If this is enhanced to 10 IU kg−1 three times per week, this will then start reaching reductions in the ‘time at risk’ of ~60%. If paradigms could be changed completely and find practical and convenient ways found to administer CFC once a day then even with doses as low 5 IU kg−1 day−1 one could maintain >1% at all times with an annual dose well below 2000 IU kg−1. All the evidence suggests that any prophylaxis that reduces ABR should help improve long-term outcomes. After all, even in Western countries, prophylaxis had started with lower doses and they had already noted improvements in their patients before reaching current doses. There are

HDAC inhibitor also limited recent data that CFC doses as low as 10 IU kg−1 two to three times/week reduce joint bleeding in patients who previously received episodic replacement [36]. Such prophylaxis programmes need to be systematically initiated in different countries

[37]. Finally, everywhere in the world there is a need to assess outcomes with whatever replacement protocols that are followed. This has been a relatively ignored subject in the field around the world and needs to change, not only because modern medicine attempts to work on evidence but also because there is greater cost alertness from healthcare providers everywhere and high-cost diseases such as haemophilia are more likely to come under the scanner [10]. It is vital therefore that assessment of relevant outcomes with appropriate 上海皓元 tools becomes part of the care of PWH. This field has significantly advanced in the last 10 years as well JAK2 inhibitor drug [38]. Traditionally, the ABR into joints and other sites has been a simple and predictive indicator

of long-term outcome in haemophilia with regard to joint disease and overall musculoskeletal status. This continues to be used as a surrogate marker of both disease severity before intervention and an index of the efficacy of treatment provided. For standardizing bleeding assessment from other sites, tools have been developed in the past few years [39]. These have so far been used mainly to evaluate those conditions where bleeding is more skin and mucosal. There utility in haemophilia and related rarer bleeding disorders needs evaluation. The WFH has attempted to review and summarize the potential of the most relevant of these tools on a website to make them more easily accessible to the community for their use and comments. While data on ABRs are relatively easy to collect, there can be errors in a patient’s assessments and reporting. It is important therefore to always combine this with assessment of joints. The Hemophilia Joint Health Score is gradually replacing the WFH clinical score as a validated tool for the clinical assessment of joints [40, 41].