Tabelecleucel for EBV+ PTLD after allogeneic HCT or SOT in a multicenter expanded access protocol

Area of Science:

• Immunology
• Oncology
• Transplantation Medicine

Background:

• Epstein-Barr virus-positive posttransplant lymphoproliferative disease (EBV+ PTLD) has limited treatment options and poor survival rates, especially after initial treatment failure.
• Patients undergoing allogeneic hematopoietic cell transplant (HCT) or solid organ transplant (SOT) are at risk for developing EBV+ PTLD.

Purpose of the Study:

• To evaluate the efficacy and safety of tabelecleucel, an allogeneic EBV-specific cytotoxic T-lymphocyte immunotherapy, for patients with relapsed/refractory (R/R) EBV+ PTLD.
• To assess outcomes in patients who received tabelecleucel after HCT or SOT.

Main Methods:

• A multicenter expanded access protocol was conducted.
• Patients with R/R EBV+ PTLD after HCT (n=14) or SOT (n=12) received tabelecleucel treatment.
• Objective response rate and overall survival (OS) rates were assessed.

Main Results:

• An overall objective response rate of 65.4% was observed (38.5% complete response, 26.9% partial response).
• The 1- and 2-year OS rates were 70.0% overall, with higher rates in SOT recipients (81.5%) compared to HCT recipients (61.5%).
• Responders showed significantly better OS (94.1%) than non-responders (0%). Treatment was well-tolerated.

Conclusions:

• Tabelecleucel demonstrates clinically meaningful efficacy and a favorable safety profile for R/R EBV+ PTLD post-HCT and post-SOT.
• This immunotherapy represents a potentially transformative treatment advance for this patient population.
• Tabelecleucel offers an accessible therapeutic option for patients with limited alternatives.