EBMT ADWP/IEWP. 3 rd 5 th November 2017 Newcastle, UK IEWP Chairman: A Lankester IEWP Secretary: M Albert. Local Chair: A Gennery

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1 EBMT ADWP/IEWP 3 rd 5 th November 2017 Newcastle, UK IEWP Chairman: A Lankester IEWP Secretary: M Albert ADWP Chairman: J Snowden ADWP Secretary: T Alexander Local Chair: A Gennery

2 Telephone: +44 (0) Fax: +44 (0) Dear Friends and Colleagues Re: Joint ADWP / IEWP Annual Conference 3 rd 5 th November 2017, Newcastle, UK We would like to welcome you all to the 2017 Inborn Errors Working Party meeting, to be held at Newcastle, UK. We hope this will provide an excellent setting for another stimulating, educational and enjoyable IEWP meeting. This edition, the first day of the meeting will be organized in collaboration with the Autoimmune Diseases Working Party. The educational morning session will be dedicated to the interface between autoimmunity and primary immune deficiency. As usual the program is quite full but please contact us in advance in case you would like to present data. We have secured a considerable amount of sponsorship for this conference and once again, there is no registration fee to pay. The only cost is of booking your accommodation and travel. All meals and refreshments are included at the conference for the three days. We would like to thank all those who have sponsored the meeting for their generous contributions. Best wishes, Michael Albert, John Snowden, Tobias Alexander, Andy Gennery

3 Inborn Errors WP/Autoimmune Diseases WP Educational Day Friday 3 rd November : Welcome and scope Summary of IEWP database and activity Summary of ADWP database and activity PID as cause of autoimmunity Case reports by participants Interface between Autoimmunity and PID John Snowden / John Snowden Sophie Hambleton (Newcastle) Tea/Coffee 10:50 Treating AID/PID by allo-sct (prototypes) 11:10 Treating AID/PID with precision medicine 11:30 asct in Crohn s disease: who benefits? 11:50 a-sct in SSc: which patients and how? a-sct in JIA is there a role? 13:35 Patterns/mechanisms of immune reconstitution/modulation Auto-SCT - What have we learned from immune reconstitution studies post auto-hsct in MS, rheumatological diseases and Crohn s disease Allo-SCT - Allo SCT in autoimmune diseases - What have we learned from PID Specific cases/diseases 15:00 Tea/coffee 15: Keynote lecture Allo-HSCT for JIA and autoinflammatory disease Mary Slatter (Newcastle) Stephan Ehl (Freiburg, Germany) Montserrat Rovira (Barcelona) Dominique Farge (St Louis, Paris) Nico Wulffraat (Utrecht) Tobias Alexander (Berlin) Raffaella Greco (Milan) Andy Gennery (Newcastle) Mario Abinun (Newcastle) ADWP business meeting IEWP studies committee meeting Coordinated by John Snowden Coordinated by :00 Evening program - Dinner Palace of Arts, Exhibition Park

4 08:30 SCT in metabolic diseases Hurler (LT) MLD (SCT/GT) Saturday 4 th November 2017 Coordinated by Rob Wynn/Jaap-Jan Boelens 10:00 Tea/coffee DNA repair disorders SCT outcome in NBS/AT Fanconi/GT DNA Ligase I def Malignancies in PID: meeting report 11:45 HSCT for emerging indications - update APDS STAT1-GOF STAT3-GOF LRBA 13:00 Lunch 13:45 PID session pcid update Newborn screening update (various countries) SCETIDE update Coordinated by Claire/Despina Moshous Andy Gennery Claire Booth Austen Worth Coordinated by Bénédicte Neven / Manfred Hönig Coordinated by Mary Slatter/Carsten Speckmann Carsten Speckmann Francesca Ferugia Nizar Mahlaoui/ 15:15 Tea/coffee Hb pathy session Introduced by Josu de la Fuente Keynote Session Georg Hollander (Oxford) 17:30 Finish 19:00 Evening Networking Dinner Discovery Museum 08:30 Haplo/mmUD SCT: novel strategies TCD ex vivo (TCRαβ ) TCD in vivo (PTCy) CD45RA Precursor T co-tx Sunday 5 th November 2017 Coordinated by Michael Albert / Isabelle André- Schmutz Alice Bertaina Jean-Sebastian, Diana Bénédicte Neven Isabelle André-Schmutz Tea/coffee Conditioning-complications-TMA Coordinated by Andy Gennery/Robbert Bredius 11:15 IEWP studies and proposals: SCETIDE PID update Joint IEWP/PIDTC - Long term outcome SCID: - IL7R-/- Others New proposals Coordinated by /Michael Albert Bénédicte Neven 12:30 Final Remarks Andy Gennery,

5 VENUE Conference venue: Crowne Plaza Stephenson Quarter Hawthorn House, Forth Banks Newcastle upon Tyne NE1 3SA For further information please contact: Yvonne Wijkhuisen Roz Scott SPONSORS