ational observational study to monitor the new guideline concerning treatment of atypical hemolytic uremic syndrome
Recruiting
- Conditions
- acute kidney failurecomplement mediated hemolytic uremic syndrome1002142910029149
- Registration Number
- NL-OMON44007
- Lead Sponsor
- Radboud Universitair Medisch Centrum
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 50
Inclusion Criteria
1. Patients of all ages, suspected of or diagnosed with aHUS
2. Treated conform the new Dutch guideline for aHUS.
3. Subject and/or his parents is able and willing to sign the Informed Consent before screening evaluations.
Exclusion Criteria
1, Subject and/or his parents is not able or willing to sign the Informed Consent before start of the study.
2. Patients with other etiological forms of HUS than aHUS
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>During four years the new guideline will be monitored and evaluation will take<br /><br>place after these two years. With the new guideline we aim to demonstrate that<br /><br>dosing regimens can be safely adapted or even discontinued, and hereby<br /><br>optimizing the treatment of aHUS patients. According the new guideline,<br /><br>patients will be treated with eculizumab during three months (six-nine gifts)<br /><br>and after this period therapy will be adjusted or discontinued. Clinical and<br /><br>laboratorial data of the patients will be evaluated by the national Working<br /><br>Group aHUS-eculizumab. Data are assembled in an online webbased database,<br /><br>Castor.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Two add-on studies are submitted with this research protocol.<br /><br>1. To gather more inside in the pharmacokinetics and dynamics of eculizumab.<br /><br>2. To test the psychometric properties of the questionnaire: medication-related<br /><br>patient-reported experience measure (PREM) that is aimed to better understand<br /><br>patient experiences with (expensive) medications.</p><br>