Correlations between patient-reported outcomes and self-reported characteristics in adults with hemophilia B and caregivers of children with hemophilia B: analysis of the B-HERO-S study

Figures & data

Table

Survey Question	BPI Average Pain^b	BPI Current Pain^b	BPI Pain Severity^c	BPI Pain Interference^d	HAL Upper Extremity^e	HAL Basic Lower Extremity^e	HAL Complex Lower Extremity^e	HAL Overall Score^e	PHQ-9 Total Score^f
Not working (vs working full or part-time)	0.019^g	−0.077^g	−0.075^g	−0.247^g	0.112^g	0.160^g	0.254^g	0.145^g	−0.531
Not working due to complications from hemophilia	0.365^g	0.428^g	0.459	0.394^g	−0.522	−0.529	−0.538	−0.563	0.346^g
Current treatment allows me to work	−0.403	−0.349	−0.398	−0.428	0.238	0.238	0.256	0.278	−0.506
Increasing negative experiences with work/career	0.443	0.467	0.471	0.52	−0.594	−0.564	−0.499	−0.617	0.125^g
Increasing satisfaction with support from employer or manager	−0.338	−0.378	−0.35	−0.328	0.357	0.234	0.245	0.322	0.002^g
Negative experience with telling employer or manager	0.54	0.62	0.601	0.572	−0.693	−0.561	−0.45	−0.654	0.034^g

Notes: ^aRange, −0.011–1.0 (higher scores indicate better quality of life or functional status). ^bRange, 0–10 (higher scores indicate greater pain severity or pain interference). ^cPain severity score is the average of 4 severity scores (worst, least, average, current). ^dPain interference score is the average of 7 interference scores. ^eRange, 0–100 (higher scores indicate better quality of life or functional status). ^fRange, 0–27 (higher scores indicate more severe depression). ^gNot significant. Negative attributes are shown in red. Inverse correlations are highlighted in gray. Correlations highlighted in bold are considered high (r≥0.37).

Abbreviations: BPI, Brief Pain Inventory v2 Short Form; HAL, Hemophilia Activities List; PHQ-9, Patient Health Questionnaire; VAS, visual analog scale.

Table 1 Pearson correlation assessment between hemophilia impact on work and PRO scores in adults with hemophilia B

Survey Question	EQ-5D-5L Mobility	EQ-5D-5L Self-care	EQ-5D-5L Usual Activities	EQ-5D-5L Pain/Discomfort	EQ-5D-5L Anxiety/Depression	EQ-5D-5L Index Score^a	EQ-5D-5L Overall Health VAS Score	BPI Worst Pain^b	BPI Least Pain^b
Not working (vs working full or part-time)	−0.291	−0.055^g	−0.119^g	−0.315	−0.127^g	0.155^g	−0.244^g	−0.285	0.106^g
Not working due to complications from hemophilia	0.506	0.201^g	0.183^g	0.472	0.545	−0.533	−0.487	0.472	0.305^g
Current treatment allows me to work	−0.266	−0.257	−0.426	−0.402	−0.323	0.343	0.208	−0.353	−0.36
Increasing negative experiences with work/career	0.523	0.445	0.492	0.449	0.399	−0.591	−0.587	0.367	0.439
Increasing satisfaction with support from employer or manager	−0.319	−0.292	−0.321	−0.19	−0.282	0.375	0.314	−0.219	−0.315
Negative experience with telling employer or manager	0.511	0.51	0.511	0.357	0.352	−0.57	−0.678	0.424	0.573

Table

Survey Question	BPI Average Pain^b	BPI Current Pain^b	BPI Pain Severity^c	BPI Pain Interference^d	HAL Upper Extremity^e	HAL Basic Lower Extremity^e	HAL Complex Lower Extremity^e	HAL Overall Score^e	PHQ-9 Total Score^f
Increasing limitation in functional abilities and engagement in activities	0.441	0.438	0.46	0.467	−0.47	−0.468	−0.417	−0.508	0.248
Sometimes/always used cane/crutches/walker in the past 6 months	0.594	0.582	0.644	0.618	−0.483	−0.446	−0.403	−0.52	0.551
Sometimes/always used wheelchair in the past 6 months	0.566	0.582	0.609	0.59	−0.409	−0.408	−0.344	−0.468	0.432
Increasing negative impact of hemophilia on ability to engage in activities	0.34	0.342	0.373	0.446	−0.516	−0.488	−0.53	−0.548	0.16
Increasing treatment adjustment to accommodate participation in activities	0.492	0.472	0.496	0.477	−0.434	−0.421	−0.304	−0.442	0.204

Notes: ^aRange, −0.011–1.0 (higher scores indicate better quality of life or functional status). ^bRange, 0–10 (higher scores indicate greater pain severity or pain interference). ^cPain severity score is the average of 4 severity scores (worst, least, average, current). ^dPain interference score is the average of 7 interference scores. ^eRange, 0–100 (higher scores indicate better quality of life or functional status). ^fRange, 0–27 (higher scores indicate more severe depression). Negative attributes are shown in red. Inverse correlations are highlighted in gray. Correlations highlighted in bold are considered high (r≥0.37).

