Azacitidine as a bridge to transplantation in juvenile myelomonocytic leukemia
Azacitidine as a bridge to transplantation in juvenile myelomonocytic leukemia
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阿扎胞苷作为幼年粒单核细胞白血病移植的桥梁
DOI：
10.1111/ped.14929
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发表时间：
2022
影响因子：
1.4
通讯作者：
Kawasaki Yukihiko
中科院分区：
文献类型：
--
作者：
Takebayashi Akira;Yamamoto Masaki;Igarashi Keita;Muramatsu Hideki;Kawasaki Yukihiko
异基因造血干细胞移植(HSCT)是大多数患者的唯一治疗方法，但尚未建立合适的HSCT前桥接疗法。自从Furlan等人1报道了氮胞替丁在HSCT前JMML中的有效性以来，已经发表了几篇报告。分子分析证实外周血单核细胞中存在KRAS p.G13D突变。表1造血干细胞移植前诊断的临床病程第1周期第2周期第3周期第4周期第5周期第6周期第7周期造血干细胞移植前第8周期(/μL)18 500 5,700 4,500 5,200 4,400 4,200 3,300 2,700 3,400 3,300单核细胞(/μL)8,510 2,907 675 1,664 968 882 180 231 297 68 198%母细胞(PB)2 1 4 2 2 3 1 0 0 0%单核细胞(BM)8.2-3-1.4%单核细胞7(PB；FISH)77-23 46 35 28 47 13 14-9%单核细胞鱼)93-92-28血小板(×104/μL)1.7 1.8 1.6 3.5 8.3 4.7 5.1 6.7 14.9 12.1 18.1肝*1(Cm)4 10 8 7 6 6 6 3 00 0脾*2(Cm)0.5 10 8 7 6 6 6 30 1 0血小板输注，每日1次，2天1次，每3天1次，右肋缘下1号*2左肋缘下1个缩写：aza，azacitidine；骨髓；FISH，荧光原位杂交；造血干细胞移植；外周血；WBC，白细胞。
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative therapy for most patients, but no appropriate pre-HSCT bridging therapy has been established. Since Furlan et al 1 reported the effectiveness of azacitidine in pre-HSCT JMML, several reports have been published. Molecular analysis identified a KRAS p. G13D mutation in peripheral blood mononuclear cells. Table 1 Clinical course At diagnosis Before AZA 1st cycle 2nd cycle 3rd cycle 4th cycle 5th cycle 6th cycle 7th cycle 8th cycle Before HSCT WBCs (/μL) 18 500 5,700 4,500 5,200 4,400 4,200 3,000 3,300 2,700 3,400 3,300 Monocytes (/μL) 8,510 2,907 675 1,664 968 882 180 231 297 68 198% Blasts (PB) 2 1 4 2 2 3 1 0 0 0 0% Blasts (BM) 8.2–3–––––––1.4% Monosomy7 (PB; FISH) 77–23 46 35 28 47 13 14–9% Monosomy7 (BM; FISH) 93–92–––––––28 Platelet (× 104/μL) 1.7 1.8 1.6 3.5 8.3 4.7 5.1 6.7 14.9 12.1 18.1 Liver* 1 (cm) 4 10 8 7 6 6 6 3 0 0 0 Spleen* 2 (cm) 0.5 10 8 7 6 6 6 3 1 0 0 Platelet infusion Once a day Once a day Every 2 days Every 2 days Every 3 days Every 3 days Every 3 days No No No No* 1below right costal margin* 2below left costal margin Abbreviations: AZA, azacitidine; BM, bone marrow; FISH, fluorescent in situ hybridization; HSCT, hematopoietic stem cell transplantation; PB, peripheral blood; WBC, white blood cell.