title: Resolution of Budd-Chiari syndrome following bone marrow transplantation for paroxysmal nocturnal haemoglobinuria.
abstract: Thrombosis of the hepatic veins (Budd-Chiari syndrome) is a life-threatening thrombotic complication which can occur in patients with paroxysmal nocturnal haemoglobinuria (PNH). Despite aggressive medical and surgical therapy, mortality from Budd-Chiari syndrome remains high. We report a boy with PNH who developed Budd-Chiari syndrome and underwent syngeneic bone marrow transplantation (BMT). Now, 3 years following BMT, he has had dramatic clinical and radiographic evidence of resolution of the thrombosis. We suggest that BMT for PNH can successfully correct life-threatening thrombosis in patients with PNH.
authors.affiliation: Department of pediatrics, Duke University Medical Center, Durham, North Carolina 27710, USA.
authors.forename: M L
authors.initials: ML
authors.lastname: Graham
authors.forename: W F
authors.initials: WF
authors.lastname: Rosse
authors.forename: E C
authors.initials: EC
authors.lastname: Halperin
authors.forename: C R
authors.initials: CR
authors.lastname: Miller
authors.forename: R E
authors.initials: RE
authors.lastname: Ware
mesh_terms: D016026:Bone Marrow Transplantation; D006502:Budd-Chiari Syndrome / Q000209:etiology / Q000473:pathology / Q000628:therapy*; D002648:Child; D006457:Hemoglobinuria, Paroxysmal / Q000150:complications / Q000473:pathology / Q000628:therapy*; D006801:Humans; D008099:Liver / Q000473:pathology; D008279:Magnetic Resonance Imaging; D008297:Male