INTRODUCTION:Therapeutic apheresis (TA) is an important treatment for certain conditions. TA refers to various procedures in which either plasma (plasmapheresis) or specific blood cell fractions (cyta-pheresis) are removed. These include, among others, extracorporeal photopheresis (EP), erythrocytapheresis (EA), therapeutic plasma exchange (TPE), and therapeutic erythrocyte exchange (TEE). The indications for these procedures in specific diseases are published by the American Society for Apheresis (ASFA) as well as by hematology societies such as the American Society of Hematology (ASH) and the British Society of Haematology (BSH). There are only a few indications for therapeutic apheresis as first-line therapy for hematologic diseases, either as primary treatment or in combination with other therapies, for which strong evidence (Grade 1A-1C) exists. These indications include TPE for symptomatic hyperviscosity syndrome, hyperviscosity syndrome prophylaxis during rituximab therapy for Waldenström's macroglobulinemia, TEE for acute complications of sickle cell disease such as stroke and for the prophylaxis of such events, as well as the use of TPE for thrombotic thrombocytopenic purpura. Less well-known indications with strong evidence include the treatment of erythrocytosis in polycythemia vera and the treatment of iron overload in hereditary hemochromatosis using EA. Similarly, EP is an established treatment option for mycosis fungoides and Sézary syndrome. The aim of this paper is to summarize these indications.
INTRODUCTION:Immune checkpoint inhibitors have fundamentally transformed the treatment of numerous malignant diseases and are now part of standard therapy plans across many tumor entities. With their increasing use, physicians outside specialized oncology centres are more frequently confronted with immune-related adverse events. These adverse events result from enhanced immune activity and can, in principle, affect any organ system. The clinical presentation is often non-specific and ranges from mild symptoms to potentially life-threatening conditions. Moreover, immune-related adverse events may occur not only during treatment but also with a delayed onset after therapy has been disconti-nued. In clinical practice, the early recognition of immune-related adverse events represents a key challenge, particularly in general practice and internal medicine settings. This review provides an overview of the pathophysiology, typical clinical manifestations, and important warning signs of immune-related adverse events. Its aim is to raise awareness of these complications and to promote timely referral for specialized evaluation.
INTRODUCTION:Historically, the discovery of new pharmaceutical therapies has constituted a lengthy and cost-intensive process. Between the initial identification of a drug candidate and successful regulatory approval, 10 to 15 years commonly elapse, with development costs reaching the order of billions of dollars. Research activities, which are still largely empirical in nature, are frequently marked by setbacks, for example due to toxicological findings or insufficient efficacy in preclinical disease models. Technological breakthroughs, such as the sequencing of the human genome, have given rise to largely unmet expectations that medicines could be developed more rapidly and efficiently. Accordingly, expectations for the application of artificial intelligence (AI) in pharmaceutical research and development are high. Indeed, workflows in the preclinical phase are currently being fundamentally transformed by AI. In the following, the individual stages of preclinical research are analysed, and the potential applications of AI are described. In addition, selected critical aspects are discussed to realistically contextualize expectations regarding AI. As a result of the integration of AI, both preclinical and clinical research are undergoing profound transformation. There is justified hope that effective and safe therapies can finally be developed more rapidly and cost-efficiently.
INTRODUCTION:Drug development is undergoing profound change. Accelerated regulatory processes, gene and cell therapies, the use of artificial intelligence, and the increasing use of real-world evidence are fundamentally transforming the pharmaceutical innovation system. These developments offer patients faster access to effective therapies, but at the same time raise significant ethical and regulatory questions: How can the protection of trial participants be reconciled with the desire for rapid innovation transfer? How can an evidence-based risk-benefit assessment be successful when pivotal trials are small and short? What mechanisms ensure the equitable distribution of high-cost therapies in a solidarity-based healthcare system such as Switzerland's? This overview outlines the key trends and provides guidance for practising doctors on the use of innovative medicines.
