The Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids
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: CBC.
- Кому может быть актуально
- Состояния в реестре: Persistent or Chronic ITP Not Respnding to Steroids. Базовые параметры: 1 год — 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
- Египет
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Обзор
Immune thrombocytopenic purpura (ITP) is an autoimmune disease characterized by a low platelet count, purpura, and hemorrhagic episodes caused by antiplatelet autoantibodies. The diagnosis is typically made by excluding the known causes of thrombocytopenia. IgG autoantibodies sensitize the circulating platelets. It leads to the accelerated removal of these cells by antigen-presenting cells (macrophages) of the spleen and sometimes the liver or other components of the monocyte-macrophage system. The bone marrow compensates for platelet destruction by increasing platelet production. ITP most often occurs in healthy children and young adults within a few weeks following a viral infection . New treatment guidelines have supported a shift from corticosteroids and splenectomy to newer medical treatments that mitigate the thrombocytopenia and avoid splenectomy. The thrombopoietin receptor agonists (TPO-RA), romiplostim, eltrombopag have markedly altered the treatment of ITP. The thrombopoietin receptor agonists (TPO-Ras) romiplostim and eltrombopag have shown high therapeutic activity In primary ITP. Romiplostim, a thrombopoiesis-stimulating peptibody, represents a new therapeutic option in adult refractory chronic immune thrombocytopenia (ITP). This study aimed to assess the short-term efficacy and safety of romiplostim in children withChronic ITP. The most commonly reported drug-related adverse effects include headache, nausea, and hepatobiliary laboratory abnormalities. Long-term safety data in children are limited, and studies in adults have not revealed a clinically significant increased incidence of thrombosis, marrow fibrosis, or cataract formation. TPO-RA do not need to be continued forever; about a third of patients In the first year and about another third after two years have a remission. Whether TPO-RA affect the ITP pathophysiology and directly cause remission remains unclear. This review provides a personal overview of the diagnosis and treatment of ITP with a focus on the mechanism of action of TPO-RA, their place in the treatment algorithm, unique aspects of their clinical use, adverse effects.
Вмешательства
- Диагностический тест CBC
Platelets counts in pediatric patient with perisistent or chronicITP unresponsive to steroids
Первичные конечные точки
- Platelets counts in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids on thrombopoietin receptor agonists [Срок оценки: 12 months]
Критерии участия
Критерии включения
pediatric patients with persistent or chronic Itp on thrombopoietin receptor agonists unresponsive to steroid.
Критерии исключения
Any patient with other causes of thrombocytopenia. Any patient with chronic disease . Any patient with Acute ITP . Any newly diagnosed patient with ITP.
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Распределение
- Не применимо
- Модель
- Параллельные группы
- Маскирование
- Открытое
- Основная цель
- Организация здравоохранения
Центры проведения
Египет · 1 центр
- Sohag University hospitals — Sohag
Публикации
- Zaja F, Barcellini W, Cantoni S, Carpenedo M, Caparrotti G, Carrai V, Di Renzo N, Santoro C, Di Nicola M, Veneri D, Simonetti F, Liberati AM, Ferla V, Paoloni F, Crea E, Volpetti S, Tuniz E, Fanin R. Thrombopoietin receptor agonists for preparing adult patients with immune thrombocytopenia to splenectomy: results of a retrospective, observational GIMEMA study. Am J Hematol. 2016 May;91(5):E293-5. PMID 26910388
- Mokhtar GM, Tantawy AA, El Sherif NH. Romiplostim therapy in children with unresponsive chronic immune thrombocytopenia. Platelets. 2012;23(4):264-73. doi: 10.3109/09537104.2011.619601. Epub 2012 Apr 3. PMID 22471399
- Bussel JB, Soff G, Balduzzi A, Cooper N, Lawrence T, Semple JW. A Review of Romiplostim Mechanism of Action and Clinical Applicability. Drug Des Devel Ther. 2021 May 26;15:2243-2268. doi: 10.2147/DDDT.S299591. eCollection 2021. PMID 34079225
- Giordano P, Lassandro G, Barone A, Cesaro S, Fotzi I, Giona F, Ladogana S, Miano M, Marzollo A, Nardi M, Notarangelo LD, Pession A, Ruggiero A, Russo G, Saracco P, Spinelli M, Tolva A, Tornesello A, Palladino V, Del Vecchio GC. Use of Eltrombopag in Children With Chronic Immune Thrombocytopenia (ITP): A Real Life Retrospective Multicenter Experience of the Italian Association of Pediatric Hematolo PMID 32181255
Идентификаторы
NCT: NCT06568913 · Soh-Med-24-07-12MS