A model for predicting day-100 stem cell transplant-related mortality in AL amyloidosis Journal Article


Authors: Muchtar, E.; Dispenzieri, A.; Sanchorawala, V.; Hassan, H.; Mwangi, R.; Maurer, M.; Buadi, F.; Lee, H. C.; Qazilbash, M.; Kin, A.; Zonder, J.; Arai, S.; Chin, M. M.; Chakraborty, R.; Lentzsch, S.; Magen, H.; Shkury, E.; Sarubbi, C.; Landau, H.; Schönland, S.; Hegenbart, U.; Gertz, M.
Article Title: A model for predicting day-100 stem cell transplant-related mortality in AL amyloidosis
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. © The Author(s), under exclusive licence to Springer Nature Limited 2025.
Keywords: immunohistochemistry; adult; controlled study; aged; middle aged; major clinical study; overall survival; clinical trial; mortality; area under the curve; cytarabine; outcome assessment; follow up; sensitivity analysis; mass spectrometry; progression free survival; etoposide; cohort analysis; melphalan; autologous stem cell transplantation; stem cell transplantation; hematopoietic stem cell transplantation; retrospective study; prediction; carmustine; alkaline phosphatase; albumin; multicenter study; blood analysis; allogeneic hematopoietic stem cell transplantation; systolic blood pressure; heart arrhythmia; echocardiography; heart left ventricle ejection fraction; forced expiratory volume; decision making; immunoglobulin light chain; logistic regression analysis; lung function test; peripheral nerve; therapy; autonomic nerve; receiver operating characteristic; diagnostic test accuracy study; clinical outcome; albumin blood level; amino terminal pro brain natriuretic peptide; troponin t; al amyloidosis; estimated glomerular filtration rate; humans; human; male; female; article; immunofluorescence assay; random forest; ecog performance status; all cause mortality; diffusing capacity for carbon monoxide; immunoglobulin light-chain amyloidosis; interventricular septum
Journal Title: Bone Marrow Transplantation
Volume: 60
Issue: 5
ISSN: 0268-3369
Publisher: Nature Publishing Group  
Date Published: 2025-05-01
Start Page: 595
End Page: 602
Language: English
DOI: 10.1038/s41409-025-02535-z
PUBMED: 39994333
PROVIDER: scopus
DOI/URL:
Notes: Article -- Source: Scopus
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  1. Heather Jolie Landau
    418 Landau