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Abstract(s)
Contexto: A doença das células falciformes (DCF) é um conjunto de anemias hemolíticas crónicas, com mutação no gene da cadeia β-hemoglobina, a qual condiciona a formação de hemoglobina mutada - HbS. Esta conduz a alterações na forma e rigidez dos eritrócitos, quando sob certas situações, como a hipoxia - processo denominado por falciformação. Complicações agudas e crónicas foram descritas associadas a este fenómeno (Inusa et al., 2019).
A abordagem destas complicações frequentemente requer transfusões sanguíneas – simples, de permuta manual ou automatizada (Tzounakas et al., 2018). A eritracitaferese (ECF), permuta automatizada, é a terapêutica recomendada na prevenção de acidente vascular cerebral (AVC), síndrome torácico agudo (STA) e de crise vaso-oclusiva (CVO) refratária (Koehl et al., 2016). Estudos sugerem que esta técnica está associada a níveis de sobrecarga de ferro inferiores, efeito adverso comum de transfusões crónicas (Fasano et al., 2016).
Objetivos: Comparar a sobrecarga de ferro nos doentes pediátricos com DCF sob terapêutica transfusional crónica - ECF vs transfusão simples (TS) - com seguimento no Hospital Universário Lisboa Norte nos últimos cinco anos. Como objetivo secundário foi avaliada a eficácia da ECF na prevenção de AVC, STA e CVO, até à data, sem estudos semelhantes na literatura.
Métodos: Utilizou-se os valores de ferritina pré e pós procedimento, de ambos os grupos, aos quais se aplicou o Teste Mann-Whitney U, com nível de confiança de 95%. Foram contabilizadas as complicações AVC, STA e CVO nos dois grupos.
Resultados: A variação de ferritina entre os grupos foi significativa (p =0.006), de -45,7% na ECF e de 98% na TS. Verificou-se uma diminuição da ocorrência/recorrência de CVO, sob ECF, apesar de não significativa (p=0.361).
Conclusão: A ECF é uma técnica que reduz a incidência da sobrecarga de ferro nos doentes com DCF cronicamente transfundidos. Adicionalmente, aparenta ser mais eficaz na prevenção de CVO.
Background: Sickle cell disease (SCD) is a group of inherited disorders caused by a mutation on the haemoglobin-β gene, which leads to an abnormal haemoglobin protein in red blood cells - HbS. HbS is responsible for sickling - an alteration on the form and rigidity of red blood cells that occurs with certain triggers, such as hypoxia. Several complications associated with this phenomenon have been described (Inusa et al, 2019). These complications often requires chronic blood transfusions which can be simple, manual, or automated exchange, also known as erythrocytapheresis (Tzounakas et al., 2018). Erythrocytapheresis is the recommended therapy in the prevention of stroke, thoracic acute syndrome (TAS), and non-responsive vaso-occlusive crisis (VOC) (Koehl et al., 2016). This technique is associated with lower iron overload levels, a common side effect of chronic transfusions (Fasano et al., 2016). Aim: We aim to compare the iron overload levels on chronic transfused children with SCD who underwent simple transfusion (ST) versus erythrocytapheresis followed on the Hospital Universitário Lisboa Norte in the last 5 years. We also aim to compare the effiency of erythrocytapheresis regarding stroke, TAS and VOC (major complications), study never done before, according to literature. Methods: Iron overload was measured using ferritin levels before and after transfusions in both groups. Significant variations were investigated with Mann-Whitney U test, for a defined confidence level of 95%. Inventory of major complications was made in both groups. Results: The variation of ferritin between the groups was significant (p=0.006), with a ferritin median variation of 98% on ST vs -47.5% on erythrocytapheresis group. Reduction of incidence of CVO on erythrocytapheresis group was found, nevertheless not significant (p=0.361). Conclusion: Erythrocytapheresis is a technique which reduces the incidence of iron overload on chronic transfused patients with SCD. Erythrocytapheresis is also associated with reduction of incidence of VOC.
Background: Sickle cell disease (SCD) is a group of inherited disorders caused by a mutation on the haemoglobin-β gene, which leads to an abnormal haemoglobin protein in red blood cells - HbS. HbS is responsible for sickling - an alteration on the form and rigidity of red blood cells that occurs with certain triggers, such as hypoxia. Several complications associated with this phenomenon have been described (Inusa et al, 2019). These complications often requires chronic blood transfusions which can be simple, manual, or automated exchange, also known as erythrocytapheresis (Tzounakas et al., 2018). Erythrocytapheresis is the recommended therapy in the prevention of stroke, thoracic acute syndrome (TAS), and non-responsive vaso-occlusive crisis (VOC) (Koehl et al., 2016). This technique is associated with lower iron overload levels, a common side effect of chronic transfusions (Fasano et al., 2016). Aim: We aim to compare the iron overload levels on chronic transfused children with SCD who underwent simple transfusion (ST) versus erythrocytapheresis followed on the Hospital Universitário Lisboa Norte in the last 5 years. We also aim to compare the effiency of erythrocytapheresis regarding stroke, TAS and VOC (major complications), study never done before, according to literature. Methods: Iron overload was measured using ferritin levels before and after transfusions in both groups. Significant variations were investigated with Mann-Whitney U test, for a defined confidence level of 95%. Inventory of major complications was made in both groups. Results: The variation of ferritin between the groups was significant (p=0.006), with a ferritin median variation of 98% on ST vs -47.5% on erythrocytapheresis group. Reduction of incidence of CVO on erythrocytapheresis group was found, nevertheless not significant (p=0.361). Conclusion: Erythrocytapheresis is a technique which reduces the incidence of iron overload on chronic transfused patients with SCD. Erythrocytapheresis is also associated with reduction of incidence of VOC.
Description
Trabalho Final do Curso de Mestrado Integrado em Medicina, Faculdade de Medicina, Universidade de Lisboa, 2023
Keywords
Ferritina Doença das células falciformes Eritracitaferese Complicações major Sobrecarga de ferro Pediatria