Journal: Blood advances
This real-world study from the Center for International Blood and Marrow Transplant Research evaluated tisagenlecleucel, a CD19-directed CAR T-cell therapy, in 768 pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) over a median follow-up of 32.1 months.
Key findings include:
- Overall complete remission rate: 86.0%
- At 12 months: Relapse-free survival (RFS) was 61.8% and overall survival (OS) was 79.4%
- At 24 months: RFS was 50.3% and OS was 63.8%
- Better outcomes observed in: Patients under 18 years and those with lower disease burden (<50% bone marrow blasts)
- Poorer prognosis predicted by: Prior treatment with inotuzumab and KMT2A rearrangement
- Adverse events: Older patients (≥18 years) had higher rates of cytokine release syndrome (CRS), which correlated with greater disease burden
- Lower toxicity in younger children: Both CRS and neurotoxicity were less common in children under 3 years
The data support sustained efficacy and a favorable safety profile of tisagenlecleucel in this patient population.