title: A prospective study of health-related quality of life of boys with severe haemophilia A in China: comparing on-demand to prophylaxis treatment.
abstract: BACKGROUND
  Treatment for boys with haemophilia in China is rapidly improving; however, comprehensive outcomes have not been examined prospectively.
  
  
  OBJECTIVE
  The aim of this study was to evaluate the effect of short-term full-dose prophylaxis compared to on-demand treatment, on the Health-Related Quality of Life (HR-QoL) of boys with severe haemophilia A (HA) in China.
  
  
  METHODS
  Boys with severe HA (FVIII<1%) completed 3 months of on-demand treatment and 3 months of full-dose prophylaxis (25 FVIII IU per kg 3x per week). The primary outcomes were child- and parent-reported Canadian Hemophilia Outcomes - Kids Life Assessment Tool (CHO-KLAT) scores. The number and type of bleeds and Activities Scale for Kids (ASK) scores were also recorded.
  
  
  RESULTS
  Analyses included 23 boys between 4 and 15.9 years of age. The number of bleeds decreased by 94% on prophylaxis (P < 0.0001, Wilcoxon Signed-Rank test). The mean child-reported CHO-KLAT scores for boys ≥7 years (n = 20) was 61.4 (±10.9) during on-demand treatment and 61.9 (±11.4) following short-term prophylaxis (P = 0.72, paired t-test). The mean parent-reported CHO-KLAT score during the on-demand phase was 54.4 (±10.5) with an increase of 3.8 points (±8.1; P = 0.04, paired t-test) following prophylaxis.
  
  
  CONCLUSIONS
  Child-reported CHO-KLAT scores were lower in boys with severe HA in China than reported in countries with access to full-dose prophylaxis. Boys reported higher HR-QoL scores than their parents. Small improvements in ASK scores were noted following the prophylaxis phase. These changes were only significant in the parent-reported CHO-KLAT scores. Longer term prospective clinical trials are needed in China to determine the impact of prophylaxis on HR-QoL in boys with severe HA.
authors.affiliation: Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, Beijing, China.|Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing, China.
authors.forename: R
authors.initials: R
authors.lastname: Wu
authors.affiliation: Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China.
authors.forename: J
authors.initials: J
authors.lastname: Sun
authors.affiliation: Department of Pediatrics, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing, China.
authors.forename: J
authors.initials: J
authors.lastname: Xiao
authors.affiliation: Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China.
authors.forename: Y
authors.initials: Y
authors.lastname: Liu
authors.affiliation: Thrombosis & Hemostasis center, Institute of Hematology, Blood Diseases Hospital Chinese Academy of Medical Sciences, Peking Union Medical College, Tianjin, China.
authors.forename: F
authors.initials: F
authors.lastname: Xue
authors.affiliation: Department of Hematology, Wuhan Union Medical College Hospital, Huazhong University of Science and Technology, Wuhan, China.
authors.forename: H
authors.initials: H
authors.lastname: Wang
authors.affiliation: Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, Beijing, China.|Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics, Ministry of Education, Beijing, China.
authors.forename: L
authors.initials: L
authors.lastname: Tang
authors.affiliation: Department of Hematology, Hemophilia clinic, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing, China.
authors.forename: Y
authors.initials: Y
authors.lastname: Zhao
authors.affiliation: Department of Hematology, Hemophilia clinic, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College, Beijing, China.
authors.forename: K
authors.initials: K
authors.lastname: Li
authors.affiliation: Thrombosis & Hemostasis center, Institute of Hematology, Blood Diseases Hospital Chinese Academy of Medical Sciences, Peking Union Medical College, Tianjin, China.
authors.forename: R
authors.initials: R
authors.lastname: Yang
authors.affiliation: Department of Hematology, Wuhan Union Medical College Hospital, Huazhong University of Science and Technology, Wuhan, China.
authors.forename: Y
authors.initials: Y
authors.lastname: Hu
authors.affiliation: Department of Pediatrics, Laboratory Medicine and Pathology, Division of Hematology and Oncology, Children Hospital of Eastern Ontario, University of Ottawa, Ottawa, ON, Canada.
authors.forename: K-H
authors.initials: KH
authors.lastname: Luke
authors.affiliation: Departments of Medicine, Pediatrics and Oncology, Foothills Hospital, Alberta Health Services, University of Calgary and Southern Alberta Rare Blood and Bleeding Disorders Comprehensive Care Program, Calgary, AB, Canada.
authors.forename: M-C
authors.initials: MC
authors.lastname: Poon
authors.affiliation: Division of Hematology/Oncology, The Hospital for Sick Children, Toronto, ON, Canada.|Department of Pediatrics, University of Toronto, Toronto, ON, Canada.
authors.forename: V S
authors.initials: VS
authors.lastname: Blanchette
authors.affiliation: School of Rural and Northern Health and the Research Centre for Evaluating Children's Health Outcomes (ECHO), Laurentian University, Sudbury, ON, Canada.
authors.forename: K
authors.initials: K
authors.lastname: Usuba
authors.affiliation: School of Rural and Northern Health and the Research Centre for Evaluating Children's Health Outcomes (ECHO), Laurentian University, Sudbury, ON, Canada.
authors.forename: N L
authors.identifier: http://orcid.org/0000-0002-1739-3299
authors.initials: NL
authors.lastname: Young
mesh_terms: D000293:Adolescent; D002648:Child; D002681:China; D004305:Dose-Response Relationship, Drug; D005169:Factor VIII / Q000494:pharmacology / Q000627:therapeutic use; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy* / Q000503:physiopathology / Q000517:prevention & control*; D006470:Hemorrhage / Q000150:complications; D006801:Humans; D008297:Male; D011446:Prospective Studies; D011788:Quality of Life; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D005169:Factor VIII
keywords: haemophilia; health-related quality of life; on-demand treatment; outcomes; paediatrics; prophylaxis