异基因造血干细胞移植联合化疗治疗儿童髓系肉瘤7例临床分析

doi:10.12372/jcp.2025.25e1007

Abstract

Abstract:

Objective To explore the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) combined with chemotherapy in children with myeloid sarcoma (MS). Methods A retrospective analysis was conducted on the clinical diagnosis, treatment and follow-up data of 7 children with MS who were diagnosed and treated at the hematology and oncology center from January 1, 2020 to December 31, 2023. Results There were 3 male and 4 female patients included in this study. The median age of diagnosis was 62 months (10 months - 14 years). Of the 7 patients, only 1 was initially presented with a "local mass," while the remaining 6 were diagnosed with acute myeloid leukemia (AML) and subsequently discovered to have extramedullary involvement, including 4 in the head and face, 2 in the mediastinum, and 1 in the ilium. All 7 patients received CCLG-AML-2019 protocol-based chemotherapy and bridging allo-HSCT. All children received a myeloablative pretreatment regimen prior to transplantation. Five of the 7 children were treated with post-infusion cyclophosphamide (PTCY), and 2 with busulfan combined with cyclophosphamide (BUCY). The median number of mononuclear cells infused was 19.87 (7.4-24.28)×10⁸/kg, and the median number of CD34⁺ cells infused was 12.87 (7.1-14.7)×10⁶/kg. The median time of neutrophil engraftment was +15(13-15) days, and the median time of platelet engraftment was +12(11-17) days. As of June 30, 2025, The median follow-up time was 1008 (371-1814) d. Five patients were alive, and 2 died. There was no death related to allo-HSCT. The estimated 3-year OS rate was 71%. Conclusion There are still challenges in the early diagnosis of MS in children, and the standard chemotherapy bridging allo-HSCT in the treatment of MS in children can obtain better efficacy.

Key words: myeloid sarcoma, acute myeloid leukemia, allogeneic hematopoietic stem cell transplantation, child

CLC Number:

WU Jinjun, XIONG Hao, ZENG Hui, CHEN Zhi, YANG Li, SUN Ming, WANG Zhuo, DU Yu, QI Shanshan, WANG Wei, ZHANG Lannan. Clinical analysis of allogeneic hematopoietic stem cell transplantation on pediatric myeloid sarcoma[J].Journal of Clinical Pediatrics, 2026, 44(1): 44-50.

