Health-related quality of life and health status in persons with haemophilia A with inhibitors

A prospective, multicentre, non-interventional study (NIS)

Johnny Mahlangu, Johannes Oldenburg, Michael U. Callaghan, Midori Shima, Maria Elisa Mancuso, Peter Trask, Michael Recht, Claudia Garcia, Renchi Yang, Michaela Lehle, Harrison Macharia, Elina Asikanius, Gallia G. Levy, Rebecca Kruse-Jarres, Sylvia von Mackensen

Research output: Contribution to journalArticle

Abstract

Introduction: Real-world data (RWD) on health-related outcomes in persons with haemophilia A (PwHA) provide insights into patient needs and can guide clinical study design. A global, prospective, non-interventional study (NIS; NCT02476942) collected detailed RWD on bleeding outcomes, health-related quality of life (HRQoL) and health status in PwHA treated per local routine clinical practice. Aim: To report HRQoL and health status in the adult/adolescent PwHA with inhibitors cohort in the NIS. Methods: This cohort enrolled PwHA aged ≥12 years with high-titre factor VIII inhibitor history. Participants remained on their usual treatment (no protocol-specified interventions). Health-related outcomes: Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL), Haemophilia-specific Quality of Life Questionnaire for Children Short Form (Haemo-QoL SF), EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) index utility score (IUS) and visual analogue scale (EQ-VAS). Results: One hundred three participants were enrolled on episodic (n = 75) or prophylactic treatment (n = 28); median (range) age, 31 (12-75) years; median (range) observation time, 26 (4-70) weeks. Haem-A-QoL scores indicated impairments in HRQoL aspects; comparable between episodic/prophylactic regimens and relatively consistent over time. Haemo-QoL SF scores with both regimens varied over time, and appeared poorer with episodic than prophylactic treatment. IUS and EQ-VAS were comparable between regimens, stable over time and lower on bleeding days. Mean proportions of missed work and school days were 16% and 23%, respectively; mean (standard deviation) number of days hospitalized was 3.2 (8.8) (comparable between groups). Conclusions: These RWD demonstrate that PwHA with inhibitors have impaired HRQoL, despite standard treatment, and that more effective treatment options are needed.

Original languageEnglish (US)
JournalHaemophilia
DOIs
StatePublished - Jan 1 2019

Fingerprint

Hemophilia A
Health Status
Quality of Life
Heme
Hemorrhage
Health
Factor VIII
Therapeutics
Clinical Protocols
Visual Analog Scale
History
Observation

Keywords

  • alloantibodies
  • haemophilia
  • health-related quality of life
  • inhibitors
  • non-interventional

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

Health-related quality of life and health status in persons with haemophilia A with inhibitors : A prospective, multicentre, non-interventional study (NIS). / Mahlangu, Johnny; Oldenburg, Johannes; Callaghan, Michael U.; Shima, Midori; Mancuso, Maria Elisa; Trask, Peter; Recht, Michael; Garcia, Claudia; Yang, Renchi; Lehle, Michaela; Macharia, Harrison; Asikanius, Elina; Levy, Gallia G.; Kruse-Jarres, Rebecca; von Mackensen, Sylvia.

In: Haemophilia, 01.01.2019.

Research output: Contribution to journalArticle

Mahlangu, J, Oldenburg, J, Callaghan, MU, Shima, M, Mancuso, ME, Trask, P, Recht, M, Garcia, C, Yang, R, Lehle, M, Macharia, H, Asikanius, E, Levy, GG, Kruse-Jarres, R & von Mackensen, S 2019, 'Health-related quality of life and health status in persons with haemophilia A with inhibitors: A prospective, multicentre, non-interventional study (NIS)', Haemophilia. https://doi.org/10.1111/hae.13731
Mahlangu, Johnny ; Oldenburg, Johannes ; Callaghan, Michael U. ; Shima, Midori ; Mancuso, Maria Elisa ; Trask, Peter ; Recht, Michael ; Garcia, Claudia ; Yang, Renchi ; Lehle, Michaela ; Macharia, Harrison ; Asikanius, Elina ; Levy, Gallia G. ; Kruse-Jarres, Rebecca ; von Mackensen, Sylvia. / Health-related quality of life and health status in persons with haemophilia A with inhibitors : A prospective, multicentre, non-interventional study (NIS). In: Haemophilia. 2019.
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abstract = "Introduction: Real-world data (RWD) on health-related outcomes in persons with haemophilia A (PwHA) provide insights into patient needs and can guide clinical study design. A global, prospective, non-interventional study (NIS; NCT02476942) collected detailed RWD on bleeding outcomes, health-related quality of life (HRQoL) and health status in PwHA treated per local routine clinical practice. Aim: To report HRQoL and health status in the adult/adolescent PwHA with inhibitors cohort in the NIS. Methods: This cohort enrolled PwHA aged ≥12 years with high-titre factor VIII inhibitor history. Participants remained on their usual treatment (no protocol-specified interventions). Health-related outcomes: Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL), Haemophilia-specific Quality of Life Questionnaire for Children Short Form (Haemo-QoL SF), EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) index utility score (IUS) and visual analogue scale (EQ-VAS). Results: One hundred three participants were enrolled on episodic (n = 75) or prophylactic treatment (n = 28); median (range) age, 31 (12-75) years; median (range) observation time, 26 (4-70) weeks. Haem-A-QoL scores indicated impairments in HRQoL aspects; comparable between episodic/prophylactic regimens and relatively consistent over time. Haemo-QoL SF scores with both regimens varied over time, and appeared poorer with episodic than prophylactic treatment. IUS and EQ-VAS were comparable between regimens, stable over time and lower on bleeding days. Mean proportions of missed work and school days were 16{\%} and 23{\%}, respectively; mean (standard deviation) number of days hospitalized was 3.2 (8.8) (comparable between groups). Conclusions: These RWD demonstrate that PwHA with inhibitors have impaired HRQoL, despite standard treatment, and that more effective treatment options are needed.",
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T1 - Health-related quality of life and health status in persons with haemophilia A with inhibitors

