residual or subclinical haemarthroses , and hence at risk of subsequent arthropathy , due to insufficient trough FVIII levels , and those who request it after having learned about the benefits of these new products .
A monocentre case study in France showed that , out of 48 patients who switched to rFVIIIFc , 32 changed their dosing schedules , reducing the injection frequency or introducing prophylaxis . Of those 48 patients , 40 received long-term prophylaxis , without any obvious preference for a particular treatment pre-switch , and 8 were treated on demand . After switching to rFVFc , prophylaxis was continued or initiated in 47 patients , with a good / excellent qualitative assessment of efficacy in 44 out of 45 patients evaluated . As of now , FVIII trough levels ( 1 – 4 %) were shown to be consistent with the clinical outcomes , and no inhibitor development was observed . Although larger and longer surveys need to be conducted to determine QoL and health economic outcomes , rFVIIIFc may optimise outcomes in some patients by increasing the level of protection in patients at risk of residual bleedings , and by making prophylaxis acceptable and feasible among patients reluctant to changes in their treatment plans .
Reconsider treatment goals in haemophilia B : Clinical experience with rFIXFc One of the major achievements of prophylaxis regimens for haemophilia patients is sustained protection from bleeds ; however , among the treatment goals reported by patients to be important to them is decreased injection frequency to reduce the treatment burden and improve QoL . In addition , having weekly injections allows patients and / or their carers to remember to take their medication , which contributes to improved adherence . However , these patient-related benefits are related to clinical outcomes . The anticipated desired outcomes with rFIXFc are to achieve and maintain low ABRs , as well as attain adequate FIX levels ( individualised for the patient ’ s bleeding phenotype , joint status , and activity levels ), prevent bleeding when undergoing surgical or invasive medical procedures , and enhance patient experience .
A study was conducted in the Republic of Ireland to assess FIX usage among patients with haemophilia B on conventional or EHL FIX after switching to rFIXFc At baseline , 22 and 6 patients were receiving prophylaxis and on-demand treatment with FIX , respectively , compared with 26 and 0 patients on rFIXFc . The mean prophylactic doses were higher for the conventional agents , and some of the patients were on quite high weekly doses . After three months on rFIXFc prophylaxis , FIX trough levels increased compared with rFIX trough levels before switching , and the median ABR after six months of follow-up was 3 for the standard agents versus 1 for rFIXFc , with approximately half of the patients on rFIXFc experiencing zero bleeds . Moreover , weekly consumption in patients on doses > 98 IU / kg / week of rFIX decreased significantly .
A total of eight patients underwent minor surgical procedures , and only one experienced bleeding and required local homeostasis and more than one injection in this study . With regard to patient experience , ease of use , efficacy and confidence were reported by individual patients , although these were not formally quantified .
It is important to identify patients who are more likely to benefit from treatment with rFIXFc : those with poor venous access or needle phobia , or those who show poor adherence to prior therapy , those who are not receiving adequate treatment because of fast clearance , those with breakthrough bleeds and / or synovitis with conventional replacement treatment , or those with a very active life . It is also important to agree in advance on the individual treatment goals , taking into consideration what constitutes a good measure of success ( for example , increased protection , fewer bleeding episodes , or improved convenience ) and re-evaluating goals and outcomes at three and six months post-switch .
Key insights Current prophylaxis regimens provide significant benefits to patients , but may not be optimal if other aspects beyond bleeds prevention are considered , such as joints protection , patients ’ adherence and QoL . The new EHL-Fc clotting factors could facilitate at least maintaining adequate protection , even in cases of fast drug clearance , and improve adherence , as well as patient confidence and experience . The question that remains is if the available clinical trial data will translate into the clinical practice .
Based on the data and the experience presented , the audience felt that the priority when setting treatment goals for patients was the reduction of breakthrough and joint bleeds , but the importance of trough levels as an indicator of a favourable direction of treatment must be emphasised , and noted that relief of patient treatment burden can lead to better protection . Therefore , although bleeding remains at the cornerstone of expected treatment outcomes , ABRs alone should not be considered as the only treatment objective . Joint health , QoL and increased / maintained physical activity are also important goals , although QoL is not usually assessed in clinical practice .
Finally , the audience in this symposium presentation voted real-world evidence to be most useful when evaluating new treatments for haemophilia . Pivotal clinical trials undoubtedly provide fundamental data for a specific patient population , whereas real-world studies can contribute valuable information reflecting use in real-world conditions , outside of a clinical trial setting . Both rFVIIIFc and rFIXFc have the longest real-world experience among EHL products , and the many ongoing real-world experiences may complement these data and help shed light on the best strategies to improve outcomes for patients with haemophilia .
The satellite symposium Evolving Protection in Haemophilia and Helping to Advance the Standard of Care was held on 7 February 2018 at the 11th Annual Congress of the European Association for Haemophilia and Allied Disorders , Madrid , Spain
16 HHE 2018 | hospitalhealthcare . com
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