Source | Zhejiang University Institute
On October 28th, the National Childrens Health and Disease Clinical Medicine Research Center, National Childrens AreaMedical Center and Childrens Hospital of Zhejiang University Medical College (hereinafter referred to as Zhejiang University Childrens Hospital) have good news: the worlds first CAR-T the treatment of refractory younger dermatitis (JDM) secondary dry syndrome, QingqingPseudonym) has successfully stopped the medicine and was discharged smoothly!
CAR-T to treat refractory younger dermatitis clinical project leaders, Lu Meiping, director of rheumatism and immunology department of Zhejiang University, said: "QingqingThe successful stopping of the drug will be a new hope for other patients with dermatitis to completely get rid of disease torture and drug dependence.Steriitis is a more common childrens autoimmune disease with systemic lupus erythematosus.The disease can cause childrens skin and muscles to inflammation, as well as other systems such as lungs and gastrointestinal tract. Children have to rely on drugs such as hormones and immunosuppressants to alleviate the disease.However, at the same time, some children still have a poor response to these treatment. The condition has repeatedly prolonged, skin ulcer/, severe interproval lesions, gastrointestinal perforation, and even life-threatening.
In June 2024, an article from Arthritis Rheumatol magazine reported the worlds first JDM case that was successfully used for anti-CD19 CAR-T cells.In order to help more children with cure younger dermatitis, Director Lu Meiping followed the international pace and led the Rheumatology and Immune Team to conduct CAR-T the treatment of refractory younger dermatitis projects.
This is another clinical study of CAR-T treatment after completing the first global largest sample CAR-T to treat childrens systemic lupus erythematosus project.Qingqing, a child, has also become the first successful case of a secondary dry syndrome in the world for the treatment of refractory younger dermatitis (JDM).
"Zhejiang University Pediatric Academy as the national dual center, comprehensive strength ranks in the forefront of the first party in China, and is the clinical trial of CAR-T technologyThe application provides a strong guarantee.Below, we hope that through clinical trials, we will expand the field of CAR-T therapy and help children with dermatitis in refractory younger age to relieve more pain. "Director Lu Meiping said.
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16 -year -old Qingqing is a child with cure younger dermatitis. In a beautiful age, she, sheHowever, he has always endured the pain and torture of youth dermatitis ...
More than 8 years ago, the purple -red rashes with edema suddenly appeared around the eyelids and the orbits.There are also bright red scales, and it is also accompanied by muscle soreness, muscle decreased, and high muscle enzymes.
In order to control the onset, in these 8 years, Qingqing has been constantly receiving hormone, immunosuppressives, C -ball and multiple targeting biological agents.However, the prognosis is always not ideal, the condition is always repeated, and the childs physical and mental is tortured.
Especially in the past two years, Qingqings activity endurance has declined significantly. It should have been the most capable age. Now it ’s hard to climb a staircase.There are also symptoms of dry mouth, dry eye, toothache, dental caries and repeated mumps.The test results showed that the positive anti -nuclear antibodies and myositis antibody were positive in Qingqings body, and the pungent pneumonic dysfunction was accompanied by a reduction function, and the lungs were meticulous.
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"I took the child to multiple hospitals, tried various treatments, invested a lot of time, money, but the childs condition did not improve, and even the body was still in the body.It ’s worse and worse, we really do n’t know what to do.
Try CAR-T clinical trials
After 17 daysDis -hospitalIn August 2024, the unusual Qingqing family came to the Rheumatology and Immunology Department of Zhejiang University of Zhejiang University, which was visited before seeking treatment.
CAR-T clinical trial project launch meeting of the treatment of refractory JDM
In the mouth of the medical staff, after learning that the Rheumatology and Immunology Team of Zhejiang University had just approved the clinical research project of CAR-T to treat refractory young dermatitis, Qingqings parents applied for Qingqing as soon asThis new treatment method is to solve the childs illness.
"You had received the corresponding treatment in our hospital before Qingqing. At that time, after using conventional treatment such as hormones, immunosuppressants, C -balls and various targeting biological agents,The condition is always repeated and the effect is very limited.After conducting a comprehensive evaluation, the application for the entrance test was passed.
CAR-T therapy, which is targeted at CD19 chimeric antigen receptor T lymphocyte therapy, is a relatively new immunotherapy.It extracts the T cells of the patients autoimmune system. After in vitro culture and transformation, special molecules are used for these T cells, enabling them to identify and attack specific target cells, and then inject the transformed T cells back to the patients body.Tr cells are used to destroy target cells.
