A latest clinical study covering 12 pediatric hematology-oncology centers in China showed that CD19 CAR-T cell therapy achieved a complete remission (CR) rate of 92% in children with relapsed or refractory acute lymphoblastic leukemia (ALL), with a two-year event-free survival (EFS) rate of 63% and a median follow-up of 18 months. The results were published online in the *Chinese Journal of Pediatrics* in May 2025, drawing attention from the international pediatric oncology community.
The study enrolled 187 children aged 1-18 years, using a humanized anti-CD19 CAR-T product. The incidence of cytokine release syndrome (CRS) ≥ grade 3 was 14%, and neurotoxicity ≥ grade 3 was 6%, with no treatment-related deaths. All patients were from a refractory/relapsed population that had failed at least two prior lines of chemotherapy, with a median of 3 prior treatment lines.
Background and Significance
Although the overall cure rate for pediatric ALL has exceeded 85%, 15%-20% of children still face relapse or drug resistance. Long-term survival after conventional salvage chemotherapy is below 30%, and allogeneic hematopoietic stem cell transplantation is limited by donor availability and transplant-related complications. CAR-T, which genetically engineers T cells to precisely target the CD19 antigen, provides these children with an opportunity for bridging to transplant or independent cure. The high remission rate and manageable safety profile of this study further validate the efficacy of this strategy in the Chinese pediatric population and support its prioritization as a second-line or later treatment option.
Compared with similar products in Europe and the United States, this humanized CAR-T reduces immunogenicity, prolongs in vivo persistence, and lowers production costs by approximately 40%. Currently, this regimen has received breakthrough therapy designation from the National Medical Products Administration and is advancing toward registered clinical trials.
Significance for International Patients
For families from overseas, especially those from regions with limited access to CAR-T therapy, childhood leukemia CAR-T treatment in China offers significant appeal: first, the treatment cost is approximately 1/5 to 1/3 of that in the United States, with a total cost of about 200,000-300,000 RMB without insurance coverage; second, China has multiple experienced pediatric hematology-oncology centers, such as Shanghai Children's Medical Center and Beijing Children's Hospital, each performing over 100 CAR-T treatments annually with high technical proficiency; third, support is available for international patient visa coordination, remote consultations, and Chinese translation accompaniment.
The study also showed that children who received bridging transplantation after CAR-T had a two-year EFS of 71%, superior to the CAR-T consolidation-only group (52%), providing evidence for optimizing treatment pathways. Overseas families can use this information to develop individualized plans with their clinicians.
How MedBridge Shanghai Can Help
MedBridge Shanghai, as a cross-border medical platform, provides one-stop services for international patients:
- Matching appropriate pediatric hematology-oncology centers and CAR-T clinical experts, scheduling remote video consultations, and assessing eligibility for enrollment.
- Assisting with medical visa applications, international transfer, and post-arrival accommodation arrangements, along with bilingual Chinese-English medical interpretation.
- Providing cost estimation services that transparently list all expenses for CAR-T therapy, hospitalization, laboratory tests, and more, while also facilitating connections with charitable assistance programs.
- During treatment, MedBridge Shanghai continuously coordinates follow-up care, medication management, and rehabilitation support to ensure patient safety.
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