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Advisor(s)
Abstract(s)
Introdução: A diabetes mellitus tipo 1 (DM1) é uma doença autoimune caracterizada
pela destruição das células beta do pâncreas, o que, por sua vez, resulta numa diminuição
da produção de insulina. A incidência da DM1 tem vindo a aumentar ao longo dos últimos
anos, sendo, na atualidade, uma das doenças crónicas mais prevalentes na idade
pediátrica. Esta patologia está associada a uma elevada morbilidade e mortalidade, uma
vez que os pacientes apresentam complicações tanto a curto como a longo prazo
relacionadas com o controlo glicémico inadequado. Atualmente, o tratamento consiste na
administração de esquemas intensivos de insulina, tratamento esse que apenas trata os
sintomas e não a causa. Torna-se, portanto, essencial o desenvolvimento de novas
terapêuticas capazes de alterar o curso natural da doença. Os anticorpos monoclonais
anti-CD3 mostraram-se eficazes em prevenir ou até mesmo reverter o início da DM1 em
modelos animais. Dentro desta classe, o Teplizumab destacou-se como o mais promissor
em termos de resultados.
Objetivo: O objetivo desta revisão é, assim, avaliar tanto a segurança como a eficácia do
Teplizumab na prevenção e tratamento da DM1.
Material e Métodos: O presente trabalho teve como base a pesquisa bibliográfica de
artigos científicos na base de dados “PubMed/MEDLINE” com uso das palavras-chaves
"Type 1 Diabetes" e "Teplizumab". A pesquisa ficou restrita a ensaios clínicos
randomizados cegos ou duplamente-cegos e estudos realizados em seres humanos. Foram,
ainda, consultadas as listas de referências bibliográficas dos artigos selecionados.
Resultados: Este estudo identificou um total de 13 ensaios clínicos, nos quais a
terapêutica com Teplizumab demonstrou vantagens significativas tanto em indivíduos
com elevado risco de desenvolver DM1, como em pacientes recentemente diagnosticados
com a patologia. Nos pacientes considerados de alto risco, observou-se que o Teplizumab é
capaz de retardar o diagnóstico da doença, enquanto preserva os níveis de peptídeo-C. Em
pacientes recém-diagnosticados com DM1, a terapêutica evidenciou a capacidade de
manter os níveis de peptídeo-C e reduzir as exigências insulínicas durante, pelo menos, 1 a
2 anos. Além disso, o fármaco revelou-se seguro quando utilizado em dose adequada, com
a ocorrência de efeitos adversos leves e autolimitados na maioria dos casos.
Discussão/Conclusão: O Teplizumab representa, assim, uma nova esperança para os
doentes com DM1, na medida em que, pela primeira vez na história, se vislumbra a possibilidade de efetivamente prevenir o desenvolvimento da doença. Contudo, é
imperativo obter mais evidências científicas para definir com precisão o papel terapêutico
do Teplizumab na DM1.
Introduction: Type 1 Diabetes Mellitus (DM1) is an autoimmune disease characterized by the destruction of pancreatic beta cells, resulting in a decreased production of insulin. The incidence of DM1 has been increasing over the past years, making it currently one of the most prevalent chronic diseases in pediatric age. This condition is associated with high morbidity and mortality, as patients experience both short-term and long-term complications related to inadequate glycemic control. Presently, the treatment involves the administration of intensive insulin regimens, addressing symptoms but not the root cause. Hence, it becomes essential to develop new therapeutics capable of altering the natural course of the disease. Monoclonal antibodies targeting CD3 have proven effective in preventing or even reversing the onset of DM1 in animal models. Among this class, Teplizumab has emerged as the most promising in terms of outcomes. Objective: The objective of this review is, therefore, to evaluate both the safety and efficacy of Teplizumab in the prevention and treatment of DM1. Material and Methods: This study was based on a literature review of scientific articles in the "PubMed/MEDLINE" database using the keywords "Type 1 Diabetes" and "Teplizumab." The search was limited to blinded or double-blinded randomized clinical trials and studies conducted in humans. Additionally, the reference lists of selected articles were consulted. Results: This study identified a total of 13 clinical trials in which Teplizumab therapy demonstrated significant advantages in both individuals at high risk of developing DM1 and in patients recently diagnosed with the condition. In individuals considered at high risk, Teplizumab was observed to delay the onset of the disease while preserving C-peptide levels. In recently diagnosed DM1 patients, the therapy exhibited the ability to maintain Cpeptide levels and reduce insulin requirements for, at least, 1 to 2 years. Furthermore, the drug proved to be safe when administered at an appropriate dose, with the occurrence of mild and self-limiting adverse effects in most cases. Discussion/Conclusion: Teplizumab, thus represents a new hope for DM1 patients, as for the first time in history, the possibility of effectively preventing the development of the disease is envisaged. However, it is imperative to obtain more scientific evidence to precisely define the therapeutic role of Teplizumab in DM1.
Introduction: Type 1 Diabetes Mellitus (DM1) is an autoimmune disease characterized by the destruction of pancreatic beta cells, resulting in a decreased production of insulin. The incidence of DM1 has been increasing over the past years, making it currently one of the most prevalent chronic diseases in pediatric age. This condition is associated with high morbidity and mortality, as patients experience both short-term and long-term complications related to inadequate glycemic control. Presently, the treatment involves the administration of intensive insulin regimens, addressing symptoms but not the root cause. Hence, it becomes essential to develop new therapeutics capable of altering the natural course of the disease. Monoclonal antibodies targeting CD3 have proven effective in preventing or even reversing the onset of DM1 in animal models. Among this class, Teplizumab has emerged as the most promising in terms of outcomes. Objective: The objective of this review is, therefore, to evaluate both the safety and efficacy of Teplizumab in the prevention and treatment of DM1. Material and Methods: This study was based on a literature review of scientific articles in the "PubMed/MEDLINE" database using the keywords "Type 1 Diabetes" and "Teplizumab." The search was limited to blinded or double-blinded randomized clinical trials and studies conducted in humans. Additionally, the reference lists of selected articles were consulted. Results: This study identified a total of 13 clinical trials in which Teplizumab therapy demonstrated significant advantages in both individuals at high risk of developing DM1 and in patients recently diagnosed with the condition. In individuals considered at high risk, Teplizumab was observed to delay the onset of the disease while preserving C-peptide levels. In recently diagnosed DM1 patients, the therapy exhibited the ability to maintain Cpeptide levels and reduce insulin requirements for, at least, 1 to 2 years. Furthermore, the drug proved to be safe when administered at an appropriate dose, with the occurrence of mild and self-limiting adverse effects in most cases. Discussion/Conclusion: Teplizumab, thus represents a new hope for DM1 patients, as for the first time in history, the possibility of effectively preventing the development of the disease is envisaged. However, it is imperative to obtain more scientific evidence to precisely define the therapeutic role of Teplizumab in DM1.
Description
Keywords
Anticorpo Monoclonal Anti-Cd3 Diabetes Mellitus Tipo 1 Endocrinologia Prevenção e Tratamento Teplizumab