Ultomiris use leads to life quality similar to US general population
PNH patients on approved therapy achieve same daily activity levels: Study
Treatment with Ultomiris (ravulizumab) enabled paroxysmal nocturnal hemoglobinuria (PNH) patients to achieve the same level of physical and mental health, daily activity, and sleep duration displayed by healthy individuals from the U.S. general population, according to the findings of an observational study.
In the study, people with PNH completed weekly online questionnaires over the course of about seven months and also were tracked with a digital wearable device. According to the researchers, this was the first study in PNH to make use of digital monitoring technology to collect data on physical activity and sleep among patients with the rare disease.
The data were then compared with those from healthy individuals in the U.S.
“The findings indicate that [Ultomiris] treatment enables patients with PNH to achieve activity levels (heart rate, sleep duration, step count) and quality of life that are comparable to those of the US general population,” the researchers wrote.
In clinical trials, Ultomiris has been shown to improve survival and quality of life in people with PNH. However, its impact on “sleep, physical and mental health, and ability to work needs further research,” the team wrote.
“Using a digital activity-tracking device to monitor patient activity continuously enabled the evaluation of PNH symptoms in a novel, objective way and limited any potential perception bias that may be associated with survey data,” the researchers wrote.
The study, “Patient-reported outcomes and daily activity assessed with a digital wearable device in patients with paroxysmal nocturnal hemoglobinuria treated with ravulizumab: REVEAL, a prospective, observational study,” was published in the journal Health and Quality of Life Outcomes.
Patients’ heart rate, step count tracked with FitBit wearable device
PNH is marked by red blood cell destruction, or hemolysis, driven by the abnormal activation of the complement cascade, a part of the immune system. Patients often have low red blood cell levels, or anemia, and many develop blood clots. Additional common symptoms include fatigue and weakness as well as poor sleep quality.
The mainstay treatment for PNH are medications that block complement activation, such as Ultomiris. Clinical trials have demonstrated that Ultomiris can reduce the risk of blood clot formation and fatigue, enhance patients’ survival, and exert a positive impact on quality of life.
However, according to the researchers, “information is limited on how it impacts sleep and physical activity.”
To learn more, the researchers analyzed data from PNH patients enrolled in a U.S. observational study called REVEAL. The research was funded by Alexion, Astrazeneca Rare Disease, the company that markets Ultomiris.
The patients were followed for 32 weeks, or about seven months, while on treatment with Ultomiris or Soliris (eculizumab). Soliris is another complement inhibitor therapy approved for PNH and also marketed by Alexion.
The participants were asked to complete a series of online questionnaires on a weekly basis throughout treatment to assess their fatigue, sleep, and physical and mental health, as well as their ability to work. During this period, they also wore a wrist Fitbit wearable device to track their resting heart rate, daily step count, and sleep duration.
“This was the first study to use digital monitoring technology to collect data on physical activity and sleep in patients with PNH,” the researchers wrote.
The results of the questionnaires and the Fitbit data were then compared with those from healthy individuals of the U.S. general population reported in the literature.
Ultomiris-treated patients show better physical, mental health
Altogether, the researchers analyzed data from 28 people with PNH, who had a median age of 34. All were treated with Ultomiris on an eight-week infusion schedule.
At the end of the 32-week treatment period, 82% of the surveys were completed and returned by patients. The scores of each of the surveys — assessing fatigue, physical and mental health, sleep-related impairments and disturbances, and overall work impairment — were similar to those of the U.S. general population.
Fitbit data also showed similar results across the different parameters analyzed. Indeed, the mean resting heart rate (67 vs. 65.5 beats per minute), mean daily step count (7,476 vs. 7,271 steps), and mean sleep duration ( 7.7 vs. 7.2 hours) were all similar when comparing values from PNH patients to normative values.
The researchers then assessed how the activity data recorded with the Fitbit correlated with patient-reported outcomes in the different surveys. A higher number of daily steps showed a weak, but positive, correlation with the PROMIS Global Physical Health survey and the PROMIS Global Mental Health scores. In both surveys, higher scores indicate better health.
The findings from REVEAL indicate that patients with PNH treated with [Ultomiris] reached quality of life scores (tiredness, sleep, and physical and mental health) that were within the range of the healthy US general population.
A weak, positive correlation was also seen between a lower resting heart rate and the PROMIS Global Physical Health score. Meanwhile, longer sleep duration showed a weak positive correlation with the Work Productivity and Activity Impairment Questionnaire – Specific Health Problem (WPAI-SHP). Here, higher scores indicate greater impairment and less productivity.
“The findings from REVEAL indicate that patients with PNH treated with [Ultomiris] reached quality of life scores (tiredness, sleep, and physical and mental health) that were within the range of the healthy US general population,” the researchers wrote.
Importantly, the team added that “the study also supports the use of wearable devices for the collection of activity data in clinical trials for such patients.”
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