Children's Hospital Of Philadelphia Researchers Find Monoclonal Antibody May Control Crohn's Disease In Children

In addition to providing significant medical benefits in children, it also has the potential to significantly reduce the need for steroids -- a treatment associated with significant side effects. Side effects of current treatments -- corticosteroids and drugs affecting immune responses -- include acne, weight gain, impaired growth, osteoporosis, the risk of hepatitis, and bone marrow suppression.

"Monoclonal antibodies and other biological agents are a technology that we're going to see much more from in the future," said Robert Baldassano, M.D., director of the Center for Inflammatory Bowel Disease at The Children's Hospital of Philadelphia and primary investigator of the study. "They will revolutionize care in many fields of medicine, and we're just at the beginning of what monoclonal antibodies have to offer." Biological agents are chemicals naturally produced in the body that are genetically altered for use as drugs.

The study, a retrospective chart and database review, looked at 82 patients who received monoclonal antibody treatment at The Children's Hospital of Philadelphia. This study population was the largest cohort of pediatric patients who received this treatment for Crohn's disease.

Forty patients were being treated with corticosteroid at the start of treatment with the monoclonal antibody; 33 were eligible to participate. Nineteen of those 33 patients were able to discontinue corticosteroid usage. Few complications or problems were reported in the study. "This study provides strong evidence for the use of infliximab in short-term therapy for children with Crohn's disease," said Dr. Baldassano. "It shows promise that we may soon reduce the need for steroid treatment."

Crohn's disease is a chronic bowel disorder characterized by inflammation of the gastrointestinal tract. Tumor necrosis factor alpha (TNF-a), a protein produced by the immune system, is among the causes of inflammation. It is becoming a common chronic pediatric disease with the average age of onset occurring much younger than before. The average age for diagnosis is 10-11 years, though it can also occur during the adolescent and adult years. Symptoms may include pain, severe abdominal discomfort and more than 30 bowel movements each day. Most children with CD experience ongoing disease activity over the course of their lives, which may be complicated by the need for prolonged medication and numerous surgeries.

Infliximab is a genetically engineered monoclonal antibody that acts against TNF-a. Monoclonal antibodies are naturally occurring human antibodies that are genetically altered in a laboratory, cloned in large numbers and introduced into the patient to target disease sites. Infliximab is engineered from both human and mouse material.

Robert Baldassano, M.D., director of the Center for Pediatric Inflammatory Bowel Disease at The Children's Hospital of Philadelphia, was the primary investigator of the study. Researchers also included Michael C. Stephens, M.D., Melissa A. Shepanski, M.S., Petar Mamula, M.D., Jonathan E. Markowitz. M.D., and Kurt A. Brown, M.D.

The Center for Pediatric Inflammatory Bowel Disease at The Children's Hospital of Philadelphia was established in 1996 and has cared for more than 3,000 children with IBD, making it the largest center of its kind in the country. As a result of its comprehensive services, patients from all over the nation and the world are referred to the Center at Children's Hospital.

Founded in 1855 as the nation's first pediatric hospital, The Children's Hospital of Philadelphia is ranked today as the best pediatric hospital in the nation by a comprehensive Child magazine survey. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country, ranking second in National Institutes of Health funding. In addition, its unique family-centered care and public service programs have brought the 381-bed hospital recognition as a leading advocate for children and adolescents from before birth through age 19.