title: Clinical characteristics and outcome of refractory/relapsed myeloid leukemia in children with Down syndrome.
abstract: Myeloid leukemia in Down syndrome (ML-DS) is associated with good response to chemotherapy and favorable prognosis. Because little research has been focused on refractory/relapsed (R/R) cases, we conducted a retrospective analysis for R/R ML-DS. Among ML-DS patients diagnosed between 2000 and 2010 in Japan, 26 relapsed (25 in the BM and 1 in the skin), and 3 refractory patients were enrolled. The male/female ratio was 18/11. The median age at initial diagnosis of ML-DS was 2 years, and the median time to relapse was 8.6 months. Each patient initially had been treated with ML-DS-specific protocols. Thirteen of the 26 patients achieved complete remission with various kinds of reinduction chemotherapies; 2 of 8 survived without further recurrence after receiving allogeneic hematopoietic stem cell transplantation, and 4 of 5 maintained complete remissions with chemotherapy alone. Treatment failures mostly were associated with disease progression rather than treatment-related toxicities. The 3-year OS rate was 25.9% ± 8.5%. A longer duration from initial diagnosis to relapse was a significant favorable prognostic factor (P < .0001). We conclude that clinical outcome for patients with R/R ML-DS generally are unfavorable, even in those receiving hematopoietic stem cell transplantation. Novel methods to identify poor prognostic factors for ML-DS are necessary.
authors: Department of Pediatrics, Shiga University of Medical Science, Shiga, Japan. ttaga@belle.shiga-med.ac.jp
authors: Takashi
authors: T
authors: Taga
authors: Akiko Moriya
authors: AM
authors: Saito
authors: Kazuko
authors: K
authors: Kudo
authors: Daisuke
authors: D
authors: Tomizawa
authors: Kiminori
authors: K
authors: Terui
authors: Hiroshi
authors: H
authors: Moritake
authors: Akitoshi
authors: A
authors: Kinoshita
authors: Shotaro
authors: S
authors: Iwamoto
authors: Hideki
authors: H
authors: Nakayama
authors: Hiroyuki
authors: H
authors: Takahashi
authors: Akio
authors: A
authors: Tawa
authors: Akira
authors: A
authors: Shimada
authors: Tomohiko
authors: T
authors: Taki
authors: Hisato
authors: H
authors: Kigasawa
authors: Katsuyoshi
authors: K
authors: Koh
authors: Souichi
authors: S
authors: Adachi
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018450:Disease Progression; D004314:Down Syndrome / Q000150:complications* / Q000235:genetics; D019008:Drug Resistance, Neoplasm; D005260:Female; D050982:GATA1 Transcription Factor / Q000235:genetics; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D059785:Karyotype; D007951:Leukemia, Myeloid / Q000150:complications / Q000235:genetics / Q000628:therapy*; D008297:Male; D015999:Multivariate Analysis; D009154:Mutation; D011379:Prognosis; D012008:Recurrence; D012189:Retrospective Studies; D016019:Survival Analysis; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D050982:GATA1 Transcription Factor; C494688:GATA1 protein, human