Study protocol for developing a patient-reported outcome measure for infection screening in hematological patients with secondary immunodeficiency

Patients with chronic lymphocytic leukemia (CLL) or multiple myeloma (MM) frequently develop secondary immunodeficiencies (SID), on the basis of both their respective disease, as well as immunosuppressive therapies, leading to increased infection risks and impaired health-related quality of life (HRQOL). While timely infection detection is essential, outpatient symptom monitoring remains challenging. Electronic patient-reported outcome (ePRO) tools have shown promise for real-time symptom monitoring, enhancing early detection and management. We describe a study protocol for developing an ePRO screening tool to monitor infections and assessing HRQOL in patients with SID and chronic lymphocytic leukemia (CLL) or multiple myeloma (MM). The study consists of two phases. We first developed an item list to measure infection-related symptoms and a mobile application for the daily assessment of the item list. The study will be conducted with 120 patients across multiple centers in Austria and Germany, observing patient-reported symptoms and HRQOL over a 12-month period. The primary outcome is the predictive accuracy of the PRO screening tool for infection detection. Secondary outcomes include health-related quality of life, the incidence rate of infections, and completion rates of the scheduled PRO assessments during the study period. The PRO-SID study will evaluate whether remote symptom monitoring for infections can help detect infections in patients with hematological malignancies with SID, a population where early detection is critical for reducing morbidity and mortality. By potentially improving infection detection in outpatient settings, our study has the potential to make treatment safer and more effective for patients, ultimately enhancing clinical outcomes and reducing the risk of severe infection-related complications. https://clinicaltrials.gov/study/NCT06821880 .