Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease

Oregon Health and Science University6 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Rheologic Disease Sickle Cell Disease Paroxysmal Nocturnal Hemoglobinuria

Interventions

Eligibility

Sex: ALLMin age: 19 YearsHealthy volunteers:

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Microvascular perfusion

1. Myocardial microvascular blood flow (MBF) and capillary blood velocity (CBV) on study day. 2. Renal MBF on study day 3. Skeletal muscle MBF and CBV on study day 4. Myocardial MBF and CBV during hyperemia 5. Skeletal muscle CBV during hyperemia

Time frame: 1 hour