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Daily Medical Update
Immune thrombocytopenic purpura
Tuesday, May 05, 2026
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🔬 Practice-Changing Findings
Evidence from RCTs and meta-analyses published in the
last 12 months.
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JAMA (2025) - Randomized Controlled Trial
Key Findings
- Sustained platelet response occurred in 65% of children receiving eltrombopag versus 35% with standard therapy, a 30% absolute difference.
- There was no increase in adverse events versus standard therapy, and the number and type of events were similar between groups.
📋 Practice Implication: Eltrombopag is a reasonable front-line treatment option for children with newly diagnosed ITP who need medication but do not need an immediate rescue-level platelet rise.
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The Lancet. Haematology (2025) - Randomized Controlled Trial
Key Findings
- Durable platelet response was achieved in 28% of patients on avatrombopag versus 0% on placebo.
- Any platelet response occurred in 81% with avatrombopag versus 0% with placebo, with no deaths, thromboembolic events, or grade 3 or higher bleeding reported.
📋 Practice Implication: For pediatric persistent or chronic ITP after prior treatment failure, avatrombopag adds an oral TPO-RA option with clear efficacy and a reassuring short-term safety signal.
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Expert review of hematology (2025) - Network Meta-Analysis
Key Findings
- Across 29 randomized trials, avatrombopag 20 mg had the highest platelet response versus placebo with a risk ratio of 12.23.
- Eltrombopag plus danazol reduced bleeding risk the most, avatrombopag 5 mg had the lowest serious adverse event rate, and dose-adjusted romiplostim showed one of the most favorable overall benefit-risk profiles.
📋 Practice Implication: Second-line ITP therapy can be selected more deliberately by matching the clinical goal to the comparative signal, such as maximizing platelet response, minimizing bleeding, or limiting serious toxicity.
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Thrombosis research (2025) - Systematic Review
Key Findings
- Among 100,446 patients with ITP, the pooled thrombosis incidence was 6.03% and increased to 10.43% after adjustment for publication bias.
- Thrombotic risk increased with advanced age, lupus anticoagulant positivity, elevated anticardiolipin IgG, hypertension, multiple prior therapies, secondary ITP, and thrombopoietin receptor agonist use.
📋 Practice Implication: ITP management should include explicit thrombosis risk assessment rather than focusing only on bleeding, especially when considering TPO-RAs or treating older patients with vascular comorbidity.
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BMJ (Clinical research ed.) (2025) - Randomized Controlled Trial
Key Findings
- Overall response at week 8 was 83% with CM313 versus 20% with placebo.
- Median time to platelet counts of at least 50 x 10^9/L was 1 week with CM313 and was not reached with placebo, while median cumulative response duration was 18 weeks versus 3 weeks.
📋 Practice Implication: CM313 appears promising for adults with persistent or chronic primary ITP after glucocorticoid failure and may become a useful salvage option if larger trials confirm benefit and safety.
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Summary
Recent ITP evidence sharpens second-line decision-making while expanding pediatric thrombopoietin receptor agonist use into both newly diagnosed and persistent or chronic disease. Meta-analytic data also reinforce that thrombosis is a clinically relevant comorbidity in ITP, and an early phase anti-CD38 study suggests another immunologic option for adults with persistent or chronic disease.
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