Successful treatment with rituximab for acute refractory thrombotic thrombocytopenic purpura related to acquired ADAMTS13 deficiency: a pediatric report and literature review.
OBJECTIVE
To report the case of a child with severe autoimmune thrombotic thrombocytopenic purpura (TTP) resistant to plasma exchange and steroids who was successfully treated with rituximab.


METHODS
Case report and review of the literature on pediatric acquired TTP. The report was approved by an independent local ethics committee.


METHODS
Pediatric intensive care unit in a tertiary care children's hospital.


METHODS
A 10-yr-old boy was referred to the emergency unit with fever, vomiting, confusion, hemolytic anemia, thrombocytopenia, and mild acute renal failure. An atypical hemolytic uremic syndrome was suspected, and plasma exchange was started urgently. The patient was refractory to plasma therapy and presented critical complications. After a diagnosis of acquired TTP attributable to anti-ADAMTS13 autoantibodies had been made, he was treated with rituximab, which resulted in a stable clinical remission.


METHODS
Rituximab therapy.


RESULTS
Clinical remission.


CONCLUSIONS
TTP is a rare but life-threatening condition in children that is characterized by hemolytic anemia, thrombocytopenia, and signs of ischemic organ dysfunction. If renal involvement is present, TTP may be misdiagnosed as hemolytic uremic syndrome, but reliable screening for ADAMTS13 activity and anti-ADAMTS autoantibodies allow us to distinguish the two entities and provide adequate therapy.
Harambat
Jérôme
J
Service de Pédiatrie Hôpital Pellegrin-Enfants, CHU de Bordeaux and Université de Bordeaux, Bordeaux, France. jerome.harambat@chu-bordeaux.fr
Lamireau
Delphine
D
Delmas
Yahsou
Y
Ryman
Anne
A
Llanas
Brigitte
B
Brissaud
Olivier
O
D051722:ADAM Proteins / Q000172:deficiency* / Q000276:immunology; D000071120:ADAMTS13 Protein; D058846:Antibodies, Monoclonal, Murine-Derived / Q000627:therapeutic use*; D002648:Child; D006801:Humans; D007155:Immunologic Factors / Q000627:therapeutic use*; D015278:Intensive Care Units, Pediatric; D008297:Male; D017063:Outcome Assessment, Health Care; D011697:Purpura, Thrombotic Thrombocytopenic / Q000188:drug therapy* / Q000503:physiopathology; D000069283:Rituximab
D058846:Antibodies, Monoclonal, Murine-Derived; D007155:Immunologic Factors; D000069283:Rituximab; D051722:ADAM Proteins; D000071120:ADAMTS13 Protein; C099604:ADAMTS13 protein, human