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

Overview

Journal Br J Haematol

Specialty Hematology

Date 2012 Feb 25

PMID 22360602

Citations 14

Authors

Tapan M Kadia

Gautam Borthakur

Guillermo Garcia-Manero

Stefan Faderl

Elias Jabbour

Zeev Estrov

Sergerrne York

Xuelin Huang

Sherry Pierce

Mark Brandt

Charles Koller

Hagop M Kantarjian

Farhad Ravandi

Affiliations

Soon will be listed here.

Abstract

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.

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