6-year-old Yang Yang (pseudonym) is sitting in a wheelchair, his small face is tightly covered by a mask, revealing a pair of eyes that are turning, curiously looking at the familiar and unfamiliar adults in front of him. Only a slightly yellowish complexion faintly revealed his unusualness.
In the past 10 months, Yang Yang has experienced nine deaths: he has not subsided due to high fever, white blood cells have dropped to zero, three times into the ICU rescue, and is about to face the situation of multi-organ bleeding and functional failure throughout the body.
Guangzhou Daily Xinhuacheng reporter learned from Sun Yat-sen Memorial Hospital of Sun Yat-sen University on March 31 that after the conventional treatment was ineffective, in order to save this small life, the team of the Children's Medical Center of the hospital gave it a go with the resolute support of the child's father, and the "super instructions" used the immunotherapy drug PD-1 antagonist, which finally got Yangyang out of danger.
At present, Yangyang has completed hematopoietic stem cell transplantation and is receiving anti-graft-versus-host disease treatment. The successful experience of treating Yang yang has also been applied by the hospital to treat three other children with the same disease. At present, these children have been successfully discharged from the hospital.
White blood cells fall to zero! This disease causes cells to attack the body itself!
In early June 2021, Yang Yang, who was only 5 years old at the time, went to a hospital in Guangdong for a sustained high fever of 42 °C and was diagnosed with "Epstein-Barr virus-related hemophagocytic syndrome". It is an excessive inflammatory response syndrome caused by immune abnormalities with a mortality rate of up to 70%. After four weeks of standardized chemotherapy with the HLH-1994 regimen, Yangyang's condition still did not improve.
At the end of July, Yangyang, who had a high fever, was transferred to the Children's Medical Center of Sun Yat-sen Memorial Hospital of Sun Yat-sen University for hospitalization.
According to Zhou Dunhua , director of the Children's Medical Center of the hospital (pictured), EBV is a type of herpes virus, after some people with congenital genetic defects or abnormal immune systems are infected, lymphocytes and macrophages that were originally used to "defend against foreign enemies" are abnormally activated and proliferated, secreting a large number of inflammatory cytokines, forming a "cytokine storm", attacking the body's own tissues and organs, causing extremely serious damage.
"Treatment of refractory severe Epstein-Barr virus-related hemophagocytic syndrome is tricky, and hematopoietic stem cell transplantation is currently the only cure for congenital, refractory hemophagocytic syndrome (HLH) in children." Professor Zhou Dunhua said, but this "bridge transplant" treatment plan has a premise: patients should first achieve remission through drug therapy, otherwise it may lead to transplant failure.
However, even with the DEP rescue plan, the situation in Yangyang is still not satisfactory. "The child has a high fever, the mouth and perianal ulceration, the bone marrow cytology test is in a state of extreme inhibition, and for five months, the white blood cells in the blood drop to zero, relying heavily on red blood cells and platelets." Zhou Dunhua recalled that the child's condition was unusually serious, his life characteristics were unstable, and he faced the possibility of bleeding and failure of multiple organs.
Good dad does not abandon the delivery of takeaway to pay back 250,000 yuan in arrears
Yangyang has a strong and affectionate father. Raising YangYang alone after his divorce, he tried everything to raise the cost of treatment for the child, borrowing 250,000 yuan in the village alone. During Yang Yang's hospitalization, his grandmother took care of him in the hospital, and his father got up early and greedily delivered takeaways and did odd jobs, hoping to repay the loans of the villagers as soon as possible.
The medical staff pitied Yang Yang's condition and tried their best to help him: under the coordination of the hospital, Yang Yang received a donation of 30,000 yuan from another family member who treated the child, but it was still a drop in the bucket. For Yangyang's condition, the father and grandmother never wanted to give up, fully supported the treatment of medical staff, and said: "No matter how difficult we are, we must insist on treating the child."
In the most dangerous moments, the family supports the doctor to give it a try
On September 28, 2021, YangYang, who continued to have a high fever, was short of breath and mentally weak. This means that the child's life has reached its most dangerous moment.
The doctors immediately organized in-department consultations and invited authoritative experts to conduct remote online consultations to actively seek new treatment options. At present, the use of PD1 antagonists has been explored in China to treat adult patients with EBV-related hemophagosis, but can this treatment plan be used in children?
After full communication with the family, the child's father supported the doctor to take a risk. In September 2021, under the premise of making a response plan, medical staff used PD1 antagonists on Yangyang. On the second day of medication, the child with persistent high fever began to have a fever. After 3 days of medication, peripheral hemoleukemia, hemoglobin and platelets also began to recover, and then the plasma EB-DNA turned negative.
After evaluation, the hemophagocytic index of Yangyang has been partially relieved and can be bridged transplantation. On December 21, 2021, the transplant specialty of the Children's Medical Center of Sun Yat-sen Memorial Hospital of Sun Yat-sen University performed a cord blood transplant donated by volunteers for Yangyang.
The situation reappeared four days after transplantation
No one expected that after the transplant, Yangyang would be in danger again.
From the 4th day after the infusion of umbilical cord blood, Yangyang began to have symptoms such as persistent high fever, increased heart rate, diarrhea, and convulsive attacks, and his life was in danger.
In response to his "severe perimplantation syndrome, systemic inflammatory response syndrome, transplant-related thrombotic microangiopathy", doctors immediately adjusted the immunosuppressant medication regimen for him, plasma exchange and other treatments. The proportion of implantation also reached 100% after transplantation d+17, and the proportion of whole blood implantation monitored thereafter was 100%.
In the end, Yangyang, who had entered the ICU three times, successfully survived a difficult time.
Grandma accompanied Yang Yang to the hospital, and the medical staff sent toys to help him relieve his nervousness
Under the careful care of doctors and nurses, Yang Yang went from being timid and afraid to speak to loving to smile and coddle the nurse sister, he also liked to paint, and gave his paintings to the doctor to express his gratitude.
Yang Yang sent the painting to Professor Fang Jianpei, a pediatric hematology and oncology expert
Anti-rejection therapy is required after discharge
Looking back at the treatment of Yangyang, Professor Fang Jianpei, the academic leader of the Children's Medical Center of Sun Yat-sen Memorial Hospital of Sun Yat-sen University, believes that yangyang transplantation has many comorbidities, which are related to multiple chemotherapy before transplantation, repeated infections, and serious organ damage.
"If conventional treatment is ineffective, whether the use of PD-1 antagonists as early as possible can reduce the damage to the child's body by repeated chemotherapy and win more time and opportunities for subsequent hematopoietic stem cell transplantation, which is worth further study." Professor Fang Jianpei stressed that whether it is suitable for using the new treatment plan must be determined according to the specific situation of the child.
At present, Yangyang can be discharged from the hospital after another plasma exchange therapy, but it is necessary to insist on taking anti-rejection drugs in the future. "It costs about 5-7,000 yuan per month, and generally requires continuous treatment for 6 to 9 months." Professor Fang Jianpei introduced.
Guangzhou Daily Xinhuacheng reporter learned from the hospital that Yangyang's father was already saddled with heavy debts. The medical staff appealed that caring people can reach out their hands to help the Yangyang family tide over the difficulties.
Text, photo/ Guangzhou Daily Xinhuacheng Reporter: Ren Shanshan Intern Zheng Han Correspondents: Lin Weiyin, Zhang Yang
Guangzhou Daily Xinhuacheng Editor: Li Jin