No registrations found.
ID
Source
Brief title
Health condition
atypische hemolytisch uremisch syndroom, atypical hemolytic uremic syndrome, complement system, eculizumab, restrictive treatment regimen, orphan drugs, weesgeneesmiddel
Sponsors and support
Intervention
Outcome measures
Primary outcome
Monitoring and evaluation of the Dutch guideline for treatment of aHUS in children and adults during two years.
Secondary outcome
Two add-on studies are submitted with this research protocol.
1. To gather more inside in the pharmacokinetics and dynamics of eculizumab.
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.
Background summary
Atypical hemolytic uremic syndrome (aHUS) is a rare, but severe form of thrombotic
microangiopathy, and is considered the consequence of complement dysregulation. Atypical HUS has
a poor outcome with mortality up to 10% and over 50% of patients developing end stage renal
disease. Since the end of 2012, these outcomes have greatly improved with the introduction of
eculizumab.
The European Medicines Agency has approved eculizumab for the treatment of aHUS
patients. The guideline advocates lifelong treatment. However, there is no hard evidence to support
this advice. Historically, a substantial number of aHUS patients were weaned of plasma therapy,
often without disease recurrence. Moreover, the long-term consequences of eculizumab treatment
are unknown.
Recently, a new guideline concerning therapy in aHUS patients and hereby addressing
therapy adjustment and/or discontinuation in aHUS patients is implemented in the Netherlands. In
this national, multicenter, observational study we will include all aHUS, both pediatric and adults,
patients who are treated conform this new guideline. During four years this guideline will be
monitored and evaluated.
Study objective
-
Study design
Observational study for two years
Intervention
None
Kioa Wijnsma
Radboudumc, huispostnummer 804 Postbus 9101
Nijmegen 6500 HB
The Netherlands
-
CUREiHUS@Radboudumc.nl
Kioa Wijnsma
Radboudumc, huispostnummer 804 Postbus 9101
Nijmegen 6500 HB
The Netherlands
-
CUREiHUS@Radboudumc.nl
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
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
Design
Recruitment
IPD sharing statement
Followed up by the following (possibly more current) registration
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL5833 |
| NTR-old | NTR5988 |
| CCMO | NL52817.091.15 |
| OMON | NL-OMON44007 |