A pediatric prognostic score for patients undergoing allogeneic hematopoietic stem cell transplantation

Purpose: 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 mu M.s (22% vs. 67%, P = 0.001), gamma-glutamyl transpeptidase >1 mu M.s (43% vs. 68%, P = 0.035), and cholinesterase < 60 mu M.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.