AMERICAN JOURNAL OF HEMATOLOGY 年发文量-刊鹿论文编译

Smoldering mastocytosis: Survival comparisons with indolent and aggressive mastocytosis

Abstract:

To the Editor: The 2016 World Health Organization (WHO) system classifies systemic mastocytosis (SM) into five categories: indolent (ISM), smoldering (SSM), SM with an associated hematological neoplasm (SM-AHN), aggressive (ASM), and mast cell leukemia (MCL). WHO morphologic distinctions within SM were based on presence or absence of B (high mast cell burden, evidence of nonmast cell morphologic dysplasia or myeloproliferation, organomegaly without functional consequence) and C (cytopenia related to mast cell infiltration, palpable organomegaly with functional consequence, skeletal involvement with large osteolytic lesions, and malabsorption with weight loss secondary to gastrointestinal mast cell infiltrates) findings, presence, or absence of an associated hematological neoplasm, and presence or absence of criteria for MCL (≥20% mast cells on bone marrow aspirate smears or ≥ 10% circulating mast cells). In general, C findings are absent in both ISM and SSM and present in ASM; SSM is distinguished from ISM by the presence of ≥2 B findings including high mast cell burden, organomegaly, and evidence for multilineage involvement. Given that SSM is clearly distinguished from ISM, as outlined above, we sought to examine the impact of this distinction on survival. The current study was approved by the institutional review board. Study patients were recruited from the Mayo Clinic, Rochester, MN database for SM. Diagnoses of SM and its morphological subcategories were confirmed by both clinical and bone marrow examinations, in line with the 2016 WHO criteria. Particular attention was paid to potential C findings, in order to avoid inadvertent labeling of ASM as SSM; patients with subthreshold complete blood counts were scrutinized to clarify the contribution of bone marrow mast cell infiltrates. Next-generation sequencing was performed in a subset of the study population. Statistical analyses considered clinical and laboratory data collected at the time of initial diagnosis at the Mayo Clinic. Conventional statistics was used for calculation of survival and comparison of disease features. The JMP Pro 13.0.0 software from SAS Institute, Cary, NC, was used for all analysis. A total of 376 consecutive patients with SSM (n = 17), ISM (n = 274) or ASM (n = 85) constituted the study population. Respective values for SSM vs ISM vs ASM were age 64, 47, and 61 years; male sex percentage 47%, 43%, and 45%; median hemoglobin level 12.6, 14, and 12.3 g/dL; median platelet count 217, 260, and 219 × 10/L; presence of sex-adjusted anemia in 47%, 12%, and 52%; platelet count <150 × 10/L in 13%, 4%, and 27%; serum alkaline phosphatase (ALP) above the upper limit of normal in 82%, 35%, and 71%; and serum albumin <3.5 g/dL in 6%, 12%, and 25%. Among 69 informative cases, adverse mutations (ie, ASXL1, RUNX1, and NRAS) were not seen in 4 SSM or 39 ISM cases but were present in 4 (15%) of 26 patients with ASM. Among 191 patients with available cytogenetic studies, abnormal karyotype was seen in 12 (6%) total patients, including none of 3 cases with SSM, 8 (6%) of 139 cases with ISM, and 4 (8%) of 49 patients with ASM. Compared to ISM, SSM was associated with older age (P < .001) and, as expected from disease definition, with higher bone marrow mast cell burden (P < .001), higher serum tryptase level (<.001), higher prevalence of palpable hepatomegaly (P < .001), and palpable splenomegaly (P < .001; Supporting Information Table S1). SSM, as opposed to ISM, was also associated with adverse disease features including anemia (P = .001), thrombocytopenia (P = .03), and higher serum ALP level (P < .001); in contrast, there was no significant difference in the expression of mast cell mediator symptoms, urticaria pigmentosa, abnormal karyotype or KIT mutational frequency; the number of informative cases was too small to comment of the prevalence of non-KIT mutations, although the higher incidence of TET2 mutations in SSM would be consistent with the observed difference in age distribution (Supporting Information Table S1). In a separate analysis comparing SSM (n = 17) with ASM (n = 85), age distribution was similar (P = .4), as were most other disease features, including prevalence of KIT and non-KITmutations. After median follow-up of 32 months for ISM, 39 months for SSM, and 26 months for ASM, a total of 86 (23%) deaths and only one (0.3%) leukemic transformation was recorded; the single case of leukemic transformation occurred in a patient with ASM. In univariate analysis, survival appeared significantly shorter in SSM vs ISM (HR 5.5, 95% CI 2.8-10.2) whereas it was similar between SSM and ASM (HR 0.9, 95% CI 0.5-1.8; Figure 1). However, the significant difference in survival between ISM and SSM was not sustained during age-adjusted multivariable analysis that also included other previously established risk factors in SM, which instead confirmed the independent adverse survival effect of advanced age (P < .001) and hemoglobin level below the lower limit of the sex-adjusted hemoglobin level (P < .001). In contrast, the difference in survival between ASM and ISM remained significant during a similar multivariable analysis; HR (95% CI) were 2.0 (1.1-3.5) for ASM vs ISM, 2.2 (1.3-3.8) for sexadjusted anemia), 2.2 (1.2-3.9) for thrombocytopenia <150 × 10/L, and 2.3 (1.2-4.9) for ALP above upper limit of normal; P value for age as a continuous variable was <.001. The current study highlights the older age distribution of patients with SSM (median 64 years), which was similar to that of patients with ASM (median 61 years) but significantly different than that of patients Received: 23 September 2018 Accepted: 26 September 2018

