Committed to providing breakthrough treatment for endocrine patients and improving the level of diagnosis and treatment of endocrine-related diseases in China, the first "Vitriosteria-Visceral Endocrinology Summit Forum" was held online from March 28 to April 1, 2022, focusing on growth hormone deficiency (GHD), hypochondriatics (ACH), hypoparathyroidism (HP), obesity, type 1 diabetes (T1DM) and other hot endocrine issues for a five-day academic week.
Highlights Sneak Peek:
1
In children/adults with growth hormone deficiency, long-acting growth hormone may be a good choice
2
There are no specific drugs for ACH treatment, and targeted therapy is expected to improve clinical outcomes in patients
3
Traditional alternative hp treatments face five major dilemmas, with transCon PTH emerging to improve patient survival
4
Pay attention to the basis of life management, avoid hereditary obesity missed diagnosis, and prevention is more important than treatment
T1DM emphasizes multidisciplinary consultation, and monoclonal antibody drugs initially show preventive effects
The conference specially invited Professor Fu Junfen of children's hospital affiliated to Zhejiang University School of Medicine, Professor Luo Xiaoping of Tongji Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology, Professor Gu Xuefan of Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Professor Xia Weibo of Peking Union Medical College Hospital, Professor Gong Chunxiu of Beijing Children's Hospital affiliated to Capital Medical University, and Professor Luo Feihong of Children's Hospital Affiliated to Fudan University as the hosts of the conference, bringing together big coffee and innovation leaders in the field of endocrinology at home and abroad to attend and deliver wonderful speeches and share international cutting-edge information.
DAY1
From children to adults, explore optimal management of growth hormone deficiencies
Growth hormone from research to the application of growth disorders treatment has spanned more than a century, the mainland clinical application is mainly based on short-acting growth hormone, its effectiveness, safety is relatively certain, but the treatment of GHD (growth hormone deficiency) is a long-term process, the inconvenience of daily injections, pain, and injection fear seriously affect treatment compliance, patients and families tend to choose a less frequent injection regimen [1].
Professor Gong Chunxiu of Beijing Children's Hospital affiliated to Capital Medical University pointed out that in order to improve such clinical phenomena, long-acting growth hormone (LAGH) preparations may represent a more advanced program than daily injection of growth hormone, with the potential to improve compliance and outcomes, there are mainly safety concerns in the current research and development, and technologies that do not change molecular structure such as sustained-release microsphere administration systems and TransCon temporary connection technology may have certain advantages, in addition to dose adjustment, Long-term effectiveness is also a matter of consideration.
For LAGH TransCon hGH ( Lombeuil Growth Hormone), Professor Bradley Miller of the University of Minnesota School of Medicine presents the latest findings: In children with GHD, it has been shown that its weekly administration is superior to that of short-acting growth hormone, while its safety and tolerability are similar [2].
GHD is not just about child growth, it can occur from the transition period to adulthood. Professor Chen Xiaobo of the Children's Hospital Affiliated to the Capital Institute of Pediatrics mentioned that transitional growth hormone deficiency (TGHD) should be carefully evaluated for patients' clinical manifestations and laboratory results after the application of recombinant human growth hormone (rhGH) to enable patients with TGHD to maintain normal body composition and glycolipid metabolic balance in adulthood, and to consider long-term or even lifelong rhGH replacement therapy [3]. According to Professor Zhu Huijuan of Peking Union Medical College Hospital, the use of rhGH can improve clinical symptoms and reduce the occurrence of complications in patients with AGHD, improve body composition and motor capacity, reduce cardiovascular risk, increase bone quality, and improve quality of life [4].
Expert Discussion Session: Identify the pain points of transitional diagnosis and treatment that are not enough for GDH to manage the whole process, and look forward to the application prospects of LAGH
Focusing on the pain points of the whole process of GDH management, experts believe that the docking between child/adult endocrinologists, especially the diagnosis and treatment of the transitional period, including the lack of follow-up in the long-term management process, is a deficiency in the current clinical practice, so fully understand the importance of full life cycle management, work closely together, and jointly complete the treatment and management of GHD. In addition, for the application prospects of LAGH, experts combined with their own clinical experience to say that it effectively strengthens the acceptance and compliance of patients, from the current monitoring data the overall safety is relatively good, the future is also looking forward to the emergence of sufficient safety data, and constantly summarize the experience, do a good job in laGH dose selection rationalization, standardization.
DAY2
The prevalence is low but the harm is large, how to break through the underdevelopment of cartilage in mainland China?
Limited data from China indicate that the prevalence of ACH (hypoplasia of cartilage) in mainland China is low, but the disease is more harmful, and although the children have good intellectual and physical development, most children have manifestations such as asymmetrical short stature and are accompanied by complications such as spinal deformities [5]. Professor Liu Li of Guangzhou Women and Children's Medical Center introduced that there is currently a lack of large sample data in China, sponsored by the China Rare Disease Alliance, funded by Weisheng Pharmaceutical, and participated in a national multi-center registration study (ApproaCH) in ACH patients, which is currently in progress, and the preliminary outlook will bring in 2025 to depict the demographic and clinical characteristics, growth and development standards, comorbidities of Chinese ACH patients. Comprehensive reporting of data on the status of diagnostic and medical interventions.
