Kymriah (tisagenlecleucel-T)

Vignettes explored access to evidence-informed but not universally funded therapies: blinatumomab for low-risk relapse of B-cell acute lymphoblastic leukemia (B-ALL), larotrectinib for TRK-fused soft tissue sarcoma, PBT for unresectable head-and-neck sarcoma, and tisagenlecleucel for first relapse of B-ALL in a patient with Down syndrome. Access to evidence-informed cancer therapies for Canadian children remains variable. Universal funding, simplified approval processes, and the establishment of Canadian PBT centres to reduce travel burden, would ensure timely, equitable access to high-cost therapies.

P2, N=121, Active, not recruiting, Novartis Pharmaceuticals | Trial primary completion date: Aug 2025 --> Oct 2027

In a single-institution retrospective cohort of pediatric and young adult patients with relapsed or refractory acute B-lymphoblastic leukemia (B-ALL) treated with tisagenlecleucel between November 2023 and April 2025, two of 12 patients (17%) developed IEC-HS after CRS resolution, marked by recurrent fever, coagulopathy, hepatic dysfunction, cytopenias, and profound elevations in CXCL9, IL-18, and soluble C5b-9. Treatment with emapalumab led to rapid clinical and biomarker improvement. These findings support IEC-HS as an interferon-γ-driven syndrome and suggest targeted IFN-γ blockade as a potentially effective therapeutic strategy.

P=N/A, N=30, Recruiting, Novartis Pharmaceuticals | Not yet recruiting --> Recruiting

These findings highlight the importance of early leukapheresis, ideally before intensive treatments, to optimize T-cell yield, product quality, and therapeutic efficacy.

We conducted a single-center retrospective study of 69 patients treated with Tisagenlecleucel, to evaluate the prognostic impact of TP53 alterations (TP53Alt), including mutations and/or deletions...These findings identify TP53 alterations as a strong adverse prognostic factor in patients with r/r B-ALL treated with CAR-T therapy. Screening for TP53Alt may guide risk-adapted strategies, including early consolidation with hematopoietic stem cell transplantation or alternative therapies.

P4, N=75, Not yet recruiting, Universitair Ziekenhuis Gent

P3, N=331, Completed, Novartis Pharmaceuticals | Active, not recruiting --> Completed

P=N/A, N=500, Recruiting, Novartis Pharmaceuticals | N=179 --> 500

P3, N=200, Recruiting, Novartis Pharmaceuticals | Trial completion date: Mar 2026 --> Apr 2027 | Trial primary completion date: Mar 2026 --> Mar 2027

P3, N=109, Active, not recruiting, Novartis Pharmaceuticals | Recruiting --> Active, not recruiting

P1, N=20, Recruiting, Stanford University | Trial completion date: Nov 2025 --> Jul 2026 | Trial primary completion date: Nov 2025 --> Jul 2026