Skinner M. W., Kucher A., Clearfield E., Curtis R. G., Germini F., Iorio A., Nichol M., Page D., Stonebraker J. Development of Women, Girls, and People Who Have or Had the Potential to Menstruate (WGPPM) questionnaire for PROBE study. (2025), Book of Abstracts. Haemophilia, 31: 21-109. https://doi.org/10.1111/hae.70032
Objective
There is limited comparative quality-of-life (QoL) data for women and girls with hemophilia A or B who have the potential to menstruate (WGwH). To advance management and treatment it is essential to collect patient-reported health outcomes data. The validated PROBE questionnaire collects health-related QoL data from WGwH, including carriers, and women and girls who have the potential to mensurate without a bleeding disorder (NoBD) who serve as a control group. We compared the QoL of WGwH with NoBD.
Methods
This was a cross-sectional study from the PROBE database. We included WGwH ≥ age 13 who self-reported hemophilia A or B, as a carrier with a factor level <40%, or NoBD that were identified in the PROBE database. We used descriptive statistics to provide a summary of participant characteristics (Table 1).
Results
From 2015 to 2024, 232 WGwH indicated that they had severe (n=70), moderate (n=26) or mild (n=136) factor deficiency and 407 NoBD were available for comparison. Prophylaxis use was 65.71% in WGwH classified as severe, 19.23% in moderate, and 5.15% in mild. Use of mobility aids was 14.29% in severe, 38.46% in moderate, 14.71% in mild WGwH, and 8.85% in NoBD. Participants with moderate and mild hemophilia reported that this condition impacted important QoL metrics for: chronic pain (CP), target/problem joints (TJ/PJ), reduced joint range of motion (ROM) and difficulties with activities of daily living (ADL) (CP 61.54%, 44.85%; TJ/PJ 50%, 21.32%; ROM 50.0%, 19.85%; ADL 46.15%, 21.32% respectively). See Table 1.
Conclusions
Our study suggests a worse QoL for WGwH compared to NoBD, with a worsening trend with increasing severity. This analysis confirms prior research (Chai-Adisakosopha Haemophilia 2020) showing that non-severe hemophilia is not benign. WGwH are affected by their bleeding disorders. PROBE data collection for WGwH and further analysis are on-going.
| Table 1. Participant Characteristics | ||||
| Severe | Moderate | Mild | No BD | |
| n1 | 70 | 26 | 136 | 407 |
| Age (SD) | 37.19 (11.52) | 41.69 (14.77) | 45.09 (14.83) | 44.58 (14.75) |
| Years of education (SD) | 15.87 (4.87) | 15.4 (3.88) | 15.84 (4.5) | 16.87 (4.47) |
| Hemophilia A1 | 65 (92.86%) | 21 (80.77%) | 118 (86.76%) | n/a |
| Regular prophylaxis | 46 (65.71%) | 5 (19.23%) | 7 (5.15%) | n/a |
| Intermediate prophylaxis | 5 (7.14%) | 8 (30.77%) | 7 (5.15%) | n/a |
| Episodic (On-demand) treatment | 12 (17.14%) | 7 (26.92%) | 64 (47.06%) | n/a |
| No treatment available | 3 (4.29%) | 4 (15.38%) | 36 (26.47%) | n/a |
| Married | 50 (71.43%) | 19 (73.08%) | 97 (71.32%) | 304 (74.69%) |
| Have children | 56 (80.0%) | 19 (73.08%) | 107 (78.68%) | 284 (69.78%) |
| Used a mobility aid or assistive device (past 12 months) | 10 (14.29%) | 10 (38.46%) | 20 (14.71%) | 36 (8.85%) |
| Reported acute pain (past 12 months) | 30 (42.86%) | 15 (57.69%) | 73 (53.68%) | 149 (36.61%) |
| Reported chronic pain (past 12 months) | 27 (38.57%) | 16 (61.54%) | 61 (44.85%) | 158 (38.82%) |
| Currently have a Target Joint(s) or Problem Joint(s)2 | 30 (42.86%) | 13 (50.0%) | 29 (21.32%) | n/a |
| Report chronic pain in a Target Joint(s) or Problem Joint(s)2 | 18 (60%) | 7 (53.85%) | 15 (65.52%) | n/a |
| Currently have Joint(s) with reduced Range of Motion (ROM) | 29 (41.43%) | 13 (50.0%) | 27 (19.85%) | n/a |
| Ever had Joint Surgery or Other Invasive Procedure | 23 (32.86%) | 15 (57.69%) | 59 (43.38%) | 129 (31.7%) |
| Current difficulty with Activities of Daily Living (ADL) affected | 21 (30.0%) | 12 (46.15%) | 29 (21.32%) | 70 (17.2%) |
| Unemployed or on long-term disability (not a student) | 5 (7.14%) | 3 (11.54%) | 3 (2.21%) | 15 (3.69%) |
| Working full or part-time | 39 (55.71%) | 5 (19.23%) | 87 (63.97%) | 239 (58.72%) |
| Retired | 3 (4.29%) | 2 (7.69%) | 15 (11.03%) | 40 (9.83%) |
| Stay at home parent | 11 (15.71%) | 2 (7.69%) | 8 (5.88%) | 39 (9.58%) |
| Student (full or part-time) | 13 (18.57%) | 4 (15.38%) | 14 (10.29%) | 52 (12.78%) |
| Average days missed school or work due to health (SD) | 29.74 (86.41) | 24.8 (72.62) | 15.45 (49.03) | 10.5 (48.13) |
| EQ-5D-5L | 0.867 | 0.806 | 0.650 | 0.897 |
| PROBE Score | 0.848 | 0.766 | 0.817 | 0.872 |
| Annual Bleed Rate | ||||
| 0 | 17 (24.29%) | 4 (15.38%) | 74 (54.41%) | n/a |
| 1 | 5 (7.14%) | 2 (7.69%) | 24 (17.65%) | n/a |
| 2-3 | 23 (32.86%) | 7 (26.92%) | 15 (11.03%) | n/a |
| 4-7 | 9 (12.86%) | 5 (19.23%) | 11 (8.09%) | n/a |
| 8-10 | 3 (4.29%) | 4 (15.38%) | 0 | n/a |
| 11-15 | 4 (5.71%) | 2 (7.69%) | 3 (2.21%) | n/a |
| 16-30 | 4 (5.71%) | 1 (3.85%) | 4 (2.94%) | n/a |
| More than 30 | 3 (4.29%) | 0 | 2 (1.47%) | n/a |
| Bleeding in the past 2 weeks | 25 (35.71%) | 7 (26.92%) | 16 (11.76%) | n/a |
| Life threatening bleed (past 12 months) | 10 (14.29%) | 5 (19.23%) | 4 (2.94%) | n/a |
| 1. WGwH who self-report having hemophilia or as carriers with a factor level <40.
2. Beginning with PROBE questionnaire Version 3, the generic question asking about target joints was revised to ask about ‘Problem Joint(s)’. A Problem Joint is defined as having chronic joint pain and/or limited range of movement due to compromised joint integrity (e.g. chronic synovitis and/or hemophilic arthropathy), with or without persistent bleeding. |
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Oral Presentation: HERE