Professor Yu Yongguo of Xinhua Hospital affiliated to Shanghai Jiao Tong University School of Medicine said that there are currently no specific drugs for ACH treatment, and most of the new drugs for the molecular causes of ACH are currently in the preclinical research stage, of which two C-type natriuretic peptide analogues (Vosoritide and TransCon CNP) have entered the clinical trial stage. Professor Irving Melita of the Guy and St. Thomas National Health Trust highlighted ACH molecular targets and emerging treatment options, such as TransCon CNP, which maintains CNP levels, is effective and well tolerated[6], and anti-FGFR3 therapy, which has the potential to alter the natural history of cartilage hypoplasia and improve clinical outcomes in children and adults with cartilage hypoplasia.
Expert Discussion Session: The sooner ACH treatment, the better, and we are looking forward to the results of the ApproaCH study
Around the problem of the best treatment time window and treatment cycle of ACH, experts believe that the earlier the treatment, the better, and the epiphyseal fusion in the adult stage has lost its therapeutic significance, and after early treatment, it is maintained until adulthood, and if there is a complication, it is still necessary to continue treatment, which requires the current research and development of drugs to enter the clinic, as well as the accumulation of more evidence, and combined with the safety of long-term medication to determine. For the application prospects of approved and under-development drugs, experts believe that although ACH is a rare disease, there is still a considerable number of patients, and while looking forward to the results of the ApproaCH study, they also hope to include ACH in the Chinese rare disease directory, thus bringing more protection and greater help to patients.
DAY3
Current status and progress in the treatment of hypoparathyroidism by "symptoms" and "causes"
HP (Hypoparathyroidism) is a rare endocrine disease that can be complicated by long-term renal insufficiency, kidney stones, cataracts, and intracranial calcifications. Professor Wu Yangfeng of the Clinical Research Institute of Peking University introduced that the epidemiological data on the chronic HP population in the mainland is almost zero, and its disease, diagnosis, treatment, control and even the demand and cost of medical treatment are still blank. The Current Status Study on the Diagnosis and Treatment of Hypoparathyroidism in China (PaTHway R), which is currently being conducted by 7 top hospitals in seven regions of the mainland, is expected to obtain chronic HP prevalence, clinical features, missed diagnosis and misdiagnosis, complication rate, treatment rate, compliance rate, medical cost, patient quality of life score and unmet needs, in order to fully understand the clinical diagnosis and treatment information of chronic HP patients in mainland China, in order to enrich the evidence-based medical evidence in this field. Provide an important reference for the national designation of relevant rare disease diagnosis and treatment guidelines and policies.
Professor Xie Zhongjian of the Second Xiangya Hospital of Central South University proposed five major treatment dilemmas of the current hp traditional alternative treatment, including difficult to stabilize blood calcium levels, difficult to balance blood calcium and urine calcium levels, increasing the risk of kidney complications, unable to reduce the blood phosphorus and calcium phosphorus product, difficult to improve ectopic calcification, unable to correct abnormal calcium and phosphorus metabolism in bones and kidneys, and seriously impaired quality of life. In the face of these dilemmas, 84 weeks of data from PaTH Forward OLE (still in the study phase) support TransCon PTH as a potential alternative therapy for HP patients, with more than 93% of subjects discontinuing traditional treatments and blood calcium levels maintained within the normal range, significant reductions in plasma levels of blood phosphorus, blood calcium, and calcium phosphorus, reduced urinary calcium excretion, normalization of bone remodeling, and significant improvement in quality of life.
Expert Discussion Session: Listen to patient needs and strengthen awareness of the disease and the ideal hormone replacement therapy
Around the management of HP's existing treatment, experts believe that for the hormone deficiency caused by HP, there is currently no hormone that can be replaced, and it can only be treated by supplementing calcium, vitamin D and other alternative methods, which is also a helpless move; at the same time, doctors need to listen more to the needs of patients and pay attention to the quality of life. For the possible impact of ideal hormone replacement therapy, experts believe that on the one hand, primary doctors need to strengthen their understanding of HP, avoid missed diagnosis and misdiagnosis, and rationally use drugs, on the other hand, patients still need long-term management, and also look forward to the listing of TransCon CNP to improve the survival status of HP patients.
DAY4
Pediatric and adult experts join hands to talk about obesity management
Overweight/obesity is increasingly a serious public health problem in mainland China, predicting that by 2030, the prevalence of overweight/obesity among mainland adults will reach 65.3%, 31.8% of school-age children and adolescents, and 15.6% of preschoolers [7].
Professor Chen Lulu of Union Medical College Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology and Professor Chen Wei of Peking Union Medical College Hospital said that the growth of overweight/obesity in China is driven by systemic dynamic factors and environmental determinants through unhealthy diet and lack of physical activity[8], and lifestyle management is the basis for obesity treatment, especially diet, exercise and behavior management [9].
