Characterisation of a Population of Adults Suffering From Cystic Fibrosis in a Belgian Reference Center
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- Это наблюдательное исследование: исследуемое лечение участникам по протоколу не назначают.
- Кому может быть актуально
- Состояния в реестре: Quality of Lifte, Cystic Fibrosis (CF), Diabetes. Базовые параметры: от 18 лет · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- Бельгия
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Официальное название
Characterisation of a Population of Adults Suffering From Cystic Fibrosis in a Belgian Center of Reference and Evaluation of the Impact of Diabetes and CFTR Modulators on Respiratory Function, Nutritional Status and Quality of Life
Обзор
This research project aims to better understand the consequences of diabetes on the quality of life, respiratory function, and nutritional status of patients with cystic fibrosis followed at a Belgian reference center and to compare the quality of life of patients with cystic fibrosis depending on whether or not they have diabetes.
Подробное описание
This is a monocentric observational cohort study combining retrospective medical record review and cross-sectional assessment using quality-of-life questionnaires in patients with cystic fibrosis(CF) with or without cystic fibrosis-related diabetes.
This study aims to investigate and compare the characteristics of patients with cystic fibrosis according to the presence or absence of cystic fibrosis-related diabetes (CFRD), based on patients currently followed in one of the Belgian reference centers. The impact of diabetes, its early diagnosis, and its metabolic control will be assessed in relation to patients' nutritional and respiratory status, as well as their quality of life.
The influence of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy on diabetes control will also be evaluated by comparing glycemic control parameters in patients receiving modulators with those not receiving them, and by examining these parameters before and after treatment initiation within the treated group. In addition, the impact of CFTR modulators on nutritional and respiratory status will be assessed in patients without CFRD.
Quality of life will be evaluated using the SF-36 questionnaire in patients with cystic fibrosis, with and without CFRD. The quality of life of patients with CFRD will also be compared with that of an age-matched sample of patients with type 1 diabetes followed at the same hospital. Additional diabetes-related outcomes will be assessed and compared between the two groups of diabetic patients, including hypoglycemia awareness, satisfaction with diabetes treatment, and satisfaction with blood glucose monitoring.
Previous studies suggest that patients with CFRD tend to report a lower overall quality of life, poorer acceptance of insulin therapy and glucose monitoring, but a better perception of hypoglycemia and a lower perceived impact of hypoglycemia on daily life.
Первичные конечные точки
- Comparison of quality of life between patients with cystic fibrosis according to the presence or absence of diabetes [Срок оценки: cross-sectional assessment at study inclusion]
- Comparison of ppFEV₁ between patients with cystic fibrosis according to the presence or absence of diabetes. [Срок оценки: FEV₁ data will be collected retrospectively from medical records, using the value closest to the time of study inclusion]
Вторичные конечные точки (3)
- Comparison of BMI between patients with cystic fibrosis according to the presence or absence of diabetes [Срок оценки: Data will be collected retrospectively from medical records, using the value closest to the time of study inclusion]
- Hypoglycemia awareness and treatment satisfaction will be assessed using validated questionnaires and compared between patients with CFTR-related disease and those with type 1 diabetes. [Срок оценки: Cross-sectional assessment at study inclusion]
- Comparison of glycemic control parameters in patients with Cystic Fibrosis Related Diabetes before and after initiation of CFTR modulator therapy. [Срок оценки: Glycemic parameters will be recorded retrospectively at the visit closest to the initiation of CFTR modulator treatment and at the visit closest to study inclusion]
Критерии участия
Критерии включения
Adults ≥18 years Confirmed diagnosis of cystic fibrosis Followed at the participating CF reference center Ability to complete quality-of-life questionnaires Signed informed consent (if required by EC)
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Критерии исключения
Refusal to participate Inability to complete questionnaires
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Модель наблюдения
- Когортное
Центры проведения
Бельгия · 1 центр
- Hôpital Erasme - HUB 808 route de Lennik 1070 Brussels Belgium — Brussels
Публикации
- Gupta N, Gupta A, Narayanan M R V. Current status of nitrous oxide use in pediatric patients. World J Clin Pediatr. 2022 Mar 9;11(2):93-104. doi: 10.5409/wjcp.v11.i2.93. eCollection 2022 Mar 9. PMID 35433304
- Marks BE, Kilberg MJ, Aliaj E, Fredkin K, Hudson J, Riva D, Roman C, Kelly A, Putman MS. Perceptions of Diabetes Technology Use in Cystic Fibrosis-Related Diabetes Management. Diabetes Technol Ther. 2021 Nov;23(11):753-759. doi: 10.1089/dia.2021.0201. Epub 2021 Jul 20. PMID 34185606
- Tierney S, Webb K, Jones A, Dodd M, McKenna D, Rowe R, Whitehouse J, Deaton C. Living with cystic fibrosis-related diabetes or type 1 diabetes mellitus: a comparative study exploring health-related quality of life and patients' reported experiences of hypoglycaemia. Chronic Illn. 2008 Dec;4(4):278-88. doi: 10.1177/1742395308094240. PMID 19091936
- Tsabari R, Elyashar HI, Cymberknowh MC, Breuer O, Armoni S, Livnat G, Kerem E, Zangen DH. CFTR potentiator therapy ameliorates impaired insulin secretion in CF patients with a gating mutation. J Cyst Fibros. 2016 May;15(3):e25-7. doi: 10.1016/j.jcf.2015.10.012. Epub 2015 Nov 4. PMID 26547591
Идентификаторы
NCT: NCT07442682 · HUB2025082