Debunking Misconceptions and Understanding the Stages of Type 1 Diabetes

In a Pharmacy Times^® interview, Jennifer Goldman, PharmD, CDCES, BC-ADM, FCCP, professor of pharmacy practice, clinical pharmacist, Massachusetts College of Pharmacy and Health Sciences, highlights teplizumab (Tzield; Sanofi), the first FDA-approved therapy to delay type 1 diabetes progression. It targets stage 2 type 1 diabetes, modulating T cells to delay the onset of stage 3 by 2 to 3 years, and in some patients, up to 5 to 7 years. Goldman emphasizes the importance of early identification and screening, noting that approximately 59% of type 1 diabetes cases occur in patients aged 20 to 40 years. Pharmacists play a crucial role in patient education, infusion coordination, and long-term follow-up, with the goal of preventing crisis situations and improving disease management.

Pharmacy Times: Can you introduce yourself and provide an overview on the different stages of diabetes?

Jennifer Goldman, PharmD, CDCES, BC-ADM, FCCP: My name is Jennifer Goldman. I've been practicing for 35 years, and I'm professor of pharmacy practice at the Massachusetts College of Pharmacy in Boston, and a clinical pharmacist and director of the cardiometabolic services at Well Life Medical in Peabody, Massachusetts. I am so excited to talk about teplizumab (Tzield; Sanofi). It's the first and only FDA-approved disease-modifying therapy that can delay the onset of stage 3 type 1 diabetes in people aged 8 [years] and over who are in stage 2.

To understand where [teplizumab] fits in, it's actually helpful to explain the 3 stages of type 1 diabetes, because this is something most of us did not learn in school. So stage 1, this is when the immune system has started attacking the pancreas, and 2 or more islet autoantibodies are present, but blood sugar is still normal and patients do not have symptoms—so, asymptomatic. Stage 2, there's still no outward symptoms, but the glucose tolerance is now impaired, so the autoimmune attack continues, and beta cell destruction is accelerating... Stage 3 is the clinical diagnosis stage, this is what we learned in school. This is where blood sugars are consistently high, symptoms like thirst, frequent urination, weight loss, that's when it becomes evident, and many individuals are diagnosed in stage 3 during an emergency room visit, often with diabetic ketoacidosis (DKA).

[Teplizumab] is given in stage 2 as a 14-day infusion, where it works by modulating T cells to delay the progression to stage 3 by an average of 2 to 3 years, with some cases showing delay of 5 to 7 years. That gives individuals more time before requiring insulin [and] it reduces the risk of DKA at that diagnosis. So, as pharmacists, we are uniquely positioned across community, ambulatory care, infusion centers, [and] hospitals really to support early identification, patient education, infusion coordination, and long-term follow-up.

Pharmacy Times: Who is teplizumab approved for, and at what ages are people most commonly diagnosed with type 1 diabetes?

Goldman: [Teplizumab] is FDA-approved for people 8 years and older with stage 2 type 1 diabetes, meaning they have 2 or more islet autoantibodies and impaired glucose tolerance, but they have not developed symptoms or received a formal diagnosis of diabetes. It is a myth that most type 1 cases are diagnosed in childhood, while the peak clinical onset is aged 10 to 14 years old. In one very large longitudinal study—that actually had over 30,000 people—59% were over the age of 20, typically between the ages of 20 and 40, but many are even diagnosed later in life. And that's why both pediatric and adult screenings are vital, especially for people with a family history of autoimmune conditions. I do think it's important for people to understand that about 40% of adults with type 1 diabetes are initially misdiagnosed, [of which] 75% of those are misdiagnosed with type 2 diabetes. That leads to poor disease management from diagnosis. Pharmacists can help identify at-risk individuals and refer them for screening early when they're 10 or 35, it doesn't matter what age they are, refer them for screening.

Pharmacy Times: Why is delaying stage 3 type 1 diabetes with teplizumab considered so clinically meaningful?

Goldman: Delaying stage 3 allows patients and families time. Time to mentally and physically prepare, time to avoid emergency diagnoses—such as DKA—and to reduce the long-term complications. It also preserves beta cell functions, so endogenous insulin production, which translates to less glycemic variability, reducing insulin needs, and a longer "honeymoon phase" without an immediate need for insulin. So, for many, it's the difference between crisis and control. Pharmacists play a vital role in making this opportunity accessible and meaningful, essentially buying time for that patient—time to prepare [and] time to become educated. And, what if you buy time for another new, great breakthrough?

Pharmacy Times: Any final remarks?

Goldman: Up to 62% of patients that are initially diagnosed with type 1 diabetes without prior screening have DKA at the time of that type 1 diagnosis. DKA can have long-term negative effects beside increased morbidity and mortality. These patients have poor neurocognitive outcomes, increased risk for cardiomyopathy—[notably] in young patients—psychological distress, and a sustained negative effect on glycemic control over time. So, screening and monitoring of these presymptomatic type 1 diabetes patients can help reduce DKA rates at stage 3 type 1 diabetes onset at 93.5%. The American Diabetes Association, the International Society for Pediatric and Adolescent Diabetes, and Breakthrough [T1D], formerly the [Juvenile Diabetes Research Foundation], recommends screening first-degree relatives of individuals with type 1 diabetes and individuals with personal or family history of selected autoimmune diseases.

Pharmacists have a central role at every step from identifying high-risk individuals, initiating screening, educating families—which can help prevent DKA from being that event that leads to that diagnosis—and they can help with coordinating infusions, and ensuring long-term monitoring. Teplizumab has opened the door to proactive disease prevention [and] proactive diabetes care, so pharmacists are essential in walking patients through that door.