Impact of High-Dose Glucocorticoid Therapy on Long-Term Health
Neurocrine Biosciences has announced the publication of a comprehensive narrative review exploring the complexities of traditional glucocorticoid (GC) treatment for classic congenital adrenal hyperplasia (CAH) and the potential advantages of introducing novel therapies that target non-glucocorticoid mechanisms.
This review, titled Glucocorticoid Therapy in Classic Congenital Adrenal Hyperplasia: Traditional and New Treatment Paradigms, was published in the esteemed journal Expert Review of Endocrinology & Metabolism and offers valuable insights for healthcare providers navigating the evolving treatment landscape of classic CAH.
Classic congenital adrenal hyperplasia (CAH) is a genetic disorder characterized by an enzyme deficiency that leads to abnormal adrenal steroid production. One of the key challenges in managing this condition is balancing the need to control excess adrenocorticotropic hormone (ACTH) and androgen levels.
Historically, this has been achieved by administering high doses of glucocorticoids, but this approach comes with its own set of risks and complications. Over time, the use of high-dose glucocorticoid therapy has been associated with various adverse effects, making it a lifelong challenge for both patients and healthcare providers to find the most effective and balanced treatment regimen.
As a result of these challenges, there has been an increasing interest in alternative therapies that could complement or reduce the need for high-dose glucocorticoid treatment. The publication in Expert Review of Endocrinology & Metabolism highlights the emerging role of non-glucocorticoid treatments,
Which can help to control ACTH and adrenal androgen levels while allowing for a reduction in glucocorticoid dosing. The review emphasizes how this approach may not only improve treatment outcomes but also reduce the long-term risks associated with chronic glucocorticoid exposure, such as cardiovascular, metabolic, and skeletal complications.
Dr. Irina Bancos, M.S., M.D., a Professor of Medicine at the Mayo Clinic and an expert in endocrinology, explained the importance of finding the right balance in managing CAH. “The treatment paradigm for CAH involves continually monitoring and balancing ACTH and androgen levels and glucocorticoid-dosing to optimize treatment,” said Dr. Bancos.
“New CAH medications that control excess ACTH and adrenal androgens through a non-glucocorticoid mechanism allow for glucocorticoid dose reduction, with the cumulative reduction over time translating into a significant decrease in the risk for complications that can occur when higher doses are used over a patient’s lifetime.”
One of the most significant concerns with traditional therapy is the long-term health risks that arise from chronic exposure to high doses of steroids. High-dose glucocorticoid therapy can lead to a range of serious side effects, including cardiovascular disease, metabolic disorders such as diabetes, bone density loss, and skeletal complications.
Additionally, the mental health of patients can be impacted, and their quality of life can suffer as a result of the side effects associated with long-term glucocorticoid use. Dr. Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences, addressed these concerns, noting, “Chronic exposure to high-dose treatment can result in severe cardiovascular, metabolic and skeletal complications, as well as negatively impact the mental health and quality of life of patients.”
Emerging Therapies: Non-Glucocorticoid Mechanisms for Managing CAH
Dr. Roberts also highlighted the importance of reducing doses over time to mitigate these risks. “Over a lifetime, even modest reductions in daily doses can reduce the risk of these complications and lessen the overall burden of exposure,” she said. This statement underscores the potential benefit of exploring alternative therapies that can help reduce reliance on glucocorticoids and enhance the overall well-being of individuals living with classic CAH.
For many years, the treatment options for CAH have been limited, and managing the condition has largely depended on high-dose glucocorticoid therapy. This has presented a significant unmet need in the medical community, as the risks associated with chronic glucocorticoid use continue to raise concerns. As such, there has been increasing demand for innovative treatments that can better address the underlying causes of CAH while minimizing the adverse effects associated with long-term glucocorticoid use.
In recent developments, the U.S. Food and Drug Administration (FDA) approved CRENESSITY™ (crinecerfont), a new adjunctive treatment for adults and pediatric patients aged four years and older with classic CAH. CRENESSITY is a potent and selective oral corticotropin-releasing factor type 1 receptor (CRF1) antagonist. This therapy directly reduces excess ACTH levels and, as a result, helps to decrease the production of adrenal androgens. By targeting the root cause of the condition, CRENESSITY offers a novel approach to managing classic CAH while reducing the need for high-dose glucocorticoid therapy.
The approval of CRENESSITY marks a significant milestone in the treatment of classic CAH, offering a new option for patients that could improve the overall management of the disease. By reducing the reliance on glucocorticoids, CRENESSITY has the potential to lower the risks of side effects associated with steroid use, offering patients a more balanced and physiologic treatment option. This approval highlights the growing interest in non-glucocorticoid therapies for CAH and represents a promising advancement in the field of endocrinology.
The introduction of non-glucocorticoid therapies such as CRENESSITY has sparked optimism among healthcare providers and patients alike. These new treatments have the potential to revolutionize the management of classic CAH by addressing the underlying hormonal imbalances without relying on high doses of glucocorticoids. By offering a more targeted approach to treatment, these therapies not only improve clinical outcomes but also reduce the overall burden of disease management for patients.
For patients living with classic CAH, the approval of CRENESSITY and the ongoing development of additional therapies offer hope for a better quality of life. The ability to reduce glucocorticoid doses while still effectively managing the symptoms of CAH can lead to improved health outcomes and a reduction in long-term complications. Moreover, these advancements provide a more patient-centered approach to care, focusing on minimizing side effects and promoting overall well-being.
As the field of endocrinology continues to evolve, the need for novel therapies to manage conditions like classic CAH is becoming increasingly evident. The publication of this review by Neurocrine Biosciences, combined with the FDA approval of CRENESSITY, underscores the progress being made in developing more effective and safer treatments for rare and complex endocrine disorders. Healthcare providers now have more options than ever before to optimize the treatment of CAH and improve the lives of patients affected by this challenging condition.
In conclusion, the publication of this narrative review and the approval of CRENESSITY are pivotal moments in the ongoing effort to improve the management of classic CAH. These developments highlight the importance of reducing glucocorticoid exposure and the potential benefits of introducing new non-glucocorticoid therapies to the treatment landscape.
As more innovative treatments become available, patients with classic CAH can look forward to better health outcomes and a more balanced, less burdensome approach to managing their condition. Neurocrine Biosciences continues to lead the way in advancing treatments that improve the lives of patients, offering hope for a brighter future in the management of congenital adrenal hyperplasia.
