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The aim is to evaluate safety and efficacy of Romiplostim in the treatment of chronic ITP in children
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Safety and efficacy of Romiplostim in Treatment of chronic ITP in children | Drug | 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 regularly for efficacy (platelet count response, bleeding events) and safety (adverse events, laboratory parameters). |
| Measure | Description | Time Frame |
|---|---|---|
| Durable Platelet Response in Patients Receiving Treatment for Chronic ITP | The proportion of patients achieving a durable platelet response, defined as platelet counts ≥50 × 10⁹/L for at least 6 of the final 8 weeks of a 48-week treatment period, without the need for rescue therapy (e.g., IVIG, corticosteroids, platelet transfusion). | Up to 48 weeks of treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Initial Platelet Response | Time (in days) from initiation of Romiplostim therapy to the first platelet count ≥50×10⁹/L without rescue medication in the preceding 7 days. | Up to 48 weeks of treatment. |
| Need for Rescue Medications |
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Inclusion Criteria:
Exclusion Criteria:
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The study will include pediatric patients aged 1 to 18 years with a diagnosis of chronic immune thrombocytopenia (ITP) who are refractory to or intolerant of first-line therapies (e.g., corticosteroids, intravenous immunoglobulin, or oral eltrombopag). Eligible patients must have a platelet count <30,000/µL, or evidence of bleeding/risk of bleeding with platelet count <50,000/µL. Patients with secondary or congenital thrombocytopenia, aplastic anemia, age <1 year or >18 years, or known hypersensitivity to romiplostim will be excluded. A total of 100 patients will be enrolled from the Pediatric Department at Assiut University Hospital.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Mostafa Refat Mostafa, Principal Investigator | Contact | +201140514880 | moustafa.17298696@aun.edu.eg |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 19005182 | Result | 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. 2009 Mar 12;113(11):2386-93. doi: 10.1182/blood-2008-07-162503. Epub 2008 Nov 12. | |
| 34313512 |
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| ID | Term |
|---|---|
| D012449 | Safety |
| ID | Term |
|---|---|
| D000056 | Accident Prevention |
| D000059 | Accidents |
| D011634 | Public Health |
| D004778 | Environment and Public Health |
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Proportion of patients requiring administration of rescue medications (e.g., corticosteroids, intravenous immunoglobulin) during Romiplostim therapy.
| Up to 48 weeks of treatment. |
| Rate of Treatment-Free Remission | Proportion of patients who maintain platelet counts ≥50×10⁹/L for at least 24 consecutive weeks after discontinuation of Romiplostim without rescue therapy. | Up to 48 weeks of treatment. |
| Result |
| 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. |
| 35201417 | Result | 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. |
| 36658588 | Result | 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. |
| 33669423 | Result | 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. |
| 34487679 | Result | 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. |