Monitoring and evaluation of the Dutch guideline for treatment of aHUS in children and adults during two years.
ID
Source
Brief title
Condition
- Immune system disorders congenital
- Nephropathies
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
During four years the new guideline will be monitored and evaluation will take
place after these two years. With the new guideline we aim to demonstrate that
dosing regimens can be safely adapted or even discontinued, and hereby
optimizing the treatment of aHUS patients. According the new guideline,
patients will be treated with eculizumab during three months (six-nine gifts)
and after this period therapy will be adjusted or discontinued. Clinical and
laboratorial data of the patients will be evaluated by the national Working
Group aHUS-eculizumab. Data are assembled in an online webbased database,
Castor.
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 measure (PREM) that is aimed to better understand
patient experiences with (expensive) medications.
Background summary
The hemolytic uremic syndrome (HUS) is a rare, but severe thrombotic
microangiopathy (TMA), that is characterized by the trias hemolytic anemia,
thrombocytopenia, and acute renal failure. Atypical HUS (aHUS), an ultra orphan
disease, is seen in 5-10% of all HUS cases (estimated prevalence 1-9/1.000.000
cases per year in the Netherlands), occurs at any age and has a very poor
outcome: mortality in the acute phase of the disease is 2-10% and up to 50% of
patients will develop end stage renal disease. Since end 2012, there is a new
drug, named eculizumab, available for the treatment of aHUS. Eculizumab is a
monoclonal antibody against C5 and subsequently inhibits formation of the
terminal complement complex . With the use of Eculizumab, the outcome
perspectives have drastically improved for patients with aHUS. However, this
drug is very expensive and may cost up to ยค500.000 per adult patient per year
when following the dosing regimen of the European Medicines Agency. Recently a
new guideline concerning therapy in aHUS patients and hereby addressing also
the adjustment and/or discontinuation of eculizumab in aHUS is implemented in
the Netherlands. This enables the physician to adapt the treatment and hereby
the possibility of individualized and personalized therapy.
Study objective
Monitoring and evaluation of the Dutch guideline for treatment of aHUS in
children and adults during two years.
Study design
Multicentre, prospective, observational, cohort study.
Study burden and risks
This observational cohort study, for monitoring and evaluation of
effectiveness of the new guideline for treatment of aHUS, does not include
risks for the participants. For all add-on studies (invasive venapuncture
and/or questionnaire) informed consent is gathered separately. Atypical HUS is
a very rare disease with up to 10-15 new aHUS patients each year of which 3-5
children. In this study, the possibility of personalized therapy is
investigated which makes a substantial difference for the treatment with
eculizumab of these same patients in the future.
Geert grooteplein 10
Nijmegen 6525 GA
NL
Geert grooteplein 10
Nijmegen 6525 GA
NL
Listed location countries
Age
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
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
In other registers
| Register | ID |
|---|---|
| CCMO | NL52817.091.15 |
| OMON | NL-OMON22544 |