Treatment for POEMS (polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes) syndrome

POEMS (polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes) syndrome is a rare cause of demyelinating and axonal mixed neuropathy with multiorgan involvement and monoclonal plasma cell-proliferative disorder. The pathogenesis of POEMS syndrome is not well understood, but overproduction of vascular endothelial growth factor (VEGF), probably secreted by plasmacytomas, is likely to be responsible for most of the characteristic symptoms. POEMS syndrome is a potentially fatal disease, and patients' quality of life deteriorates because of progressive neuropathy, massive pleural effusion or ascites, or thromboembolic events. There is a need for efficacious therapy to improve prognosis.

To provide the best available evidence from randomised controlled trials on treatment for POEMS syndrome.

We searched the Cochrane Neuromuscular Disease Group Trials Register (March 2008), MEDLINE (from January 1966 to March 2008), EMBASE (from January 1980 to March 2008) and CINAHL (from January 1982 to March 2008) for randomized controlled trials, quasi-randomized trials, historically controlled studies, and trials with concurrent controls. We adapted this strategy to search MEDLINE from 1966 and EMBASE from 1980 for comparative cohort studies, case-control studies and trials, and case series.

All randomized and quasi-randomized controlled trials, and non-randomized controlled studies were sought. Since we discovered no such clinical trials , we assessed and summarized all retrospective case series including five or more patients in the 'Discussion' section.

Two authors independently reviewed and extracted details of all potentially relevant trials with any treatment for POEMS syndrome. We then collated and summarized information on the outcome.

We found no randomized or non-randomized prospective controlled trials of treatment for POEMS syndrome. We summarized the results of retrospective case series containing five or more patients in the Discussion section.

There are no randomized or quasi-randomized controlled clinical trials of treatment for POEMS syndrome on which to base practice.