Pioneering CAR-T Treatment for Refractory Juvenile Dermatomyositis Secondary to Sjögren's Syndrome
China Health Trip takes you through the latest medical breakthroughs in China, showcasing the possibilities of advanced healthcare. Read on for more insights.
On October 28 2024, the National Clinical Research Center for Child Health and Disease, the National Children’s Regional Medical Center, and the Children’s Hospital Affiliated to Zhejiang University School of Medicine (hereinafter referred to as ZJU Children’s Hospital) received good news: Qingqing (pseudonym), the world’s first CAR-T treatment for refractory juvenile dermatomyositis (JDM) with secondary Sjögren’s syndrome, has successfully stopped medication and was discharged smoothly!
CAR-T treatment advancements in China
Lu Meiping, the head of the CAR-T treatment of refractory juvenile dermatomyositis clinical project and director of the Department of Rheumatology and Immunology at Zhejiang University Children’s Hospital, said: “Tingting’s successful discontinuation of medication will bring new hope of complete recovery for other children with refractory juvenile dermatomyositis to completely get rid of the torture of the disease and drug dependence.”
Refractory juvenile dermatomyositis is a common pediatric autoimmune disease second only to systemic lupus erythematosus. The disease causes inflammation of the child’s skin and muscles, and may affect other systems such as the lungs and gastrointestinal tract. Children have to rely on drugs such as hormones and immunosuppressants to alleviate the condition. But at the same time, some children still respond poorly to these treatments, with repeated procrastination, skin ulcers/calcification, severe interstitial lung lesions, gastrointestinal perforation, and even life-threatening.
In June 2024, an article in the journal Arthritis Rheumatol reported the world’s first successful case of JDM treated with anti-CD19 CAR-T cells. In order to help more children with refractory juvenile dermatomyositis, Director Lu Meiping followed the international pace and led the rheumatology and immunology team to conduct a CAR-T treatment project for refractory juvenile dermatomyositis.
This is another clinical study of CAR-T treatment by Zhejiang University Children’s Hospital after completing the world’s largest sample CAR-T treatment project for pediatric systemic lupus erythematosus. The patient Qingqing also became the world’s first successful case of CAR-T treatment of refractory juvenile dermatomyositis (JDM) secondary to Sjögren’s syndrome.
“As a national dual center, the comprehensive strength of Zhejiang University Children’s Hospital ranks first in China, providing strong support for the clinical trial application of CAR-T technology. During the clinical trial, we also received strong support from Professor Mao Jianhua, Vice President of Zhejiang University Children’s Hospital and Director of the Kidney and Urology Center, the hospital ethics committee, and the cell apheresis of the Department of Hematology and Oncology. It is with everyone’s joint efforts that we hope to expand the field of CAR-T treatment through clinical trials and help children with refractory juvenile dermatomyositis relieve more pain.” Director Lu Meiping said.
A 16-year-old girl suffers from refractory juvenile dermatomyositis
16-year-old Qingqing is a child with refractory juvenile dermatomyositis. At the age of flowers, she has been enduring the pain and torture of juvenile dermatomyositis…
More than 8 years ago, Qingqing suddenly developed a purple-red rash with edema around her eyelids and eye sockets, and a bright red scaly rash appeared on the edges of her finger joints, accompanied by muscle soreness, decreased muscle strength, and increased muscle enzymes. After being sent to the hospital, she was diagnosed with juvenile dermatomyositis.
In order to control the onset of the disease, Qingqing has been receiving hormones, immunosuppressants, immunoglobulins and various targeted biological agents for more than 8 years. However, the prognosis has always been less than ideal, and the condition has always recurred, and the child’s body and mind have suffered.
Especially in the past two years, Qingqing’s activity endurance has declined significantly. She should be at the age when she can run and jump the most, but now she has difficulty climbing stairs. She feels chest tightness and shortness of breath after a slight activity, and has symptoms of dry mouth, dry eyes, toothache, caries and recurrent parotitis. The examination results showed that Qingqing had multiple antinuclear antibodies and myositis antibodies in her body, pulmonary ventilation dysfunction with reduced diffusion function, and interstitial lung lesions… Qingqing was finally diagnosed with refractory JDM secondary to Sjögren’s syndrome.
“We took the child to many hospitals, tried various treatments, and invested a lot of time and money, but the child’s condition did not improve, and even his health got worse and worse. We really don’t know what to do.” Qingqing’s mother said desperately.
