RT Journal Article
SR Electronic
T1 Clinical characteristics, risk factors, and outcomes of POEMS syndrome
JF Neurology
JO Neurology
FD Lippincott Williams & Wilkins
SP e268
OP e279
DO 10.1212/WNL.0000000000009940
VO 95
IS 3
A1 Keddie, Stephen
A1 Foldes, David
A1 Caimari, Francisca
A1 Baldeweg, Stephanie E.
A1 Bomsztyk, Joshua
A1 Ziff, Oliver J.
A1 Fehmi, Janev
A1 Cerner, Aviva
A1 Jaunmuktane, Zane
A1 Brandner, Sebastian
A1 Yong, Kwee
A1 Manji, Hadi
A1 Carr, Aisling
A1 Rinaldi, Simon
A1 Reilly, Mary M.
A1 D'Sa, Shirley
A1 Lunn, Michael P.
YR 2020
UL http://n.neurology.org/content/95/3/e268.abstract
AB Objective POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin lesions) is a paraneoplastic disorder resulting in severe neurologic disability. Understanding the clinical, laboratory, neurophysiologic, and histopathologic features as well as treatment responses of POEMS will assist in more accurate and timely diagnosis, risk stratification, and effective management.Methods This was a retrospective longitudinal cohort study from 1998 to March 2019, with 7,184 person-months of follow-up time. Hospital databases were used to collate presenting features, investigations, therapies, and response.Results One hundred patients were included with a median follow-up time of 59 months (range, 1–252). Mean symptom onset to diagnosis was 15 months (range, 1–77), with 54% of patients initially misdiagnosed with chronic inflammatory demyelinating polyneuropathy. Median number of multisystem features at diagnosis was 7. Ninety-six (96%) presented with neuropathy, which was length-dependent in 93 (93%) and painful in 75 (75%). At diagnosis, 35% of patients were wheelchair or bedbound, with median Overall Neuropathy Limitation Score of 6, improving to 3 following treatment (p < 0.05). Five-year survival was 90% and 82% at 10 years, with 5- and 10-year progression-free survival of 65% and 53%. Nontreatment with autologous stem cell transplantation, nonhematologic response, and non–vascular endothelial growth factor response are significant risk factors in multivariate analysis to predict progression or death. Risk factors are incorporated to develop a risk score enabling stratification of high- and low-risk cases.Conclusions POEMS syndrome is a rare multisystem condition with delayed diagnosis and poor neurologic function at presentation. Therapy has favorable outcomes. Patients at high risk of death or progression can be identified, which may allow for more active monitoring and influence management.ASCT=autologous stem cell transplantation; CI=confidence interval; CIDP=chronic inflammatory demyelinating polyneuropathy; CR=complete response; HR=hazard ratio; IQR=interquartile range; M-protein=monoclonal protein; mRS=modified Rankin Scale; NE=not evaluable; NR=no response; ONLS=Overall Neuropathy Limitation Score; OS=overall survival; PFS=progression-free survival; PR=partial response; SFLC=serum free light chains; SPE=serum protein electrophoresis; SUV=standardized uptake value; sVEGF=serum vascular endothelial growth factor; UCLH=University College London Hospital; VEGF=vascular endothelial growth factor