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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

6 participants

Study Classification

OBSERVATIONAL

Study Start Date

2011-02-28

Study Completion Date

2014-10-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Rheologic Disease Sickle Cell Disease Paroxysmal Nocturnal Hemoglobinuria

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

PROSPECTIVE

Study Groups

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Control

Age matched healthy subjects

Imaging

Intervention Type OTHER

Contrast ultrasound perfusion imaging and complete echocardiography

Sickle Cell Patients

Patients with established SCD

Imaging

Intervention Type OTHER

Contrast ultrasound perfusion imaging and complete echocardiography

Paroxysmal Nocturnal Hemoglobinuria patients

Patients with PNH

Imaging

Intervention Type OTHER

Contrast ultrasound perfusion imaging and complete echocardiography

Interventions

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Imaging

Contrast ultrasound perfusion imaging and complete echocardiography

Intervention Type OTHER

Eligibility Criteria

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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

Minimum Eligible Age

19 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes