Takeda China's First Pediatric High-Level Forum Successfully Held, Showcasing Landmark '4001 Study' on Central Precocious Puberty

Shanghai, July 14, 2020 /PRNewswire/ -- From July 10 to 12, 2020, the inaugural "Pressing Forward with Courage, Building Dreams Without Limits" Pediatric Summit, hosted by Takeda China, was grandly launched. More than 1,500 pediatric professionals from China and abroad gathered virtually to witness the landmark release of "Study 4001," the largest prospective, multicenter study in terms of sample size conducted in China on central precocious puberty (CPP) in children. Participants also engaged in discussions on hot topics such as quality control in pediatric diagnosis and treatment, professional development of early- and mid-career physicians, and child health management in the post-pandemic era.

“Forging Ahead, Building Infinite Dreams” Pediatric Summit is Takeda China’s response to the “Healthy Children” initiative proposed in the Outline of the “Healthy China 2030” Planning Program.^[1]This pediatric summit in the field of child health was initiated to strengthen early childhood development, enhance pediatric care infrastructure, and intensify prevention and treatment efforts for key pediatric diseases. It is committed to building an international academic exchange platform for medical experts and young-to-middle-aged physicians in pediatrics, working together to safeguard the future health of children in China. At this forum, several renowned experts attended, including Professor Luo Xiaoping, Vice Chairman of the Pediatric Branch of the Chinese Medical Association and Director of the Department of Pediatrics at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology; Professor Fu Junfen, Head of the Endocrinology, Genetics, and Metabolism Group of the Pediatric Branch of the Chinese Medical Association, Executive Vice President of the National Clinical Research Center for Child Health and Diseases, and Affiliated Children's Hospital of Zhejiang University School of Medicine; Professor Gong Chunxiu, Director of the Department of Endocrinology, Genetics, and Metabolism at Beijing Children's Hospital, Capital Medical University (National Center for Children's Health), and Chairman of the Pediatric Endocrinology, Genetics, and Metabolism Committee of the Chinese Medical Doctor Association; and Dr. Maria G. Vogiatzi, Associate Professor of Pediatrics and Endocrinology Expert at Children's Hospital of Philadelphia. They engaged in in-depth discussions on frontier hot topics and research advances, such as the treatment of precocious puberty in children, pooling wisdom from multiple parties to jointly realize the Chinese Dream of child health.

4001 Study First Published, Providing Strong Clinical Support for Research on the Treatment of Precocious Puberty in China

# Diagnosis and Treatment of Central Precocious Puberty (CPP) in Children: Core Topics of the Summit ForumCentral precocious puberty (CPP) is a common pediatric endocrine disorder caused by the premature activation of the hypothalamic-pituitary-gonadal axis, leading to early secretion of gonadotropin-releasing hormone from the hypothalamus. This results in rapid development of internal and external genitalia and the appearance of secondary sexual characteristics before the age of 8 in girls and before the age of 9 in boys.^[2]Its effects on the body mainly manifest as follows: Precocious sexual development leads to early menarche in girls; accelerated skeletal maturation results in a bone age exceeding chronological age and premature epiphyseal closure, thereby compromising the child’s final adult height; premature development of secondary sexual characteristics and sexual maturity may give rise to corresponding psychological issues or social behavioral abnormalities, severely affecting children’s physical and mental health, which warrants urgent attention.^[3]。

The safety and efficacy of treatment for central precocious puberty (CPP) in children have long been a focal point of widespread concern. Since 2015, Takeda China has supported Professor Luo Xiaoping, Deputy Chairman of the Pediatric Branch of the Chinese Medical Association and Director of the Department of Pediatrics at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, in leading the initiation of the “Study 4001,” the largest prospective, multicenter study in terms of sample size in the field of CPP in China. This study aims to further investigate the safety and efficacy of standard treatment regimens for CPP. Spanning two years, Study 4001 is designed to evaluate the safety and efficacy of leuprorelin, a gonadotropin-releasing hormone agonist (GnRHa) widely used globally for the treatment of children with CPP.

The results of Study 4001 showed that during the 96-week treatment period, all drug-related adverse events were expected, with an incidence rate of adverse reactions of only 10.7%; the safety profile observed in the study was consistent with the known safety profile, and there were no concerns regarding long-term administration, fully demonstrating the good tolerability of leuprorelin. In terms of efficacy, the incidence rate of Tanner stage regression or lack of progression at Week 96 compared to baseline among female subjects was 83.5%; at Week 96, 90.1% of subjects exhibited suppressed peak LH levels following the stimulation test, and 95.4% of subjects exhibited suppressed peak FSH levels.

