Erschienen in:
22.08.2016 | Original Article
Azacitidine-lenalidomide (ViLen) combination yields a high response rate in higher risk myelodysplastic syndromes (MDS)—ViLen-01 protocol
verfasst von:
Moshe Mittelman, Kalman Filanovsky, Yishai Ofran, Hanna Rosenbaum, Pia Raanani, Andrei Braester, Neta Goldschmidt, Ilya Kirgner, Yair Herishanu, Chava Perri, Martin Ellis, Howard S. Oster, for The Israel Myelodysplastic Syndrome Working Group (MDS-WG)
Erschienen in:
Annals of Hematology
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Ausgabe 11/2016
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Abstract
Azacitidine treatment is effective in higher risk MDS (HR-MDS), with less than 50 % response, lasting 2 years. Aza and lenalidomide (Len) have a potential synergistic effect. ViLen-01 phase IIa trial includes 6-month induction (Aza 75 mg/m2/day, days 1–5, Len 10 mg/day, days 6–21, every 28 days), 6-month consolidation (Aza 75 mg/m2/day, days 1–5, every 28 days), and 12-month maintenance (Len 10 mg/day, days 1–21, every 28 days). Response was evaluated according to IWG criteria. Totally, 25 patients enrolled, with an average of 76.3 years old (60–87), and 88 % with major comorbidities. Thirteen patients completed induction, 7 proceeded for consolidation, and 2 for maintenance. The overall response rate (ORR) was 72 % (18/25), with 6 (24 %) for CR, 3 (12 %) for marrow CR, and 9 (36 %) for hematologic improvement (HI). The 7 non-responding patients were on the study 3 days to 4.1 months. At 6 months, 4 of 6 evaluable patients achieved complete cytogenetic response and 2 with del (5q) at diagnosis. Adverse events (AEs) were as expected in these patients: grades III–IV, mainly hematologic—thrombocytopenia (20 patients) and neutropenia (13 patients). The common non-hematologic AEs were infections (14 patients), nausea (7), vomiting (7), diarrhea (7), and skin reactions (5). The median progression-free survival (PFS) was 12 ± 1.36 months, with median overall survival (OS) of 12 ± 1.7 months. Quality of life (FACT questionnaire) data were available for 12 patients with a tendency towards improved QoL. This trial with elderly HR-MDS patients with an expected poor prognosis demonstrates a high (72 %) response rate and a reasonable expected safety profile but a relatively short PFS and OS.