Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3

Alfred P. Gillio, Lawrence B. Faulkner, Blanche P. Alter, Laura Reilly, Robert Klafter, Glenn Heller, Diane C. Young, Jeffrey M. Lipton, Malcolm A S Moore, Richard J. O'Reilly

Research output: Contribution to journalArticlepeer-review

45 Scopus citations


This report describes the response of eighteen Diamond-Blackfan anemia (DBA) patients to recombinant human interleukin-3 (rhlL-3). rhlL-3 was administered subcutaneously once daily on an escalating dose schedule (0.5 to 10 Mg/kg/d). The rhlL-3 dose was escalated every 21 days until erythroid response was attained, grade III or IV nonhematologic toxicity was observed, or the maximum rhlL-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 rhlL-3 response. rhlL-3 administered at 5 to 10 μg/kg/d was associated with an increase in total white blood cell count, secondary to increases in neutrophils, eosinophils, and lymphocytes. Patients experienced a dose-dependent elevation in absolute eosinophils across the entire dose range. Two of the responding patients remain on maintenance rhlL-3, without diminution of effect at 244 and 370+ days. rhlL-3 was discontinued in the other two responders, because of the development of deep venous thrombi.

Original languageEnglish (US)
Pages (from-to)744-751
Number of pages8
Issue number3
StatePublished - Aug 1 1993
Externally publishedYes

ASJC Scopus subject areas

  • Hematology


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