Boy, 12, recovering after groundbreaking transplant
A 12-year-old boy suffering a rare disease because of genetic disorder was transferred to ordinary ward on Monday after a successful stem cell transplant.
He is the first patient in China to receive a transplant for PIK3CD mutation, which causes primary immunodeficiency, officials from the Children's Hospital of Fudan University said.
Nicknamed Hao Hao, the boy from Jiangsu Province had frequent infections from 3 months old and had begun to suffer damage to his kidneys and other organs along with lung problems as doctors were unable to identify his disease.
Hao Hao’s younger brother also showed the same symptoms and the parents had been to several hospitals to have them checked.
In 2017 Hao Hao and his brother were confirmed to have PIK3CD mutation, which causes recurrent infections, immune problems and a higher chance of tumors.
The disease was only discovered in 2013 and, so far, there have been only 100 to 200 cases reported worldwide.
Doctors at the children's hospital said conventional treatments for the disease — medicine and therapy — had no positive effects on the two boys.
“We decided on a stem cell transplant for Hao Hao and his brother after the medicine failed. Stem cells are an effective therapy for blood and immune disease. If successful, the patients can be cured,” said Dr Wang Xiaochuan, director of the hospital’s immunity department. “But there is no report of using stem cell transplants to treat patient with PIK3CD mutation in China. So we were very careful in each step, as the stem cell transplant also has risks such as rejection.”
Hao Hao’s brother died in June as his condition was too serious but a match for Hao Hao, a 37-year-old Shanghai man, was found in the city’s stem cell donors' bank and he received the transplant on September 25.
The donor said he was glad to help a young boy and a family which had just lost a child.
Doctors said the transplant was successful and Hao Hao’s immunity function has recovered.
“We are expecting to declare a full recovery in three months after Hao Hao shows no further symptoms of rejection and other complications," said Dr Qian Xiaowen, who is in charge of Hao Hao's stem cell transplant.
Hao Hao's parents expressed their gratitude to the hospital and the donor, who had given Hao Hao a new life. "We will educate him to become a useful person in the future and benefit society and help other people," his father said.
Hao Hao, seen here with his mother and father, is now in an ordinary ward after a successful stem cell transplant.
