Evans syndrome in children. Long-term outcome in a prospective French national observational cohort.

• Main Authors: Nathalie eAladjidi (Author), Helder eFernandes (Author), Thierry eLeblanc (Author), Amelie eVareliette (Author), Frédéric eRieux-Laucat (Author), Yves eBertrand (Author), Hervé eChambost (Author), Marlène ePasquet (Author), Françoise eMazingue (Author), Corinne eGuitton (Author), Isabelle ePellier (Author), Françoise eRoqueplan-Bellmann (Author), Corinne eArmari-Alla (Author), Caroline eThomas (Author), Aude eMarie-Cardine (Author), Odile eLejars (Author), Fanny eFouyssac (Author), Sophie eBayart (Author), Patrick eLutz (Author), Christophe ePiguet (Author), Eric eJeziorski (Author), Pierre eRohrlich (Author), Philippe eLemoine (Author), Damien eBodet (Author), Catherine ePaillard (Author), Gerard eCouillault (Author), Frédéric eMillot (Author), Alain eFischer (Author), Yves ePerel (Author), Guy eLeverger (Author)
• Format: Book
• Published: Frontiers Media S.A., 2015-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Summary

Evans syndrome (ES) is a rare autoimmune disorder whose long-term follow-up characteristics are unknown. Patients under 18 at the time of diagnosis of a first autoimmune cytopenia have been included since 2004 in a national prospective observational cohort. In 2014, 156 children diagnosed between 1981 and 2014 with ES, were analyzed. The median age at initial cytopenia was 5.4 (0.2-17.2) years old. For 85 sequential cases, the median delay between the episodes of AIHA and ITP was 2.4 years (0.1-16.3). The median follow-up since ES diagnosis was 6.5 years (0.1-28.8). ES revealed underlying diseases in 10% of children; in 60% of patients, various associated immune manifestations were observed, and ES remained primary in 30%. Five-year ITP and AIHA relapse-free survival were respectively 25% and 61%. In all, 69% of children required one or more than one second-line immune treatment and 15 patients (10%) died at a median age of 14.3 years (1.7-28.1).This national work provides the first consistent clinical description for ES and underscores the high percentage of associated immune manifestations, the long-term complications, and treatment toxicities. Current challenges include the identification of underlying genetic immune dysregulations and better characterization of subgroups of patients and of second-line therapy strategies.

Item Description

2296-2360
10.3389/fped.2015.00079