Abbreviations: BPI, Brief Pain Inventory v2 Short Form; HAL, Hemophilia Activities List; PHQ-9, Patient Health Questionnaire; VAS, visual analog scale.

Table 2 Pearson correlation assessment between hemophilia impact on functional and recreational activities and PRO scores in adults with hemophilia B

Survey Question	EQ-5D-5L Mobility	EQ-5D-5L Self-care	EQ-5D-5L Usual Activities	EQ-5D-5L Pain/Discomfort	EQ-5D-5L Anxiety/Depression	EQ-5D-5L Index Score^a	EQ-5D-5L Overall Health VAS Score	BPI Worst Pain^b	BPI Least Pain^b
Increasing limitation in functional abilities and engagement in activities	0.496	0.44	0.508	0.348	0.45	−0.521	−0.502	0.395	0.415
Sometimes/always used cane/crutches/walker in the past 6 months	0.474	0.509	0.628	0.6	0.535	−0.59	−0.424	0.547	0.631
Sometimes/always used wheelchair in the past 6 months	0.511	0.524	0.538	0.566	0.519	−0.581	−0.484	0.498	0.579
Increasing negative impact of hemophilia on ability to engage in activities	0.469	0.341	0.405	0.416	0.358	−0.45	−0.502	0.425	0.287
Increasing treatment adjustment to accommodate participation in activities	0.391	0.453	0.426	0.378	0.507	−0.537	−0.457	0.418	0.439

(Continued)

Survey Question	BPI Average Pain^b	BPI Current Pain^b	BPI Pain Severity^c	BPI Pain Interference^d	HAL Upper Extremity^e	HAL Basic Lower Extremity^e	HAL Complex Lower Extremity^e	HAL Overall Score^e	PHQ-9 Total Score^f
Hemophilia affected relationship with (prospective) partners	0.287	0.28	0.307	0.274	−0.398	−0.251	−0.147	−0.323	−0.114^g
Increasing satisfaction with support from partner regarding hemophilia	−0.516	−0.494	−0.56	−0.528	0.537	0.426	0.234	0.473	−0.127^g
Hemophilia affected the quality of sex life	0.508	0.479	0.468	0.369	−0.302	−0.233^g	−0.162^g	−0.253	−0.119^g
Increasing satisfaction with support from friends	−0.387	−0.397	−0.419	−0.426	0.458	0.326	0.222	0.4	0.002^g
Negative experiences with telling friends about hemophilia	0.388	0.44	0.428	0.41	−0.49	−0.449	−0.29	−0.478	−0.009^g
Bullied by peers because of hemophilia	0.459	0.509	0.49	0.442	−0.527	−0.508	−0.334	−0.52	−0.080^g
Negative experiences with telling colleagues at work/school about hemophilia	0.351	0.384	0.368	0.367	−0.436	−0.333	−0.253	−0.397	−0.074^g
Bullied by colleagues at work/school because of hemophilia	0.286	0.321	0.315	0.309	−0.408	−0.363	−0.215	−0.391	−0.208

Notes: ^aRange, −0.011–1.0 (higher scores indicate better quality of life or functional status). ^bRange, 0–10 (higher scores indicate greater pain severity or pain interference). ^cPain severity score is the average of 4 severity scores (worst, least, average, current). ^dPain interference score is the average of 7 interference scores. ^eRange, 0–100 (higher scores indicate better quality of life or functional status). ^fRange, 0–27 (higher scores indicate more severe depression). ^gNot significant. Negative attributes are shown in red. Inverse correlations are highlighted in gray. Correlations highlighted in bold are considered high (r≥0.37).

Abbreviations: BPI, Brief Pain Inventory v2 Short Form; HAL, Hemophilia Activities List; PHQ-9, Patient Health Questionnaire; VAS, visual analog scale.

