Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3 Journal Article
| Authors: | Gillio, A. P.; Faulkner, L. B.; Alter, B. P.; Reilly, L.; Klafter, R.; Heller, G.; Young, D. C.; Lipton, J. M.; Moore, M. A. S.; O'Reilly, R. J. |
| Article 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 μg/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 μg/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. |
| Keywords: | adolescent; adult; child; clinical article; child, preschool; paresthesia; dose response; erythropoietin; erythropoiesis; gastrointestinal symptom; myalgia; granulocyte macrophage colony stimulating factor; hemoglobin; immunoglobulin; steroid; bone pain; deep vein thrombosis; dose-response relationship, drug; arthralgia; chill; fever; pruritus; hypotension; recombinant proteins; blood transfusion; erythema; headache; leukocyte count; cyclosporin a; injection site; ferritin; fanconi anemia; recombinant interleukin 3; hemoglobin f; interleukin-3; hematological parameters; subcutaneous drug administration; human; male; female; priority journal; article; blackfan diamond anemia; support, non-u.s. gov't; support, u.s. gov't, p.h.s. |
| Journal Title: | Blood |
| Volume: | 82 |
| Issue: | 3 |
| ISSN: | 0006-4971 |
| Publisher: | American Society of Hematology |
| Date Published: | 1993-08-01 |
| Start Page: | 744 |
| End Page: | 751 |
| Language: | English |
| PUBMED: | 8338944 |
| PROVIDER: | scopus |
| DOI: | 10.1182/blood.V82.3.744.744 |
| DOI/URL: | |
| Notes: | Source: Scopus |
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