Treatment of Dysphagia in Patients With Dystrophic Epidermolysis Bullosa With Budesonide: a Retrospective Bicentric Study Nice - Paris St Louis / Necker (EBUDE Study)

Обзор

Budesonide is an anti-inflammatory medication that works by decreasing the immune system's overreaction. In the treatment of eosinophilic esophagitis, especially with Jorveza or a preparation made in a pharmacy, it reduces the production of substances responsible for inflammation in the esophagus. This action reduces the accumulation of certain inflammatory cells (eosinophils) and thus improves the symptoms and lesions of the esophagus. Dystrophic epidermolysis bullosa (EBD) is a rare genetic disease that makes the skin and mucous membranes very fragile. Blisters, sores and wounds can appear as a result of even minor trauma. Scarring is often abnormal and can cause tissue to shrink or shrink. To date, only two studies involving a total of eight children with EBD have evaluated a budesonide-based preparation. The results show an improvement in swallowing difficulties (dysphagia) as well as good tolerance of the treatment. However, there are currently no published data regarding the use of Jorveza in the form of an orally disintegrating tablet in adults with EBD. Through the follow-up of patients treated at the MAGEC reference center, specialized in rare genetic diseases of the skin and mucous membranes, we aim to evaluate the interest and effects of budesonide treatment in individuals with EBD presenting with dysphagia.

Подробное описание

Budesonide is a non-halogenated glucocorticoid that has an anti-inflammatory effect by binding to glucocorticoid receptors. In the treatment of eosinophilic esophagitis, whether as Jorveza or as a compounded formulation, it inhibits the antigen-induced secretion of various cytokines and pro-inflammatory molecules such as thymic stromal lymphopoietin, interleukin-13, and eotaxin-3 in the esophageal epithelium (1). This action leads to a significant reduction in inflammatory eosinophil infiltrates in the esophagus.

Dystrophic epidermolysis bullosa (DEB) is a rare genetic disease that causes fragile skin and mucous membranes, leading to blisters and wounds after trauma. Healing is abnormal and often associated with scarring and contractures.

The literature only reports two pediatric studies, with a total of eight DEB patients treated with a compounded formulation (CF) budesonide-based. These studies have shown good tolerance and satisfactory effectiveness on dysphagia (2, 3). To date, there is no data on the use of Jorveza in orally disintegrating tablets in adults with EBD, even though it is recommended for this indication in official guidelines (4). Our goal is to evaluate, within a cohort followed at the MAGEC reference center, the usefulness of budesonide in PM/orally disintegrating tablets, in patients of all ages with EBD.

Вмешательства

• Другое The retrospective data collected will be entered into an eCRF and then analyzed centrally
  there is no treatment for the study but the patient take a treatment out of MMA and the retrospectives datas collected will be entered into an eCRF and then analyzed centrally by the sponsor

Первичные конечные точки

• overall patient satisfaction with the treatment [Срок оценки: month 1]
• overall patient satisfaction with the treatment [Срок оценки: month 3]

Вторичные конечные точки (5)

• The assessment of budesonide treatment tolerance in patients [Срок оценки: Month 3]
• Reduce meal time [Срок оценки: Month 0]
• Reduce meal time [Срок оценки: Month 1]
• oesophagal expansion number [Срок оценки: Month 0]
• oesophagal expansion number [Срок оценки: Month 1]

Критерии участия

Критерии включения

Patients of both sexes, of any age, with EBD in dominant or recessive form, treated for at least one month with budesonide for dysphagia resistant to usual treatments.

A description of the clinical form must be available in the file. The patient must assess the benefit of treatment at 1 and/or 3 months of Budesonide.

Patients must be affiliated with social security. For adults, consent of non-opposition is required, and for minors, non-opposition from one of the parents or the person holding parental authority must be collected.

Критерии исключения

No precise clinical or biological diagnosis. Treatment with Budesonide < 1 month. No evaluation criteria in the file / Objection to the use of data (withdrawal of non-opposition)

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Франция · 3 центра

• CHU de Nice — Nice
• APHP Necker — Paris
• APHP St Louis — Paris

Идентификаторы

NCT: NCT07684105 · 26-PP-03 · IDRCB

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