Management of Evans syndrome

Abstract

SummaryEvans syndrome is an uncommon condition defined by the combination (either simultaneously or sequentially) of immune thrombocytopenia (ITP) and autoimmune haemolytic anaemia (AIHA) with a positive direct antiglobulin test (DAT) in the absence of known underlying aetiology. This condition generally runs a chronic course and is characterised by frequent exacerbations and remissions. First-line therapy is usually corticosteroids and/or intravenous immunoglobulin, to which most patients respond; however, relapse is frequent. Options for second-line therapy include immunosuppressive drugs, especially ciclosporin or mycophenolate mofetil; vincristine; danazol or a combination of these agents. More recently a small number of patients have been treated with rituximab, which induces remission in the majority although such responses are often sustained for <12 months and the longterm effects in children are unclear. Splenectomy may also be considered although long-term remissions are less frequent than in uncomplicated ITP. For very severe and refractory cases stem cell transplantation (SCT) offers the only chance of long-term cure. The limited data available suggest that allogeneic SCT may be superior to autologous SCT but both carry risks of severe morbidity and of transplant-related mortality. Cure following reduced-intensity conditioning has now been reported and should be considered for younger patients in the context of controlled clinical trials.

Management of autoimmune hemolytic anemia in children and adolescents: A single center experience

“…Some researchers emphasized the exclusion of an underlying disease for the definition of true ES [16], whereas others defined secondary ES based on underlying disease; we agree with the latter definition. ES is more than a coincidental combination of immune cytopenias; rather, it is a chronic state of profound dysregulation of the immune system [17].…”

“…Prior to his diagnosis of AIHA, despite thrombocytopenia he did not have any bleeding and perianal infections rarely had a negative affect on his quality of life. In contrast to our observation, some researchers reported that the role of splenectomy has never been established in ES and remissions last only 1-2 months without support [16,[18][19][20]. In a survey of ES mainly from the US and Canada 15 patients underwent splenectomy at a median age of 10 years (range: 1.2-26.5 years).…”

“…Despite discrepancies in descriptions, all researchers conclude that cytopenias are recurrent, often refractory to IVIG, corticosteroids, and splenectomy, and that the syndrome may be fatal [18]. In very severe and refractory cases hematopoietic stem cell transplantation (HSCT) offers long-term cure, but again carries the risk of severe morbidity and transplant-related mortality [16]. The present study included 3 patients with primary ES, all of whom had a chronic course.…”

“…Northon et al evaluated the response to rituximab in 8 pediatric patients (aged 0.3-16 years) with ES from the literature [16]. Seven had complete remission during a follow-up period of 12-15 months, but 2 had relapses and achieved a second complete remission with rituximab.…”

Diagnosis and management of chronic ITP: comments from an ICIS expert group

“…Steroids and IVIg are the usual first-line therapy of the ITP; anti-D is contraindicated. Rituximab, cyclosporine, mycophenolate mofetil, splenectomy (not recommended in children) and combination chemotherapy have been used in second-line treatment [14].…”