Patients cite aHUS treatment effectiveness, safety as top priorities
Survey shows patients prefer oral therapies, less frequent dosing
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People with atypical hemolytic uremic syndrome (aHUS) consider treatment effectiveness and safety as top priorities when choosing medications, an international survey showed.
Most patients also said they would prefer oral therapies and less frequent dosing.
The findings, based on responses from 122 patients, revealed a substantial burden from treatment-related appointments and concerns about disease recurrence, particularly among women considering pregnancy. The researchers said this highlights the importance of involving patients in treatment decisions.
“The survey confirms that patients with aHUS desire to be involved in therapeutic decision and voice clear preferences on drug choice,” the researchers wrote.
The study, “Atypical hemolytic uremic syndrome patients’ perspective in the era of complement inhibitors,” was published in Kidney International Reports.
Infusion visits, costs add to treatment burden
aHUS is caused by excessive activation of the complement system, a part of the immune system. The development of complement inhibitors, including Soliris (eculizumab) and Ultomiris (ravulizumab), has substantially improved outcomes for people with aHUS.
However, both treatments are administered through intravenous (into-the-vein) infusions, which can require repeated hospital visits. Their high costs and the burden of long-term treatment also remain important challenges.
To better understand how people with aHUS perceive their disease and its impact on daily life, researchers conducted an international online survey, based on input from patients, healthcare professionals, and a psychologist. The questionnaire covered treatment preferences, genetic testing, pregnancy, quality of life, and psychological well-being,
Some 55% of the 122 patients who completed the survey had adult-onset aHUS, and 24% developed the disease during childhood. Their mean age at the time of the survey was 30, and 67% were women. Genetic variants were identified in 67% of participants, more commonly in the CFH and C3 genes.
More than half had high blood pressure and required dialysis — a procedure that involves filtering waste from the blood when the kidneys cannot function properly — although both were more prevalent in people with adult-onset aHUS. Overall, 23% underwent kidney transplantation.
Most respondents with available treatment information had received or were currently receiving Soliris (85%), and 47% took Ultomiris. About one-third of respondents had at least one aHUS relapse, and 21% experienced a relapse after kidney transplantation.
From the patient’s perspective, the factors that mattered most when choosing treatment were efficacy (mean score of 90 out of 100), safety (88), and access to medication (80). Side effects, dosing frequency, number of medical visits, and treatment costs, received intermediate scores (61 to 65).
The route of administration scored lowest in importance, with a mean value of 55. However, 76% of the responders preferred oral medications. Most (63%) also favored long-acting treatments, administered every six to eight weeks, compared with daily medication (24%) and treatment every one to three weeks (13%).
Participants also expressed greater interest in switching from shorter- to longer-acting complement inhibitors and from intravenous to oral treatment, and a large proportion (88%) would like to receive treatment at home. Yet only 30% felt well informed about drug development.
Although most participants felt informed about the risk of meningitis associated with complement inhibitors, concerns about infection remained relevant. About 69% reported receiving preventive antibiotics, and 15% experienced infections.
More than 60% of respondents reported attending medical appointments or receiving treatment more than once a month, with mean treatment and travel-to-treatment times of eight and five hours monthly. About one-third of the participants discontinued treatment, but they did not identify a dominant factor influencing this decision.
Scores for satisfaction with mental health and coping with the disease were generally higher, while concerns about worsening disease, work-related stress, fear of dying, and social isolation received intermediate scores.
Among women who answered the pregnancy-related questions, 67% expressed concern about future pregnancies, while 57% reported refraining from further pregnancies. Most (92%) associated pregnancy with a high risk of aHUS recurrence.
The researchers said the findings provide insight into what matters most to people living with aHUS and how emerging treatments might better address their needs.
“Disease burden is high and efforts to reduce medical time with innovative approaches such as home perfusions or development of oral drugs are crucial,” they wrote.
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