Who is eflornithine suitable for?

Eflornithine tablets (eflornithine) is an oral ornithine decarboxylase (ODC) inhibitor. It is mainly suitable for patients with high-risk neuroblastoma (HRNB) who have partially responded or stabilized after treatment, especially those who are still at risk of recurrence after multiple modes of treatment. High-risk neuroblastoma is a highly aggressive neuroectodermal tumor that occurs frequently in children. Its treatment often includes complex regimens such as chemotherapy, stem cell transplantation, radiotherapy, and immunotherapy. Although these treatments are initially successful in relieving the disease, once relapse occurs, the prognosis is generally poor. Therefore, how to further maintain the efficacy and prolong disease-free survival has become a key goal in the post-treatment period.

The role of eflornithine in maintenance therapy is through long-term inhibition ofODC activity, reducing polyamine synthesis, interfering with tumor cell proliferation and differentiation, thereby inhibiting the development of potential residual lesions. It is suitable for patients who have completed previous multi-line treatments, including GD2-targeted immunotherapy, chemoradiotherapy, and hematopoietic stem cell transplantation, and have shown partial response (PR) or stable (SD) on imaging or biomarkers. Although the main lesions have been controlled in such patients, latent tumor cells may still relapse at any time, and eflornithine provides a maintenance strategy that is non-cytotoxic but has clear biological function regulation.

It is worth mentioning that although the drug was mainly used in infectious diseases such as African trypanosomiasis in the early days, its repositioning and development in the field of tumor maintenance treatment marks the in-depth expansion of precision medicine in the treatment of children's solid tumors. Eflornithine is particularly suitable for younger patients whose physical functions have not fully recovered after long-term treatment. It has higher tolerance than traditional cytotoxic drugs. This drug is currently being studied as a long-term maintenance drug for neuroblastoma in many European and American centers.

Reference materials:https://www.drugs.com/mtm/eflornithine.html