3 was administered s.c. once daily on an escalating dose schedule (0.5-10 micrograms/kg/day). The rhIL-3 dose was escalated every 21 days until erythroid response was attained, grade III or IV nonhematologic toxicity was observed, or the maximal rhIL-3 dose was reached. Four patients experienced clinically significant erythroid responses. Two of the responders were steroid-dependent and transfusion-independent, while two were steroid-independent and transfusion-dependent. Baseline clinical or laboratory parameters, in particular in vitro bone marrow erythroid progenitor assays, were not useful in predicting rhIL-3 response. Two of the responding patients remain on maintenance rhIL-3 without diminution of effect at 490 and 855+ days. rhIL-3 was discontinued in the other two responders because of the development of deep venous thrombi.