Patient Identification

Key factors that inform eligibility

CONSIDER TREATMENT FOR PATIENTS WHO MEET THESE CRITERIA1:

  • Diagnosed with TDT1
  • Who do not have a β00 genotype1
  • Patients ≥12 years of age1
  • Appropriate for HSCT1
  • No HLA–matched related HSC donor available

Consider additional clinical aspects

Which patients are eligible for ZYNTEGLO?

Mila*, 16 YEARS


Mila refuses to accept lifelong transfusions as the only option.

Discover more about Mila and see how she can be a candidate for treatment with ZYNTEGLO.

See Patient Profile

Mila refuses to accept lifelong transfusions as the only option.

Discover more about Mila and see how she can be a candidate for treatment with ZYNTEGLO.

See Patient Profile

Christos*, 32 YEARS


Christos worries about his future with chronic transfusions.

Learn why Christos could be eligible for treatment with ZYNTEGLO.

See Patient Profile

Christos worries about his future with chronic transfusions.

Learn why Christos could be eligible for treatment with ZYNTEGLO.

See Patient Profile

*These are not real patients

Consider additional clinical aspects

Further examining a patient’s full clinical picture, including iron overload and comorbidities, is important to inform eligibility for treatment with ZYNTEGLO.

Iron overload is cited as a leading reason for ZYNTEGLO referral.2

However, the impact of iron overload should be evaluated. While high Liver Iron Concentration (LIC) does not eliminate a patient from being a candidate, it does introduce additional precautions.1

While ZYNTEGLO does not directly reduce iron, the elimination of RBC transfusions allows for post-treatment iron management options to clear previously accumulated iron.1

In clinical studies, patients treated with ZYNTEGLO had a range of serum ferritin at baseline of 784 to 22517 pmol/L, a range of liver iron concentration of 1.0 to 41.0 mg/g.1

It is recommended that patients with MRI results demonstrating liver iron content ≥15 mg/g undergo liver biopsy for further evaluation. If the liver biopsy demonstrates bridging fibrosis, cirrhosis, or active hepatitis, HSC transplantation with myeloablative conditioning is not appropriate.1

Similar to the limitations of allo-HSCT, comorbidities and organ damage will impact consideration as patients must be fit for myeloablative conditioning.1

Myeloablative conditioning is not appropriate for patients with TDT who have evidence of severely elevated iron in the heart, i.e., patients with cardiac T2* <10 msec by magnetic resonance imaging (MRI).1

MRI of the liver should be performed on all patients prior to myeloablative conditioning. It is recommended that patients with MRI results demonstrating liver iron content ≥15 mg/g undergo liver biopsy for further evaluation. If the liver biopsy demonstrates bridging fibrosis, cirrhosis, or active hepatitis, HSC transplantation with myeloablative conditioning is not appropriate.1

Patients should be assessed for renal impairment defined as creatinine clearance ≤70 mL/min/1.73 m2 to ensure HSC transplantation is appropriate.1

In clinical studies, all patients treated with ZYNTEGLO had a Karnofsky/Lansky performance score ≥80 and the majority (18/32, 56.3%) had a performance score of 100 at baseline. Cardiac T2* at baseline for all these patients was >20 msec.1

Consider patient's motivation

Patient’s understanding of their disease and dissatisfaction with
their condition and disease progression
Compliance issues with current treatment
Life goals, including transfusion independence
Patient’s understanding of their disease and dissatisfaction with
their condition and disease progression
Compliance issues with current treatment
Life goals, including transfusion independence

Patient motivation can stem from life goals limited by current treatments, compliance issues with transfusions and chelation, level of dissatisfaction with standard of care, and a desire to be free of transfusions. Ultimately, a patient’s motivation for change could play a factor when identifying a candidate.3


Consider discussing the following questions with your patients to help determine if they are ready for a change in their treatment:

  • How does coming into the office for regular transfusions affect your daily life?
  • What type of impact does your treatment have on your family, professional, and social life?
  • Have you been unable to complete parts of your daily routine or other activities?

If they are ready for change, consider presenting all up-to-date available options and appropriate treatments, with the varying risk-benefit profiles, length of treatment, along with any required long-term follow-up.


Patient profiles

MILA*, 16 YEARS

Clinical Presentation

  • β0/β+ IVS-1-6
  • No HLA-matched donor
  • Regular red blood cell transfusion: 15 transfusions/year
  • Chelation with deferasirox
  • Serum ferritin 850 μg/L
  • LIC: 4.5 mg/g dry weight
  • T2* >38 msec
  • No comorbidities

Mila*, 16 YEARS


Clinical Presentation

  • β0+ IVS-1-6
  • No HLA-matched donor
  • Regular red blood cell transfusion: 15 transfusions/year
  • Chelation with deferasirox
  • Serum ferritin 850 μg/L
  • LIC: 4.5 mg/g dry weight
  • T2* >38 msec
  • No comorbidities

Patient Journey


Mila doesn’t remember a time in her life where she wasn’t receiving transfusions. Just last year, she had 18 transfusions in total. During the week prior to Mila’s transfusion, she feels tired and has difficulties with her normal activities. 

Mila is compliant with her treatments and maintains a positive attitude. Chelation has allowed her iron levels to be adequately controlled. When Mila was younger, her parents explored HSCT. However, they did not pursue this treatment because no matched sibling donor was available.

Mila does not want transfusion dependence to affect her future. Transfusions are a burden for Mila, and she dreams of entering university and not having to deal with transfusions. She has become increasingly involved in her care plan and continues to inquire about new treatments.

Recently, her doctor suggested a one-time gene therapy called ZYNTEGLO, which has the potential to make Mila transfusion independent. Mila understands ZYNTEGLO treatment will require chemotherapy and is prepared for its side effects and for fertility preservation. 

Are you treating patients like Mila who want to be transfusion independent?

CHRISTOS*, 32 YEARS

Clinical Presentation

  • Codon 44 (-C)/IVS-I-6 (T>C)
  • Transfusion: 17 transfusions/year
  • Deferasirox 1440 mg
  • Splenomegaly
  • Serum ferritin 975 μg/L
  • LIC: 14.5 mg/g dry weight
  • T2* >40 msec

CHRISTOS*, 32 YEARS


Clinical Presentation

  • Codon 44 (-C)/IVS-I-6 (T>C)
  • Transfusion: 17 transfusions/year
  • Deferasirox 1440 mg
  • Splenomegaly
  • Serum ferritin 975 μg/L
  • LIC: 14.5 mg/g dry weight
  • T2* >40 msec

Patient Journey


Christos started transfusions during his first year of life. At times he struggles with his treatment, especially with adherence to his chelation, which he always disliked for tolerability reasons. Due to this, Christos’ LIC has been increasing in the past few years.

Right now, Christos works at his family’s business and looks forward to the day when he will manage the family business on his own. His family has always supported him, but he worries about complications due to transfusions, and how this can affect his ability to take care of his family and the business.

Christos’ haematologist suggested ZYNTEGLO, a one-time gene therapy, approved for patients like him.

Do you have patients like Christos that are worried about their future with chronic transfusions?
*These are not real patients
CONTRAINDICATIONS
  • Hypersensitivity to the active substance or to any of the excipients
  • Pregnancy and breastfeeding
  • Previous treatment with HSC gene therapy
  • Contraindications to the mobilisation agents and the myeloablative conditioning agent must be considered
Please consult the full EU Summary of Product Characteristics (EU SmPC) and the EU Abbreviated Prescribing Information (EU API) before prescribing.