Factor VIII concentrate-responsive thrombocytopenia, hemolytic anemia, and nephropathy. Evidence that factor VIII:von Willebrand factor is involved in its pathogenesis.

Abstract

A 4-year-old Japanese girl had a congenital disorder that was characterized by recurrent thrombocytopenia, hemolytic anemia, hematuria, and proteinuria, which were repeatedly improved by the infusion of factor VIII concentrate. She developed the similar symptoms within 1 h after 1-desamino-8-D-arginine vasopressin (DDAVP) administration. Coagulation studies 30 and 60 min after DDAVP infusion showed a disappearance of large factor VIII:von Willebrand factor (VIII:vWF) multimers, which was the same abnormality that was observed at acute episodes. There were no significant changes in the plasma levels of 6-keto-prostaglandin F1 alpha and thromboxane B2 before and after DDAVP infusion. These results provide further support that VIII:vWF is directly involved in the pathogenesis of this congenital disorder.

• Authors: T Hara, A Kitano, T Kajiwara, T Kondo, K Sakai, Y Hamasaki
• Journal: The American journal of pediatric hematology/oncology (Am J Pediatr Hematol Oncol) Vol. 8 Issue 4 Pg. 324-8 ( 1986) ISSN: 0192-8562 [Print] United States
• PMID: 3099591 (Publication Type: Case Reports, Journal Article)

Chemical References

• Macromolecular Substances
• von Willebrand Factor
• Factor VIII
• Deamino Arginine Vasopressin

Topics

• Anemia, Hemolytic (therapy)
• Child, Preschool
• Deamino Arginine Vasopressin (pharmacology)
• Factor VIII (metabolism, therapeutic use)
• Female
• Humans
• Kidney Diseases (therapy)
• Macromolecular Substances
• Recurrence
• Thrombocytopenia (therapy)
• von Willebrand Factor (metabolism)