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Sickle cell disease is a hereditary multiorgan disease that is considered rare in the EU. In 2017, the Rare Diseases Plan was implemented within the EU and 24 European Reference Networks (ERNs) were created, including the ERN on Rare Haematological Diseases (ERN-EuroBloodNet), dedicated to rare haematological diseases. This EU initiative has made it possible to accentuate existing collaborations and create new ones. The project also made it possible to list all the needs of people with rare haematological diseases not yet covered health-care providers in the EU to allow optimised care of individuals with rare pathologies, including sickle cell disease. This Viewpoint is the result of joint work within 12 EU member states (ie, Belgium, Cyprus, Denmark, France, Germany, Greece, Ireland, Italy, Portugal, Spain, Sweden, and The Netherlands), all members of the ERN-EuroBloodNet. We describe the role of the ERN-EuroBloodNet to improve the overall approach to and the management of individuals with sickle cell disease in the EU through specific on the pooling of expertise, knowledge, and best practices; the development of training and education programmes; the strategy for systematic gathering and standardisation of clinical data; and its reuse in clinical research. Epidemiology and research strategies from ongoing implementation of the Rare Anaemia Disorders European Epidemiological Platform is depicted., Competing Interests: Declaration of interests MdMMP declares grant funding or contracts from EuroBloodNet Association (RADeep) and Agios Pharmaceuticals (PKDeep) and participation in an advisory board for Agios Pharmaceuticals. RC declares grant funding or contracts from Global Blood Therapeutics, EU grants in the framework of Horizon 2020 and Horizon Europe, and EuroBloodNet Association (RADeep); consulting fees from Global Blood Therapeutics and Novartis; honoraria from Physician's Education Resources and Global Blood Therapeutics; participation on a data safety monitoring board or advisory board for Vertex, Addmedica, and Novonordisk; being a member of the steering committee of the Italian Association of Pediatric Hematology Oncology and coordinator of the Red Cell Disorder Working Group. PB declares grant funding or contracts from EuroBloodNet Association (RADeep). EC declares support for the Spanish Registry of Rare Anemias, partly supported by Novartis; grant funding or contracts from Vall d'Hebrin Research Institute (Integra) and EuroBloodNet Association (RADeep); honoraria from Terumo BCT, Chiesi, and Bionest; support for attending meetings or travel to the European Hematology Association Hemoglobinopathy Working Party 2023 by the European Hematology Association and to the Sociedad Española de Hematología y Oncología Pediátricas 2022 by Novartis; and participation on a data safety monitoring board or advisory board of Novartis (Adakveo) and Vertex. AC declares grant funding or contracts from EuroBloodNet Association (RADeep) and consulting fees from Novartis. LD declares grant funding or contracts from EuroBloodNet Association (RADeep). AG declares grant funding or contracts from Bristol Myers Squibb, Novo Nordisk, and EuroBloodNet Association (RADeep) and participation on a data safety monitoring board or advisory board of Novo Nordisk and Agios Pharmaceuticals. VGV declares grant funding or contracts from EuroBloodNet Association (RADeep). AK declares grant funding or contracts from Novartis and BMS; consulting fees from Agios Pharmaceuticals, Amgen, Bristol Myers Squib/Celgene, Crispr/Vertex, Ionis Pharmaceuticals, Novartis, and Vifor; honoraria from Novartis, Bristol Myers Squib/Celgene, Chiesi, and Crisp/Vertex; and support for attending meetings or travel from Bristol Myers Squib/Celgene. JK declares grant funding or contracts from the Deutsche Kinderkrebsstiftung (German Childhood Cancer Foundation), Pfizer, and Novartis; support for attending meetings or travel for American Society of Hematology Annual Meeting 2020 with support from Art Tempi Communications; participation on a data safety monitoring board or advisory board of Novartis, Global Blood Therapeutics, and Bluebird Bio; and leadership or fiduciary role of the Konsortium Sichelzellkrankheit. SL declares consulting fees for advisory board of Novartis, BlueBird Bio, AddMedica, Global Blood Therapeutics, and Agios; honoraria from Novartis and BlueBird Bio; participation on a data safety monitoring board or advisory board of Novartis, BlueBird Bio, AddMedica, Global Blood Therapeutics, and Agios; leadership or fiduciary role at Vertex CTX001 study program steering committee, in the German National Disease Management Program, and in the German National Treatment Guideline. SR declares grant funding or contracts from EuroBloodNet Association (RADeep). GR declares participation on a data safety monitoring board or advisory board of Biovalley. MS declares payment for expert testimony from Novartis; support for attending meetings or travel to ASCAT 2022 from ERN-EuroBloodNet; leadership or fiduciary role as patient representative for the UK in the Red Cell Network, Patient and