Patient and Family Engagement during Treatment Change to Emicizumab at a Hemophilia Treatment Center
Authors: Lattimore Susan U , Hofstetter Madolyn T 
Published: November 2019

Introduction: Treatment options to prevent bleeding events in people with hemophilia A expanded with the FDA-approval of emicizumab. Safety and efficacy of emicizumab have been reported, but patient-specific health goals and expectations for switching to emicizumab are unpublished.
Federally-supported Hemophilia Treatment Centers (HTCs), under the Maternal Child Health Bureau's Regional Hemophilia Network, are working to increase patient and family engagement in care decisions through quality improvement initiatives. Placing patients and families at the center of their care has been found to correlate with the ability to undertake personal health maintenance, adhere to complex treatment regimens and improve health outcomes.
Objective: We sought to engage patients and families in their care by capturing patient-reported goals and gaps in education when changing treatment to emicizumab prophylaxis in people with hemophilia A at The Hemophilia Center at Oregon Health & Science University.
Methods: A member of the HTC team not directly involved in the patient's care asked baseline questions at emicizumab initiation. Two follow-up questions were asked 5 weeks and 3 months after baseline, in clinic or via phone. Five open-ended baseline questions facilitated conversation and addressed patient/caregiver stated goals of treatment change and unanswered questions regarding emicizumab. Two follow-up questions assessed progress toward goals identified at baseline and information they wish they knew before switching. Participation was voluntary. No age or disease severity exclusions, but patients with active inhibitor were not included in this pilot project. For patients age < 18, both patient and caregiver were able to respond.
Results: Thirty patients changing treatment to emicizumab were engaged. Fourteen patients and 5 caregivers responded independently. Eleven pediatric patients had a response by both patient and caregiver to total 41 baseline responses. Eighteen patients were <18 years and mean age was 19 (range: 1 to 70). 20 patients had severe hemophilia; 10 had moderate hemophilia. Most patients and caregivers said they were well informed prior to treatment change regarding the drug, administration, safety, and reported experience. Unanswered questions were categorized and included: side effects/drug interactions (17%), treating bleeds/amount of factor to have on hand (20%), and subcutaneous injections (13%). 47% had no outstanding questions at baseline. Patient-/caregiver-reported goals were categorized. Among all goals, 43% aligned with an increase/change in physical activity, 23% decrease in bleeding events, 17% less bruising, 10% fewer infusions, 3% more convenience, and 3% becoming more independent.
Follow up data is available for 26 (87%) patients. At follow up visits, gaps in pre-switch education included unexpected painful injections (27%), ordering emicizumab and/or supplies (14%), maintaining peripheral infusion skills (4.5%), preparing emicizumab (4.5%), and assessing/treating bleeds (4.5%). 50% of patients/caregivers reported no outstanding education needs.
All patients/caregivers (100%) stated they met their baseline-identified goals by month 3. Additional information derived from conversations prompted by the questions and offered by patients and families included reports of increased convenience, decreased parental worry, increased activity, fewer bleeding events, fewer work absences, and decreased pain.
Discussion: Patients and caregivers are receptive to providing information regarding expectations surrounding treatment change to emicizumab. Respondents were able to identify personal goals in regard to their care and provide input into additional education that may inform other patients switching to emicizumab. Progress on patient-reported goals remains ongoing at this time. Although 50% of respondents indicated that there was information they wished they knew prior to changing treatment, none of the patients approached under this project have discontinued the use of emicizumab. It is feasible to engage patients and families with bleeding disorders in their care, as exampled by progress under this quality improvement initiative. Further analysis is needed to understand longer-term engagement of patients who use emicizumab for treatment of hemophilia A.

Disclosures
No relevant conflicts of interest to declare.