title: Hepatic GVHD after HLA-haploidentical bone marrow transplantation in children with severe combined immunodeficiency: the effect of ursodeoxycholic acid.
abstract: We describe two children with a severe combined immune deficiency (SCID) with B cells. Following a T-cell-depleted haploidentical bone marrow transplantation (BMT), they both developed a chronic graft-versus-host disease (GVHD) of the skin and a severe persisting hyperbilirubinaemia and elevated liver enzymes. The diagnosis of a vanishing bile duct syndrome was confirmed by liver biopsies. Because corticosteroids and cyclosporin A induced only a partial response, ursodeoxycholic acid (UDCA) was added to their treatment schedule. Serum bilirubin and liver enzymes returned to normal within months. A control liver biopsy showed normal and proliferating bile ducts without cholestatic damage. We conclude that UDCA was well tolerated and may be of value as an additional treatment for hepatic GVHD in SCID.
authors.affiliation: Department of Immunology, University Hospital for Children Het Wilhelmina Kinderziekenhuis, Utrecht, The Netherlands.
authors.forename: N M
authors.initials: NM
authors.lastname: Wulffraat
authors.forename: E
authors.initials: E
authors.lastname: Haddad
authors.forename: M
authors.initials: M
authors.lastname: Benkerrou
authors.forename: W G
authors.initials: WG
authors.lastname: Spliet
authors.forename: N
authors.initials: N
authors.lastname: Patey
authors.forename: A
authors.initials: A
authors.lastname: Fischer
authors.forename: B R
authors.initials: BR
authors.lastname: de Graeff-Meeder
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D001649:Bile Duct Diseases / Q000188:drug therapy; D001663:Bilirubin / Q000097:blood; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000379:methods*; D002756:Cholagogues and Choleretics / Q000627:therapeutic use; D016572:Cyclosporine / Q000627:therapeutic use; D005355:Fibrosis / Q000188:drug therapy; D006086:Graft vs Host Disease / Q000209:etiology*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D007231:Infant, Newborn; D008107:Liver Diseases / Q000209:etiology*; D008297:Male; D016511:Severe Combined Immunodeficiency / Q000628:therapy*; D012871:Skin Diseases / Q000209:etiology*; D014580:Ursodeoxycholic Acid / Q000627:therapeutic use
chemical_list: D000305:Adrenal Cortex Hormones; D002756:Cholagogues and Choleretics; D007166:Immunosuppressive Agents; D014580:Ursodeoxycholic Acid; D016572:Cyclosporine; D001663:Bilirubin