Professor Luo Xiaoping stated, “Precocious puberty is one of the diseases that seriously affect the healthy growth of children in China. However, there is currently a significant lack of relevant research on the treatment of precocious puberty in China, particularly large-sample studies. It is gratifying to see that the results of the large-sample Study 4001 have fully demonstrated the safety and efficacy of leuprorelin (a GnRH agonist) in treating children with central precocious puberty (CPP), filling a gap in this field and providing strong clinical support for CPP diagnosis and treatment research in China.”

Standardize Quality Control in Diagnosis and Treatment, Explore New Knowledge through Multi-party Collaboration, and Promote Comprehensive Development of Pediatric Care

In recent years, precocious puberty in children has been on the rise in China.^[4], the incidence rate has been rising year by year, becoming a focal point of social concern. Data shows that the prevalence of precocious puberty among children in China is 0.43%, with approximately 350,000 affected children nationwide.^[5]However, in the diagnosis and treatment of diseases, issues such as low consultation rates and non-standardized treatments still persist in China. At the conference, Professor Fu Junfen, Executive Deputy Director of the National Clinical Medical Research Center for Children's Health and Diseases and Project Leader of the Chinese Pediatric Growth and Development Quality Control Program, discussed how to establish a national quality control platform for growth and development. She emphasized, "The demand for diagnosis and treatment of precocious puberty in children has shown an increasing trend in recent years, posing higher requirements for standardizing diagnostic and therapeutic procedures and strengthening resource integration within the field of pediatric endocrinology in China. We hope that by establishing a national quality control platform for growth and development, we can effectively enhance the overall level of diagnosis and treatment for precocious puberty in children across China, promote comprehensive and coordinated disciplinary development, and enable more Chinese pediatric patients to receive timely, standardized, and efficient care."

During the three-day forum, experts from various sectors also held in-depth discussions on specialized topics, including the professional development of young and middle-aged physicians and children’s health management in the post-pandemic era. According to Professor Gong Chunxiu, during the pandemic, children were prone to obesity and subsequent precocious puberty due to unhealthy lifestyle habits, such as increased food intake and reduced physical activity, a matter that warrants greater attention from parents. She appealed, “In recent years, there has been a rising incidence of precocious puberty in children driven by poor lifestyle habits. There is an urgent need to enhance public awareness of this condition and promote early diagnosis and treatment. Meanwhile, in facing the health management challenges of the post-pandemic era, multiple stakeholders should jointly explore new models for the development of pediatric diagnosis and treatment, and flexibly implement long-acting and convenient therapeutic regimens to ensure that affected children receive scientific, continuous, and standardized effective care.”

Building Dreams Without Limits: Takeda Safeguards the Healthy Growth of Children in China

Children’s health is one of the key areas Takeda continues to prioritize. In China, Takeda actively collaborates with various stakeholders to enhance public awareness and understanding of pediatric diseases, safeguarding children’s healthy growth. Since 2017, Takeda China has supported the “Care & Growth – Public Health Education on Precocious Puberty” initiative, hosted by the China Health Promotion and Education Association. This program has reached 200 primary schools, 20 hospitals, and more than 60,000 parents of patients across China, improving public understanding of the condition from multiple dimensions and fostering correct and standardized diagnosis and treatment concepts. It has also established online and offline communication platforms for families of affected children and healthcare providers, guiding them to seek timely medical care. Since 2018, Takeda has partnered with professional societies specializing in pediatric endocrinology to train community physicians and pediatricians, assisted numerous hospitals in establishing pediatric endocrinology departments, and provided specialized training on growth and development disorders to nearly 80,000 pediatricians, thereby enhancing the professional competence of pediatric endocrinologists.

Mr. Shan Guohong, President of Takeda China, stated, “Children are the future of our nation. Supporting their growth and safeguarding their health is an enduring responsibility for Takeda. With extensive professional expertise and innovative therapeutic solutions in pediatric health management, we aim to leverage this high-level summit to bring together authoritative academic resources, establish a cutting-edge platform for academic exchange, fully utilize Takeda’s professional strengths, and contribute to the ‘Healthy China 2030’ initiative.”

[1] Chapter 10, Section 1 of the "Outline of the Healthy China 2030 Plan," issued by the Central Committee of the Communist Party of China and the State Council in 2016.

[2] Subspecialty Group of Endocrinology, Hereditary and Metabolic Diseases, the Society of Pediatrics, Chinese Medical Association. Consensus on the Diagnosis and Treatment of Central Precocious Puberty (2015).

[3] Ke Jiangwei (reviewed by Duan Rong), “Research Progress on Precocious Puberty and Its Risk Factors.” Experimental and Laboratory Medicine, Vol. 30, No. 3, June 2016.

[4] Writing Group for the Pubertal Development Survey of the Endocrinology, Genetics and Metabolism Subspecialty Group, Society of Pediatrics, Chinese Medical Association. Chinese Journal of Endocrinology and Metabolism. 2010;26

[5] China Statistical Yearbook 2016.