Abstracts – Page 3

Impact on Pain and Activities of Daily Living after Switching to Emicizumab: Insight from PROBE

Kucher A, Clearfield E, Germini F, Dubé E, Merlen C, Bonnefoy A, St-Louis J, Rivard G.E, Skinner M. Impact on Pain and Activities of Daily Living after Switching to Emicizumab: Insight from PROBE.(2023), POSTER ABSTRACTS (PO177). Haemophilia, 29: 24-202. https://doi.org/10.1111/hae.14715

Introduction

Emicizumab for treatment of people with severe hemophilia A (SHA) was recently approved by Health Canada. A prospective observational study was initiated to evaluate the efficacy of emicizumab and assess health-related quality of life (HRQoL).

Methods

HRQoL outcomes were collected using the Patient Reported Outcomes Burdens and Experiences (PROBE) questionnaire. Eight male SHA (median age 22.5, range 17-55 years) completed PROBE pre- and ≥ 10 months post-switching to emicizumab. Acute and chronic pain were assessed as any occurrence (recall: 12 months) and during 8 activities (walking, stair climbing, nighttime, resting, weight bearing, playing, after falling/trauma, other). Participants reported pain interference in 11 aspects of life (general activity, walking ability, normal work, attending school, relations with others, sleep, enjoyment of life, playing/participating in sports/exercising, lifting, other). PROBE also includes a 24-item activities of daily living (ADL) list; current difficulty is reported. Descriptive statistics present results for pain outcomes and difficulty with ADL.

Results

The number of SHA reporting any acute pain, chronic pain, or ADL impairment was reduced post switch (7
[87.5%] to 4 [50.0%], 5[62.5%] to 4 [50%], 8 [100%] to 3 [37.5%], respectively). Switching was associated with improvement on self-reported acute and chronic pain occurrence during activities post-switch. Pre-switch SHA reporting acute pain indicated “yes” to the acute pain occurrence in 22 vs 8 cases post-switch. Chronic pain was similarly reduced from 20 to 7. No SHA reported acute or chronic pain occurrences for nighttime and resting post-switch. Reports of pain interference were also reduced: there were more affirmative reports of acute and chronic pain interfering pre-switch compared to post (42 vs 12 and 28 vs 15, respectively). Data also show an association with improvements in ADL from 72 activities reported difficult to perform pre-switch vs 20 post-switch. The most improved ADL post-switch were getting up from sitting, playing games or participating in sport, sleeping/resting, doing heavy domestic tasks.

Discussion/Conclusion

PROBE demonstrated that the introduction of emicizumab is associated with a significant decrease in self-reported acute and chronic pain, as well as reduced interference with activities of daily living.

Disclosure of Interest: Noone D.

View Poster: HERE

Exploring Regional Variations in the Cross‐cultural, International Implementation of the Patient Reported Outcomes Burdens and Experience (PROBE) Study

Chai‐Adisaksopha C, Noone D, Curtis R, Frick N, Nichol M, O’Mahony B, Page D, Stonebraker J, Iorio, A, Skinner MW. Exploring Regional Variations in the Cross‐cultural, International Implementation of the Patient Reported Outcomes Burdens and Experience (PROBE) Study. Abstract PB189. Res Pract Thromb Haemost. 2018;2(S1):1-368. https://doi.org/10.1002/rth2.12125

Background

The Patient Reported Outcomes Burdens and Experience (PROBE) study has developed and validated the multilingual PROBE questionnaire for assessing patient reported outcomes in people living with hemophilia (PWH) and participants without bleeding disorders (NOBD). The PROBE questionnaire translations into local languages account for site specific language and cultural peculiarities in the management and life experience of hemophilia.

Aims

To explore the regional variations in the international implementation of the PROBE questionnaire and examine the hypothesis that the PROBE score will not be confounded by cross-cultural aspects.

Methods

Data were collected from participants in four regions (Western Pacific, South America, North America and Europe). Participants were able to select from 23 localized language versions of the PROBE questionnaire based on their first language. We used analysis of variance methods and multivariable regression to determine the relative contribution of the variance explained by region controlling for hemophilia diagnosis, age group and level of education. We also explored interactions between region and the other components.

