title: A pediatric prognostic score for patients undergoing allogeneic hematopoietic stem cell transplantation.
abstract: OBJECTIVE
  The purpose of this study was to find and evaluate risk factors influencing the outcome of allogeneic hematopoietic stem cell transplantation (HSCT) in children and to develop a score stratifying patients by their risk of mortality.
  
  
  METHODS
  We retrospectively analyzed the effects of patient and donor characteristics as well as laboratory data including liver, iron, and inflammation parameters on the overall survival of 131 children and young adults with malignant and non-malignant diseases undergoing allogeneic HSCT.
  
  
  RESULTS
  In univariate analysis, 5-yr overall survival decreased significantly in patients with high disease risk (38% vs. 74%, P < 0.001), peripheral blood as graft source (47% vs. 73% for bone marrow, P < 0.001), ferritin >1500 ng/mL (41% vs. 79%, P = 0.001), C-reactive protein >10 mg/L (55% vs. 69%, P = 0.019), lactate dehydrogenase >6 μm∙s (22% vs. 67%, P = 0.001), gamma-glutamyl transpeptidase >1 μm∙s (43% vs. 68%, P = 0.035), and cholinesterase <60 μm∙s (36% vs. 70%, P = 0.002). For HLA recipient-donor match, there was a 5-yr overall survival of 81% for matched related, 58% for matched unrelated, 56% for mismatched unrelated, and 50% for haploidentical related donors (P = 0.020). We subsequently developed a score of parameters significant in multivariate analysis, that is, disease risk (HR = 4.1, P = 0.027), ferritin (HR = 6.4, P = 0.002), and cholinesterase (HR = 5.3, P = 0.027). For this score, 5-yr overall survival was 92% for the low-risk group, 66% for the intermediate-risk group, and 17% for the high-risk group (P < 0.001).
  
  
  CONCLUSIONS
  Disease risk, ferritin, and cholinesterase are factors decisively influencing the prognosis after HSCT and should be evaluated in further trials.
authors.affiliation: Section for Hematology and Oncology, Department of Pediatrics, Jena University Hospital, Jena, Germany.
authors.forename: Clemens
authors.initials: C
authors.lastname: Arndt
authors.forename: James F
authors.initials: JF
authors.lastname: Beck
authors.forename: Bernd
authors.initials: B
authors.lastname: Gruhn
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D008875:Middle Aged; D009026:Mortality; D063868:Patient Outcome Assessment; D011379:Prognosis; D012189:Retrospective Studies; D012307:Risk Factors; D014019:Tissue Donors; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
keywords: children; cholinesterase; disease risk; hematopoietic stem cell transplantation; score; serum ferritin