CD19 is widely expressed on the surface of B lymphocytes. While killing the pathogenic target cells, CD19 CAR-T can also cause the depth of other CD19+B cells, which is expected to achieveAutoimmune diseases include immune reconstruction of patients with younger dermatitis, the status quo of long -term use of hormones and immunosuppressants for long -term use of such patients, and reaching no medicine to relieve or even cure.
The nursing team lost CAR-T cells for children
September 20, September 20,The rheumatism and immunology team achieved a single mining for Qingqing.
On October 11, it was a CAR-T cell for Qingqings return to the back of the loser.
Figure 1 is left for migrant gland swelling photos (before CAR-T); right for 14 days after CAR-T treatment, comparison of the parotid glands back and forth, comparison of the parotid glands before and after the parotid glands.After the area narrowed before
After completing CAR-T cell therapy, Qingqings overall situation is good.After all the drugs were discontinued, there was no fever and toothache, and the condition of the perfusion of the parotid glands was reduced compared to the previous, and the rash and muscle weakness did not worsen after stopping the drug.
On October 28, Qingqing was discharged smoothly. Since then, she no longer needs to rely on drug life.
CAR-T clinical trials of refractory young dermatitis
Recruiting subjects
Director Lu Meiping said that CAR-T cell therapy is expensive and the market price reaches one million, but the children are difficult to treat by the admission of CAR-T treatment.The clinical trials of dermatitis (JDM) are supported by scientific research funds and related companies provide free services. Families in children only need to pay conventional medical expenses other than CAR-T cell therapy, and the burden of medical care has been greatly reduced.
"At present, the clinical trials of CAR-T the treatment of refractory younger dermatitis (JDM) are still recruiting subjects for free.The project can bring breakthroughs for more similar patients! "Director Lu Meiping said.
Project Name: A targeted CD19 church antigen receptor T lymphocyte (CAR-T) to treat refractory younger dermatitis.
Selected criteria: The subject must meet all the following standards to be selected:
(1) Age ≥ 5 years;
(2) Diagnose diagnosis according to the Bohan and Peter standards as young dermatitis (JDM);
(3) meet the classification standards of RJDM, meet 1And 2 ~ 5 any conditions:
① At least 2 immunization for glycogenic cortex (1 ~ 2 mg/kg/d or equivalent dose)Inhibitors are not tolerated or reactless, hormone therapy duration is at least 6 months;
② The disease progresses rapidly and/or organs such as lungs, hearts, and gastrointestinal tract;
③ calcification of subcutaneous or muscle and joint tissue;
④ Repeated rash or skin ulcer;
(4) For those who are involved in muscles, childrens myogitis evaluation table (CMAS) scores & lt; 48 points, and belowAt least 2 abnormal results of the 5 core measurement indicators: the overall evaluation of the physician (PHGA) ≥2cm, the patients overall evaluation (PTGA) ≥2cm, the disease activity scoring score table (DAS) ≥2 points, and the child health assessment questionnaire (C C (C C-HAQ) Total score ≥0.25 points, muscle enzyme level & gt; 1.5 × upper limit (ULN);
(5) The necrotic muscles of the necrotic muscles of SRP or HMGCR antibody -positive immune mediaThe patient meets RJDM and can be directly included in;
(6) The function of important organs is basically normal:
① Heart function: Left room shootingBlood score (LVEF) ≥55%, there is no obvious abnormality in the electrocardiogram;
② renal function: EGFR ≥ 30ml/min/1.73m2;
③ liver function: AST and Alt≤3.0uln, total bilirubin ≤ 2.0 × uln (except for primary diseases);
④ lung function: SPO2 ≥92%;
(7) With the standard of single mining or venous blood collection, and no other cell collection taboos;
(8) Early age -age period sufferingTest the results of the results of the urine pregnancy test are negative, and agreed to take effective contraceptive measures during the test until 1 year after the infusion;The test and signed the informed consent, indicating that it understands the purpose and procedure of this clinical trial and is willing to participate in the study.
Number of subjects: It is expected that 4-11 cases are enrolled in this study
Project contact person: Dr. Ma QianTel: 0571-81732564, 0571-81732562, 19511273786
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