Axicabtagene ciloleucel for aggressive B-cell lymphoma outside clinical trials

This study looked at axicabtagene ciloleucel (Yescarta) to treat B-cell non-Hodgkin lymphoma which has returned after previous treatment outside of clinical trials. It found that most patients responded to this immune therapy and that some patients had a complete response.

B-cell non-Hodgkin lymphomas (NHL) are a group of cancers of white blood cells known as B-cells. The most common variety is diffuse large B-cell lymphoma (DLBCL), which is a fast-growing cancer. DLBCL and other aggressive NHL can be treated with chemotherapy or with targeted therapies. However, it is more difficult to treat NHL which has relapsed (returned) after multiple treatments.

Axicabtagene ciloleucel (api-cel) is an immune therapy that uses a patient’s own T-cells (immune cells). Killer T-cells are white blood cells that remove cancer, bacteria, and other problematic cells. Each killer T-cell recognizes a specific molecule, and it will only attack cells which have that molecule. The api-cel treatment modifies the patient’s T-cells to recognize their cancer. Api-cel has been shown to treat relapsed B-cell NHL.

Api-cel takes an extended amount of time to modify the T-cells, which may be an issue for patients whose cancer is developing particularly quickly. The studies used to approve api-cel did not include all patients. It is not clear how api-cel works for the general patient population.

This study included 122 patients with B-cell NHL. The most common types of lymphoma were DLBCL (43%) and transformed follicular lymphoma (FL; 27%). The majority of patients (62%) would not have qualified for the ZUMA-1 trial which led to the approval of api-cel. Patients were followed for an average of 10.4 months.

70% of patients responded to api-cel. 50% of patients had a complete response, meaning the cancer was not detectable. Patients went an average of 4.5 months before the cancer progressed (worsened). Among patients who had a complete response, 79% did not have signs of cancer after six months. 

Patients with low inflammation prior to starting api-cel (C-reactive protein under 30 mg/L) went a significantly longer time before the cancer progressed.

93% of patients experienced cytokine release syndrome, which is inflammation throughout the body. 70% of patients had neurologic side effects, such as headaches, speech problems, or changes in vision.

This study found that api-cel is an effective treatment for relapsed B-cell lymphoma outside of clinical trials. 

Because this study lasted a short time, it is too soon to tell how long on average patients with a complete response have until the cancer progresses.