Blood Adv. 2024 Aug 2:bloodadvances.2024013484. doi: 10.1182/bloodadvances.2024013484. Online ahead of print.
ABSTRACT
Several attempts have been made to optimize pre-transplant risk assessment to improve hematopoietic stem cell transplantation (HSCT) decision-making and to predict outcome post- HSCT. However, its relevance to the pediatric population remains unclear. We report the results of revalidation of the HCT-CI in 874 children who received 944 HSCTs for malignant or non-malignant diseases at a single centre. After finding the HCT-CI invalid in our patient population; we proposed a modified pediatric adapted scoring system that captures risk factors (RF) and comorbidities (CoM) relevant to pediatrics. Each RF/CoM was assigned an integer weight based on its hazard ratio (HR) for TRM; 0 (HR <1.2), 1 (1.2 ≥HR <1.75), 2 (1.75 ≥HR <2.5), 3 (HR ≥2.5) .Using these weights, the pediatric adapted HSCT-RI (PARI) was devised, and patients were divided into 4 risk groups; group 1 without RF/CoM, group 2: scores 1-2, group 3: scores 3-4, group 4: scores ≥5. There was a linear increase in 2-year TRM from group 1 to 4 (TRM= 6.2% in group 1, 50.9% in group 4). PARI was successfully validated on an internal and external cohort of pediatric patients. Comparing models using c-statistics, PARI was found to be a better model than HCT-CI in predicting 2-year TRM in children with Akaike’s and Schwarz’s Bayesian information criteria (AIC and BIC) of 1069.245 and 1073.269; respectively using PARI vs 1223.158 and 1227.051; respectively using HCT-CI. We believe that PARI will be a valuable tool enabling better counselling and decision making for pediatric HSCT patients.
PMID:39093984 | DOI:10.1182/bloodadvances.2024013484