Results

We analyzed 862 questionnaires from 21 countries. Mean age of participants was 40.03 years (SD 13.89) and 65.20% were PWH and 34.80% were NOBD. After adjusting for hemophilia diagnosis, age group and level of education, region contributed 0.44% to 7.98% of the variance component in sub-item scores and 0.26% in the PROBE score. Years of education contributed 0.34% in the PROBE score. Age and diagnosis (NOBD, mild, moderate, severe) contributed 3.42% and 22.42% of the PROBE score. 70.74% of the variance was explained by inter-individual variation.

Conclusions

Variance partitioning for the PROBE score is similar to that for EQ5D. The results demonstrate that the PROBE questionnaire is valid to implement for assessing health status among PWH and participants without bleeding disorders across regions.

Validation of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study Questionnaire

Skinner M, Chai-Adisaksopha C, Noone D, Curtis R, Frick N, Nichol M, O’Mahony B, Page D, Pastarnak A, Stonebraker J, Iorio A and the Patient Reported Outcomes Burdens and Experiences (PROBE) Study Investigator Group. Validation of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study Questionnaire (Abstract). HAAB (2019).

Introduction

The interest of health care agencies, private payers and policy makers for patient-reported outcomes (PRO) is continuously increasing. There has been a substantial need to improve capacity to collect and interpret relevant PRO data to support implementation of patient-centered research and optimal care in hemophilia.

Objectives

The aims were to implement a structured data collection of PRO across countries to build a robust evidence base for comparative eﬀectiveness research, evidence-based decision making, and advocacy. Three intermediate objectives were identiﬁed:

develop a patient-led research network;
develop a standardized questionnaire to gather PRO; and
perform a feasibility study of implementing the PROBE questionnaire.

Additional aims were to explore the measurement properties of the PROBE questionnaire and demonstrate PROBEis valid to implement for assessing health status among PWH and participants without bleeding disorders acrossregions.

Methods:

Data collection was initiated 8 April 2015 and completed on 23 February 2017. In total, 2,101 surveys were collected through all phases of the study. Of these, 1,541 met study criteria for the relevant phases of research and were suitable for analysis.

Clinical Trial registration: NCT02439710.

Results

Validation studies for the PROBE questionnaire have been completed. The questionnaire was assessed for face validity, relevance, clarity and completeness(i); test-retest reliability (reproducibility) confirmed(ii); a core analytic framework (psychometric properties) established(iii); and cross-cultural validation demonstrated(iv). Outcomes of importance to PWH and metrics to consider for measurement were determined. The PROBEquestionnaire consists of four major sections (demographic data, general health problems, hemophilia-related healthproblems and health-related quality of life). PROBE questionnaire validation studies establish:

Face validity, relevance, clarity and completeness
Test-retest reliability (reproducibility)
A core analytic framework (psychometric properties)
Cross-cultural validation

Conclusions

The PROBE questionnaire is a valid and reliable tool for assessing health status among PWH and participants without bleeding disorders across regions. PROBE assesses patient-important PROs with ademonstrated short completion time. PROBE proved the feasibility to engage diverse patient communities instructured generation of real-world outcome data. The web‐based questionnaire and the paper‐based version maybe used interchangeably. The known group property of PROBE will allow its use in future clinical trials, longitudinalstudies, health technology assessment studies, routine clinical care or registries. Additional studies are planned totest responsiveness and sensitivity to change.

