New long-term follow-up data from the phase Ib/II CARTITUDE-1 study demonstrated that one-third of patients in the study with relapsed or refractory multiple myeloma treated with the chimeric antigen receptor T-cell therapy ciltacabtagene autoleucel achieved progression-free survival of 5 years or more with a single infusion and no maintenance or subsequent antimyeloma therapy.
In a subset of 12 patients who underwent serial evaluations at a single site, all were minimal residual disease–negative and negative on imaging throughout 5 years of posttreatment follow-up. The findings were presented at the 2025 ASCO Annual Meeting (Abstract 7507)and simultaneously published by Jagannath et al in the Journal of Clinical Oncology.
"This new evidence shows how a single infusion of ciltacabtagene autoleucel can help patients survive without disease progression much longer than previously thought possible in this setting, and without any maintenance or subsequent treatment," said Peter M. Voorhees, MD, Clinical Professor of Hematology and Oncology at Atrium Health, Levine Cancer Institute at Wake Forest University School of Medicine. "In a heavily pretreated population, a third of patients remained treatment- and progression-free for at least 5 years."
The phase Ib/II CARTITUDE-1 study (n = 97) evaluated ciltacabtagene autoleucel for the treatment of heavily pretreated patients with relapsed or refractory multiple myeloma. Patients who remained progression-free for at least 5 years (n = 32) had received a median of six prior lines of therapy and included subgroups with high-risk cytogenetics (23.3%), extramedullary disease (12.5%), triple-class refractory (90.6%), and pentadrug-refractory (46.9%). At a median follow-up of 61.3 months, median overall survival was 60.7 months (95% confidence interval = 41.9 months to not estimable), highlighting the depth and durability of response with ciltacabtagene autoleucel.
With longer follow-up, the safety profile in CARTITUDE-1 was consistent with the known safety profile of ciltacabtagene autoleucel with no new safety signals observed. There were two newly reported second primary malignancies (both solid tumors) and no new Parkinsonism events or cranial nerve palsies.
Additional data CARTITUDE-4, presented at the 2025 ASCO Annual Meeting (Abstract 7539), evaluated progression-free survival and overall survival with ciltacabtagene autoleucel vs standard of care in prespecified subgroups, including patients with standard and high-risk cytogenetics, extramedullary disease, and by line of therapy. Results demonstrated that ciltacabtagene autoleucel improved progression-free survival and overall survival across subgroups. In patients with standard-risk disease after one to three prior lines of treatment, progression-free survival curves indicate stability in survival rates.
These results will also be presented at the upcoming European Hematology Association (EHA) 2025 Congress.
Disclosure: For full disclosures of the study authors, visit coi.asco.org.
