Lenalidomide and dexamethasone in patients with POEMS syndrome: results of a prospective, open‐label trial

• DOI: 10.1111/bjh.14966
• Publication Date: 2017-10-19T10:42:38Z
• Authors: Andrea Nozza; Fabrizia Terenghi; Francesca Gallia; Fausto Adami; Chiara Briani; Giampaolo Merlini; Laura Giordano; Armando Santoro; Eduardo Nobile‐Or...
• Keywords: POEMS syndrome; Regimen; Clinical endpoint

Summary Given its anti‐angiogenic activity, lenalidomide may have a role in the treatment of POEMS ( P eripheral neuropathy, O rganomegaly, E ndocrinopathy, M onoclonal plasma cell disorder and S kin changes) syndrome. This prospective, open‐label, pilot study evaluated combination + dexamethasone RD ) 18 syndrome patients (13 pre‐treated, 5 newly‐diagnosed but ineligible for high‐dose therapy). Treatment consisted six cycles (25 mg/day 21 days followed by 7 rest) plus (40 mg/once week). Patients responding after continued until progression or unbearable toxicity. The primary endpoint was proportion with either neurological clinical improvement. considered as deserving further evaluation if 9 first 15 responded. Ten responses were observed among enrolled patients, meeting endpoint. Fifteen (83%) completed cycles: 13 (72%) responded nine had both Among who cycles, four still on 25‐month follow‐up. At 39 months follow‐up, all alive 3‐year progression‐free survival 59%. No patient discontinued Overall, regimen showed high incidence prolonged symptoms improvement well tolerated most patients.