Bone · 2026
Development of the Fibrodysplasia Ossificans Progressiva FLare EXperience Revised (FOP-FLEX-R©) Questionnaire: A Patient-Reported Outcome Measure.
СтатьяНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Pignolo Robert J, Hsiao Edward C, Al Mukaddam Mona, Brova Monica, Olonilua Daniella, Shields Alan L, LaBranche Timothy P, Sherwin Cori Ann, Boing Elaine A, Karimian Negar, Solanke Oyinkan, Kaplan Frederick S
Аннотация
BACKGROUND: Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, genetic disorder mainly characterized by progressive heterotopic ossification (HO). Flare-ups in FOP, involving soft tissue swelling, pain, warmth, and stiffness, often precede HO. We report development of the FOP-FLEX-R© questionnaire (Ipsen, 2026), providing clinical researchers with a comprehensive tool to assess patient experiences during flare-ups, and address the limitations of existing patient-reported outcome measures (PROMs).
METHODS: The FOP-FLEX questionnaire was developed through: a targeted literature review to identify signs, symptoms, and impacts of FOP; concept selection and questionnaire development activities; validation of content and readability in expert consultations and patient interviews; revisions to create the FOP-FLEX-R© questionnaire.
RESULTS: The literature review identified 42 'sign and symptom' and 28 'impact' concepts. Ten concepts were selected for inclusion in the initial FOP-FLEX: swelling, pain, skin warmth, stiffness, skin redness, impaired daily activities, frustration, bother, fatigue/tiredness, and appetite loss. Consultations with clinical experts (n = 5) and interviews with patients (n = 12) affirmed these concepts. Cognitive debriefing with the same patients indicated the FOP-FLEX was understandable and easy to complete, though some revisions were made, including those to enable the assessment to be completed by younger patients and to remove non-flare-up items.
CONCLUSIONS: The FOP-FLEX-R© questionnaire offers a comprehensive, content-validated PROM for use in clinical trials, to understand the lived experience of flare-ups in FOP and evaluate the benefit of new therapies.
METHODS: The FOP-FLEX questionnaire was developed through: a targeted literature review to identify signs, symptoms, and impacts of FOP; concept selection and questionnaire development activities; validation of content and readability in expert consultations and patient interviews; revisions to create the FOP-FLEX-R© questionnaire.
RESULTS: The literature review identified 42 'sign and symptom' and 28 'impact' concepts. Ten concepts were selected for inclusion in the initial FOP-FLEX: swelling, pain, skin warmth, stiffness, skin redness, impaired daily activities, frustration, bother, fatigue/tiredness, and appetite loss. Consultations with clinical experts (n = 5) and interviews with patients (n = 12) affirmed these concepts. Cognitive debriefing with the same patients indicated the FOP-FLEX was understandable and easy to complete, though some revisions were made, including those to enable the assessment to be completed by younger patients and to remove non-flare-up items.
CONCLUSIONS: The FOP-FLEX-R© questionnaire offers a comprehensive, content-validated PROM for use in clinical trials, to understand the lived experience of flare-ups in FOP and evaluate the benefit of new therapies.