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. Par- ticipants 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 reli- ability, 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 a = .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 psy- chometric properties, indicating potential future utility for surveying patient-reported symptoms of SFPN regardless of its cause.