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Chronic relapsing thrombotic thrombocytopenic purpura in infants with large von Willebrand factor multimers during
M M Chintagumpala1, R L Hurwitz, J L Moake
1Department of Pediatrics (Hematology/Oncology), Baylor College of Medicine, Houston, TX.
Insights
Two children with recurrent hemolytic anemia and thrombocytopenia improved with fresh-frozen plasma. This suggests a chronic relapsing disorder responsive to plasma infusions, unlike typical thrombotic thrombocytopenic purpura treatments.
Area of Science:
- Hematology
- Pediatrics
- Genetics
Background:
- Recurrent schistocytic hemolytic anemia and thrombocytopenia in neonates present diagnostic challenges.
- Thrombotic thrombocytopenic purpura (TTP) is a rare, life-threatening condition characterized by microangiopathic hemolytic anemia, thrombocytopenia, and organ damage.
Observation:
- Two neonates presented with recurrent episodes of hemolytic anemia and thrombocytopenia.
- One patient experienced a stroke during a thrombotic thrombocytopenic purpura episode.
- Unusually large von Willebrand factor multimers were detected during remission periods in both children.
Findings:
- Standard treatments like corticosteroids and intravenous immune globulin were ineffective.
- Both patients showed immediate improvement following fresh-frozen plasma infusion.
- Thrombocytopenia recurred regularly without periodic plasma infusions, indicating a chronic relapsing course.
Implications:
- This suggests a distinct chronic relapsing disorder, potentially a variant of TTP, responsive to plasma therapy.
- Early diagnosis and plasma-based treatment may be crucial for managing these rare neonatal hematologic conditions.
- Further research into the underlying mechanisms of von Willebrand factor multimer abnormalities in these patients is warranted.
Abstract:
We studied two children with recurrent schistocytic hemolytic anemia and thrombocytopenia beginning in the neonatal period. One patient had a stroke during one of the episodes of thrombotic thrombocytopenic purpura. The presence of unusually large von Willebrand factor multimers was demonstrated in both children during clinical and hematologic remissions. Treatment with corticosteroids and intravenous injections of immune globulin was unsuccessful in the one patient who received it. Immediate improvement occurred in both patients after the infusion of fresh-frozen plasma. Symptoms of thrombocytopenia continue to recur at regular intervals in the absence of periodic fresh-frozen plasma infusions. One of these children apparently has chronic relapsing thrombotic thrombocytopenic purpura; the second has a chronic relapsing disorder similar to thrombotic thrombocytopenic purpura.