Inborn Errors Working Party (IEWP)

IEWP - Newsletter August 2016

by Andrew Gennery

Dear all,

I write as summer approaches to update you on the year to date and inform you of future events.

The IEWP session during 42nd EBMT meeting, Valencia, 3-6 April was well attended - 270 participants were present in the IEWP plenary session and 215 attended the oral abstracts. Alexandra Laberko, a previous  ESID Medium Term Fellow, presented the SCID longterm  follow up study at the Presidential Symposium, and was a winner of ‘EBMT Young Investigator of the Year’

The IEWP educational session during EBMT PD/IEWP Meeting, Rhodes 26-28 May was well attended – Despina Moshaus talked on HSCT for HLH, Mary Slatter talked on HSCT for Monogenic Autoimmunity, Francesca Ferrua presented data on Gene Therapy for ADA – Longterm Results, Shahrzad Bakhtiar presented the joint PDWP/IEWP study on Allo HSCT for Leucocyte Adhesion Deficiency and Robbert Bredius presented Pharmacokinetics/-dynamics of Alemtuzumab in HSCT.

Future meetings this year are:

  1. IEWP sessions during ESID 17th Biennial meeting, Barcelona 21-24 September – workshop sessions on Haplo HSCT for PID and ADA gene therapy update on Thursday morning and Longterm SCID follow up results on Friday.
  2. IEWP Autumn meeting, Ghent, 4-6 November – a draft programme has been circulated, preceded on Friday morning by a half day work shop – ‘Approach to transplant of a patient with metabolic disease’
  3. IEWP 2nd Thymic workshop, Leiden, 11 November – a draft programme has been circulated – contact Roz Gale ( ) if you wish to register as places are limited

Publications 2016

Morillo-Gutierrez B, Beier R, Rao K, Burroughs L, Schulz A, Ewins A-M, Gibson B, Sedláček P, Krol L, Strahm B, Zaidman I, Kalwak K, Talano J-A, Woolfrey A, Fraser C, Meyts I, Müller I, Wachowiak J, Bernardo ME, Veys P, Sykora K-W, Gennery A, Slatter M. (2016) Treosulfan based conditioning for allogeneic haematopoietic stem cell transplantation in children with Chronic Granulomatous Disease. Multicentre experience on behalf of the EBMT Inborn Errors Working Party. Blood 2016;128:440-448

In revision:

Nademi Z, Slatter MA, Dvorak CC , Neven B, Suarez F, Fischer A,  Booth C, Rao K, Laberko A, Bertand Y, Kołtan S, Dębski R, Rodina J, Flood T, Abinun M, Gennery AR, Hambleton S, Ehl S, Cant AJ on behalf of the Inborn Error Working Party of EBMT and ESID.  Haematopoietic Stem Cell Transplant in Patients with Activated PI3K Delta Syndrome. J Allergy Clin Immunol

Yeates L, Slatter MA, Bonanomi S, Lim WIFL, Ong SY, Dalissier A, Barberi W, Shulz A, Duval M, Heilmann C, Willekens A, Hwang HYW, Uderzo C, Bader P, Gennery AR. Use of Defibrotide to treat Transplant-Associated Thrombotic Microangiopathy – a Retrospective Study of the Paediatric Diseases and Inborn Errors Working Parties of the European Society of Blood and Marrow Transplantation. Bone Marrow Transplant

I wish you a restful summer and look forward to meeting you in the Autumn.

Andy Gennery

Chair - IEWP