Is Besudil mesylate tablets safe for children with cGVHD? Can be used by those under 12 years old?

Belumosudil mesylate tablets (Belumosudil) is an oral Rho-associated coiled-coil-forming protein kinase 2 (ROCK2) inhibitor, mainly used to treat adults and children over 12 years old who still have chronic graft-versus-host disease (cGVHD) after multiple lines of systemic therapy. This drug plays a dual role of immunomodulation and anti-inflammatory by regulating T cells, B cells and fibrosis signaling pathways. It is one of the few new targeted drugs that has shown sustained efficacy in cGVHD. Although clinical studies and FDA approval support its use in children 12 years and older, there is currently insufficient data to clarify its safety and efficacy in children under 12 years old.

In the United StatesIn the official instructions of the FDA, the indication scope of Besudil Mesylate Tablets is limited to patients 12 years old and above. This is mainly based on the age limit of participants in clinical trials. Most studies do not include children under the age of 12. Therefore, its use in younger children is still in the research or caution stage as the scientific evidence is not yet complete. Although ROCK2 inhibition may theoretically bring benefits to patients in a wider age range, immature immune systems may respond differently to the drug, especially in long-term use, where potential side effects such as abnormal liver function and increased risk of infection require closer monitoring.

In addition, there are currently no authoritative pediatric studies proving whether the risks and benefits of long-term use of this drug are balanced in patients under 12 years old. Therefore, for children with cGVHD under 12 years old, the necessity of treatment and potential risks must be weighed before using besudil. It is recommended that the decision whether to use it on a case-by-case basis be made under the guidance of experienced transplantation experts and must be carried out under strict monitoring.

Reference: https://www.drugs.com/pro/rezurock.html