Successful treatment of Diamond‐Blackfan anemia with interleukin 3 Conference Paper
| 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. |
| Title: | Successful treatment of Diamond‐Blackfan anemia with interleukin 3 |
| Conference Title: | Stem Cell Factor and Related Cytokines in Bone Marrow Congenital Dysplasias |
| 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 m̈g/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. Copyright © 1993 AlphaMed Press |
| Keywords: | child; clinical article; controlled study; treatment outcome; prednisone; dose response; conference paper; animal; mice; stem cell factor; erythropoietin; erythropoiesis; gastrointestinal symptom; hemoglobin; steroid; arthralgia; chill; fever; hypotension; mononuclear cell; recombinant proteins; thrombocyte count; hematopoiesis; headache; blood cell count; leukocyte count; erythrocyte transfusion; growth factor; irritability; reticulocyte count; fanconi anemia; recombinant interleukin 3; erythroid progenitor cells; eosinophilia; biological response modifiers; interleukin 3; interleukin-3; thrombophlebitis; colony stimulating factor; burst forming unit e; subcutaneous drug administration; human; male; female; leukocyte differential count; blackfan diamond anemia; support, non-u.s. gov't; support, u.s. gov't, p.h.s.; hematopoietic cell growth factors; pure red cell aplasia; diamond‐blackfan anemia |
| Journal Title | Stem Cells |
| Volume: | 11 |
| Issue: | Suppl. 2 |
| Conference Dates: | 1993 Mar 8-9 |
| Conference Location: | Cattolica, Italy |
| ISBN: | 1066-5099 |
| Publisher: | AlphaMed Press |
| Date Published: | 1993-07-01 |
| Start Page: | 123 |
| End Page: | 130 |
| Language: | English |
| DOI: | 10.1002/stem.5530110820 |
| PUBMED: | 7691318 |
| PROVIDER: | scopus |
| DOI/URL: | |
| Notes: | Article -- Export Date: 1 March 2019 -- Source: Scopus |
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