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Идёт набор NCT05919433

Detection Program for Patients With Primary Biliary Cholangitis Lost in the System

— Наблюдательное Primary Biliary Cholangitis

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: Primary Biliary Cholangitis. 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
Испания
Следующий шаг
Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Обзор

Primary biliary cholangitis (PBC) has been considered a rare disease and its management has been limited by the lack of therapeutic alternatives. PBC is a slowly progressing chronic liver disease characterized by an immune-mediated destruction of the intrahepatic bile ducts, which leads to cholestasis, portal inflammation, and ultimately liver cirrhosis and its associated complications (ascites, portal hypertension, etc), if not treated effectively. Thus, early diagnosis and close management of these patients with PBC is essential. First-line treatment with ursodeoxycholic acid (UDCA) improves liver biochemical parameters, delays histological progression, and increases liver transplant-free survival and overall survival. However, up to 40% of patients are non-responders to UDCA. Obeticholic acid (OCA) is recommended as second-line therapy in combination with UDCA for patients with an inadequate response to UDCA or as monotherapy in cases of UDCA intolerance. According to current clinical guidelines, the diagnosis of PBC includes a combination of elevated alkaline phosphatase (ALP) levels and the presence of anti-mitochondrial antibodies (AMA) (titer \>1:40) and/or anti-nuclear antibodies (ANA) anti-gp210 or anti-sp100. AMA are highly sensitive and specific for PBC and are detected in nearly 95% of PBC patients. A liver biopsy is not necessary unless there is an elevation of ALP without the presence of specific AMA and/or anti-gp210 or anti-sp100 ANA or if coexistence with other liver diseases is suspected (autoimmune hepatitis, hepatic steatosis). The incidence of PBC has increased in recent years due to an increase in the diagnosis of cases in the initial phases, better awareness in the medical community and the development of more sensitive diagnostic tests. However, up to 31% of patients with PBC are lost without follow-up. The correct identification of patients with PBC is essential so that they can benefit from an adequate treatment and modify disease progression. To date, two studies (one Spanish and one Portuguese) showed that 27% and 45.5% of the patients lost with PBC presented advanced fibrosis, respectively. The objective of this study is to identify, through computerized data, patients with PBC who may be lost in the system and evaluate their clinical, analytical and demographic characteristics, and in a second phase, provide access to follow-up in specialized consultations.

Первичные конечные точки

  • Percentage of patients correctly diagnosed with PBC and monitored in specialized health care compared to lost to follow-up PBC patients [Срок оценки: at inclusion]

Критерии участия

Критерии включения

  • Patients with positive AMA and/or with positive ANA anti-gp210 or anti-sp100 identified in the computer databases of each of the participating centers.
  • Adults (≥18 years)

Критерии исключения

  • Patients with Overlap Syndrome (PBC overlap with Autoimmune Hepatitis (AIH))

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

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения
Только случаи

Центры проведения

Испания · 1 центр
  • Hospital Universitari MútuaTerrassa — Terrassa

Публикации

  • Carey EJ, Ali AH, Lindor KD. Primary biliary cirrhosis. Lancet. 2015 Oct 17;386(10003):1565-75. doi: 10.1016/S0140-6736(15)00154-3. Epub 2015 Sep 11. PMID 26364546
  • Kaplan MM, Gershwin ME. Primary biliary cirrhosis. N Engl J Med. 2005 Sep 22;353(12):1261-73. doi: 10.1056/NEJMra043898. No abstract available. PMID 16177252
  • European Association for the Study of the Liver. EASL Clinical Practice Guidelines: The diagnosis and management of patients with primary biliary cholangitis. J Hepatol. 2017 Jul;67(1):145-172. doi: 10.1016/j.jhep.2017.03.022. Epub 2017 Apr 18. PMID 28427765
  • Mayo MJ. Mechanisms and molecules: What are the treatment targets for primary biliary cholangitis? Hepatology. 2022 Aug;76(2):518-531. doi: 10.1002/hep.32405. Epub 2022 Mar 10. PMID 35152430
  • Pares A; Leading PBC Group. Practical management of primary biliary cholangitis. Rev Esp Enferm Dig. 2022 Jul;114(7):410-417. doi: 10.17235/reed.2021.8219/2021. PMID 34663072
  • Leung KK, Hirschfield GM. Autoantibodies in Primary Biliary Cholangitis. Clin Liver Dis. 2022 Nov;26(4):613-627. doi: 10.1016/j.cld.2022.06.004. PMID 36270719
  • Olveira-Martin A, Yebra-Carmona J, Amaral-Gonzalez C, Tejedor M, Eiras P, Hernandez-Perez M, Suarez-Cabredo C, Spigarelli-de Rabago I, Suarez-Ferrer C, Morales-Arraez D, Chico I, Diaz-Flores F, Rodriguez R, Llorente S, Molina-Perez E, Hernandez-Guerra de Aguilar MN. Retrieval and treatment of patients with primary biliary cholangitis who are lost in the health system. Rev Esp Enferm Dig. 2021 Nov; PMID 34470449
  • Garrido I, Liberal R, Cardoso MJ, Macedo G. The impact of undiagnosed primary biliary cholangitis. Eur J Gastroenterol Hepatol. 2021 Dec 1;33(1S Suppl 1):e1027-e1031. doi: 10.1097/MEG.0000000000002268. PMID 34402472

Идентификаторы

NCT: NCT05919433 · RESCAT

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