Can Neuro-Qol Inform Us of Symptomatic Burden and Functional Disability Experienced by Patients Living with Stiff Person Syndrome Spectrum Disorders?
Hanyeh Afshar1, Herbert Chen2, Mayaa Appiah1, Scott Newsome1
1Johns Hopkins University School of Medicine, 2Baylor College of Medicine
Objective:

To evaluate the potential use of the Quality of Life in Neurological Disorders (Neuro-QoL) questionnaires for assessment of symptomatic burden and functional disability in stiff person syndrome spectrum disorders (SPSD).

Background:

SPSD are rare, disabling neurological disorders that profoundly impact patients’ social, emotional, and physical well-being. Common complaints outside of impaired mobility include anxiety, sleep disturbances, and fatigue. Despite improving quality of life being central to SPSD care, there remains an unmet need for standardized health-related quality of life measures.

Design/Methods:

Data were collected across 10 Neuro-QoL domains for a convenience sample of 19 patients. Demographics, clinical characteristics, and T25FW measures were also collected. Spearman correlation or one-sample t-tests (reference mean=50) were used to determine associations as appropriate.

Results:

Nineteen patients with median age 60 [IQR 55-65] participated (13 classic SPS, 5 SPS-plus, 1 PERM). Initial symptom onset was 12 [IQR 9, 20] years. Most patients were female (84%) and White (74%). SPSD patients scored lower in social participation (t=-4.0, p=0.001) and satisfaction (t=-3.7, p=0.002). They also showed mild upper extremity and moderate lower extremity (LE) functional impairments (t=-3.7, p=0.002; t=-5.1, p=0.000, respectively). There was a strong negative correlation between LE and T25FW (rho=-0.92, p=0.001 for unassisted T25FW and rho=-0.82, p=0.034 for T25FW with bilateral assistance). Participants also scored worse in stigma (t=2.5, p=0.02). No other statistically significant differences were observed.

Conclusions:
Neuro-QoL questionnaires may provide clinicians with a valuable tool for assessing QoL in SPSD, supporting more patient-centered care. The Neuro-QoL limb function domains can be used to measure patients’ current capabilities across these areas, while other domains may be more suited for monitoring changes over time due to their use of a defined recall period. Given these complementary strengths, Neuro-QoL measures may also be useful in clinical trials to capture both functional status and QoL outcomes in a standardized, patient-reported format.
10.1212/WNL.0000000000217334
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