The fiber-specific domains of the QOL-DN demonstrated acceptable reliability and ability to discriminate between subjects with and without neuropathy. Not surprisingly, the DN group scored significantly (P < 0.05) higher than either of the two control groups (i.e., greater impairment). The positive scores for the DC group in the ADL and autonomic domains suggest that diabetes per se impacts these aspects of QOL.

The Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire is an instrument to assess QOL in diabetic polyneuropathy. The objective of this observational, cross-sectional study in 61 patients with V30M transthyretin familial amyloid polyneuropathy (TTR-FAP) and 16 healthy volunteers was to validate the Norfolk QOL-DN for assessment of QOL in TTR-FAP. Comparisons were conducted to identify the best items to discriminate disease stages and assess which individual Norfolk domains (symptoms, large fiber, small fiber, autonomic, and activities of daily living) would be most affected by disease stage. Analysis of individual items revealed a significant pattern of discrimination among disease stages (p < 0.001). Total QOL scores increased (indicating worsening) with duration of symptoms, with a steeper increase observed earlier in the course of disease. Significant correlations were observed between each Norfolk domain and other measures of neurological function. Limitations include cross-sectional study design, low patient numbers in this rare disease, and the ordinal-based character of the metric used; future areas to explore include item response theory approaches such as Rasch analysis. These results suggest the Norfolk QOL-DN is a reliable indicator of the impact of disease severity on QOL in patients with TTR-FAP.

BackgroundThis 52-week study evaluated the long-term safety and tolerability of capsaicin 8% w/w (179 mg) patch repeat treatment plus standard of care (SOC) versus SOC alone in painful diabetic peripheral neuropathy (PDPN).MethodsPhase 3, multinational, open-label, randomised, controlled, 52-week safety study, conducted in Europe. Patients were randomised to capsaicin 8% patch repeat treatment (30 or 60 min; 1–7 treatments with ≥ 8-week intervals) to painful areas of the feet plus SOC, or SOC alone. The primary objective was the safety of capsaicin 8% patch repeat treatment (30 min and 60 min applications) plus SOC versus SOC alone over 52 weeks, assessed by changes in Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) total score from baseline to end of study (EOS). Secondary safety endpoints included Utah Early Neuropathy Scale (UENS) assessments and standardised testing of sensory perception and reflex function.ResultsOverall, 468 patients were randomised (30 min plus SOC, n = 156; 60 min plus SOC, n = 157; SOC alone, n = 155). By EoS, mean changes in Norfolk QOL-DN total score from baseline [estimated mean difference versus SOC alone; 90% CI for difference] were: 30 min plus SOC, −27.6% [−20.9; −31.7, −10.1]; 60 min plus SOC, −32.8% [−26.1; −36.8, −15.4]; SOC alone, −6.7%. Mean changes [difference versus SOC alone] in UENS total score by EoS versus baseline were: 30 min plus SOC, −2.1 [−0.9; −1.8, 0.1]; 60 min plus SOC, −3.0 [−1.7; −2.7, −0.8]; SOC alone, −1.2. No detrimental deterioration was observed in any of the Norfolk or UENS subscales by EoS with capsaicin. Also, no worsening in sensory perception testing of sharp, warm, cold and vibration stimuli was found with capsaicin by EoS. Capsaicin treatment was well tolerated and the most frequent treatment-emergent adverse events were application site pain (30 min, 28.2%; 60 min, 29.3%), burning sensation (30 min, 9.0%; 60 min, 9.6%) and application site erythema (30 min, 7.7%; 60 min, 8.9%).ConclusionIn patients with PDPN, capsaicin 8% patch repeat treatment plus SOC over 52 weeks was well tolerated with no negative functional or neurological effects compared with SOC alone.Trial RegistrationClinicalTrials.gov registration: NCT01478607. Date of registration November 21, 2011; retrospectively registered.Electronic supplementary materialThe online version of this article (doi:10.1186/s12883-016-0752-7) contains supplementary material, which is available to authorized users.

NIS-LL, Norfolk QOL-DN score, composite endpoints of nerve fiber function, and mBMI are valid, reliable measures of TTR-FAP severity. Muscle Nerve 55: 323-332, 2017.

Background For older adults, falls are a serious health problem with over 30% of people over 65 suffering a fall at least once a year. One element often overlooked in the assessment of falls is whether a person’s balance, walking ability and overall falls risk is affected by performing activities of daily living such as walking. Objective This study assessed the immediate impact of incline walking at a moderate pace on falls risk, leg strength, reaction time, gait and balance in 75 healthy adults from 30 to 79 years of age. Subjects were subdivided into five equal groups based upon their age (Group 1, 30–39 years; Group 2, 40–49 years; Group 3, 50–59 years; Group 4, 60–69 years; Group 5, 70–79 years). Methods Each person’s falls risk (using the Physiological Profile Assessment), simple reaction time, leg strength, walking ability and standing balance were assessed prior to and following a period of incline walking on an automated treadmill. The walking task consisted of three 5-minute trials at a faster than preferred pace. Fatigue during walking was elicited by increasing the treadmill incline in increments of 20 (from level) every minute to a maximum of 80. Results As predicted, significant age-related differences were observed prior to the walking activity. In general, increasing age was associated with declines in gait speed, lower limb strength, slower reaction times and increases in overall falls risk. Following the treadmill task, older adults exhibited increased sway (path length 60–69 yrs; 10.2±0.7 to 12.1±0.7 cm: 70–79 yrs; 12.8±1.1 to 15.1±0.8 cm), slower reaction times (70–79 yrs; 256±6 to 287±8 ms), and declines in lower limb strength (60–69 yrs; 36±2 to 31±1 kg: 70–79 yrs; 32.3±2 to 27±1 kg). However, a significant increase in overall falls risk (pre; 0.51±0.17: post; 1.01±0.18) was only seen in the oldest group (70–79 years). For all other persons (30–69 years), changes resulting from the treadmill-walking task did not lead to a significant increase in falls risk. Conclusions As most falls occur when an individual is moving and/or fatigued, assessing functional properties related to balance, gait, strength and falls risk in older adults both at rest and following activity may provide additional insight.