Sidra Medicine pioneers groundbreaking treatment protocol for infant with congenital hyperinsulinism

DOHA: Sidra Medicine announced that it has pioneered a new treatment protocol for a young infant with congenital hyperinsulinism the first-of-its-kind milestone globally, and the findings have been published in the prestigious New England Journal of Medicine1.

Division Chief of Endocrinology at Sidra Medicine Prof. Khalid Hussain said the case refers to a three-month-old infant from Kuwait, whose condition was deteriorating because of congenital hyperinsulinism as she was experiencing severe hypoglycemia (low blood sugar).

He highlighted that although in some mild cases, the condition can be managed with medication, however for most children hypoglycemia cannot be controlled with conventional medications. In such cases, he said, surgery to remove either part or all of their pancreas (pancreatectomy) remains the only option. This is a major procedure, and the children then must live with lifelong diabetes mellitus and pancreatic exocrine insufficiency.

To manage her low blood sugar, the patient was first put on conventional medical therapies and medications as well as a combination of intravenous dextrose therapy, and continuous gastrostomy-tube feeding to maintain acceptable blood glucose levels. Since the infant wasn't responding to the conventional medicines, she was at increased risk of having to undergo a pancreatectomy, Prof. Hussain pointed out, adding that the Sidra Medicine team were keen to try other methods and decided to trial a new treatment protocol using 'Alpelisib' drug.

It should be noted that 'Alpelisib' in an infant with congenital hyperinsulinism is the first of its kind. It was trialled, following previous case reports where adult patients with breast cancer, being treated with the same drug, were shown to have experienced high blood glucose profiles. It is a protein-targeted medication that is used for treating breast cancer and overgrowth disorders in children.

The medical team avoided performing a pancreatectomy that would drastically impact the young girl's future growth and lifestyle. The strategy applied was to take an existing medication like 'Alpelisib', which is used to treat breast cancer and overgrowth disorders and repurpose its adverse effects of hyperglycemia - to address hypoglycemia i.e. low blood sugar levels, he said.

The young patient was put on the new treatment protocol and within six weeks, started to show a vast improvement in her blood sugar levels. She also continued maintaining a healthy weight and length without impacting her growth. She was discharged with minimal low blood sugar levels with the management of her condition requiring three-hourly oral feeds and two daily doses of the medication.

For her part, the CEO of Sidra Medicine DrIyabo Tinubu-Karch said this was the first time in the world, a medication had been re-purposed for treating severe low blood sugar levels in an infant.