Revolutionary 'Pulsatility' Therapy Offers Hope for Adrenal Insufficiency Patients

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A new hormone replacement therapy has shown promise in treating patients with adrenal conditions. The therapy, called ‘Pulsatility’, is a first-of-its-kind treatment that more closely replicates the natural circadian and ultradian rhythms of our hormones. The University of Bristol-led clinical trial, published in the Journal of Internal Medicine, revealed that the therapy improved symptoms in patients with adrenal insufficiency conditions. Low levels of cortisol, a key hormone that regulates a range of vital processes, can trigger symptoms of debilitating fatigue, nausea, muscle weakness, dangerously low blood pressure, and depression. Although existing oral hormone replacement treatment can restore cortisol levels, it is still associated with an impaired quality of life for patients. Scientists believe this is because the current treatment does not mimic the body’s normal physiological timing, missing cortisol’s anticipatory rise and lacking its underlying ultradian and circadian rhythms.

The new ‘Pulsatility’ therapy is designed to deliver standard hydrocortisone replacement to patients via a pump that replicates more closely cortisol’s natural rhythmic secretion pattern. The pulsatile subcutaneous pump has now revealed promising results in its first clinical trial. Twenty participants aged 18 to 64 years with adrenal insufficiency conditions were assessed during the double-blinded PULSES six-week trial and treated with usual dose hydrocortisone replacement therapy administered either via the pump or the standard three times daily oral treatment. While only psychological and metabolic symptoms were assessed during the trial, results revealed the pump therapy decreased fatigue by approximately 10%, improved mood, and increased patient energy levels by 30% first thing in the morning — a key time frame when many patients struggle. Patient MRI scans also revealed alteration in the way that the brain processes emotional information.

The study was funded by the Medical Research Council and Bristol & Weston Hospitals Charity (formerly Above & Beyond). The team now hopes to conduct a larger trial looking at personalization of steroid replacement therapy using hormone secretion patterns from over 200 healthy volunteers. The new therapy offers greater hope for the thousands of people living with hormone insufficiency conditions, and it is one of the first major advances in adrenal insufficiency treatment to date.

Adrenal Insufficiency: An Overview

Introduction

Adrenal insufficiency is a condition characterized by inadequate production of hormones by the adrenal glands. These small glands located atop each kidney are responsible for producing vital hormones, including cortisol, aldosterone, and adrenaline. A deficiency in these hormones can have significant physiological impacts, affecting metabolic processes, blood pressure regulation, and immune system function, among other bodily systems.

Etiology and Classification

Adrenal insufficiency is broadly classified into primary, secondary, and tertiary types, each with distinct etiological factors:

Primary Adrenal Insufficiency (Addison’s Disease)

This form is caused by a dysfunction within the adrenal glands themselves. Autoimmune disorders are the most common etiological factor, but it can also be due to infections like tuberculosis, congenital disorders, or adrenal hemorrhage.

Secondary Adrenal Insufficiency

This form arises due to inadequate secretion of adrenocorticotropic hormone (ACTH) from the pituitary gland. Causes may include pituitary tumors, surgical removal of pituitary tissue, or certain medications like glucocorticoids that suppress ACTH production.

Tertiary Adrenal Insufficiency

This form is due to a deficiency in corticotropin-releasing hormone (CRH) from the hypothalamus. Chronic use of exogenous steroids, head injuries, or tumors affecting the hypothalamus are common causes.

Clinical Manifestations

Symptoms of adrenal insufficiency often manifest gradually and may include fatigue, weakness, weight loss, hypotension, and hyperpigmentation of the skin. Acute adrenal crisis, a severe manifestation of the condition, is a medical emergency and may present with vomiting, diarrhea, hypovolemic shock, and altered mental status.

Diagnosis and Investigation

Diagnostic investigations generally include blood tests to measure cortisol, ACTH, and aldosterone levels. Additional tests like the ACTH stimulation test can also be conducted. Imaging studies such as CT scans or MRIs may be useful in identifying structural abnormalities in the adrenal glands or pituitary.

