复发/难治性弥漫大 B 细胞淋巴瘤患者治疗偏好：一项离散选择实验

Aim: We quantified patient preferences for second-line diffuse large B-cell lymphoma therapies, including attributes of chimeric antigen receptor (CAR) T-cell therapy. Materials & methods: Using a discrete choice experiment, we surveyed 224 diffuse large B-cell lymphoma patients from the USA and Europe. Patients chose between two treatment options defined by six attributes with predefined levels for overall survival, adverse events (severe cytokine-release syndrome, severe neurological toxicities, severe infection) and time to return to pre-treatment functioning. Results: Increasing the probability of 1-year survival was most important to patients, followed by avoiding risks of cytokine-release syndrome and neurological toxicities. Respondents required a 13-14 percentage point increased 1-year survival probability to accept risks of treatment-associated adverse events. Conclusion: Patients prioritize survival and will accept certain adverse event risks to gain survival improvements.

Chimeric antigen receptor (CAR) T-cell therapy is a new treatment for patients with diffuse large B-cell lymphoma. CAR T-cell therapies are made from a patient s own cells, modified in a laboratory and used to attack cancer cells. While CAR T-cell therapies may increase long-term survival, they can also cause temporary but serious side effects, including neurological issues (e. g. , headache, confusion, brain swelling) and cytokine-release syndrome (CRS), an inflammatory condition that can cause fever, breathing difficulties and organ dysfunction.

To understand how patients perspectives of CAR T-cell therapy compared with their perspectives on other treatments for diffuse large B-cell lymphoma, we surveyed 224 patients in the USA and Europe. They were asked to choose between two treatments in a series of choice sets, each displaying varying levels of aspects of cancer therapies, including survival and risks of serious side effects. Their choices allowed us to measure which factors were most important to patients when making decisions about treatment.

We found that increasing the probability of survival was most important, followed by avoiding risks of neurological complications and CRS. Patients were willing to accept increased risks of neurological toxicities and CRS if they could obtain a 13 14 percentage point increase in the probability of surviving for at least 1 year after treatment.