Risk factors for and outcomes of patients with POEMS syndrome who experience progression after first-line treatment

• Published in: Leukemia 2016-05, Vol.30 (5), p.1079-1085
• Main Authors: Kourelis, T V, Buadi, F K, Gertz, M A, Lacy, M Q, Kumar, S K, Kapoor, P, Go, R S, Lust, J A, Hayman, S R, Rajkumar, V, Zeldenrust, S R, Russell, S J, Dingli, D, Lin, Y, Leung, N, Hwa, Y L, Gonsalves, W, Kyle, R A, Dispenzieri, A
• Format: Article
• Language: English
• Subjects: 692/420; 692/499; 692/699/1541; 692/699/1541/1990/804; 692/700/565/1436/99; Adult; Aged; Aged, 80 and over; Albumins; Ascites; Cancer Research; Chemotherapy; Combined Modality Therapy; Critical Care Medicine; Diagnosis; Disease Management; Disease Progression; Disease-Free Survival; Endocrine disorders; Failure analysis; Female; Forecasts and trends; Growth factors; Health aspects; Health services; Hematology; Humans; Intensive; Internal Medicine; Leukemia; Lymphoproliferative disorders; Male; Medicine; Medicine & Public Health; Middle Aged; Mortality; Oncology; original-article; Patient outcomes; Patients; Peripheral neuropathy; Plasma; POEMS Syndrome - diagnosis; POEMS Syndrome - mortality; POEMS Syndrome - pathology; POEMS Syndrome - therapy; Polyneuropathy; Proteins; Quality of life; Radiation therapy; Recurrence; Retrospective Studies; Risk analysis; Risk Factors; Salvage Therapy - methods; Signs and symptoms; Survival; Survival Rate; Treatment Outcome; Vascular endothelial growth factor; Vascular Endothelial Growth Factor A - blood; Young Adult
• ISSN: 0887-6924; 1476-5551
• DOI: 10.1038/leu.2015.344

Although clinical improvement is almost universal with therapy in patients with POEMS (an acronym for polyneuropathy, organomegaly, endocrinopathies, monoclonal protein and a variety of skin changes) syndrome, outcomes and management of patients who relapse or progress (R/P) after first-line treatment have not been described. We retrospectively identified 262 patients with POEMS syndrome treated at the Mayo Clinic from 1974 to 2014 and who had follow-up information. The 5-year progression-free survival (PFS) and overall survival (OS) was 58% and 78%, respectively. Median time to R/P was 42 months. Seventy-nine patients (30%) had an R/P, with 52 (19%) experiencing a symptomatic R/P. Eighteen patients relapsed with symptoms or signs that were not documented at diagnosis. Median times to vascular endothelial growth factor, hematologic, radiographic and clinical R/P were 35 months (range, 4–327 months), 72 months (range, 4–327 months), 51 months (range, 4–327 months) and 48 months (range, 6–311 months), respectively. On multivariate analyses, low albumin at diagnosis and failure to achieve a complete hematologic response to first-line therapy were independent risk factors for PFS. Thirty patients had documentation of a second R/P at a median of 26 months from diagnosis of the first R/P. An early R/P was a risk factor for death, but most patients with an R/P had salvageable disease. A majority of patients are still without R/P at 5 years from diagnosis.