Treatment and Management

Treatment aims to replace deficient hormones. Hydrocortisone or prednisone may be used to replace cortisol, while fludrocortisone is often used for aldosterone replacement. In cases of acute adrenal crisis, immediate intravenous administration of hydrocortisone along with fluids and electrolytes is essential.

Epidemiology and Prevalence

The prevalence of Addison’s disease is estimated to range between 100 and 140 cases per million population (Erlich, 2020). Secondary adrenal insufficiency is more common but precise epidemiological data are limited.

Future Directions

Research is ongoing to improve diagnostic accuracy, treatment options, and the understanding of long-term outcomes for adrenal insufficiency. Advances in genetic research may also provide insights into novel therapeutic targets.

Case Study: The Impact of Crono P Pump on Addison’s Disease—A Patient’s Perspective

Introduction

The case study presented herein examines the experiences of Joe Miles, a 40-year-old male diagnosed with Addison’s Disease. This case aims to shed light on the transformational effects of a novel therapeutic approach, the Crono P pump, in managing Addison’s disease. Mr. Miles participated in the PULSES study, which evaluated the efficacy of the Crono P pump as compared to traditional oral tablets. The patient narrative serves as a valuable data point in understanding the impact of this new therapy on the quality of life for individuals with adrenal insufficiency.

Background: Adrenal Insufficiency and Addison’s Disease

Addison’s disease is a specific form of primary adrenal insufficiency. In the UK, approximately 8,500 individuals suffer from primary adrenal insufficiency due to adrenal gland malfunction, while around 10,000 have secondary adrenal insufficiency, a pituitary gland problem. Tertiary adrenal insufficiency, though its prevalence is unknown, arises due to long-term steroid use (Medical Research Council, UK).

The Case of Joe Miles: Clinical History and Diagnosis

Joe began experiencing unexplained exhaustion approximately 14 years ago. This exhaustion was so extreme it led to blackouts and significant weight loss. His initial consultation with a General Practitioner (GP) did not result in a concrete diagnosis, leading him to seek a second opinion. The second GP identified Joe’s condition as Addison’s disease and emphasized its severity, even driving him to the emergency department for immediate intervention.

Introduction to Crono P Pump via PULSES Study

Joe had managed his Addison’s disease using oral tablets for nearly eight years before enrolling in the PULSES study, where he was introduced to the Crono P pump. He reported a sudden surge in energy levels, marking a significant improvement in his quality of life. After the study concluded, Joe made concerted efforts to obtain the Crono P pump through private prescription, making him the first person in the UK to use the device outside of a clinical trial.

Patient Advocacy and Peer Testimonies

Given the transformative impact of the Crono P pump on his life, Joe advocated for the device and introduced it to others with Addison’s disease. According to these individuals, the pump was also “life-changing,” facilitating a transition from a state of chronic illness to markedly improved well-being.

Discussion: The Role of Charitable Organizations and Medical Research

Institutions like the Medical Research Council and Bristol & Weston Hospitals Charity play a pivotal role in advancing medical research and enhancing patient care. Their support often extends to funding innovative treatments and technologies, potentially transforming the landscape of healthcare for chronic conditions like adrenal insufficiency.

Conclusion: Implications for Future Research and Treatment

The case of Joe Miles serves as a compelling testimony to the potential of the Crono P pump in enhancing the lives of individuals with Addison’s disease. While this single case cannot establish the efficacy of the device definitively, it underscores the importance of further research to validate its effectiveness on a broader scale. Such validation could lead to the inclusion of Crono P pumps in standard treatment guidelines, thus serving a larger population of individuals with adrenal insufficiency.

References

Erlich, D. R. (2020). Prevalence and incidence of adrenal insufficiency. Endocrinology and Metabolism, 35(2), 217-225.
Medical Research Council, UK. “Adrenal Insufficiency Statistics.”
Bristol & Weston Hospitals Charity. “Investing in Medical Research and Patient Care.”
Image Credit: Pixabay

Note: Please consult healthcare professionals for accurate diagnosis and treatment of adrenal insufficiency. This information is intended for educational purposes.