Recordati Rare Diseases Academy

Advancing knowledge in rare diseases:
independent, professional, education and training

Transplantation in inherited metabolic disease

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Lille, France, 8 – 10 June 2017
Thursday, November 17, 2016

Solid organ and bone marrow transplantation have become more prominent in the field of inherited metabolic medicine over the past decade. Major advances in transplantation have allowed partial or complete cure in some inherited metabolic diseases for children and adults. Careful selection and planning are required for optimal survival. Longer term survivors now reveal more questions to be answered in terms of optimal monitoring and follow up. A multidisciplinary approach is essential in selecting and managing cases peri- and post-operatively to achieve good outcomes.

LEARNING OBJECTIVES

  • Insights into the concepts of solid organ and bone marrow transplantation in children and adults with IMD.
  • Understanding indications for different transplant types in specific IMD’s.
  • Understanding current practices peri-and post-operatively in IMD and discuss existing guidelines.
  • To enable participants to present difficult clinical scenarios.
  • To enable participants and faculty to attend targeted workshops with case presentations and interactive discussions.

Institutes of main organisers - providers

  • Evelina Children’s Hospital, St Thomas’ Hospital, London, United Kingdom
  • Reference centre for inherited metabolic diseases, Jeanne de Flandre Hospital, Lille, France
  • Royal Manchester Children’s Hospital, Manchester, United Kingdom
  • Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, United Kingdom

Scientifc Organising Committee

  • Roshni Vara, Evelina Children’s Hospital, St Thomas’ Hospital, London, United Kingdom
  • Karine Mention, Reference centre for inherited metabolic diseases, Jeanne de Flandre Hospital, Lille, France
  • Robert Wynn, Royal Manchester Children’s Hospital, Manchester, United Kingdom
  • Robin Lachman, Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, United Kingdom

Contact RRD Foundation

Cecilia Kellquist
Recordati Rare Diseases Fondation d'entreprise
Immeuble ‘Le Wilson’
70 avenue du Général de Gaulle
92800 PUTEAUX, France
Telephone : +33 1 47 73 86 11
Fax : +33 1 49 00 18 00
Email : ckellquist@rrd-foundation.org

Programme

Thursday 8 June

Solid organ transplantation in IMD

14:00 Liver transplantation - surgical aspects
Nigel Heaton
14:40 Liver transplantation in IMD - an overview
Jerry Vockley
15:50 Hepatocyte transplantation
Anil Dhawan
16:30 Current experience of liver transplantation in PA / MMA
Roshni Vara

Friday 9 June

Solid organ transplantation in IMD

09:00 Transplantation in Glycogen Storage Disease - an update
Philippe Labrune
09:40 Liver transplantation in MSUD - an update
Karine Mention
10:50 Renal transplantation - an overview in IMD
Mohan Shenoy
11:30 Cardiac transplantation in IMD
Jacob Simmonds
13:45 - 17:20 Workshops: 1hr each - participants split in 3 groups
1. Guidelines for management pre, peri and post liver transplantation in UCD and OA
Roshni Vara, Stephanie Grünewald
2. Types of liver transplantation and ideal timing in IMD
Jerry Vockley, Dino Hadzic
3. Pre-transplant preparation of the Hurler patient - patient preparation, donor selection, conditioning therapy
Robert Wynn, Jaap Jan Boelens

Saturday 10 June

Bone marrow transplantation in IMD

08:30 Principles and Practice of Bone Marrow Transplant in Hurler Syndrome and other LSD
Robert Wynn
09:10 Multi-disciplinary follow up of Transplanted Hurler: Setting up a Clinic
Simon Jones
09:50 Transplant in other lysosomal storage disorders
Jaap Jan Boelens
11:00 Towards Gene Therapies of IEM
Maria Ester Bernardo
11:40 X-ALD
Wolfgang Koehler
12:20 Late effects of BMT
Rachel Windsor

 

Registration details

Target audience and participant profile

Experienced metabolists, hepatologists, haematologists, transplant surgeons and any other allied healthcare professional or trainees with an interest in transplantation.

Fees

The standard course fee of 450€ covers:

  • 2 nights hotel accommodation including breakfast
  • Lunch, coffee and dinner during the course

A local fee of 315€ is granted if accommodation is not needed.

Participants are responsible for their own travel arrangements to and from the course. Fees are not refundable

Registration process and deadline

The registration form should be completed on-line and submitted with your curriculum vitae in English. No payment is required at this stage.

Deadline for registration is 10th of April 2017.

Selection criteria and review process

Candidates will be selected based on their background and experience.
The scientific organising committee will review the applications and select participants.
Selection decisions will be announced within 10 days following the deadline for registration.

CME accreditation:

An application will be made for to the EACCME for CME accreditation.