Publicação
Autologous hematopoietic stem cell transplantation in multiple sclerosis: revision of clinical practice
| Resumo: | Background In the last years, several disease-modifying therapies (DMTs) have been studied and approved for the treatment of multiple sclerosis (MS). However, some groups of patients presented a phenotype called "aggressive", now named highly active MS. The severe nature of this phenotype requires different treatment decisions. This led to the investigation of potentially more efficient therapies, such as autologous hematopoietic stem cell transplantation (aHSCT). Despite several evidence demonstrating that aHSCT is an effective therapy, this procedure has not been widely implemented in clinical practice, since the optimal population remains poorly studied. Thus, this systematic review aims to demonstrate in which groups of patients aHSCT can bring therapeutic benefits. Methods A comprehensive online search was undertaken using Pubmed, Science Direct, and Spocus databases (2017 to 2021). The original articles included in this review were randomized controlled trials or observational studies that evaluate progression-free survival using the Expanded Disability Status Scale (EDSS). Results The systematic electronic search of the literature identified 158 articles, of which 15 were eligible for this review after screening and assessment, with a total of 1381 patients included. All of the studies that include the relapsing form of MS demonstrate that autologous hematopoietic stem cell transplantation is beneficial because it improves the EDSS score of these patients. Moreover, four studies focused on the aggressive/high active relapsing-remitting multiple sclerosis treatment and showed a significant decrease of disability progression after aHSCT. In addition, several studies showed that transplantation may play an important role in MS refractory to conventional therapy. Conclusion Autologous hematopoietic stem cell transplantation is an alternative therapy to the currently used DMTs, which may allow a significant decrease of the disability progression, in patients with multiple sclerosis. After reviewing the literature, we concluded that this therapy will be more effective in patients with highly active MS. Other factors associated with better aHSCT results are younger age, lower baseline EDSS and to be submitted to a small number of previous treatments. |
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| Autores principais: | Rafaela Cristina Coelho da Silva |
| Assunto: | Medicina clínica Clinical medicine |
| Ano: | 2022 |
| País: | Portugal |
| Tipo de documento: | dissertação de mestrado |
| Tipo de acesso: | acesso restrito |
| Instituição associada: | Universidade do Porto |
| Idioma: | inglês |
| Origem: | Repositório Aberto da Universidade do Porto |
| Resumo: | Background In the last years, several disease-modifying therapies (DMTs) have been studied and approved for the treatment of multiple sclerosis (MS). However, some groups of patients presented a phenotype called "aggressive", now named highly active MS. The severe nature of this phenotype requires different treatment decisions. This led to the investigation of potentially more efficient therapies, such as autologous hematopoietic stem cell transplantation (aHSCT). Despite several evidence demonstrating that aHSCT is an effective therapy, this procedure has not been widely implemented in clinical practice, since the optimal population remains poorly studied. Thus, this systematic review aims to demonstrate in which groups of patients aHSCT can bring therapeutic benefits. Methods A comprehensive online search was undertaken using Pubmed, Science Direct, and Spocus databases (2017 to 2021). The original articles included in this review were randomized controlled trials or observational studies that evaluate progression-free survival using the Expanded Disability Status Scale (EDSS). Results The systematic electronic search of the literature identified 158 articles, of which 15 were eligible for this review after screening and assessment, with a total of 1381 patients included. All of the studies that include the relapsing form of MS demonstrate that autologous hematopoietic stem cell transplantation is beneficial because it improves the EDSS score of these patients. Moreover, four studies focused on the aggressive/high active relapsing-remitting multiple sclerosis treatment and showed a significant decrease of disability progression after aHSCT. In addition, several studies showed that transplantation may play an important role in MS refractory to conventional therapy. Conclusion Autologous hematopoietic stem cell transplantation is an alternative therapy to the currently used DMTs, which may allow a significant decrease of the disability progression, in patients with multiple sclerosis. After reviewing the literature, we concluded that this therapy will be more effective in patients with highly active MS. Other factors associated with better aHSCT results are younger age, lower baseline EDSS and to be submitted to a small number of previous treatments. |
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