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ASTCT & CIBMTR 2026 | Dr. Huaying Liu: 90.1% Overall Survival in Pediatric R/R ALL with CAR-T–Bridged TCRαβ-Depleted Haploidentical Transplantation

2026-2-6

From February 4–7, 2026, the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood and Marrow Transplant Research (CIBMTR) jointly hosted the 2026 Tandem Meetings in Salt Lake City, USA. As one of the most influential international conferences in hematopoietic stem cell transplantation and cellular therapy, the meeting brought together leading experts worldwide to present the latest advances in transplantation, CAR-T therapy, and related translational research.

At this year’s meeting, the research team led by Dr. Huaying Liu from the GoBroad Chunfu Hematology Research Institute, GoBroad Healthcare Group, delivered an oral presentation highlighting a comprehensive treatment strategy for pediatric relapsed/refractory acute lymphoblastic leukemia (R/R ALL). The study demonstrated that CAR-T therapy followed by TCRαβ-depleted haploidentical hematopoietic stem cell transplantation (TDH) combined with prophylactic haploidentical donor lymphocyte infusion (haplo-DLI) achieved an overall survival (OS) rate of 90.1%.

Dr. Liu's team has remained deeply engaged in the field of hematologic malignancy transplantation and cellular therapy, with related work presented at the ASTCT & CIBMTR meetings for three consecutive years, offering the international community a systematic view of their evolving clinical strategy and outcomes.

Background

TCRαβ-depleted haploidentical transplantation is a potentially curative option for children with R/R ALL. Achieving minimal residual disease (MRD) negativity prior to transplantation is strongly associated with a reduced risk of relapse. The team proposed an integrated approach: CAR-T therapy before transplant to achieve MRD negativity, followed by prophylactic haplo-DLI after transplant, aiming to further decrease relapse and improve long-term outcomes.

Methods

Sixty-five pediatric patients with R/R ALL who underwent TDH between December 2020 and July 2025 were included. Fifty-five patients received CAR-T therapy before transplantation and achieved MRD negativity. All patients received myeloablative conditioning, TCRαβ-depleted grafts, no routine post-transplant immunosuppression, and scheduled prophylactic haplo-DLI with escalating doses.

Results

With a median follow-up of 29 months, the cumulative relapse rate was 9.5%, non-relapse mortality 7.9%, overall survival 90.1%, and leukemia-free survival 83.2%. Patients transplanted in earlier remission stages showed superior outcomes. Among patients in ≥CR2, those receiving CAR-T prior to TDH had significantly lower relapse rates and better leukemia-free survival compared with those without CAR-T bridging. The incidences of grade II–IV acute GVHD and chronic GVHD were 9.1% and 12.7%, respectively.

Conclusion

TCRαβ-depleted haploidentical transplantation provides durable disease control with low GVHD rates in pediatric R/R ALL, particularly when preceded by CAR-T therapy achieving MRD negativity. This integrated strategy offers a favorable balance between anti-leukemic efficacy and long-term safety.

Investigator's Perspective

“Our previous studies have shown that CAR-T therapy followed by TDH and prophylactic haplo-DLI can effectively reduce relapse in pediatric R/R ALL. In this study, we further confirmed that careful optimization of key steps before and after transplantation allows sustained graft-versus-leukemia effects without routine GVHD prophylaxis, resulting in excellent survival outcomes. These findings provide valuable clinical evidence to guide transplant decision-making in children with R/R ALL,” said Dr. Huaying Liu.

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