ERN-EuroBloodNet at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition!

The 66th edition of the American Society of Hematology (ASH) annual meeting has been held December 7-10, 2024, in San Diego, California with a great number of ERN-EuroBloodNet experts attending and presenting actions linked to the ERN.

The 66^th edition of the American Society of Hematology (ASH) annual meeting has been held December 7-10, 2024, in San Diego, California with a great number of ERN-EuroBloodNet experts attending and presenting actions linked to the ERN.

The following abstracts carried out in collaboration with ERN-EuroBloodNet have been presented:

• uRADAR: European Patients Referral Frame to Improve Access to New Drugs and Therapies in Ultra-Rare Anemia Disorders and Severe Hereditary Spherocytosis

The Rare Anemia Disorders European Epidemiological Platform (RADeep) together with the ERN-EuroBloodNet have joint efforts to establish the uRADAR initiative aiming to develop a referral frame for patients affected by uRADs in the European Union. The uRADAR ultimate goal is to enable access to clinical trials (CT), including drug repurposing i.e. ERN-EuroBloodNet SATISFY Phase 2 Trial. ERN-EuroBloodNet Scientific Director and RADeep coordinator, María del Mar Mañú Pereira presented the results.

• An Artificial Intelligence-Based Federated Learning Platform to Boost Precision Medicine in Rare Hematological Diseases: An Initiative By GenoMed4all and Synthema Consortia

Genomics and Personalized Medicine for all (GenoMed4All) and Synthetic Generation of Haematological Data Over Federated Computing Frameworks (SYNTHEMA) consortia has the goal of developing an innovative FL platform for rare hematological diseases. This platform enables the development of novel Artificial Intelligence (AI) models for personalized medicine without data sharing, to be implemented in the referral centers of ERN-EuroBloodNet. The aims of the project were: 1) to develop robust federated models for personalized prediction using multicentric, real-world datasets; 2) to protect patients' privacy; and 3) to enhance collaboration between institutions while avoiding the creation of centralized data repositories.

• Data Driven Research through the European RADeep Registry and the Use of Artificial Intelligence Towards Personalized Medicine in Sickle Cell Disease

Artificial Intelligence (AI) based models for SCD progression, severity classification, response to treatments and generation of synthetic data are being developed through Genomics and Personalized Medicine for all (GenoMed4All) and Synthetic Generation of Haematological Data Over Federated Computing Frameworks (SYNTHEMA) EU-funded projects in collaboration with ERN-EuroBloodNet.

• Oxygen Gradient Ektacytometry Is Associated with Markers of Hemolysis and Inflammation in a Large Sickle Cell Disease Cohort within the GenoMed4ALL Project

Ongoing studies aim to explore how laboratory data and clinical complications of all patients within the GenoMed4ALL project are associated with oxygen gradient ektacytometry by implementing a generalized hierarchical linear mixed model. This approach will improve our understanding of SCD pathophysiology and enable personalized medicine in SCD.

• Satisfy: A Eurobloodnet Multicenter, Single-Arm Phase 2 Trial of Mitapivat in Adult Patients with Erythrocyte Membranopathies and Congenital Dyserythropoietic Anemia Type II - Results from the 8-Week Dose-Escalation Period

The SATISFY study (Mitapivat in red blood cell membranopathies​) is the first ERN-EuroBloodNet sponsored clinical trial. The trial is now open in Denmark and the Netherlands, providing targeted treatments to patients with hereditary spherocytosis, hereditary stomatocytosis, and congenital dyserythropoietic anemia II. Expansion to other rare hereditary anaemias is foreseen.

Moreover, ERN-EuroBloodNet has been also present in:

• Diagnosis and Management of Hereditary Hemochromatosis: Lifestyle Modification, Phlebotomy, and Blood Donation Session – Presented by Domenico Girelli

We are thankful for the collaboration of the ERN-EuroBloodNet community and particularly to the speakers that represented ERN-EuroBloodNet during the 66^th ASH Meeting!

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