Descriptive Analysis of Pediatric Studies Included in the European Union Post-Authorization Study Register from 2010 to 2023.

Background/objectives: This work aimed to analyze pediatric Post-Authorization Studies (PASs) registered in the European Union electronic Register of Post-Authorization Studies (EU PAS Register) from September 2010 to April 2023 to identify trends in terms of timing, age groups, and therapeutic areas and to discuss pediatric specificities and sources of funding for the PASs.

Methods: A screening process identified PASs conducted exclusively on the pediatric population, and instructions were provided to ensure standardized data collection from the EU PAS Register. A univariate linear regression descriptive analysis was performed to assess trends over time, while a multivariate linear regression analysis helped explore additional characteristics of these studies.

Effects of the Paediatric Regulation funding on the development of off-patent medicines in children.

Introduction: In paediatrics, medicines repurposing is a particularly advantageous approach, offering a route to address unmet medical needs and turn off-label use into evidence-based treatments for paediatric populations. This study analysed the effects of funds provided under the Seventh Framework Programme for Research (FP7-FRP), issued by the European Commission from 2007 to 2013 according to the European Paediatric Regulation, in terms of new paediatric marketing authorisations (MAs) including paediatric Use Marketing Authorisations (PUMAs). Additionally, we investigated which funded projects included repurposing initiatives.

Donor-specific anti-HLA antibodies (DSAs) in patients undergoing allogeneic hematopoietic stem cell transplantation from mismatched donors on behalf of GITMO and AIBT.

Background: Antibodies directed against donor-specific HLA allele(s)/antigen(s) (DSAs) represent a known risk factor for hematopoietic stem cell transplantation (HSCT) engraftment. Still, the overall management needs to be standardized.

Material And Methods: GITMO and AIBT ran a survey on DSAs in Italian Transplant Programs including mismatched HSCT performed between January 2014 and June 2017.

Post Transplant Cyclophosphamide as GvHD Prophylaxis in Patients Receiving Mismatched Unrelated HCT: the PHYLOS trial.

Post-transplant high-dose cyclophosphamide (PTCy) is effective in overcoming the negative impact of HLA disparity in the haploidentical setting. In the light of these results, we investigated the efficacy and safety of PTCy, with a calcineurine inhibitor and mycophenolate mofetil, in improving clinical outcomes of haematopoietic cell transplantation (HCT) from mismatched unrelated donor (MMUD) in patients with acute myeloid malignancies by reducing aGvHD incidence and severity. A prospective single arm, phase II study (PHYLOS - NCT03270748) was conducted by the Italian GITMO.

Prospective Validation of CAR-HEMATOTOX and a Simplified Version Predict Survival in Patients with Large B-Cell Lymphoma Treated with Anti-CD19 CAR T-Cells: Data from CART-SIE Study.

Background: Anti-CD19 CAR T-cells have revolutionized outcomes in relapsed/refractory large B-cell lymphomas. Long-term follow-up underscored the role of hematological toxicity in nonrelapse mortality, largely driven by infections, leading to the development of the CAR-HEMATOTOX (HT) score for predicting neutropenia. The European scientific community (EHA/EBMT) later reached a consensus, defining a new entity: immune effector cell-associated hematotoxicity (ICAHT).