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The team celebrated after the patient finished his 14-day treatment. |
Because of a mother’s deep concern for her child and a physician’s willingness to listen, a little boy may be able to postpone treatment for Type 1 diabetes – including daily needle sticks and strict food restrictions – for several years.
After a series of tests revealed that a patient was a candidate for a new treatment, pediatric endocrinologists at USA Health successfully administered its first Teplizumab infusion to the patient in Mobile, marking a milestone in the fight against Type 1 diabetes.
The treatment, completed in late July after a 14-day course, is designed to delay the onset of symptomatic stage 3 Type 1 diabetes by as much as two years.
Type 1 diabetes mellitus (T1D), formerly called juvenile diabetes, is an autoimmune disorder that leads to loss of the pancreas's ability to produce insulin, a critical hormone for the body's glucose use. Children with symptomatic T1D present with health conditions including hyperglycemia, polyuria, and polydipsia, requiring the initiation of insulin replacement therapy.
“This is the first available disease-modifying therapy available for these patients and presents as a light of hope to currently delay – but maybe one day potentially cure – T1D,” said Melissa Perez-Garcia, M.D., a pediatric endocrinologist and assistant professor of pediatrics at USA Health who worked with the patient and his family. The patient treated at USA Health is the health system’s first pediatric patient to receive the therapy.
After seeking care in other facilities in the region, the patient’s family came to see Perez-Garcia for a third opinion. The pediatric endocrinologist sat with the mother and took a detailed history, asking questions that revealed her son could have Type 1 diabetes. Tests confirmed her suspicion.
Fortunately, when detected early enough, the disease can be delayed with treatment.
In 2022, the U.S. Food and Drug Administration approved Teplizumab as the first therapy shown to delay the progression to symptomatic T1D. Delaying the onset of the disease could have a profound impact on a child’s quality of life, allowing them more years free from the burden of frequent injections and glucose monitoring.
“This is an exciting moment for our pediatric endocrine team,” said Benjamin Estrada, M.D., interim chair of pediatrics, professor of pediatrics, and assistant dean for medical education at the Frederick P. Whiddon College of Medicine at USA. “By offering this therapy, we hope to delay the need for insulin treatment and give our patient and his family more time before facing the daily challenges of Type 1 diabetes.”
After pre-treatment testing, the treatment required a daily IV infusion for 14 days, which was administered in the pediatric infusion center at USA Health Children’s & Women’s Hospital.
“We are filled with optimism that this sweet 8-year-old boy will be a responder to the medication,” Perez-Garcia said, “and won’t have to think about insulin injections or blood glucose checks for at least two more years.”