The reporter recently learned from the Children's Medical Center of the Affiliated Hospital of Guizhou Medical University that the center has successfully completed gene therapy for a patient with severe hemophilia B, and the patient's coagulation activity is stable and the effect is remarkable. This marks a major breakthrough in the field of gene therapy for hemophilia in Guizhou Province.
According to Jin Jiao, director of the Department of Pediatric Hematology and director of the Hemophilia Diagnosis and Treatment Center of the Affiliated Hospital of Guizhou Medical University, the patient is now 19 years old and has a history of severe hemophilia for 10+ years. Half a year ago, gene therapy was evaluated for him, and after follow-up, the patient's current coagulation factor activity was stable at about 100%.
Jin Jiao said that patients with severe hemophilia B need to be treated with intravenous injection of coagulation factors 1~2 times a week. Even so, their coagulation factor activity can only reach about 1~3%. Although bleeding can be significantly reduced, it does not completely prevent bleeding from occurring. Moreover, patients need to use intravenous access for long-term blood transfusion and transfusion of coagulation factors, and their quality of life will also be reduced.
"We evaluated and screened this patient before treating him, and first determined that he was eligible for gene therapy. Gene therapy began in October last year, and the infusion process went smoothly, and the patient did not have any adverse reactions in the following days. Kim Kyauk said.
Professor He Zhixu, an expert in pediatric hematology, chairman of the Guizhou Provincial Pediatric Branch, and the leader of the Children's Medical Center of the Affiliated Hospital of Guizhou Medical University, said in an interview that gene therapy, as an emerging treatment method, has shown great potential in the treatment of hereditary diseases such as hemophilia. The successful implementation of gene therapy has not only brought significant therapeutic effects to patients, but also provided valuable experience for future research and treatment in the field of hemophilia.
Hemophilia is a hereditary disorder of the blood that occurs at a relatively high rate in children. The cause is a lack of coagulation factor VIII or factor IX. in the body, which causes lifelong coagulation abnormalities. Hemophilia caused by factor eight deficiency is called hemophilia A, and cause factor nine deficiency is called hemophilia B. Because of the need to receive long-term coagulation factor injections, the cost of treatment is relatively high, therefore, gene therapy will become a trend in the treatment of hemophilia.
"Gene therapy is the construction of factor 9 in hemophilia B patients outside the body and then re-infused into the patient. It is targeted and integrated into liver cells and continuously secretes the corresponding hemophilia factor. In the treatment of hemophilia, gene therapy can correct the defects of clotting factors in the patient's body, thereby restoring their normal coagulation function. Therefore, this treatment can be done once and for all, and the purpose of lifelong treatment can be achieved. Professor He Zhixu said.
Professor He Zhixu said that the first case of gene therapy for children with hemophilia successfully implemented by the Affiliated Hospital of Guizhou Medical University is the first gene therapy for hemophilia patients in southwest China, and it is also the earliest institution in China to carry out gene therapy for hemophilia. The success of this technology marks that the Department of Pediatric Hematology of the Affiliated Hospital of Guizhou Medical University has reached the international advanced level in the diagnosis and treatment of hemophilia, and has opened up a new way for the treatment of hemophilia. In the future, with the continuous development and improvement of gene therapy technology, it is believed that more hemophilia patients will benefit from this advanced treatment.
(Reporter: Zhou Qian)