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Alemtuzumab is safe and effective as immunosuppressive treatment for aplastic anaemia and single-lineage marrow failure : a pilot study and a survey from the EBMT WPSAA
JournalArticle (Originalarbeit in einer wissenschaftlichen Zeitschrift)
ID
1193571
Author(s)
Risitano, Antonio Maria; Selleri, Carmine; Serio, Bianca; Torelli, Giovanni Fernando; Kulagin, Alexander; Maury, Sébastien; Halter, Jörg; Gupta, Vikas; Bacigalupo, Andrea; Sociè, Gerard; Tichelli, André; Schrezenmeier, Hubert; Marsh, Judith; Passweg, Jakob; Rotoli, Bruno; Working Party Severe Aplastic Anaemia (WPSAA) of the European Group for Blood and Marrow Transplantation (EBMT)
Alemtuzumab is safe and effective as immunosuppressive treatment for aplastic anaemia and single-lineage marrow failure : a pilot study and a survey from the EBMT WPSAA
Journal
British journal of haematology
Volume
148
Number
5
Pages / Article-Number
791-6
Keywords
alemtuzumab, aplastic anaemia, pure red cell aplasia, pure white cell aplasia, bone marrow failure
Abstract
An alemtuzumab-based experimental immunosuppressive treatment (IST) regimen was investigated in 35 patients with severe aplastic anaemia (SAA), pure red cell (PRCA) or pure white cell aplasia (PWCA). Alemtuzumab total dose was 73-103 mg s.c., followed by cyclosporine. No serious toxicity due to the regimen was observed. Adverse events were clinically irrelevant; infectious events were rare. The total response rate was 58%, 84% and 100% in SAA, PRCA and PWCA, respectively, with corresponding 6 months cumulative response probabilities of 84%, 84% and 100%. Subcutaneous alemtuzumab is a feasible and sufficiently safe IST regimen for patients suffering from immune-mediated marrow failures.