Daily variation in symptoms and functioning in Huntington disease: Feasibility and variability - PubMed (original) (raw)

Daily variation in symptoms and functioning in Huntington disease: Feasibility and variability

Noelle E Carlozzi et al. Parkinsonism Relat Disord. 2025 Apr.

Abstract

Introduction: The day-to-day experience of symptoms and functioning are largely understudied in people with Huntington disease (HD). Real-world capture of day-to-day symptoms and functioning could be valuable as future HD outcome measures. Therefore, the purpose of this study was to 1) examine the feasibility of a weeklong intensive study design; and 2) understand the daily variation of symptoms and functioning in individuals with premanifest and manifest HD.

Methods: During this 8-day study, 52 individuals with HD completed real-time symptom reports 3 times each day (chorea, sleep, fatigue, anxiety), wore a wrist-worn device (PRO-Diary sleep and physical activity), and completed daily diaries of symptoms (chorea, sleep, fatigue, anger, depression, anxiety) and functioning (physical activity, speech/swallowing, and social participation) each night. We examined rates of missing data, and multilevel models were used to analyze data.

Results: Rates of missing data were 23-52 % for the real-time symptom reports and 19-35 % for the daily diaries. Average PRO-Diary wear compliance was 64 % for manifest and 67 % for pre-manifest HD for sleep, and 71 % for pre-manifest and 78 % for manifest HD for physical activity. Within-subject variability was greater over the week (day-to-day median range 1.9-4.4 SDs) than over the day (within-a-day median range 0-2 SDs). Associations between baseline and real-time reports of the same constructs were generally consistent.

Conclusions: Rates of missing data were consistent with similar study designs in other clinical populations, supporting feasibility. Missingness was highest for the real-time symptom reports. HD participants experienced more day-to-day variation in symptoms and function, with less variability within a day, suggesting that once-daily assessments may suffice.

Copyright © 2025 Elsevier Ltd. All rights reserved.

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Conflict of interest statement

Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Conflict of interest statement Noelle E. Carlozzi, PhD has been a consultant to Teva Pharmaceuticals Industries, Ltd.; she also receives compensation for her role on the CHDI Foundation scientific advisory board. No other authors have potential conflicts to disclose. Funding sources and conflicts of interest Carlozzi, N.E. Dr. Carlozzi received salary support for this project from Teva Pharmaceuticals Industries, Ltd.; Dr. Carlozzi also receives compensation for her CHDI Foundation scientific advisory board role. She has no other potential conflicts to report. Troost, J.P. Dr. Troost received salary support for this proposal from Teva Pharmaceuticals Industries, Ltd. He has no potential conflicts to report. Miner, J.A. Ms. Miner received salary support for this proposal from Teva Pharmaceuticals Industries, Ltd. She has no potential conflicts to report. Singh, K. Dr. Singh received salary support for this proposal from Teva Pharmaceuticals Industries, Ltd. He has no potential conflicts to report. Padmanabhan, A.A. Mr. Padmanabhan received salary support for this proposal from Teva Pharmaceuticals Industries, Ltd. He has no potential conflicts to report. Lombard, W. Mrs. Lombard has no potential conflicts to report. Dayalu, P. Dr. Dayalu received salary support for this proposal from Teva Pharmaceuticals Industries, Ltd. He has no potential conflicts to report. Financial disclosures for the previous 12 Months In the past 12 months, Dr. Noelle E Carlozzi has received funding from the National Institutes of Health, the Agency for Healthcare Research and Quality, the Patient-Centered Outcomes Research Institute, the Craig H. Neilsen Foundation, the Alzheimer's Association, the CHDI Foundation, and Teva Pharmaceutical Industries, as well as Health and Human Services, the Department of Centers for Medicare and Medicaid Services, and the Department of Food and Drug Administration. In the past 12 months, Dr. Jonathan P Troost has received funding from the National Institutes of Health, the Craig H. Neilsen Foundation, the Alzheimer's Association, Teva Pharmaceutical Industries, and the Gerber Foundation. In the past 12 months, Jennifer A. Miner has received funding from the National Institutes of Health, the Patient-Centered Outcomes Research Institute, the Alzheimer's Association, the Craig H. Neilsen Foundation, Teva Pharmaceutical Industries, the Centers for Medicare and Medicaid Services, and the Department of Food and Drug Administration. In the past 12 months, Dr. Karandeep Singh has received funding from the National Institutes of Health, the Rheumatology Research Foundation, the American Society for Surgery, the American Heart Association, and Teva Pharmaceutical Industries. In the past 12 months, Arjun Padmanabhan has received funding from Teva Pharmaceutical Industries. In the past 12 months, Wendy Lombard has received funding from the National Institutes of Health. In the past 12 months, Dr. Praveen Dayalu has received funding from the CHDI Foundation, Teva Pharmaceutical Industries, uniQure Biopharmaceuticals, and Neurocrine Biosciences funding through the Huntington Study Group.

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