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Published on: May 4, 2017
[Clinical analysis of lower doses rituximab for children primary immune thrombocytopenia]
Xiaofan Liu1, Yueting Huang1, Yunfei Chen1
1Thrombosis and Hemostasis Center, Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC, Tianjin 300020, China.
Insights
Lower doses of rituximab (375 mg/m²×1) demonstrated good therapeutic effects in children with chronic immune thrombocytopenia (ITP). Patients with anti-GPIIb/IIIa autoantibodies showed improved response, and the treatment was well-tolerated with no serious side effects.
Area of Science:
- Pediatric Hematology
- Immunology
- Pharmacology
Background:
- Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by low platelet counts.
- Chronic and persistent ITP in children often requires effective treatment options.
- Rituximab, a monoclonal antibody, has shown promise in treating ITP.
Purpose of the Study:
- To assess the efficacy and safety of a lower dose regimen of rituximab (375 mg/m²×1) in pediatric patients with persistent and chronic ITP.
- To identify factors influencing treatment outcomes in children with ITP receiving rituximab.
- To evaluate the tolerability and side effect profile of low-dose rituximab therapy.
Main Methods:
- A cohort of 50 children with persistent and chronic ITP were treated with a single lower dose of rituximab.
- Efficacy was measured by complete response (CR), response (R), and overall response (OR) rates at 3 and 6 months.
- Side effects and factors associated with treatment outcomes, including autoantibody status, were analyzed.
Main Results:
- An overall response rate of 58% at 3 months and 64% at 6 months was observed.
- Complete response was achieved in 34% of patients, with sustained platelet counts.
- Mild, transient side effects occurred in six patients; no serious adverse events were reported. Patients with anti-GPIIb/IIIa autoantibodies showed a better overall response.
Conclusions:
- Lower dose rituximab (375 mg/m²×1) is effective for children with persistent and chronic ITP.
- The presence of anti-GPIIb/IIIa autoantibodies predicts a better treatment response.
- Rituximab is a safe and well-tolerated treatment option for pediatric ITP.
Objective:
To evaluate the efficacy and safety of lower doses rituximab(375 mg/m²×1) in primary children immune thrombocytopenia (ITP).
Methods:
Fifty children [23 male and 27 female, the median age was 9.5 years (rage 3.5-17.0 years)]with persistent and chronic ITP were treated with lower doses rituximab from January 2009 to January 2013 in our hospital. Efficacy and side effects of lower doses rituximab was studied, and factors related to the outcomes were analyzed.
Results:
Among fifty patients, 17/50(34%) achieved a complete response (CR) and 15/50 (30%) patients got response (R). Patients with CR continued to maintain a platelet count above 50×10⁹/L at a median 12.3 (6-40) months. Patents with R continued to maintain a platelet count above 30×10⁹/L at a median 6 (2-12) months. The overall response (OR) in 3 and 6 months were 58% (29/50), 64% (32/50) respectively. Six patients have mild and transient side effects, including urticarial rash and fever, which were promptly resolved with appropriate therapy. Sex, age at diagnosis, interval from diagnosis to initial treatment with rituximab, platelet count at treatment and CD19+B cell count did not influence the overall response and complete response (P>0.05). Patients with anti-GPIIb/IIIa autoantibody had a better OR (P<0.05).
Conclusion:
Children with persistent and chronic ITP treated by lower doses rituximab had better therapeutic effects. Patients with anti-GPIIb/IIIa autoantibody had better response. Rituximab was well tolerated and no related serious side effects were recorded in the study.
