ITP, Romiplostim N01
Conditions
Brief summary
The aim is to evaluate safety and efficacy of Romiplostim in the treatment of chronic ITP in children
Interventions
DRUGSafety 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 regularly for efficacy (platelet count response, bleeding events) and safety (adverse events, laboratory parameters).
Sponsors
Assiut University
Study design
Observational model
OTHER
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
No
Inclusion criteria
* 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)
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Durable Platelet Response in Patients Receiving Treatment for Chronic ITP | Up to 48 weeks of treatment | 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). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to Initial Platelet Response | Up to 48 weeks of treatment. | Time (in days) from initiation of Romiplostim therapy to the first platelet count ≥50×10⁹/L without rescue medication in the preceding 7 days. |
| Need for Rescue Medications | Up to 48 weeks of treatment. | Proportion of patients requiring administration of rescue medications (e.g., corticosteroids, intravenous immunoglobulin) during Romiplostim therapy. |
| Rate of Treatment-Free Remission | Up to 48 weeks of treatment. | Proportion of patients who maintain platelet counts ≥50×10⁹/L for at least 24 consecutive weeks after discontinuation of Romiplostim without rescue therapy. |
Contacts
Primary ContactMostafa Refat Mostafa, Principal Investigator
Outcome results
None listed