“After diagnosing a rare fetal disease in a 7-year-old patient, we were faced with two challenging and distressing options. We could commit the patient to lifelong blood apheresis, a demanding process that involves repeatedly filtering harmful cholesterol from the chilld’s blood or pursuing a liver transplant, which itself comes with significant risks and potential complications,” said Dr. Narmeen AlBashrawi, Pediatric Endocrinologist and the child’s physician. “Fortunately, after the team decided to try a groundbreaking medication, this young patient has been spared from both of those burdensome treatments and can now enjoy a carefree childhood.”
The profound transformation in the child's life was achieved through a multidisciplinary collaboration between the JHAH Pediatric Endocrinology Team, Pharmacy, the Drug & Therapeutics Committee and Supply Chain, working collectively to safeguard the child’s medical, emotional and overall well-being.
The team decided to try a medication that had never been used for pediatric patients in the Kingdom outside of a trial study, and the results were astonishing. Before the medicine, the child’s cholesterol level was almost 4 -5 times above normal, and within three months, the patient’s cholesterol levels had become normal. As a result, the child can now be a child.
“This is yet another great achievement by our JHAH teams,” said Salem AlShehry, Chief Operating Officer. “It was a truly heartwarming success story. My deepest appreciation to all teams involved.”