Single-center retrospective study of anaplastic lymphoma kinase inhibitors for neuroblastoma in adults

Adult-onset neuroblastoma (AON) differs significantly in biology and clinical behavior from childhood-onset disease. AON poses therapeutic challenges since tolerance of intensive multimodality therapies that are standard of care for pediatric neuroblastoma is poor. AON is enriched for somatic mutation including anaplastic lymphoma kinase (ALK), deemed to be an oncogenic driver in neuroblastoma. A retrospective study at Memorial Sloan Kettering Cancer Center (New York, NY) evaluated ALK inihibitors (ALKis) for AON. Fifteen patients were treated with a variety of ALKis. Of the 13 patients treated with lorlatinib, nine (65%) had complete or partial response, including among those previously treated with other ALKis. Lorlatinib was associated with significant adverse events, several requiring dose reductions; however, ongoing responses were seen even at doses below the recommended adult dose. Mediane survival form start of the first ALKi was 43 ± 26 months. The authors conclude that, while not without toxicity, ALKi is overall well tolerated in patients with AON, and its toxicity profile allows consideration of combination with chemotherapy.