NPM1‐mutated myeloid neoplasms are a unique entity not defined by bone marrow blast percentage

净现值1 医学 骨髓 化疗 髓样 内科学 胃肠病学 髓系白血病 白血病 肿瘤科 核型 生物 基因 生物化学 染色体
作者
Georgina Gener‐Ricos,Àlex Bataller,Juan José Rodríguez‐Sevilla,Kelly S. Chien,Andres Quesada,Emmanuel Almanza‐Huante,Danielle Hammond,Koji Sasaki,Courtney D. DiNardo,Tapan M. Kadia,Naval Daver,Gautam Borthakur,Ghayas C. Issa,Nicholas J. Short,Rashmi Kanagal‐Shamanna,Hagop M. Kantarjian,Guillermo Garcia‐Manero,Guillermo Montalban‐Bravo
出处
期刊:Cancer [Wiley]
标识
DOI:10.1002/cncr.35433
摘要

Abstract Introduction NPM1 ‐mutated ( NPM1 mut ) myeloid neoplasms (MNs) with <20% bone marrow (BM) blasts ( NPM1 mut MNs<20) are uncommon, and their classification remains inconsistent. Methods The clinicopathologic features of 54 patients with NPM1 mut MNs <20 were evaluated and compared with wild‐type NPM1 MNs <20 and NPM1 mut MNs≥20, respectively. Results NPM1 mut MNs had similar features regardless of blast percentage, except for higher IDH2 (29% vs 7%, p = .023) and FLT3 (70% vs 11%, p < .001) frequency in patients with ≥20% BM blasts. Thirty‐three (61%) patients with NPM1 mut MNs <20 received low‐intensity chemotherapy (LIC) and 12 (22%) received intensive chemotherapy (IC). Higher complete remission rates (75% vs 27%, p = .006) and median overall survival (mOS) (not reached vs 30.4 months, p = .06) were observed with IC compared to LIC. Young patients (age <60 years) did not reach mOS either when treated with LIC or IC. Stem cell transplant was associated with increased survival only in patients treated with LIC (HR, 0.24; p = .025). No differences in mOS were observed by BM blast strata (32.2 months, not reached and 46.9 months for <10%, 10%–19%, and ≥20% blasts, p = .700) regardless of treatment modality (LIC: p = .900; IC: p = .360). Twenty‐three patients (43%) with NPM1 mut MNs <20 had marrow blast progression to ≥20%. Conclusions Overall, NPM1 mut MNs define a unique entity independent of BM blast percentage.
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