Svirca I, Kukaj E, Svirca L, Rotellini D, Harvey-Simi K, et al. Patient-reported outcomes in hemophilia in Kosovo: results from the PROBE questionnaire. Haemophilia. 2026;32:4-224. Abstract PP-317. doi:10.1111/hae.70232.
Introduction
National-level patient-reported outcomes (PROs) for people with hemophilia (PWH) in Kosovo are limited. The PROBE (Patient-Reported Outcomes, Burdens and Experiences) questionnaire captures treatment patterns, pain, function, and health status.
The aim is to characterize the Kosovo cohort with PROBE and compare key outcomes between severe and moderate/mild hemophilia contextualized against a local control group with no bleeding disorder (PwNBD).
Methods
We conducted a cross-sectional analysis of participants in Kosovo who completed PROBE during the National Bleeding Disorders Foundation and Kosovo Hemophilia Foundation (KHF) World Federation of Hemophilia (WFH) twinning program. A formal framework of cooperation between KHF, WFH, and the Ministry of Health of Kosovo was executed. Data were collected between April 2023 and May 2024. Outcomes included treatment regimen, acute and chronic pain, missed work or school, EQ-5D index, and PROBE total score. Participants were grouped as Severe, Moderate/Mild, or PwNBD, and results were summarized descriptively.
Results
Thirty-three participants were included in the initial pilot data collection: Severe (n=6), Moderate/Mild (n=13), and No BD/Carriers (n=12). Acute pain was reported by 66.7% of Severe versus 46.2% of Moderate/Mild participants; chronic pain by 50.0% versus 53.9%. Mean days missed from work or school in the previous six months were higher in the PWH group than in PwNBD. The EQ-5D index (mean) was 0.558 in Severe, 0.859 in Moderate/Mild, and 0.950 in PwNBD. PROBE total scores (mean) were 0.794, 0.843, and 0.906, respectively, indicating worse reported outcomes in hemophilia groups versus controls. Additional patient characteristics are described in Table 1.
Conclusion
Using the PROBE instrument to measure quality of life, individuals with severe hemophilia in Kosovo reported lower health status (EQ-5D) and higher burden (PROBE score) with greater functional impact (missed work/school) than those with moderate/mild disease, and both groups fared worse than the PwNBD control group. Only one-third of Severe participants were on regular prophylaxis, with half relying on intermittent regimens and some reporting no treatment availability. Findings highlight persistent unmet needs in access to regular prophylaxis, pain management, and joint health. Ongoing data collection will support longitudinal evaluation to guide national health policy for PWH.
Table. Patient Characteristics
| Severe | Moderate / Mild | PwNBD | ||||
| n (%) / Mean (SD) | 95% CI | n (%) / Mean (SD) | 95% CI | n (%) / Mean (SD) | 95% CI | |
| n | 6 | 13 | 12 | |||
| Age average | 29.5 (8.90) | 22.377 – 36.623 | 29.46 (8.47) | 24.856 – 34.067 | 36.25 (11.17) | 29.932 – 42.568 |
| Years of Education average | 12.17 (3.76) | 9.158 – 15.175 | 12.62 (3.20) | 10.876 – 14.355 | 14.92 (4.33) | 12.467 – 17.366 |
| Hemophilia A | 4 (66.67 %) | 7 (53.85%) | n/a | |||
| Treatment Regiment | ||||||
| Regular Prophylaxis | 2 (33.33%) | 2 (15.39%) | n/a | |||
| Interm. PROXY | 3 (50%) | 3 (23.08%) | n/a | |||
| On Demand | 0 | 3 (23.08%) | n/a | |||
| No treatment available | 1 (16.67%) | 0 | n/a | |||
| Married | 3 (50%) | 9 (69.23%) | 10 (83.33%) | |||
| Children | 4 (66.67 %) | 5 (38.46%) | 8 (66.67%) | |||
| Used a mobility device in last 12 months | 2 (33.33%) | 0 | 0 | |||
| Pain med Frequency | ||||||