Table 3 Pearson correlation assessment between hemophilia impact on family and relationships and PRO scores in adults with hemophilia B

Survey Question	EQ-5D-5L Mobility	EQ-5D-5L Self-care	EQ-5D-5L Usual Activities	EQ-5D-5L Pain/Discomfort	EQ-5D-5L Anxiety/Depression	EQ-5D-5L Index Score^a	EQ-5D-5L Overall Health VAS Score	BPI Worst Pain^b	BPI Least Pain^b
Hemophilia affected relationship with (prospective) partners	0.153	0.302	0.29	0.119	0.245	−0.294	−0.451	0.146	0.38
Increasing satisfaction with support from partner regarding hemophilia	−0.358	−0.464	−0.502	−0.343	−0.486	0.499	0.532	−0.397	−0.575
Hemophilia affected the quality of sex life	0.209^g	0.131^g	0.26	0.362	0.427	−0.389	−0.477	0.246^g	0.431
Increasing satisfaction with support from friends	−0.331	−0.296	−0.323	−0.187	−0.366	0.409	0.488	−0.258	−0.466
Negative experiences with telling friends about hemophilia	0.414	0.402	0.379	0.293	0.388	−0.478	−0.492	0.282	0.436
Bullied by peers because of hemophilia	0.438	0.463	0.366	0.281	0.426	−0.529	−0.543	0.343	0.463
Negative experiences with telling colleagues at work/school about hemophilia	0.303	0.377	0.358	0.191	0.346	−0.424	−0.498	0.248	0.358
Bullied by colleagues at work/school because of hemophilia	0.272	0.368	0.272	0.125	0.278	−0.393	−0.49	0.254	0.299

(Continued)

Table

Survey Question	BPI Average Pain^b	BPI Current Pain^b	BPI Pain Severity^c	BPI Pain Interference^d	HAL UpperExtremity^e	HAL Basic Lower Extremity^e	HAL Complex Lower Extremity^e	HAL Overall Score^e	PHQ-9 Total Score^f
Experienced stressful events: loss of job	0.667	0.639	0.668	0.65	−0.269	−0.275	−0.162	−0.304	0.694
Received psychological treatment in the past 5 years	0.67	0.629	0.681	0.676	−0.311	−0.308	−0.248	−0.364	0.7
Psychological treatment related to hemophilia	0.332	0.29	0.371	0.39	−0.239	−0.104^g	0.001^g	−0.135^g	0.307

Notes: ^aRange, −0.011–1.0 (higher scores indicate better quality of life or functional status). ^bRange, 0–10 (higher scores indicate greater pain severity or pain interference). ^cPain severity score is the average of 4 severity scores (worst, least, average, current). ^dPain interference score is the average of 7 interference scores. ^eRange, 0–100 (higher scores indicate better quality of life or functional status). ^fRange, 0–27 (higher scores indicate more severe depression). ^gNot significant. Negative attributes are shown in red. Inverse correlations are highlighted in gray. Correlations highlighted in bold are considered high (r≥0.37).

Abbreviations: BPI, Brief Pain Inventory v2 Short Form; HAL, Hemophilia Activities List; PHQ-9, Patient Health Questionnaire; VAS, visual analog scale.

Table 4 Pearson correlation assessment between the psychological/stress impact of hemophilia and PRO scores in adults with hemophilia B

Survey Question	EQ-5D-5L Mobility	EQ-5D-5L Self-care	EQ-5D-5L Usual Activities	EQ-5D-5L Pain/Discomfort	EQ-5D-5L Anxiety/Depression	EQ-5D-5L Index Score^a	EQ-5D-5L Overall Health VAS Score	BPI Worst Pain^b	BPI Least Pain^b
Experienced stressful events: loss of job	0.401	0.393	0.613	0.581	0.621	−0.582	−0.445	0.539	0.599
Received psychological treatment in the past 5 years	0.448	0.441	0.632	0.624	0.606	−0.597	−0.488	0.588	0.62
Psychological treatment related to hemophilia	0.015^g	0.228	0.151^g	0.176	0.328	−0.211	−0.271	0.232	0.394

Table

Survey Question	BPI Average Pain^b	BPI Current Pain^b	BPI Pain Severity^c	BPI Pain Interference^d	HAL Upper Extremity^e	HAL Basic Lower Extremity^e	HAL Complex Lower Extremity^e	HAL Overall Score^e	PHQ-9 Total Score^f
Access to and responsibility for factor/treatment
Self-responsibility for your hemophilia care (vs somebody else)	−0.481	−0.494	−0.506	−0.44	0.503	0.381	0.236	0.466	0.045^g
Routine factor treatment (vs on-demand)	0.486	0.471	0.498	0.43	−0.459	−0.452	−0.292	−0.456	−0.011^g
Difficulty obtaining or concerns about factor products’ availability or affordability
Last 5 years	0.611	0.564	0.631	0.625	−0.257	−0.157	−0.123	−0.246	0.657
Next 5 years	0.41	0.414	0.432	0.422	−0.44	−0.299	−0.186	−0.38	−0.006^g
Lack of control^h	0.028^g	−0.022^g	−0.004^g	−0.081^g	0.445	0.45	0.403	0.449	0.31
Access to HTC
Fewer visits to HTC in the past year	0.272	0.283	0.295	0.293	0.079^g	−0.024^g	−0.025^g	0.009^g	0.633

