IntroductionChronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), a rare autoimmune disorder affecting peripheral nerves, is associated with substantial societal costs. This study assesses healthcare utilization and productivity losses across varying levels of patient disability.MethodsData were obtained from Adelphi’s CIDP Disease Specific Programme™ (September 2022–April 2023), a digital, international, real-world survey involving neurologists and their patients (UK, France, Germany, Italy, Spain). Disability was measured using the Inflammatory Neuropathy Cause and Treatment (INCAT; range: 0–10), and categorized as mild (≤2), moderate (3–4), or severe (≥5). Neurologists provided data on treatment, informal care, hospitalizations, mobility aids, and home modifications for 542 patients, while lost productivity was reported by a subset of patients (n = 199). Associations between disability level and healthcare utilization and lost productivity were evaluated using linear regression, adjusting for age and time since diagnosis.ResultsOf the 542 patients, 236 (43.5%) had mild, 189 (34.9%) moderate, and 117 (21.6%) severe disability. Treatment rates increased with severity (receiving treatment: mild 78.4%, moderate 89.9%, severe 92.3%; p < 0.001). The proportion of patients receiving informal care rose sharply with increasing disability (mild 7.2%, moderate 31.2%, severe 62.9%; p < 0.001). Hospitalizations in the 12 months prior to the survey were more frequent with greater disability (mild 7.0%, moderate 14.5%, severe 21.6%; p < 0.001), as were emergency admissions (1.0, 9.9, 13.7% respectively; p < 0.001) and ICU stays (0.0, 0.7, 3.9% respectively; p = 0.001). The use of mobility aids (19.1, 56.1, 89.7% respectively) and home modifications (12.8, 45.8, 74.2% respectively) also rose significantly with severity (p < 0.001 for both). Finally, work productivity losses among employed patients and activity impairment rose as disability increased (average percentage of productivity loss: mild 21.8%, moderate 44.0%, severe: 67.3%, p < 0.001; average daily activity impairment: mild 28.6%, moderate: 44.6%, severe: 68.1%, p < 0.001).ConclusionCIDP is associated with substantial healthcare utilization that increases with increasing disability, highlighting the progressive functional decline associated with more severe disease. In addition to direct healthcare demands, CIDP imposes considerable caregiver support needs, changes in living conditions, and productivity losses. Together, these findings underscore the wide-ranging impact of CIDP and the need for comprehensive management strategies.

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive disease predominantly affecting older men and is associated with increasing care needs. Previous international studies have reported that, as ATTR-CM progresses, caregivers experience substantial physical and emotional burden, as well as productivity impairment. However, real-world evidence on caregiver burden in Japan remains limited. This study aimed to characterize multidimensional caregiver and patient burden in Japan. Data were drawn from the Adelphi ATTR Disease Specific Programme™, a cross-sectional survey of cardiologists and their consulting patients with ATTR-CM, conducted in Japan between October 2024 and January 2025. Cardiologists completed patient record forms, including patient demographics, clinical characteristics, and caregiving status. Patients and their non-professional caregivers also completed voluntary self-reported questionnaires. Caregiver outcomes included caregiving time, work productivity, and activity impairment (WPAI), and Zarit Burden Interview (ZBI) scores. Patient health status was assessed using the Kansas City Cardiomyopathy Questionnaire (KCCQ). Analyses were descriptive. Twenty-five physicians provided data for 120 patients (mean age 74.7 years; 78.3