T2 - A prospective, multicentre, non-interventional study (NIS)

AU - Mahlangu, Johnny

AU - Oldenburg, Johannes

AU - Callaghan, Michael U.

AU - Shima, Midori

AU - Mancuso, Maria Elisa

AU - Trask, Peter

AU - Recht, Michael

AU - Garcia, Claudia

AU - Yang, Renchi

AU - Lehle, Michaela

AU - Macharia, Harrison

AU - Asikanius, Elina

AU - Levy, Gallia G.

AU - Kruse-Jarres, Rebecca

AU - von Mackensen, Sylvia

PY - 2019/1/1

Y1 - 2019/1/1

N2 - Introduction: Real-world data (RWD) on health-related outcomes in persons with haemophilia A (PwHA) provide insights into patient needs and can guide clinical study design. A global, prospective, non-interventional study (NIS; NCT02476942) collected detailed RWD on bleeding outcomes, health-related quality of life (HRQoL) and health status in PwHA treated per local routine clinical practice. Aim: To report HRQoL and health status in the adult/adolescent PwHA with inhibitors cohort in the NIS. Methods: This cohort enrolled PwHA aged ≥12 years with high-titre factor VIII inhibitor history. Participants remained on their usual treatment (no protocol-specified interventions). Health-related outcomes: Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL), Haemophilia-specific Quality of Life Questionnaire for Children Short Form (Haemo-QoL SF), EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) index utility score (IUS) and visual analogue scale (EQ-VAS). Results: One hundred three participants were enrolled on episodic (n = 75) or prophylactic treatment (n = 28); median (range) age, 31 (12-75) years; median (range) observation time, 26 (4-70) weeks. Haem-A-QoL scores indicated impairments in HRQoL aspects; comparable between episodic/prophylactic regimens and relatively consistent over time. Haemo-QoL SF scores with both regimens varied over time, and appeared poorer with episodic than prophylactic treatment. IUS and EQ-VAS were comparable between regimens, stable over time and lower on bleeding days. Mean proportions of missed work and school days were 16% and 23%, respectively; mean (standard deviation) number of days hospitalized was 3.2 (8.8) (comparable between groups). Conclusions: These RWD demonstrate that PwHA with inhibitors have impaired HRQoL, despite standard treatment, and that more effective treatment options are needed.

AB - Introduction: Real-world data (RWD) on health-related outcomes in persons with haemophilia A (PwHA) provide insights into patient needs and can guide clinical study design. A global, prospective, non-interventional study (NIS; NCT02476942) collected detailed RWD on bleeding outcomes, health-related quality of life (HRQoL) and health status in PwHA treated per local routine clinical practice. Aim: To report HRQoL and health status in the adult/adolescent PwHA with inhibitors cohort in the NIS. Methods: This cohort enrolled PwHA aged ≥12 years with high-titre factor VIII inhibitor history. Participants remained on their usual treatment (no protocol-specified interventions). Health-related outcomes: Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL), Haemophilia-specific Quality of Life Questionnaire for Children Short Form (Haemo-QoL SF), EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) index utility score (IUS) and visual analogue scale (EQ-VAS). Results: One hundred three participants were enrolled on episodic (n = 75) or prophylactic treatment (n = 28); median (range) age, 31 (12-75) years; median (range) observation time, 26 (4-70) weeks. Haem-A-QoL scores indicated impairments in HRQoL aspects; comparable between episodic/prophylactic regimens and relatively consistent over time. Haemo-QoL SF scores with both regimens varied over time, and appeared poorer with episodic than prophylactic treatment. IUS and EQ-VAS were comparable between regimens, stable over time and lower on bleeding days. Mean proportions of missed work and school days were 16% and 23%, respectively; mean (standard deviation) number of days hospitalized was 3.2 (8.8) (comparable between groups). Conclusions: These RWD demonstrate that PwHA with inhibitors have impaired HRQoL, despite standard treatment, and that more effective treatment options are needed.

KW - alloantibodies

KW - haemophilia

KW - health-related quality of life

KW - inhibitors

KW - non-interventional

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