Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy
Jennifer Quinn,1 Kathleen A Delaney,2 Wing Yen Wong,2 Wolfgang Miesbach,3 Monika Bullinger4 1BioMarin Pharmaceuticals UK Ltd, London, UK; 2BioMarin Pharmaceutical Inc., Novato, CA, USA; 3Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt, Germany; 4Departme...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
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Dove Medical Press
2022-07-01
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| Series: | Patient Related Outcome Measures |
| Subjects: | |
| Online Access: | https://www.dovepress.com/psychometric-validation-of-the-haemo-qol-a-in-participants-with-hemoph-peer-reviewed-fulltext-article-PROM |
| _version_ | 1818509166186070016 |
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| author | Quinn J Delaney KA Wong WY Miesbach W Bullinger M |
| author_facet | Quinn J Delaney KA Wong WY Miesbach W Bullinger M |
| author_sort | Quinn J |
| collection | DOAJ |
| description | Jennifer Quinn,1 Kathleen A Delaney,2 Wing Yen Wong,2 Wolfgang Miesbach,3 Monika Bullinger4 1BioMarin Pharmaceuticals UK Ltd, London, UK; 2BioMarin Pharmaceutical Inc., Novato, CA, USA; 3Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt, Germany; 4Department of Medical Psychology, University Medical Center Hamburg Eppendorf, Hamburg, GermanyCorrespondence: Jennifer Quinn, BioMarin Pharmaceuticals UK Ltd, 10 Bloomsbury Way, London, WC1A 2SL, UK, Tel +44 7976 129 039, Email jennifer.quinn@bmrn.comPurpose: The hemophilia-specific health-related quality of life (HRQOL) questionnaire (Haemo-QOL-A) is validated for detecting QOL changes following standard therapy for hemophilia A, but has not been rigorously evaluated after gene therapy. This post hoc analysis evaluated the psychometric properties of Haemo-QOL-A in adult people with severe hemophilia A (PWSHA) receiving valoctocogene roxaparvovec (AAV5-hFVIII-SQ) in 2 clinical trials (phase 1/2, NCT02576795; phase 3, NCT03370913).Patients and Methods: Adult PWSHA (factor VIII levels ≤ 1 IU/dL) received 1 AAV5-hFVIII-SQ infusion (6× 1013 vg/kg). Participants were assessed using the Haemo-QOL-A and the EuroQOL (EQ)-5D-5L and visual analog scale (VAS) questionnaires pre- and post-infusion. Psychometric analyses included convergent and discriminant validity, internal consistency, and reliability. Clinically important difference (CID) was estimated using 3-point change in EQ-5D-5L VAS as anchor.Results: Haemo-QOL-A data were analyzed from 7 (phase 1/2, 3-year follow-up) and 16 participants (phase 3, 26-week analysis). Change in Haemo-QOL-A Total Scores correlated with EQ-5D-5L VAS score change at 26 weeks (Pearson’s correlation 0.77). At 26 weeks, increased Haemo-QOL-A Physical Functioning was associated with decreased EQ-5D-5L Pain and Discomfort and decreased Anxiety and Depression (Spearman’s Rank correlations − 0.73 and − 0.62, respectively, P < 0.01). Internal consistency analysis showed good reliability for all domains (Cronbach’s alpha > 0.7) except Treatment Concern (Cronbach’s alpha = 0.31). Anchor-based CID estimates were met for Haemo-QOL-A Total Score (≥ 5.5) and domain scores (≥ 6) for Consequences of Bleeding, Physical Functioning, Role Functioning, and Worry.Conclusion: Our preliminary results suggest that the Haemo‐QOL‐A is a valid, reliable instrument for HRQOL assessment in PWSHA undergoing gene therapy. Future research should be undertaken to confirm these findings in a larger number of participants.Keywords: severe hemophilia A, gene therapy, quality of life, Haemo-QOL-A, psychometric testing, clinically important difference |
| first_indexed | 2024-12-10T22:41:46Z |
| format | Article |
| id | doaj.art-e442d1c5190c4fcbad82bf5c332307eb |
| institution | Directory Open Access Journal |
| issn | 1179-271X |
| language | English |
| last_indexed | 2024-12-10T22:41:46Z |
| publishDate | 2022-07-01 |
| publisher | Dove Medical Press |
| record_format | Article |
| series | Patient Related Outcome Measures |
