| Abstract |
Autoimmune hemolytic anemia (AIHA) is characterized by premature destruction of erythrocytes, due to auto-antibodies directed against antigens on surface, with or without complement involvement. The clinical course of AIHA varies from mild to severe or even life-threatening forms. Based on the pathogenic antibody class, the disease is classified as warm, cold, mixed AIHA or paroxysmal cold hemoglobinuria (PCH). AIHA can be idiopathic or secondary to underlying systemic illnesses. First line therapy includes corticosteroids, with response in 81-100% of children with primary or secondary AIHA, while better studied second line treatment is rituximab. The aim of the present retrospective study is to report on clinical course and treatment outcome of pediatric patients diagnosed with AIHA and followed in a single pediatric hematology department in Northern Greece, during the period 2002-2020, as well as review of related literature. Thirty-five children and adolescents were enrolled. Demographic, clinical and laboratory data as well as therapy administered at diagnosis and during follow up period were collected. Sex ratio was 1:1, mean age of diagnosis 5.5 years and mean follow up period 5.1 years. Out of 35 patients, 22 (63%) experienced a single AIHA episode and the rest 13 (39%) presented one or more relapses, with mean number of relapses 3.3. Mean hemoglobin (Hb) level was 7.7g/dl at diagnosis, whilst 10 (32.2%) patients presented with severe anemia. Warm AIHA was diagnosed in the majority of them (28/35, 80%) while PCH in the remaining 7 (20%). Three patients (8.6%) presented with a known underlying condition at diagnosis, while 11 children were diagnosed with secondary AIHA during follow up. As for treatment, 7 (20%) patients did not require any therapy, 20 (57,1%) received
ΠΜΣ Αιματολογία - Ογκολογία Παιδιών και Εφήβων Ιατρική Σχολή – Πανεπιστήμιο Κρήτης
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combined therapy with corticosteroids and intravenous immunoglobulin, 4 (11,4%) corticosteroids, 2 (5.7%) intravenous immunoglobulin and 2 (5.7%) corticosteroids with second line treatment due to known underlying disease. Complete response after first line treatment was present in 24 out of 26 (92.3%) patients. Partial or no response was recorded in 2 (7.7%) children. All patients were alive at last follow up. Thirty-three (94.3%) patients demonstrated continuous complete response without need for further treatment and 2 (5.7%) still received immunomodulatory drugs at the end of study. Even though study results largely agree with previous similar studies, key difference of the present study is the absence of reported mortality. Furthermore, diagnosis of secondary AIHA was revealed in many cases remarkably during follow up period, mostly as a result of genetic analysis progress. With regards to literature data available on second line treatment, it is scarce, with most immunomodulatory drugs remaining as off label for use in pediatric AIHA. As pediatric AIHA is a rare disease with potentially complicated clinical course or even fatal outcome and as clinical trials are missing, establishing an international multicenter registry would be of great value, in order to report on knowledge and experience and guide disease management worldwide.
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Αυτοάνοση αιμολυτική αναιμία στην παιδική ηλικία : Καταγραφή της εμπειρίας ενός κέντρου και ανασκόπηση της βιβλιογραφίας
