Stem cells do more! The first case of transplantation of stem cells to treat pik3cd gene defect in China has been successful!

The National Children's medical center and the pediatric hospital affiliated to Fudan University explored stem cell transplantation for patients with ineffective targeted drug treatment. On the same day, 12-year-old Hao Hao completed stem cell transplantation and returned to the general ward. Stem cell transplantation has not been reported in China before.

Since his birth, Haohao has repeatedly suffered from diarrhea, hepatosplenomegaly, pulmonary infection and other symptoms, kidney damage, thyroid dysfunction and so on. His parents took him around, and finally in 2015 he was diagnosed with a rare disease of primary immunodeficiency. Xuanxuan, 5 years younger than Haohao, was also a patient with the disease. After the definite diagnosis, the two brothers began to receive targeted drug treatment, but they were not sensitive to drug treatment and the condition was repeated. My younger brother died before because of his rapid progress.

The stem cell transplantation team and the Immunology Department of the pediatric hospital decided to adopt stem cell transplantation after finding that Haohao was not sensitive to targeted drug treatment. Professor Qian Xiaowen, deputy director of Hematology Department of pediatric hospital, said that stem cell transplantation is to replace patients with healthy human stem cells, so that the blood cells and immune cells with congenital defects in patients with rare diseases can become the cells of healthy donors, so that the blood diseases and immune system diseases with congenital defects can be cured and patients can be cured.

Hao Hao successfully matched a 37 year old Shanghai man in the Shanghai Branch of China bone marrow bank. At the end of September, Haohao received stem cell reinfusion. In the past four weeks, new cells grew little by little in his body. Professor Qian Xiaowen said that at present, Haohao has no rejection reaction after transplantation, no other transplantation related complications, and the blood routine has gradually recovered. After more than one month's observation, the child can be discharged from the hospital. Professor Wang Xiaochuan said that after the blood cells recovered, the immune function began to recover slowly, but it usually took more than one year to recover to the normal level.