Rare Disease Report

Explaining the Challenges of Pediatric Ulcerative Colitis

DECEMBER 21, 2017
Paul P. Doghramji, M.D.
Paul P. Doghramji, M.D., is cofounder of Brookside Family Practice & Pediatrics, a current affiliate of Pottstown Medical Specialists, in Pottstown, PA. He has also been Attending Physician in Family Practice, Chair of the Utilization Management Committee, and Physician Advisor at Pottstown Memorial Medical Center.

With 3 potential therapies (cobitolimod; INN-108; SER-287) for pediatric ulcerative colitis receiving Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) since August, Rare Disease Report requested to sit down with Doghramji to discuss the outlook for this patient population.

Doghramji: The whole field of medicine changes as decades go by, whether it’s environmental or whether it’s genetic, things occur. We know that a lot of diseases that are going on now didn’t occur decades ago and vice versa. As far as ulcerative colitis, it’s difficult to know why we’re finding more of it, either in the pediatric or adult populations. Whether it has to do with the microbiomes in the intestines, we’re not really sure. What a clinician needs to know – in primary care, especially – is when children come in, or even young adults in their teens come in, with gastrointestinal symptoms, it’s really important to rule out some of the critical and some of the more difficult problems that need to be diagnosed early and treated early. Ulcerative colitis is one of them. Children come in with severe abdominal pain that’s unrelenting, with weight loss, or maybe even with bloody bowel movements, they need to be worked on thoroughly. A good pediatric gastroenterologist is very important to be referred to very quickly for them.

Right now, from what I know about some of the orphan drugs in the pediatric population for GI diseases, there are some very promising ones coming out, primarily in the area of ulcerative colitis. It’s not that common of a disease in the pediatric population, but from what I see, there are some very interesting ones. When it does occur in a pediatric patient, it can be devastating. It can be really difficult, and result in surgeries, etc. Identifying those patients early, and getting them the right medication and treatment plan is important; some of the newer medications seem very promising.


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