title: Management of type 2b von Willebrand disease in the neonatal period.
abstract: Von Willebrand disease (VWD) is the most common inherited bleeding disorder, affecting one in 1,000 people. Type 2b VWD is a less common subtype caused by a gain-of-function mutation in von Willebrand factor (VWF) that leads to the formation of large, ineffective VWF-platelet multimers in circulation. This unique pathophysiology creates diagnostic and treatment dilemmas. There is limited information on the management of type 2b VWD in the neonatal period. This report describes the management of a neonate with type 2b VWD with an emphasis on the added benefit of concomitant platelet transfusion and factor replacement therapy over factor replacement therapy alone.
authors.affiliation: Pediatrics Residency Program, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA.
authors.forename: Lindsay
authors.initials: L
authors.lastname: Proud
authors.affiliation: Division of Hematology/Oncology, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA.
authors.forename: A Kim
authors.initials: AK
authors.lastname: Ritchey
mesh_terms: D003131:Combined Modality Therapy; D019468:Disease Management; D006801:Humans; D007231:Infant, Newborn; D008297:Male; D017713:Platelet Transfusion; D011379:Prognosis; D056728:von Willebrand Disease, Type 2 / Q000628:therapy*; D014841:von Willebrand Factor / Q000008:administration & dosage*
chemical_list: D014841:von Willebrand Factor
keywords: management; neonatal; platelet transfusion; von Willebrand disease