title: Hematologic Response to Vorinostat Treatment in Relapsed Myeloid Leukemia of Down Syndrome.
abstract: Children with Down syndrome are at high risk to develop myeloid leukemia (ML-DS). Despite their excellent prognosis, children with ML-DS particularly suffer from severe therapy-related toxicities and for relapsed ML-DS the cure rates are very poor. Here we report the clinical course of one child with ML-DS treated with the histone deacetylase (HDAC) inhibitor vorinostat (suberoylanilide hydroxamic acid) after second relapse. The child had previously received conventional chemotherapy and stem cell transplantation, yet showed a remarkable clinical and hematologic response. Thus, HDAC inhibitor may represent an effective class of drugs for the treatment of ML-DS.
authors.affiliation: Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany.
authors.forename: Carina
authors.initials: C
authors.lastname: Scheer
authors.affiliation: Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany.
authors.forename: Christian
authors.initials: C
authors.lastname: Kratz
authors.affiliation: Pediatric Oncology, German Cancer Research Center (DKFZ), University Hospital and National Center for Tumor Diseases, Heidelberg, Germany.
authors.forename: Olaf
authors.initials: O
authors.lastname: Witt
authors.affiliation: Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany.
authors.forename: Ursula
authors.initials: U
authors.lastname: Creutzig
authors.affiliation: Clinic for Pediatrics III, University Hospital Essen, Essen, Germany.
authors.forename: Dirk
authors.initials: D
authors.lastname: Reinhardt
authors.affiliation: Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany.
authors.forename: Jan-Henning
authors.initials: JH
authors.lastname: Klusmann
mesh_terms: D002675:Child, Preschool; D003430:Cross-Sectional Studies; D004314:Down Syndrome / Q000150:complications*; D056572:Histone Deacetylase Inhibitors / Q000627:therapeutic use*; D006801:Humans; D006877:Hydroxamic Acids / Q000627:therapeutic use*; D007951:Leukemia, Myeloid / Q000097:blood / Q000188:drug therapy*; D008297:Male; D000077337:Vorinostat
chemical_list: D056572:Histone Deacetylase Inhibitors; D006877:Hydroxamic Acids; D000077337:Vorinostat
keywords: AML; Down syndrome; HDAC; HDACi; ML-DS; histone deacetylase inhibitor; vorinostat