Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease
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Details and patient eligibility
About
The purpose of this study is to examine how abnormal blood flow in the small vessels (microvessels) of the heart, muscle and kidney in paroxysmal nocturnal hemoglobinuria (PNH) or sickle cell disease leads to poor functioning of the heart and kidney. To test this question, the investigators will perform imaging tests (contrast ultrasound perfusion imaging) to look at the flow and function of these microvessels and compare this information to heart and kidney function. To further look at this question, patients who have PNH will be studied before and after starting a new drug (Soliris) that decreases damage to blood cells. In patients with sickle cell disease, patients will be studied at baseline (not during a pain crisis) and also during a pain crisis if one develops.
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
- Established diagnosis of PNH or SCD Age > 18 years old
Exclusion criteria
- Pregnant or lactating women Presence of significant right to left shunting Allergy to ultrasound contrast agent Reactive airways disease Significant peripheral or coronary artery disease
Trial design
6 participants in 3 patient groups
Trial contacts and locations
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Data sourced from clinicaltrials.gov
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