Initial Development and Validation of a Patient-Reported Symptom Survey for Small-Fiber Polyneuropathy

Roi Treister, Mette Lodahl, Magdalena Lang, Shelley S. Tworoger, Shlomo Sawilowsky, Anne Louise Oaklander

Research output: Contribution to journalArticlepeer-review


Small-fiber polyneuropathy (SFPN) affects unmyelinated and thinly myelinated peripheral axons. Several questionnaires have been developed to assess polyneuropathy from diabetes or chemotherapy, but none for SFPN from other or unknown causes. A comprehensive survey could help clinicians diagnose and assess treatment responses, define prevalence natural history and cures, and identify research subjects. Thus, we developed the 1-page Small-Fiber Symptom Survey, using input from patients and 21 medical/scientific experts. Participants comprised consenting consecutive patients evaluated for SFPN at the Massachusetts General Hospital plus normal control subjects. Participants SFPN status was stratified on the basis of the results of their objective diagnostic tests (distal leg skin biopsy and autonomic function testing). We measured internal consistency, test retest reliability, convergent validity, and performed a receiver operating curve analysis. The 179 participants averaged 46.6 ± 15.6 years old; they were 73.2% female and 92.2% Caucasian. Eighty-five had confirmed SFPN, mostly idiopathic. Principal component analysis revealed 5 symptom clusters. The questionnaire had good internal consistency (Cronbach α = .893), excellent test retest reliability (r = .927, P < .001) and good to fair convergent validity. Participants with confirmed SFPN had more severe symptoms than others (P = .009). The Small-Fiber Symptom Survey has satisfactory psychometric properties, indicating potential future utility for surveying patient-reported symptoms of SFPN regardless of its cause. Perspective This article reports the initial development and early psychometric validation of a new patient-reported outcome measure intended to capture the wide range of multisystem symptoms of SFPN. When further developed, it could potentially help clinicians diagnose and monitor patients, and help advance research.

Original languageEnglish
Pages (from-to)556-563
Number of pages8
JournalJournal of Pain
Issue number5
StatePublished - 1 May 2017

Bibliographical note

Funding Information:
This work was supported by the National Institutes of Health (R01-NS093653 and UL1 TR001102), the Lundbeck Foundation Scholarship in Neurology, and the U.S. Department of Defense (GW140169).

Publisher Copyright:
© 2017 American Pain Society


  • Peripheral neuropathy
  • dysautonomia
  • neuropathic pain
  • sensory
  • skin biopsy

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology
  • Anesthesiology and Pain Medicine


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