PMID- 33576546
OWN - NLM
STAT- MEDLINE
DCOM- 20210924
LR  - 20210924
IS  - 1365-2516 (Electronic)
IS  - 1351-8216 (Linking)
VI  - 27
IP  - 3
DP  - 2021 May
TI  - Health-related quality of life and health status in adolescent and adult people 
      with haemophilia A without factor VIII inhibitors-A non-interventional study.
PG  - 398-407
LID - 10.1111/hae.14270 [doi]
AB  - INTRODUCTION: Real-world data on health-related outcomes in persons with 
      haemophilia A (PwHA) can provide useful information for improving patient care. 
      The global, non-interventional study (NIS; NCT02476942) prospectively collected 
      high-quality data in PwHA, including those without factor VIII (FVIII) inhibitors 
      treated according to local routine clinical practice. AIM: To report 
      health-related quality of life (HRQoL) and health status of adult/adolescent PwHA 
      without FVIII inhibitors. METHODS: Participants were PwHA without FVIII 
      inhibitors age >/=12 years; they remained on existing episodic treatment or 
      prophylaxis. HRQoL was assessed by Haemophilia Quality of Life Questionnaire for 
      Adults (Haem-A-QoL) or Haemophilia-Specific Quality of Life Assessment for 
      Children and Adolescents Short Form (Haemo-QoL-SF II). Health status was assessed 
      through EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) index utility score and visual 
      analogue scale (EQ-VAS). RESULTS: Ninety-four participants enrolled; median age 
      was 34.0 years (range 12-76). Forty-five received episodic treatment and 49 
      received prophylaxis for a median time of 27.7 weeks and 30.4 weeks, 
      respectively. Mean (standard deviation) baseline Haem-A-QoL total scores were 
      40.1 (17.0) for the episodic group and 26.6 (14.6) for the prophylaxis group, 
      indicating impairments in HRQoL, which remained consistent over time. Mean 
      EQ-5D-5L IUS scores were similar between treatment regimens (0.8 episodic; 0.9 
      prophylaxis) and consistent over time. The mean EQ-VAS scores were similar 
      between treatment regimens, and lower on days when bleeding occurred (79.0 vs 
      85.0 for episodic treatment; 77.0 vs 82.0 for prophylaxis, respectively). 
      CONCLUSIONS: Adult and adolescent PwHA without FVIII inhibitors had HRQoL 
      impairments regardless of whether they were treated with episodic or prophylactic 
      standard care with FVIII.
CI  - (c) 2021 The Authors. Haemophilia published by John Wiley & Sons Ltd.
FAU - Oldenburg, Johannes
AU  - Oldenburg J
AUID- ORCID: 0000-0002-1585-4100
AD  - Department of Experimental Haematology and Transfusion Medicine, University 
      Clinic Bonn, Bonn, Germany.
FAU - Tran, Huyen
AU  - Tran H
AD  - The Alfred Hospital, Melbourne, Vic., Australia.
FAU - Peyvandi, Flora
AU  - Peyvandi F
AUID- ORCID: 0000-0001-7423-9864
AD  - Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Fondazione Ca, 
      Granda Ospedale Maggiore Policlinico, Milan, Italy.
AD  - Department of Pathophysiology and Transplantation, University of Milan, Milan, 
      Italy.
FAU - Nunez, Ramiro
AU  - Nunez R
AD  - Hospital Universitario Virgen Del Rocio, Seville, Spain.
FAU - Trask, Peter
AU  - Trask P
AD  - Genentech, Inc., South San Francisco, CA, USA.
FAU - Chebon, Sammy
AU  - Chebon S
AD  - F. Hoffmann-La Roche Ltd, Basel, Switzerland.
FAU - Mahlangu, Johnny N
AU  - Mahlangu JN
AUID- ORCID: 0000-0001-5781-7669
AD  - Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of 
      the Witwatersrand and NHLS, Johannesburg, South Africa.
FAU - Lehle, Michaela
AU  - Lehle M
AD  - F. Hoffmann-La Roche Ltd, Basel, Switzerland.
FAU - Jimenez-Yuste, Victor
AU  - Jimenez-Yuste V
AUID- ORCID: 0000-0003-3937-3499
AD  - La Paz University Hospital, Autonoma University, Madrid, Spain.
FAU - von Mackensen, Sylvia
AU  - von Mackensen S
AUID- ORCID: 0000-0002-5926-0478
AD  - Department of Medical Psychology, University Medical Centre Hamburg-Eppendorf, 
      Hamburg, Germany.
LA  - eng
GR  - F. Hoffmann-La Roche Ltd/
GR  - Chugai Pharmaceutical Co., Ltd/
PT  - Journal Article
DEP - 20210212
PL  - England
TA  - Haemophilia
JT  - Haemophilia : the official journal of the World Federation of Hemophilia
JID - 9442916
RN  - 9001-27-8 (Factor VIII)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - Child
MH  - Factor VIII/therapeutic use
MH  - Health Status
MH  - *Hemophilia A/drug therapy
MH  - Humans
MH  - Infant, Newborn
MH  - Middle Aged
MH  - *Quality of Life
MH  - Surveys and Questionnaires
MH  - Young Adult
OTO - NOTNLM
OT  - factor VIII
OT  - haemophilia A
OT  - health-related quality of life
OT  - inhibitors
OT  - non-interventional study
OT  - real-world data
EDAT- 2021/02/13 06:00
MHDA- 2021/09/25 06:00
CRDT- 2021/02/12 08:41
PHST- 2021/01/20 00:00 [revised]
PHST- 2020/08/07 00:00 [received]
PHST- 2021/01/21 00:00 [accepted]
PHST- 2021/02/13 06:00 [pubmed]
PHST- 2021/09/25 06:00 [medline]
PHST- 2021/02/12 08:41 [entrez]
AID - 10.1111/hae.14270 [doi]
PST - ppublish
SO  - Haemophilia. 2021 May;27(3):398-407. doi: 10.1111/hae.14270. Epub 2021 Feb 12.