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Treatment of Diamond-Blackfan anemia with recombinant human interleukin- 3
[see comments]
AP Gillio, LB Faulkner, BP Alter, L Reilly, R Klafter, G Heller, DC Young, JM Lipton, MA Moore and RJ O'Reilly
Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York,
NY 10021.
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.
Volume 82,
Issue 3,
pp. 744-751,
08/01/1993
Copyright © 1993 by The American Society of Hematology
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