Müller-Ladner U, Lange U
Lehrstuhl für Innere Medizin mit Schwerpunkt Rheumatologie, Justus-Liebig-Universität Gießen, Gießen, Deutschland,
Internist (Berl). 2014 Apr;55(4):377-81. doi: 10.1007/s00108-013-3417-2.
Because the development of a novel drug or therapeutic strategy can be monitored using the respective databases such as clinicaltrials.gov, true surprises in the field of therapeutic advances have become a very rare event. On the other hand, owing to the common variability of the individual entities and the large number of rare diseases within rheumatology and clinical immunology, introducing a novel drug is a very challenging task. This problem also applies specifically to the first phase after approval, as the usually strict in- and exclusion criteria of a clinical trial only match a small portion of the population with the target disease. Therefore, the numerous novel treatments that have been or are about to be introduced into clinical rheumatology are more than welcome. Key examples are rituximab for ANCA-associated vasculitides, ustekinumab for psoriatic arthritis, or interleukin-1 inhibitors for complicated gout--with more to come including a large group of intracellular kinase or extracellular macrophage inhibitors.
由于可以使用诸如clinicaltrials.gov等相应数据库来监测新型药物或治疗策略的研发情况，治疗进展领域真正的意外已变得极为罕见。另一方面，由于个体实体的常见变异性以及风湿病和临床免疫学中大量罕见疾病的存在，引入新型药物是一项极具挑战性的任务。这个问题在批准后的第一阶段也尤为突出，因为临床试验通常严格的纳入和排除标准仅适用于一小部分患有目标疾病的人群。因此，众多已引入或即将引入临床风湿病领域的新型治疗方法备受欢迎。关键例子包括用于抗中性粒细胞胞浆抗体相关血管炎的利妥昔单抗、用于银屑病关节炎的乌司奴单抗，或用于复杂性痛风的白细胞介素-1抑制剂，未来还会有更多，包括一大批细胞内激酶或细胞外巨噬细胞抑制剂。
