title: Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3.
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.
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: 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
chemical_list: D007377:Interleukin-3; D011994:Recombinant Proteins