title: Successful treatment of Diamond-Blackfan anemia with interleukin 3.
abstract: 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.
authors.affiliation: Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, NY 10021.
authors.forename: A P
authors.initials: AP
authors.lastname: Gillio
authors.forename: L B
authors.initials: LB
authors.lastname: Faulkner
authors.forename: B P
authors.initials: BP
authors.lastname: Alter
authors.forename: L
authors.initials: L
authors.lastname: Reilly
authors.forename: R
authors.initials: R
authors.lastname: Klafter
authors.forename: G
authors.initials: G
authors.lastname: Heller
authors.forename: D C
authors.initials: DC
authors.lastname: Young
authors.forename: J M
authors.initials: JM
authors.lastname: Lipton
authors.forename: M A
authors.initials: MA
authors.lastname: Moore
authors.forename: R J
authors.initials: RJ
authors.lastname: O'Reilly
mesh_terms: 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
chemical_list: D016298:Hematopoietic Cell Growth Factors; D007155:Immunologic Factors; D007377:Interleukin-3; D011994:Recombinant Proteins; D019089:Stem Cell Factor; D004921:Erythropoietin