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Haemophilia. 2019 Feb 6. doi: 10.1111/hae.13655. [Epub ahead of print]

Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective non-interventional study in a real-world setting.

Author information

1
Washington Center for Bleeding Disorders at Bloodworks Northwest, Seattle, Washington.
2
Universitätsklinikum Bonn, Bonn, Germany.
3
Centro Emofilia e Trombosi A. Bianchi Bonomi, IRCCS Fondazione Ca' Granda, Milano, Italy.
4
Department of Pediatrics, Nara Medical University, Kashihara, Japan.
5
Department of Hematology and Medical Oncology, Emory School of Medicine, Atlanta, Georgia.
6
Lombardi Comprehensive Cancer Center Georgetown University Medical Center, Washington, District of Columbia.
7
F. Hoffmann-La Roche Ltd, Basel, Switzerland.
8
Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Parktown, Johannesburg, South Africa.

Abstract

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.

KEYWORDS:

blood coagulation factor inhibitors; factor VIII; haemophilia A; non-interventional study; observational study; prospective study

PMID:
30724422
DOI:
10.1111/hae.13655

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