Juvenile myelomonocytic leukemia-A comprehensive review and recent advances in management

幼年粒单核细胞白血病 医学 PTPN11型 单核细胞增多 骨髓增生性肿瘤 癌症研究 免疫学 髓样 内科学 白血病 造血干细胞移植 肿瘤科 骨髓 移植 克拉斯 骨髓纤维化 造血 干细胞 癌症 生物 遗传学 结直肠癌
作者
Aditya Gupta,Jagdish Prasad Meena,Anita Chopra,Pranay Tanwar,Rachna Seth
出处
期刊:American journal of blood research 卷期号:11 (1): 1-21 被引量:24
链接
标识
摘要

Juvenile myelomonocytic leukemia (JMML) is a rare pediatric myelodysplastic/myeloproliferative neoplasm overlap disease. JMML is associated with mutations in the RAS pathway genes resulting in the myeloid progenitors being sensitive to granulocyte monocyte colony-stimulating factor (GM-CSF). Karyotype abnormalities and additional epigenetic alterations can also be found in JMML. Neurofibromatosis and Noonan's syndrome have a predisposition for JMML. In a few patients, the RAS genes (NRAS, KRAS, and PTPN11) are mutated at the germline and this usually results in a transient myeloproliferative disorder with a good prognosis. JMML with somatic RAS mutation behaves aggressively. JMML presents with cytopenias and leukemic infiltration into organs. The laboratory findings include hyperleukocytosis, monocytosis, increased hemoglobin-F levels, and circulating myeloid precursors. The blast cells in the peripheral blood/bone-marrow aspirate are less than 20% and the absence of the BCR-ABL translocation helps to differentiate from chronic myeloid leukemia. JMML should be differentiated from immunodeficiencies, viral infections, intrauterine infections, hemophagolymphohistiocytosis, other myeloproliferative disorders, and leukemias. Chemotherapy is employed as a bridge to HSCT, except in few with less aggressive disease, in which chemotherapy alone can result in long term remission. Azacitidine has shown promise as a single agent to stabilize the disease. The prognosis of JMML is poor with about 50% of patients surviving after an allogeneic hematopoietic stem cell transplant (HSCT). Allogeneic HSCT is the only known cure for JMML to date. Myeloablative conditioning is most commonly used with graft versus host disease (GVHD) prophylaxis tailored to the aggressiveness of the disease. Relapses are common even after HSCT and a second HSCT can salvage a third of these patients. Novel options in the treatment of JMML e.g., hypomethylating agents, MEK inhibitors, JAK inhibitors, tyrosine kinase inhibitors, etc. are being explored.
最长约 10秒,即可获得该文献文件

科研通智能强力驱动
Strongly Powered by AbleSci AI
更新
大幅提高文件上传限制,最高150M (2024-4-1)

科研通是完全免费的文献互助平台,具备全网最快的应助速度,最高的求助完成率。 对每一个文献求助,科研通都将尽心尽力,给求助人一个满意的交代。
实时播报
坚强亦丝应助hooke采纳,获得10
2秒前
可靠的书桃应助刘斌采纳,获得10
3秒前
liu完成签到 ,获得积分10
3秒前
田様应助健忘溪流采纳,获得10
4秒前
4秒前
8秒前
8秒前
10秒前
本之上课发布了新的文献求助10
11秒前
11秒前
我是老大应助雷声有点响采纳,获得20
13秒前
14秒前
robi发布了新的文献求助10
16秒前
zhaoyingxin完成签到,获得积分10
18秒前
19秒前
19秒前
无花果应助不一样是怎样采纳,获得10
19秒前
20秒前
顾矜应助邵竺采纳,获得10
22秒前
22秒前
amnsd000完成签到 ,获得积分10
22秒前
彪yu完成签到 ,获得积分10
22秒前
23秒前
23秒前
马大翔应助不二采纳,获得20
26秒前
derek发布了新的文献求助10
26秒前
ycp完成签到,获得积分10
28秒前
言不得语发布了新的文献求助10
29秒前
我是老大应助robi采纳,获得30
29秒前
LHD发布了新的文献求助10
30秒前
derek完成签到,获得积分10
34秒前
zx完成签到,获得积分10
38秒前
40秒前
伊可完成签到 ,获得积分10
43秒前
不配.应助桀桀采纳,获得10
43秒前
大模型应助irisjlj采纳,获得10
43秒前
0x1orz完成签到,获得积分10
44秒前
小媛完成签到 ,获得积分20
46秒前
doctorbin完成签到 ,获得积分10
47秒前
47秒前
高分求助中
Sustainability in Tides Chemistry 2800
The Young builders of New china : the visit of the delegation of the WFDY to the Chinese People's Republic 1000
Rechtsphilosophie 1000
Bayesian Models of Cognition:Reverse Engineering the Mind 888
Le dégorgement réflexe des Acridiens 800
Defense against predation 800
Very-high-order BVD Schemes Using β-variable THINC Method 568
热门求助领域 (近24小时)
化学 医学 生物 材料科学 工程类 有机化学 生物化学 物理 内科学 纳米技术 计算机科学 化学工程 复合材料 基因 遗传学 催化作用 物理化学 免疫学 量子力学 细胞生物学
热门帖子
关注 科研通微信公众号,转发送积分 3134935
求助须知:如何正确求助?哪些是违规求助? 2785802
关于积分的说明 7774295
捐赠科研通 2441699
什么是DOI,文献DOI怎么找? 1298093
科研通“疑难数据库(出版商)”最低求助积分说明 625075
版权声明 600825