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HEALTH SERVICES RESEARCH-NON-MALIGNANT CONDITIONS | NOVEMBER 13, 2019

Patient and Family Engagement during Treatment

Change to Emicizumab at a Hemophilia Treatment Center
*,1 *,1
Susan U Lattimore, Madolyn T Hofstetter, BS
1
The Hemophilia Center, Oregon Health & Science University, Portland, OR

bloodjournal Blood blood (2019) 134 (Supplement_1) : 5796.

http://doi.org/10.1182/blood-2019-131030

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health goals and expectations for switching to emicizumab are unpublished.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.

Author notes
*Asterisk with author names denotes non-ASH members.

© 2019 by the American Society of Hematology