Public Voice Group. EvB declares grant funding or contracts from Agios Pharmaceuticals, Horizon Europe, Sikkelcelfonds, and EuroBloodNet Association (RADeep); honoraria from Agios and GSK; support for attending meetings or travel from European Rare Disease Research Coordination and Support Action consortium and EuroBloodNet Association (RADeep); participation on a data safety monitoring board or advisory board at Imara, BMS, Agios, and Forma therapeutics; leading the ERN-EuroBloodNet Transversal Field of Action on Clinical Trials and Research for non-oncological rare haematological diseases; and being a board member of the Benign Hematology Section of the Dutch Society for Hematology (NVVH) and chair of the Dutch Patient Registry for Sickle Cell Disease (SCORE). PK declares grant funding or contracts from EuroBloodNet Association (RADeep). BG declares grant funding or contracts from EuroBloodNet Association (RADeep). All other authors declare no competing interests., (Copyright © 2023 Elsevier Ltd. 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In the UK and Northern Europe, ripening oats can become contaminated with T-2 and HT-2 mycotoxins, produced mainly by Fusarium langsethiae. There are indicative levels related to the maximum limits for oat grain for these toxins. The objectives of this study were to examine the effect of interacting conditions of temperature (10-30 °C) and water activity (a w , 0.995-0.90) on (a) lag times prior to growth, (b) growth and (c) T-2 and HT-2 toxins by two strains of F. langsethiae isolated from oats in the UK and compare this with the type strain (Fl201059) which has been genomically sequenced, and (d) develop (and validated with published data) a probabilistic models for impacts of temperature × a w on growth and toxin production. All three strains had an optimum a w range and temperature of 0.995-0.98 and 25 °C for growth. For T-2 + HT-2 production these were 0.995 a w and 20 °C. Overall, the type strain produced higher amounts of T-2 + HT-2 with a HT-2/T-2 ratio of up to 76. Using this study data sets and those from the literature, probabilistic models were developed and validated for growth and T-2 + HT-2 toxin production in relation to temperature × a w conditions. These models, when applied in stored oats, will be beneficial in determining the conditions on the relative level of risk of contamination with these two toxins in the context of the EU indicative maximum levels., (Copyright © 2021 Elsevier B.V. All rights reserved.)

Sickle cell disease (SCD) describes a set of chronic inherited anemias characterized by hemolysis, episodes of vaso-occlusion, and high infectious risk, with high morbidity and mortality. Newborn screening (NBS) for SCD allows family health education and early start of infectious prophylaxis. In the Community of Madrid, a pilot universal NBS study found that the SCA birth prevalence was 1/5851 in newborns, higher than expected, confirming the need to include early detection in the NBS program. The aim of the present prospective single-center study is to analyze the results of newborn SCD screening in Madrid in terms of epidemiological data and its inclusion in a comprehensive care program during the last 15 years, between 1st of May 2003 and 1st of May 2018. During the study period, 1,048,222 dried bloodspots were analyzed. One hundred ninety-seven patients were diagnosed with possible SCD (HPLC phenotype of FS, FSA, FSC, FSE, FSD Punjab , FSO Arab ), with 187 patients finally confirmed (birth prevalence 1/5552 newborns, 0.18 per 1000 live births), and 1 out of 213 infants carried Hb S. All of them were seen by a specialist clinician; median age at the first visit consultation was 35 days and median age at the beginning of penicillin treatment was 66 days. The Madrid SCD NBS program achieved high rates of sensitivity and specificity and good quality of care assistance. Establishing a good relationship with the family, a strong education program, and a multidisciplinary team that includes social workers and a psychologist are needed to ensure the success of early intervention.

Sickle Cell Disease (SCD) is an increasing global health problem and presents significant challenges to European health care systems. Newborn screening (NBS) for SCD enables early initiation of preventive measures and has contributed to a reduction in childhood mortality from SCD. Policies and methodologies for NBS vary in different countries, and this might have consequences for the quality of care and clinical outcomes for SCD across Europe. A two-day Pan-European consensus conference was held in Berlin in April 2017 in order to appraise the current status of NBS for SCD and to develop consensus-based statements on indications and methodology for NBS for SCD in Europe. More than 50 SCD experts from 13 European countries participated in the conference. This paper aims to summarise the discussions and present consensus recommendations which can be used to support the development of NBS programmes in European countries where they do not yet exist, and to review existing programmes., (© 2018 British Society for Haematology and John Wiley & Sons Ltd.)