A prognostic score including mutation profile and clinical features for patients with CMML undergoing stem cell transplantation
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A prognostic score including mutation profile and clinical features for patients with CMML undergoing stem cell transplantation. / Gagelmann, Nico; Badbaran, Anita; Beelen, Dietrich W; Salit, Rachel B; Stölzel, Friedrich; Rautenberg, Christina; Becker, Heiko; Radujkovic, Aleksandar; Panagiota, Victoria; Bogdanov, Rashit; Christopeit, Maximilian; Park, Yong; Nibourel, Olivier; Luft, Thomas; Koldehoff, Michael; Corsten, Maarten; Heuser, Michael; Finke, Jürgen; Kobbe, Guido; Platzbecker, Uwe; Robin, Marie; Scott, Bart L; Kröger, Nicolaus.
In: BLOOD ADV, Vol. 5, No. 6, 23.03.2021, p. 1760-1769.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - A prognostic score including mutation profile and clinical features for patients with CMML undergoing stem cell transplantation
AU - Gagelmann, Nico
AU - Badbaran, Anita
AU - Beelen, Dietrich W
AU - Salit, Rachel B
AU - Stölzel, Friedrich
AU - Rautenberg, Christina
AU - Becker, Heiko
AU - Radujkovic, Aleksandar
AU - Panagiota, Victoria
AU - Bogdanov, Rashit
AU - Christopeit, Maximilian
AU - Park, Yong
AU - Nibourel, Olivier
AU - Luft, Thomas
AU - Koldehoff, Michael
AU - Corsten, Maarten
AU - Heuser, Michael
AU - Finke, Jürgen
AU - Kobbe, Guido
AU - Platzbecker, Uwe
AU - Robin, Marie
AU - Scott, Bart L
AU - Kröger, Nicolaus
N1 - © 2021 by The American Society of Hematology.
PY - 2021/3/23
Y1 - 2021/3/23
N2 - 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.
AB - 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.
KW - Hematopoietic Stem Cell Transplantation
KW - Humans
KW - Leukemia, Myelomonocytic, Chronic/diagnosis
KW - Mutation
KW - Prognosis
KW - Stem Cell Transplantation
U2 - 10.1182/bloodadvances.2020003600
DO - 10.1182/bloodadvances.2020003600
M3 - SCORING: Journal article
C2 - 33755092
VL - 5
SP - 1760
EP - 1769
JO - BLOOD ADV
JF - BLOOD ADV
SN - 2473-9529
IS - 6
ER -
