How many patients with thalassemia do you actively manage?

Are you knowledgeable about the clinical data for Aqvesme (mitapivat) in thalassemia, Pyrukynd (mitapivat) in pyruvate kinase deficiency, and mitapivat in sickle cell disease?

• Yes
• No

How frequently do you typically see your thalassemia patients?

• Quarterly
• Bi-annually
• Annually
• Something else, please specify

Approximately what percentage (%) of your thalassemia patients are transfusion-dependent (TDT)?

What percentage (%) of your transfusion-dependent thalassemia (TDT) patients could reasonably benefit from an additional agent beyond standard care?

What percentage (%) of your non-transfusion-dependent thalassemia (NTDT) patients could reasonably benefit from an additional agent beyond standard care?

To what percentage (%) of your transfusion-dependent thalassemia (TDT) patients would you offer Aqvesme (mitapivat)?

To what percentage (%) of your non-transfusion-dependent thalassemia (NTDT) patients would you offer Aqvesme (mitapivat)?

What is the primary reason a transfusion-dependent thalassemia (TDT) patient would not receive Aqvesme (mitapivat)?

• Safety/tolerability concerns on liver injury
• Non-liver safety/tolerability concern (e.g. headache, reproductive hormone changes, drug-drug interactions, etc)
• Efficacy concerns on lack of functional benefit (i.e. transfusion burden reduction)
• Payor restrictions on reimbursement without functional benefit (i.e., transfusion burden reduction)
• Burden of the REMS program
• Something else, please specify

What is the primary reason a non-transfusion-dependent thalassemia (NTDT) patient would not receive Aqvesme (mitapivat)?

• Safety/tolerability concerns on liver injury
• Non-liver safety/tolerability concern (e.g. headache, reproductive hormone changes, drug-drug interactions, etc)
• Efficacy concerns on lack of functional benefit (i.e. fatigue score improvement)
• Payor restrictions on reimbursement without functional benefit (fatigue score improvement)
• Burden of the REMS program
• Something else, please specify

To what percentage (%) of transfusion-dependent thalassemia (TDT) patients do you plan to offer Aqvesme (mitapivat) in the first 12 months?

To what percentage (%) of non-transfusion-dependent thalassemia (NTDT) patients do you plan to offer Aqvesme (mitapivat) in the first 12 months?

Do you plan to reach out proactively to transfusion-dependent thalassemia (TDT) patients to offer Aqvesme (mitapivat)?

• Yes — I will proactively reach out to eligible patients
• I will discuss it only if raised by the patient
• No — I will not actively offer it
• Not sure

Do you plan to reach out proactively to non-transfusion-dependent thalassemia (NTDT) patients to offer Aqvesme (mitapivat)?

• Yes — I will proactively reach out to eligible patients
• I will discuss it only if raised by the patient
• No — I will not actively offer it
• Not sure

To what extent would lack of approval of mitapivat in sickle cell disease influence your use of Aqvesme (mitapivat) in thalassemia?

• Not at all–I consider mitapivat’s use case in thalassemia distinct from its use case in sickle cell disease
• Partially–Use of mitapivat in thalassemia could partly be influenced by non-approval in sickle cell disease
• Largely–I consider mitapivat’s use case in thalassemia highly correlated to its use case in sickle cell disease
• Something else, please specify