Thromboprophylaxis in congenital nephrotic syndrome: 15 year experience from a national cohort

Dobbie, L. J., Lamb, A., Eskell, L., Ramage, I. J. and Reynolds, B. C. (2021) Thromboprophylaxis in congenital nephrotic syndrome: 15 year experience from a national cohort. Pediatric Nephrology, 36(5), pp. 1183-1194. (doi: 10.1007/s00467-020-04793-z) (PMID:33089377)

Abstract

Introduction: Congenital nephrotic syndrome (CNS) is an ultra-rare disease associated with a pro-thrombotic state and venous thromboembolisms (VTE). There is very limited evidence evaluating thromboprophylaxis in patients with CNS. This study aimed to determine the doses and duration of treatment required to achieve adequate thromboprophylaxis in patients with CNS. Methods: From 2005 to 2018 children in Scotland with a confirmed genetic or histological diagnosis of CNS were included if commenced on thromboprophylaxis. The primary study endpoint was stable drug monitoring. Secondary outcomes included VTE or significant haemorrhage. Results: Eight patients were included; all initially were commenced on low-molecular weight heparin (enoxaparin). Four patients maintained therapeutic anti-Factor Xa levels (time 3–26 weeks, dose 3.2–5.07 mg/kg/day), and one patient developed a thrombosis (Anti-Factor Xa: 0.27 IU/ml). Four patients were subsequently treated with warfarin. Two patients maintained therapeutic INRs (time 6–11 weeks, dose 0.22–0.25 mg/kg/day), and one patient had two bleeding events (Bleed 1: INR 6, Bleed 2: INR 5.5). Conclusions: Achieving thromboprophylaxis in CNS is challenging. Similar numbers of patients achieved stable anticoagulation on warfarin and enoxaparin. Enoxaparin dosing was nearly double the recommended starting doses for secondary thromboprophylaxis. Bleeding events were all associated with supra-therapeutic anticoagulation.

• Item Type: Articles
• Status: Published
• Refereed: Yes
• Glasgow Author(s) Enlighten ID: Ramage, Dr Ian and Reynolds, Dr Ben
• Authors: Dobbie, L. J., Lamb, A., Eskell, L., Ramage, I. J., and Reynolds, B. C.
• College/School: College of Medical Veterinary and Life Sciences > School of Medicine, Dentistry & Nursing
• Journal Name: Pediatric Nephrology
• Publisher: Springer
• ISSN: 0931-041X
• ISSN (Online): 1432-198X
• Published Online: 21 October 2020
• Copyright Holders: Copyright © 2020 The Authors
• First Published: First published in Pediatric Nephrology 36(5): 1183-1194
• Publisher Policy: Reproduced under a Creative Commons Licence