Understanding Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and serious blood disorder. It causes the destruction of red blood cells within blood vessels, a process called intravascular hemolysis. This disorder also affects white blood cells and platelets, increasing the risk of blood clots, kidney damage, and other complications. Common symptoms of PNH include fatigue, abdominal pain, and difficulty swallowing, all of which negatively impact a patient's quality of life.
Read more about PNH here.
Ravulizumab: A Breakthrough for PNH
Ravulizumab is an FDA-approved treatment for PNH that works by targeting a specific protein in the immune system known as complement component 5 (C5). Clinical trials have demonstrated that ravulizumab helps reduce the risk of blood clots, improve survival rates, and enhance the overall quality of life for people living with PNH. However, further research is needed to understand its impact on other aspects of life, such as sleep, physical and mental health, and the ability to work.
The REVEAL Study: Tracking Quality of Life in PNH Patients
A recent observational study called the REVEAL trial, published in BMC Health and Quality of Life Outcomes, looked at how ravulizumab affects patients with PNH. The study focused on measuring fatigue, sleep, physical health, and mental health among patients receiving ravulizumab. Interestingly, the study found that PNH patients treated with ravulizumab experienced a quality of life similar to that of the general healthy US population.
The REVEAL study was conducted over 32 weeks and involved PNH patients receiving ravulizumab treatment in the US. Importantly, this study was conducted virtually, allowing participants to complete all tasks from home. Using wrist-worn Fitbitâ„¢ devices, the study collected data on participants heart rates, daily steps, and sleep duration. Additionally, participants filled out weekly surveys that assessed:
Fatigue (FACIT-Fatigue)
Physical and mental health (PROMIS Global Health)
Sleep quality (PROMIS Sleep-Related Impairment and Sleep Disturbance)
Work productivity (WPAI-SHP)
The data collected from the Fitbits and surveys were compared to values from the general US population.
Results: How PNH Patients Compared to the General Population
The study included 28 patients, with a median age of 34 years, and 54% were female. The results were encouraging: patients’ scores on quality of life indicators, such as fatigue, physical health, mental health, and sleep, were comparable to those of the healthy US population. Here’s how the PNH patients compared to the general US population in key areas:
Quality of Life Indicators | Patients treated with ravulizumab | Population Normative Value |
Fatigue (FACIT-Fatigue score) | 40.0 | 43.7 |
Physical Health (PROMIS Global score) | 51.0 | 51.6 |
Mental Health (PROMIS Global score) | 51.0 | 48.5 |
Sleep Impairment score | 50.0 | 50.0 |
Sleep Disturbance score | 49.0 | 50.0 |
Overall Work Impairment (WPAI-SHP) | 22.0 | 17.0 |
Resting heart rate | 67 BPM | 65.5 BPM |
Daily step count | 7,476 steps | 7,271 steps |
Sleep duration | 7.7 hours | 7.2 hours |
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These values were comparable to the average values seen in the general population. Additionally, the study found a positive link between physical activity (daily step count) and physical and mental health scores.
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Conclusion
This study was the first to use wearable technology, like Fitbits, to track physical activity and sleep in people living with PNH. The results show that ravulizumab enables patients to achieve activity levels and a quality of life similar to healthy individuals. Although the study sample was small and focused on patients with well-controlled PNH, the findings highlight the potential of wearable devices in future clinical trials for rare diseases like PNH.
While the REVEAL study offers encouraging insights, the small sample size and focus on patients with controlled PNH may limit its wider application. Additionally, the study faced challenges like potential selection bias and self-reporting bias. Despite these limitations, the study showcases the value of using digital monitoring tools to track health outcomes in rare disease patients.
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