Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3 - PubMed (original) (raw)
Clinical Trial
. 1993 Aug 1;82(3):744-51.
Affiliations
- PMID: 8338944
Free article
Clinical Trial
Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3
A P Gillio et al. Blood. 1993.
Free article
Abstract
This report describes the response of eighteen Diamond-Blackfan anemia (DBA) patients to recombinant human interleukin-3 (rhIL-3). rhIL-3 was administered subcutaneously once daily on an escalating dose schedule (0.5 to 10 micrograms/kg/d). The rhIL-3 dose was escalated every 21 days until erythroid response was attained, grade III or IV nonhematologic toxicity was observed, or the maximum 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. rhIL-3 administered at 5 to 10 micrograms/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 rhIL-3, without diminution of effect at 244 and 370 + days. rhIL-3 was discontinued in the other two responders, because of the development of deep venous thrombi.
Comment in
- Sustained response after recombinant interleukin-3 in diamond blackfan anemia.
Bastion Y, Bordigoni P, Debré M, Girault D, Leblanc T, Tchernia G, Ball S, McGuckin C, Gordon-Smith EC, Békassy A, et al. Bastion Y, et al. Blood. 1994 Jan 15;83(2):617-8. Blood. 1994. PMID: 8286756 No abstract available.