R) within 3 months after infusion, the duration of CR, and proportion of patients with CR and minimal residual disease (MRD) < 0.01% by flow cytometry (MRD-negative) (Table 8). Among the 63 infused patients, 52 (83%) achieved CR/CRi, all of which were MRD-negative. With a median follow-up of 4.8 months from response, the median duration of CR/CRi was not reached (range: 1.2 to 14.1+ months). Median time to onset of CR/CRi was 29 days with onset of CR/CRi between 26 and 31 days for 50/52 (96%) responders. The stem cell transplantation rate among those who achieved CR/CRi was 12% (6/52). Table 8 shows the efficacy results from this study.
Table 8. Efficacy Results in Pediatric and Young Adult Patients with r/r B-cell ALL
1CR/CRi was calculated based on all patients who received KYMRIAH and completed at least 3 months follow-up, or discontinued earlier prior to the data cut-off. Requires remission status to be maintained for at least 28 days without clinical evidence of relapse.
2 The null hypothesis of CR/CRi less than or equal to 20% was rejected.
3CR (complete remission) was defined as less than 5% of blasts in the bone marrow, no evidence of extramedullary disease, and full recovery of peripheral blood counts (platelets greater than 100,000/microliter and absolute neutrophil counts [ANC] greater than 1,000/microliter) without blood transfusion.
4CRi (complete remission with incomplete blood count recovery) was defined as less than 5% of blasts in the bone marrow, no evidence of extramedullary disease, and without full recovery of peripheral blood counts with or without blood transfusion.
5MRD (minimal residual disease) negative was defined as MRD by flow cytometry less than 0.01%.
6The null hypothesis of MRD-negative remission rate less than or equal to 15% was rejected.
7DOR (duration of remission) was defined as time since onset of CR or CRi to relapse or death due to underlying cancer, whichever is earlier, censoring for new cancer therapy including stem cell transplantation (N = 52).
8Not Estimable.
Results N = 63
CR/CRi1,2
95% CI 52 (83%)
(71%, 91%)
p < 0.0001
CR3 40 (63%)
CRi4 12 (19%)
CR or CRi with MRD-negative bone marrow5,6
95% CI
52 (83%)
(71%, 91%)
p < 0.0001
Duration of Remission7 N = 52
Median (months) Not reached
95% CI (7.5, NE8)
14.2 Adult Relapsed or Refractory (r/r) Diffuse Large B-cell Lymphoma (DLBCL)
The efficacy and safety of KYMRIAH was eva luated in an open-label, multicenter, single-arm trial (JULIET; NCT02445248). Eligible patients were ≥ 18 years of age with relapsed or refractory DLBCL, who received ≥ 2 lines of chemotherapy, including rituximab and anthracycline, or relapsed following autologous hematopoietic stem cell transplantation (HSCT). The study excluded patients with active central nervous system malignancy, prior allogenic HSCT, an ECOG performance status ≥ 2, a creatinine clearance < 60, alanine aminotransferase > 5 times normal, cardiac ejection fraction < 45%, or absolute lymphocyte concentration less than 300/µL.
Following 2 to 11 days after completion of lymphodepleting (LD) chemotherapy consisting of either fludarabine (25 mg/m2
