西罗莫司治疗儿童弥漫性肺淋巴管瘤病1例报告

doi:10.12372/jcp.2025.24e0399

Abstract

Abstract:

Objective To explore the clinical characteristics and treatment strategies of diffuse pulmonary lymphangiomatosis in children, in order to improve the early diagnosis and treatment level and the prognosis of children. Methods The clinical data of a child with diffuse pulmonary lymphangiomatosis admitted to the respiratory department of our hospital were retrospectively analyzed. The clinical manifestations, diagnostic process, and treatment outcomes were systematically summarized. Furthermore, current comprehensive treatment options and prognostic factors were discussed in light of relevant literature. Results The child received sirolimus treatment for 3 years, and the clinical symptoms were significantly relieved. Chest CT showed significant improvement. Conclusion Sirolimus demonstrates promising therapeutic efficacy in the management of diffuse pulmonary lymphangiomatosis in children, with a favorable safety profile and the potential to significantly enhance long-term prognosis.

Key words: diffuse pulmonary lymphangiomatosis, sirolimus, child

CLC Number:

YE Zehui, JIANG Xiaoli. A case report of sirolimus in the treatment of diffuse pulmonary lymphangiomatosis in children[J].Journal of Clinical Pediatrics, 2025, 43(9): 705-709.

Figures/Tables 6

Figure 1

Figure 2

Table 1

Figure 3

Figure 4

Table 2

References 24

[1]	沈秋英, 农光民, 顾永耀. 儿童弥漫性肺淋巴管瘤病二例[J]. 中华儿科杂志, 2016, 54(10): 781-782.
	Shen QY, Nong GM, Gu YY. Two cases of diffuse pulmonary lymphangioma in children[J]. Zhonghua Erke Zazhi, 2016, 54(10): 781-782.
[2]	Ernotte C, Médart L, Collignon L. Diffuse pulmonary lymphangiomatosis[J]. J Belg Soc Radiol, 2018, 102(1): 64. doi: 10.5334/jbsr.1603 pmid: 30324183
[3]	Gurskytė V, Zeleckienė I, Maskoliūnaitė V, et al. Successful treatment of diffuse pulmonary lymphangiomatosis with sirolimus[J]. Respir Med Case Rep, 2020, 29: 101014.
[4]	Triana P, Dore M, Cerezo VN, et al. Sirolimus in the treatment of vascular anomalies[J]. Eur J Pediatr Surg, 2017, 27(1): 86-90. doi: 10.1055/s-0036-1593383 pmid: 27723921
[5]	Luisi F, Torre O, Harari S. Thoracic involvement in generalised lymphatic anomaly (or lymphangiomatosis)[J]. Eur Respir Rev, 2016, 25(140): 170-177. doi: 10.1183/16000617.0018-2016 pmid: 27246594
[6]	Zheng G, Tang H, Su R, et al. A gene missense mutation in diffuse pulmonary lymphangi- omatosis with thrombocytopenia: a case report[J]. Medicine (Baltimore), 2020, 99(39): e21941.
[7]	Faul JL, Berry GJ, Colby TV, et al. Thoracic lymphangiomas, lymphangiectasis, lymphangiomatosis, and lymphatic dysplasia syndrome[J]. Am J Respir Crit Care Med, 2000, 161(3Pt 1): 1037-1046.
[8]	Moreno RP, Hernández Y, Garrido P, et al. Diffuse pulmonary lymphangiomatosis with pleural and pericardial involvement. Pediatric case report[J]. Arch Argent Pediatr, 2021, 119(3): e264-e268.
