title: Azacitidine as a bridge to allogeneic hematopoietic cell transplantation in a pediatric patient with Fanconi anemia and acute myeloid leukemia.
abstract: HCT is the definitive therapy for patients with FA and AML. Conventional cytotoxic agents cause potential DNA damage, and currently, there is no established regimen for these patients prior to HCT. A 13-year-old male with FA and refractory AML was given azacitidine, achieved morphologic remission and underwent HCT. At 95 days after HCT, he relapsed. Azacitidine along with DLI was used as first salvage therapy. Azacitidine was overall well tolerated with minimal side effects. In patients with AML and FA, azacitidine can be considered an alternative to conventional chemotherapy.
authors.affiliation: Pediatric Hematology-Oncology and Bone Marrow Transplantation, University Hospitals Rainbow Babies & Children's Hospital/Case Medical Center, Cleveland, OH, USA.
authors.forename: Hilda
authors.initials: H
authors.lastname: Ding
authors.affiliation: Pediatric Hematology-Oncology and Bone Marrow Transplantation, University Hospitals Rainbow Babies & Children's Hospital/Case Medical Center, Cleveland, OH, USA.
authors.forename: Hasan
authors.initials: H
authors.lastname: Hashem
authors.affiliation: Pediatric Hematology-Oncology and Bone Marrow Transplantation, University Hospitals Rainbow Babies & Children's Hospital/Case Medical Center, Cleveland, OH, USA.
authors.forename: Linda
authors.initials: L
authors.lastname: Cabral
authors.affiliation: Pediatric Hematology Oncology and Bone Marrow Transplant, Nationwide Children's Hospital, Columbus, OH, USA.
authors.forename: Hemalatha
authors.initials: H
authors.lastname: Rangarajan
authors.affiliation: Pediatric Hematology/Oncology and Bone Marrow Transplant, University of Iowa Children's Hospital, Iowa City, IA, USA.
authors.forename: Ghada
authors.initials: G
authors.lastname: Abusin
authors.affiliation: Department of Medicine-Hematology and Oncology, University Hospitals Case Medical Center, Cleveland, OH, USA.
authors.forename: Hillard M
authors.initials: HM
authors.lastname: Lazarus
authors.affiliation: Pediatric Hematology Oncology and Bone Marrow Transplant, Nationwide Children's Hospital, Columbus, OH, USA.
authors.forename: Rolla
authors.initials: R
authors.lastname: Abu-Arja
mesh_terms: D000293:Adolescent; D001374:Azacitidine / Q000627:therapeutic use*; D003603:Cytotoxins / Q000627:therapeutic use; D004249:DNA Damage; D005199:Fanconi Anemia / Q000150:complications* / Q000628:therapy*; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000150:complications* / Q000628:therapy*; D008297:Male; D012074:Remission Induction; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D003603:Cytotoxins; D001374:Azacitidine
keywords: Fanconi anemia; acute myeloid leukemia; azacitidine; donor lymphocyte infusion; hematopoietic cell transplant; pediatric