Paroxysmal Nocturnal Haemoglobinuria
Conditions
Brief summary
Paroxysmal nocturnal haemaoglobinuria (PNH) is a rare disease which results in breakdown of the red blood cells and bone marrow failure. It is associated with an increased risk of blood clots. Until recently, treatment has been with blood transfusions and in patients with a blood clot, blood thinners. A new treatment called eculizumab is now standard for patients who require regular blood transfusions. It works in the majority of patients by preventing the breakdown of red blood cells. This can eliminate the need for blood transfusion and reduce the risk of blood clots. It is not well understood why patients with PNH are at high risk of blood clots. The investigators plan to use specialised blood tests to assess the stickiness of the blood before starting eculizumab treatment and monthly after starting treatment. The investigators will compare these tests with standard tests of clotting.
Interventions
None listed
Sponsors
King's College Hospital NHS Trust
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Presence of a PNH clone
Exclusion criteria
* Longterm anticoagulation for previous venous thrombosis
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To establish the role of thrombin generation in assessing the prothrombotic phenotype of PNH | After sample is obtained |
Secondary
| Measure | Time frame |
|---|---|
| To establish the role of thromboelastometry in evaluating the prothrombotic phenotype of PNH | After sample is obtained |
| To establish effects of eculizumab treatment on thrombin generation and thromboelastometry | 3-6 months |
Countries
United Kingdom
Outcome results
None listed