PNH treatments effective, but can create a heavy time burden for patients
Administration time should be a factor in selecting a therapy, researchers say
Written by |
While people with paroxysmal nocturnal hemoglobinuria (PNH) have access to a range of approved medications that effectively control the rare acquired disease, their administration can be time-consuming and may disrupt patients’ work, travel, daily activities, and family life, creating a large time burden.
That’s according to a new study that investigated the impact of available complement inhibitors, treatments that have been found to ease disease symptoms and substantially reduce the risks of life-threatening complications in PNH. Two mainstay complement inhibitors are given through infusion into a vein, typically lasting 30-60 minutes.
“Given the multiple treatment options available for patients with PNH, treatment decisions should be guided not only by efficacy, but also by how the treatment, including the time required to receive treatment, impacts patients,” the researchers wrote.
The study, “Time-Related Burden of Treatment With Parenterally Administered Complement Inhibitors: A Mixed Methods Observational Study Exploring Experiences among Patients With Paroxysmal Nocturnal Hemoglobinuria,” was published as a communication in the journal Health Science Reports.
The work was funded by Novartis, which markets Fabhalta (iptacopan), an oral complement inhibitor approved for PNH that’s taken as oral capsules twice per day. Four of the study’s 12 authors are employed by Novartis, and another five work for Iqvia, a data science company.
The researchers concluded that treatment-related time burden should be considered by clinicians and patients when selecting among different therapies for PNH.
In PNH, red blood cells lack surface proteins that normally signal the complement system, part of the immune system, not to attack them. Without these proteins, the complement system destroys red blood cells, a process known as hemolysis.
Complement inhibitors block elements of the complement system, preventing hemolysis and helping reduce the symptoms of PNH.
Getting treatment takes more than 3 hours each time
Among complement inhibitors, AstraZeneca’s Soliris (eculizumab) and Ultomiris (ravulizumab) are both given intravenously, or delivered directly into the patient’s vein. Soliris is usually infused every two weeks, while Ultomiris is given every eight weeks, or about every two months. Another such therapy, Empaveli (pegcetacoplan), is self-administered at home, under the skin or subcutaneously. It’s given up to three times each week, with each treatment lasting as long as one hour. This contrasts with Fabhalta, an oral therapy that may be a more convenient option for patients.
Because people with PNH have several treatment options, the research team suggested that doctors and patients should consider not only how well each treatment works but also how much time and effort are required to administer it.
To learn more, this study looked at how individuals with PNH experience the time burden of treatment. The study involved 61 adults with a mean age of 46.9. Each had been receiving either Soliris, Ultomiris, or Empaveli for at least six months.
All patients completed an online survey about the time they spent on treatment-related activities, and 25 also took part in individual interviews to describe their experiences in more detail. Slightly more than half (61%) were receiving Ultomiris, while fewer were receiving Empaveli (21%) or Soliris (18%).
Overall, it took patients a median of three hours and 15 minutes to receive each treatment. Individuals on Ultomiris spent the most time (three hours and 45 minutes), while those receiving Empaveli at home spent the least time (one hour and 15 minutes). Even so, of the eight patients on Empaveli who were interviewed, two said the time associated with the infusions was “more than they would like to spend,” the researchers wrote.
“I don’t love the infusion day, in terms of how long it takes. … It takes like [five or six hours] by the time I get there, get the blood work done … then I have to wait an hour for the order to come. …[After the infusion] they have an observation hour,” said one 57-year-old woman taking Ultomiris.
Anothger woman, age 62, taking Empaveli, said: “You have to do it twice a week. … You have to at least allow an hour … to … warm up your medication. … You have to get your supplies ready to do it and that takes another half an hour.”
Time burden also involves travel, waiting, and paperwork
Patients receiving intravenous infusions also spent additional time preparing for appointments, traveling to treatment centers, waiting for appointments, and completing paperwork.
“It becomes a very long day very quickly,” a 29-year-old woman on Ultomiris said.
For about half of the patients interviewed, treatment limited their daily lives, the data showed. These individuals noted that they had to plan work schedules, family activities, vacations, and travel around their treatment appointments.
Those on Empaveli found it easier to travel because they could bring the medication with them. Still, they faced challenges, such as keeping the medication cold and carrying supplies.
“If we go away for the weekend, we make sure that I’m back in time to do the infusion. … If we go to a hotel, we have to make sure we have a place to store the medication. … Plus you’ve got the needles, so it’s not like you can travel with needles, especially on an airplane,” said one woman taking the self-administered therapy.
Among the 35 employed patients, the median time away from work per treatment was three hours. Interviews showed that many patients had to rearrange their work schedules or use vacation time for medical appointments, making treatment disruptive even when it was effective.
The findings … add to the body of evidence indicating that the time associated with [complement inhibitors that are injected or infused] is burdensome.
Nearly half (43%) of patients said they needed help from a spouse, partner, family member, or another caregiver. Helpers spent a median of two hours assisting with treatment-related activities, such as driving patients to appointments or providing post-treatment support. Some patients felt uncomfortable relying on others and worried that their treatment placed a heavy burden on family members, the researchers noted.
“The findings … add to the body of evidence indicating that the time associated with [complement inhibitors that are injected or infused] is burdensome,” the researchers wrote. “As new treatments are developed, the findings from this study serve to support investigations into more patient-friendly treatment administration options, potentially easing the treatment burden of patients with PNH.”
Leave a comment
Fill in the required fields to post. Your email address will not be published.