Core-binding factor acute myeloid leukemia with t(8;21) Risk factors and a novel scoring system (I-CBFit)

Celalettin Ustun; Elizabeth Morgan; Erica E. M. Moodie; Sheeja Pullarkat; Cecilia Yeung; Sigurd Broesby-Olsen; Robert Ohgami; Young Kim; Wolfgang Sperr; Hanne Vestergaard; Dong Chen; Philip M. Kluin; Michelle Dolan; Krzysztof Mrozek; David Czuchlewski; Hans-Peter Horny; Tracy I. George; Thomas Kielsgaard Kristensen; Nam K. Ku; Cecilia Arana Yi; Michael Boe Moller; Guido Marcucci; Linda Baughn; Ana-Iris Schiefer; J. R. Hilberink; Vinod Pullarkat; Ryan Shanley; Jessica Kohlschmidt; Janie Coulombe; Amandeep Salhotra; Lori Soma; Christina Cho; Michael A. Linden; Cem Akin; Jason Gotlib; Gregor Hoermann; Jason Hornick; Ryo Nakamura; Joachim Deeg; Clara D. Bloomfield; Daniel Weisdorf; Mark R. Litzow; Peter Valent; Gerwin Huls; Miguel-Angel Perales; Gautam Borthakur

doi:10.1002/cam4.1733

Core-binding factor acute myeloid leukemia with t(8;21) Risk factors and a novel scoring system (I-CBFit)

Celalettin Ustun^*, Elizabeth Morgan, Erica E. M. Moodie, Sheeja Pullarkat, Cecilia Yeung, Sigurd Broesby-Olsen, Robert Ohgami, Young Kim, Wolfgang Sperr, Hanne Vestergaard, Dong Chen, Philip M. Kluin, Michelle Dolan, Krzysztof Mrozek, David Czuchlewski, Hans-Peter Horny, Tracy I. George, Thomas Kielsgaard Kristensen, Nam K. Ku, Cecilia Arana YiMichael Boe Moller, Guido Marcucci, Linda Baughn, Ana-Iris Schiefer, J. R. Hilberink, Vinod Pullarkat, Ryan Shanley, Jessica Kohlschmidt, Janie Coulombe, Amandeep Salhotra, Lori Soma, Christina Cho, Michael A. Linden, Cem Akin, Jason Gotlib, Gregor Hoermann, Jason Hornick, Ryo Nakamura, Joachim Deeg, Clara D. Bloomfield, Daniel Weisdorf, Mark R. Litzow, Peter Valent, Gerwin Huls, Miguel-Angel Perales, Gautam Borthakur

^*Corresponding author for this work

Research output: Contribution to journal › Article › Academic › peer-review

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Abstract

Background: Although the prognosis of core-binding factor (CBF) acute myeloid leukemia (AML) is better than other subtypes of AML, 30% of patients still relapse and may require allogeneic hematopoietic cell transplantation (alloHCT). However, there is no validated widely accepted scoring system to predict patient subsets with higher risk of relapse.

Methods: Eleven centers in the US and Europe evaluated 247 patients with t(8;21) (q22;q22).

Results: Complete remission (CR) rate was high (92.7%), yet relapse occurred in 27.1% of patients. A total of 24.7% of patients received alloHCT. The median diseasefree (DFS) and overall (OS) survival were 20.8 and 31.2 months, respectively. Age, KIT D816V mutated (11.3%) or nontested (36.4%) compared with KIT D816V wild type (52.5%), high white blood cell counts (WBC), and pseudodiploidy compared with hyper- or hypodiploidy were included in a scoring system (named I-CBFit). DFS rate at 2 years was 76% for patients with a low-risk I-CBFit score compared with 36% for those with a high-risk I-CBFit score (P <0.0001). Low- vs high-risk OS at 2 years was 89% vs 51% (P <0.0001).

Conclusions: I-CBFit composed of readily available risk factors can be useful to tailor the therapy of patients, especially for whom alloHCT is not need in CR1 (ie, patients with a low-risk score).

Original language	English
Pages (from-to)	4447-4455
Number of pages	9
Journal	Cancer medicine
Volume	7
Issue number	9
DOIs	https://doi.org/10.1002/cam4.1733
Publication status	Published - Sept-2018

Keywords

acute myeloid leukemia
core-binding factor
disease-free survival
KIT mutation
predictive value
relapse
scoring system
C-KIT MUTATIONS
PROGNOSTIC IMPACT
GENE-MUTATIONS
GROUP-B
FLT3
AML
INV(16)
SURVIVAL
CANCER
ADULTS

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Ustun, C., Morgan, E., Moodie, E. E. M., Pullarkat, S., Yeung, C., Broesby-Olsen, S., Ohgami, R., Kim, Y., Sperr, W., Vestergaard, H., Chen, D., Kluin, P. M., Dolan, M., Mrozek, K., Czuchlewski, D., Horny, H.-P., George, T. I., Kristensen, T. K., Ku, N. K., ... Borthakur, G. (2018). Core-binding factor acute myeloid leukemia with t(8;21) Risk factors and a novel scoring system (I-CBFit). Cancer medicine, 7(9), 4447-4455. https://doi.org/10.1002/cam4.1733

