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Soluble TNF receptor fusion protein (etanercept) for the treatment of myelodysplastic syndrome: A pilot study

Abstract

Blockade of tumor necrosis factor (TNF)α by a soluble TNF receptor fusion protein (etanercept; Enbrel) improved in vitro hemopoiesis from the marrow of patients with myelodysplastic syndrome (MDS). Therefore, we enrolled 14 MDS patients (4 RA, 2 RARS, 6 RAEB, 2 CMML), 44–80 (median 60) years old, in a pilot trial. Etanercept, 25 mg, was given twice a week s.c. for 16 weeks (increased to three times a week if no response at 8 weeks). Among 12 evaluable patients, four had rises in hemoglobin by 1–1.5 gm/dl (three) or decreased transfusion requirements (one). Two patients had increased platelet counts (54% and 73%), and two increased neutrophils (63% and 120%). Baseline TNFα levels, determined in all patients, did not correlate with responses. Among eight marrows available for sequential in vitro assays, four showed increases in CFU-GM of 1.5- to 5-fold at 8 weeks, whereas three showed 3- to 10-fold decrements relative to baseline. Thus, etanercept treatment resulted in moderate improvements of cytopenias in some patients, while cell counts declined in others. Additional trials are needed to evaluate its clinical efficacy in MDS.

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Acknowledgements

We thank all referring physicians, and physicians and staff caring for these patients, for their contributions. We appreciate the administrative help of Medo Schlemmer and Monina Almeda. We thank B Larson and H Crawford for typing the manuscript. This work was supported in part by grants CA87948 from the National Cancer Institute, and HL36444 from the National Heart Lung and Blood Institute, NIH, DHHS, Bethesda, MD, and by a grant-in-aid from Immunex Corp. HJD also received support from the Gabrielle Rich Leukemia Fund.

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Deeg, H., Gotlib, J., Beckham, C. et al. Soluble TNF receptor fusion protein (etanercept) for the treatment of myelodysplastic syndrome: A pilot study. Leukemia 16, 162–164 (2002). https://doi.org/10.1038/sj.leu.2402356

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