Validation of a new instrument to measure disease‐related distress among patients with haemophilia

Abstract

Introduction In patients with haemophilia, general psychological distress as measured by the National Comprehensive Cancer Network (NCCN) distress thermometer has been associated with pain, disability and increased healthcare utilization. Aims To develop and validate a measure of haemophilia‐related distress. Methods After qualitative interviews, the Hemophilia‐Related Distress Questionnaire (HRDq) was developed. To validate the HRDq, adults (≥18 years) with haemophilia were enrolled, reported demographic and clinical information, and completed the HRDq and other questionnaires that measured similar constructs. Analysis included factor analysis and assessment of internal consistency using Cronbach's α, convergent validity using Pearson's correlation coefficient, and discriminant validity by comparing subgroups of patients. Test‐retest reliability was assessed using an intraclass correlation coefficient (ICC). Results Among 130 enrolled participants, 126 (median age=32.7 years) completed the 24 item HRDq in a median time of 5.4 minutes with overall HRDq scores ranging from 2 to 83 (median score=31.5; higher scores indicating higher distress). Assessment of convergent validity demonstrated a strong correlation (ρ>.60) of the HRDq total score with the NCCN Distress Thermometer, Haem‐A‐QoL total Score, and PROMIS‐29 Profile social role domain and a mild to moderate correlation with all other questionnaire domains (.3‐.59, p < .05). Distress was higher among those who had less education, were not employed, and were disabled and was not significantly different among those with severe compared with non‐severe disease. Assessment of test‐retest reliability demonstrated an ICC value of .84 (95% CI .71‐.91) for the total score. Conclusions The HRDq demonstrates good internal consistency, construct and discriminant validity, and retest reliability with a low responder burden.

Generic PROMIS item banks in adults with hemophilia for patient‐reported outcome assessment: Feasibility, measurement properties, and relevance

“…[15][16][17][18] However, PROMIS item banks have not yet been validated in adults with hemophilia and were to date seldom applied in hemophilia research projects. [19][20][21] Therefore, this study aimed to evaluate (i) feasibility, (ii) measurement properties, and (iii) relevance of nine PROMIS CATs and short forms for Dutch adult patients with hemophilia. We hypothesized that PROMIS CATs and short forms Results: Overall, 142 of 373 invited patients (mean age, 47 [range, 18-79], 49% severe hemophilia, 46% receiving prophylaxis) responded.…”

“…A large set of PROMIS item banks has been translated and validated in the Dutch general population and several patient populations 15‐18 . However, PROMIS item banks have not yet been validated in adults with hemophilia and were to date seldom applied in hemophilia research projects 19‐21 . Therefore, this study aimed to evaluate (i) feasibility, (ii) measurement properties, and (iii) relevance of nine PROMIS CATs and short forms for Dutch adult patients with hemophilia.…”

Cross‐sectional study evaluating the association of haemophilia‐related distress and clinically relevant outcomes

“…Domain scores can range from 0 to 35 for domains 1, 3 and 4; domain 2 scores range from 0 to 15. Higher scores overall and within each domain indicate increased haemophilia‐related distress 7 …”

“…Target joint bleeding may vary over time depending on the quality of the prescribed treatment regimen and the patient's adherence to the treatment regimen. Pain was previously shown to be associated with haemophilia‐related distress 7 . Based on the cross‐sectional design, it is not possible to determine whether pain leads to increased haemophilia‐related distress or haemophilia‐related distress contributes to increased pain.…”

“…Pain was previously shown to be associated with haemophilia-related distress. 7 Based on the cross-sectional design, it is not possible to determine whether pain leads to increased haemophilia-related distress or haemophilia-related distress contributes to increased pain. Future prospective studies will be needed to sort out the relationship.…”