MON-453 Dramatic Clinical Response to Lenvatinib in a Pediatric Patient with Advanced Metastatic Papillary Thyroid Carcinoma

Abstract Introduction: Papillary thyroid cancer (PTC) is the most common thyroid tumor in childhood. Most patients are referred with locally advanced and/or distant disease at the time of diagnosis. Whenever possible, these patients should be offered total thyroidectomy and radioiodine remnant ablation; however, this approach is not always feasible, rendering these tumors unresectable. These critical cases could benefit from neoadjuvant treatment with multikinase inhibitors (MKI) so that standard treatment can be performed. Lenvatinib is an MKI recently approved in many countries throughout the world for the treatment of radioiodine refractory adult differentiated thyroid cancer. Only few pediatric cases have been reported. Case report: A 10-year-old female patient with locally advanced PTC and metastasis to the lungs, who required 3 liters of oxygen due to respiratory failure caused by bilateral miliary lung disease, mistakenly treated as tuberculosis two months previously and referred to our Hospital. A large thyroid mass adhered to deep tissues was confirmed on CT scan, showing a large heterogeneous neck mass with multiple microcalcifications associated with multiple lymph nodes. Both lungs had multiple micro-nodular disease with interstitial involvement. Total thyroidectomy together with lymph-node dissection was planned, but extensive local infiltration made the lesion unresectable and surgery was limited to a thyroid biopsy. The patient required respiratory assistance. Histopathology confirmed the presence of a PTC (diffuse sclerosing variant) with a RET-PTC3 oncogene rearrangement. Eight days after surgery the patient was critical and we decided to indicate the compassionate use of Lenvatinib. The patient was started on oral lenvatinib at a dose of 14 mg daily (14 mg/m/day). Three days later, she clinically improved and nine days post-lenvatinib initiation, the patient was discharged from hospital without need for oxygen therapy. Lab studies showed a rise in thyroglobulin levels in the first month of treatment followed by a significant drop. All Lung Function Test parameters significantly improved. The patient initially had severe restrictive breathing and due to the resting dyspnea with hypoxemia she could not perform the walking test. Two months after treatment onset she could walk 360 meters in six minutes with 96% oxygen saturation. After 4 months on Lenvatinib, imaging studies showed a stable thyroid mass while the pulmonary nodules appeared stable to slightly smaller without evidence of new or progressive disease. Conclusion: On lenvatinib treatment, our patient showed significant clinical improvement, arrest of disease progression, and stable disease on imaging studies. This case shows that lenvatinib may be a beneficial option for children with advanced PTC not amenable to surgery/RAI treatment and may be used as a bridge to these first-line therapies.