Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3.
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.
Gillio
A P
AP
Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, NY 10021.
Faulkner
L B
LB
Alter
B P
BP
Reilly
L
L
Klafter
R
R
Heller
G
G
Young
D C
DC
Lipton
J M
JM
Moore
M A
MA
O'Reilly
R J
RJ
D000293:Adolescent; D000328:Adult; D001803:Blood Transfusion; D002648:Child; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D004920:Erythropoiesis / Q000187:drug effects; D005199:Fanconi Anemia / Q000188:drug therapy*; D005260:Female; D006801:Humans; D007377:Interleukin-3 / Q000009:adverse effects / Q000627:therapeutic use*; D007958:Leukocyte Count; D008297:Male; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use
D007377:Interleukin-3; D011994:Recombinant Proteins