In January 2021, the FDA approved Xalkori (crizotinib; from Pfizer), an oral ALK inhibitor, for the treatment of young patients aged 1 to 21 years who are diagnosed with relapsed or refractory systemic anaplastic large-cell lymphoma (ALCL) and ALK mutation.
Xalkori was previously approved for patients with metastatic non–small-lung cancer and ALK or ROS1 mutation.
This new indication for the targeted therapy Xalkori was based on the results of a single-arm clinical trial in 26 patients aged 1 to 21 years who had been diagnosed with relapsed or refractory ALCL and ALK mutation. The study did not include older people with this diagnosis. All the patients had previously received at least 1 systemic treatment for this diagnosis.
In the study, 20 patients received treatment with Xalkori at a dose of 280 mg/m2 twice daily and the other 6 patients received Xalkori at a dose of 165 mg/m2 twice daily, until their disease progressed or until they had unacceptable side effects. The patients were allowed to discontinue treatment with Xalkori therapy so they could have a stem-cell transplant if they wanted.
Of the 26 patients in the study, 23 (88%) patients had a response to Xalkori therapy; furthermore, 81% of these patients had a complete remission (no sign of disease). Among the 23 patients who had a response to therapy, 39% had a response lasting 6 months or more, and 22% had a response lasting 12 months or more.
The most common side effects were diarrhea, vomiting, nausea, vision disorder, headache, musculoskeletal pain, stomatitis, fatigue, decreased appetite, fever, abdominal pain, cough, and pruritus.
Serious side effects, including neutropenia and infection, occurred in 35% of the patients.