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Long-term eltrombopag in children with chronic immune thrombocytopenia: A single-centre extended real-life
Zhifa Wang1, Nan Wang2, Ouyang Juntao3
1Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Insights
Long-term eltrombopag (ELT) use in children with chronic immune thrombocytopenia (cITP) shows sustained responses and no new safety concerns. Early treatment with thrombopoietin receptor agonists (TPO-RAs) in pediatric cITP demonstrates favorable outcomes.
Area of Science:
- Pediatric Hematology
- Immunology
- Pharmacology
Background:
- Eltrombopag (ELT) is effective for pediatric chronic immune thrombocytopenia (cITP).
- Long-term data and early use insights for TPO-RAs in children are limited.
- Further real-world evidence is needed to guide treatment strategies.
Purpose of the Study:
- To evaluate long-term treatment patterns and outcomes of eltrombopag in pediatric cITP patients.
- To assess the safety and efficacy of extended eltrombopag therapy.
- To identify potential predictive factors for loss of response to eltrombopag.
Main Methods:
- Retrospective review of a large institutional cohort of pediatric cITP patients.
- Analysis of treatment patterns, outcomes, and long-term follow-up data (up to June 2023).
- Comparison of patient characteristics between those with loss of response and those achieving sustained response.
Main Results:
- Over 3 years of follow-up, eltrombopag demonstrated sustained efficacy and safety in pediatric cITP.
- Nearly 30% of patients discontinued ELT due to stable response, with some achieving sustained response off-treatment.
- Loss of response occurred in 18.46% of patients, with initial platelet count and time to response potentially predicting LOR.
Conclusions:
- Long-term eltrombopag therapy is safe and effective for children with cITP.
- Sustained responses off-treatment are achievable for a subset of pediatric patients.
- Platelet count at ELT initiation and time to response may predict loss of response, guiding future treatment adjustments.
Abstract:
We have previously confirmed the efficacy and safety of eltrombopag (ELT) in children with chronic immune thrombocytopenia (cITP). However, data on both long-term exposure and early use of TPO-RAs are lacking, so further 'field-practice' evidence on treatment is required. Here, we report the long-term follow-up results (between September 2018 and June 2023) of our previous study. The main objective of this study was to retrospectively review our large institutional experience with ITP patients previously enrolled in our paediatric cITP study. We had more than 3 years of follow-up by June 2023 for treatment patterns and outcomes. A total of 65 patients (28 males) were enrolled, with a median age at ELT initiation of 6.34 (range 1.65, 14.13) years and a follow-up of 47.07 (36.00, 57.00) months, with 40.36 (10.53, 56.83) months of ELT therapy at the time of analysis. In total, 29.23% (19/65) of patients discontinued ELT due to stable response, and 18.46% (12/65) of patients switched to other ITP therapies due to loss of response (LOR) after 19.13 (14.53, 26.37) months. Of the 19 patients who discontinued ELT due to a stable response, 24.62% (16/65) achieved a 12 m sustained response off-treatment (SRoT); the last recorded platelet count ranged from 56 to 166 × 109 /L (median 107 × 109/L); and 4.62% (3/65) patients relapsed at 5, 6 and 9 months after discontinuation. Of the 12 patients who LOR to ELT after 19.13 (14.53, 26.37) months of therapy, four switched to avatrombopag, three switched to hetrombopag, two switched to traditional Chinese medicine (TCM), one underwent splenectomy and two received additional prednisolone under ELT treatment. Thirty-four patients who tapered and maintained a durable response. The patients with LOR and the patients with tapering were compared; the platelet count at the start of ELT is lower, and the time to response is longer in the patients with LOR. The platelet count at the start of ELT and the time to response may be the predictive factors for LOR during ELT treatment. We report more than 3 years of long-term clinical data on children with cITP using ELT. These data do not raise any new safety concerns regarding the long-term use of ELT in children with cITP.
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