Long‐term joint outcomes in adolescents with moderate or severe haemophilia A

• Published in: Haemophilia : the official journal of the World Federation of Hemophilia 2022-11, Vol.28 (6), p.1054-1061
• Main Authors: Schmidt, David E., Michalopoulou, Aikaterini, Fischer, Kathelijn, Motwani, Jayashree, Andersson, Nadine G., Pergantou, Helen, Ranta, Susanna
• Format: Article
• Language: English
• Subjects: Adolescent; Adolescents; Adult; Ankle; Ankle - diagnostic imaging; arthropathy; Cartilage; Children; Clinical Medicine; Cross-Sectional Studies; haemophilia A; Hemarthrosis - complications; Hematologi; Hematology; Hemophilia; Hemophilia A - complications; Humans; Joint Diseases - complications; Joint Diseases - etiology; Klinisk medicin; Medical and Health Sciences; Medicin och hälsovetenskap; Original; paediatrics; Patients; Teenagers; Ultrasonic imaging; Ultrasonography; Ultrasound
• ISSN: 1351-8216; 1365-2516
• DOI: 10.1111/hae.14636

Introduction Favourable joint outcomes are expected with modern haemophilia A (HA) management. Evaluation of long‐term treatment outcomes is hampered by the delay between bleeding episodes during childhood and resulting joint outcomes in adulthood. Aim To measure the long‐term joint health of adolescents with moderate and severe HA, according to severity and inhibitor status. Methods Pilot cross‐sectional study of five European PedNet centres in moderate and severe HA patients aged 10–19 years. Structured assessment of joint status by physical examination (HJHS) and ultrasound (HEAD‐US). Results In total, 141 HA patients were evaluable, 100 without inhibitors (81 severe, 19 moderate HA), and 41 severe HA with current/past inhibitors. On physical examination, 12/81 (15%) of severe HA without inhibitors, 3/19 (16%) of moderate HA, and 13/41 (32%) of severe HA patients with inhibitors exhibited joint abnormalities. Inhibitor persistence, longer inhibitor duration, and a high peak inhibitor level were associated with impaired joint health. Ultrasound showed joint damage (bone or cartilage) in 13/49 (27%) of severe HA without inhibitors, 1/12 (8%) of moderate HA, and 10/28 (36%) of severe HA patients with inhibitors. A discordant ankle evaluation by ultrasound versus physical examination was present in 53/169 joints (31%). Conclusions Most adolescents with severe or moderate HA show favourable joint health. Future research with combined ultrasound and/or MRI is needed to better understand joint outcomes in the remaining patients. Patents with inhibitors showed a two‐fold increased proportion with joint deterioration. Ultrasound paired with physical examination increases sensitivity for detection of joint damage.