2025 Volume 62 Issue 1 Pages 14-19
The Platelet Committee of the Japanese Society of Pediatric Hematology/Oncology published the Guidelines for Childhood Immune Thrombocytopenia in 2022. Besides changes in the name and stage of a disease, a modified Buchanan Bleeding Severity Classification system was introduced to assess bleeding symptoms. Based on a multidimensional assessment of platelet count, bleeding symptoms, activity level, lifestyle, and access to healthcare, the guidelines propose therapeutic interventions aimed at improving patients’ health-related quality of life. Presently, a more individualized approach is required. Aside from differential diagnosis recommendations, a system that facilitates collaboration with hematologists to determine treatment options is needed.
In Japan, the TPO receptor agonist rituximab, positioned as second-line therapy, has not yet received a dosage regimen for pediatric ITP, and each institution was forced to consider its introduction in accordance with its ethical guidelines. However, with the support of these guidelines, these drugs were approved for pediatric use in April of the same year. None of the treatments after third-line therapy are indicated for children, and new drugs that are safer and more effective should be developed. Target molecules for new drugs include spleen tyrosine kinase, Bruton’s tyrosine kinase inhibitors, and fetal Fc receptor inhibitors. We look forward to future developments in the treatment of pediatric ITP.
We will continue to disseminate these guidelines, examine the changes in clinical practice choices, and reflect on these changes in the following revision of the guidelines.