title: Azacitidine in the treatment of pediatric therapy-related myelodysplastic syndrome after allogeneic hematopoietic stem cell transplantation.
abstract: We herein present a case of pediatric therapy-related myelodysplastic syndrome (t-MDS) with complex karyotype who was treated with azacitidine (AZA) for AML1-EVI1 fusion transcript as minimal residual disease after allogeneic hematopoietic stem cell transplantation (HSCT). The patient was started on AZA 41 days after the HSCT without having achieved complete remission. After 9 cycles of AZA, the AML1-EVI1 fusion transcript disappeared, and there was no manifestation of graft versus host disease during AZA treatment. Preemptive AZA treatment for minimal residual disease has an acceptable safety profile and appears to be an effective strategy for preventing or substantially delaying hematological relapse in pediatric patients with high-risk myelodysplastic syndrome after HSCT.
authors.affiliation: Department of Pediatrics, Osaka Medical College, Takatsuki, Osaka, Japan.
authors.forename: Akiko
authors.initials: A
authors.lastname: Inoue
authors.forename: Chihiro
authors.initials: C
authors.lastname: Kawakami
authors.forename: Kimitaka
authors.initials: K
authors.lastname: Takitani
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Tamai
mesh_terms: D000964:Antimetabolites, Antineoplastic / Q000627:therapeutic use*; D001374:Azacitidine / Q000627:therapeutic use*; D002648:Child; D006086:Graft vs Host Disease / Q000188:drug therapy* / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D008297:Male; D009190:Myelodysplastic Syndromes / Q000150:complications* / Q000628:therapy; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy* / Q000209:etiology; D018365:Neoplasm, Residual / Q000188:drug therapy* / Q000209:etiology; D011379:Prognosis; D014184:Transplantation, Homologous
chemical_list: D000964:Antimetabolites, Antineoplastic; D001374:Azacitidine