Sustained Therapeutic Efficacy of Intravenous Plasminogen Concentrate in Pediatric Patients With Type 1 Plasminogen Deficiency: An Analysis of Dosing Parameters and Clinical Outcomes.
Pediatr Blood CancerJul 29, 2026 (epub)
Charles Nakar, Karen Thibaudeau, Mary Mathias et al.
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In 30 seconds
This Phase II/III clinical trial analyzed the efficacy of intravenous plasminogen concentrate in 17 pediatric patients with Type 1 plasminogen deficiency (PLGD-1). The treatment significantly elevated plasminogen levels, leading to lesion resolution in patients, with baseline plasminogen activity ranging from <2% to 40% (median: 27%).
Key findings
- All patients in compassionate use programs had at least one lesion at enrollment.
- 79% of patients with lesions had conjunctival lesions, and 43% had respiratory lesions.
- Therapeutic plasminogen levels were achieved and maintained in children.
Why it matters
Understanding the efficacy of intravenous plasminogen concentrate is crucial for clinicians managing pediatric patients with PLGD-1, as timely intervention can prevent severe complications and improve patient outcomes.