Real-world experience with tabelecleucel within and beyond the approval label

Abstract

Tabelecleucel (tab-cel) is an allogeneic Epstein-Barr virus (EBV)–specific T-cell therapy approved in the European Union for relapsed or refractory (R/R) EBV-associated posttransplantation lymphoproliferative disorder (PTLD) after rituximab or immunochemotherapy failure. Despite its promise as the first approved option for treating the disorder, real-world evidence remains limited. The aim of this study was to investigate the scope of use and outcomes of tab-cel treatment in the real-world setting across Germany, Austria, and Switzerland. Eleven patients with EBV+ PTLD or other EBV-associated lymphoproliferative disorders (LPD) from 9 academic centers were included for a detailed analysis of baseline characteristics and survival parameters. Of these, 8 patients with PTLD (6 after solid organ transplantation and 2 after allogeneic hematopoietic cell transplantation) and 3 patients with EBV-associated LPDs were treated with a median of 2 cycles of tab-cel (range, 1-3). After a median follow-up of 15.1 months, 6 of 11 (55%) patients remained alive. Initially, 7 of 11 patients (64%) achieved an objective response, with best response occurring at a median of 31 days after the first tab-cel infusion (ie, end of cycle 1). Relapse and progression after tab-cel occurred in 7 of 11 (64%) patients. The estimated median overall survival was 13.4 months (confidence interval, 4.4-14.7), likely because of successful subsequent salvage treatments. Immunotherapy-related adverse events were rare. Tab-cel showed efficacy and low toxicity in patients with R/R PTLD/LPD, a vulnerable cohort of immunodeficient or patient who underwent transplantations. These real-world data help elucidate the optimal integration of tab-cel in the management of R/R PTLD.