NPM1 突变的髓系肿瘤是一种独特的实体，并非由骨髓原始细胞百分比来定义。

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

作者信息

Gener-Ricos Georgina, Bataller Alex, Rodriguez-Sevilla Juan Jose, Chien Kelly S, Quesada Andres E, Almanza-Huante Emmanuel, Hammond Danielle, Sasaki Koji, DiNardo Courtney, Kadia Tapan, Daver Naval, Borthakur Gautam, Issa Ghayas C, Short Nicholas J, Kanagal-Shamanna Rashmi, Kantarjian Hagop M, Garcia-Manero Guillermo, Montalban-Bravo Guillermo

机构信息

Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.

Department of Hematopathology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.

出版信息

Cancer. 2024 Oct 15;130(20):3452-3462. doi: 10.1002/cncr.35433. Epub 2024 Jun 19.

DOI:10.1002/cncr.35433

PMID:38896064

Abstract

INTRODUCTION

NPM1-mutated (NPM1) myeloid neoplasms (MNs) with <20% bone marrow (BM) blasts (NPM1 MNs<20) are uncommon, and their classification remains inconsistent.

METHODS

The clinicopathologic features of 54 patients with NPM1 MNs <20 were evaluated and compared with wild-type NPM1 MNs <20 and NPM1 MNs≥20, respectively.

RESULTS

NPM1 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 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 MNs <20 had marrow blast progression to ≥20%.

CONCLUSIONS

Overall, NPM1 MNs define a unique entity independent of BM blast percentage.

摘要

引言

骨髓母细胞<20%的NPM1突变（NPM1）髓系肿瘤（MNs）（NPM1 MNs<20）并不常见，其分类仍不一致。

方法

评估54例NPM1 MNs<20患者的临床病理特征，并分别与野生型NPM1 MNs<20和NPM1 MNs≥20患者进行比较。

结果

无论母细胞百分比如何，NPM1 MNs具有相似的特征，但骨髓母细胞≥20%的患者中IDH2（29%对7%，p = 0.023）和FLT3（70%对11%，p < 0.001）频率较高。33例（61%）NPM1 MNs<20患者接受了低强度化疗（LIC），12例（22%）接受了强化化疗（IC）。与LIC相比，IC观察到更高的完全缓解率（75%对27%，p = 0.006）和中位总生存期（mOS）（未达到对30.4个月，p = 0.06）。年轻患者（年龄<60岁）接受LIC或IC治疗时均未达到mOS。干细胞移植仅在接受LIC治疗的患者中与生存期增加相关（HR，0.24；p = 0.025）。无论治疗方式如何（LIC：p = 0.900；IC：p = 0.360），按骨髓母细胞分层未观察到mOS有差异（<10%、10%-19%和≥20%母细胞分别为32.2个月、未达到和46.9个月，p = 0.700）。23例（43%）NPM1 MNs<20患者的骨髓母细胞进展至≥20%。