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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(s) -
Vepsäläinen K.,
Riikonen P.,
Lassila R.,
Arola M.,
Huttunen P.,
Lähteenmäki P.,
Möttönen M.,
Selander T.,
Martikainen J.
Publication year - 2018
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/hae.13447
Subject(s) - medicine , pediatrics , haemophilia , haemophilia a , medical record , surgery
Aim For previously untreated patients ( PUP s) 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 PUP s from birth to adolescence, including immune tolerance induction ( ITI ). Methods From the medical records of all PUP s 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. Results All 62 patients received early regular prophylaxis; totally, they have had treatment for nearly 700 patient‐years. The median age of starting home treatment was 1.1 years. The mean ( SD ) annual treatment costs (€ per kg) were 4391€ (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 ITI s were successful. The mean costs for ITI were 383 448€ (259 085). The expected ITI payback period was 1.81 (95% CI 0.62‐12.12) years. Conclusions Early high‐dose prophylaxis leads to excellent long‐term clinical outcomes, and early childhood ITI therapy seems to turn cost‐neutral generally already in 2 years.