Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3

Alfred P. Gillio, Lawrence B. Faulkner, Blanche P. Alter, Laura Reilly, Robert Klafter, Glenn Heller, Diane C. Young, Jeffrey M. Lipton, Malcolm A S Moore, Richard J. O'Reilly

Research output: Contribution to journalArticle

Abstract

This report describes the response of eighteen Diamond-Blackfan anemia (DBA) patients to recombinant human interleukin-3 (rhlL-3). rhlL-3 was administered subcutaneously once daily on an escalating dose schedule (0.5 to 10 Mg/kg/d). The rhlL-3 dose was escalated every 21 days until erythroid response was attained, grade III or IV nonhematologic toxicity was observed, or the maximum rhlL-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 rhlL-3 response. rhlL-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 rhlL-3, without diminution of effect at 244 and 370+ days. rhlL-3 was discontinued in the other two responders, because of the development of deep venous thrombi.

Original languageEnglish (US)
Pages (from-to)744-751
Number of pages8
JournalBlood
Volume82
Issue number3
StatePublished - Aug 1 1993
Externally publishedYes

Fingerprint

Diamond-Blackfan Anemia
Diamond
Interleukin-3
Steroids
Lymphocytes
Eosinophils
Toxicity
Assays
Bone
Blood
Cells
Therapeutics
Leukocyte Count
Appointments and Schedules
Neutrophils
Thrombosis
Bone Marrow
Maintenance

ASJC Scopus subject areas

  • Hematology

Cite this

Gillio, A. P., Faulkner, L. B., Alter, B. P., Reilly, L., Klafter, R., Heller, G., ... O'Reilly, R. J. (1993). Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3. Blood, 82(3), 744-751.

Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3. / Gillio, Alfred P.; Faulkner, Lawrence B.; Alter, Blanche P.; Reilly, Laura; Klafter, Robert; Heller, Glenn; Young, Diane C.; Lipton, Jeffrey M.; Moore, Malcolm A S; O'Reilly, Richard J.

In: Blood, Vol. 82, No. 3, 01.08.1993, p. 744-751.

Research output: Contribution to journalArticle

Gillio, AP, Faulkner, LB, Alter, BP, Reilly, L, Klafter, R, Heller, G, Young, DC, Lipton, JM, Moore, MAS & O'Reilly, RJ 1993, 'Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3', Blood, vol. 82, no. 3, pp. 744-751.
Gillio AP, Faulkner LB, Alter BP, Reilly L, Klafter R, Heller G et al. Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3. Blood. 1993 Aug 1;82(3):744-751.
Gillio, Alfred P. ; Faulkner, Lawrence B. ; Alter, Blanche P. ; Reilly, Laura ; Klafter, Robert ; Heller, Glenn ; Young, Diane C. ; Lipton, Jeffrey M. ; Moore, Malcolm A S ; O'Reilly, Richard J. / Treatment of Diamond-Blackfan anemia with recombinant human interleukin-3. In: Blood. 1993 ; Vol. 82, No. 3. pp. 744-751.
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