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Tratamento da degenerescência macular da idade: passado, presente e futuro
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A degenerescência macular da idade (DMI) é uma das principais causas de perda visual irreversível na população idosa, com impacto significativo na autonomia, qualidade de vida e nos sistemas de saúde. Nas últimas décadas, registaram-se avanços relevantes na compreensão da fisiopatologia e no tratamento desta doença multifatorial. Esta revisão tem como objetivo oferecer uma visão abrangente da evolução terapêutica da DMI, destacando as principais abordagens atualmente disponíveis e perspetivas promissoras para o futuro.
Historicamente, as opções terapêuticas para a DMI eram limitadas e pouco eficazes na contenção da progressão da doença. A introdução dos agentes anti-fator de crescimento endotelial vascular (anti-VEGF) constituiu um marco no tratamento da forma neovascular da DMI, traduzindo-se em melhorias significativas na acuidade visual. No entanto, a necessidade de injeções intravítreas frequentes, os custos elevados e a resposta variável entre os doentes permanecem como desafios consideráveis. Acresce que, até ao momento, não existem tratamentos curativos para a atrofia geográfica, forma avançada da DMI seca, apenas terapias que podem retardar a sua progressão e ajudar a controlar a doença.
Este trabalho revê as terapias farmacológicas atualmente aprovadas, assim como os tratamentos em investigação clínica e pré-clínica. Entre as estratégias emergentes, destacam-se a terapia génica mediada por vetores virais e as terapias celulares, especialmente aquelas que utilizam células do epitélio pigmentado da retina (EPR) derivadas de induced pluripotent stem cells (iPSC). Estas abordagens visam não apenas travar a progressão da doença, mas também restaurar a função retiniana comprometida, com potencial aplicação nas formas neovascular e atrófica.
Apesar do grande potencial destas terapias inovadoras, a sua translação para a prática clínica exigirá evidência robusta quanto à sua segurança, eficácia e custo-benefício a longo prazo.
Em conclusão, embora persistam desafios significativos, o panorama terapêutico da DMI encontra-se em rápida evolução. As intervenções emergentes poderão oferecer opções mais eficazes, duradouras e personalizadas, com potencial para redefinir os cuidados prestados e melhorar substancialmente o prognóstico dos doentes.
Age-related macular degeneration (AMD) is one of the leading causes of irreversible vision loss in the elderly population, with a significant impact on autonomy, quality of life, and healthcare systems. In recent decades, there have been significant advances in understanding the pathophysiology and treatment of this multifactorial disease. This review aims to provide a comprehensive overview of the therapeutic evolution of AMD, highlighting the main approaches that are currently available as well as promising prospects for the future. Historically, therapeutic options for AMD were limited and ineffective in halting disease progression. The introduction of anti-vascular endothelial growth factor (anti-VEGF) agents marked a turning point in the treatment of the neovascular form of AMD, resulting in significant improvements in visual outcomes. However, the need for frequent intravitreal injections, high costs, and variable response among patients remain considerable challenges. Moreover, to date, there are no curative treatments for geographic atrophy, an advanced form of dry AMD, only therapies that can slow its progression and help control the disease. This study reviews currently approved pharmacological therapies, as well as treatments undergoing clinical and preclinical investigation. Among the emerging strategies, viral vector-mediated gene therapy and cell therapies stand out, especially those employing retinal pigment epithelial (RPE) cells derived from induced pluripotent stem cells (iPSCs). These approaches aim not only to halt disease progression, but also to restore impaired retinal function, with potential application in neovascular and atrophic forms of AMD. Despite the great potential of these innovative therapies, their translation into clinical practice will require robust evidence of their long-term safety, efficacy, and cost-effectiveness. In conclusion, although significant challenges remain, the therapeutic landscape for AMD is rapidly evolving. Emerging interventions may offer more effective, long-lasting, and personalized treatment options with the potential to redefine care and substantially improve patient outcomes.
Age-related macular degeneration (AMD) is one of the leading causes of irreversible vision loss in the elderly population, with a significant impact on autonomy, quality of life, and healthcare systems. In recent decades, there have been significant advances in understanding the pathophysiology and treatment of this multifactorial disease. This review aims to provide a comprehensive overview of the therapeutic evolution of AMD, highlighting the main approaches that are currently available as well as promising prospects for the future. Historically, therapeutic options for AMD were limited and ineffective in halting disease progression. The introduction of anti-vascular endothelial growth factor (anti-VEGF) agents marked a turning point in the treatment of the neovascular form of AMD, resulting in significant improvements in visual outcomes. However, the need for frequent intravitreal injections, high costs, and variable response among patients remain considerable challenges. Moreover, to date, there are no curative treatments for geographic atrophy, an advanced form of dry AMD, only therapies that can slow its progression and help control the disease. This study reviews currently approved pharmacological therapies, as well as treatments undergoing clinical and preclinical investigation. Among the emerging strategies, viral vector-mediated gene therapy and cell therapies stand out, especially those employing retinal pigment epithelial (RPE) cells derived from induced pluripotent stem cells (iPSCs). These approaches aim not only to halt disease progression, but also to restore impaired retinal function, with potential application in neovascular and atrophic forms of AMD. Despite the great potential of these innovative therapies, their translation into clinical practice will require robust evidence of their long-term safety, efficacy, and cost-effectiveness. In conclusion, although significant challenges remain, the therapeutic landscape for AMD is rapidly evolving. Emerging interventions may offer more effective, long-lasting, and personalized treatment options with the potential to redefine care and substantially improve patient outcomes.
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Degenerescência macular da idade terapias emergentes anti-VEGF terapia génica terapia celular retina Age-related macular degeneration emerging therapies gene therapy cell therapy