Clinical Trial: Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease

Brief Summary: 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.

Detailed Summary:
Sponsor: Oregon Health and Science University

Current Primary Outcome: Microvascular perfusion [ Time Frame: 1 hour ]

  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


Original Primary Outcome: 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


Current Secondary Outcome:

Original Secondary Outcome:

Information By: Oregon Health and Science University

Dates:
Date Received: February 11, 2011
Date Started: February 2011
Date Completion:
Last Updated: October 27, 2014
Last Verified: October 2014