At the same time, Professor Gong Chunxiu of Beijing Children's Hospital affiliated to Capital Medical University and Professor Wu Wei of Children's Hospital Affiliated to Zhejiang University School of Medicine shared clinical weight loss examples, and said that while affirming that intensive diet and exercise can effectively reduce weight, we should also pay attention to medical history collection and related examinations, and timely discover rare hereditary obesity to avoid missed diagnosis.
Expert discussion session: Emphasize the change in adolescent obesity concepts, prevention is more important than treatment
Around the causes of adolescent obesity, experts believe that mainland adolescent obesity is mainly nutritional obesity, which is closely related to lifestyle, and is inseparable from the mainland public's resistance to children and adolescents to lose weight, so it is necessary to change the concept, educate what is the real state of health, and find safer ways to lose weight for children and adolescents. For the differences in obesity management between children and adults, experts believe that it is mainly reflected in the more vague diagnosis of children, the lack of initiative in children's weight loss, the lack of intervention methods for children and their susceptibility to external influences, so it is more difficult for children to lose weight, and it also calls for children's obesity to be mainly prevented.
DAY5
Pediatric and endemic specialists worked together to analyze the prevention and treatment of type 1 diabetes
On the mainland, children and adolescents are still dominated by T1DM (type 1 diabetes), a major pediatric endocrine disorder that endangers children's health [10]. Professor Luo Feihong of the Children's Hospital of Fudan University and Professor Yang Tao of Jiangsu Provincial People's Hospital proposed that in different stages of the natural course of T1DM, the treatment plan is different, and in addition to the clinical characteristics such as age of onset, acute and slow onset, severity of onset, and metabolic indicators, it is also necessary to do a good job in the evaluation of islet function, immunological indicators, and genetic genes, which needs to be fully considered.
At the meeting, Professor Francisco Leon of Provention Bio Company in the United States shared an experimental drug that delays and treats early T1DM - anti-CD3 monoclonal antibody (Teplizumab), and the research phase results show that the drug can reverse the decline of C-peptide and delay clinical T1DM by about 3 years.
Expert Discussion Session: T1DM referral in children focuses on multidisciplinary consultation, expecting further results from prophylactics
Around the issue of pediatric to adult T1DM referral, experts believe that for children generally pediatric follow-up to 18 years old, if the initial diagnosis age is over 14 years old, you can directly visit the adult department, if the child patient encounters ketoacidosis, renal complications, etc., will also be introduced to the corresponding adult department for treatment, and pay attention to the organization of multidisciplinary consultation (MDT). For the prevention prospect of T1DM, experts believe that in the early stage of diabetes β cell function is better, the use of immune agents targeted therapy for preservation, is the direction of future prevention, the current anti-CD3 monoclonal antibody appears to show the preventive effect for clinicians to establish confidence, but also look forward to further research results.
epilogue
The five-day academic week allows more clinicians to have a deeper understanding of hot endocrine problems such as GHD, ACH, HP, obesity, and T1DM, and further understand the clinical pain points, diagnosis and treatment status of related diseases in the mainland, as well as promising treatment methods in the future.
Taking this as an opportunity, it is believed that the interaction and exchange of children and adult endocrinologists in mainland China can be further promoted, with a view to helping the overall improvement of the diagnosis and treatment level of endocrine diseases in mainland China and improving the survival status and clinical outcomes of endocrine patients in mainland China.
This article is for the reference of medical and health professionals only
Approval code: VS-MA-ENDOSUMMIT-2022-013
Content Planner: Chen Jing/Ma Yan
Content Review: Zhou Jie
Legal Review: Zhou Jie
Title image source: Stand Cool Helo
bibliography:
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[3] Endocrinology Genetics and Metabolism Group, Science Branch of Chinese Medical Association, Editorial Board of Chinese Journal of Pediatrics. Expert consensus on the diagnosis and treatment of transitional growth hormone deficiency[J]. Chin J Pediatrics,2020,58(6):455-460.
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[5] Dai Weiqian, et al. Clinical genetic characteristics analysis and growth and development curve exploration of 210 children with cartilage hypoplasia in China[J]. Chin J Pediatrics, 2020, 58(6):7.
[6] Breinholt VM, et al. TransCon CNP, a sustained-release C-Type Natriuretic Peptide prodrug, a potentially safe and efficacious new therapeutic modality for the treatment of comorbidities associated with FGFR3-related skeletal dysplasias. J Pharmacol Exp Ther. 2019 Sep;370(3):459-471.
[7] Youfa Wang et al. Health policy and public health implications of obesity in China. Lancet Diabetes Endocrinol. 2021 Jul;9(7):446-461.
[8] Pan XF, et al. Epidemiology and determinants of obesity in China. Lancet Diabetes Endocrinol 2021;9: 373–392
[9] American association of cilinical enodcrinologists and American college of endocrinology comprehensive clinical practice guidelines for medical care of patients with obesity, Endocr Pract. 2016 Jul;22(7):842-84.
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