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References 20

[1]	Khoury JD, Solary E, Abla O, et al. The 5th edition of the world health organization classification of haematolymphoid tumours: myeloid and histiocytic/dendritic neoplasms[J]. Leukemia, 2022, 36(7): 1703-1719. doi: 10.1038/s41375-022-01613-1
[2]	Chung YJ, Bertoli R, Cao D, et al. Disruption of normal stem cell function and transmission of myelodysplastic syndrome by self-renewal of committed myeloid lineage cells[J]. Stem Cell Reports, 2025: 102571.
[3]	Ramia DCM, Chen W. Myeloid sarcoma: an overview[J]. Semin Diagn Pathol, 2023, 40(3): 129-139. doi: 10.1053/j.semdp.2023.04.009 pmid: 37149396
[4]	Goyal G, Bartley AC, Patnaik MM, et al. Clinical features and outcomes of extramedullary myeloid sarcoma in the United States: analysis using a national data set[J]. Blood Cancer J, 2017, 7(8): e592.
[5]	Bianchi S, Capria S, Trisolini SM, et al. Myeloid sarcoma: diagnostic and treatment tools from a monocentric retrospective experience[J]. Acta Haematol, 2022, 145(1): 84-88. doi: 10.1159/000517389
[6]	Shallis RM, Gale RP, Lazarus HM, et al. Myeloid sarcoma, chloroma, or extramedullary acute myeloid leukemia tumor: a tale of misnomers, controversy and the unresolved[J]. Blood Rev, 2021, 47: 100773. doi: 10.1016/j.blre.2020.100773
[7]	中华医学会血液学分会干细胞应用学组, 中华医学会儿科学分会. 异基因造血干细胞移植治疗儿童急性淋巴细胞白血病中国专家共识(2022年版)[J]. 中华血液学杂志, 2022, 43(10): 793-801.
	Chinese expert consensus of the allogeneic hematopoietic stem cell transplantation for pediatric acute myeloid leukemia (not APL) (2022)[J]. Zhonghua Xue Ye Xue Za Zhi, 2022, 43(10): 802-809.
[8]	Wang Y, Chang Y, Chen J, et al. Consensus on the monitoring, treatment, and prevention of leukaemia relapse after allogeneic haematopoietic stem cell transplantation in China: 2024 update[J]. Cancer Lett, 2024, 605: 217264. doi: 10.1016/j.canlet.2024.217264
[9]	Gao L, Zhang Y, Wang S, et al. Effect of rhG-CSF combined with decitabine prophylaxis on relapse of patients with high-risk MRD-negative AML after HSCT: an open-label, multicenter, randomized controlled trial[J]. J Clin Oncol, 2020, 38(36): 4249-4259. doi: 10.1200/JCO.19.03277 pmid: 33108244
[10]	张宏, 于彤, 王岩, 等. 儿童髓系肉瘤的CT和MRI影像学表现分析[J]. 中国小儿血液与肿瘤杂志, 2022, 27(6): 383-389.
	Zhang H, Yu T, Wang Y, et al. Analysis of CT and MRI imaging manifestations of pediatric myeloid sarcoma[J]. Zhongguo Xiaoer Xueye Yu Zhongliu Zazhi, 2022, 27(6): 383-389.
[11]	Duminuco A, Maugeri C, Parisi M, et al. Target therapy for extramedullary relapse of FLT3-ITD acute myeloid leukemia: emerging data from the field[J]. Cancers (Basel), 2022, 14(9): 2186. doi: 10.3390/cancers14092186
[12]	Magdy M, Abdel KN, Eldessouki I, et al. Myeloid sarcoma[J]. Oncol Res Treat, 2019, 42(4): 224-229. doi: 10.1159/000497210 pmid: 30840960
[13]	Zhao H, Dong Z, Wan D, et al. Clinical characteristics, treatment, and prognosis of 118 cases of myeloid sarcoma[J]. Sci Rep, 2022, 12(1): 6752. doi: 10.1038/s41598-022-10831-7 pmid: 35474239
[14]	Zorn KE, Cunningham AM, Meyer AE, et al. Pediatric myeloid sarcoma, more than just a chloroma: a review of clinical presentations, significance, and biology[J]. Cancers (Basel), 2023, 15(5): 1443. doi: 10.3390/cancers15051443
[15]	Al Semari MA, Perrotta M, Russo C, et al. Orbital myeloid sarcoma (chloroma): Report of 2 cases and literature review[J]. Am J Ophthalmol Case Rep, 2020, 19: 100806.
[16]	潘虹宇, 吴南海, 吴敏媛, 等. 儿童髓系肉瘤临床特征及BCH-AML05方案治疗效果观察[J]. 人民军医, 2019, 62(6): 558-562.
	Pan HY, Wu NH, Wu MY, et al. Clinical characteristics of pediatric myeloid sarcoma and observation on the therapeutic effect of BCH-AML05 regimen[J]. Renmin Junyi, 2019, 62(6): 558-562.
[17]	Lazzarotto D, Candoni A, Fili C, et al. Clinical outcome of myeloid sarcoma in adult patients and effect of allogeneic stem cell transplantation. Results from a multicenter survey[J]. Leuk Res, 2017, 53: 74-81. doi: 10.1016/j.leukres.2016.12.003
[18]	Ye F, Zhang H, Zhang W, et al. Clinical characteristics, pathology features and outcomes of pediatric myeloid sarcoma: a retrospective case series[J]. Front Pediatr, 2022, 10: 927894. doi: 10.3389/fped.2022.927894
[19]	江亚军, 张春芳, 王红霞, 等. 原发性髓系肉瘤遗传学和分子学改变的临床意义[J]. 中国实验血液学杂志, 2024, 32(1): 27-32.
	Jiang YJ, Zhang CF, Wang HX, et al. Clinical significance of genetic and molecular alterations in primary myeloid sarcoma[J]. Zhongguo Shiyan Xueyexue Zazhi, 2024, 32(1): 27-32.
[20]	Chiu AM, Yoon JG, Tirumani SH, et al. Myeloid sarcoma: a primer for radiologists[J]. J Comput Assist Tomogr, 2023, 47(3): 475-484. doi: 10.1097/RCT.0000000000001440 pmid: 36877785