Introduction:Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) is a rare autoimmune disorder affecting the peripheral nerves, typically characterized by muscle weakness and sensory deficits. This study seeks to describe CIDP patients' journey to diagnosis alongside factors influencing misdiagnosis and time to diagnosis. Methods:We analyzed demographics and diagnostic data reported by neurologists and their patients in Adelphi's CIDP Disease Specific Programme™. This digital, multinational real-world survey was held in the UK, France, Germany, Italy and Spain between September 2022 and April 2023 (n = 542). Results:Mean (SD) age was 54.0 (12.4) years; 62% of patients were male. Half of the patients reported at least one comorbidity, with anxiety, depression and diabetes being the most common. The mean (SD) number of diagnostic procedures undergone per patient was 19.6 (9.4). An electromyogram and nerve conduction study (98%), complete blood count (82%) and administration of anti-ganglioside antibodies (78%) were carried out most frequently. Most patients had been diagnosed with typical CIDP (68%) and 37% had been misdiagnosed at least once. The most common misdiagnosis was Guillain-Barré syndrome, in 37% of cases. No significant associations were found between misdiagnosis and the variables sex, disease severity at symptom onset, age category, BMI or CIDP subtype. The median (Q1 - Q3) time between symptom onset and diagnosis was 7.0 (3.2-13.0) months. A multiple linear analysis on the log-transform of the time to diagnosis indicated that patients with a long time to diagnosis more often presented with mild symptoms at onset, had variant CIDP and had been misdiagnosed. Conclusion:Median time to diagnosis for CIDP patients was 7 months; over a third had at some point been misdiagnosed. Mild symptoms, having variant CIDP and having been misdiagnosed were associated with longer time to diagnosis. Further research into the causes of diagnostic delay and the impact of late diagnosis and treatment is needed.

INTRODUCTION:Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) is an immune-mediated disorder characterized by proximal and distal motor weakness and sensory deficits. This study investigated the impact of fatigue and disability on health-related quality of life (HRQoL) in individuals with CIDP. METHODS:Matched physician-patient data were analyzed from Adelphi's CIDP Disease Specific Programme™, an international survey conducted in France, Germany, Italy, and Spain (n = 199; April 2022-April 2023). Patients completed the FACIT-Fatigue, I-RODS (limitations in daily activities), and EQ-5D-5L (HRQoL) questionnaires. Physicians assessed disability using the INCAT score. Spearman correlations and multiple linear regression were used to assess associations between INCAT, I-RODS, FACIT-Fatigue, and EQ-5D-5L utilities. RESULTS:Participants' mean age was 54.0 years (SD: 12.4); 62% were male. The mean EQ-5D-5L utility value was 0.65 (SD: 0.22). Moderate to severe fatigue was reported by 67.2% of patients, 27.7% reported substantial activity limitations, and 48.3% were rated as having moderate to severe disability. Greater disability (INCAT: r = -0.568; I-RODS: r = 0.770), and higher fatigue (FACIT-Fatigue: r = 0.799) were significantly correlated with lower HRQoL, as reflected by EQ-5D-5L utility values. In regression analysis, higher INCAT disability (β = -0.024, 95% CI [-0.036, -0.013]), higher I-RODS disability (β = 0.004, 95% CI [0.002, 0.005]), and more severe fatigue (β = 0.009, 95% CI [0.006, 0.012]) were all independently associated with lower HRQoL. CONCLUSION:Both fatigue and disability were independently associated with reduced HRQoL in CIDP. These findings suggest that fatigue is an important consideration in the management of HRQoL, with further research needed to evaluate its potential as a therapeutic target.

BACKGROUND:The Revised Amyotrophic Lateral Sclerosis (ALS) Functional Rating Scale (ALSFRS-R) is a clinician-reported outcome measure monitoring disease progression in people living with ALS (pALS). This study examined the relationship of ALSFRS-R scores with disease progression and independence levels for activities of daily living (ADLs) among pALS. METHODS:Real-world data, including the ALSFRS-R, were drawn from a cross-sectional survey of US neurologists treating pALS (Adelphi ALS Disease Specific Programme™), conducted between July 2020 and March 2021. ALSFRS-R scores were modeled against 11 pre-defined disease milestones. The relationship between ALSFRS-R scores and levels of independence in 24 ADLs was examined using ordered logistic regression. RESULTS:Fifty-nine neurologists provided data for 379 pALS (mean age: 59.5 years; mean disease duration: 16.1 months). Estimated mean ALSFRS-R total score decreased (worsened) from 46.1 at first consultation regarding ALS symptoms to 25.1 upon receipt of a feeding tube. In general, pALS were likely to be completely dependent in most ADLs when their ALSFRS-R total scores were ≤ 25. A 1-point decrease in ALSFRS-R total score was associated with increased risks of losing independence across all ADLs. For each ADL, a 1-point decrease in domain score was associated with varying risks of losing independence across different domains. CONCLUSIONS:There is a correlation between ALSFRS-R scores and levels of independence in ADLs among pALS, facilitating score interpretation for monitoring disease and function status. Yet, the relevance of the ALSFRS-R total score diminishes in advanced stages of ALS, indicating a need for additional measures to provide comprehensive evaluation.

