EXPLORING BURDEN IN RARE DISEASES: EXAMINING NEUROPSYCHOLOGICAL SYMPTOMS, FLARES, AND SLEEP QUALITY

Author(s)

Hiba Anwar, MPH, Samantha McStocker, BS, Sarah Villard, PhD, Merika Sanders, PhD, Amanda Healey, MPH, Connie Zhang, MPH;
Folia Health, Boston, MA, USA
OBJECTIVES: IgA nephropathy (IgAN), complement 3 glomerulopathy (C3G), and paroxysmal nocturnal hemoglobinuria (PNH) are rare diseases that present heterogeneously. Patients with these conditions may experience neuropsychological symptoms (NS) and sleep disruptions, as well as symptom flares. Home-reported outcomes (HROs) enable granular, longitudinal tracking of flares, NS, and sleep quality, creating an opportunity to explore potential relationships between them.
METHODS: As of January 2026, 91 patients across these conditions (33 IgAN, 20 C3G, 38 PNH) have tracked their health using the Folia Health (FH) app. FH users report their symptom severity (0-5 Likert scale), denote a flare day, and record their sleep quality. Two analyses were performed to understand the relationship between NS and flares, and the impact of NS on sleep quality.
RESULTS: Brain fog, fatigue, anxiety, and irritability were the most commonly reported NS. Across all conditions, average reported severity was consistently higher on flare days than on non-flare days. For example, brain fog average (SD) severity on flare days vs non-flare days was 2.3 (1.26) vs 1.98 (0.91) in IgAN, 2.78 (0.39) vs 2.25 (0.86) in C3G, and 2.61 (1.18) vs 1.85 (1.21) in PNH. Furthermore, NS severity was associated with sleep quality. For example, in C3G patients, higher average fatigue severity of 2.93 (0.74) was associated with “restless sleep”, whereas lower average fatigue severity of 1.81 (0.62) was associated with “calm sleep”.
CONCLUSIONS: There appears to be a relationship between NS severity and flare incidence, as well as between NS severity and sleep quality. Understanding the relationship between symptom flares, NS, and sleep quality can provide insight into the holistic burden of these diseases and how treatment utilization may affect these important quality-of-life domains.

Conference/Value in Health Info

2026-05, ISPOR 2026, Philadelphia, PA, USA

Value in Health, Volume 29, Issue S6

Code

PCR111

Topic

Patient-Centered Research

Topic Subcategory

Patient-reported Outcomes & Quality of Life Outcomes

Disease

SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain), SDC: Urinary/Kidney Disorders

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