References

i.Skinner M, Chai-Adisaksopha C, Curtis R, Frick N, Nichol M, Noone D, et al. The Patient Reported Outcomes, Burdens and Experiences (PROBE) Project: development and evaluation of a questionnaire assessing patient reported outcomes in people with haemophilia. Pilot and Feasibility Studies. 2018;4(1):58.

ii.Chai-Adisaksopha C, Skinner MW, Curtis R, Frick N, Nichol MB, Noone D, et al. Test-retest properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire and its constituent domains. Haemophilia.0(0).

iii.Chai-Adisaksopha C, Skinner MW, Curtis R, Frick N, Nichol MB, Noone D, et al. Psychometric properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire. BmjOpen. 2018;8(8):10.

iv.Chai-Adisaksopha C, Noone D, Curtis R, Frick N, Nichol M, O’Mahony B, et al. Exploring regional variations in the cross-cultural, international implementation of the patient reported outcomes burdens and experience (PROBE) study. Research and Practice in Thrombosis and Haemostasis. 2018;2(S1):92.

View Poster: Validation of the PROBE Study Questionnaire

Comparison of the Measurement Properties of the PROBE and EQ5D on Pain

Skinner M, Chai-Adisaksopha C, Noone D, Curtis R, Frick N, Nichol M, O’Mahony B, Page D, Pastarnak A, Stonebraker J, Iorio A and the Patient Reported Outcomes Burdens and Experiences (PROBE) Study Investigator Group. Comparison of the Measurement Properties of the PROBE and EQ5D on Pain [Oral Presentation]. HTAi (2019).

Background

The Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire has been developed for assessing patient reported outcomes in people living with hemophilia (PWH). The PROBE questionnaire consists of 29 questions involving the following domains: hemophilia-related problems, general health problems and health-related quality of life. The PROBE questionnaire contains 3 questions assessing pain (current use of pain medications, occurrence and interference of acute and chronic pain).

Objectives

This study aims to investigate the measurement properties of pain assessment of the PROBE questionnaire compared with the pain and discomfort domain of EQ5D-5L.

Methods

The participants of the PROBE study were recruited via national patient organizations from 21 countries. Participants who did not state status of hemophilia (having hemophilia or not) or those who did not report severity of disease were excluded from the analysis. Descriptive data were reported as a proportion and mean (standard deviation) as appropriate. We calculated the correlation coefficient between EQ5D-5L (pain domain) and the occurrence and inference of acute pain and chronic pain from the PROBE questionnaire. We investigated the discriminative property of the pain domain of the PROBE questionnaire and EQ5D-5L.

Results

A total of 1675 participants were included in the analysis (PWH 68.7%, 31.3% participants without bleeding disorders). Mean age was 37.5 years (SD 17.4). Data from the PROBE revealed that during the past 12 months, 60.5% and 51.1% of participants reported they have acute and chronic pain, respectively. 79.6% of participants reporting the use of pain medications. Acute pain occurred when walking (30.5%) followed by night time and weight bearing. Acute pain interfered with general activities the most (38.3%) followed by walking ability and mood. Chronic pain occurred when walking (38.9%), followed by stairclimbing and weight bearing. Chronic pain interfered with general activities the most (35.7%), followed by walking ability and mood. Table 1 shows the correlation coefficient of the counts of occurrence and inference of acute and chronic pain (PROBE) versus the pain domain of the EQ5D-5L. The correlations were moderate between acute pain (PROBE) and the pain domain on EQ5D-5L, whereas the correlations were strong between chronic pain (PROBE) and the pain domain on EQ5D-5L. When classifying participants to 4 groups (controls, mild-, moderate- and severe hemophilia), the discriminative property of PROBE (figure 1) and EQ5D-5L-pain (figure 2) was excellent. Both tools can differentiate participants with hemophilia (varied severity) and without hemophilia, ANOVA p-value<0.05.

Conclusions

The pain questions on the PROBE questionnaire are well correlated with the pain domain on EQ5D-5L. The discriminative property of both tools is found to be excellent to separate people with various severities of hemophilia as well as people without bleeding disorders. The strength of the pain domain on the PROBE questionnaire is that it provides more informative data on the use of pain medication, occurrence and interference of acute and chronic pain. Therefore, the PROBE questionnaire is a disease-specific patient reported outcome measure, which will provide more insightful information regarding pain status in PWH.

