2026-07-03
[POST-ASCO 2026] Large-Scale Real-World Data Confirms 5-Year Long-term Survival Benefit of Axi-cel in R/R LBCL, Reinforcing Its Curative Potential
The 2026 American Society of Clinical Oncology (ASCO) Annual Meeting convened in Chicago, Illinois, USA, from May 29 to June 2, 2026. As one of the world's largest, most academically prestigious, and authoritative gatherings in clinical oncology, the ASCO Annual Meeting annually unites leading oncology experts, clinical researchers, and industry representatives from across the globe to share and discuss cutting-edge scientific achievements in clinical oncology.
As the first approved CD19-targeted autologous CAR-T cell therapy for relapsed/refractory large B-cell lymphoma (R/R LBCL), Axicabtagene Ciloleucel (Axi-cel) has built a strong track record of efficacy and safety, moving from registration trials to solid real-world validation -3.
At the registration trial level, the pivotal ZUMA-1 study established Axi-cel's therapeutic position based on outstanding efficacy in 101 patients with refractory LBCL: achieving an objective response rate (ORR) of 83% and a complete response (CR) rate of 58%. At a median follow-up of 63.1 months, the 5-year overall survival (OS) rate reached 42.6%, escalating to 64.4% among patients who achieved a CR [1,3]. No new safety signals were observed during the 5-year follow-up period [3]. A subsequent analysis of the 6-year follow-up data from the ZUMA-1 study further consolidated Axi-cel's long-term curative potential [4]: the 5-year lymphoma-related event-free survival (LREFS) rate was 33.5%, and the estimated 6-year disease-specific survival (DSS) rates reached a striking 94.4% and 100% for patients who maintained a CR at Month 12 and Month 24, respectively.
In the real-world setting, a post-marketing requirement study based on the Center for International Blood and Marrow Transplant Research (CIBMTR) registry [2] evaluated the efficacy and safety of Axi-cel in a large-scale real-world cohort for the first time. The study enrolled 1,297 patients treated with commercial Axi-cel across 78 centers, with a median follow-up of 12.9 months. Notably, 57% of the patients fell into the ineligible group based on ZUMA-1 enrollment criteria (including ECOG PS ≥ 2, moderate-to-severe organ dysfunction, active infection, or prior malignancies). However, the efficacy in this ZUMA-1 ineligible subgroup was comparable to that of the eligible subgroup, demonstrating excellent consistency of Axi-cel's efficacy across a broader, more heavily co-morbid patient population. Long-term follow-up data from the US Lymphoma CAR-T Consortium Real-World Study (RWS) also confirmed the consistency of Axi-cel's efficacy between real-world and registration studies; at a median follow-up of 58 months, the 5-year OS rate stood at 40.3% (N = 275) [5].
Nevertheless, whether Axi-cel can replicate the long-term survival benefits observed in clinical trials within a real-world setting—encompassing a broader and medically more complex patient population—lacks systematic validation. The release of long-term real-world follow-up data carries crucial evidence-based value in confirming the durable advantages of Axi-cel as a single-infusion, potentially curative therapy. The CIBMTR 5-year follow-up data analysis [6] presented at this year's ASCO Annual Meeting has unveiled the answer.
Abstract No.: #7028
Title
Long-term real-world outcomes of axicabtagene ciloleucel (axi-cel) inrelapsed/refractory (R/R) large B-cell lymphoma (LBCL) [6]
Methodology
A total of 1500 adults from 79 US centers receiving commercial axi-cel after ≥2 LOT for LBCL from Oct 2017-Aug 2020 were enrolled in a post-marketing requirement study. A protocol was developed prior to the studyʼs implementation, reviewed by Kite and CIBMTR. This is a secondary analysis of said study. Key outcomes were progression-free survival (PFS), overall survival (OS), time to next therapy (TTNT), disease-specific mortality (competing risks were non-relapse deaths), subsequent malignancies, and non-relapse mortality (NRM). Outcomes were analyzed descriptively. Cases with missing data were excluded separately for each endpoint.
Patient Baseline Characteristics
As of Aug 2025, 1446 patients (pts) were included in the analysis. Median age at infusion was 62.2 y (range, 19.6-90.8:38% of pts ≥65 y); 65% were male. Five percent of pts had ECOG PS ≥2 and 16% had high-grade lymphoma. Pts had a median of 3 (range, 2-4) prior LOT, 28% had prior autologous hematopoietic cell transplantation, and 50% received bridging therapy.
