Narrative-based Decision-making: Co-development of a Graphic Educational Aid for Sickle Cell Disease Treatment Options
Liza-Marie Johnson, Ambria Williams, Abagail Cohen, Meaghann Weaver, Belinda Mandrell, Jami Gattuso, Mary Caples, Yoram Unguru, Yvonne Carroll, Gary Ashwal, Alex Thomas, Lakiea Bailey, Dominique Goodson, Amanda Young, Benjamin Wilfond, Akshay SharmaAbstract
Background
Parents and individuals with sickle cell disease (SCD) may feel overwhelmed when weighing the risks and benefits of the various treatment options, particularly as clinical trials exploring new treatments are offered. Gene therapy (GT) and hematopoietic stem cell transplantation (HSCT) may be discussed as potentially transformative treatment options for children and adults with moderate to severe SCD. The FDA approved two gene therapies for SCD in late 2023 for eligible patients ages 12 and older, bringing national attention to SCD gene therapies to lay audiences. Additional gene therapies are currently in development for SCD and other rare genetic diseases. While GT and HSCT are promising options for many, these therapies may not be suitable for every eligible patient. Additionally, ethical issues around consent and informed choice can arise in the face of uncertainty. As such, there is a need to promote shared decision-making and ensure patients are adequately counseled in the risk/benefit analysis of treatment. The purpose of this project was to develop patient-centered educational resources to ensure a high-quality informed consent process for these therapies.
Methods
Through a community partnership research approach, a regionally diverse panel of parents of children with SCD and adult patients with SCD were recruited through purposive sampling to participate in an eight-part series of virtual focus groups to discuss communication and educational needs related to transformative therapies. An interdisciplinary expert stakeholder advisory group was also formed to discuss the implementation of the communication and educational needs highlighted by the focus groups. An in-person meeting between the members of both focus group and stakeholder advisory group was held to elicit design and utility input for the development of an educational website specifically highlighting transformative treatments for SCD. After the creation of the website, usability testing was conducted to elicit feedback on the layout and content. Usability testing participants reviewed the website, completed the Patient Educational Materials Assessment Tool (PEMAT) survey, and were interviewed to provide detailed feedback.
Results
Twelve individuals participated in the patient and parent focus groups. Of these, seven (58%) were female and five (42%) were male. The focus groups revealed that patient-caregivers weigh the benefits and risks of all their treatment options alongside GT and HSCT. As a result, one narrative-based website, myscdstory.org, was developed detailing four SCD treatment options: GT, HSCT, chronic transfusion therapy, and hydroxyurea. Users can choose a treatment pathway and follow the fictional patient’s journey from ages 13 to 34 for all four treatment options. A comparative risk and benefits page, along with additional resources are provided on the website. Eleven parents of a child with SCD participated in website usability testing. Out of this participant group, nine (82%) were female and two (18%) were male. For the PEMAT survey results, 100% of participants said that the website was easily viewable on their devices and that it identified at least one possible action users can take regarding treatment decision-making. Additionally, 91% of usability testing participants reported that the purpose of the website was completely evident. In the interviews, nearly all parents who were asked (9/10), said that the website would make them more confident in speaking with their child’s doctor about treatment options. All parents asked (10/10), said that it was easy to navigate. Participants said the website was informative, with realistic depictions of the treatment options, including transformative therapies like HSCT, and about two-thirds (64%) and said that they would want to discover the website in their hematology clinic.
Conclusions
Informed directly by patient-caregivers and a working group of SCD transformative treatment experts, graphic educational tools such as myscdstory.org can serve as engaging assistive aids to support informed discussions about treatment options. The website is currently undergoing pilot testing for implementation into clinical practice. In addition to the website, one science fiction short story and one fairytale short story were developed to aid patients ages 7-14, along with their parents, to learn about the complex ethical and social themes relevant to transformative treatment. This work critically demonstrates that, through community engagement, educational graphic stories can be developed to facilitate patient understanding around novel treatment options.