Transcript IEWP Business Meeting

IEWP – EBMT 2015
Business Meeting
Monday 08.00 23rd March 2015
#EBMT2015
www.ebmt.org
Agenda
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IEWP sessions at EBMT 2015
Leuven Workshop – “How to transplant a patient with PID” April 24th
IEWP Autumn meeting – Seville October 16th-18th
Metabolic diseases – update
Gene Therapy – update
Recent publications
Update on current studies
New study proposals
News from EBMT/ESID
AOB
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IEWP Sessions – Istanbul 2015
Tuesday 24th March 2015
Paediatrics WP 09.45 – 10.00 - Çamlica Hall
• Marina Cavazzano – Gene therapy clinical trials: Recent progress
Inborn Errors WP - 11.00-12.30 - Çamlica Hall
• Beatriz Morillo-Gutierrez – Treosulfan-based conditioning for CGD
• Giovanna Lucchini – SCT in AML: Does the conditioning regimen
matter? Christina Peters – Myeoablative chemoconditioning for
allogeneic SCT in children with ALL
• Arjan Lankester – HSCT 1968 - 2013 for SCID below age two: an update
by the European Registry (SCETIDE)
• Jaap Jan Boelens – Outcome of Duke-Eurocord study on outcomes of
CBT for leukodystrophy
• Bénédicte Neven - HSCT For Patients With PID In Europe: An Update
By The European Registry (SCETIDE)
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Metabolic Diseases sub-committee - Update
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Gene Therapy Sub-committee - Update
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Publications
• Gungor T, et al. Reduced-intensity conditioning and HLA-matched
haemopoietic stem-cell transplantation in patients with chronic
granulomatous disease: a prospective multicentre study. Lancet.
2014;383:436-48
IF 30.209
• Bontant T, et al. Survey of CMV management in pediatric allogeneic
HSCT programs, on behalf of the inborn errors, infectious diseases and
pediatric diseases working parties of EBMT. Bone Marrow Transplant.
2014;49:276-9
IF 3.466
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Publications
• Angelucci E, et al. Hematopoietic stem cell transplantation in
thalassemia major and sickle cell disease: indications and management
recommendations from an international expert panel. Haematologica.
2014;99:811-20
IF 5.868
• Fuchs S, et al. Patients with T⁺/low NK⁺ IL-2 receptor γ chain deficiency
have differentially-impaired cytokine signaling resulting in severe
combined immunodeficiency. Eur J Immunol. 2014;44:3129-40
IF 4.518
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Publications
• Wehr C, et al. Multicenter experience in hematopoietic stem cell
transplantation in 25 patients with common variable immunodeficiency. J
Allergy Clin Immunol 2015 (in press)
IF 11.248
• Aydin SE, et al. DOCK8 deficiency: clinical and immunological
phenotype and treatment options. J Clin Immunol 2015;35:189-198
IF 2.654
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Update on current studies
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DNA repair defects - outcome of HSCT: an EBMT IEWP/ CIBMTR study –
manuscript draft almost ready to be circulated
HSCT for CD40 ligand deficiency: results from an EBMT IEWP study
– study closed, presented at ESID, to be presented at EBMT, manuscript in
preparation**
The PCID Study: Update And Baseline Features Of The First Fifty Patients ESID presentation, manuscript in process
Nijmegen Breakage Syndrome: clinical and immunological features, long-term
outcome and treatment options. Submitted to J Clin Immunol – revision in
progress
Cord Blood Tx in Leukodystrophies (IEWP/Eurocord) – – Oral presentation at
EBMT
Rituximab post HSCT
Defibrotide treatment for TMA (IEWP/PDWP) – data analysis commencing
Reticular Dysgenesis: International Survey on Clinical Presentation,
Transplantation and Outcome – Manuscript prepared
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Update on current studies
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Retrospective outcome of CGD (Tx or no) correlated with residual NADPH
function (joint IEWP/PIDTC) - commencing
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Analyse the next data set from SCETIDE (SCID/PID) – Oral presentations at
EBMT, Manuscript commencing
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Outcome of HSCT with Treosulfan conditioning for CGD - Oral presentations at
EBMT, Manuscript commencing
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Longterm (>20yr) outcome of HSCT for SCID (IEWP/PIDTC) – ESID fellow
commencing April 2015
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HSCT for VPS45 deficiency - commencing
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IPEX – Outcome +/- HSCT (with PIDTC) – commencing
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New Study Proposals
– HSCT for DOCK8 - commencing
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New Study Proposals
Inborn Errors Working Party – Study Proposal Form
Title of study:
Date submitted:
Principle investigator:
Co-investigators:
Joint IEWP/PDWP/PIDTC/other (delete as appropriate)
Objectives:
Outcomes:
Brief methods (SCETIDE/EBMT data base, new questionnaire etc):
Timeline:
Expected start date:
Expected end date:
Target meetings for presentations:
Target journals for publication:
IEWP study committee comments:
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New Studies
• Leiden Statistical unit – Arjan Lankester update
• Ethical approval
• Ideas for a prospective study………
– CGD Treo or Bu (need Treop PK first)
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