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.
Publication types
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Clinical Trial
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Research Support, Non-U.S. Gov't
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Research Support, U.S. Gov't, P.H.S.
MeSH terms
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Animals
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Blood Cell Count / drug effects
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Child
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Eosinophilia / chemically induced
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Erythroid Precursor Cells / drug effects
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Erythropoiesis / drug effects
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Erythropoietin / pharmacology
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Fanconi Anemia / therapy*
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Female
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Hematopoietic Cell Growth Factors / pharmacology
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Humans
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Hypotension / chemically induced
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Immunologic Factors / administration & dosage
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Immunologic Factors / adverse effects
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Immunologic Factors / therapeutic use*
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Interleukin-3 / administration & dosage
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Interleukin-3 / adverse effects
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Interleukin-3 / pharmacology
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Interleukin-3 / therapeutic use*
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Male
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Mice
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Recombinant Proteins / administration & dosage
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Recombinant Proteins / adverse effects
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Recombinant Proteins / pharmacology
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Recombinant Proteins / therapeutic use
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Stem Cell Factor
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Thrombophlebitis / chemically induced
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Treatment Outcome
Substances
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Hematopoietic Cell Growth Factors
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Immunologic Factors
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Interleukin-3
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Recombinant Proteins
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Stem Cell Factor
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Erythropoietin