After 17 days, she can stop taking the medicine and be discharged smoothly
In August 2024, Qingqing’s family, who are at their wits’ end, come to the Rheumatology and Immunology Department of Zhejiang University Children’s Hospital, where they have been treated before, to seek treatment again.
When Qingqing’s parents learned from the medical staff that the Rheumatology and Immunology Department of Zhejiang University Children’s Hospital had just been approved for a clinical research project on CAR-T for the treatment of refractory juvenile dermatomyositis, they immediately applied for Qingqing, hoping to solve their child’s pain by trying CAR-T, a new treatment method.
“Qingqing had received corresponding treatment in our hospital before. At that time, after using conventional treatment methods such as hormones, immunosuppressants, immunoglobulins and various targeted biological agents, the condition kept recurring and the effect was very limited. For Qingqing, who has no medicine to treat, the new method of CAR-T treatment may help her rebuild her own immunity and achieve the possibility of self-healing.” Director Lu Meiping conducted a comprehensive assessment of Qingqing’s situation and approved the application for enrollment in the trial.
CAR-T therapy is a relatively new type of immunotherapy, which is a chimeric antigen receptor T lymphocyte therapy targeting CD19. It extracts T cells from the patient’s own immune system, cultures and modifies them in vitro, equips these T cells with special molecules so that they can recognize and attack specific target cells, and then injects the modified T cells back into the patient’s body to eliminate the target cells.
CD19 is widely expressed on the surface of B lymphocytes. While CD19 CAR-T kills pathogenic target cells, it can also cause deep depletion of other CD19+ B cells, thereby achieving immune reconstruction for patients with autoimmune diseases including juvenile dermatomyositis, completely changing the current situation of such patients needing to take hormones and immunosuppressants for a long time, and achieving drug-free relief or even cure.
On September 20, the rheumatology and immunology team achieved single collection for Qingqing.
On October 11, Qingqing was reinfused with CAR-T cells, and the entire reinfusion process was very smooth.
On October 28, Qingqing was discharged smoothly. Since then, she no longer needs to rely on drugs to live, and only needs regular follow-up.
Pioneering CAR-T Treatment for Refractory Juvenile Dermatomyositis Secondary to Sjögren's Syndrome
China Health Trip takes you through the latest medical breakthroughs in China, showcasing the possibilities of advanced healthcare. Read on for more insights.
On October 28 2024, the National Clinical Research Center for Child Health and Disease, the National Children’s Regional Medical Center, and the Children’s Hospital Affiliated to Zhejiang University School of Medicine (hereinafter referred to as ZJU Children’s Hospital) received good news: Qingqing (pseudonym), the world’s first CAR-T treatment for refractory juvenile dermatomyositis (JDM) with secondary Sjögren’s syndrome, has successfully stopped medication and was discharged smoothly!
CAR-T treatment advancements in China
Lu Meiping, the head of the CAR-T treatment of refractory juvenile dermatomyositis clinical project and director of the Department of Rheumatology and Immunology at Zhejiang University Children’s Hospital, said: “Tingting’s successful discontinuation of medication will bring new hope of complete recovery for other children with refractory juvenile dermatomyositis to completely get rid of the torture of the disease and drug dependence.”
Refractory juvenile dermatomyositis is a common pediatric autoimmune disease second only to systemic lupus erythematosus. The disease causes inflammation of the child’s skin and muscles, and may affect other systems such as the lungs and gastrointestinal tract. Children have to rely on drugs such as hormones and immunosuppressants to alleviate the condition. But at the same time, some children still respond poorly to these treatments, with repeated procrastination, skin ulcers/calcification, severe interstitial lung lesions, gastrointestinal perforation, and even life-threatening.
In June 2024, an article in the journal Arthritis Rheumatol reported the world’s first successful case of JDM treated with anti-CD19 CAR-T cells. In order to help more children with refractory juvenile dermatomyositis, Director Lu Meiping followed the international pace and led the rheumatology and immunology team to conduct a CAR-T treatment project for refractory juvenile dermatomyositis.
This is another clinical study of CAR-T treatment by Zhejiang University Children’s Hospital after completing the world’s largest sample CAR-T treatment project for pediatric systemic lupus erythematosus. The patient Qingqing also became the world’s first successful case of CAR-T treatment of refractory juvenile dermatomyositis (JDM) secondary to Sjögren’s syndrome.