患儿	形态学		免疫学			细胞遗传学	分子生物学
	异常细胞比例	异常细胞种类	异常细胞比例		免疫表型		基因变异与融合基因
	异常细胞比例	异常细胞种类	初诊	移植+90 d	免疫表型		基因变异与融合基因
例1	8%	原始细胞	3.6%	<0.1%	CD33、CD34、CD38、CD117、MPO、CD9	46,XY[20]	WT1(15.3%)； PHF6(25.3%)； SUZ12(15.0%)
例2	83%	原始细胞	71.5%	<0.1%	HLA-DR、CD4、CD11c、CD15、CD45、CD64、TDT(din)、CD11b、CD33、CD38(dim)、CD56(dim)、CD99、CD123(dim)	44~46,XX，-X，add(2) (q11.2),add(3)(q21), -10,add(11)(q23), +mar,inc[cp20]	NRAS(7.6%)； FLT3(17.5%)； ASXL1(9.5%)； BCOR(10.7%)
例3	73.5%	早幼粒细胞	96.5%	<0.1%	HLA-DR、CD2、CD3、CD4、CD5、CD6、、CD7、CD8、CD10、CD11b、CD13、CD14、CD15、CD16、CD19、CD20、CD22、CD33、CD34、CD38、CD56、CD58、CD64、CD71、CD117、CD123、CD45	46, XY, t(5;13;17)(q35; q14;q21)[20]	NRAS(43.8%); NPM1::RaRa
例4	81%	原始细胞	85.1%	<0.1%	CD33、CD34、CD41a、CD42b、CD43、CD56、CD61、CD117	46,XX[16]	PIK3CA(18.3%)
例5	12%	原始细胞	17.5%	<0.1%	CDC13、CD33、CD34、CD117、HLA-DR，CD4(dim)、CD64、MPO(dim)	46,XY[15]	NRAS(36.2%)； CSF3R(29.9%)； KIT(4.1%)
例6	66%	原始细胞	61.4%	<0.1%	CD13、CD15、CD33、CD34、CD117;CD4(dim)、CD9、CD11c(dim)、CD64(dim)、HLA-DR、MPO	46,XX,inv(16)(p13q22)[20]	CBFB::MYH11
例7	62%	原始细胞	67.2%	<0.1%	CD9、CD11c、CD13、CD19dim、CD33、CD34、CD38、CD56、CD64、CD117、HLA-DR、MPO	46,XX,del(7)(q22q32), t(8;21)(q22;q22)[18]/46,XX[2]	RUNX1::RUNX1T1

患儿	影像学检查	部位	肿瘤边界	穿刺活检术	外科手术 (移植前)	大小
患儿	影像学检查	部位	肿瘤边界	穿刺活检术	外科手术 (移植前)	初诊	移植+90 d
例1	CT	前上纵隔	不清	是	否	70 mm×52 mm×138 mm	28mm×10mm×23mm
例2	CT	额部中线区近囟门处	清晰	否	是	16.1 mm×6.8 mm×14.5 mm	-
例3	CT	左侧第7后肋近肋椎关节	清晰	否	是	31 mm×15 mm×17 mm	-
例4	CT	双侧髂骨周围	清晰	否	否	45 mm×47 mm×57 mm(右) 36 mm×20 mm×27 mm(左)	27mm×18mm×50mm(右)
例4	CT	双侧髂骨周围	清晰	否	否	45 mm×47 mm×57 mm(右) 36 mm×20 mm×27 mm(左)	31mm×15mm×26mm(左)
例5	CT	双侧颞部、眶内、颧骨及下颌骨体及颅底诸骨周围	清晰	否	是	36 mm×7 mm×33 mm(右) 32 mm×7 mm×42 mm(左)	-
例6	MRI	左眼部及颞部	不清	否	否	87 mm×69 mm×99 mm	-
例7	CT	双侧颌面部、眼眶周围、颅骨内外板	不清	否	否	-	-

患儿	性别	诊断年龄 /岁	诊断至移植时间间隔 /d	HLA 匹配	供者	ABO血型		移植物	CD34⁺ (10⁶/kg)	MNC (10⁸/kg)	植入时间/d		TOMO	预处理方案	aGVHD 发生	结局
患儿	性别	诊断年龄 /岁	诊断至移植时间间隔 /d	HLA 匹配	供者	供者	受者	移植物	CD34⁺ (10⁶/kg)	MNC (10⁸/kg)	粒系	血小板	TOMO	预处理方案	aGVHD 发生	结局
例1	男	14.0	110	9/10	无关供者	O+	B+	PBSC	7.1	7.4	13	12	是	BUCY	II˚肠道	存活
例2	女	0.8	93	8/12	父亲	A+	O+	PBSC	13.21	20.37	14	11	否	PTCY	I˚肠道	复发死亡
例3	男	1.5	141	5/10	父亲	A+	A+	PBSC	14.6	33.9	15	15	是	BUCY	无	存活
例4	女	2.4	572	6/10	母亲	B+	B+	PBSC	14.7	19.87	15	17	是	PTCY	II˚皮肤	复发死亡
例5	男	2.9	101	5/10	父亲	A+	A+	PBSC	12.87	18.26	14	12	否	PTCY	II˚皮肤	存活
例6	女	9.6	84	6/12	父亲	A+	O+	PBSC	12.11	16.13	15	13	否	PTCY	I˚肠道	存活
例7	女	5.3	117	9/12	父亲	A+	A+	PBSC	10.01	24.28	15	12	是	PTCY	无	存活

Clinical analysis of allogeneic hematopoietic stem cell transplantation on pediatric myeloid sarcoma

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