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Идёт набор NCT06107582

Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)

— Наблюдательное Primary Immune Thrombocytopenia

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
В протоколе указаны: data collection.
Кому может быть актуально
Состояния в реестре: Primary Immune Thrombocytopenia. Базовые параметры: 6 лет — 17 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
Китай
Следующий шаг
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Официальное название

Clinical Characteristics, Prognosis and Prognostic Model of Pediatric Immune Thrombocytopenia: a Prospective, Multicenter, Observational Cohort Study

Обзор

Immune thrombocytopenic purpura (ITP) is a kind of rare childhood disease that involve autoimmune destruction of platelets.The current Pediatric ITP cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in pediatric ITP. The purpose of this study is to analyze the clinical characteristics of Pediatric ITP, the treatment methods, prognosis and prognostic model of these patients in China.

Подробное описание

Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.). ITP is one of the most common hemorrhagic diseases. At present, the treatment response of ITP is not good, and a considerable number of patients need drug maintenance treatment, which seriously affects the quality of life of patients and increases the economic burden of patients. Longitudinal Cohort allows to describe the long-term clinical characteristics of pediatric ITP patients, to study the benefit-risk balance of treatments, including the growing development of targeted therapies and to analyze the prognostic factors and attempts to establish prognostic models.

The study will include pediatric patients diagnosed with primary immune thrombocytopenia in the investigating hospitals, and collect basic information, diagnostic and treatment information from medical records. The study will use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.

Вмешательства

  • Другое data collection
    The study will collect basic information, diagnostic and treatment information from medical records and use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.

Первичные конечные точки

  • Overall response rate [Срок оценки: 3 years]
  • Time to onset response [Срок оценки: 3 years]
  • Duration of response [Срок оценки: 3 years]
  • Sustained response rate [Срок оценки: 3 years]
  • Emergency treatment [Срок оценки: 3 years]
  • Number of subjects with clinically significant bleeding as assessed using the bleeding scale for pediatric patients with ITP after initial administration of certain treatment [Срок оценки: 3 years]
  • Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale after initial administration of certain treatment [Срок оценки: 3 years]
  • Recurrence-free survival rate [Срок оценки: 3 years]
Вторичные конечные точки (7)
  • Incidence [Срок оценки: 3 years]
  • Distribution [Срок оценки: 3 years]
  • Prognosis related factors selected from transcriptome data [Срок оценки: 3 years]
  • Prognosis related factors selected from proteomics data [Срок оценки: 3 years]
  • Prognosis related factors selected from metabolomics data [Срок оценки: 3 years]
  • Prognosis related factors selected from microbiome data [Срок оценки: 3 years]
  • Incidence, severity, and relationship of treatment emergent adverse events after treatment [Срок оценки: 3 years]

Критерии участия

Критерии включения

  • Age 6-17 years old (including both ends), male and female;
  • Diagnosis of ITP.

Критерии исключения

  • Secondary thrombocytopenia caused by various reasons, such as connective tissue disorders, bone marrow hematopoietic failure disease, myelodysplastic syndrome, malignancy, drugs, inherited thrombocytopenia, common variable immune deficiency, lymphoma, etc.;
  • The expected follow-up period is less than 3 months.

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

Дизайн исследования

Модель наблюдения
Когортное

Центры проведения

Китай · 1 центр
  • Chinese Academy of Medical Science and Blood Disease Hospital — Тяньцзинь

Идентификаторы

NCT: NCT06107582 · IIT2023044

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