Innovative Drug Brings New Hope: Beijing Children's Hospital Helps 'Ultra-Rare Child Patient' Escape 'Infusion Bag' Dependence

Background

Short bowel syndrome (SBS) is a rare and serious condition that occurs when a large portion of the small intestine is missing or non-functional, often due to surgical resection. Patients are unable to absorb sufficient nutrients and fluids from food, leading to dependence on parenteral nutrition (PN) delivered intravenously. For children, this can result in significant growth delays, developmental issues, frequent hospitalizations, and risks including infections and liver complications from long-term PN use. SBS in the pediatric population is considered ultra-rare, with adult prevalence in China estimated at approximately 0.73 per 1,000,000 and an increasing trend, while incidence data for children remain limited.

One such case involved a child known as Mingming (pseudonym). Following multiple surgeries shortly after birth to treat an intestinal disease, a substantial part of the small intestine was removed. This led to SBS, requiring the child to receive daily parenteral nutrition support at the hospital. The child was unable to leave the facility and showed growth and development markedly behind that of peers. The General Surgery Department at Beijing Children's Hospital, Capital Medical University, which frequently manages rare and complex pediatric cases as part of the National Children's Medical Center, addressed the case.

What was achieved

In late 2025, the General Surgery Department applied teduglutide for this pediatric SBS patient. Teduglutide is reported as the world's first and currently the only approved innovative drug for short bowel syndrome in patients aged one year and older. It is a glucagon-like peptide-2 (GLP-2) analog.

According to Huang Liuming, chief physician in general surgery, after three months of treatment with the drug, the child completely discontinued parenteral nutrition and escaped dependence on the infusion bag. Autonomous intestinal function was restored. Huang Liuming stated that Beijing Children's Hospital, as a national children's medical center, regularly encounters rare and complex cases. This successful application accumulated valuable clinical experience for the department and contributed to the hospital's broader rare disease diagnosis and treatment framework. Future expansion of innovative drug applications is expected to benefit more such children.

How it works

In SBS, the shortened or damaged intestine lacks adequate surface area to absorb nutrients, fluids, and electrolytes from oral or tube feeding. Standard management has relied on PN, where a nutrient solution is infused directly into the bloodstream through a vein, often requiring a central line and daily or frequent administration. While life-sustaining, prolonged PN carries substantial risks and limits normal childhood activities.

Teduglutide works by mimicking the action of natural GLP-2, an intestinal hormone. It stimulates growth and adaptation of the remaining bowel tissue. It increases the length of intestinal villi—the microscopic projections responsible for absorption—and the depth of crypts, where new intestinal lining cells are generated. These changes enlarge the absorptive surface area, enhance barrier function, and improve the gut's capacity to utilize enteral nutrition. Consequently, patients can gradually increase feeds from food while decreasing PN volume, potentially achieving full independence from intravenous support. In this Beijing Children's Hospital case, the process enabled complete weaning from PN within three months.

What it means for international patients

Beijing Children's Hospital manages numerous complex and rare pediatric conditions through its Center for Complex and Rare Case Consultation, multidisciplinary teams, and high-volume pediatric surgery expertise. The hospital accepts international and foreign patients, who register using passports, and maintains an English-language website along with special needs outpatient services to support access for overseas families.

For cases involving short bowel syndrome or therapies such as teduglutide, suitability depends on individual factors including the child's age, post-surgical intestinal adaptation period, residual bowel length, and overall clinical stability. The hospital's documented experience with this pediatric case illustrates its capacity in rare disease management and innovative applications. International patients should contact the hospital directly to confirm eligibility, undergo case review by the relevant specialists such as those in general surgery, and discuss individualized treatment plans. Availability and appropriateness must be verified with the hospital, as outcomes vary and require ongoing follow-up.

About the hospital

Beijing Children's Hospital, Capital Medical University, located in Beijing, is designated as the National Center for Children's Health and serves as the primary hospital for the National Children's Medical Center. It is a Grade 3A comprehensive pediatric hospital with origins tracing to 1942, founded by pioneers of modern Chinese pediatrics including Zhu Futang. The institution integrates medical care, research, education, and child health services across multiple campuses, including the main Xicheng campus with approximately 970 beds.

It handles an average of approximately 2.9 million outpatient visits, around 80,000 inpatient admissions, and more than 35,000 surgical procedures annually. The hospital features the national key discipline of pediatrics, 10 National Clinical Key Specialties including pediatric surgery, pediatric critical care, and others, along with multiple national research platforms. It is recognized for expertise in managing difficult, rare, and complex pediatric cases, including through specialized consultation centers, and its pediatric surgery has been noted as top-ranked in relevant national reputation assessments.