Data collection in expanded access: real-world examples 29 Results A total of 48 expanded access requests from nine countries for patients with Epstein-Bar Virus driven diseases were received. Twenty-two patients from seven countries consented to this research: 16 Epstein-Bar Virus +PTLD and six Epstein-Bar Virus +non-PTLD. We primarily focused on the PTLD patients. Of the 16 PTLD patients, 15 received at least one dose of tabelecleucel. One patient had not started treatment at data cut-off date. Safety No adverse events were reported as related to tabelecleucel by the treating physician. There were no reports of tumor flare reaction, infusion-related reaction, cytokine release syndrome, marrow rejection, or transmission of infectious diseases, including cytomegalovirus. There were no events of graft versus host disease or organ rejection reported as related to tabelecleucel. Efficacy The overall response rate as assessed by the treating physicians was 60% (9/15, 95% CI: [32% , 83%]), which is comparable to the overall response rate of 51% (22/43, 95% CI: [35%, 66%]) observed in the pivotal regulatory studies.55,57 Limitations 1. Due to the heterogeneity of the disease population, only the results for patients with PTLD are presented. Even within the PTLD-population, variability across disease characteristics, such as viral load or prior therapies, makes it difficult to interpret the results from the expanded access program. 2. Not all patients consented to the research study and are hence lacking from the analysis. Nonetheless, as every cycle response was known, these data are available. How the exclusion of these patients effects the outcomes remains unclear. 3. Assessment of efficacy was done per clinical judgement by each individual treating physician. There was no central blinded review of the outcome results. Conclusion The successful implementation and the patient participation of this European program demonstrates the feasibility of administering an off-the-shelf, allogeneic, T-cell therapy and the unmet medical need in this disease area. Although the setting of evidence generation in this program was unblinded, and uncontrolled, the results across benefits and risks reflect the results obtained in the regulatory trials.57 All adverse events were consistent with the underlying diseases of the patient and were considered unrelated to tabelecleucel.The outcome data for patients with relapsed/refractory PTLD are comparable to data observed in clinical trials. The European expanded access program with Atara Biotherapeutics ended when tabelecleucel received marketing authorization for PTLD patients.55
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