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
100 HHE 2018 | hospitalhealthcare. com
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