Atypical Hemolytic-Uremic Syndrome (aHUS) Registry

Conditions

Atypical Hemolytic-Uremic Syndrome

Keywords

Atypical Hemolytic-Uremic Syndrome, aHUS, Thrombotic Microangiopathy, TMA

Brief summary

Post-marketing safety data on patients treated and untreated with eculizumab or ravulizumab.

Detailed description

The study will capture post-marketing safety data on patients treated with eculizumab or ravulizumab. Additionally, the study will collect information on the progression of disease in all patients.

Anja Gaeckler, Imad Al‐Dakkak, Nuria Saval, Hans Dieperink, Margriet Eygenraam et al. (8 authors)

Clinical Transplantation2025-08-28

10.1111/ctr.70278

Global aHUS Registry Analysis of Patients Switching to Ravulizumab From Eculizumab.

Schaefer F, Al-Dakkak I, Anokhina K, Cohen D, Greenbaum LA, Ariceta G.

2024-06-207 citations

10.1016/j.ekir.2024.06.020

#243 Characteristics and safety in patients with atypical haemolytic uraemic syndrome switching to ravulizumab from eculizumab: a global registry analysis

Franz Schaefer, Imad Al‐Dakkak, Nuria Saval, David J. Cohen, Laurence Greenbaum et al. (6 authors)

Nephrology Dialysis Transplantation2024-05-01

10.1093/ndt/gfae069.011

Characterization of Patients with aHUS and Triggering/Associated Events, with and without Complement Pathogenic Variants or Anti-Cfh Antibodies: A Global aHUS Registry Analysis

Andrew M. Siedlecki, Imad Al‐Dakkak, Katerina Anokhina, Nicole M. Isbel, Véronique Frémeaux‐Bacchi et al. (12 authors)

Blood2022-11-151 citations

10.1182/blood-2022-155814

Clinical characteristics and outcomes of a patient population with atypical hemolytic uremic syndrome and malignant hypertension: analysis from the Global aHUS registry

Jean‐Michel Halimi, Imad Al‐Dakkak, Katerina Anokhina, Gianluigi Ardissino, Christoph Licht et al. (10 authors)

Journal of Nephrology2022-09-2424 citations

10.1007/s40620-022-01465-z

Résultats à long terme chez les patients transplantés rénaux traités par l’eculizumab inscrits dans le registre mondial du syndrome hémolytique et urémique atypique

M. Rongy, G. Ariceta, I. Al-Dakkak, K. Anokhina, G. Ardissino et al. (10 authors)

Néphrologie & Thérapeutique2022-09-01

10.1016/j.nephro.2022.07.211

FC 115: Long-Term Outcomes in Eculizumab-Treated Kidney Transplant Patients Enrolled in the Global Atypical Haemolytic Uraemic Syndrome Registry

María Gema Ariceta Iraola, Imad Al‐Dakkak, Katerina Anokhina, Gianluigi Ardissino, Laurence Greenbaum et al. (9 authors)

Nephrology Dialysis Transplantation2022-05-011 citations

10.1093/ndt/gfac123.004

Pregnancy in Women with Atypical Hemolytic Uremic Syndrome

Éric Rondeau, Gianluigi Ardissino, Marie‐Pierre Caby‐Tosi, Imad Al‐Dakkak, Fádi Fakhouri et al. (8 authors)

The Nephron journals/Nephron journals2021-09-0724 citations

10.1159/000518171

MO107CLINICAL CHARACTERISTICS OF A PATIENT POPULATION WITH ATYPICAL HAEMOLYTIC URAEMIC SYNDROME AND MALIGNANT HYPERTENSION: THE GLOBAL AHUS REGISTRY ANALYSIS

Jean‐Michel Halimi, Imad Al‐Dakkak, Katerina Anokhina, Gianluigi Ardissino, Christoph Licht et al. (10 authors)

Nephrology Dialysis Transplantation2021-05-01

10.1093/ndt/gfab106.0016

Pregnancy-triggered atypical hemolytic uremic syndrome (aHUS): a Global aHUS Registry analysis

Fádi Fakhouri, Marie Scully, Gianluigi Ardissino, Imad Al‐Dakkak, Benjamin L. Miller et al. (6 authors)

Journal of Nephrology2021-04-0753 citations

10.1007/s40620-021-01025-x

Systemic Involvement at Entry into the Global Atypical Hemolytic Uremic Syndrome (aHUS) Registry

Marie Scully, Sally Johnson, Varant Kupelian, Laurence Greenbaum

Blood2016-12-022 citations

10.1182/blood.v128.22.3729.3729

An innovative and collaborative partnership between patients with rare disease and industry-supported registries: the Global aHUS Registry.

Woodward L, Johnson S, Walle JV, Beck J, Gasteyger C, Licht C, Ariceta G, aHUS Registry SAB.

2016-11-2124 citations

10.1186/s13023-016-0537-5

The global aHUS registry: methodology and initial patient characteristics

Christoph Licht, Gianluigi Ardissino, Gema Ariceta, David J. Cohen, J. Alexander Cole et al. (12 authors)

BMC Nephrology2015-12-0177 citations

10.1186/s12882-015-0195-1

Vers l’amélioration de la compréhension du syndrome hémolytique urémique atypique avec le registre international : caractéristiques des patients inclus

Véronique Frémeaux‐Bacchi, Stuart Johnson, Gema Ariceta, Gianluigi Ardissino, Masahiro Ogawa et al. (6 authors)

Néphrologie & Thérapeutique2014-09-01

10.1016/j.nephro.2014.07.214

Interventions

None listed

Study design

Observational model

OTHER

Time perspective

OTHER

Eligibility

Sex/Gender

ALL

Healthy volunteers

No

Inclusion criteria

* Male or female patients of any age, including minors, who have been diagnosed with aHUS * Patients with or without an identified complement pathogenic variant or anti-complement factor antibody * Able to give written informed consent. Patient or patient's parent/legal guardian must be willing and able to given written informed consent and the patient (if minor) must be willing to give written informed assent \[if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees (IRB/IEC)\]. * ADAMTS13 \> 5%, if performed.

Exclusion criteria

* Hemolytic Uremic Syndrome (HUS) only due to Shiga Toxin producing Escherichia coli (STEC). * Unable to give written informed consent. Patient or patient's parent/legal guardian unable to give written informed consent. Patient (if minor) unable to give written informed assent (if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees \[IRB/IEC\]).

Design outcomes

Primary

Measure	Time frame	Description
Proportion of patients who experience specified events	10 years	To collect and evaluate safety and effectiveness data specific to the use of eculizumab or ravulizumab in aHUS patients.
Time to first and subsequent occurrence of specified events.	5 years	To assess the long term manifestations of thrombotic microangiopathy (TMA) complications of aHUS as well as other clinical outcomes, including morbidity and mortality in aHUS patients, receiving eculizumab or ravulizumab treatment or other disease management approaches

Countries

Australia, Belgium, Canada, Denmark, France, Germany, Israel, Italy, Poland, Russia, South Korea, Spain, Switzerland, Taiwan, Turkey (Türkiye), United Kingdom, United States

Contacts

Primary ContactAlexion Pharmaceuticals, Inc. (Sponsor)

clinicaltrials@alexion.com1-855-752-2356

Outcome results