Sickle cell disease (SCD) is a severe non-malignant disorder of hemoglobin and is inherited in an autosomal-recessive manner

Sickle Cell Disease (SCD) is the most common monogenic disorder globally but qualifies as a rare disease in Germany. In 2012, the German Society for Paediatric Oncology and Haematology (GPOH) mandated a consortium of five university hospitals to develop a disease management program for patients with SCD. Besides other activities, this consortium issued treatment guidelines for SCD that strongly favour the use of hydroxyurea and propagated these guidelines in physician and patient education events. In order to quantify the effect of these recommendations, we made use of claims data that were collected by the research institute (WIdO) of the major German insurance company, the Allgemeine Ortskrankenkasse (AOK), and of publicly accessible data collected by the Federal Statistical Office (Statistisches Bundesamt, Destatis). While the number of patients with SCD in Germany increased from approximately 2200 in 2011 to approximately 3200 in 2019, important components of the recently issued treatment guidelines have been largely implemented. Specifically, the use of hydroxyurea has more than doubled, resulting in a proportion of approximately 44% of all patients with SCD being treated with hydroxyurea in 2019. In strong negative correlation with the use of hydroxyurea, the frequency of acute chest syndromes decreased. Similarly, the proportion of patients who required analgesics and hospitals admissions declined. In sum, these data demonstrate an association between the dissemination of treatment guidelines and changes in clinical practice. The close temporal relationship between the increased use of hydroxyurea and the reduction in the incidence of acute chest syndrome in a representative population-based analysis implies that these changes in clinical practice contributed to an improvement in key measures of disease activity.

Lung cancer continues to have one of the highest morbidity and mortality rates of any cancer. Although radiochemotherapy, in combination with immunotherapy, has significantly improved overall survival, new treatment options are urgently needed. However, preclinical radiotherapy testing is often performed in animal models, which has several drawbacks, including species-specific differences and ethical concerns. To replace animal models, this study used a micro-extrusion bioprinting approach to generate a three-dimensional (3D) human lung cancer model consisting of lung tumor cells embedded in human primary lung fibroblasts for radiotherapy research. The models were placed in a mouse phantom, i.e., a 3D-printed mouse model made of materials that mimic the X-ray radiation attenuation rates found in mice. In radiotherapy experiments, the model demonstrated a selective cytotoxic effect of X-rays on tumor cells, consistent with findings in 2D cells. Furthermore, the analysis of metabolic activity, cell death, apoptosis, and DNA damage-induced γH2AX foci formation revealed different results in the 3D model inside the phantom compared to those observed in irradiated models without phantom and 2D cells. The proposed setup of the bioprinted 3D lung model inside the mouse phantom provides a physiologically relevant model system to study radiation effects.

The epidemic of childhood allergic disorders has been associated to the decline of infectious disease. However, exposure to many triggers (airborne viruses, tobacco smoke, pollution, indoor allergens, etc.) contribute to the disease. Breast feeding practices, nutrition, dietary and obesity also play a multifaceted role in shaping the observed worldwide trends of childhood allergies. Guidelines for treatment are available, but their implementation is suboptimal. Then developed countries are slowing learning integrating the development of suitable guidelines with implementation plans. Awareness, psychosocial and family factors strongly influence asthma and food allergy control. Moreover, monitoring tools are necessary to facilitate self-management. By taking into consideration these and many other pragmatic aspects, national public health programs to control the allergic epidemic have been successful in reducing its impact and trace the need for future research in the area.

In recent years, Germany has become one of the most popular destinations for international migrants and refugees, mostly from countries with a high prevalence of haemoglobinopathies. The concurrent lack of awareness among healthcare professionals, of systematic data, and of targeted screening carry the risk of disparities in patient care. In this context, a change in public health policy is urgently needed. In this article, we argue for a tailored prenatal screening algorithm supported by an analysis of open-source data showing a significant increase in pregnancies at risk of carrying a haemoglobinopathy. We also advocate for multifaceted interventions including the expansion of counselling services, healthcare professional education, community engagement and dedicated research funding to establish a robust framework for the management of haemoglobinopathies in Germany.

Zusammenfassung Antisense-Oligonukleotide (ASO) haben sich als vielversprechende Therapieoption für seltene genetische Erkrankungen erwiesen. Ihre patientenspezifische Anpassung ist jedoch mit erheblichen zeitlichen und finanziellen Herausforderungen verbunden und stellt eine große Hürde bei der Entwicklung von Behandlungen seltener Erkrankungen. Eine neue Studie von J. C. Means et al. (Nature (2025) 638: 237) stellt eine skalierbare Plattform vor, die die präklinische Evaluation von personalisierten ASOs in patientenabgeleiteten Organoiden ermöglicht.