Premium
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
Author(s) -
Kadia Tapan M.,
Borthakur Gautam,
GarciaManero Guillermo,
Faderl Stefan,
Jabbour Elias,
Estrov Zeev,
York Sergerrne,
Huang Xuelin,
Pierce Sherry,
Brandt Mark,
Koller Charles,
Kantarjian Hagop M.,
Ravandi Farhad
Publication year - 2012
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/j.1365-2141.2012.09064.x
Subject(s) - medicine , ciclosporin , anti thymocyte globulin , myelodysplastic syndromes , gastroenterology , granulocyte colony stimulating factor , globulin , aplastic anemia , chemotherapy , surgery , bone marrow , immunology
Summary 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 ‐ DR 15 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.