Vagace JM, Bajo R, Gervasini G. Diagnostic and therapeutic challenges of primary autoimmune haemolytic anaemia in children. Arch Dis Child. 2014 Jul;99(7):668-73.
Autoimmune haemolytic anaemias (AIHAs) are extracorpuscular haemolytic anaemias produced by antierythrocyte autoantibodies which cause a shortened red blood cell life span. There are several reasons why the diagnosis and treatment of AIHAs in children represent a bigger challenge than in adult patients, including the presence of particular AIHA types, the uncertainty of serological tests and the limited clinical experience. All these facts have added up to a poor understanding and management of some topics in childhood AIHA. We discuss some of these questions, for example, the occurrence of AIHA with negative direct antiglobulin (Coombs) test, the correct diagnosis and actual incidence of paroxysmal cold haemoglobinuria, the most appropriate second-line therapy of AIHA in childhood or the management of transfusion procedures in these patients. This review takes a practical point of view, providing with some ground rules on how to identify and deal with these paediatric patients.
Teachey DT, Lambert MP. Diagnosis and management of autoimmune cytopenias in childhood. Pediatr Clin North Am. 2013 Dec;60(6):1489-511.
The diagnosis and management of children with autoimmune cytopenias can be challenging. Children can present with immune-mediated destruction of a single-cell lineage or multiple cell lineages, including platelets (immune thrombocytopenia [ITP]), erythrocytes (autoimmune hemolytic anemia), and neutrophils (autoimmune neutropenia). Immune-mediated destruction can be primary or secondary to a comorbid immunodeficiency, malignancy, rheumatologic condition, or lymphoproliferative disorder. Treatment options generally consist of nonspecific immune suppression or modulation. This nonspecific approach is changing as recent insights into disease biology have led to targeted therapies, including the use of thrombopoietin mimetics in ITP and sirolimus for cytopenias associated with autoimmune lymphoproliferative syndrome.
The clinical and hematological profile and treatment outcome of children with warm autoimmune hemolytic anemia (AIHA) were assessed using retrospective case record analysis. There were 26 (17 idiopathic; 9 secondary) patients with a median age of 11 years. Pallor (100%), fever (39%), and jaundice (59%) were the main presenting complaints. Jaundice was much more common in idiopathic (70%) compared to secondary (44%). Direct antiglobulin test was negative in 3 patients. Oral prednisolone produced remission in 81% patients. Four patients relapsed after a median period of 7 months (2 months to 2 year) after response. All responded to a second course of steroids in median 14 days. One child required cyclosporin A in addition. No correlation was found between response and parameters such as age, sex, jaundice, low pretreatment hemoglobin, reticulocyte count, total leukocyte count, platelet count, subtype of AIHA, and hepatosplenomegaly. Relapse correlated with increased duration between the onset of symptoms and treatment. This study indicates that oral prednisolone is an effective therapy for autoimmune hemolytic anemia. In refractory cases cyclosporine A may be useful.