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

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, Mrózek K, Czuchlewski D, Horny HP, George TI, Kristensen TK, Ku NK, Yi CA, Møller MB, Marcucci G, Baughn L, Schiefer AI, 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 MA, Borthakur G
Cancer Med 7 4447-4455 09/01/2018


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 disease-free (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 I-CBFit score).

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