Severe haemophilia A children on low‐dose tertiary prophylaxis showed less joint deterioration and better maintenance of functional independence than children on on‐demand treatment: A 6‐year follow‐up study

• Published in: Haemophilia : the official journal of the World Federation of Hemophilia 2020-09, Vol.26 (5), p.779-785
• Main Authors: Liu, Ying, Chen, Lixia, Li, Kuixing, Shi, Mingnan, Poon, Man‐Chiu
• Format: Article
• Language: English
• Subjects: Bleeding; Children; functional independence score in hemophilia; haemophilia; Hemophilia; hemophilia joint health score; low‐dose prophylaxis; on‐demand treatment; Prophylaxis
• ISSN: 1351-8216; 1365-2516
• DOI: 10.1111/hae.14016

Background In countries with limited resource, haemophilia patients have to choose low‐dose prophylaxis or on‐demand treatment (ODT) because of economic constraints. Whether low‐dose prophylaxis can achieve better joint function outcome than ODT over long‐term remains unclear. Aim To investigate the long‐term effect of low‐dose tertiary prophylaxis versus ODT on joint health in severe haemophilia A children. Methods This was a retrospective study. We enrolled and followed 34 severe haemophilia boys in China receiving on‐demand treatment (n = 18) or low‐dose prophylaxis (10‐15 IU/kg, 2‐3 times per week) for a medium‐term (6‐18 months, n = 9) or longer‐term (19‐30 months, n = 7). We evaluated their haemophilia joint health score (HJHS) 2.1 and functional independence score in haemophilia (FISH) at baseline and at their 6‐year follow‐up visits. Their annual bleeding rate (ABR) and annual joint bleeding rate (AJBR) were also recorded. Results During the 6‐year follow‐up period, ABR and AJBR were similar between the 2 prophylaxis groups, with each of the 2 prophylaxis group rates being significantly better (lower) than the ODT group (P