INTRODUCTION:Progressive supranuclear palsy (PSP) is a rare, relentlessly progressive, ultimately fatal neurodegenerative disease. Clinical phenotypes have been defined to describe observed heterogeneity in clinical characteristics of PSP patients; it is of interest to assess the potential of using datasets such as the one used in this study to determine predominant phenotypes of patients with PSP and enable epidemiological studies. We applied Movement Disorder Society (MDS)-PSP and Multiple Allocation eXtinction (MAX) algorithms for designating patients to PSP phenotypes using real-world data and describe the distribution of these phenotypes. METHODS:Data were drawn from the Adelphi PSP Disease Specific Programme, a real-world cross-sectional study of neurologists and people living with PSP in the USA, France, Germany, Italy, Spain, and the UK. Patients were allocated to PSP phenotypes using MDS criteria and MAX Rules. RESULTS:Data from 892 patients with PSP were evaluated (mean age: 68.9 years; 60.7 % male), and 816 (91 %) patients could be allocated to >1 phenotype (mean: 7.3 per patient). After applying MAX Rules, mean number of phenotypes per patient reduced to 1.0, with only 2 % of patients still allocated multiple phenotypes. While 42 % of patients were initially designated as PSP-Richardson syndrome (RS), this increased to 75 % when signs throughout the entire patient record were considered. CONCLUSIONS:In the largest study done to date, these findings in real-world data confirm that applying of MAX Rules to MDS-PSP criteria can yield a predominant phenotype for majority of PSP patients and reinforce suggestions that PSP-RS becomes the predominant phenotype over time.

Background: Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare form of heart failure. Symptoms can be burdensome and management often involves caregiver support. However, real-world data describing burden in ATTR-CM are limited. Objective: To describe the burden of ATTR-CM among patients and their caregivers in the United States (US). Methods: Secondary analyses of data from the Adelphi Real World ATTR Disease Specific Programme™, a cross-sectional survey of ATTR-treating physicians and their patients in the US from August 2024 – February 2025. Physicians reported patient demographics and caregiver support. Patients and their accompanying non-professional caregivers were invited to report disease burden, including the Work Productivity and Activity Impairment questionnaire (WPAI), and the Zarit Burden Interview (ZBI) for caregivers. Analyses were descriptive. Results: Overall, 33 physicians provided data for 256 patients – of whom 58 self-reported data – and 15 caregivers provided data. Mean (standard deviation; SD) patient age was 63.9 (13.2) years and 60.9% of patients were male. At survey, 21.9% of patients were New York Heart Association (NYHA) class I, 54.3% class II, 19.9% class III, and 3.9% class IV. Median (interquartile range; IQR) disease duration was 1.9 (1.3–3.0) years. Overall, 42.2% of patients had a caregiver, commonly their partner/spouse (61.7%), their child (21.0%), and/or a professional caregiver (16.0%). Caregiver support was often required with housecleaning/home maintenance (68.0%), transportation (65.3%), and shopping/meal preparation (64.0%). Patients cited fear about symptoms getting worse (94.6%) and loss of independence (87.9%) as negatively impacting quality of life (QoL). For the WPAI ( Figure 1 ), patients reported a mean (SD) percent work time missed of 12.5% (19.2%). For the WPAI ( Figure 2 ), caregivers reported a mean (SD) percent work time missed of 53.8% (50.0%). For the ZBI, caregivers reported a mean (SD) score of 39.1 (20.1). Conclusion: Despite an average disease duration of >2 years, over two fifths of patients required caregiver support. Most patients reported loss of independence as negatively impacting their QoL, with work and activity impairment evident in both patient and caregiver WPAI data. This substantial burden on patients and their caregivers underscores the need for interventions to address clinical outcomes and reduce caregiver dependence.