| Never (0% of the time) | 2 (33.33%) | 1 (7.69%) | 6 (50%) | |||
| Rarely (1–5% of the time) | 0 | 3 (23.08%) | 3 (25%) | |||
| Occasionally (6–25% of the time) | 2 (33.33%) | 6 (46.15%) | 1 (8.33%) | |||
| Sometimes (26%–50% of the time) | 1 (16.67%) | 3 (23.08%) | 2 (16.67%) | |||
| Frequently (51%–75% of the time) | 1 (16.67%) | 0 | 0 | |||
| Very Frequently (76%–99% of the time) | 0 | 0 | 0 | |||
| All the time (100%) | 0 | 0 | 0 | |||
| Acute pain | 4 (66.67 %) | 6 (46.15%) | 6 (50%) | |||
| Mean number of activities where Acute Pain Occurs | 1.33 (1.37) | 0.234 – 2.433 | 1.38 (1.73) | 0.518 – 2.252 | 0.08 (0.28) | -0.138 – 0.304 |
| Mean number of activities where Acute Pain interferes | 2.67 (2.43) | 0.725 – 4.608 | 1.69 (2.58) | 1.179 – 3.988 | 0.17 (0.55) | -0.146 – 0.479 |
| Chronic pain | 3 (50%) | 7 (53.85%) | 4 (33.33%) | |||
| Mean number of activities where Chronic Pain Occurs | 1.17 (1.34) | 0.0915 – 2.242 | 1.38 (1.60) | 0.518 – 2.252 | 0.50 (0.87) | 0.0100 – 0.990 |
| Mean number of activities where Chronic Pain Interferes | 1.50 (1.80) | 0.0575 – 2.942 | 1.85 (2.48) | 0.500 – 3.192 | 0.82 (1.34) | 0.0731 – 1.594 |
| Chronic pain in a target joint | 4 (66.67 %) | 6 (46.15%) | n/a | |||
| ADL affected | 4 (66.67 %) | 7 (53.85%) | 2 (16.67%) | |||
| Mean number of ADL’s effected | 2.17 (2.19) | 0.413 – 3.921 | 1.77 (2.29) | 0.523 – 3.015 | 0.25 (0.60) | -0.087 – 0.587 |
| Employment and Education | ||||||
| Unemployed/ long-term disability/ not a student | 2 (33.33%) | 1 (7.69%) | 0 | |||
| Unemployed due to health | 2/2 (100%) | 1/1 (100%) | 0/2 (0%) | |||
| Working full time / working part time | 2 (33.33%) | 11 (84.62%) | 7 (58.33%) | |||
| Work part-time due to health | 0 | 2/5 (40%) | 3/3 (100%) | |||
| Retired | 0 | 0 | 0 | |||
| Retirement due to health | 0 | 0 | 0 | |||
| Stay at home parent | 1 (16.67%) | 0 | 2 (16.67%) | |||
| Student (full time or part-time) | 3 (50%) | 3 (23.08%) | 4 (33.33%) | |||
| Education/Career decision due to health | 3 (50%) | 3 (23.08%) | 0 | |||
| Average of missed days of school or/and work | 25.67 (42.81) | -8.587 – 59.920 | 10.85 (10.41) | 5.190 – 16.503 | 7.42 (10.75) | 1.334 – 13.499 |
| Joint Surgery or Other Procedure | 2 (33.33%) | 3 (23.08%) | 1 (8.33%) | |||
| Mean of Joint Surgery or Other Procedure | 1.17 (2.19) | -0.587 – 2.921 | 0.31 (0.61) | -0.022 – 0.637 | 0.08 (0.28) | -0.073 – 0.240 |
| EQ5D | 0.558 (0.296) | 0.322 – 0.795 | 0.859 (0.169) | 0.767 – 0.950 | 0.95 (0.07) | 0.913 – 0.988 |
| PROBE score | 0.794 (0.105) | 0.710 – 0.877 | 0.843 (0.108) | 0.784 – 0.901 | 0.906 (0.07) | 0.864 – 0.948 |
| Annual Bleed Rate | ||||||
| 0 | 1 (16.67%) | 4 (30.77%) | n/a | |||
| 1 | 0 | 0 | n/a | |||
| 2-3 | 0 | 1 (7.69%) | n/a | |||
| 4-7 | 0 | 4 (30.77%) | n/a | |||
| 8-10 | 3 (50%) | 1 (7.69%) | n/a | |||
| 11-15 | 1 (16.67%) | 0 | n/a | |||
| 16-30 | 1 (16.67%) | 1 (7.69%) | n/a | |||
| More than 30 | 0 | 2 (15.39%) | n/a | |||
| Bleeding in last 2 weeks | 5 (83.33%) | 6 (46.15%) | n/a | |||
| Target Joints/Problem Joints | 5 (83.33%) | 11 (84.62%) | n/a | |||
| Mean number of Target Joints/Problem Joints | 4.00 (3.16) | 1.470 – 6.530 | 2.54 (2.68) | 1.083 – 3.994 | n/a | |
| Joint with a reduced Range of motion | 5 (83.33%) | 8 (61.54%) | n/a | |||
| Mean number of joints with a reduced Range of Motion | 3.00 (2.24) | 1.211 – 4.789 | 1.77 (1.89) | 0.743 – 2.795 | n/a | |
| Life threatening bleed in the last 12 months | 4 (66.67 %) | 2 (15.39%) | n/a | |||
| Mean number of Life threatening bleed in the last 12 months | 1.00 (1.00) | 0.200 – 1.800 | 0.31 (0.72) | -0.085 – 0.700 | n/a | |
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