POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin lesions) is associated with a relatively high 5- and 10-year survival rate with appropriate therapy, according to a study in Neurology,

POEMS is a rare paraneoplastic disorder that causes severe neurologic disability. The objective of the study was to gain a further understanding of clinical, laboratory, neurophysiologic, and histopathologic features, and treatment responses in this disorder. To accomplish this goal, researchers reviewed hospital databases to collate clinical features at presentation, investigations, treatments, and recorded responses.

This retrospective study included 100 patients with POEMS who presented to the University College London Hospital between 1998 to March 2019. These patients were assessed over a median follow-up time of 59 months (range, 1–252). Overall, patients had 7184 person-months of follow-up time available for review.

The mean time between symptom onset to POEMS diagnosis in these patients was 15 months (range, 1–77). Approximately 54% initially received a misdiagnosis of chronic inflammatory demyelinating polyneuropathy. At time of diagnosis, a median of 7 multisystem features were recorded. Almost all (96%) patients presented with neuropathy. The neuropathy was length-dependent in 93% of patients and painful in 75% of patients.


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Up to 35% of patients were either wheel chair or bed bound at the time of diagnosis. The median Overall Neuropathy Limitation Score was 6 at diagnosis, but significantly improved to 3 after appropriate therapy (P <.05). The 5- and 10-year survival rates were 90% and 82%, respectively. In contrast, the 5-year progression-free survival (PFS) was 65%, whereas the 10-year PFS was 53%. Significant risk factors for death and progression were hematologic nonresponse (hazard ratio [HR], 4.8; 95% CI, 2.0–11.7; P =.000), vascular endothelial growth factor nonresponse (HR, 2.7; 95% CI, 1.0–7.1; P =.033), and therapy other than autologous stem cell transplantation (ASCT) at any stage of the patients’ treatment (HR, 4.5; 95% CI, 1.6–12.4; P =.003).

Limitations of this study included the rarity of the disorder, which resulted in a limited number of enrolled patients, as well as its retrospective nature. In addition, younger patients with fewer comorbidities were selected for ASCT, a factor which possibly skewed results toward favorable outcomes.

The researchers concluded that a greater understanding of the clinical features and “treatment responses of POEMS will assist in more accurate and timely diagnosis, risk stratification, and effective management.”

Reference

Keddie S, Foldes D, Caimari F, et al. Clinical characteristics, risk factors, and outcomes of POEMS syndrome: A longitudinal cohort study. Neurology. 2020;95(3):e268-e279.