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Actor Portrayal
Patient identification
ZYNTEGLO® is indicated for the treatment of adult and pediatric patients with beta-thalassemia who require regular RBC transfusions.1
Made using the patient’s
own hematopoietic stem cells.
own hematopoietic stem cells.
Studied in both adult and
pediatric patients.*
pediatric patients.*
Assessed across all genotypes
(β0/β0 and non-β0/β0).
(β0/β0 and non-β0/β0).
*ZYNTEGLO was studied in patients 4 to 34 years of age. The safety and efficacy in children <4 years of age have not been established; no data are available.1
Topics to consider when identifying potential patients for ZYNTEGLO
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
- 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
- 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
Do you have patients like the profiles below for whom ZYNTEGLO
could be a treatment option?
could be a treatment option?
CLINICAL PRESENTATION
- Genotype: non-β0/β0
- LIC: 7 mg/g (LIC increased from 4.2 mg/g at last evaluation)
- Serum ferritin: 4121 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 | 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: 4121 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 | 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: 4121 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 | 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: 4121 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 | Age 12
ACTOR PORTRAYAL
DANIEL'S STORY
- Diagnosed after suggestive newborn screening result
- Has been receiving regular blood transfusions since 2 years old
- Struggles with chelation therapy—has expressed frustration with his condition and current therapy interfering with his active lifestyle while his parents have mentioned it has caused him to miss class as well
LIC = Liver iron concentration.
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?
Feeling burdened by his current treatment, Daniel's parents are wanting to know about additional treatment options. Consider discussing with them about the efficacy and safety profile of ZYNTEGLO as a potential option.CLINICAL PRESENTATION
- Genotype: non-β0/β0
- LIC: 5.5 mg/g
- Serum ferritin: 6412 pmol/L
- Cardiac T2*: >38 msec
- No comorbidities
CURRENT TREATMENT
- Chelation with deferasirox
- Blood transfusion (2 units) every 4 weeks
Mila | Age 18
ACTOR PORTRAYAL
CLINICAL PRESENTATION
- Genotype: non-β0/β0
- LIC: 5.5 mg/g
- Serum ferritin: 6412 pmol/L
- Cardiac T2*: >38 msec
- No comorbidities
CURRENT TREATMENT
- Chelation with deferasirox
- Blood transfusion (2 units) every 4 weeks
Mila | Age 18
ACTOR PORTRAYAL
CLINICAL PRESENTATION
- Genotype: non-β0/β0
- LIC: 5.5 mg/g
- Serum ferritin: 6412 pmol/L
- Cardiac T2*: >38 msec
- No comorbidities
CURRENT TREATMENT
- Chelation with deferasirox
- Blood transfusion (2 units) every 4 weeks
Mila | Age 18
ACTOR PORTRAYAL
CLINICAL PRESENTATION
- Genotype: non-β0/β0
- LIC: 5.5 mg/g
- Serum ferritin: 6412 pmol/L
- Cardiac T2*: >38 msec
- No comorbidities
CURRENT TREATMENT
- Chelation with deferasirox
- Blood transfusion (2 units) every 4 weeks
Mila | Age 18
ACTOR PORTRAYAL
MILA'S STORY
- Diagnosed at 12 months and began receiving regular blood transfusions
- Started chelation therapy at age 3
- Has explored allo-HSCT with the help of her parents, but 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
HLA = human leukocyte antigen; LIC = liver iron concentration.
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 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 her and her family are seeking. As she is preparing to begin college, consider discussing the benefits and risks associated with ZYNTEGLO.CLINICAL PRESENTATION
- Genotype: β0/β0
- LIC: 5.2 mg/g
- Serum ferritin: 2785 pmol/L
- Cardiac T2*: >40 msec
- Comorbidity: splenomegaly and hypothyroidism
CURRENT TREATMENT
- Chelation with deferasirox
- Blood transfusion (3 units) every 4 weeks
Alex | Age 23
ACTOR PORTRAYAL
CLINICAL PRESENTATION
- Genotype: β0/β0
- LIC: 5.2 mg/g
- Serum ferritin: 2785 pmol/L
- Cardiac T2*: >40 msec
- Comorbidity: splenomegaly and hypothyroidism
CURRENT TREATMENT
- Chelation with deferasirox
- Blood transfusion (3 units) every 4 weeks
Alex | Age 23
ACTOR PORTRAYAL
CLINICAL PRESENTATION
- Genotype: β0/β0
- LIC: 5.2 mg/g
- Serum ferritin: 2785 pmol/L
- Cardiac T2*: >40 msec
- Comorbidity: splenomegaly and hypothyroidism
CURRENT TREATMENT
- Chelation with deferasirox
- Blood transfusion (3 units) every 4 weeks
Alex | Age 23
ACTOR PORTRAYAL
CLINICAL PRESENTATION
- Genotype: β0/β0
- LIC: 5.2 mg/g
- Serum ferritin: 2785 pmol/L
- Cardiac T2*: >40 msec
- Comorbidity: splenomegaly and hypothyroidism
CURRENT TREATMENT
- Chelation with deferasirox
- Blood transfusion (3 units) every 4 weeks
Alex | Age 23
ACTOR PORTRAYAL
ALEX'S STORY
- Diagnosed at 8 months and has received regular transfusions ever since
- Demonstrated strong adherence to treatment and proactively self monitors
- Cautious about any changes in current, familiar treatments
- Adjustments in chelation therapy have always been confusing to him
LIC = liver iron concentration.
HOW IS ALEX NOW?
Recently graduated from college, Alex is about to start a new job that will require traveling. Before he starts his new, professional life, Alex reaches out to his doctor to determine how he can maintain his current treatment with his new job. Alex also asks his doctor if other treatment options exist that would also work with his new schedule.WHAT WOULD BE YOUR NEXT STEP TO HELP ALEX?
Alex is curious about ZYNTEGLO and what potential transfusion independence would mean for him. He wonders if there's anything that could help him reduce his need for regular blood transfusions. Discussing the possibilities with ZYNTEGLO allows Alex to work with his doctor to reach out to a Qualified Treatment Center to gather information on the benefits and risks of gene therapy.
Patient profiles do not encompass all characteristics for ZYNTEGLO eligibility.
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