PMID- 30724422
OWN - NLM
STAT- MEDLINE
DCOM- 20190429
LR  - 20190429
IS  - 1365-2516 (Electronic)
IS  - 1351-8216 (Linking)
VI  - 25
IP  - 2
DP  - 2019 Mar
TI  - Bleeding and safety outcomes in persons with haemophilia A without inhibitors: 
      Results from a prospective non-interventional study in a real-world setting.
PG  - 213-220
LID - 10.1111/hae.13655 [doi]
AB  - INTRODUCTION: Prospectively collected real-world data on bleeds, haemophilia 
      treatment and safety in persons with haemophilia A (PwHA) without factor VIII 
      (FVIII) inhibitors are limited. A global, non-interventional study (NIS; 
      NCT02476942) prospectively collected real-world data in PwHA who were treated per 
      local routine clinical practice. AIM: Assess annualized bleeding rate (ABR), 
      haemophilia treatment practices and adverse events (AEs) in adult/adolescent PwHA 
      without inhibitors. METHODS: Eligible participants aged >/=12 years with severe HA 
      without history of inhibitors prospectively collected bleeding and treatment 
      information. RESULTS: Ninety-four participants were enrolled (median [range] age, 
      34 [12-76] years) and monitored for a median (range) of 29.8 (12.4-47.7) weeks. 
      In the episodic (n = 45) and prophylactic (n = 49) treatment groups, 
      respectively, 872/1066 (81.8%) and 151/189 (79.9%), bleeds were treated; ABRs 
      (95% confidence interval) were 36.1 (30.8-42.3) and 5.0 (3.3-7.5), respectively, 
      for treated bleeds and 43.1 (36.5-50.9) and 6.2 (4.2-9.2), respectively, for all 
      bleeds, and median (interquartile range) ABRs were 31.1 (19.8-51.6) and 1.9 
      (0.0-8.2), respectively, for treated bleeds and 35.3 (21.7-62.9) and 2.7 
      (0.0-9.4), respectively, for all bleeds. Half of the participants on FVIII 
      prophylaxis had relatively high adherence to treatment, using 2.9 and 2.1 median 
      doses/wk of standard and extended half-life FVIII, respectively. Serious AEs 
      included gastrointestinal polyp haemorrhage and haemarthrosis; the most common AE 
      was viral upper respiratory tract infection. CONCLUSION: PwHA without inhibitors 
      continue to bleed on prophylaxis, consistent with the literature, and require 
      treatment for breakthrough bleeds. This prospective NIS demonstrates the need for 
      more efficacious haemostatic approaches.
CI  - (c) 2019 John Wiley & Sons Ltd.
FAU - Kruse-Jarres, Rebecca
AU  - Kruse-Jarres R
AUID- ORCID: 0000-0001-6547-5240
AD  - Washington Center for Bleeding Disorders at Bloodworks Northwest, Seattle, 
      Washington.
FAU - Oldenburg, Johannes
AU  - Oldenburg J
AUID- ORCID: 0000-0003-3038-2145
AD  - Universitatsklinikum Bonn, Bonn, Germany.
FAU - Santagostino, Elena
AU  - Santagostino E
AD  - Centro Emofilia e Trombosi A. Bianchi Bonomi, IRCCS Fondazione Ca' Granda, 
      Milano, Italy.
FAU - Shima, Midori
AU  - Shima M
AD  - Department of Pediatrics, Nara Medical University, Kashihara, Japan.
FAU - Kempton, Christine L
AU  - Kempton CL
AUID- ORCID: 0000-0002-2391-2016
AD  - Department of Hematology and Medical Oncology, Emory School of Medicine, Atlanta, 
      Georgia.
FAU - Kessler, Craig M
AU  - Kessler CM
AD  - Lombardi Comprehensive Cancer Center Georgetown University Medical Center, 
      Washington, District of Columbia.
FAU - Lehle, Michaela
AU  - Lehle M
AD  - F. Hoffmann-La Roche Ltd, Basel, Switzerland.
FAU - Chebon, Sammy
AU  - Chebon S
AD  - F. Hoffmann-La Roche Ltd, Basel, Switzerland.
FAU - Selak Bienz, Nives
AU  - Selak Bienz N
AD  - F. Hoffmann-La Roche Ltd, Basel, Switzerland.
FAU - Asikanius, Elina
AU  - Asikanius E
AD  - F. Hoffmann-La Roche Ltd, Basel, Switzerland.
FAU - Mahlangu, Johnny
AU  - Mahlangu J
AUID- ORCID: 0000-0001-5781-7669
AD  - Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of 
      the Witwatersrand and NHLS, Parktown, Johannesburg, South Africa.
LA  - eng
GR  - F. Hoffmann-La Roche Ltd/
PT  - Journal Article
DEP - 20190206
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/adverse effects/pharmacokinetics/*therapeutic use
MH  - Gastrointestinal Hemorrhage/etiology
MH  - Half-Life
MH  - Hemarthrosis/etiology
MH  - Hemophilia A/*diagnosis/drug therapy
MH  - Humans
MH  - Male
MH  - Medication Adherence
MH  - Middle Aged
MH  - Prospective Studies
MH  - Respiratory Tract Infections/etiology
MH  - Young Adult
OTO - NOTNLM
OT  - blood coagulation factor inhibitors
OT  - factor VIII
OT  - haemophilia A
OT  - non-interventional study
OT  - observational study
OT  - prospective study
EDAT- 2019/02/07 06:00
MHDA- 2019/04/30 06:00
CRDT- 2019/02/07 06:00
PHST- 2018/08/17 00:00 [received]
PHST- 2018/11/09 00:00 [accepted]
PHST- 2019/02/07 06:00 [pubmed]
PHST- 2019/04/30 06:00 [medline]
PHST- 2019/02/07 06:00 [entrez]
AID - 10.1111/hae.13655 [doi]
PST - ppublish
SO  - Haemophilia. 2019 Mar;25(2):213-220. doi: 10.1111/hae.13655. Epub 2019 Feb 6.