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Scientific report 2020 of the Paediatric Diseases Working Party (PDWP)

Major achievements

The year 2020 was particularly embossed by the need to switch all meeting to virtual events. Despite these major changes, the PDWP maintained a high number of oral presentations during the annual meeting (eight = identical to the previous year).

The scientific output in 2020 increased further compared to 2019 (IF 86.01) and reached an all-time. The most important publication of this year are the results of the randomized FORUM trial in childhood ALL in the Journal of Clinical Oncology by Christina Peters et al, clearly demonstrating the superiority of TBI over chemotherapy-based conditioning. Albeit surprising and disappointing, it is a seminal publication and a paradigm for the power of prospective clinical trials and evidence-based medicine.

Another highlight was the open-access publication of 12 articles plus an Editorial, summarizing the major contributions from the Educational Meeting on Sickle Cell Disease in Regensburg 2019 in a Special Issue Edition of Hematology/Oncology and Stem Cell Therapy.

The PDWP maintains strong links and interacts with the IEWP and the SAAWP resulting in very important collaborations and consensus manuscripts on pediatric issues. With the unequivocal commitment for education the Midterm Focus Meeting on non-malignant diseases, from July 8th to 10th, was a huge collaborative success of the PDWP, IEWP and the SAAWP. With 46 speakers and chairs, albeit virtually, it attracted more than 370 attendees from over 40 countries and in particularly the Q&A sessions received an enormous interest from the scientific community.

The Education Program for 2021 foresees new online formats such as educational sessions with expert presentations and case conferences for discussion of complex clinical cases.

Moreover, the Cancer Predisposition Meeting originally planned for 2020, will be held in October 2021.

Principal research studies

  1. Outcome after graft failure of allogeneic HSCT in patients with SCD. PI: Josu de la Fuente
  2. A phase II stratified trial to assess haploidentical T-depleted stem cell transplantation in patients with sickle cell disease with no available sibling donor (prospective). PI: Selim Corbacioglu
  3. Haploidentical stem cell transplantation using PTCY for children with acute leukemia. PI: Annalisa Ruggeri
  4. Late effects after hematopoietic stem cell transplantation in patients with HLH. PI: AnnaCarin Horne
  5. Eculizumab for Thrombotic Microangiopathy. PI: Peter Svec
  6. The use of TPO agonist post pediatric HSCT. PI: Asaf Yanir
  7. ABO incompatibility in hemoglobinopathies. PI: Katharina Kleinschmidt
  8. Allogeneic HSCT for BCR/ABL1-negative myeloproliferative neoplasms in children. PI: Jacek Wachowiak
  9. Trend and outcome of HSCT in Thalassemia inside the EBMT. PI: Baronciani Donatella, Emanuele Angelucci
  10. Impact of HLA-B polymorphism on outcomes of stem cell transplantation for Sickle Cell Disease. PI: Eliane Gluckman, Barbara Capelli
  11. ALL-SCT Forum Trial (prospective). PI: Christina Peters

Impact factor_(Pediatric Diseases)

  2015 2016 2017 2018 2019 2020
Oral presentations 6 8 6 5 8 8
Poster presentations 3 7 1 7 1 0
International educational events 2 1 1 3 1 1

Major educational courses

  1. Midterm Focus Meeting on Non-Malignant Diseases - 8-10 July, 2020 / [Virtual].