| spelling | doaj.art-e442d1c5190c4fcbad82bf5c332307eb2022-12-22T01:30:41ZengDove Medical PressPatient Related Outcome Measures1179-271X2022-07-01Volume 1316918076652Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene TherapyQuinn JDelaney KAWong WYMiesbach WBullinger MJennifer Quinn,1 Kathleen A Delaney,2 Wing Yen Wong,2 Wolfgang Miesbach,3 Monika Bullinger4 1BioMarin Pharmaceuticals UK Ltd, London, UK; 2BioMarin Pharmaceutical Inc., Novato, CA, USA; 3Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt, Germany; 4Department of Medical Psychology, University Medical Center Hamburg Eppendorf, Hamburg, GermanyCorrespondence: Jennifer Quinn, BioMarin Pharmaceuticals UK Ltd, 10 Bloomsbury Way, London, WC1A 2SL, UK, Tel +44 7976 129 039, Email jennifer.quinn@bmrn.comPurpose: The hemophilia-specific health-related quality of life (HRQOL) questionnaire (Haemo-QOL-A) is validated for detecting QOL changes following standard therapy for hemophilia A, but has not been rigorously evaluated after gene therapy. This post hoc analysis evaluated the psychometric properties of Haemo-QOL-A in adult people with severe hemophilia A (PWSHA) receiving valoctocogene roxaparvovec (AAV5-hFVIII-SQ) in 2 clinical trials (phase 1/2, NCT02576795; phase 3, NCT03370913).Patients and Methods: Adult PWSHA (factor VIII levels ≤ 1 IU/dL) received 1 AAV5-hFVIII-SQ infusion (6× 1013 vg/kg). Participants were assessed using the Haemo-QOL-A and the EuroQOL (EQ)-5D-5L and visual analog scale (VAS) questionnaires pre- and post-infusion. Psychometric analyses included convergent and discriminant validity, internal consistency, and reliability. Clinically important difference (CID) was estimated using 3-point change in EQ-5D-5L VAS as anchor.Results: Haemo-QOL-A data were analyzed from 7 (phase 1/2, 3-year follow-up) and 16 participants (phase 3, 26-week analysis). Change in Haemo-QOL-A Total Scores correlated with EQ-5D-5L VAS score change at 26 weeks (Pearson’s correlation 0.77). At 26 weeks, increased Haemo-QOL-A Physical Functioning was associated with decreased EQ-5D-5L Pain and Discomfort and decreased Anxiety and Depression (Spearman’s Rank correlations − 0.73 and − 0.62, respectively, P < 0.01). Internal consistency analysis showed good reliability for all domains (Cronbach’s alpha > 0.7) except Treatment Concern (Cronbach’s alpha = 0.31). Anchor-based CID estimates were met for Haemo-QOL-A Total Score (≥ 5.5) and domain scores (≥ 6) for Consequences of Bleeding, Physical Functioning, Role Functioning, and Worry.Conclusion: Our preliminary results suggest that the Haemo‐QOL‐A is a valid, reliable instrument for HRQOL assessment in PWSHA undergoing gene therapy. Future research should be undertaken to confirm these findings in a larger number of participants.Keywords: severe hemophilia A, gene therapy, quality of life, Haemo-QOL-A, psychometric testing, clinically important differencehttps://www.dovepress.com/psychometric-validation-of-the-haemo-qol-a-in-participants-with-hemoph-peer-reviewed-fulltext-article-PROMsevere hemophilia agene therapyquality of lifehaemo-qol-apsychometric testingclinically important difference |
| spellingShingle | Quinn J Delaney KA Wong WY Miesbach W Bullinger M Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy Patient Related Outcome Measures severe hemophilia a gene therapy quality of life haemo-qol-a psychometric testing clinically important difference |
| title | Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy |
| title_full | Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy |
| title_fullStr | Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy |
| title_full_unstemmed | Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy |
| title_short | Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy |
| title_sort | psychometric validation of the haemo qol a in participants with hemophilia a treated with gene therapy |
| topic | severe hemophilia a gene therapy quality of life haemo-qol-a psychometric testing clinically important difference |
| url | https://www.dovepress.com/psychometric-validation-of-the-haemo-qol-a-in-participants-with-hemoph-peer-reviewed-fulltext-article-PROM |
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