Patient-reported outcome measure for neuromyelitis optica: pretesting of preliminary instrument and protocol for further development in accordance with international guidelines

Abstract

ObjectiveThis study outlines the development of a patient-reported outcome measure (PROM), an instrument to obtain self-reported health status for neuromyeltis optica (NMO), a disabling neurological condition.DesignDevelopment was conducted in accordance with international guidance for PROMs including systematic review of existing literature, item generation guided by qualitative interviews, health-related quality of life conceptual framework and clinical expert panel and cognitive interviews with NMO patients.SettingParticipants were identified through a national NMO clinic in a tertiary NHS neurosciences service.Participants15 individuals with NMO participated in cognitive interviews requiring review and ranking of proposed PROM items and qualitative feedback on content, layout and response options.ResultsParticipants endorsed the draft instrument as reflecting their experience of the condition and as being easy to understand. Rating and ranking of item relevance and importance reduced the draft instrument from 106 to 48 items. Participant feedback on overlapping items eliminated a further 2 items and resulted in a preliminary instrument of 46 items. As a direct result of participant feedback ordering of the 10 domains was revised, a 4 option Likert scale was employed and a 4-week recall period for impact of symptoms was selected.ConclusionsA 46-item instrument developed in accordance with international PROM development guidelines through literature review, developed by subject matter experts and refined through pretesting examining content validity provides a preliminary measure for assessing patient-report of health status in NMO. Further evaluation is proposed including sensitivity to clinical change, and international contributions to evaluating the measure are encouraged.

Neuromyelitis optica spectrum disorder

“…These themes are concordant with those of previous studies. 22,23,41 Of interest, patients receiving nonspecific immune-suppressing treatments tended to rate their regimens more negatively, whereas those on target-specific treatments (e.g., biologics) rated their treatments more positively. Impact of NMO on QoL extended beyond physical and emotional costs; respondents reported a high financial burden, particularly for prescription medicines, travel costs, hospitalization, and specialist care.…”

“…[18][19][20][21] By comparison, few studies have systematically examined the impact of NMOSD on quality of life (QoL) in well-characterized cohorts. [22][23][24][25] Therefore, The Guthy-Jackson Charitable Foundation, Alexion Pharmaceuticals, Chugai Pharmaceutical Co., MedImmune/Viela Bio, and Ipsos Public Affairs conducted a cooperative study of NMOSD patient experience and QoL. Through an interactive survey format, patient-reported clinical, demographic, and experiential data were systematically collected from geographically dispersed patients with NMOSD across North America.…”

Vision Loss from Atypical Optic Neuritis: Patient and Physician Perspectives

“…However, it covers only three domains of quality of life, and the composite summary score lacks psychometric validity, and is not amenable to parametric statistical analysis [8,9]. More recently, a 46-item instrument was developed for neuromyelitis optica spectrum disorder, one rare cause of optic neuritis [10]. This includes questions on a large number of quality of life domains, including vision and vision-related functioning, impacts on life goals and roles, general health-related functioning, mobility, bladder function, bowel function, sexual function, mood, pain, general fatigue, and cognition.…”

<p>The use of patient-reported outcome research in modern ophthalmology: impact on clinical trials and routine clinical practice</p>

“…The Children’s Visual Function Questionnaire has been proposed as a secondary endpoint for clinical trials in children with NF1-associated optic pathway gliomas 76. Disease-specific instruments have been developed for patients with neuromyelitis optica spectrum disorders 97,98. The first use of a PROM information system utilizing CAT in patients with neurofibromatosis has also been reported 99…”