The aim is to evaluate safety and efficacy of Romiplostim in the treatment of chronic ITP in children
Study Type
OBSERVATIONAL
Enrollment
100
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).
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).
Time frame: Up to 48 weeks of treatment
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.
Time frame: Up to 48 weeks of treatment.
Need for Rescue Medications
Proportion of patients requiring administration of rescue medications (e.g., corticosteroids, intravenous immunoglobulin) during Romiplostim therapy.
Time frame: 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.
Time frame: Up to 48 weeks of treatment.
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