Can Surgery Cure Congenital Hyperinsulinism?

In the recent review, it was shown that surgery can cure nearly all patients with the focal or localized form of congenital hyperinsulinism and prevent complications in patients with the diffuse from in which insulin-producing cells occur throughout the pancreas.

In the recent review conducted by N. Scott Adzick MD, Surgeon-in-Chief at Children’s Hospital of Philadelphia (CHOP), et al., it was shown that surgery can cure nearly all patients with the focal or localized form of congenital hyperinsulinism and prevent complications in patients with the diffuse from in which insulin-producing cells occur throughout the pancreas.

The review, “Surgical Treatment of Congenital Hyperinsulinism: Results from 495 Pancreatectomies in Neonates and Children,” represents the world’s largest single-center case series of patients with congenital hyperinsulinism. Results were presented at the 2018 American Pediatric Surgical Association Annual Meeting.

Congenital hyperinsulinism is a rare genetic disease in which inherited mutations halt the insulin-secreting beta cells, causing insulin levels to become excessive. The excess insulin levels cause recurrent hypoglycemia and can cause irreversible brain damage or death if not treated properly. Essentially, congenital hyperinsulinism is the opposite of diabetes.

The review included approximately 500 cases of infants with severe congenital hyperinsulinism who underwent partial or near-total removal of their pancreas for persistent hypoglycemia at CHOP. Forty-seven percent of patents aged between 10 and 20 years with surgically treated congenital hyperinsulinism have diabetes, and all those who developed the disease experienced near-total pancreatectomies to treat their congenital hyperinsulinism.

Adzick commented on the review’s positive findings as well as the potential for diffuse hyperinsulinism treatments in a press release. "Our team's approach is able to distinguish focal from diffuse HI, localize focal lesions, and cure almost all focal patients with a partial pancreatectomy, and while we cannot cure diffuse HI, we can help prevent brain damage and severe hypoglycemia commonly associated with the disease. When all is said and done, the children treated with our approach to focal HI have been able to lead normal lives and not worry about the effects of this rare condition.”

Other aspects of the disease are also being studied for several reasons, including the potential to provide better treatment options for patients with diffuse congenital hyperinsulinism, as well as the underlying genetic causes of the disease. Additionally, uses of modern technology for monitoring and treating hypo- and hyperglycemia in children and adults with hyperinsulinism are being investigated.

Co-author of the paper and pediatric endocrinologist and Director of CHOP's Congenital Hyperinsulinism Center, Diva De León-Crutchlow, MD, shared his sights on forward-looking therapies for patients with diffuse hyperinsulinism. "We are actively pursuing clinical trials for new therapies that may improve the quality of life for patients with diffuse HI, including an investigational drug called exendin-(9-39) that can increase fasting blood glucose in these patients. The therapy has been shown to be effective in older patients and we are currently studying its effects in younger patients."

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