@article{bce51693afb84d20bf80848478ae3a84,

title = "Core-binding factor acute myeloid leukemia with t(8;21) Risk factors and a novel scoring system (I-CBFit)",

abstract = "Background: Although the prognosis of core-binding factor (CBF) acute myeloid leukemia (AML) is better than other subtypes of AML, 30% of patients still relapse and may require allogeneic hematopoietic cell transplantation (alloHCT). However, there is no validated widely accepted scoring system to predict patient subsets with higher risk of relapse.Methods: Eleven centers in the US and Europe evaluated 247 patients with t(8;21) (q22;q22).Results: Complete remission (CR) rate was high (92.7%), yet relapse occurred in 27.1% of patients. A total of 24.7% of patients received alloHCT. The median diseasefree (DFS) and overall (OS) survival were 20.8 and 31.2 months, respectively. Age, KIT D816V mutated (11.3%) or nontested (36.4%) compared with KIT D816V wild type (52.5%), high white blood cell counts (WBC), and pseudodiploidy compared with hyper- or hypodiploidy were included in a scoring system (named I-CBFit). DFS rate at 2 years was 76% for patients with a low-risk I-CBFit score compared with 36% for those with a high-risk I-CBFit score (P <0.0001). Low- vs high-risk OS at 2 years was 89% vs 51% (P <0.0001).Conclusions: I-CBFit composed of readily available risk factors can be useful to tailor the therapy of patients, especially for whom alloHCT is not need in CR1 (ie, patients with a low-risk score).",

keywords = "acute myeloid leukemia, core-binding factor, disease-free survival, KIT mutation, predictive value, relapse, scoring system, C-KIT MUTATIONS, PROGNOSTIC IMPACT, GENE-MUTATIONS, GROUP-B, FLT3, AML, INV(16), SURVIVAL, CANCER, ADULTS",

author = "Celalettin Ustun and Elizabeth Morgan and Moodie, {Erica E. M.} and Sheeja Pullarkat and Cecilia Yeung and Sigurd Broesby-Olsen and Robert Ohgami and Young Kim and Wolfgang Sperr and Hanne Vestergaard and Dong Chen and Kluin, {Philip M.} and Michelle Dolan and Krzysztof Mrozek and David Czuchlewski and Hans-Peter Horny and George, {Tracy I.} and Kristensen, {Thomas Kielsgaard} and Ku, {Nam K.} and Yi, {Cecilia Arana} and Moller, {Michael Boe} and Guido Marcucci and Linda Baughn and Ana-Iris Schiefer and Hilberink, {J. R.} and Vinod Pullarkat and Ryan Shanley and Jessica Kohlschmidt and Janie Coulombe and Amandeep Salhotra and Lori Soma and Christina Cho and Linden, {Michael A.} and Cem Akin and Jason Gotlib and Gregor Hoermann and Jason Hornick and Ryo Nakamura and Joachim Deeg and Bloomfield, {Clara D.} and Daniel Weisdorf and Litzow, {Mark R.} and Peter Valent and Gerwin Huls and Miguel-Angel Perales and Gautam Borthakur",

year = "2018",

month = sep,

doi = "10.1002/cam4.1733",

language = "English",

volume = "7",

pages = "4447--4455",

journal = "Cancer medicine",

issn = "2045-7634",

publisher = "Wiley",

number = "9",

}

Ustun, C, Morgan, E, Moodie, EEM, Pullarkat, S, Yeung, C, Broesby-Olsen, S, Ohgami, R, Kim, Y, Sperr, W, Vestergaard, H, Chen, D, Kluin, PM, Dolan, M, Mrozek, K, Czuchlewski, D, Horny, H-P, George, TI, Kristensen, TK, Ku, NK, Yi, CA, Moller, MB, Marcucci, G, Baughn, L, Schiefer, A-I, Hilberink, JR, Pullarkat, V, Shanley, R, Kohlschmidt, J, Coulombe, J, Salhotra, A, Soma, L, Cho, C, Linden, MA, Akin, C, Gotlib, J, Hoermann, G, Hornick, J, Nakamura, R, Deeg, J, Bloomfield, CD, Weisdorf, D, Litzow, MR, Valent, P, Huls, G, Perales, M-A & Borthakur, G 2018, 'Core-binding factor acute myeloid leukemia with t(8;21) Risk factors and a novel scoring system (I-CBFit)', Cancer medicine, vol. 7, no. 9, pp. 4447-4455. https://doi.org/10.1002/cam4.1733

TY - JOUR

T1 - Core-binding factor acute myeloid leukemia with t(8;21) Risk factors and a novel scoring system (I-CBFit)

AU - Ustun, Celalettin

AU - Morgan, Elizabeth

AU - Moodie, Erica E. M.

