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A prognostic score including mutation profile and clinical features for patients with CMML undergoing stem cell transplantation

The inclusion of mutation status improved risk stratification for newly diagnosed patients with chronic myelomonocytic leukemia (CMML). Stem cell transplantation is a potentially curative treatment option, and patient selection is critical because of relevant transplant-related morbidity and mortali...

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Main Authors: Gagelmann, Nico (Author) , Badbaran, Anita (Author) , Beelen, Dietrich W. (Author) , Salit, Rachel B. (Author) , Stölzel, Friedrich (Author) , Rautenberg, Christina (Author) , Becker, Heiko (Author) , Radujković, Aleksandar (Author) , Panagiota, Victoria (Author) , Bogdanov, Rashit (Author) , Christopeit, Maximilian (Author) , Park, Yong (Author) , Nibourel, Olivier (Author) , Luft, Thomas (Author) , Koldehoff, Michael (Author) , Corsten, Maarten (Author) , Heuser, Michael (Author) , Finke, Jürgen (Author) , Kobbe, Guido (Author) , Platzbecker, Uwe (Author) , Robin, Marie (Author) , Scott, Bart L. (Author) , Kröger, Nicolaus (Author)
Format: Article (Journal)
Language:English
Published: 23 March 2021
In: Blood advances
Year: 2021, Volume: 5, Issue: 6, Pages: 1760-1769
ISSN:2473-9537
DOI:10.1182/bloodadvances.2020003600
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1182/bloodadvances.2020003600
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Author Notes:Nico Gagelmann, Anita Badbaran, Dietrich W. Beelen, Rachel B. Salit, Friedrich Stölzel, Christina Rautenberg, Heiko Becker, Aleksandar Radujkovic, Victoria Panagiota, Rashit Bogdanov, Maximilian Christopeit, Yong Park, Olivier Nibourel, Thomas Luft, Michael Koldehoff, Maarten Corsten, Michael Heuser, Jürgen Finke, Guido Kobbe, Uwe Platzbecker, Marie Robin, Bart L. Scott and Nicolaus Kröger
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Summary:The inclusion of mutation status improved risk stratification for newly diagnosed patients with chronic myelomonocytic leukemia (CMML). Stem cell transplantation is a potentially curative treatment option, and patient selection is critical because of relevant transplant-related morbidity and mortality. We aimed to evaluate the impact of mutation status together with clinical presentations on posttransplant outcome. Our study included 240 patients with a median follow-up of 5.5 years. A significant association with worse survival was identified for the presence of mutations in ASXL1 and/or NRAS. In multivariable analysis, ASXL1- and/or NRAS-mutated genotype (hazard ratio [HR], 1.63), marrow blasts >2% (HR, 1.70), and increasing comorbidity index (continuous HR, 1.16) were independently associated with worse survival. A prognostic score (CMML transplant score) was developed, and the following points were assigned: 4 points for an ASXL1- and/or NRAS-mutated genotype or blasts >2% and 1 point each for an increase of 1 in the comorbidity index. The CMML transplant score (range, 0-20) was predictive of survival and nonrelapse mortality (P < .001 for both). Up to 5 risk groups were identified, showing 5-year survival of 81% for a score of 0 to 1, 49% for a score of 2 to 4, 43% for a score of 5 to 7, 31% for a score of 8 to 10, and 19% for a score >10. The score retained performance after validation (concordance index, 0.68) and good accuracy after calibration. Predictions were superior compared with existing scores designed for the nontransplant setting, which resulted in significant risk reclassification. This CMML transplant score, which incorporated mutation and clinical information, was prognostic in patients specifically undergoing transplantation and may facilitate personalized counseling.
Item Description:Gesehen am 18.07.2021
Physical Description:Online Resource
ISSN:2473-9537
DOI:10.1182/bloodadvances.2020003600

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