ZYNTEGLO™ Patient Identification
ZYNTEGLO is indicated for the treatment of adult and pediatric patients with beta-thalassemia who require regular RBC transfusions.1
Talk to your patients about ZYNTEGLO
Create a discussion guide to help support conversations with your patients and their loved ones. Get started by answering the following 5 questions. This tool is not intended to determine treatment eligibility or provide medical advice.
What is your patient's age group?
Talk to your patients about ZYNTEGLO
Have you spoken to your patient about any challenges or concerns with their transfusion regimen?
Talk to your patients about ZYNTEGLO
Are their iron levels under control?
Have you noticed, or has your patient expressed, any difficulties with maintaining their chelation therapy?
Identifying potential
patients for ZYNTEGLO
Considerations
Iron Overload
Iron Overload
Iron Overload
Iron Overload
- What impact has iron overload had on my patient?
- How might my patient’s iron level affect their ability to undergo myeloablative conditioning?
ORGAN DAMAGE/
COMORBIDITIES
ORGAN DAMAGE/
COMORBIDITIES
ORGAN DAMAGE/
COMORBIDITIES
ORGAN DAMAGE/
COMORBIDITIES
- Does my patient have organ damage from iron overload or beta-thalassemia?
- How do my patient’s comorbidities affect their ability to receive treatment?
Life Milestones
Life Milestones
Life Milestones
Life Milestones
- What are my patient’s thoughts on having a family?
- What upcoming life changes may affect my patient’s desire to receive treatment?
Explore Patient Profiles
Age 12
ACTOR PORTRAYAL
Age 12
ACTOR PORTRAYAL
CLINICAL PRESENTATION
- Genotype: non-β0/β0
- LIC: 7 mg/g (LIC increased from 4.2 mg/g at last evaluation)
- Serum ferritin: 3212 pmol/L
- Cardiac T2*: >38 msec
- No comorbidities
CURRENT TREATMENT
- Chelation with deferiprone (switched from subcutaneous chelator)
- Blood transfusion (3 units) every 4 weeks
DANIEL'S STORY
- Diagnosed after suggestive newborn screening result
- Has been receiving regular blood transfusions since 2 years old
- Frustrated with his condition and struggles with chelation therapy interfering with his active lifestyle
- Parents mentioned that blood transfusions have caused him to miss classes
HOW IS DANIEL NOW?
Daniel’s parents have seen how his frequent blood transfusions can interfere with his active lifestyle, and have begun asking about what other treatment options are available. The family has read a little on gene therapy and are curious to know more, wondering if it’s a treatment option that could potentially help Daniel become transfusion independent.
WHAT WOULD BE YOUR NEXT STEP TO HELP DANIEL?
Consider providing Daniel and his family the ZYNTEGLO Patient and Caregiver Brochure today, so that next time they come in you can discuss whether ZYNTEGLO may help meet their treatment goals.
LIC = liver iron concentration.
Age 18
ACTOR PORTRAYAL
Age 18
ACTOR PORTRAYAL
CLINICAL PRESENTATION
- Genotype: non-β0/β0
- LIC: 5.5 mg/g
- Serum ferritin: 3412 pmol/L
- Cardiac T2*: >20 msec
- No comorbidities
CURRENT TREATMENT
- Chelation with deferasirox
- Blood transfusion (2 units) every 4 weeks
MILA'S STORY
- Diagnosed at 12 months and began receiving regular blood transfusions
- Started chelation therapy at age 3
- As a child, explored allo-HSCT with the help of her parents, but was unsuccessful in finding an HLA-matched donor
- After being accepted into an out-of-state college, she is focused on her future and is concerned her current treatment plan will affect her ability to attend class in college
HOW IS MILA NOW?
When Mila was in high school, she had a great relationship with the teachers who understood her disease and the need to occasionally miss class for treatment. Now that Mila is about to move across the country to start college at a very large university, she’s nervous about being in an environment with people who are not familiar with her needs.
WHAT WOULD BE YOUR NEXT STEP TO HELP MILA?
Mila and her family have expressed a desire to pursue allo-HSCT in the past, but did not have access to an appropriate donor. Gene therapy with ZYNTEGLO is an option that does not require a donor and may offer Mila the outcomes she and her family are seeking. As she is preparing to begin college, consider discussing the benefits and risks associated with ZYNTEGLO.
HLA = human leukocyte antigen; LIC = liver iron concentration.
Age 23
ACTOR PORTRAYAL
Age 23
ACTOR PORTRAYAL
CLINICAL PRESENTATION
- Genotype: β0/β0
- LIC: 9 mg/g
- Serum ferritin: 8785 pmol/L
- Cardiac T2*: unknown
- Comorbidity: splenomegaly and hypothyroidism
CURRENT TREATMENT
- Chelation with deferasirox
- Blood transfusion (3 units) every 3 weeks
ALEX'S STORY
- Diagnosed at 8 months and has received regular transfusions ever since
- Recently moved to the United States; has struggled with adherence to his transfusion schedule and chelation therapy as he tries to establish himself in a new country
- Medical records are incomplete
HOW IS ALEX NOW?
After being referred by his primary care physician, Alex begins working with you to manage his condition. During his appointment, you uncover that Alex is having difficulty with maintaining his current treatment as he juggles work and finishes up moving. Alex asks you if there are any other treatment options that could help him reduce his need for regular blood transfusions.
WHAT WOULD BE YOUR NEXT STEP TO HELP ALEX?
Alex is curious about ZYNTEGLO and what potential transfusion independence would mean for him. Consider discussing the benefits and risks of ZYNTEGLO with Alex, begin working with him to improve his adherence to current treatment, and complete his medical history. Consider consulting with a Qualified Treatment Center to confirm eligibility. Use the Qualified Treatment Center Locator Tool or connect with a Patient Navigator from my bluebird support to help identify which centers may work for Alex.
LIC = liver iron concentration.
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