Kite, a Gilead Company, announced compelling real-world evidence supporting the safety and effectiveness of outpatient administration of Yescarta (axicabtagene ciloleucel) for patients with relapsed/refractory large B-cell lymphoma (R/R LBCL). The data, presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, demonstrates that patients treated in outpatient settings achieved comparable outcomes to those receiving inpatient care.
Study Design and Patient Population
The analysis utilized data from the Center for International Blood and Marrow Transplant Research (CIBMTR) registry, collected between July 2021 and November 2023. Researchers evaluated 238 patients with R/R LBCL from 75 treatment centers, comparing 119 individuals assigned to receive Yescarta in outpatient settings to 119 patients assigned to inpatient treatment.
Patient groups were carefully matched using propensity scoring across multiple variables including age, sex, comorbidities, lactate dehydrogenase (LDH) levels, bulky disease, prior lines of therapy, chemosensitivity, and infusion year. The outpatient group had a median age of 63 years, with 25% being 70 years or older, 66% male, and 67% having at least one comorbidity. Half had elevated LDH levels, 73% had received one prior line of therapy, 3% had bulky disease, and 60% had chemo-resistant disease.
Safety and Effectiveness Outcomes
With a median follow-up of 12 months, the study found no general differences between outpatient and inpatient groups in rates of cytokine release syndrome, neurologic events, or immune effector cell-associated neurotoxicity syndrome grade ≥3. These findings are particularly significant given that these toxicities represent the primary safety concerns with CAR-T cell therapy.
"The encouraging results from this study corroborate reports from treatment centers and prior studies in the real world that illustrate the potential feasibility of administering axi-cel in the outpatient setting for people with relapsed or refractory large B-cell lymphoma," said Dr. Fateeha Furqan, lead investigator at The University of Texas MD Anderson Cancer Center.
Healthcare System Impact
The study revealed notable benefits for healthcare resource utilization. Almost one-fourth of the 119 patients assigned to receive Yescarta in the outpatient setting did not require hospital admission within 30 days, while half did not require hospital admission within 3 days. These findings suggest substantial potential for reducing healthcare system burden while maintaining patient safety.
Dr. Furqan emphasized the cost-effective advantages, noting that "outpatient administration of axi-cel has cost-effective advantages over inpatient treatment, including less burden on hospitals. These clinical results reflect the fact that the knowledge and experience needed to safely administer the therapy has only grown since axi-cel was first approved in 2017."
Clinical Context and Implications
Large B-cell lymphoma represents the most common type of non-Hodgkin lymphoma globally, with more than 18,000 people diagnosed annually in the United States. Approximately 30-40% of patients with LBCL require second-line treatment due to disease relapse or refractoriness to initial therapy.
Yescarta is a CD19-directed genetically modified autologous T cell immunotherapy approved for multiple indications, including adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or relapses within 12 months of first-line treatment, and patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy.
Expanding Treatment Access
Dominique Tonelli, M.D., Vice President and Global Head of Medical Affairs at Kite, highlighted the broader implications: "We are seeing a growing body of evidence that suggests outpatient administration of Yescarta could be reliable and safe in suitable settings, with benefits for the patient, their family and healthcare system. We are confident that these promising results will help inform providers to expand the number of patients who can achieve the curative potential of a one-time treatment with Yescarta."
The real-world data presented are not currently reflected in the U.S. Prescribing Information, but may inform future clinical practice guidelines and regulatory considerations for CAR-T cell therapy administration settings.
Safety Profile Considerations
Despite the encouraging outpatient data, Yescarta carries significant safety warnings, including boxed warnings for cytokine release syndrome, neurologic toxicities, and secondary hematological malignancies. The therapy is available only through a restricted Risk Evaluation and Mitigation Strategy (REMS) program that requires healthcare facilities to have immediate access to tocilizumab and trained personnel for managing potential complications.
The study's findings represent an important step toward optimizing CAR-T cell therapy delivery while maintaining the rigorous safety standards essential for this potentially curative treatment approach.
