External validation and comparison of multiple prognostic scores in allogeneic hematopoietic stem cell transplantation

Abstract

Clinical decisions in allogeneic hematopoietic stem cell transplantation (allo-HSCT) are supported by the use of prognostic scores for outcome prediction. Scores vary in their features and in the composition of development cohorts. We sought to externally validate and compare the performance of 8 commonly applied scoring systems on a cohort of allo-HSCT recipients. Among 528 patients studied, acute myeloid leukemia was the leading transplant indication (44%) and 46% of patients had a matched sibling donor. Most models successfully grouped patients into higher and lower risk strata, supporting their use for risk classification. However, discrimination varied (2-year overall survival area under the receiver operating characteristic curve [AUC]: revised Pretransplantation Assessment of Mortality [rPAM], 0.64; PAM, 0.63; revised Disease Risk Index [rDRI], 0.62; Endothelial Activation and Stress Index [EASIx], 0.60; combined European Society for Blood and Marrow Transplantation [EBMT]/Hematopoietic Cell Transplantation-specific Comorbidity Index [HCT-CI], 0.58; EBMT, 0.58; Comorbidity-Age, 0.58; HCT-CI, 0.55); AUC ranges from 0.5 (random) to 1.0 (perfect prediction). rPAM and PAM, which had the greatest predictive capacity across all outcomes, are comprehensive models including patient, disease, and transplantation information. Interestingly, EASIx, a biomarker-driven model, had comparable performance for nonrelapse mortality (NRM; 2-year AUC, 0.65) but no predictive value for relapse (2-year AUC, 0.53). Overall, allo-HSCT prognostic systems may be useful for risk stratification, but individual prediction remains a challenge, as reflected by the scores’ limited discriminative capacity.

Cardiopulmonary performance in allogeneic hematopoietic cell transplantation recipients—evaluation of pre-transplant risk assessments

“…In this context, calculating individual risk scores to predict overall survival (OS) and non-relapse mortality (NRM) are used prior to alloHCT. Commonly used scores are [4,5] the European Blood and Bone Marrow Transplantation (EBMT) score [6], the Hematopoietic Cell Transplantation-Comorbidity-Index (HCT-CI) [7] the revised Disease Risk Index (rDRI) [8] and the revised Pretransplant Assessment of Mortality score (rPAM) [9]. As none of these scores covers all important variables, the concurrent use of various scores is recommended to include various aspects of risk prediction.…”

“…As none of these scores covers all important variables, the concurrent use of various scores is recommended to include various aspects of risk prediction. However, according to an analysis by Shouval et al [5] the forecast reliability of those scores ranges from moderate to random. To specify the individual risk-benefit ratio and provide an additional basis for medical decisions, e.g., the intensity of conditioning therapy, patients' organ functions are usually determined by medical tests.…”

Endothelial activation and stress index (EASIX) is a reliable predictor for overall survival in patients with multiple myeloma

“…They subsequently proposed that EASIX could predict the survival outcome in lower-risk myelodysplastic syndrome which is not a candidate for allogeneic stem cell transplantation [ 6 ]. The prognostic impact of EASIX in allogeneic stem cell transplantation was externally validated in generalized population cohorts [ 7 – 9 ]. Platelet count, serum creatinine and LDH, which make up EASIX, are well-known prognostic factors for MM.…”

Pre-transplant depression decreased overall survival of patients receiving allogeneic hematopoietic stem cell transplantation: a nationwide cohort study

“…Besides primary hematologic disorder and disease relapse after transplantation, transplant-related mortality is an important obstacle influencing treatment outcome of allo-HSCT 4 . Thus, careful assessment of risk and accurate prediction of mortality are necessary before undertaking allo-HSCT 5 .…”