[9]	Liu JR, Shen WB, Wen Z, et al. Clinical analysis of two cases with diffuse pulmonary lymphatic disease[J]. Zhonghua Erke Zazhi, 2016, 54(5): 360-364.
[10]	Ricci KW, Iacobas I. How we approach the diagnosis and management of complex lymphatic anomalies[J]. Pediatr Blood Cancer, 2022, 69(Suppl 3): e28985.
[11]	曾祥妮, 吴爱民, 李岚, 等. 一例弥漫性肺淋巴管瘤病支气管镜下表现及分析[J]. 临床儿科杂志, 2024, 42(12): 1047-1050..
	Zeng XN, Wu AM, Li L, et al. A case report of a rare case of diffuse pulmonary lymphangiomatosis with bronchoscopic presentation[J]. Linchuang Erke Zazhi, 2024, 42(12): 1047-1050.
[12]	Sun X, Shen W, Xia S, et al. Diffuse pulmonary lymphangiomatosis: MDCT findings after direct lymphangiography[J]. AJR Am J Roentgenol, 2017, 208(2): 300-305.
[13]	潘登, 丛金鹏, 王芳芳, 等. 弥漫性肺淋巴管瘤病二例报[J]. 临床医学进展, 2020, 10(7): 1397-1404.
	Pan D, Cong JP, Wang FF, et al. Diffuse pulmonary lymphangiomatosis: report of two cases[J]. Linchuang Yixue Jinzhan, 2020, 10(7): 1397-1404.
[14]	Biscotto I, Rodrigues RS, Forny DN, et al. Diffuse pulmonary lymphangiomatosis[J]. J Bras Pneumol, 2019, 45(5): e20180412.
[15]	Lim HJ, Han J, Kim HK, et al. A rare case of diffuse pulmonary lymphangiomatosis in a middle-aged woman[J]. Korean J Radiol, 2014, 15(2): 295-299.
[16]	Ozeki M, Nozawa A, Yasue S, et al. The impact of sirolimus therapy on lesion size, clinical symptoms, and quality of life of patients with lymphatic anomalies[J]. Orphanet J Rare Dis, 2019, 14(1): 141. doi: 10.1186/s13023-019-1118-1 pmid: 31196128
[17]	Ricci KW, Hammill AM, Mobberley-Schuman P, et al. Efficacy of systemic sirolimus in the treatment of generalized lymphatic anomaly and Gorham-Stout disease[J]. Pediatr Blood Cancer, 2019, 66(5): e27614.
[18]	Rodriguez-Laguna L, Agra N, Ibañez K, et al. Somatic activating mutations in PIK3CA cause generalized lymphatic anomaly[J]. J Exp Med, 2019, 216(2): 407-418. doi: 10.1084/jem.20181353
[19]	Reinglas J, Ramphal R, Bromwich M. The successful management of diffuse lymphangio matosis using sirolimus: a case report[J]. Laryngoscope, 2011, 121(9): 1851-1854.
[20]	Sun X, Lu C, Huang Z, et al. Diagnosis and treatment of diffuse pulmonary lymphangioma in children: a case report[J]. Exp Ther Med, 2023, 25(4): 175. doi: 10.3892/etm.2023.11874 pmid: 37006871
[21]	Dimiene I, Bieksiene K, Zaveckiene J, et al. Effective initial treatment of diffuse pulmonary lymphangiomatosis with sirolimus and propranolol: a case report[J]. Medicina (Kaunas), 2021, 57(12): 1308.
[22]	Amodeo I, Colnaghi M, Raffaeli G, et al. The use of sirolimus in the treatment of giant cystic lymphangioma: four case reports and update of medical therapy[J]. Medicine (Baltimore), 2017, 96(51): e8871.
[23]	Ferrara N. Vascular endothelial growth factor: basic science and clinical progress[J]. Endocr Rev, 2004, 25(4): 581-611. doi: 10.1210/er.2003-0027 pmid: 15294883
[24]	Onyeforo E, Barnett A, Zagami D, et al. Diffuse pulmonary lymphangiomatosis treated with bevacizumab[J]. Respirol Case Rep, 2018, 7(1): e00384.

