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Early prophylaxis in children with severe haemophilia A: clinical and ultrasound imaging outcomes
Author(s) -
Altisent C.,
Martorell M.,
Crespo A.,
Casas L.,
Torrents C.,
Parra R.
Publication year - 2016
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.12792
Subject(s) - medicine , haemophilia , ultrasound , ankle , arthropathy , haemophilia a , cohort , surgery , osteoarthritis , radiology , alternative medicine , pathology
Aim This observational study was undertaken with the aim to describe the characteristics and evaluate the outcomes of prophylactic treatment in children with severe haemophilia A ( HA ) treated at our centre. Methods Twenty‐five patients aged 4–19 years with severe HA , no history of inhibitors and treated with at least two infusions of factor VIII (FVIII) per week were studied. Prophylactic doses and annual joint bleeding rate ( AJBR ) were retrospectively evaluated over the last 5 years. Current joint status was assessed using the Haemophilia Joint Health Score ( HJHS ) (136 joints of 23 patients) and the Haemophilia Early Arthropathy Detection with Ultrasound ( HEAD ‐ US ) procedure (124 joints of 21 patients). Results Median AJBR was 0.2 and median prophylaxis dose 65.4 IU −1 kg −1 week −1 . Median total HJHS was 0 (range 0–13) and total HEAD ‐ US 1 (0–8). At the joint level, 85.3% of joints were normal on HJHS and 79.0% on US . The ankle was the joint most commonly affected, considering bleeding and ultrasound results. Correlation was found between HEAD ‐ US scores and bleeding scores but not between HEAD ‐ US and HJHS scores. HJHS and HEAD ‐ US scores were concordant in 91/124 (73.4%) joints (86 joints normal and five abnormal). Ultrasound detected minimal changes in 19.6% of joints with normal physical function, whereas 12.2% of joints considered normal on ultrasound showed changes at HJHS . Conclusion A well‐preserved joint status was found in our cohort. High‐resolution US detected a higher percentage of abnormalities than the physical evaluation, but the clinical implications of these findings still need to be ascertained.