View Presentation: PROBE and EQ5D-5L on pain assessment

Part-Time Employment and Early Retirement In People With Severe Haemophilia: Insights From The PROBE Study

Skinner M, Chai-Adisaksopha C, Noone D, Curtis R, Frick N, Nichol M, O’Mahony B, Page D, Pastarnak A, Stonebraker J, Iorio A and the Patient Reported Outcomes Burdens and Experiences (PROBE) Study Investigator Group. Part-Time Employment and Early Retirement In People With Severe Haemophilia: Insights From The PROBE Study. Poster Presentations #P204 Haemophilia. 2019;25(S1):35-188. https://doi.org/10.1111/hae.13666

Introduction

Despite treatment advances economic, social and educational barriers still remain for people with severe haemophilia. Contextual factors such as the lifetime impact of haemophilia on employment and overall work life are not well understood.

Methods

People with severe haemophilia A/B (PwSH) and controls with no bleeding disorder (NoBD) reporting working part-time or havingretired early due to their health were compared with those reporting working full-time. Patient Reported Outcome Burdens and Experiences (PROBE) Study data of 1008 participants age 18 and over from 21 countries were analyzed (550 PwSH, 458 NoBD). Descriptive statistics were used to present results as n (%), and odds ratio (95% CI) were calculated for the associations for participants with any health problems—use of mobility aids, use of pain medication, having acute or chronic pain, difficulties with activities of daily living and history of joint surgery—and assessed for their statistical significance.

Results

250 PwSH (45.5%) and 263 NoBD (57.4%) reported working full time. 86 PwSH (15.6%) and 80 NoBD (17.5%) reported working part-time. 27 of the 86 PwSH (31.4%) and 3 of the 80 NoBD (3.8%) reported working part-time due to health. 52 PwSH (9.5%) and 28 NoBD (6.1%) reported taking early retirement. 25 of the 52 PwSH (48.1%) and 1 of the 28 NoBD (3.6%) reported taking early retirement due to health. Association between reporting a health-related problem and working part-time or taking early retirement due to health were[n (%), Odds Ratio (95% CI), p-value]: use of mobility aids 77.7 (3.8-1645) 0.0005, having acute pain 41.2 (2-831.8) 0.01, use of pain medication 23 (2.05-258.1) 0.01, participants experiencing any health problems 22.5 (2-252.6) 0.01, having chronic pain 16.5 (1.5-179.2) 0.02, difficulty with activities of daily living (ADL) 16.5 (1.5-179.2) 0.02, and history of joint surgery 7.3 (0.4-148) 0.197. Mean participant age: PwSH 39 (14.4 SD) and NoBD 45.3 (13.7 SD).

Discussion/Conclusion

Haemophilia has a significant negative impact on work life. PwSH report a higher rate of retiring early or working part-time due to health than age-matched controls. Use of mobility aids, acute / chronic pain, difficulty with ADL and history of joint surgery are associated with retiring early or working part-time. The lifetime impact of haemophilia on employment should be more fully considered within health technology assessments.

Disclosure of Interest

M. Skinner Grant/Research support from: PROBE is an independent investigator led research project with grant / research support from: Baxalta, now part of Shire; Bayer; Bioverativ, a Sanofi Company; CSLBehring; Novo Nordisk, Roche and Sobi and the collaboration of the US National Hemophilia Foundation., C. Chai-Adisaksopha: None Declared, D. Noone: None Declared, R. Curtis: None Declared, N. Frick: None Declared, M. Nichol: None Declared, B. O’Mahony: None Declared, D. Page: None Declared, A. Pastarnak: None Declared, J. Stonebraker: None Declared, A. Iorio: None Declared

View Poster: Early Retirement and Part-time Employment

Predictors For Acute And Chronic Pain In Patients With Severe Haemophilia In The PROBE Cohort

Noone D, Chai-Adisaksopha C, Curtis R, Frick N, Nichol M, O’Mahony B, Page D, Pastarnak A, Stonebraker J, Iorio A, Skinner M. Predictors For Acute And Chronic Pain In Patients With Severe Haemophilia In The PROBE Cohort. Poster Presentations #P080. Haemophilia. 2019;25(S1):35-188. https://doi.org/10.1111/hae.13666

Introduction

For people with severe haemophilia, acute and chronic pain is often part of their daily reality. We looked at the extent to which this pain was influenced by annual bleed rate, presence of target joints or joints with reduced range of motion.

