title: Initial Development and Validation of a Patient-Reported Symptom Survey for Small-Fiber Polyneuropathy.
abstract: 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.
  
  
  
  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.
authors.affiliation: Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts. Electronic address: treister.roi@gmail.com.
authors.forename: Roi
authors.initials: R
authors.lastname: Treister
authors.affiliation: Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts.
authors.forename: Mette
authors.initials: M
authors.lastname: Lodahl
authors.affiliation: Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts.
authors.forename: Magdalena
authors.initials: M
authors.lastname: Lang
authors.affiliation: Channing Division of Network Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts; Department of Epidemiology, Harvard T.H. Chan School of Public Health, Boston, Massachusetts.
authors.forename: Shelley S
authors.initials: SS
authors.lastname: Tworoger
authors.affiliation: College of Education, Wayne State University, Detroit, Michigan.
authors.forename: Shlomo
authors.initials: S
authors.lastname: Sawilowsky
authors.affiliation: Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts; Department of Pathology (Neuropathology), Massachusetts General Hospital, Boston, Massachusetts.
authors.forename: Anne Louise
authors.initials: AL
authors.lastname: Oaklander
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010147:Pain Measurement / Q000379:methods*; D025341:Principal Component Analysis; D011594:Psychometrics; D012372:ROC Curve; D015203:Reproducibility of Results; D000071075:Small Fiber Neuropathy / Q000175:diagnosis / Q000503:physiopathology* / Q000523:psychology*; D011795:Surveys and Questionnaires; D055815:Young Adult
keywords: Peripheral neuropathy; dysautonomia; neuropathic pain; sensory; skin biopsy