Health Related Quality of Life (HrQoL) in Patients with Bleeding Disorder of Unknown Cause (BDUC)

Background: Patients with an increased bleeding tendency without any abnormalities in laboratory hemostasis tests are classified as having a bleeding disorder of unknown cause (BDUC). Data on health related quality of life (HrQoL) in BDUC patients and other patients with an increased bleeding tendency are scarce. The aim of this study was to investigate HrQoL in patients with BDUC and by extension in other patients with an increased bleeding tendency. Methods: 237 patients from the prospective Predictors of Bleeding Evaluation in Adult Hematologic Patients with Bleeding Tendencies cohort (ProBe-AHP study), in which patients with an increased bleeding tendency are included, were invited to complete the 36-item Short Form Health Survey (RAND-36). This questionnaire consists of 36 questions within eight subcategories including physical functioning, energy, emotional wellbeing, social functioning, pain, general health and role limitations due to physical health or emotional problems. These subcategories can be combined into the physical component summary (PCS) and the mental component summary (MCS). Scores range from 0-100, with 0 being the lowest HrQoL and 100 the highest. The ProBe-AHP study population consists of patients with an established bleeding disorder (BD), patients with BDUC and patients with no bleeding disorder (NBD). Data from a Dutch RAND-36 population survey were used as a reference (general population). Results: A total of 97/237 RAND-36 questionnaires were completed and returned. From the 97 returned questionnaires, 20% (n=19) were filled in by NBD patients, 34% (n=33) by BDUC patients and 46% (n=45) by BD patients. The median PCS score and the median MCS score was not statistically significant different between these three groups (Median PCS score [IQR]: NBD (84 [66-93], BDUC (61 [35-90]) and BD (72 [34-88]), median MCS score [IQR]: NBD (79 [53-87]), BDUC (75 [51-86]) and BD (68 [41-82]). Compared to the general population, BDUC patients had a statistically significant decreased score on the role limitations in physical health, energy, social functioning, pain and general health subcategories. Conclusion: HrQoL is impaired in BDUC and BD compared to the general population. No statistically significant difference in RAND-36 score was found between NBD, BDUC and BD patients in the ProBE-AHP cohort. These results suggest that HRQoL-related issues should be an integral part of the personalized management of BDUC and BD patients.