World Cord Blood Congress IV and Innovative Therapies for Sickle Cell Disease

Chairs: E. Gluckman (Paris), M. Cavazzana (Paris)
Monaco – October 2013, 24-27

Session I: CORD BLOOD STEM CELL

Session II: METHODS OF GRAFT FACILITATION

State of the art – E.J. Shpall
Generation and expansion of human T-lymphoid progenitors from cord blood CD34+ cells in a feeder-cell-free DL-4 culture system – I. André-Schmutz
Building myeloid bridges to enhance engraftment in cord blood transplant and beyond – C. Delaney
Nicord expanded hematopoietic progenitor cells can outcompete the unmanipulated cord blood unit following myeloablative dual umbilical cord blood transplantation in adults with hematological malignancies – J. Kurtzberg
Hematopoietic stem cell expansion to overcome non-engraftment – J. Wagner

Session III: NEW INNOVATIVE THERAPIES

Gene therapy for hemoglobinopathies: obstacles, expectancy, availability – M. Cavazzana
The present and future challenges of gene therapy for sickle cell disease – F. Mavilio
Biomarker – based personalized medicine – S. Paczesny
Engineering cord blood derived T cells to prevent infection after cord blood transplantation – C. Bollard
Cytokine environment analysis can modify the outcome of hematopoietic stem cell transplantation – F. Touzot
Tregs for immune modulation – J. Wagner
Gene expression of hematopoietic stem cells (CD34+) in post-transplant hematopoiesis – F. Frassoni
Assesment of HLA diversity in Marseille cord blood bank – C. Chabannon
Human cord blood (CB) derived unrestricted somatic stem cells (USSCS) significantly improves survival and wound healing in an animal model of recessive dystrophic epidermolysis bullosa (RDEB) – M. Cairo
Combinations of inhibitory receptors are expressed on cd8⁺ t cells in pediatric subjects following bone marrow or umbilical cord blood transplantation – H. Soudeyns
Crosstalk between umbilical cord/cord blood endothelial precursors and mesenchymal stromal cells in promoting new blood vessel formation – S. Watt
Treatment of mesenchymal stromal cells with immunosuppressive drugs increases their immunoregulatory potency – J. Girdlestone
The Italian Cord Blood Platelet Gel project – P. Rebulla

Session IV: ROLE OF HLA AND OTHER GENETIC MARKERS

Overview: State of the art – E. Petersdorf
Role of high resolution HLA typing for cord blood transplantation – M. Eapen
Increasing use of hematopoietic stem cell transplantation for ethnic minorities – M. Oudshoorn
Role of non HLA markers for donor selection – R. Tamouza
Criteria of donor choice for cord blood transplantation – A. Ruggeri

Session V: CORD BLOOD BANKING

State of the art – E. Baudoux
Assessment of parameters affecting the quality of cord blood units – G. Koegler
Updating the cord blood unit inventory to meet current needs – S. Querol
Improving outcomes of cord blood transplantation by screening potency of cord blood units before release from the cord blood bank – J. Kurtzberg
Eurocord registry, a real support for cord blood bank’s accreditation – I. Ionescu
Role of registries to analyze outcomes data – Eurocord – Netcord – V. Rocha

Session VI: INTERNATIONAL OBSERVATORY ON SICKLE CELL DISEASE

Sickle cell disease global perspective – E. Gluckman
Hydroxycarbamide in the treatment of sickle cell disease – M. de Montalembert
Is sickle cell disease an autonomic nervous system disorder: what triggers the transition from steady state to crisis? – T. Coates
Sickle cell disease: genetic etiologies for phenotypic diversity – J. Elion
Registry of pregnant women with sickle cell disease – M. Cavazzana
Hematopoietic stem cell transplant for sickle cell disease – F. Bernaudin
Haplo-identical related hematopoietic stem cell transplant for sickle cell disease – R. Jones
New directions in allogeneic stem and cellular therapy in patients with sickle cell disease – M. Cairo
Nicord expanded hematopoietic progenitor cells bridge hematopoiesis and facilitate engraftment after myeloablative chemotherapy for patients sickle cell disease – J. Kurtzberg
Hematopoietic stem cell transplant for sickle cell disease in Brazil – B. Simoes
Haemoglobinopathies and malaria in Africa – D. Diallo
Epidemiology and treatment of sickle cell in Senegal including hydroxycarbamide, transfusions and chelation
in children – I. Diagne
in adults – S. Diop
Long term outcome of paediatric patients undergoing haploidentical and matched related haematopoietic cell transplantation for sickle cell disease – M. Dallas

Session VII: HOW TO IMPROVE OUTCOME OF UNRELATED CORD BLOOD TRANSPLANT

State of the art – J. Wagner
Optimization of cord blood transplant for hematologic malignancies: graft, conditioning, graft versus host disease – J. Barker
Results of single and double cord blood in adult malignancies: cost efficiency study – A. Ruggeri
Conditioning for cord blood transplant – G. Sanz
Cord blood transplant in children with non malignant disease – E. Gluckman
Cord blood transplantation for adults: what have we learned – D.Weisdorf
Unrelated donor umbilical cord blood transplantation (UCBT) for hemoglobinopathy using reduced intensity conditioning (RIC) – A. Abraham
Outcome analysis of unrelated cord blood transplantation in Korea – Y.H. Lee
Reduced intensity conditioning regimen combined with single unit cord blood transplantation is effective and safe for children with inherited metabolic disorders and combined immunodeficiency diseases – P. Szabolcs
Long term survival in 365 pediatric recipients of unrelated cord blood transplantation (UCBT) with a single 4/6 matched cord blood unit after myeloablative therapy: Impact of HLA and race/ethnicity – V. Prasad
Allogeneic hematopoietic stem cell transplantation (HSCT) for treatment of high risk hemoglobinopathy patients lacking a fully HLA-matched sibling donor using a novel myeloablative, reduced toxicity conditioning regimen – S. Kharbanda

Session VIII: COMPARISON OF CORD BLOOD TRANSPLANTS WITH OTHER SOURCES OF STEM CELLS

Haplo versus cord blood: pros cord blood – J. Wagner
Haplo versus cord blood: pros Haplo – R. Jones
Comparison of cord blood transplants with other sources of stem cells – V. Rocha
Algorythm of donor choice– K. Ballen
The RTN Policy: a comprehensive algorythm of the allogeneic hematopoietic stem cell transplant for adults with hematological malignancies – W. Arcese
Comparison between related haploidentical transplants in children- F. Locatelli

SATELLITE WORKSHOP ON SICKLE CELL DISEASE IN EMERGING COUNTRIES : 14:00 – 17:30

Session I: SICKLE CELL DISEASE IN EMERGING COUNTRIES

Sickle cell disease in India – D. Jain
Networks, research tools and cooperation: the example of network research on sickle cell disease in Central Africa –L. Tshilolo
Treatment with hydroxycarbamide, transfusion and iron chelation: issues specific to Africa – L. Tshilolo
Problems related to the availability of opioids in Africa – E. Kafando

Session II: Words from the family support groups