An electronically gathered patient reported outcomes (ePRO) system customized for paroxysmal nocturnal hemoglobinuria (PNH) and aplastic anemia (AA) and may be a feasible data collection and patient guidance solution in light of high adherence, satisfaction, and performance, according to an article published in the journal Annals of Hematology.

For the purpose of this single-center study, the researchers recruited 9 patients with PNH and/or AA and their corresponding medical team at the University Hospital Basel, Switzerland, between November and December 2019. The participants were assigned to a particular symptom monitoring program based on their diagnosis and sex (AA, PNH female, PNH male).

The ePRO system provided by Kaiku Health, previously utilized in cancer patients, was customized for symptom monitoring in PNH and AA patients.

Each week, the patients were required to complete a questionnaire on 11 core symptoms, as well as measure and document their blood pressure, pulse, and temperature. During the first and last study weeks, they were also asked to fill out an health-related quality of life (HR-QoL) questionnaire.

Moreover, the participants had the option to report within the application their current blood counts and, if applicable, the time of eculizumab infusion.

In case of minor or moderate symptoms, automatic self-management advice would be sent to the patient. Severe symptoms would generate emergency first aid instructions and a warning to urgently seek medical assistance.

The application was tested for a total of 6 months by both the patients and their medical staff members.

Read more about PNH patient education

According to the results, the participants had a high adherence rate to the weekly symptom reporting (72%) and were satisfied with the application. The system was rated well for usage, comprehensibility, and integration into daily life. The majority of patients (78%) stated they would continue using the application after the study ended as well as recommend it to other PNH/AA patients.

Although the technical performance was rarely an issue and healthcare providers evaluated ePRO-AA-PNH as a useful supplement, the lack of integration of the ePRO system and the hospital information system was reported as a major barrier to usage.

“Symptom monitoring with ePROs may have the potential to detect symptoms and life-threatening complications earlier, thereby improving disease management. In these rare entities, ePROs may also help to guide patients remotely when living a greater distance from their specialized medical center,” Bänziger and colleagues wrote.

ePRO systems have previously been found to improve cancer patients’ outcomes and are becoming increasingly recognized in health care. However, their usage in rare hematological diseases has been much less studied.


Bänziger S, Weisshaar K, Arokoski R, et al. Feasibility of electronic patient-reported outcome monitoring and self-management program in aplastic anemia and paroxysmal nocturnal hemoglobinuria-a pilot study (ePRO-AA-PNH)Ann Hematol. Published online November 3, 2022. doi:10.1007/s00277-022-05012-5