OBJECTIVE: To understand patient experience in essential tremor (ET), describe meaningful concepts related to treatment benefit, and compare concepts to existing clinical outcome assessments (COAs). Background: ET is among the most common movement disorders, but patient experience in ET is understudied and has not been mapped to COAs.
METHOD: Eligible participants aged ≥18 years, recruited through the International Essential Tremor Foundation, were either individuals with self-reported diagnosed ET or care partners. One-hour, semi-structured, concept elicitation telephone interviews were conducted. Transcripts were coded for themes and summarized against 2 existing ET-specific COAs used in clinical trials: The Essential Tremor Rating Assessment Scale (TETRAS) and Quality of Life in Essential Tremor (QUEST).
RESULTS: Eighteen individuals with ET and 2 care partners completed interviews (N=20). Participants’ self-report represented the full range of severity (5 mild, 8 moderate, 7 severe/very severe). All participants reported hand tremor, 75% had additional symptoms; 30% utilized caregiver support. The most common impacts on activities of daily living (ADLs) were pouring liquid (100%), writing/typing (100%), grooming/hygiene (85%), drinking (85%), dressing (80%), eating (80%), reading (80%), using keys (75%), work/school (70%), chores (60%), carrying/lifting (60%), and hobbies (55%). The most bothersome impacts were writing (45%) and eating (30%). Overall, 90% of participants indicated ≥1 emotional impact of ET; 75% reported tremor-related worry or anxiety. Decreasing the severity and frequency of tremor were described as meaningful treatment benefits to all participants, particularly related to reducing its impact on ADLs. Most concepts reported by participants were captured in either TETRAS or QUEST; however, balance, stigma, frustration, irritability, work relationships, and need for caregiver support were not robustly assessed.
CONCLUSION: Participants articulated a range of symptoms and impacts related to ET, including physical, mental, and emotional aspects of overall health and quality of life. Existing COAs generally capture the health concepts most relevant and meaningful to patients, suggesting suitability for evaluating treatment benefits. Further research should evaluate whether ET treatments enable patients to achieve meaningful benefits.
