Current prescription of prophylactic factor infusions and perceived adherence for children and adolescents with haemophilia: a survey of haemophilia healthcare professionals in the United States

Abstract

The primary goal of prophylaxis in patients with severe haemophilia is to convert the phenotype from severe to moderate and to prevent the development of chronic arthropathy. Prior studies have demonstrated that prophylaxis decreases episodes of joint bleeds and chronic arthropathy. Effectiveness depends on prescription of prophylaxis and adherence to the prescribed regimen. The aim of this study was to determine if prescription of prophylaxis for children with haemophilia and perceptions of adherence to prophylaxis have changed since publication of the Joint Outcome Study (JOS). A questionnaire was sent, in electronic and written formats, to health professionals who provide care to children with haemophilia at US haemophilia treatment centres (HTCs). The response rate was 56 of 128 (44%) of the targeted HTCs. There were a few missing data and denominators are provided. All responses agreed with the results of the JOS and 30/55 (55%) reported the JOS increased their prescription of prophylaxis. Nineteen of 56 (34%) physicians or HTC staff reported that they had not prescribed prophylaxis within the last year due to concerns about adherence, and 19/56 (34%) reported they had stopped prophylaxis due to concerns about adherence within the last year. Predicted adherence decreased with increasing age. Prescription of prophylaxis appears to be increasing since publication of the JOS. Strategies to improve adherence may increase the likelihood of physician prescription of prophylaxis and make prophylaxis easier to implement for individual patients, thereby improving the clinical outcome of children and adults with haemophilia.

Both Hemophilia Health Care Providers and Hemophilia A Carriers Report That Carriers Have Excessive Bleeding

“…Finally, the survey response rate of health care providers was moderately low at 44%, which introduces concern of selection bias. Despite these limitations, the survey response rate was the same or higher than previously published manuscripts (14, 15). …”

Influence of medical insurance schemes and charity assistance projects on regular prophylaxis treatment of the boys with severe haemophilia A in China

“…20 This was even though treatment efficacy should ultimately be the most important factor for treatment adherence/ compliance. [21][22][23][24] The availability of haemophilia outpatient medical insurance in China since 2007 has allowed low-dose prophylaxis possible to improve quality of life in HA children. The majority of our patients in this study were covered by medical insurance, and the upper limit for over 80% of our patients covered by medical insurance was sufficient to cover the cost of annual FVIII consumption for the low-dose prophylaxis regimen.…”

Haemophilia Experiences, Results and Opportunities (HERO) study: treatment‐related characteristics of the population

“…2). PWH reported a mean/median (IQR) of 17.8/7 (2)(3)(4)(5)(6)(7)(8)(9)(10)(11)(12)(13)(14)(15)(16)(17)(18)(19)(20) bleeds in the past year; 8% reported no bleeds in the past year. PWH reported a mean of 15.41 joint and 5.84 muscle bleeds.…”

“…PWH reported a mean of 15.41 joint and 5.84 muscle bleeds. Of note, median (IQR) bleeds per year were similar in PWH with and without inhibitors; median (IQR) bleeds per year were similar on on demand (13.89 [1-16.5]) and prophylaxis (14.81 [2][3][4][5][6][7][8][9][10][11][12][13][14][15]), but much higher with on-demand plus short-term prophylaxis (28.85 [6.5-35.5]). A lot less than was instructed by the haemophilia or nurse Parents reported a mean/median (IQR) of 8.67/4 (2-10) bleeds in the past year; 12% reported no bleeds in the past year.…”

“…In contrast to PWH, children with inhibitors had a higher mean/ median (IQR) reported bleeds per year than those without inhibitors (14.34/9.5 [4][5][6][7][8][9][10][11][12][13][14][15] vs. 8.19/4 [2][3][4][5][6][7][8][9][10]) and fewer had no bleeds in the past year (4% vs. 13%). Children on prophylaxis had a lower mean/ median (IQR) reported bleeds per year than those on demand (6.25/3 [1][2][3][4][5][6][7] vs. 11.96/6 [2][3][4][5]) and lower mean rates of joint and muscle bleeding. As seen in PWH, parents reported higher bleed rates with ondemand plus short-term prophylaxis (16.47 [4-20]) than either on demand or prophylaxis.…”