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Myelodysplasias

Phase II study of rabbit anti-thymocyte globulin, cyclosporine and granulocyte colony-stimulating factor in patients with aplastic anemia and myelodysplastic syndrome

Leukemia volume 23, pages 1297–1302 (2009)Cite this article

Abstract

We investigated efficacy and safety of rabbit anti-thymocyte globulin (rATG), cyclosporine and granulocyte colony-stimulating factor (G-CSF) as first-line therapy for patients with aplastic anemia (AA) and low or intermediate-1 or hypoplastic myelodysplastic syndrome (MDS). rATG 3.5 mg/kg (or 2.5 mg/kg per day for patients ⩾55 years with MDS) was given for 5 days. Cyclosporine (5 mg/kg) and G-CSF (5 μg/kg) were given daily and continued for up to 6 months or longer. Responses were assessed about 3 and 6 months after therapy. Thirty-six patients have been enrolled on study and 32 patients treated; 25 were evaluable for a response (13 with AA, 12 with MDS); the rest are too early. The median age was 62 years (range, 20–83) for patients with AA and 63 (range, 42–80) for patients with MDS. Of 13 patients, 12 (92%) patients with AA responded (5 complete response (CR), 7 partial response (PR)), whereas of 12 patients, 4 (33%) patients with MDS responded (1 CR, 3 PR). For patients with AA, the median time to response (TTR) was 93 days (range, 79–623), whereas in the MDS group the median TTR was 111 days (range, 77–139). Grade III/IV toxicities were mainly cytopenias and neutropenic fever. Combination of rATG, cyclosporine and G-CSF is safe and effective as first-line treatment of AA and has significant activity in low-risk MDS.

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Acknowledgements

This study was supported in part by a grant from the Genzyme Pharmaceutical company.

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Authors and Affiliations

  1. Department of Leukemia, The University of Texas, MD Anderson Cancer Center, Houston, TX, USA

    R Garg, S Faderl, G Garcia-Manero, J Cortes, C Koller, S York, S Pierce, M Brandt, M Beran, G Borthakur, H Kantarjian & F Ravandi

  2. Department of Biostatistics, The University of Texas, MD Anderson Cancer Center, Houston, TX, USA

    X Huang

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Correspondence to F Ravandi.

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Garg, R., Faderl, S., Garcia-Manero, G. et al. Phase II study of rabbit anti-thymocyte globulin, cyclosporine and granulocyte colony-stimulating factor in patients with aplastic anemia and myelodysplastic syndrome. Leukemia 23, 1297–1302 (2009). https://doi.org/10.1038/leu.2009.28

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