Safety and Efficacy of Romiplostim in Treatment of Chronic ITP in Children

Обзор

The aim is to evaluate safety and efficacy of Romiplostim in the treatment of chronic ITP in children

Вмешательства

• Препарат Safety and efficacy of Romiplostim in Treatment of chronic ITP in children
  Children diagnosed with chronic immune thrombocytopenia (ITP) will receive Romiplostim, a thrombopoietin receptor agonist administered as a once-weekly subcutaneous injection. The initial dose will be 1 µg/kg, with subsequent dose adjustments (up to a maximum of 10 µg/kg weekly) based on platelet response, in order to maintain platelet counts ≥50 × 10⁹/L while minimizing the risk of bleeding. Treatment duration will be determined according to study protocol, and patients will be monitored regula

Первичные конечные точки

• Durable Platelet Response in Patients Receiving Treatment for Chronic ITP [Срок оценки: Up to 48 weeks of treatment]

Вторичные конечные точки (3)

• Time to Initial Platelet Response [Срок оценки: Up to 48 weeks of treatment.]
• Need for Rescue Medications [Срок оценки: Up to 48 weeks of treatment.]
• Rate of Treatment-Free Remission [Срок оценки: Up to 48 weeks of treatment.]

Критерии участия

Критерии включения

• Patients aged between 1 and 18 years
• Diagnosis of chronic ITP
• Refractory to or intolerant of first-line therapies (e.g.corticosteroids , intravenous immunoglobulin \[ IVIG \] , and oral eltrombopag )
• Platelet count<30,000/µL, or evidence of bleeding or risk of bleeding with platelet count<50,000/µL.
• Written informed consent (or parental/guardian consent for minors)

Критерии исключения

• Evidence of secondary thrombocytopenia (e.g., HIV, HCV, systemic lupus erythematosus)
• congenital thrombocytopenia
• Age < 1year or more than 18 years
• Aplastic anemia
• Known hypersensitivity to romiplostim or any of its components

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

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Другое

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Публикации

• Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, Godeau B, Lechner K, Mazzucconi MG, McMillan R, Sanz MA, Imbach P, Blanchette V, Kuhne T, Ruggeri M, George JN. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 200 PMID 19005182
• Chen F, McDonald V, Newland A. Experts' review: the emerging roles of romiplostim in immune thrombocytopenia (ITP). Expert Opin Biol Ther. 2021 Nov;21(11):1383-1393. doi: 10.1080/14712598.2021.1960979. Epub 2021 Aug 12. PMID 34313512
• Vianelli N, Auteri G, Buccisano F, Carrai V, Baldacci E, Clissa C, Bartoletti D, Giuffrida G, Magro D, Rivolti E, Esposito D, Podda GM, Palandri F. Refractory primary immune thrombocytopenia (ITP): current clinical challenges and therapeutic perspectives. Ann Hematol. 2022 May;101(5):963-978. doi: 10.1007/s00277-022-04786-y. Epub 2022 Feb 24. PMID 35201417
• Liu XG, Hou Y, Hou M. How we treat primary immune thrombocytopenia in adults. J Hematol Oncol. 2023 Jan 19;16(1):4. doi: 10.1186/s13045-023-01401-z. PMID 36658588
• Singh A, Uzun G, Bakchoul T. Primary Immune Thrombocytopenia: Novel Insights into Pathophysiology and Disease Management. J Clin Med. 2021 Feb 16;10(4):789. doi: 10.3390/jcm10040789. PMID 33669423
• Sandvad M, Pedersen EA, Frederiksen H, Mannering N. Risk of infection in adult patients with primary immune thrombocytopenia (ITP): a systematic review. Expert Rev Hematol. 2021 Oct;14(10):961-974. doi: 10.1080/17474086.2021.1976635. Epub 2021 Sep 16. PMID 34487679

Идентификаторы

NCT: NCT07214025 · Romiplostim In treatmentof ITP

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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