Efficacy Analysis
At 59.7 mo median follow-up, median PFS (95% CI) was 7.9 mo (6.3-10.9) and 5-y PFS was 30% (27-32). The median OS was 25.7 mo (21.6-30.2); 5-y OS was 38% (35-41). The 5-y cumulative incidence of disease-specific mortality was51%. Among pts who were progression-free at 2, 3, 4, or 5 y post-infusion, the OS at 6 y (95% CI) was 73% (68-78), 81% (76-86), 90% (84-94), and 94% (89-97), respectively. The median duration of response (DOR) was 25.3 mo (95%CI, 20.7-32.8) and 5-y DOR was 38% (35-41). The median TTNT was 12.2 mo and 30% did not need additional LOT at 5y. Of pts who received subsequent salvage therapy, 12 (2%) received cell therapy, 39 (7%) received hematopoietic celltransplant, and 475 (90%) received other therapy. The 1, 2, 3, 4, and 5-y cumulative incidence of relapse (95% CI) was49% (46-51), 52% (50-55), 54% (52-57), 55% (53-58), and 56% (53-59), respectively.
Safety Analysis
No new safety signals were observed. The primary causes of death (n=864; 60%) were primary disease (540; 63%), infection (111; 13%), and organ failure (57; 7%). The 5-y cumulative incidence of NRM was 16% (95% CI, 14-18). At 5 y, 11% (95% CI, 10-13) had subsequent malignancies, mostly therapy-related myeloid neoplasms (6%) and non-melanomaskin cancer (3%); none were directly attributed to axi-cel.
Study Conclusions
This large-scale real-world study showed that for patients with R/R LBCL who had previously received ≥ 2 lines of therapy, Axi-cel provided a 5-year long-term survival benefit comparable to that observed in the pivotal ZUMA-1 study. Infection and organ failure were common causes of NRM. These results continue to supportthe use of axi-cel with curative intent in R/R LBCL.
Literature References:
1. Neelapu SS, et al. N Engl J Med. 2017 Dec 28;377(26):2531-2544.
2. Jacobson CA, et al. Transplant Cell Ther. 2022 Sep;28(9):581.e1-581.e8.
3. Neelapu SS, et al. Blood. 2023 May 11;141(19):2307-2315.
4. Neelapu ss, at al. ASH 2023. Abstact#4864.
5. Jain MD, et al. J Clin Oncol. 2024: JCO2302786.
6. Jacobson CA, et al. 2026ASCO. Abstract#7028.
As the first approved CD19-targeted autologous CAR-T cell therapy for relapsed/refractory large B-cell lymphoma (R/R LBCL), Axicabtagene Ciloleucel (Axi-cel) has built a strong track record of efficacy and safety, moving from registration trials to solid real-world validation -3.
At the registration trial level, the pivotal ZUMA-1 study established Axi-cel's therapeutic position based on outstanding efficacy in 101 patients with refractory LBCL: achieving an objective response rate (ORR) of 83% and a complete response (CR) rate of 58%. At a median follow-up of 63.1 months, the 5-year overall survival (OS) rate reached 42.6%, escalating to 64.4% among patients who achieved a CR [1,3]. No new safety signals were observed during the 5-year follow-up period [3]. A subsequent analysis of the 6-year follow-up data from the ZUMA-1 study further consolidated Axi-cel's long-term curative potential [4]: the 5-year lymphoma-related event-free survival (LREFS) rate was 33.5%, and the estimated 6-year disease-specific survival (DSS) rates reached a striking 94.4% and 100% for patients who maintained a CR at Month 12 and Month 24, respectively.
In the real-world setting, a post-marketing requirement study based on the Center for International Blood and Marrow Transplant Research (CIBMTR) registry [2] evaluated the efficacy and safety of Axi-cel in a large-scale real-world cohort for the first time. The study enrolled 1,297 patients treated with commercial Axi-cel across 78 centers, with a median follow-up of 12.9 months. Notably, 57% of the patients fell into the ineligible group based on ZUMA-1 enrollment criteria (including ECOG PS ≥ 2, moderate-to-severe organ dysfunction, active infection, or prior malignancies). However, the efficacy in this ZUMA-1 ineligible subgroup was comparable to that of the eligible subgroup, demonstrating excellent consistency of Axi-cel's efficacy across a broader, more heavily co-morbid patient population. Long-term follow-up data from the US Lymphoma CAR-T Consortium Real-World Study (RWS) also confirmed the consistency of Axi-cel's efficacy between real-world and registration studies; at a median follow-up of 58 months, the 5-year OS rate stood at 40.3% (N = 275) [5].