Methods

A binary regression analysis was performed to measure the association of acute and chronic pain with selected predictors from the PROBE Phase 2 data. The dependent variable (outcome) was the likelihood of reporting chronic and acute pain. The 5 predictor variables includedin the model were: age, reported annual bleeding rate (ABR), current treatment, presence of a target joint and range of motion (ROM) in a joint.

Results

There were 1287 respondents from 21 countries in total, 658 of whom had severe haemophilia and information for the predictor variables. For acute pain, the univariate logistic regression analysis showed a significant association for ABR (all bleeds) with patients reporting 2-3 bleeds/year being 2.9 times more likely to report acute pain compared to those with 0-1 bleeds/year, and those reporting more than 15 bleeds/year being 10.3 times more likely. Those reporting the presence of a target joint were 2.0 times more likely to report acute pain than thosewithout a target joint. In the full model (all 5 predictors) the impact of ABR is reduced but remains significant. Overall, the full model only predicts 13% of the variation in those who report acute pain. For chronic pain models, the univariate model predicts that those with 2-3 bleeds/year and >15 bleeds/year are 2.2 and 5.5 times more likely to report chronic pain compared to those with 0-1 bleeds/year, explaining 14% of the variance in chronic pain. In the full model however, the most responsible predictor was the presence of a joint with reduced ROM, withthose reporting a limitation being 5 times more likely to report chronic pain than those with full range of motion, which explains 23% of the variance.

Discussion/Conclusion

Our predictors were found to correlate with acute and chronic pain. While acute pain is primarily driven

by the frequency of bleeding and the presence of a target joint, chronic pain is primarily driven by the presence of a joint with reduced range of motion. Therefore, it would be worth exploring whether treatment regimens targeted to reduce annual bleeding rates or those aimed at improving the range of motion would be more effective in reducing chronic pain.

Disclosure of Interest

D. Noone Grant/Research support from: Baxalta, now part of Shire; Bayer; Bioverativ, a Sanofi Company; CSLBehring; Novo Nordisk, Roche and Sobi and the collaboration of the US National Hemophilia Foundation, Chai-Adisaksopha: None Declared, R. Curtis: None Declared, N. Frick: None Declared, M. Nichol: None Declared, B. O’Mahony: None Declared, D. Page: None Declared, A. Pastarnak: None Declared, J. Stonebraker: None Declared, A. Iorio: None Declared, M. Skinner: None Declared

View Poster: Predictors For Acute And Chronic Pain

Impact of Acute and Chronic Pain on the EQ-5D: Insights From The PROBE Study

Noone D, Chai-Adisaksopha C, Curtis R, Frick N, Nichol M, O’Mahony B, Page D, Pastarnak A, Stonebraker J, Iorio A, Skinner M. Impact of Acute and Chronic Pain on the EQ-5D: Insights From The PROBE Study. Poster Presentations #P079. Haemophilia. 2019;25(S1):35-188. https://doi.org/10.1111/hae.13666

Background

Acute and chronic pain as well as the potential access to medications, including haemophilia specific products, significantly impact the quality of life of people with haemophilia (PWH). Generic questionnaires such as the EQ-5D are used to measure the impact of the condition on life experience. EQ-5D in particular is designed to record the individual experience on the day of completion. There is little information on how the response to the pain domain in EQ-5D is driven by acute or chronic pain. The Patient Reported Outcomes, Burdens and Experiences (PROBE) is a questionnaire developed by patients to measure what matters to them in a way that allows comparison with people without bleeding disorders.

Aim and methods

We hypothesized that joint analysis of EQ-5D and PROBE measures would allow deeper understanding of the impact of acute and chronic pain on the respective scores. Descriptive and correlation analysis were performed.

