Concurrent naxitamab and lorlatinib in a patient with refractory high-risk neuroblastoma: a case report

Naxitamab is an anti-GD2 antibody currently approved in the United States as a treatment option for refractory and/or relapsed (R/R) high-risk (HR) neuroblastoma. With the identification of various mutations in tumor tissue by next-generation sequencing, patients with cancer increasingly have access to targeted therapies, including those directed at anaplastic lymphoma kinase (ALK) mutations. Here, we report a 12-year-old patient with refractory HR stage M neuroblastoma according to the International Neuroblastoma Risk Group staging system, with persistent bone and bone marrow involvement for 3 years, who was successfully treated with combined naxitamab and lorlatinib. After an ALK mutation was identified in the primary tumor tissue using the Pillar OncoReveal Multi-Cancer CNV RNA Fusion Panel, lorlatinib was initiated. Naxitamab therapy was subsequently added because of ongoing bone and bone marrow involvement. Our patient achieved complete remission with the combination of naxitamab and lorlatinib according to the International Neuroblastoma Response Criteria. Mild adverse events occurred during the combined immunotherapy, including transient hypotension managed with intravenous fluids and abdominal and leg pain that improved with analgesics. In conclusion, our case showed that combination therapy with naxitamab and lorlatinib may improve long-term outcomes and reduce chemotherapy-related toxicity.