Nevertheless, whether Axi-cel can replicate the long-term survival benefits observed in clinical trials within a real-world setting—encompassing a broader and medically more complex patient population—lacks systematic validation. The release of long-term real-world follow-up data carries crucial evidence-based value in confirming the durable advantages of Axi-cel as a single-infusion, potentially curative therapy. The CIBMTR 5-year follow-up data analysis [6] presented at this year's ASCO Annual Meeting has unveiled the answer.
Abstract No.: #7028
Title
Long-term real-world outcomes of axicabtagene ciloleucel (axi-cel) inrelapsed/refractory (R/R) large B-cell lymphoma (LBCL) [6]
Methodology
A total of 1500 adults from 79 US centers receiving commercial axi-cel after ≥2 LOT for LBCL from Oct 2017-Aug 2020 were enrolled in a post-marketing requirement study. A protocol was developed prior to the studyʼs implementation, reviewed by Kite and CIBMTR. This is a secondary analysis of said study. Key outcomes were progression-free survival (PFS), overall survival (OS), time to next therapy (TTNT), disease-specific mortality (competing risks were non-relapse deaths), subsequent malignancies, and non-relapse mortality (NRM). Outcomes were analyzed descriptively. Cases with missing data were excluded separately for each endpoint.
Patient Baseline Characteristics
As of Aug 2025, 1446 patients (pts) were included in the analysis. Median age at infusion was 62.2 y (range, 19.6-90.8:38% of pts ≥65 y); 65% were male. Five percent of pts had ECOG PS ≥2 and 16% had high-grade lymphoma. Pts had a median of 3 (range, 2-4) prior LOT, 28% had prior autologous hematopoietic cell transplantation, and 50% received bridging therapy.
Efficacy Analysis
At 59.7 mo median follow-up, median PFS (95% CI) was 7.9 mo (6.3-10.9) and 5-y PFS was 30% (27-32). The median OS was 25.7 mo (21.6-30.2); 5-y OS was 38% (35-41). The 5-y cumulative incidence of disease-specific mortality was51%. Among pts who were progression-free at 2, 3, 4, or 5 y post-infusion, the OS at 6 y (95% CI) was 73% (68-78), 81% (76-86), 90% (84-94), and 94% (89-97), respectively. The median duration of response (DOR) was 25.3 mo (95%CI, 20.7-32.8) and 5-y DOR was 38% (35-41). The median TTNT was 12.2 mo and 30% did not need additional LOT at 5y. Of pts who received subsequent salvage therapy, 12 (2%) received cell therapy, 39 (7%) received hematopoietic celltransplant, and 475 (90%) received other therapy. The 1, 2, 3, 4, and 5-y cumulative incidence of relapse (95% CI) was49% (46-51), 52% (50-55), 54% (52-57), 55% (53-58), and 56% (53-59), respectively.
Safety Analysis
No new safety signals were observed. The primary causes of death (n=864; 60%) were primary disease (540; 63%), infection (111; 13%), and organ failure (57; 7%). The 5-y cumulative incidence of NRM was 16% (95% CI, 14-18). At 5 y, 11% (95% CI, 10-13) had subsequent malignancies, mostly therapy-related myeloid neoplasms (6%) and non-melanomaskin cancer (3%); none were directly attributed to axi-cel.
Study Conclusions
This large-scale real-world study showed that for patients with R/R LBCL who had previously received ≥ 2 lines of therapy, Axi-cel provided a 5-year long-term survival benefit comparable to that observed in the pivotal ZUMA-1 study. Infection and organ failure were common causes of NRM. These results continue to supportthe use of axi-cel with curative intent in R/R LBCL.
Literature References:
1. Neelapu SS, et al. N Engl J Med. 2017 Dec 28;377(26):2531-2544.
2. Jacobson CA, et al. Transplant Cell Ther. 2022 Sep;28(9):581.e1-581.e8.
3. Neelapu SS, et al. Blood. 2023 May 11;141(19):2307-2315.
4. Neelapu ss, at al. ASH 2023. Abstact#4864.
5. Jain MD, et al. J Clin Oncol. 2024: JCO2302786.
6. Jacobson CA, et al. 2026ASCO. Abstract#7028.