Validation and refinement of the Disease Risk Index for allogeneic stem cell transplantation

Area of Science:

• Hematology
• Oncology
• Transplantation Medicine

Background:

• Allogeneic hematopoietic cell transplantation (HCT) outcomes are heavily influenced by disease type and status.
• Accurate patient stratification by disease risk is essential for HCT success.
• The Disease Risk Index (DRI) was previously developed to address this need.

Purpose of the Study:

• To analyze a large cohort of HCT patients to validate and refine the Disease Risk Index (DRI).
• To improve the prognostic accuracy of the DRI for stratifying patients undergoing HCT.
• To establish a reliable tool for interpreting HCT outcomes and guiding clinical trial enrollment.

Main Methods:

• Analysis of 13,131 patients undergoing HCT between 2008 and 2010 from the Center for International Blood and Marrow Transplant Research (CIBMTR) database.
• A multivariable regression model was used on a training subgroup (9849 patients) to refine the DRI based on overall survival (OS).
• The refined DRI's predictive ability was validated on a separate subgroup of 3282 patients.

Main Results:

• The original DRI stratified patients into 4 groups with 2-year OS ranging from 64% to 24%.
• The DRI was identified as the strongest prognostic factor, outperforming age, conditioning intensity, graft source, or donor type.
• The refined DRI demonstrated improved prediction accuracy compared to the original DRI and other existing schemes.

Conclusions:

• The validated and refined DRI provides a robust tool for prognostication in allogeneic HCT.
• The DRI can be utilized as a 3- or 4-group index for cohort stratification and outcome interpretation.
• This refined index facilitates the comparison of studies, adjustment of center outcome data, and patient stratification in clinical trials.