Background Prompt diagnosis and early treatment prevents disability in Polyneuropathy Organomegaly Endocrinopathy Monoclonal-protein and Skin Changes (POEMS) syndrome. Delay in diagnosis is common with 55% of patients initially incorrectly diagnosed with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Patients are often treated with intravenous immunoglobulin which is both expensive and ineffective in the treatment of POEMS. Testing patients with acquired demyelinating neuropathy with serum vascular endothelial growth factor (VEGF) more accurately identifies POEMS syndrome than the current standard of care. Incorporating VEGF testing into screening could prevent misdiagnosis and reduce costs.
Methods We used observed treatment information for patients in the University College London Hospital’s POEMS syndrome database (n=100) and from the National Immunoglobulin Database to estimate costs associated with incorrect CIDP diagnoses across our cohort. We conducted a model-based cost-effectiveness analysis to compare the current diagnostic algorithm with an alternative which includes VEGF testing for all patients with an acquired demyelinating neuropathy.
Results Treatment associated with an incorrect CIDP diagnosis led to total wasted healthcare expenditures of between £808 550 and £1 111 756 across our cohort, with an average cost-per-POEMS-patient misdiagnosed of £14 701 to £20 214. Introducing mandatory VEGF testing for patients with acquired demyelinating neuropathy would lead to annual cost-savings of £107 398 for the National Health Service and could prevent misdiagnosis in 16 cases per annum.
Conclusions Misdiagnosis in POEMS syndrome results in diagnostic delay, disease progression and significant healthcare costs. Introducing mandatory VEGF testing for patients with acquired demyelinating neuropathy is a cost-effective strategy allowing for early POEMS diagnosis and potentially enabling prompt disease-directed therapy.
- health economics
- health policy & practice
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ESM and SK are joint first authors.
Contributors ESM collected data, performed the primary analysis and wrote the manuscript. SK was responsible for the paper concept and design, data collection, writing the manuscript and edits. FT-P performed data analysis and edits. SD was responsible for data acquisition and edits. MPL was responsible for the paper concept and design, data interpretation and edits.
Funding SK is funded by a Guarantors of Brain & Association of British Neurologists Clinical Training Research Fellowship. MPL is supported by the National Institute for Health Research, UCLH's Biomedical Research Centre.
Competing interests None declared.
Patient consent for publication Not required.
Ethics approval This study was approved by the London School of Hygiene and Tropical Medicine Ethics Committee.
Provenance and peer review Not commissioned; externally peer reviewed.