Communicating Sickle Cell Disease Knowledge: The Challenges of Gene Therapy

University of Memphis

Amanda Young, PhD,

Craig Stewart, PhD

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St. Jude Children’s Research Hospital

Akshay Sharma, MBBS

Yvonne Carroll, JD, RN

Mary Irvine, MPH

Mary Caples, MA, CCRP

Liza Johnson, MD, MPH, MSB

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What Does Sickle Cell Disease Look Like?

What Does Sickle Cell Disease Look Like?

• 2021 Textbook

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• Artwork was commissioned from Hertz Nazaire, a sickle cell warrior, advocate and artist.

• Most common life-limiting inherited disease in the U.S.
• Affects ~100,000 people, mostly African Americans
• Faulty hemoglobin gene limits oxygen flow
• Causes severe pain, joint and organ damage, and stroke
• Can lead to death

Sickle Cell Disease (SCD)

Two Cases of Challenging Information

SCDGENE

• A collaborative project among St. Jude and other sickle cell centers and researchers
• Purpose: Work with sickle cell warriors to create an informed consent tool and process that supports informed consent for gene therapy

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60 Minutes episode on gene therapy for SCD

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SCDGENE

Components of Project

1. A needs assessment of
• Knowledge about bone marrow transplant and gene therapy
• Acceptable risk
• Health Literacy

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• A series of focus groups of sickle cell warriors and caregivers
• Explore communication needs and preferences
• Assess attitudes and perceptions about gene therapy
• Understand warriors’ and caregivers’ experiences and perspectives

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Focus group�presentation about gene therapy and the following discussion

Work in progress.

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SCD patient

SCD patient No More

Harvest Bone Marrow Stem Cells

Grow Stem Cells in Laboratory

Gene Modification

Conditioning Regimen

Transplant Harvested and Transduced Cells into Patient

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Slide by Dr. Akshay Sharma

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Stem Cell Collection

Monthly Transfusions

“Transplant”

Follow Up

4-6 weeks in the hospital

Up to 15 years

2-3 days in the hospital

2-3 months

Slide by Dr. Akshay Sharma

Lessons from focus groups

• Trust
• My question was about the gene therapy and this thing with the Bluebird and, and they stopped it because of patients getting cancer. It’s been around for about six or seven years, is the cancer thing. Is this something new that you guys have just learned about? Or have you always known that it could cause cancer?
• Transparency
• Has this program had any success statistics, outcomes statistically or is it too new, like, how long has it been around? I guess this is a two-part question. First, how long it's been around? And then the second part is, okay, now we know what the risks are, have we had any success in practicing it?

Lessons from focus groups

• Risk
• Don't sit up there and beat around the bush. Put those risks out because I think what happens a lot of times and I know the people who have done like the bone marrow transplant or gene therapy while doing informed consent they just glaze over certain aspects
• I think the hope aspect of it is why it's even more important to spotlight or put in the forefront the risk assessment piece of it so that you can make an informed decision and not hold on to the hope. Have it counterbalanced with the facts that these are the things that you are trading off.

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Lessons from focus groups

• Tailored information
• Those consent forms are very long so if you're just sitting there and they're giving you this piece of paper. It's kind of like they want you to skip through everything and then you're signing your life away on this dotted line. You need to break it down to me. Let me take a section of it, read it, jot down my questions, and then comeback with the information that I am needing.
• Decision making burden
• It's kind of like they want you to skip through everything and then you're signing your life away on this dotted line. You need to break it down to me.

Lessons from focus groups

• Sickle cell identity
• I wasn't being considered as a person. It felt like I was just a number in their trial. And they kept talking about how oh you're the oldest one we've ever transplanted in sickle cell; it's going to be that. I'm like, excuse me. I don't care. That's great for you but you know what I mean like, "Oh you're going to be our poster child." I was like no I'm really not because I'm a person.

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60 Minutes Episode on Gene Therapy

60 Minutes

• Followed a young sickle cell warrior who received gene therapy
• Interspersed with science and interviews with scientists
• The BIG PROBLEM:
• Introducing HIV as a vector in gene therapy
• But waiting to explain why it’s safe for several sentences

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60 Minutes

• Clearly a good-faith effort to present the information
• Probably still to complex for the average viewer
• Did not consider their most important audience: the sickle cell community

First Analysis: The transcript from the program

Details were Problematic

SCD Warriors’ Reactions: CBS This Morning

Simon (the nurse practitioner): Your reactions were, you know, surprisingly, like, not exactly what I expected.

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LaPook: How so?

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Simon: I guess I was expecting a little more, like, excitement, because we’ve talked about gene therapy. And the downside, it’s really profound, and it’s scary. But I think seeing how you reacted, um, I’m still as enthusiastic as ever.

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LaPook (to the panel): What were the scary parts to you?

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Fowler: I wanna say the number one thing, we heard the HIV thing.

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Barzey-Kinard (pointing to Fowler): Right.

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Social Media Reactions

Lessons Learned

Trust is the foundation in communicating complex information

• Trust is lost
• By the use of jargon and language that is too difficult
• By not addressing the most important stake holders
• By supporting power structures that are already suspect
• Trust is built by:
• Engaging stakeholders in the creation of materials
• Transparency, especially in talking about risk
• Customizing information for the stakeholders, especially the most important stakeholders
• Using materials that reflect the people using them. “I want to see myself.”
• Creating a process that honors the individual’s needs

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To all the warriors and caregivers who shared their time, their expertise, and their hearts:��Thank you!

To all my colleagues:��Thank you for your support and collaboration.

Questions?