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Advisor(s)
Abstract(s)
As terapias CAR-T são um tratamento inovador em constante desenvolvimento científico. Ao dia de hoje, têm sido analisados e avaliados os processos de fabrico, o perfil de benefício-risco destas terapias, bem como criadas resoluções para os problemas identificados, de modo a melhorar este tipo de terapêutica biológica.
Nesta monografia, foram analisados os problemas inerentes à atual produção das terapias CAR-T, bem como o seu perfil de benefício-risco. Com as informações obtidas, realizou-se uma avaliação do risco e obtiveram-se possíveis soluções para os problemas identificados. Existem vários métodos em estudo que permitiriam melhorar tanto o alcance das terapias CAR-T, como o seu custo e tempo de produção.
O Mieloma Múltiplo, sendo uma doença que deteriora multidimensionalmente a qualidade de vida da população afetada, é alvo de muitos estudos. Terapias CAR-T foram autorizadas recentemente para esta patologia, onde têm revolucionado a qualidade de vida dos doentes que vivem com Mieloma Múltiplo Recidivante/Refratário. O ide-cel e o cilta-cel, as terapias mais recentes para esta patologia, foram alvo de comparação e análise escrutinada nesta monografia. Tendo em conta os perfis de eficácia e segurança, tanto nos estudos pivotais como nos dados a
longo prazo, bem como através das perspetivas dos doentes, foi possível comparar estudos, examinar dados, verificar relatos de doentes e muito mais. Com toda a informação angariada ao longo deste trabalho, concluiu-se que o cilta-cel tinha mais benefícios clínicos e um quadro de efeitos secundários menor, relativamente ao ide-cel. Independentemente dos dados estatísticos, as duas medicações obtiveram resultados muito benéficos e foram consideradas muito favoráveis, tanto pela análise dos dados de eficácia e segurança, como pelos relatos dos doentes.
Estas medicações surgiram como um grande avanço na terapêutica do mieloma múltiplo e, à custa delas, milhares de avanços científicos a nível das terapias com células CAR-T se encontram em estudos pré-clínicos e clínicos. Estes avanços são fulcrais para tentar mitigar os efeitos da doença na população e restaurar a sua qualidade de vida.
CAR-T therapies are an innovative treatment undergoing constant scientific development. Today, the manufacturing processes, the benefit-risk profile of these therapies have been analysed and evaluated, as well as solutions to the problems identified, in order to improve this type of biological therapy. This monograph analyses the problems inherent in the current production of CAR-T therapies, as well as their benefit-risk profile. With the information obtained, a risk assessment was carried out and possible solutions to the problems identified were obtained. There are several methods currently being studied that could improve the reach of CAR-T therapies, as well as their cost and production time. Multiple Myeloma, being a disease that multidimensionally deteriorates the quality of life of the affected population, is the subject of many studies. CAR-T therapies have recently been authorised for this pathology, where they have revolutionised the quality of life of patients living with relapsed/refractory Multiple Myeloma. Ide-cel and cilta-cel, the most recent therapies for this pathology, have been compared and scrutinised in this paper. Taking into account efficacy and safety profiles both in pivotal studies and in long-term data, as well as through the patient perspectives, it was possible to compare studies, examine data, verify patient reports and much more. With all the information gathered in the course of this work, it was concluded that cilta-cel had more clinical benefits and a lower side-effect profile than ide-cel. Regardless of the statistical data, both medications obtained very beneficial results and were considered very favourable, both by analysing the efficacy and safety data and by patient reports. These drugs have emerged as a major breakthrough in multiple myeloma therapy and, thanks to them, thousands of scientific advances in CAR-T cell therapies are in pre-clinical and clinical studies. These advances are crucial in trying to mitigate the effects of the disease on the population and restore their quality of life.
CAR-T therapies are an innovative treatment undergoing constant scientific development. Today, the manufacturing processes, the benefit-risk profile of these therapies have been analysed and evaluated, as well as solutions to the problems identified, in order to improve this type of biological therapy. This monograph analyses the problems inherent in the current production of CAR-T therapies, as well as their benefit-risk profile. With the information obtained, a risk assessment was carried out and possible solutions to the problems identified were obtained. There are several methods currently being studied that could improve the reach of CAR-T therapies, as well as their cost and production time. Multiple Myeloma, being a disease that multidimensionally deteriorates the quality of life of the affected population, is the subject of many studies. CAR-T therapies have recently been authorised for this pathology, where they have revolutionised the quality of life of patients living with relapsed/refractory Multiple Myeloma. Ide-cel and cilta-cel, the most recent therapies for this pathology, have been compared and scrutinised in this paper. Taking into account efficacy and safety profiles both in pivotal studies and in long-term data, as well as through the patient perspectives, it was possible to compare studies, examine data, verify patient reports and much more. With all the information gathered in the course of this work, it was concluded that cilta-cel had more clinical benefits and a lower side-effect profile than ide-cel. Regardless of the statistical data, both medications obtained very beneficial results and were considered very favourable, both by analysing the efficacy and safety data and by patient reports. These drugs have emerged as a major breakthrough in multiple myeloma therapy and, thanks to them, thousands of scientific advances in CAR-T cell therapies are in pre-clinical and clinical studies. These advances are crucial in trying to mitigate the effects of the disease on the population and restore their quality of life.
Description
Trabalho Final de Mestrado Integrado, Ciências Farmacêuticas, 2023, Universidade de Lisboa, Faculdade de Farmácia.
Keywords
Mieloma múltiplo (MM) CAR-T Ide-cel Cilta-cel BCMA Mestrado Integrado - 2024