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Terapêutica imunossupressora na anemia aplásica : a propósito de um caso clínico
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Resumo(s)
Na Anemia Aplásica Grave (AAG), o transplante alogénico de células hematopoéticas a partir de um dador relacionado com antigénio leucocitário humano compatível apresenta-se como o único tratamento potencialmente curativo e de sucesso demonstrado. Este é o procedimento de eleição em crianças com AAG, com dador compatível e sem comorbilidades significativas. Contudo, menos de um terço dos doentes são candidatos adequados ao transplante por ausência dos requisitos necessários. Assim, a terapêutica imunossupressora (TIS) surge como a solução mais comumente utilizada.
Esta revisão apresenta a história de uma criança diagnosticada com AAG que inicia TIS por inexistência de dador adequado. Foi instituída a TIS de primeira linha, que combina globulina anti-timócito de cavalo (GAT) e a ciclosporina, e acompanhada a sua evolução durante 12 meses. Esta terapêutica surte resposta em cerca de 70% dos doentes, no entanto, contrariamente ao expectável, o doente apresentou-se refratário. Nestas situações está preconizado um segundo curso de imunossupressão com GAT de coelho, mas os resultados não são satisfatórios pelo que serão abordadas terapêuticas, como o alemtuzumab, eltrombopag e androgéneos, que se têm demonstrado promissoras. Serão ainda discutidos agentes passíveis de aumentar a resposta à TIS de primeira linha, quando usados como alternativa ou adição a esta.
In Severe Aplastic Anemia (SAA), the allogeneic hematopoietic cell transplantion from a HLA-matched related donor is presented as the only potentially curative treatment with demonstrated success. This is the procedure of choice in children with SAA, with a compatible donor and without significant comorbidities. However, less than a third of the patients are suitable candidates for transplantation due to lack of the necessary requirements. Thus, the immunosuppressive therapy (IST) emerges as the most commonly used solution. This review presents the history of a child diagnosed with SAA that starts IST due to the absence of a suitable donor. The IST first line is established, which combines antithymocyte globulin (ATG) from a horse and cyclosporine, and its evolution is followed for 12 months. This therapeutics causes response in approximately 70% of the patients, however, contrary to expectations, the patient presented himself refractory. In these situations, it is recommended a second course of immunosuppression with rabbit ATG, but the results are not satisfactory and therefore other therapies will be addressed, such as alemtuzumab, eltrombopag and androgens, which have shown promising results. Agents likely to increase the response to IST first line, will be discussed, when used as an alternative or addition to this.
In Severe Aplastic Anemia (SAA), the allogeneic hematopoietic cell transplantion from a HLA-matched related donor is presented as the only potentially curative treatment with demonstrated success. This is the procedure of choice in children with SAA, with a compatible donor and without significant comorbidities. However, less than a third of the patients are suitable candidates for transplantation due to lack of the necessary requirements. Thus, the immunosuppressive therapy (IST) emerges as the most commonly used solution. This review presents the history of a child diagnosed with SAA that starts IST due to the absence of a suitable donor. The IST first line is established, which combines antithymocyte globulin (ATG) from a horse and cyclosporine, and its evolution is followed for 12 months. This therapeutics causes response in approximately 70% of the patients, however, contrary to expectations, the patient presented himself refractory. In these situations, it is recommended a second course of immunosuppression with rabbit ATG, but the results are not satisfactory and therefore other therapies will be addressed, such as alemtuzumab, eltrombopag and androgens, which have shown promising results. Agents likely to increase the response to IST first line, will be discussed, when used as an alternative or addition to this.
Descrição
Trabalho Final do Curso de Mestrado Integrado em Medicina, Faculdade de Medicina, Universidade de Lisboa, 2016
Palavras-chave
Anemia aplástica Transplante homólogo Hematologia Pediatria