Eli Muchtar, Angela Dispenzieri, Vaishali Sanchorawala, Hamza Hassan, Raphael Mwangi, Matthew Maurer, Francis Buadi, Hans C Lee, Muzaffar Qazilbash, Andrew Kin, Jeffrey Zonder, Sally Arai, Michelle M Chin, Rajshekhar Chakraborty, Suzanne Lentzsch, Hila Magen, Eden Shkury, Caitlin Sarubbi, Heather Landau, Stefan Schönland, Ute Hegenbart, Morie Gertz
{"title":"A model for predicting day-100 stem cell transplant-related mortality in AL amyloidosis.","authors":"Eli Muchtar, Angela Dispenzieri, Vaishali Sanchorawala, Hamza Hassan, Raphael Mwangi, Matthew Maurer, Francis Buadi, Hans C Lee, Muzaffar Qazilbash, Andrew Kin, Jeffrey Zonder, Sally Arai, Michelle M Chin, Rajshekhar Chakraborty, Suzanne Lentzsch, Hila Magen, Eden Shkury, Caitlin Sarubbi, Heather Landau, Stefan Schönland, Ute Hegenbart, Morie Gertz","doi":"10.1038/s41409-025-02535-z","DOIUrl":null,"url":null,"abstract":"<p><p>Autologous stem cell Transplant (ASCT)-related mortality (TRM) in AL amyloidosis remains elevated. AL amyloidosis patients (n = 1718) from 9 centers, transplanted 2003-2020 were included. Pre-ASCT variables of interest were assessed for association with day-100 all-cause mortality. A random forest (RF) classifier with 10-fold cross-validation assisted in variable selection. The final model was fitted using logistic regression. The median age at ASCT was 58 years. Day-100 TRM occurred in 75 patients (4.4%) with the predominant causes being shock, high-grade arrhythmia, and organ failure. Ten factors were associated with day-100 TRM on univariate analysis. RF classifier using these variables generated a model with an area under the curve (AUC) of 0.72 ± 0.12. To refine the model selection using importance hierarchy function, a 4-variable model [NT-proBNP/BNP, serum albumin, ECOG performance status (PS), and systolic blood pressure] was built with an AUC of 0.70 ± 0.12. Based on logistic regression coefficients, ECOG PS 2/3 was assigned two points while other adverse predictors 1-point each. The model score range was 0-5, with a day-100 TRM of 0.46%, 3.2%, 5.8%, and 14.5% for 0, 1, 2, and ≥3 points, respectively. This model to predict day-100 TRM in AL amyloidosis allows better-informed decision-making in this heterogeneous disease.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5000,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Bone Marrow Transplantation","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1038/s41409-025-02535-z","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Autologous stem cell Transplant (ASCT)-related mortality (TRM) in AL amyloidosis remains elevated. AL amyloidosis patients (n = 1718) from 9 centers, transplanted 2003-2020 were included. Pre-ASCT variables of interest were assessed for association with day-100 all-cause mortality. A random forest (RF) classifier with 10-fold cross-validation assisted in variable selection. The final model was fitted using logistic regression. The median age at ASCT was 58 years. Day-100 TRM occurred in 75 patients (4.4%) with the predominant causes being shock, high-grade arrhythmia, and organ failure. Ten factors were associated with day-100 TRM on univariate analysis. RF classifier using these variables generated a model with an area under the curve (AUC) of 0.72 ± 0.12. To refine the model selection using importance hierarchy function, a 4-variable model [NT-proBNP/BNP, serum albumin, ECOG performance status (PS), and systolic blood pressure] was built with an AUC of 0.70 ± 0.12. Based on logistic regression coefficients, ECOG PS 2/3 was assigned two points while other adverse predictors 1-point each. The model score range was 0-5, with a day-100 TRM of 0.46%, 3.2%, 5.8%, and 14.5% for 0, 1, 2, and ≥3 points, respectively. This model to predict day-100 TRM in AL amyloidosis allows better-informed decision-making in this heterogeneous disease.
期刊介绍:
Bone Marrow Transplantation publishes high quality, peer reviewed original research that addresses all aspects of basic biology and clinical use of haemopoietic stem cell transplantation.
The broad scope of the journal thus encompasses topics such as stem cell biology, e.g., kinetics and cytokine control, transplantation immunology e.g., HLA and matching techniques, translational research, and clinical results of specific transplant protocols. Bone Marrow Transplantation publishes 24 issues a year.