近十年來標靶藥物在視網膜疾病的應用,革命性地改變了這些疾病的治療及病人的預後。在視網膜疾病中,目前最主要的標靶藥物之目標是血管新生因子(vascular endothelial growth factor, VEGF),大幅的改善了許多視網膜疾病患者的視力預後。然而,目前的治療困境在於病患需要反覆接受這類標靶藥物的眼內注射手術,且並非所有病人對藥物治療的反應都一樣好。因此,是否有其他生物標記可以為病患選擇最佳的治療藥物、以及更長效或多重目標的新標靶藥物,都是現階段藥物開發的重點。抗血管新生因子藥物,雖然透過眼內注射局部給藥,可以減少全身藥物曝露之風險,但仍無法完全避免其副作用。此外,一些新的標靶藥物和免疫治療在癌症或其他疾病時的全身性給藥,也可能造成視網膜的副作用。
Recent advances in target therapy have led to breakthroughs in ophthalmology, revolutionizing the treatment landscape. Among them, anti-VEGF drugs play one of the most important roles in the management of retinal diseases. Through intravitreal injection, target therapy agents could be directly applied to the retina, minimizing systemic side-effects. Since studies discovered the critical role of abnormal VEGF signaling in numerous retinal diseases, such as neovascular age related macular degeneration (nAMD), diabetic macular edema, and retinal vein occlusions, VEGF inhibition has been established as a highly effective treatment and significantly improved the outcome of these patients. Yet what comes along with the success are the new problems that come to the fore. The administration of these target therapy requires repeated visits for imaging, examination and injections, over a long period of time, which is a heavy burden for both patients and physicians. Despite clinical studies demonstrated the benefit of anti-VEGF target therapy in treating retinal diseases, there is still a group of poor responders that should not be overlooked. Therefore, the availability of biomarkers to guide the treatment protocol for patients, as well as the development of new drugs with longer half-life or multiple targets are the current main focus of drug development. While these drugs hold great promise in terms of efficacy, some toxic side-effects could not be completely avoided even with local administration of the drugs, despite intraocular injection reduces the risk of systemic drug exposure. On the other hand, advances in other novel target therapy drugs such as immunotherapies for cancer are not without their risks, including potentially ocular toxicity linked to the drugs' mechanism of action.
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