The successful use of sorafenib to treat pediatric papillary thyroid carcinoma

Background: Pediatric papillary thyroid cancer (PTC) typically presents with more advanced disease as compared with adults, yet the prognosis, even in the presence of distant metastatic disease, is usually excellent. Systemic therapies other than radioactive iodine (RAI) are rarely required. Sorafenib is an orally administered multi-kinase inhibitor that has shown promise in the treatment of adults with advanced thyroid cancer, but it has not yet been studied in children with this disease. Objective: Our objective is to present an adolescent with a progressive RAI-resistant PTC who was successfully treated with sorafenib. Patient and Intervention: A 14-year-old girl had progressive lung metastases from PTC despite previous therapies with almost 400 mCi ¹³¹I. Sorafenib was initiated at a starting dose of 200 mg twice daily and decreased to a maintenance dose of 200 mg daily due to side effects that developed within 3 weeks of treatment. After 67 days on the lower dose, there was a dramatic improvement in the lung metastases. The patient was monitored while continuing thyrotropin-suppressive therapy only. Minimal regrowth of the pulmonary metastases was observed, and a second treatment course of sorafenib 200 mg every other day was equally successful in achieving a clinical response. Thyroglobulin autoantibodies declined over the course of therapy and observation. Conclusion: In the very rare case of the pediatric patient with progressive RAI-refractory PTC, for which no good systemic therapy exists, sorafenib may play an important role in the management of this disease.