Research in context
Evidence before this study
Tisagenlecleucel is an anti-CD19 chimeric antigen receptor therapy that induced complete remission in 81% of children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia treated in the ELIANA trial, and had a manageable safety profile. Before our study, there was a paucity of information about quality of life in patients treated with chimeric antigen receptor therapy. We searched PubMed for articles published in English between inception and March 20, 2019, with the following search terms: “quality of life” or “patient reported outcomes” and “chimeric antigen receptor” or “tisagenlecleucel.” The search yielded one publication providing an overview of the opportunities and challenges of incorporation of patient-reported outcomes in clinical trials of chimeric antigen receptor cell therapy. We did not identify any papers reporting the results of patient-reported outcomes or quality of life in patients receiving this therapy.
Added value of this study
We present patient-reported quality-of-life data that were prospectively collected and analysed in the global, single-arm, open-label, phase 2 ELIANA trial of tisagenlecleucel for children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia. Our study findings suggest that broad-based clinically meaningful improvements in patient-reported quality of life occur as soon as 1–3 months after treatment with tisagenlecleucel and persist for 12 months. To our knowledge, these results represent the first report of patient-reported quality of life associated with a novel treatment that has demonstrated remarkable efficacy in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukaemia.
Implications of all the available evidence
Tisagenlecleucel was approved by the US Food and Drug Administration in 2017 and European Medicines Agency in 2018 for children and young adults up to age 25 years with B-cell acute lymphoblastic leukaemia that is refractory or in second or greater relapse. The rapid improvement in patient-reported quality of life scores presented here suggest that tisagenlecleucel is a promising treatment option for this patient population. Clinical trials of tisagenlecleucel in earlier lines of therapy are warranted.