SWG Education Activities
2022 was full of educational activities organized by the SWG on Pediatric Hematology.
First of all, EHA SWG on Pediatric Hematology successfully organized a session on “Immunodeficiency-related lymphoproliferative disorders” during EHA2022 Hybrid Congress chaired by Simon Bomken (UK). During the session experts in pediatric hematology presented “Classification of immunodeficiency-related lymphoproliferative disorders” (Daphne de Jong (NL), discussed “Molecular and genetic basis of EBV-induced B-cell lymphoma” (Sylvain Latour (FR) and dived deeper into the “Treatment of immunodeficiency-related lymphoproliferative disorders” presented by Andishe Attarbaschi (AT).
Moreover, 10th EHA Pediatric Hematology Oncology Course was organized in Sorrento, October 2022.
A very successful 10th 'Focus in Pediatric Hematology-Oncology' course was held from October 19th-22nd 2022 in Sorrento in the same family-run hotel on the edge of the beautiful Bay of Naples which has supported the course since 2009. Here the warmth of the welcome and weather ensure the perfect atmosphere for learning and sharing of ideas. The course, which is particularly aimed at specialist trainees in pediatric hemato-oncology, continued the same ethos which has inspired each course: the importance of close interactions between the participants and the teaching faculty, a strong emphasis on real cases and the scientific basis of disease and its treatment.
In this 10th course, there were 64 participants from twenty countries across Europe and beyond. The course was designed and led by Professors Andrea Biondi (Monza), Irene Roberts (London), André Baruchel (Paris), Arndt Borkhardt (Dusseldorf) and Josef Vormoor (Utrecht). The invited faculty this year, many of whom have supported the course for many years, included A Attarbaschi (Aus), A Balduzzi (Ita), E Cassinerio (Ita), K-M Debatin (Ger), Jean Donadieu (Fra), B Gibson (UK), J Grainger (UK), S Izraeli (Isr), JH Klusmann (Ger), T Leblanc (Fra), F Locatelli (Ita), M de Montalembert (Fra), C Niemeyer (Ger), C Peters (Aus), K Schmiegelow (Den), and C Zeidler (Ger).
The principal topics covered were acute leukemia, myelodysplasia, bone marrow failure, immunodeficiency disorders, lymphomas, HLH, hematopoietic stem cell transplantation, late effects, red cell disorders, ITP and consultative pediatric hematology, including coagulation and neonatal hematology. Reflecting one of the most important advances in the field in recent years, an inspiring workshop on CAR-T cell therapy led by André Baruchel, Adrianna Balduzzi and Franco Locatelli was held on the final morning of the course. In addition, there were compelling guest seminars by Andrea Biondi 'Leukemia and Lymphoma treatment for children in Low and Middle-Income Countries', Klaus-Michael Debatin ‘Molecular targeting of cell death pathways in leukemia'; and Arndt Borkhardt ‘Primary Immunodeficiency Disorders '.
The emphasis of this course is on problem-solving using real clinical cases and linking basic science to the clinic. Feedback from the participants was very favorable with no negative comments and with particular appreciation of the clinical cases. An additional important objective of the course has always been to create a friendly and informal atmosphere to encourage exchange of scientific ideas for ongoing and future projects between the participants themselves and faculty members. Indeed, these courses continue to lead to exchanges of trainees between countries to experience training and research in pediatric hematology in diverse environments and ultimately, we hope, to benefit the patients we care for.
EHA & I-BFM joint session during the 4th SIOPe meeting held in Valencia on May 8-12, 2023.
Gene-editing approaches in Thalassemia and Sickle Cell Disease
The session was chaired by Maria Ester Bernardo (co-chair of the SWG on Pediatric Hematology of EHA) and included four distinguished speakers in the field of pre-clinical research and clinical transplantation strategies for hemoglobinopathies.
The first speaker was Prof. Giuliana Ferrari from San Raffaele-Telethon Institute for Gene Therapy in Milan. Prof. Ferrari has long-lasting experience and excellent track-record in the development of hematopoietic stem cell gene therapy strategies (HSC-GT) for beta-Thalassemia and has long studied the biology behind this disease. She has given a talk entitled ‘From gene addition to gene editing in the treatment of Hemoglobinopathies’ explaining the huge developments in this field in the last 10-15 years starting from the first application of lentiviral(LV)-based HSPC-GT in beta-Thalassemia to the most recent approaches under study based on gene editing of HSPC.
The second speaker was Prof. Franco Locatelli from the Department of Pediatric Hemato-Oncology of Bambino Gesù Children Hospital in Rome. Prof. Locatelli is a well-known expert in the field of hematopoietic stem cell transplantation for hemoglobinopathies, from the standard approach based on transplantation of allogeneic donor-derived HSC to the clinical application of both LV-based gene addition and gene editing strategies. During his talk Prof. Locatelli has summarized the clinical experience with both gene addition and Crispr-cas9 based gene editing in beta-Thalassemia.
The third speaker was Prof. Selim Corbacioglu from the Department of Pediatric Hemato-Oncology of Children’s Hospital in Regensburg. During his presentation Prof. Corbacioglu has reported the clinical experience with both gene addition and Crispr-cas9 based gene editing in Sickle Cell Disease.
The fourth speaker was Dr. Giulia Hardouin from Imagine Institute in Paris. Dr. Hardouin is actively involved in the development, at pre-clinical level, of novel gene editing strategies for hemoglobinopathies, including those relying on base editors and prime editors. She has reported and summarized the most recent advancements of the research in this field presenting the most promising gene therapy-based strategies that will possibly enter the clinical arena to cure hemoglobinopathies in the next years.
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