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ational observational study to monitor the new guideline concerning treatment of atypical hemolytic uremic syndrome

Conditions
atypische hemolytisch uremisch syndroom, atypical hemolytic uremic syndrome, complement system, eculizumab, restrictive treatment regimen, orphan drugs, weesgeneesmiddel
Registration Number
NL-OMON22544
Lead Sponsor
Radboud university medical center
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Pending
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.

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 non invasive
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Monitoring and evaluation of the Dutch guideline for treatment of aHUS in children and adults during two years.
Secondary Outcome Measures
NameTimeMethod
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 patient-reported experience measures (M-PREM) and the expectations measure that are aimed to better understand patient expectations, experiences and satisfaction with medications.
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