Money, logistics add to treatment burden for PNH patients in Japan
New study suggests use of more at-home or less time-consuming therapies
In interviews, people in Japan with paroxysmal nocturnal hemoglobinuria (PNH) reported financial and logistical concerns — primarily, losing time from work and spending hours waiting for examinations and infusions — that increased the treatment burden they face in living with PNH.
Patients also shared with researchers their worries about the potential impacts of their disease on others, particularly family members.
In their resulting study, the investigators noted that, besides the direct expense of PNH treatment, participants worried about costs related to transportation to hospitals and loss of income. Time spent in transit and waiting for treatment also added to the logistical burdens of the disease.
“Our study indicates that persistent burdens associated with current PNH care should be taken into account alongside therapeutic effectiveness when making treatment decisions,” the researchers wrote. “As treatment options continue to expand, healthcare providers can better address both PNH management and the impact of treatment on patients’ daily lives.”
The study, “Treatment burden in patients with paroxysmal nocturnal hemoglobinuria: an in-depth interview survey,” was published in the journal Annals of Hematology.
PNH is an acquired disease that’s marked by blood cell destruction, driven by the excessive activity of part of the immune system called the complement system.
C5 inhibitors are a mainstay PNH treatment to reduce blood cell destruction. Such therapies block complement activation by targeting a complement protein called C5.
Among approved treatments are Soliris (eculizumab) and Ultomiris (ravulizumab-cwvz). Both are administered via intravenous, or into-the-vein, infusions, typically in a hospital setting.
Fabhalta (iptacopan) is another PNH treatment that’s approved in Japan, and is an oral medication. However, the researchers noted that “as of 2024, when this interview study was conducted, home administration of C5 inhibitors was not common, and patients receiving these medications needed to visit medical institutions regularly.”
More than half of PNH patients surveyed took Ultomiris
Choosing between Soliris, Ultomiris, or other therapies, including Fabhalta, depends on a variety of factors, according to the researchers.
“To provide the best possible care for each patient, treaters need to understand PNH care from the patient perspective, in addition to therapeutic effects,” the research team wrote.
To learn more about what patients might be thinking or feeling — particularly regarding disease burden and factors impacting treatment preference — the team interviewed 30 people with PNH.
The patients had a mean age of 51.6, and about two-thirds were employed. Slightly more than half (56.7%) were taking Ultomiris, while 13.3% were on Soliris. The remainder were on another therapy or didn’t know.
Among primary concerns, 80% of the participants expressed worry about causing inconveniences to others; 86.7% felt like a burden on others.
“Since I discovered I had this illness, I have had to make my family wear masks at home, and our opportunities for travel and going out have decreased,” a male participant in his 60s told the researchers. “I feel guilty for having to make them accommodate me. If I didn’t have this illness, I would have been able to take my grandchildren to various places and do more activities.”
Concerns about work, study, and social limitations also were common. Both symptom flare-ups and scheduled treatments meant many participants had to take time off work or school, or needed time to rest. These factors also posed limitations to their personal and social activities.
“When symptoms appear or I catch a cold, the symptoms last for about a week,” said one male patient in his 40s. “During that time, I have to take time off from work. If the severe symptoms persist, I find it difficult to continue working.”
One woman in her 50s with PNH told investigators: “I feel burdened by having to schedule everything around my treatment dates.”
Financial burden reported for about 75% of those on treatment
PNH treatment resulted in a variety of financial burdens and worries for about three-quarters of the study participants. Among the patients were some who received government assistance to pay for their therapy. Some worried about how they would afford treatment if their benefits went away or they could no longer work. Several noted they needed to take time off work to go to the hospital and receive infusions, losing income.
In general, the time needed for treatment was a major concern for participants.
“It takes time to commute and be at the hospital,” said one man in his 30s. “There’s waiting time for the examination, accounting, tests, and of course, the infusion time. All in all, it consumes almost an entire day, which ultimately forces me to take time off work.”
Among participants who listed hospital waiting times as a concern, the median time spent at the hospital was four hours, the study showed.
Concerns were similar in a subset of participants receiving C5 inhibitors. Individuals who had concerns about waiting time spent a median of 2.8 hours at the hospital if they were on Soliris and four hours if they were on Ultomiris. Although all of these participants were on a government assistance program to subsidize treatment, 81% expressed financial concerns.
It takes time to commute and be at the hospital. … There’s waiting time for the examination, accounting, tests, and of course, the infusion time. All in all, it consumes almost an entire day, which ultimately forces me to take time off work.
Together, these findings suggest a number of unmet needs for people receiving PNH treatment in Japan that adds to their overall disease burden.
According to the researchers, “these unmet needs could be addressed by offering treatment options that allow for home administration or are less time-consuming in other ways.” Having such options “could reduce burdens related to patients needing to take off work or travel to receive treatment,” the team added.
The detailed, interview-based methodology was a strength of the study, according to the team. However, the researchers noted the relatively small number of participants in the study.
“Further analysis with a larger sample size is required to confirm these results,” the team wrote.
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