title: Unrelated donor hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis.
abstract: We report outcomes after unrelated donor hematopoietic cell transplantation (HCT) for 91 patients with hemophagocytic lymphohistiocytosis (HLH) transplanted in the US in 1989-2005. Fifty-one percent were <1 year at HCT and 29% had Lansky performance scores<90%. Most (80%) were conditioned with BU, CY, and etoposide (VP16) with or without anti-thymocyte globulin. Bone marrow was the predominant graft source. Neutrophil recovery was 91% at day-42. The probabilities of grades 2-4 acute GVHD at day-100 and chronic GVHD at 5 years were 41 and 23%, respectively. The overall mortality rate was higher in patients who did not receive BU/CY/VP16-conditioning regimen (RR 1.95, P=0.035). The 5-year probability of overall survival was 53% in patients who received BU/CY/VP16 compared to 24% in those who received other regimens. In the subset of patients with known disease-specific characteristics, only one of five patients with active disease at HCT is alive. For those in clinical remission at HCT (n=46), the 5-year probability of overall survival was 49%. Early mortality rates after HCT were high, 35% at day-100. These data demonstrate that a BU/CY/VP16-conditioning regimen provides cure in approximately 50% of patients and future studies should explore strategies to lower early mortality.
authors.affiliation: University of Minnesota Medical Center, Minneapolis, MN, USA.
authors.forename: K S
authors.initials: KS
authors.lastname: Baker
authors.forename: A H
authors.initials: AH
authors.lastname: Filipovich
authors.forename: T G
authors.initials: TG
authors.lastname: Gross
authors.forename: W J
authors.initials: WJ
authors.lastname: Grossman
authors.forename: G A
authors.initials: GA
authors.lastname: Hale
authors.forename: R J
authors.initials: RJ
authors.lastname: Hayashi
authors.forename: N R
authors.initials: NR
authors.lastname: Kamani
authors.forename: S
authors.initials: S
authors.lastname: Kurian
authors.forename: N
authors.initials: N
authors.lastname: Kapoor
authors.forename: O
authors.initials: O
authors.lastname: Ringdén
authors.forename: M
authors.initials: M
authors.lastname: Eapen
mesh_terms: D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000453:epidemiology; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000401:mortality; D006801:Humans; D007223:Infant; D051359:Lymphohistiocytosis, Hemophagocytic / Q000401:mortality / Q000601:surgery*; D008297:Male; D011336:Probability; D012189:Retrospective Studies; D015996:Survival Rate; D017741:Survivors; D013997:Time Factors; D014019:Tissue Donors / Q000706:statistics & numerical data; D019172:Transplantation Conditioning