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

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

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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.

Original languageEnglish (US)
Pages (from-to)1297-1302
Number of pages6
JournalLeukemia
Volume23
Issue number7
DOIs
StatePublished - Jan 1 2009
Externally publishedYes

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ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

Cite this

Garg, R., Faderl, S., Garcia-Manero, G., Cortes, J., Koller, C., Huang, X., York, S., Pierce, S., Brandt, M., Beran, M., Borthakur, G., Kantarjian, H., & Ravandi, F. (2009). Phase II study of rabbit anti-thymocyte globulin, cyclosporine and granulocyte colony-stimulating factor in patients with aplastic anemia and myelodysplastic syndrome. Leukemia, 23(7), 1297-1302. https://doi.org/10.1038/leu.2009.28