On October 23, the China News Agency reported that retinoblastoma is the most common intraocular malignant tumor in infants and young children, posing a significant threat to their lives and vision.
During an interview, it was revealed that Chinese medical experts have made a groundbreaking discovery in the treatment of this disease, which not only shortens the treatment duration but also maintains treatment effectiveness. This research was published in the latest issue of the prestigious clinical journal, The Journal of the American Medical Association (JAMA), marking a significant recognition of this innovative therapy.
Professor Qian Jiang’s team from Fudan University School of Ophthalmology and Otolaryngology collaborated with Professor Yang Huasheng’s team from Sun Yat-sen University, conducting a dual-center prospective study focusing on treating high-risk unilateral retinoblastoma patients. After more than a decade of rigorous and systematic clinical research, the team demonstrated that a treatment strategy involving three cycles of chemotherapy is just as effective as the traditional six cycles. It also showed clear advantages in safety, cost-effectiveness, and improvement in the patients’ quality of life.
Retinoblastoma is most often seen in children under five, with one in four cases affecting both eyes, and some linked to familial history. For high-risk unilateral retinoblastoma patients, enucleation (removal of the eyeball) is a common treatment, followed by systemic chemotherapy. The traditional six-cycle chemotherapy regimen lowers the risk of distant tumor metastasis and increases survival rates. However, this approach also brings considerable systemic side effects and financial burdens.
In the clinical study, researchers randomly assigned patients who underwent enucleation due to high-risk unilateral retinoblastoma into two groups: those receiving the three-cycle regimen and those receiving the traditional six-cycle regimen. Both groups underwent different chemotherapy treatments with carboplatin, etoposide, and vincristine.
With a median follow-up of 79 months and no patient lost to follow-up, the researchers found that the median age of all patients was 25 months. The five-year survival rates for the three-cycle and six-cycle groups were 90.4% and 89.2%, respectively, with no significant differences in overall survival between the two groups. This evidence supports that the three-cycle chemotherapy regimen is as effective as the standard six-cycle regimen, and data from the two study centers were highly consistent.
In terms of safety, patients in the six-cycle group experienced more adverse events, including bone marrow suppression and liver and kidney function impairment. Economically, the three-cycle regimen significantly reduced treatment costs, alleviating financial strain on patients and their families. Furthermore, the quality of life scores for patients in the three-cycle group declined less during chemotherapy, and they showed faster recovery and higher quality of life post-treatment.
JAMA has given high praise to this research. Renowned international ocular oncology expert, Professor Carol Shields, commented on the milestone importance of the three-cycle chemotherapy regimen, stating that it alleviates the financial pressure on families and increases treatment completion rates, potentially saving more children’s lives.
This study’s findings are expected to be implemented globally, improving treatment outcomes and quality of life for affected children.