Author Listing： Ayalew Tefferi;Sahrish Shah;Kaaren K Reichard;Curtis A Hanson;Animesh Pardanani

Volume： 94

Pages： None

DOI： 10.1002/ajh.25302

Language： English

Journal： American Journal of Hematology

AMERICAN JOURNAL OF HEMATOLOGY

AM J HEMATOL

影响因子：10.1

是否综述期刊：是

是否OA：否

是否预警：不在预警名单内

发行时间：1976

ISSN：0361-8609

发刊频率：Monthly

收录数据库：SCIE/Scopus收录

出版国家/地区：UNITED STATES

出版社：Wiley-Liss Inc.

期刊介绍

The American Journal of Hematology provides broad coverage of experimental and clinical features of blood diseases in humans and in animal models of human disease. The journal publishes original contributions in non-malignant and malignant hematological diseases including clinical and basic studies in hemostasis and thrombosis, immunology, blood banking, and stem cell biology. Clinical translational reports describing novel therapeutic approaches to diagnosis and treatment of hematological diseases are welcomed and encouraged. The journal includes regular original laboratory and clinical research articles, brief research reports, critical reviews, images in hematology, letters and correspondence.

《美国血液学杂志》广泛报道了人类和人类疾病动物模型中血液疾病的实验和临床特征。该杂志发表非恶性和恶性血液病的原创论文，包括止血和血栓形成、免疫学、血库和干细胞生物学的临床和基础研究。欢迎并鼓励临床转化报告描述诊断和治疗血液疾病的新治疗方法。该杂志包括定期原始实验室和临床研究文章，简短的研究报告，评论，血液学图像，信件和通信。

年发文量	175
国人发稿量	20
国人发文占比	11.43%
自引率	5.9%
平均录取率	较易
平均审稿周期	一般,3-6周
版面费	US$4800
偏重研究方向	医学-血液学
期刊官网	http://onlinelibrary.wiley.com/journal/10.1002/(ISSN)1096-8652
投稿链接	http://mc.manuscriptcentral.com/ajh

质量指标占比

研究类文章占比	OA被引用占比	撤稿占比	出版后修正文章占比
89.66%	29.12%	0.00%	0.00%

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时间	预警情况
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2020年12月发布的2020版	不在预警名单中

JCR分区 WOS分区等级：Q1区

版本	按学科	分区
WOS期刊SCI分区 WOS期刊SCI分区是指SCI官方（Web of Science）为每个学科内的期刊按照IF数值排序，将期刊按照四等分的方法划分的Q1-Q4等级，Q1代表质量最高，即常说的1区期刊。（2021-2022年最新版）
	HEMATOLOGY	Q1

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版本	大类学科	小类学科	Top期刊	综述期刊
	医学 1区	HEMATOLOGY 血液学 2区	否	是
2021年12月基础版	医学 1区	HEMATOLOGY 血液学 2区	否	是
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