“As a national dual center, the comprehensive strength of Zhejiang University Children’s Hospital ranks first in China, providing strong support for the clinical trial application of CAR-T technology. During the clinical trial, we also received strong support from Professor Mao Jianhua, Vice President of Zhejiang University Children’s Hospital and Director of the Kidney and Urology Center, the hospital ethics committee, and the cell apheresis of the Department of Hematology and Oncology. It is with everyone’s joint efforts that we hope to expand the field of CAR-T treatment through clinical trials and help children with refractory juvenile dermatomyositis relieve more pain.” Director Lu Meiping said.
A 16-year-old girl suffers from refractory juvenile dermatomyositis
16-year-old Qingqing is a child with refractory juvenile dermatomyositis. At the age of flowers, she has been enduring the pain and torture of juvenile dermatomyositis…
More than 8 years ago, Qingqing suddenly developed a purple-red rash with edema around her eyelids and eye sockets, and a bright red scaly rash appeared on the edges of her finger joints, accompanied by muscle soreness, decreased muscle strength, and increased muscle enzymes. After being sent to the hospital, she was diagnosed with juvenile dermatomyositis.
In order to control the onset of the disease, Qingqing has been receiving hormones, immunosuppressants, immunoglobulins and various targeted biological agents for more than 8 years. However, the prognosis has always been less than ideal, and the condition has always recurred, and the child’s body and mind have suffered.
Especially in the past two years, Qingqing’s activity endurance has declined significantly. She should be at the age when she can run and jump the most, but now she has difficulty climbing stairs. She feels chest tightness and shortness of breath after a slight activity, and has symptoms of dry mouth, dry eyes, toothache, caries and recurrent parotitis. The examination results showed that Qingqing had multiple antinuclear antibodies and myositis antibodies in her body, pulmonary ventilation dysfunction with reduced diffusion function, and interstitial lung lesions… Qingqing was finally diagnosed with refractory JDM secondary to Sjögren’s syndrome.
“We took the child to many hospitals, tried various treatments, and invested a lot of time and money, but the child’s condition did not improve, and even his health got worse and worse. We really don’t know what to do.” Qingqing’s mother said desperately.
After 17 days, she can stop taking the medicine and be discharged smoothly
In August 2024, Qingqing’s family, who are at their wits’ end, come to the Rheumatology and Immunology Department of Zhejiang University Children’s Hospital, where they have been treated before, to seek treatment again.
When Qingqing’s parents learned from the medical staff that the Rheumatology and Immunology Department of Zhejiang University Children’s Hospital had just been approved for a clinical research project on CAR-T for the treatment of refractory juvenile dermatomyositis, they immediately applied for Qingqing, hoping to solve their child’s pain by trying CAR-T, a new treatment method.
“Qingqing had received corresponding treatment in our hospital before. At that time, after using conventional treatment methods such as hormones, immunosuppressants, immunoglobulins and various targeted biological agents, the condition kept recurring and the effect was very limited. For Qingqing, who has no medicine to treat, the new method of CAR-T treatment may help her rebuild her own immunity and achieve the possibility of self-healing.” Director Lu Meiping conducted a comprehensive assessment of Qingqing’s situation and approved the application for enrollment in the trial.
CAR-T therapy is a relatively new type of immunotherapy, which is a chimeric antigen receptor T lymphocyte therapy targeting CD19. It extracts T cells from the patient’s own immune system, cultures and modifies them in vitro, equips these T cells with special molecules so that they can recognize and attack specific target cells, and then injects the modified T cells back into the patient’s body to eliminate the target cells.
CD19 is widely expressed on the surface of B lymphocytes. While CD19 CAR-T kills pathogenic target cells, it can also cause deep depletion of other CD19+ B cells, thereby achieving immune reconstruction for patients with autoimmune diseases including juvenile dermatomyositis, completely changing the current situation of such patients needing to take hormones and immunosuppressants for a long time, and achieving drug-free relief or even cure.
On September 20, the rheumatology and immunology team achieved single collection for Qingqing.
On October 11, Qingqing was reinfused with CAR-T cells, and the entire reinfusion process was very smooth.
On October 28, Qingqing was discharged smoothly. Since then, she no longer needs to rely on drugs to live, and only needs regular follow-up.
