TY - JOUR
T1 - Final results of the phase II study of rabbit anti-thymocyte globulin, ciclosporin, methylprednisone, and granulocyte colony-stimulating factor in patients with aplastic anaemia and myelodysplastic syndrome
AU - Kadia, Tapan M.
AU - Borthakur, Gautam
AU - Garcia-Manero, Guillermo
AU - Faderl, Stefan
AU - Jabbour, Elias
AU - Estrov, Zeev
AU - York, Sergerrne
AU - Huang, Xuelin
AU - Pierce, Sherry
AU - Brandt, Mark
AU - Koller, Charles
AU - Kantarjian, Hagop M.
AU - Ravandi, Farhad
PY - 2012/5
Y1 - 2012/5
N2 - This report describes the final results of a Phase II clinical trial investigating the efficacy of rabbit antithymocyte globulin (rATG), ciclosporin, steroids, and granulocyte colony-stimulating factor (GCSF) in patients with untreated aplastic anaemia (AA), or low to intermediate-risk and hypocellular myelodysplastic syndrome (MDS). We treated 24 patients each with AA and MDS with rATG (3·5 mg/kg/d × 5; reduced to 2·5 mg/kg/d × 5 in patients with MDS ≥55 years), ciclosporin (5 mg/kg orally daily × 6 months), steroids (1 mg/kg daily, tapered off over 1 month), and GCSF. The overall response rate in AA patients was 64% compared to 25% in MDS patients. The median time to response was 3 months in AA patients and 4 months in MDS patients. Pretreatment clinical characteristics, such as age, sex, blood counts, cellularity, cytogenetics, or HLA-DR15 status, did not predict for response. Response to therapy, however, predicted for improved overall survival (OS), with a 3-year OS of 89% vs. 43% in responders versus non-responders, respectively (P < 0·001). Infusion reactions occurred in about half the patients and were manageable. Myelosuppression, elevation in liver enzymes, and infections were common. The early mortality in MDS patients was 13% vs. 0% in AA patients.
AB - This report describes the final results of a Phase II clinical trial investigating the efficacy of rabbit antithymocyte globulin (rATG), ciclosporin, steroids, and granulocyte colony-stimulating factor (GCSF) in patients with untreated aplastic anaemia (AA), or low to intermediate-risk and hypocellular myelodysplastic syndrome (MDS). We treated 24 patients each with AA and MDS with rATG (3·5 mg/kg/d × 5; reduced to 2·5 mg/kg/d × 5 in patients with MDS ≥55 years), ciclosporin (5 mg/kg orally daily × 6 months), steroids (1 mg/kg daily, tapered off over 1 month), and GCSF. The overall response rate in AA patients was 64% compared to 25% in MDS patients. The median time to response was 3 months in AA patients and 4 months in MDS patients. Pretreatment clinical characteristics, such as age, sex, blood counts, cellularity, cytogenetics, or HLA-DR15 status, did not predict for response. Response to therapy, however, predicted for improved overall survival (OS), with a 3-year OS of 89% vs. 43% in responders versus non-responders, respectively (P < 0·001). Infusion reactions occurred in about half the patients and were manageable. Myelosuppression, elevation in liver enzymes, and infections were common. The early mortality in MDS patients was 13% vs. 0% in AA patients.
KW - Hypoplastic
KW - Immunotherapy
KW - Thymoglobulin
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U2 - 10.1111/j.1365-2141.2012.09064.x
DO - 10.1111/j.1365-2141.2012.09064.x
M3 - Article
C2 - 22360602
AN - SCOPUS:84859508819
SN - 0007-1048
VL - 157
SP - 312
EP - 320
JO - British Journal of Haematology
JF - British Journal of Haematology
IS - 3
ER -