• Nico Gagelmann, Francis Ayuk, Djordje Atanackovic, and Nicolaus Kröger.
• Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
• Eur. J. Haematol. 2020 Apr 1; 104 (4): 318-327.

IntroductionChimeric antigen receptor (CAR) T cells targeting B cell maturation antigen (BCMA) have shown impressive results in clinical studies for relapsed/refractory multiple myeloma (RRMM). We performed a systematic literature review to summarize the current body of evidence on the role of anti-BCMA CAR T cells for RRMM.Objectives And MethodsFifteen studies comprising a total of 285 patients with heavily pretreated RRMM were included using a conventional meta-analysis. Main efficacy outcomes were response, relapse, and survival. Safety outcomes were cytokine release syndrome (CRS) and neurotoxicity.ResultsAnti-BCMA CAR T cells resulted in a pooled overall response of 82% (95% confidence interval [CI], 74%-88%) and complete response of 36% (24%-50%). Higher CAR+ cell doses were associated with higher response rates. The pooled relapse rate of responders was 45% (27%-64%), and median progression-free survival was 10 months. Present extramedullary disease did not show worse outcome. Severe CRS grades 3-4 and neurotoxicity occurred in 15% (10%-23%) and 18% (10%-31%).ConclusionAnti-BCMA CAR T cells showed high response rates, even in patients with present extramedullary disease, while relapse occurred in half of the patients who achieved a response. Larger studies with longer follow-up especially evaluating the association of response and survival are needed.© 2019 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.

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