Abstract: INFO39
EARLY PRO-TECT Alport XXL Is Growing Up and Is Going International: A Worldwide Concerted Action to Improve Evidence for Preemptive Start of ACE Inhibitor Therapy in Children With Alport Syndrome
Session Information
- Informational Posters
November 04, 2021 | Location: On-Demand, Virtual Only
Abstract Time: 10:00 AM - 12:00 PM
Category: Pediatric Nephrology
- No subcategory defined
Authors
- Gross, Oliver, Georg-August-Universitat Gottingen Universitatsmedizin, Gottingen, Niedersachsen, Germany
- Boeckhaus, Jan, Georg-August-Universitat Gottingen Universitatsmedizin, Gottingen, Niedersachsen, Germany
Group or Team Name
- EARLY PRO-TECT Alport XXL team international and study group of the German Society of Pediatric Nephrology
Description
The type IV collagen disease Alport syndrome (AS) is the second most common monogenic cause of end-stage renal failure. Children with AS develop renal failure early in life. ACE-inhibition is the first-line off-label therapy and can delay renal failure in a time dependent manner. The EARLY PRO-TECT Alport trial was the first randomized and placebo-controlled trial to evaluate safety and efficacy of ACE-inhibition in children with AS. The trial indicated safety and efficacy of nephroprotective therapy without statistical significance.
The aim of this study is to analyse renal outcome and adverse effects in the longterm observation of children from the EARLY PRO-TECT Alport trial as well as other children in disease stages 0,I,II from the USA, Canada, China, France, England, Spain, South-Korea, Japan, Austria, Switzerland, The Netherlands, India and Germany.
Alport Syndrome disease stages are defined as:
0 Microhaematuria without microalbuminuria
I Microalbuminuria (30-300 mg albumin/gCrea)
II Proteinuria >300 mg albumin/gCrea
III >25% decline of normal creatinine clearance
Study Endpoints:
Primary Endpoint: time to progress of Alport Syndrome to the next disease stage under treatment
Secondary Endpoint:
Time to progress of Alport Syndrome to the next disease stage under treatment compared to children without treatment.
Using a Bayesian evidence synthesis approach, different levels of evidence will contribute to enrich the EARLY PRO-TECT Alport data in order to reach the significant levels for efficacy of pre-emptive start of ACE-inhibitor therapy in children with AS. Our international approach also aims to set up a political statement for the worldwide need for early diagnosis of children with AS.
Funding
- The observational study applied for Goverment funding.