Notes: ^aRange, −0.011–1.0 (higher scores indicate better quality of life or functional status). ^bRange, 0–10 (higher scores indicate greater pain severity or pain interference). ^cPain severity score is the average of 4 severity scores (worst, least, average, current). ^dPain interference score is the average of 7 interference scores. ^eRange, 0–100 (higher scores indicate better quality of life or functional status). ^fRange, 0–27 (higher scores indicate more severe depression). ^gNot significant. ^hRespondents rated disease control on a scale of 0–10, with 0 indicating “not at all” and 10 indicating “extremely well.” Negative attributes are shown in red. Inverse correlations are highlighted in gray. Correlations highlighted in bold are considered high (r≥0.37).

Abbreviations: BPI, Brief Pain Inventory v2 Short Form; HAL, Hemophilia Activities List; HTC, hemophilia treatment center; PHQ-9, Patient Health Questionnaire; VAS, visual analog scale.

Table 5 Pearson correlation assessment between access to and responsibility for hemophilia treatment and PRO scores in adults with hemophilia B

Survey Question	EQ-5D-5L Mobility	EQ-5D-5L Self-care	EQ-5D-5L Usual Activities	EQ-5D-5L Pain/Discomfort	EQ-5D-5L Anxiety/Depression	EQ-5D-5L Index Score^a	EQ-5D-5L Overall Health VAS Score	BPI Worst Pain^b	BPI Least Pain^b
Access to and responsibility for factor/treatment
Self-responsibility for your hemophilia care (vs somebody else)	−0.326	−0.474	−0.394	−0.244	−0.394	0.487	0.561	−0.317	−0.528
Routine factor treatment (vs on-demand)	0.36	0.453	0.335	0.293	0.414	−0.502	−0.468	0.411	0.459
Difficulty obtaining or concerns about factor products’ availability or affordability
Last 5 years	0.289	0.399	0.617	0.498	0.53	−0.483	−0.454	0.511	0.62
Next 5 years	0.323	0.351	0.399	0.232	0.366	−0.449	−0.57	0.279	0.459
Lack of control^h	−0.27	−0.309	−0.133	−0.038^g	−0.172	0.279	0.368	−0.002^g	−0.013^g
Access to HTC
Fewer visits to HTC in the past year	0.237	0.077^g	0.26	0.447	0.327	−0.229	−0.061^g	0.319	0.24

Table 6 Pearson correlation assessment between impact of hemophilia and PRO scores in caregivers of children with hemophilia B

Survey Question	PHQ-9^a	GAD-7^b
	(Depression)	(Anxiety)
Impact on work due to caring for a child with hemophilia
CG or SP had to leave job	0.338	0.393
CG or SP overlooked for promotion	0.611	0.649
CG or SP not hired for a job	0.648	0.67
CG or SP not able to work because of child’s specific treatment regimen	0.495	0.529
CG or SP not able to restrict number of hours	0.34	0.291
CG or SP not able to work flexible hours	0.416	0.37
Impact on functional and recreational activities
Increasing limitation in child’s functional abilities and engagement in activities	0.394	0.441
Increasing number of days child missed school/work because of upper extremity problems in prior 6 months^c	0.431	0.476
Increasing negative impact on your child engaging in recreational activities	0.131^d	0.182
Increasing treatment adjustment to allow for recreational activities	0.326	0.369
Impact on family and relationships
Negative impact of hemophilia on your other children	0.173^d	0.221^d
CG negative experiences telling someone that your child has hemophilia	0.399	0.465
Child had a negative reaction telling someone he/she has hemophilia	0.557	0.611
Child bullied as a result of having hemophilia	0.522	0.59
Psychological/stress
Received psychological treatment in the past 5 years	0.706	0.721
Psychological treatment related to hemophilia	0.418	0.459
Experienced stressful events
Loss of job	0.755	0.783
Financial problems	0.36	0.288
Any other stressful event	0.658	0.627
Access to factor/treatment
Difficulty obtaining or concerns about factor products’ availability or affordability
Last 5 years	0.468	0.559
Next 5 years	0.259	0.297
Access to HTC
Fewer visits to HTC in the past year	0.398	0.343
Increasing difficulty for child to visit the HTC	0.303	0.305

Notes: ^aRange, 0–27 (higher scores indicate more severe depression). ^bRange, 0–21 (higher scores indicate more severe anxiety). ^cImpact of lower extremity problems was considered independently of upper extremity problems. ^dNot significant. Correlations highlighted in bold are considered high (r≥0.37).

Abbreviations: CG, caregiver; GAD-7, Generalized Anxiety Disorder 7-item [scale]; HTC, hemophilia treatment center; PHQ-9, Patient Health Questionnaire; SP, spouse/partner.