Results

Of 1287 respondents, 686 have severe haemophilia and were examined in this article. We identified 4 subgroups based on responses to PROBE questions asking about acute and chronic pain: no pain (NP, 12.46%), acute pain (AP, 16.13%), chronic pain (CP, 16.23%) and both chronic and acute pain (A/CP, 55.13%). The mean (SD) ages for NP, AP, CP and A/CP were 27.5 (18.3), 24.8 (15.0), 39.2 (15.57) and 37.6 (16.0), respectively. There was a weak correlation (r =0.21, Pearson) between chronic and acute pain.

The mean (SD) EQ-5D utility values for NP, AP, CP and A/CP were 0.91 (0.12), 0.82 (0.21), 0.75 (0.17) and 0.65 (0.28), respectively. Standard t-tests indicated that all groups were statistically different from each other. A similar trend was seen in VAS score.

Conclusion

Acute pain has a significant impact on the quality of life of PWH. This is further exacerbated by the presence of chronic pain and the combination of both leads to the greatest decrease in quality of life. The EQ-5D does discern a difference between both types of pain. However, if chronic pain only is present at the time the EQ-5D is administered, the result may mask the full extent of the impact on the utility value unless the context and timing of using these generic tools is understood. It may lead to a misrepresentation of the true nature of pain within the severe haemophilia population.

View Poster: Impact of Acute and Chronic pain on the EQ-5D

Validation of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study Questionnaire

Skinner M, Chai‐Adisaksopha C, O’Mahony B, Noone D, Curtis R, Frick N, Nichol M, Page D, Stonebraker J, Iorio A. Patient Reported Outcomes Burdens and Experiences Study Group. Validation of the Patient Reported Outcomes Burdens and Experiences (PROBE) Study Questionnaire. Abstract PB1412. Res Pract Thromb Haemost. 2019;3(S1):1-891. https://doi.org/10.1002/rth2.12229

Background

A substantial need exists to improve capacity to collect and interpret relevant patient-reported outcomes (PRO) data to support patient-centered research and optimal care in people with hemophilia (PWH).

Aims

Implement a structured data collection of PRO across countries to build a robust evidence base for comparative eﬀectiveness research, evidence-based decision making, and advocacy. Three intermediate objectives were identiﬁed:

develop a patient-led research network;
develop a standardized questionnaire to gather PRO; and
perform a feasibility study of implementing the PROBE questionnaire.

Methods

In data collection from April 2015 to February 2017, 2,101 surveys were collected through all studyphases; 1,541 met study criteria for analysis. Clinical Trial registration: NCT02439710.

Results

Outcomes of importance to PWH and metrics to consider for measurement were determined. The PROBEquestionnaire consists of four major sections (demographic data, general health problems, hemophilia-related healthproblems and health-related quality of life). PROBE questionnaire validation studies establish:

Face validity, relevance, clarity and completeness
Test-retest reliability (reproducibility)
A core analytic framework (psychometric properties)
Cross-cultural validation

Conclusions

View Poster (English): Validation of PROBE’s Questionnaire

View Poster (Spanish): Validation of PROBE’s Questionnaire

Patient Reported Outcome Data on Switch from Standard to Extended Half Life in Irish Adults with Haemophilia

O’Mahony B, Pastarnak A, Noone A, Skinner M. Patient Reported Outcome Data on Switch from Standard to Extended Half Life in Irish Adults with Haemophilia. Abstract OC 42.4. Res Pract Thromb Haemost. 2019;3(S1):1-228. https://doi.org/10.1002/rth2.12227

Background

From 2017 to the end of 2018, all people with Haemophilia B and the majority with Haemophilia A (PwH) in Ireland were switched to extended half-life FIX (rFIXFc) or FVIII (rFVIIIFc) concentrates.

Objectives

The Irish Haemophilia Society surveyed measurable impact on their health-related quality of life.

Methods

Utilising the Patient Reported Outcomes, Burdens and Experiences (PROBE) survey, the Society surveyed members of the organisation with Haemophilia A/B pre-change and a minimum of 10 months post product change. There were 12 paired responses from people with Haemophilia B and 5 with haemophilia A.

