What is the best first-line treatment for POEMS syndrome: autologous transplantation, melphalan and dexamethasone, or lenalidomide and dexamethasone?

• Published in: Leukemia 2019-04, Vol.33 (4), p.1023-1029
• Main Authors: Zhao, Hao, Huang, Xu-fei, Gao, Xue-min, Cai, Hao, Zhang, Lu, Feng, Jun, Cao, Xin-xin, Zhou, Dao-bin, Li, Jian
• Format: Article
• Language: English
• Subjects: 13/21; 692/699/67/1990/804; 692/700/565/1436/1437; 82/1; 82/29; Adult; Aged; Antineoplastic Combined Chemotherapy Protocols - therapeutic use; Antiretroviral drugs; Autografts; Cancer Research; Combined Modality Therapy; Critical Care Medicine; Dexamethasone; Dexamethasone - administration & dosage; Female; Follow-Up Studies; Growth factors; Hematology; Hematopoietic Stem Cell Transplantation - mortality; Humans; Immunotherapy; Intensive; Internal Medicine; Lenalidomide - administration & dosage; Male; Medicine; Medicine & Public Health; Melphalan; Melphalan - administration & dosage; Middle Aged; Oncology; Patients; POEMS Syndrome - mortality; POEMS Syndrome - pathology; POEMS Syndrome - therapy; Remission; Remission Induction; Retrospective Studies; Stem cell transplantation; Stem cells; Steroids; Survival; Survival Rate; Targeted cancer therapy; Transplantation; Transplantation Conditioning; Transplantation, Autologous; Treatment Outcome; Vascular endothelial growth factor
• ISSN: 0887-6924; 1476-5551
• DOI: 10.1038/s41375-019-0391-2

POEMS syndrome is a rare plasma cell dyscrasia. This study compared the responses to and survival of 347 POEMS syndrome patients given three first-line treatment regimens: autologous stem cell transplantation (ASCT, N  = 165) and melphalan + dexamethasone (MDex, N  = 79), or lenalidomide + dexamethasone (LDex, N  = 103). After a median 45-month follow-up, overall hematologic complete remission (CR H ) was 46.4%, vascular endothelial growth factor complete remission (CR V ) was 55.1%, and neurological remission (R N ) was 93.8%. CR H was better with ASCT (49.7%) than with MDex (37.7%, p  = 0.001). CR V was better with ASCT (66.2%) than with MDex (38.5%, p  = 0.001) or LDex (47.7%, p  = 0.008). Differences in R N achieved by three regimens (91.5% vs. 100% vs. 93.8%, p  = 0.234) were not significant. Overall 3-year progression-free survival (PFS) was 80.5% and overall 3-year overall survival (OS) was 90.8%. PFS was 87.6% with ASCT and 64.9% with LDex ( p  = 0.003). OS in the three regimens did not differ ( p  = 0.079). In medium-high risk patients, ASCT had better CR H and CR V than MDex, and better PFS than LDex. Therefore, although all three treatments had reasonable responses and survivals, patients with higher risk may benefit more from ASCT treatment.