Successful treatment of Diamond-Blackfan anemia with interleukin 3.
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.
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
D000818:Animals; D001772:Blood Cell Count / Q000187:drug effects; D002648:Child; D004802:Eosinophilia / Q000139:chemically induced; D015672:Erythroid Precursor Cells / Q000187:drug effects; D004920:Erythropoiesis / Q000187:drug effects; D004921:Erythropoietin / Q000494:pharmacology; D005199:Fanconi Anemia / Q000628:therapy*; D005260:Female; D016298:Hematopoietic Cell Growth Factors / Q000494:pharmacology; D006801:Humans; D007022:Hypotension / Q000139:chemically induced; D007155:Immunologic Factors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D007377:Interleukin-3 / Q000008:administration & dosage / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D008297:Male; D051379:Mice; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use; D019089:Stem Cell Factor; D013924:Thrombophlebitis / Q000139:chemically induced; D016896:Treatment Outcome
D016298:Hematopoietic Cell Growth Factors; D007155:Immunologic Factors; D007377:Interleukin-3; D011994:Recombinant Proteins; D019089:Stem Cell Factor; D004921:Erythropoietin