Generic romiplostim for children with persistent or chronic immune thrombocytopenia: Experience from a tertiary care
Chandana Mareddy1, Manas Kalra1, Anupam Sachdeva2
1Paediatric Haematology, Oncology and BMT Unit, Sir Ganga Ram Hospital, India.
Insights
Generic romiplostim effectively treats children with chronic immune thrombocytopenia (ITP). This Indian-manufactured drug demonstrated good tolerability and efficacy, with most patients achieving sustained platelet response without serious adverse events.
Area of Science:
- Pediatric Hematology
- Pharmacology
- Immunology
Background:
- Immune thrombocytopenia (ITP) is a bleeding disorder affecting children.
- Thrombopoietin receptor agonists (TPO-RAs) are a key treatment for ITP.
- Evaluating generic TPO-RAs is crucial for accessible pediatric ITP management.
Purpose of the Study:
- To assess the efficacy and safety of a generic romiplostim in children with persistent/chronic ITP.
- To determine the treatment response and sustained remission rates.
- To evaluate the tolerability of generic romiplostim in this pediatric population.
Main Methods:
- A prospective study of 40 children with persistent/chronic ITP treated with generic romiplostim (5 mcg/kg/week) for 20 weeks.
- Platelet counts were monitored weekly and at week 26.
- Dose adjustments followed a predesigned algorithm, with tapering and discontinuation for patients achieving target platelet counts.
Main Results:
- A platelet response (≥50 × 10^9/L) was observed in 65% (26/40) of patients by week 20.
- Sustained platelet response after stopping treatment was achieved in 55% (22/40) of children.
- No serious adverse events were reported, and treatment was well-tolerated.
Conclusions:
- Generic romiplostim is an effective and safe therapeutic option for pediatric persistent/chronic ITP.
- The drug facilitates achieving and maintaining adequate platelet counts.
- This study supports the use of affordable generic TPO-RAs in managing childhood ITP.
Abstract:
Thrombopoietin receptor agonists are important therapeutic option in children with immune thrombocytopenia (ITP). We evaluated the response, efficacy, safety of a generic form of romiplostim manufactured in India for treating children with persistent/chronic ITP at our centre. Study of 45 children with persistent/chronic ITP was conducted of which 5 discontinued and 40 were included between 2019-2020. Patients received romiplostim for 20 weeks, at a dose of 5 mcg/kg/week. Platelet count at week 1, 3, 20 and 26 was assessed. Predesigned algorithm was used for dose adjustment. After 20 weeks, patients who had platelet count of 50 × 109/L or above were tapered off medication and monitored till 26 weeks. Median platelet count at enrolment was 11 x 109/L (IQR 23 X 109/L). 13/40 children had received >/= three lines of prior ITP therapy. Platelet response (platelet count rise to more than 50×109/L without rescue medications) observed in 26 (65%) patients at week 20. Rescue medication was used in 12/40 children. Sustained platelet response after tapering and stopping romiplostim observed in 22/40 children. No adverse events were considered serious or led to discontinuation of treatment. Our data demonstrated generic romiplostim is well tolerated and efficacious in children with persistent/chronic ITP.
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