This symposium took place on 5th December 2016 in Vienna, Austria
Chairpersons: Rachel J. Bishop,1 Katharina Hohenfellner,2 Hong Liang3
Speakers: Alexey Tsygin,4 Roser Torra5
1. National Eye Institute, National Institutes of Health, Washington DC, USA
2. LÄ Kindernephrologie, Klinikum Traunstein, Traunstein, Germany
3. Service III de l’hôpital des Quinze-Vingts, Equipe S12, Institut de la vision, Paris, France
4. NCZD Institute of Pediatrics, Moscow, Russia
5. Inherited Kidney Disorders, Nephrology Department, Fundació Puigvert, Instituto de Investigaciones Biomédicas Sant Pau (IIB-Sant Pau), Universitat Autònoma de Barcelona, REDinREN, Instituto de Investigación Carlos III, Barcelona, Spain
Disclosure: Dr Roser Torra has received consultancy and speaker fees from Alexion, Amicus, Genzyme-Sanofi, Novartis, Orphan Europe-Recordati, Otsuka, and Shire. Dr Hong Liang has received consultancy and speaker fees with Orphan Europe-Recordati. Dr Rachel J. Bishop, Prof Katharina Hohenfellner, and Dr Alexey Tsygin have declared no conflicts of interest.
Acknowledgements: Writing assistance was provided by Lynda McEvoy, ApotheCom.
Support: The publication of this article was funded by Orphan Europe-Recordati. The views and opinions expressed are those of the authors and not necessarily Orphan Europe-Recordati.
Citation: EMJ Nephrol. 2017;5[Suppl 4]:2-9.
This 1-day meeting was held at the Austria Trend Savoyen Hotel, Vienna, Austria. The morning session consisted of a series of presentations by experts in the field of cystinosis. Prof Katharina Hohenfellner gave an overview of nephropathic cystinosis (NC) and provided her own experience of a multidisciplinary patient follow-up. Dr Alexey Tsygin provided his long-term experience of treatment with cysteamine and highlighted the importance of treating early and the role of cysteamine in treating and preventing long-term complications of cystinosis. Dr Roser Torra presented the findings of the T-CiS.bcn project, which aimed to develop a transition plan from paediatric to adult care through a multidisciplinary team. Dr Rachel Bishop provided an overview of the ocular manifestations of cystinosis followed by a presentation on the most recent data with gel-like Cystadrops® from Dr Hong Liang.
The key themes from the morning sessions were:
- With the development of specific treatment for cystinosis, the disease paradigm has shifted from a paediatric illness where patients suffer end-stage renal disease (ESRD) by 10 years of age to a longerterm multisystemic illness with extra-renal complications
- Earlier treatment of NC with cysteamine can delay both the renal and extra-renal manifestations of the disease
- There are difficulties in transitioning from paediatric to adult care and this can lead to reduced treatment adherence; a multidisciplinary team may help to smooth the transition