Tomoki Suichi1, Sonoko Misawa1, Minako Beppu1, Sho Takahashi1, Yukari Sekiguchi1, Kazumoto Shibuya1, Hiroshi Amino1, Atsuko Tsuneyama1, Yo-Ichi Suzuki1, Keigo Nakamura1, Yasunori Sato1, Satoshi Kuwabara2. 1. From the Department of Neurology (T.S., S.M., M.B., Y. Sekiguchi, K.S., H.A., A.T., Y.-i.S., K.N., S.K.), Graduate School of Medicine, Chiba University; Clinical Research Support Center (S.T.), Jikei University School of Medicine; and Department of Preventive Medicine and Public Health (Y. Sato), School of Medicine, Keio University, Tokyo, Japan. 2. From the Department of Neurology (T.S., S.M., M.B., Y. Sekiguchi, K.S., H.A., A.T., Y.-i.S., K.N., S.K.), Graduate School of Medicine, Chiba University; Clinical Research Support Center (S.T.), Jikei University School of Medicine; and Department of Preventive Medicine and Public Health (Y. Sato), School of Medicine, Keio University, Tokyo, Japan. kuwabara-s@faculty.chiba-u.jp.
Abstract
OBJECTIVE: To elucidate current epidemiological, clinical profiles, and treatment of polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes (POEMS) syndrome. METHODS: We conducted a nationwide survey in 2015 using an established epidemiologic method. Data processing sheets were sent to all neurology and hematology specialist departments throughout Japan to identify patients with POEMS who were seen between April 2012 and March 2015. RESULTS: The estimated number of patients with POEMS was 392 (95% confidence interval [CI] 320-464), and the prevalence was 0.3 per 100,000. Detailed clinical profiles were available for 167 patients. Median age at onset was 54 years (range, 21-84 years), and the ratio of male to female was 1.5. All patients showed polyneuropathy; 89% had monoclonal plasma cell proliferation; and 84% had elevated vascular endothelial growth factor level in whom pretreatment serum or plasma was available (n = 87). Other common features were skin changes (84%), edema/effusion (81%), and organomegaly (76%). A total of 160 patients were treated with any of the following: radiation, corticosteroids, melphalan, thalidomide, lenalidomide, bortezomib, or autologous stem cell transplantation. Primary therapeutic options were thalidomide (n = 86) and autologous stem cell transplantation (n = 71). Thirty-nine patients (24%) were initially treated with corticosteroid alone. The 10-year overall survival was 93% (95% CI 86%-96%). DISCUSSION: This study showed current epidemiologic and clinical status of POEMS syndrome in Japan. A quarter of patients were still inadequately treated with corticosteroid alone, whereas either autologous stem cell transplantation or immunomodulatory drugs improved the prognosis.
OBJECTIVE: To elucidate current epidemiological, clinical profiles, and treatment of polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes (POEMS) syndrome. METHODS: We conducted a nationwide survey in 2015 using an established epidemiologic method. Data processing sheets were sent to all neurology and hematology specialist departments throughout Japan to identify patients with POEMS who were seen between April 2012 and March 2015. RESULTS: The estimated number of patients with POEMS was 392 (95% confidence interval [CI] 320-464), and the prevalence was 0.3 per 100,000. Detailed clinical profiles were available for 167 patients. Median age at onset was 54 years (range, 21-84 years), and the ratio of male to female was 1.5. All patients showed polyneuropathy; 89% had monoclonal plasma cell proliferation; and 84% had elevated vascular endothelial growth factor level in whom pretreatment serum or plasma was available (n = 87). Other common features were skin changes (84%), edema/effusion (81%), and organomegaly (76%). A total of 160 patients were treated with any of the following: radiation, corticosteroids, melphalan, thalidomide, lenalidomide, bortezomib, or autologous stem cell transplantation. Primary therapeutic options were thalidomide (n = 86) and autologous stem cell transplantation (n = 71). Thirty-nine patients (24%) were initially treated with corticosteroid alone. The 10-year overall survival was 93% (95% CI 86%-96%). DISCUSSION: This study showed current epidemiologic and clinical status of POEMS syndrome in Japan. A quarter of patients were still inadequately treated with corticosteroid alone, whereas either autologous stem cell transplantation or immunomodulatory drugs improved the prognosis.
Authors: Hana Mahallati; James Kirkland Roberts; Amer Assal; Divaya Bhutani; David C Park; Golnaz Moazami Journal: Am J Ophthalmol Case Rep Date: 2021-03-16