Results

Three patients switched from episodic treatment to regular prophylaxis (2-HB, 1-HA).

Post-switch, there were reductions in PWH reporting the use of mobility aids (67% to 53%), acute pain (78% to 47%), frequency of use of pain medications, activities of daily living (ADL) being affected, reduced range of motion (83% v 65%) and chronic pain in a target joint (patient defined) (67% to 13%).

There was a significant reduction in reported bleeds. Pre-switch 29% reported having greater than 8 bleeds/year and 41% having 1 or less bleed/year. Post-switch there we no responses greater than 8 bleeds/year and 59% having 1 or less bleed/year.

Overall, VAS (68 v 73), EQ-5D (0.71 v 0.77) and PROBE (0.72 v 0.78) score improved post-switch, which equates to ~5-6% increase in utility in a year. All dimensions of the EQ-5D showed improvement with the most significant changes seen in mobility, self-care and pain/discomfort. (Table 1).

Conclusions

From this initial data set, there is a demonstrated benefit to PWH switching to EHL products in reduced bleeding rates as well as in the physical and pain limitations that haemophilia imposes on daily life. Data collection and analysis are ongoing.

View Presentation: Data on switch from standard to extended half life in Irish adults with haemophilia

Health Status of People Living with Non- severe Hemophilia – Insights from the Patient Reported Outcomes, Burdens and Experiences (PROBE) Study

Chai-Adisaksopha C, Skinner MW, Page D, Stonebraker J, Noone D, Curtis R, Frick N, Nichol M, O’Mahony B, Iorio A, and Patient Reported Outcomes Burdens and Experiences Study Group. Health Status of People Living with Non- severe Hemophilia – Insights from the Patient Reported Outcomes, Burdens and Experiences (PROBE) Study. Abstract OC 32.4. Res Pract Thromb Haemost. 2019;3(S1):1-228. https://doi.org/10.1002/rth2/12227

Background

People living with non-severe hemophilia (PwNSH), FVIII/IX 2-40 IU/dL, generally receive factor concentrate episodically. In recent studies, life expectancy of PwNSH is comparable to the non-hemophilia male population. However, their health status has not been systematically investigated.

Aims

To evaluate the health status of PwNSH

Methods

PROBE data for PwNSH and participants without a bleeding disorder (controls) were analyzed. A match-paired analysis, using 1:1 ratio of PwNSH and controls was performed. Health status of the two groups were compared using parametric or non-parametric methods.

Results

366 participants were included in this analysis. Mean age was 44.7 (SD 17.4) years in PwNSH and 45.8 (SD 14.4) in controls. Table 1 demonstrates health status of participants. Acute pain and chronic pain were more commonly reported among PwNSH (72.4% and 68.1%) compared to controls (37.6% and 30.0%), P< 0.001. Pain medications were more commonly used among PwNSH as compared to controls (86.2% vs 68.0%, P< 0.001). Working full-time was more frequent among controls. PwNSH more frequently reported being on long-term sick or disability compared to controls. Mean sick days per year were significantly higher in PwNSH (3.7 days vs 44.9 days, P< 0.001). Self-evaluated health status using the visual analog scale (VAS) shows that PwNSH had a significantly lower VAS utility score compared to controls (72.8 vs 83.7, P< 0.001). Overall health status, which was evaluated by mean PROBE score, indicated that PwNSH had worse health status compared with controls (0.71 vs 0.89, P< 0.001).

Conclusions

PwNSH had more health-related problems, worse work/school life, and lower health-related quality of life when compared to controls. PROBE findings demonstrate non-severe hemophilia is not a benign disease. Traditional clinical outcomes, e.g., annualized bleeding rate or mortality, are less sensitive to detect health status in PwNSH. We encourage investigators and clinicians to evaluate PwNSH using a patient-reported outcome assessment tool.

View Presentation: Health Status of People Living with Non- severe Hemophilia