title: Treatment of Diamond-Blackfan anaemia with haematopoietic growth factors, granulocyte-macrophage colony stimulating factor and interleukin 3: sustained remissions following IL-3.
abstract: We have treated six transfusion-dependent, steroid-unresponsive, Diamond-Blackfan anaemia (DBA) patients with the recombinant human growth factors granulocyte-macrophage colony-stimulating factor (GM-CSF) and interleukin-3 (IL-3), administered sequentially with an interim rest period. GM-CSF was given at a dose of 500 micrograms/m2/d subcutaneously for 6 weeks. Three patients increased their absolute reticulocyte counts 1.5-35-fold (mean 20.8-fold) and into the normal range, but only one showed a reduction in transfusion requirements. Between 4 and 25 weeks after discontinuation of GM-CSF, these six patients were treated with recombinant human IL-3, at doses of 60 or 125 micrograms/m2/d subcutaneously for 4-6 weeks. Three increased their absolute reticulocyte counts from 2- to 28-fold (mean 10.6-fold) and two required fewer transfusions. One of these two patients has remained transfusion independent for over a year since completion of IL-3 therapy, and the second patient required infrequent transfusions for 9 months and then became transfusion independent for the subsequent 5 months. The sustained clinical remissions seen in two of the six patients after IL-3 therapy is very encouraging and further studies in a larger cohort of DBA patients with IL-3 alone or in combination with GM-CSF or other growth factors should be carried out.
authors.affiliation: Clinical Hematology Branch, National Heart, Lung and Blood Institute, NIH, Bethesda, MD 20892.
authors.forename: C E
authors.initials: CE
authors.lastname: Dunbar
authors.forename: D A
authors.initials: DA
authors.lastname: Smith
authors.forename: J
authors.initials: J
authors.lastname: Kimball
authors.forename: L
authors.initials: L
authors.lastname: Garrison
authors.forename: A W
authors.initials: AW
authors.lastname: Nienhuis
authors.forename: N S
authors.initials: NS
authors.lastname: Young
mesh_terms: D000293:Adolescent; D000328:Adult; D001772:Blood Cell Count; D001853:Bone Marrow / Q000473:pathology; D002648:Child; D005199:Fanconi Anemia / Q000097:blood / Q000473:pathology / Q000628:therapy*; D005260:Female; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D007377:Interleukin-3 / Q000627:therapeutic use*; D008297:Male; D011994:Recombinant Proteins / Q000627:therapeutic use*; D012074:Remission Induction; D013997:Time Factors
chemical_list: D007377:Interleukin-3; D011994:Recombinant Proteins; D016178:Granulocyte-Macrophage Colony-Stimulating Factor