AU - Pullarkat, Sheeja

AU - Yeung, Cecilia

AU - Broesby-Olsen, Sigurd

AU - Ohgami, Robert

AU - Kim, Young

AU - Sperr, Wolfgang

AU - Vestergaard, Hanne

AU - Chen, Dong

AU - Kluin, Philip M.

AU - Dolan, Michelle

AU - Mrozek, Krzysztof

AU - Czuchlewski, David

AU - Horny, Hans-Peter

AU - George, Tracy I.

AU - Kristensen, Thomas Kielsgaard

AU - Ku, Nam K.

AU - Yi, Cecilia Arana

AU - Moller, Michael Boe

AU - Marcucci, Guido

AU - Baughn, Linda

AU - Schiefer, Ana-Iris

AU - Hilberink, J. R.

AU - Pullarkat, Vinod

AU - Shanley, Ryan

AU - Kohlschmidt, Jessica

AU - Coulombe, Janie

AU - Salhotra, Amandeep

AU - Soma, Lori

AU - Cho, Christina

AU - Linden, Michael A.

AU - Akin, Cem

AU - Gotlib, Jason

AU - Hoermann, Gregor

AU - Hornick, Jason

AU - Nakamura, Ryo

AU - Deeg, Joachim

AU - Bloomfield, Clara D.

AU - Weisdorf, Daniel

AU - Litzow, Mark R.

AU - Valent, Peter

AU - Huls, Gerwin

AU - Perales, Miguel-Angel

AU - Borthakur, Gautam

PY - 2018/9

Y1 - 2018/9

N2 - Background: Although the prognosis of core-binding factor (CBF) acute myeloid leukemia (AML) is better than other subtypes of AML, 30% of patients still relapse and may require allogeneic hematopoietic cell transplantation (alloHCT). However, there is no validated widely accepted scoring system to predict patient subsets with higher risk of relapse.Methods: Eleven centers in the US and Europe evaluated 247 patients with t(8;21) (q22;q22).Results: Complete remission (CR) rate was high (92.7%), yet relapse occurred in 27.1% of patients. A total of 24.7% of patients received alloHCT. The median diseasefree (DFS) and overall (OS) survival were 20.8 and 31.2 months, respectively. Age, KIT D816V mutated (11.3%) or nontested (36.4%) compared with KIT D816V wild type (52.5%), high white blood cell counts (WBC), and pseudodiploidy compared with hyper- or hypodiploidy were included in a scoring system (named I-CBFit). DFS rate at 2 years was 76% for patients with a low-risk I-CBFit score compared with 36% for those with a high-risk I-CBFit score (P <0.0001). Low- vs high-risk OS at 2 years was 89% vs 51% (P <0.0001).Conclusions: I-CBFit composed of readily available risk factors can be useful to tailor the therapy of patients, especially for whom alloHCT is not need in CR1 (ie, patients with a low-risk score).

AB - Background: Although the prognosis of core-binding factor (CBF) acute myeloid leukemia (AML) is better than other subtypes of AML, 30% of patients still relapse and may require allogeneic hematopoietic cell transplantation (alloHCT). However, there is no validated widely accepted scoring system to predict patient subsets with higher risk of relapse.Methods: Eleven centers in the US and Europe evaluated 247 patients with t(8;21) (q22;q22).Results: Complete remission (CR) rate was high (92.7%), yet relapse occurred in 27.1% of patients. A total of 24.7% of patients received alloHCT. The median diseasefree (DFS) and overall (OS) survival were 20.8 and 31.2 months, respectively. Age, KIT D816V mutated (11.3%) or nontested (36.4%) compared with KIT D816V wild type (52.5%), high white blood cell counts (WBC), and pseudodiploidy compared with hyper- or hypodiploidy were included in a scoring system (named I-CBFit). DFS rate at 2 years was 76% for patients with a low-risk I-CBFit score compared with 36% for those with a high-risk I-CBFit score (P <0.0001). Low- vs high-risk OS at 2 years was 89% vs 51% (P <0.0001).Conclusions: I-CBFit composed of readily available risk factors can be useful to tailor the therapy of patients, especially for whom alloHCT is not need in CR1 (ie, patients with a low-risk score).

KW - acute myeloid leukemia

KW - core-binding factor

KW - disease-free survival

KW - KIT mutation

KW - predictive value

KW - relapse

KW - scoring system

KW - C-KIT MUTATIONS

KW - PROGNOSTIC IMPACT

KW - GENE-MUTATIONS

KW - GROUP-B

KW - FLT3

KW - AML

KW - INV(16)

KW - SURVIVAL

KW - CANCER

KW - ADULTS

U2 - 10.1002/cam4.1733

DO - 10.1002/cam4.1733

M3 - Article

SN - 2045-7634

VL - 7

SP - 4447

EP - 4455

JO - Cancer medicine

JF - Cancer medicine

IS - 9

ER -

Core-binding factor acute myeloid leukemia with t(8;21) Risk factors and a novel scoring system (I-CBFit)

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Keywords

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