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

Research output: Contribution to journalArticle

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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Antilymphocyte Serum
Aplastic Anemia
Myelodysplastic Syndromes
Granulocyte Colony-Stimulating Factor
Cyclosporine
Rabbits
Fever
Therapeutics
Safety

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

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

In: Leukemia, Vol. 23, No. 7, 01.01.2009, p. 1297-1302.

Research output: Contribution to journalArticle

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, vol. 23, no. 7, pp. 1297-1302. https://doi.org/10.1038/leu.2009.28
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. / Phase II study of rabbit anti-thymocyte globulin, cyclosporine and granulocyte colony-stimulating factor in patients with aplastic anemia and myelodysplastic syndrome. In: Leukemia. 2009 ; Vol. 23, No. 7. pp. 1297-1302.
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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.",
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T1 - Phase II study of rabbit anti-thymocyte globulin, cyclosporine and granulocyte colony-stimulating factor in patients with aplastic anemia and myelodysplastic syndrome

AU - Garg, R.

AU - Faderl, S.

AU - Garcia-Manero, G.

AU - Cortes, J.

AU - Koller, C.

AU - Huang, X.

AU - York, S.

AU - Pierce, S.

AU - Brandt, M.

AU - Beran, M.

AU - Borthakur, G.

AU - Kantarjian, H.

AU - Ravandi, F.

PY - 2009/1/1

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N2 - 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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