Inborn Errors Working Party (IEWP)
IEWP Newsletter - August 2015
by Andrew Gennery
Our first meeting this year was in Istanbul at the EBMT Spring meeting – where we held two very successful sessions as part of the Paediatric Day. Work is already underway in planning the programme for next years EBMT meeting in Valencia – abstract deadlines are likely to be similar to normal – around early-mid December.
Isabelle Meyts hosted a one day workshop in Leuven in April on 'How To Transplant A Patient With Primary Immunodeficiency' the audience was small but there was the usual lively and interactive discussion – we are working on publishing the proceedings as a ‘How I do…’ paper. Our Autumn meeting this year is in Seville, Spain in October – the programme is almost ready (but of course subject to change….!) – and can be found on the ESID website. I encourage you to register for the meeting with Roz ( ), and to book accommodation. As normal, there is no registration fee, but you will have to fund your accommodation and travel. We are delighted that Luigi Notarangelo will be giving the keynote lecture. On the Friday morning we will be running a selected highlights of the 'How To Transplant A Patient With Primary Immunodeficiency' – again contact Roz or Olaf Neth (firstname.lastname@example.org) for details.
We are continuing to present and publish the results of our studies. Recent publications, previously in press, include:
Wehr C, et al. Multicenter experience in hematopoietic stem cell transplantation in 25 patients with common variable immunodeficiency. J Allergy Clin Immunol 2015;135:988-97.e6.
Aydin SE, et al. DOCK8deficiency: clinical and immunological phenotype and treatment options. J Clin Immunol 2015;35:189-98
Also, just accepted for publication:
Slatter MA, Boztug H, Pötschger U, Sykora K-W, Lankester A, Yaniv I, Sedlacek P, Glogova E, Veys P, Gennery AR, Peters C on behalf of the EBMT Inborn Errors and Paediatric Diseases Working parties. (2015) Treosulfan-based conditioning regimens for allogeneic Haematopoeitic stem cell transplantation in children with non-malignant diseases. Bone Marrow Transplant (in press)
I remind you to ‘badge’ multi-centre studies with IEWP label, where possible.
We have started a survey on longterm outcome of HSCT for SCID > 20 years in the coming months – a joint project with PIDTC in North America. We are also planning a prospective non-inferiority study on outcome of WAS and CGD with a busulphan/fludarabine or treosulfan/fludarabine based regimen – further details will be discussed in October at the Autumn meeting.
I wish you a restful summer period.
With Best Wishes and I look forward to seeing you in the near future