Objective: To analyze in more detail the complement activation and regulation and especially the functional CFH activity following three types of interventions in patients known with HUS and correlate these to clinical findings. The three…
ID
Source
Brief title
Condition
- Renal and urinary tract disorders congenital
- Immune disorders NEC
- Renal disorders (excl nephropathies)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Tests: -Levels of complement factors: C3, C3b, Complement Factor H quantitative
(ELISA), Complement factor I (ELISA), CFB, sC5-9 membrane attack complex,
Presence of C3b on erythrocytes, Functional assay: Complement Factor H, MCP
expression on leucocytes (flow cytometer), tests of coagulation and endothelial
function, Micro endothelial particles, circulating DNA (nucleosomen), FSAP
(factor VII-activating protease.
Secondary outcome
not applicable
Background summary
Haemolytic uremic syndrome (HUS) is characterized by haemolysis,
thrombocytopenia and renal failure. HUS is caused by dysregulation of the
complement system. It accounts for 10% of HUS cases in children and most cases
in adults. The complement system is an ancient important component of the
innate immune system and protects against invading micro-organism or foreign
tissue. However a dysbalance in this system, which can be brought about by
multiple factors, results in the clinical syndrome of HUS. Treatment of HUS is
plasmaferesis of infusion of a monoclonal antibody directed against the C5-9
complex. Both methods are very expensive. Many uncertainties about optimal
dosing and effects of the treatment on the complement system itself remain.
Study objective
Objective: To analyze in more detail the complement activation and regulation
and especially the functional CFH activity following three types of
interventions in patients known with HUS and correlate these to clinical
findings. The three interventions are hemodialysis, plasmapheresis and infusion
of 1200 mg eculizumab (SOLIRIS®).
Study design
Study design: Single Center, single cohort, non-randomized trial.
Study burden and risks
The burden of this study due to withdrawal of a little amount of blood will
be neglect able, the more so since all patients will have an functional access
to the bloodcirculation
Meibergdreef 9
Amsterdam 1105 AZ
NL
Meibergdreef 9
Amsterdam 1105 AZ
NL
Listed location countries
Age
Inclusion criteria
- age more than 12 years
- diagnosis of atypical HUS
- treatment either with hemodialysis, plasmapheresis or eculizumab
- after giving written informed consent
Exclusion criteria
no informed consent
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL41994.018.12 |