The total quantity of CFC used in the developed countries has nea

The total quantity of CFC used in the developed countries has nearly doubled over the last decade with addition of new patients, better survival of older PWH, increasing intensity of doses and prophylaxis extending to adults as well as immune tolerance induction for those with inhibitors (Fig 1). However, this has not been matched with proper data on outcomes to show its full benefits. While observational data with prophylaxis collected over several decades at two centres (Malmo, Sweden and Utrecht, the Netherlands) has established its role in reducing bleeding

and maintaining near normal musculoskeletal status, it is important to recognize that the two worked with different philosophies – the former aiming to maintain >1% circulating factor level at Gamma-secretase inhibitor all times and the latter targeting the clinical avoidance Autophagy Compound Library clinical trial of bleeding. This resulted in the total doses at Malmo being nearly double those at Utrecht [3, 18]. Both centres have increased their total annual doses, with greater availability of CFC and evolving philosophy of intensified replacement therapy, but the proportions remain similar. Recently

performed randomized controlled studies have confirmed the obvious superiority of prophylaxis over episodic treatment in preventing bleeds and therefore improving long-term outcomes [19, 20]. While all these approaches used fairly fixed dose protocols, investigators in Canada attempted to individualize requirements with escalating

doses based on individual bleeding patterns [21]. Such approaches, however, need to be carefully designed so as not to allow too many joint bleeds before escalating replacement. learn more No major study has been attempted to prospectively compare different prophylaxis protocols. In the absence of data comparing different doses for prophylaxis, varying doses are used, based on individual experiences or conviction of what is best. There is therefore great heterogeneity in replacement therapy protocols with regard to time for initiation as well as the dose and frequency of administration, even within the same healthcare environment [22]. The irony of these practices is perhaps most obvious in Western Europe, a zone with relative socioeconomic parity in healthcare and where considerable efforts have been made to standardize care [23]. Data collected by the annual global survey of the WFH and a European Hemophilia Consortium survey have shown for some years now that the CFC use in these countries varied from less than 3 IU per capita to more than 7 IU per capita [22]. There is hardly an example of another disease where there is such variation in doses of a drug used for its treatment. National registries and databases, such as the one in the UK, that have included data on CFC use, have shown that even within the same country, there can be >twofold differences between regions or centres [24].

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