Decreased survival and increased rate of fibrotic progression in essential thrombocythemia chronicled after the FDA approval date of anagrelide

• Published in: American journal of hematology 2019-01, Vol.94 (1), p.5-9
• Main Authors: Tefferi, Ayalew, Szuber, Natasha, Vallapureddy, Rangit R., Begna, Kebede H., Patnaik, Mrinal M., Elliott, Michelle A., Christopher Hook, C., Wolanskyj, Alexandra P., Hanson, Curtis A., Ketterling, Rhett P., Pardanani, Animesh, Gangat, Naseema
• Format: Article
• Language: English
• Subjects: Busulfan; Clinical trials; FDA approval; Hematology; Hydroxyurea; Interferon; Myelofibrosis; Polycythemia; Polycythemia vera; Survival; Thrombosis
• ISSN: 0361-8609; 1096-8652
• DOI: 10.1002/ajh.25294

First‐line cytoreductive drug of choice in high risk essential thrombocythemia (ET) is currently hydroxyurea, a practice based on the results of a randomized study; second‐line drugs of choice include pegylated interferon‐α, busulfan and anagrelide. Anagrelide clinical trials were pioneered by the late Murray N. Silverstein (1928‐1998) of the Mayo Clinic whose studies led to FDA approval in March 1997. The current study represents a retrospective examination of the potential impact of anagrelide therapy on survival and disease complications in ET. 1076 patients with ET were considered (median age 58 years; females 63%); risk distribution, according to the international prognostic score for ET (IPSET), was 28% high, 42% intermediate, and 30% low. Overall (OS), myelofibrosis‐free (MFFS) and thrombosis‐free survival data were compared for ET patients diagnosed before and after the 1997 FDA approval date for anagrelide; a significant difference was apparent in OS (P = .006; HR 1.4, 95% CI 1.1‐1.7) and MFFS (P