Monitoring of Patient-Reported Outcomes in Patients with Bleeding Disorders to Assure Quality of Care: Results from a Single Outpatient Haemophilia Treatment Centre in Germany

Background: By collecting certain treatment data during patient's care and comparing these data over time, reliable findings are obtained on whether and at which points there is a need for improvement. Due to the complexity and high risk of complications in the treatment of blood coagulation disorders, an interdisciplinary care of patients is needed to ensure a high-quality and efficient therapy. The Coagulation Treatment Centre rhein-ruhr (GZRR) follows a holistic treatment concept providing cross-sectoral care through interdisciplinary cooperation. This approach is intended also to improve patients' treatment satisfaction (TS), physical functioning (PF) and health-related quality of life (HRQoL). The aim was to evaluate TS, PF and HRQoL in patients treated at the GZRR.

Methods: Since 2019 patients > 5 years attending the routine visits at the GZRR completed validated and standardised patient-reported outcomes (PROs) at each visit. Patients > 12 years filled in the generic SF-36 for the assessment of HRQoL, haemophilia-specific HRQoL is assessed with the Haem-A-QoL (adults) or age-related versions of the Haemo-QoL (kids) questionnaires. For the evaluation of TS and PF haemophilia- and age-group specific questionnaires were administered (Hemo-Sat_A+P, HEP-Test-Q).

Results: 177 patients (range 6-80 years) with bleeding disorders were enrolled, among them 55.9% had haemophilia (Haemophilia A, n=88; Haemophilia B, n=11) and 44.1% had VWD; 57.1% were severely affected. Sixty-eight were children (39%) with a mean age of 11.44±3.4 years and BMI of 18.82±3.7; 109 were adults with a mean age of 38.79±16.7 years and BMI of 25.72±5.5. Patients were relatively satisfied with their treatment, adults reported highest impairments in the domain 'burden', while parents were mainly dissatisfied concerning 'ease & convenience'. Children (_C) reported a significantly better (p<.0001) subjective physical functioning compared to adults (_A). Both age groups showed highest impairments in the domain 'endurance' (M_C=70.69±20.3 vs. M_A=51.48±25.4). Patients reported good HRQoL in the Mental Component Score of the SF-36 50.07±11.1 and showed some impairments in the Physical Component Score 45.26±11.9; significant difference were found between children and adults in all subdomains of the SF-36.

Conclusions: These findings demonstrate that the interdisciplinary approach leads to good results in PROs. Based on the findings direct short-term interventions could be administered to solve minor issues. Follow-up data could prove that this kind of quality-of-care assurance helps not only to improve the clinical situation of patients, but also their PRO.

No relevant conflicts of interest to declare.