title: Hematopoietic stem cell transplantation for patients with acute lymphoblastic leukemia and Down syndrome.
abstract: BACKGROUND
  Hematopoietic stem cell transplantation (HSCT) is one curable option for high-risk acute lymphoblastic leukemia (ALL); however, transplant-related toxicities might be severe in patients with Down syndrome and ALL (DS-ALL).
  
  
  METHODS
  HSCTs performed in patients with DS-ALL were identified in the Japan Society for Hematopoietic Cell Transplantation registry.
  
  
  RESULTS
  In the registry data, 11 patients with DS-ALL were identified. The median age at HSCT was 9 years (range: 6-22 years). Six patients underwent HSCT at non-remission status. Allogeneic grafts were utilized in all patients, including eight patients who received HSCT from unrelated donors. Reduced intensity conditioning regimens were used in three patients. All patients achieved neutrophil engraftment by a median of day 18 (range: day 11-61). Ten patients experienced grade 3 or more infectious episodes. Six patients experienced complications of the respiratory system. The incidences of II-IV or III-IV acute GVHD were nine (81.8%) or seven patients (63.6%), respectively. Chronic GVHD was observed in five (55.6%) out of nine evaluable patients. Seven patients died at a median of 6 months (range: 0-24 months) after HSCT. Two-year relapse-free and overall survival were 33.3% (95% CI: 2.5-64.1%) or 37.5% (95% CI: 5.9-69.1%), respectively. The causes of death were relapse (n = 2), infection (n = 2), bleeding (n = 1), thrombotic microangiopathy (n = 1), and chronic GVHD (n = 1).
  
  
  CONCLUSIONS
  Therapy-related mortality accounted for five out of seven deceased patients in this case series. Attempts to reduce toxicities should be considered in HSCT for patients with DS-ALL.
authors.affiliation: Division of Hemato-Oncology and Regenerative Medicine, Kanagawa Children's Medical Center, Yokohama, Japan.
authors.forename: Hiroaki
authors.initials: H
authors.lastname: Goto
authors.forename: Takashi
authors.initials: T
authors.lastname: Kaneko
authors.forename: Yoko
authors.initials: Y
authors.lastname: Shioda
authors.forename: Michiko
authors.initials: M
authors.lastname: Kajiwara
authors.forename: Kazuo
authors.initials: K
authors.lastname: Sakashita
authors.forename: Toshiyuki
authors.initials: T
authors.lastname: Kitoh
authors.forename: Akira
authors.initials: A
authors.lastname: Hayakawa
authors.forename: Mizuka
authors.initials: M
authors.lastname: Miki
authors.forename: Keisuke
authors.initials: K
authors.lastname: Kato
authors.forename: Atsushi
authors.initials: A
authors.lastname: Ogawa
authors.forename: Yoshiko
authors.initials: Y
authors.lastname: Hashii
authors.forename: Takeshi
authors.initials: T
authors.lastname: Inukai
authors.forename: Chiaki
authors.initials: C
authors.lastname: Kato
authors.forename: Hisashi
authors.initials: H
authors.lastname: Sakamaki
authors.forename: Hiromasa
authors.initials: H
authors.lastname: Yabe
authors.forename: Ritsuro
authors.initials: R
authors.lastname: Suzuki
authors.forename: Koji
authors.initials: K
authors.lastname: Kato
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D004314:Down Syndrome / Q000150:complications / Q000401:mortality / Q000628:therapy*; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000453:epidemiology* / Q000401:mortality; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015994:Incidence; D007564:Japan; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000453:epidemiology* / Q000401:mortality; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000150:complications / Q000401:mortality / Q000628:therapy*; D011379:Prognosis; D015996:Survival Rate; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D055815:Young Adult
keywords: Down syndrome; GVHD; acute lymphoblastic leukemia; relapse; transplantation