Objectives:
The longer-term clinical outcomes of patients using emicizumab in real world settings are not well documented. This study examined hemophilia patient clinical outcomes, specifically bleed rate, emergency room visits/hospitalizations, nurse resource use, adherence and emicizumab wastage in the 6 months before and after initiating emicizumab.
Methods:
This is a retrospective cohort study of patients in the United States who received an emicizumab prescription between July 1, 2018 and July 1, 2019 from a single specialty pharmacy. Patients with moderate (1% to 5% factor activity) to severe (<1% factor activity) hemophilia were included if they took emicizumab for at least 6 months and had 6 months prior use of a prophylactic factor product. Self-reported bleeds, emergency room visits, hospital admissions and nurse visits (a visit by a nurse to provide administration, education and/or infusion of the drug) were obtained from the patient’s electronic medical record. Medication adherence was assessed by calculating proportion of days covered (PDC) (the number of days in period treated divided by number of days in period) using medication dispense date and quantity from the pharmacy dispensing system. Paired Wilcoxon signed-rank and McNemar’s tests were used to assess changes in outcomes. A one-way ANOVA was used to compare emicizumab waste (the amount of drug (mg) not used per month, across the 6 months after initiating emicizumab).
Summary:
The median participant age was 15 years (range 3-53) (N=35); all were male. After initiating emicizumab, patients reported 55% fewer bleeds (m=2.9 vs. 1.3; p=0.001), 83% fewer nurse visits (m=15.5 vs. 2.8; p=0.01) and 15% greater medication adherence (m=0.84 vs. 0.97; p=0.04). When stratifying by age, the reduction in nurse visits was only significant among patients under age 18 (p = 0.01) and the increase in medication adherence was only significant among adults 18+ (p=0.01). There was no difference in emergency room/hospital visits after initiating emicizumab (m=0.40 vs. 0.23; p = 0.4). Most patients (51.4%) reported emicizumab wastage in the first month. After the initial loading dose (month 1), there was a decreasing proportion of emicizumab waste across months 2-6, though comparisons were not statistically significant (13.8%, 13.3%, 12.1%. 12.4% and 10.9%; p>0.6). Although the intent was not to assess cost savings, our study did find a 9.1% savings in drug cost over the course of one year, when comparing factor utilization pre-emicizumab.
Conclusions:
In this study, taking emicizumab was associated with a significant reduction in bleeds and nursing visits which may reduce operational costs and increase medication adherence. No significant change in emergency room/hospital visits were observed. Future prospective studies with larger samples and a control group are needed to assess long-term clinical outcomes, health-related quality of life, and total cost and quality of care among patients using emicizumab.
Objective:
To educate teens and their siblings on building skills related to self-care, medical independence and living a healthy lifestyle so that they are better prepared to be on their own as they transition to the next stage in life: adulthood.
Methods:
A collaborative, multidisciplinary team consisting of team members from the Cincinnati Children’s Hospital Medical Center Hemophilia Treatment Center (HTC) and the Tri-State Bleeding Disorder Foundation lead 14 teens attending a family bleeding disorder educational conference in an interactive, educational program.
Participants were teens (ages 12-17) with a bleeding disorder and their siblings. Participants worked in teams and visited six different booths where they engaged in interactive activities such as role playing, exercising and games. Booths were staffed by the HTC staff that included nurse care managers, pharmacists, data manager, social worker, health educator, and a physical therapist. Each booth focused on: genetics of a bleeding disorder, digital citizenship, managing home treatment independently, understanding bleeding disorders, insurance/career planning and fitness /healthy joints.
This evidenced-based educational content was based on the Medical and Scientific Advisory Council (MASAC) transition guidelines for people with a bleeding disorder (Belling e al., 2003). The content and program design was also rooted in the social cognitive theory with elements of vicarious learning, behavioral capability, and self-control.
Summary:
An interactive and evidenced-based teen transition program allowed for teens to gain knowledge in various topics related to gaining in medical independence and self-care skills. All teens that participated in the program demonstrated an increase in knowledge at the program’s end.
Conclusions:
All participants showed an increase in scores from pre-test to post-test. The average scores increased by 20%. The team plans to implement this program again in the future.
References:
Belling L., Harrop M., Kocik S., Obstein, L., Standish, D., Vlasich, W., & Zappa, S. (2003). Transition Guidelines for People with Bleeding Disorders. New York, NY: M.A.S.A.C, 147, 1.
The inherited bleeding disorders (IBD) community has witnessed significant advances in recent years thanks to novel therapeutic advances and technologies and improved diagnostic proficiency.
Yet important gaps persistent, particularly for those with rare disorders and underserved populations, including women with IBD.
A new initiative led by the National Hemophilia Foundation (NHF) and shaped by the voices of the patient community is underway to address this gap.
Our goal: to design and implement a national research blueprint that outlines actionable strategies to address the most important needs within the community and opportunities to accelerate progress through coordinated collaboration.
