Long-term clinical and economic outcomes in previously untreated paediatric patients with severe haemophilia A : A nationwide real-world study with 700 person-years

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Vepsalainen , K , Riikonen , P , Lassila , R , Arola , M , Huttunen , P , Lähteenmäki , P , Mottonen , M , Selander , T & Martikainen , J 2018 , ' Long-term clinical and economic outcomes in previously untreated paediatric patients with severe haemophilia A : A nationwide real-world study with 700 person-years ' , Haemophilia , vol. 24 , no. 3 , pp. 436-444 . https://doi.org/10.1111/hae.13447

Title: Long-term clinical and economic outcomes in previously untreated paediatric patients with severe haemophilia A : A nationwide real-world study with 700 person-years
Author: Vepsalainen, K.; Riikonen, P.; Lassila, R.; Arola, M.; Huttunen, P.; Lähteenmäki, P.; Mottonen, M.; Selander, T.; Martikainen, J.
Contributor: University of Helsinki, Clinicum
University of Helsinki, Children's Hospital
Date: 2018-05
Language: eng
Number of pages: 9
Belongs to series: Haemophilia
ISSN: 1351-8216
URI: http://hdl.handle.net/10138/304250
Abstract: AimFor previously untreated patients (PUPs) with severe haemophilia A in Finland for the past 2 decades, the standard practice has been to start early primary prophylaxis. We evaluated the long-term clinical outcomes and costs of treatment with high-dose prophylaxis in PUPs from birth to adolescence, including immune tolerance induction (ITI). MethodsFrom the medical records of all PUPs born between June 1994 and May 2013 in Finland, we retrospectively extracted data on clinical outcomes and healthcare use. Using linear mixed models, we analysed longitudinal clinical outcome data. To analyse skewed cost data, including zero costs, we applied hurdle regression. ResultsAll 62 patients received early regular prophylaxis; totally, they have had treatment for nearly 700 patient-years. The median age of starting home treatment was 1.1years. The mean (SD) annual treatment costs (Europerkg) were 4391Euro (3852). For ages 1-3, ITI comprised over half of the costs; in other groups, prophylactic FVIII treatment dominated. With these high costs, however, clinical outcomes were desirable; median (IQR) ABR was low at 0.19 (0.07-0.46) and so was AJBR at 0.06 (0-0.24). Thirteen (21%) patients developed a clinically significant inhibitor, 10 (16%) with a high titre. All ITIs were successful. The mean costs for ITI were 383448Euro (259085). The expected ITI payback period was 1.81 (95% CI 0.62-12.12) years. ConclusionsEarly high-dose prophylaxis leads to excellent long-term clinical outcomes, and early childhood ITI therapy seems to turn cost-neutral generally already in 2years.
Subject: ABJR
ABR
bleed
costs
haemophilia A
health economics
outcome
prophylaxis
PUP
IMMUNE TOLERANCE INDUCTION
PROPHYLACTIC TREATMENT
ON-DEMAND
INHIBITOR DEVELOPMENT
EPISODIC TREATMENT
CHILDREN
COMMUNICATION
DEFINITIONS
MULTICENTER
EXPERIENCE
3121 Internal medicine
3123 Gynaecology and paediatrics
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