The Effect of Mitapivat on Cerebral Perfusion and Oxygen Metabolism in Sickle Cell Disease

Conditions

Sickle cell anaemia

Brief summary

To assess the effect of mitapivat on the cerebral oxygen metabolism (CBF) as measured per MRI at 3 and 12 months

Detailed description

CBF will be measured by PCASL in SCD at 3 and 12 months, CVR will be assessed by the administration of acetazolamide which is a carbonic anhydrase inhibitor that produces a powerful cerebral vasodilatory effect comparable to 5% inhaled CO2 at 3 and 12 months, Cerebral metabolic rate of oxygen (CMRO2) is measured by MRI technique by T2 relaxation under spin tagging (TRUST) at 3 and 12 months, TRV, left atrial dilatation and LVEF + global longitudinal strain (GLS) and left ventricular end diastolic diamterer (LVED) hyperdynamic circulation as assed by trans-thoracic echocardiography after 3 and 12 months

Related papers

No related papers found yet.

Interventions

DRUGMITAPIVAT

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
To assess the effect of mitapivat on the cerebral oxygen metabolism (CBF) as measured per MRI at 3 and 12 months	—

Secondary

Measure	Time frame
CBF will be measured by PCASL in SCD at 3 and 12 months, CVR will be assessed by the administration of acetazolamide which is a carbonic anhydrase inhibitor that produces a powerful cerebral vasodilatory effect comparable to 5% inhaled CO2 at 3 and 12 months, Cerebral metabolic rate of oxygen (CMRO2) is measured by MRI technique by T2 relaxation under spin tagging (TRUST) at 3 and 12 months, TRV, left atrial dilatation and LVEF + global longitudinal strain (GLS) and left ventricular end diastolic diamterer (LVED) hyperdynamic circulation as assed by trans-thoracic echocardiography after 3 and 12 months	—

Measure

Time frame

CBF will be measured by PCASL in SCD at 3 and 12 months, CVR will be assessed by the administration of acetazolamide which is a carbonic anhydrase inhibitor that produces a powerful cerebral vasodilatory effect comparable to 5% inhaled CO2 at 3 and 12 months, Cerebral metabolic rate of oxygen (CMRO2) is measured by MRI technique by T2 relaxation under spin tagging (TRUST) at 3 and 12 months, TRV, left atrial dilatation and LVEF + global longitudinal strain (GLS) and left ventricular end diastolic diamterer (LVED) hyperdynamic circulation as assed by trans-thoracic echocardiography after 3 and 12 months

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Countries

Netherlands

Outcome results

None listed