治疗时间/月	血药浓度/ng·mL^-1	西罗莫司剂量调整
治疗3个月	5.30	0.75 mg，qd
治疗6个月	5.75	0.75 mg，qd
治疗12个月	3.00	1 mg，qd
治疗15个月	6.16	1 mg，qd
治疗18个月	4.16	0.7 mg，bid
治疗24个月	8.10	1 mg，qd
治疗35个月	6.52	停药

治疗时间/月	囊性病变大小/cm	体积/cm³
治疗前	6.3×4.2×3.7	97.9
治疗3个月	4.7×4.3×2.2	44.46
治疗6个月	4.0×2.4×2.1	20.16
治疗12个月	3.9×2.7×1.5	15.79
治疗24个月	3.5×2.4×1.8	15.12
治疗35个月	3.5×2.3×1.6	12.88

A case report of sirolimus in the treatment of diffuse pulmonary lymphangiomatosis in children

RichHTML

PDF (PC)

Like

Knowledge

Abstract

Cite this article

share this article

Figures/Tables 6

References 24

Related Articles 15

Metrics

Comments

Recommended 0

[1]	ZHANG Wei, WANG Yang, DENG Wenhua, WU Yabin. Analysis of clinical manifestations, ciliary structure and genetic characteristics of primary ciliary dyskinesia in 14 children [J]. Journal of Clinical Pediatrics, 2025, 43(9): 680-685.
[2]	XIANG Linjuan, CHEN Xuexin, JIA Yanhui, WU Yuhang, CONG Xin, LI Wei, CHEN Yingying, CHEN Sun, HUANG Lisu. Prognostic factors analysis of adenovirus type 3 pneumonia in children [J]. Journal of Clinical Pediatrics, 2025, 43(9): 686-691.
[3]	WANG Jie, WU Bin, ZHANG Lannan, CHEN Kailan. Two cases of pediatric hepatosplenic T-cell lymphoma and literature review [J]. Journal of Clinical Pediatrics, 2025, 43(9): 698-704.
[4]	DONG Suzhen, CHEN Hao, ZHANG Zhiyong, JIANG Fan. Applications of low field MRI in pediatrics and prenatal fetal diagnosis [J]. Journal of Clinical Pediatrics, 2025, 43(9): 710-715.
[5]	SHI Xiaosong, FU Shijie, HE Xiaohua, LYU Hui, CHEN Houyang, CHEN Maolin, CHEN Jie. Antigenic genotypic characteristics and antibiotic resistance analysis of Bordetella pertussis in two regions of Fujian province [J]. Journal of Clinical Pediatrics, 2025, 43(8): 575-582.
[6]	PEI Pei, LI Weihua, HUAI Wan, YAO Ruen, GE Hejia, WANG Jiwen, WANG Xiumin, JI Wei, ZHOU Yunqing, HE Yingzhong, HAN Feng. Genetic and clinical characteristics analysis of 10 children with ATP1A2/ATP1A3 gene variants [J]. Journal of Clinical Pediatrics, 2025, 43(8): 590-597.
[7]	LI Heting, SUN Ruidi, JIANG Jun. Clinical features, prognosis and neurophysiological characteristics of children with acute anti-GQ1b antibody syndrome [J]. Journal of Clinical Pediatrics, 2025, 43(8): 604-609.
[8]	ZHANG Cheng, CAO Qiaoyu, ZHENG Luyao, LI Ming, GE Hongsong. Retrospective analysis of clinical features, dermoscopic characteristics and prognosis of acquired facial hyperpigmented macules in 54 pediatric cases [J]. Journal of Clinical Pediatrics, 2025, 43(8): 610-614.
[9]	Chinese Cooperative Group for the Management of Pediatric Enuresis Affiliated to Pediatric Nephrology Committee of Chinese Medical Doctor Association. Chinese expert consensus on management of nocturnal enuresis in children [J]. Journal of Clinical Pediatrics, 2025, 43(7): 483-499.
[10]	WANG Yingshuo, CHEN Zhimin. Leveraging digital intelligence to enhance the diagnosis, treatment, and management of pediatric bronchial asthma [J]. Journal of Clinical Pediatrics, 2025, 43(7): 500-504.
[11]	ZHAO Linchao, WANG Yingjie, JING Zhaohe, MAI Yumiao, SUN Pan, QIU Simin, NIU Hongyun, CHEN Zhiwei, DONG Pengpeng, LIU Jian. Analysis of the efficacy and prognosis of hematopoietic stem cell transplantation for inherited bone marrow failure syndrome in children [J]. Journal of Clinical Pediatrics, 2025, 43(7): 505-510.
[12]	ZHANG Zhiqi, XIONG Ruolan, LI Bohan, JI Qi, WANG Qingwei, LU Jun, LI Jie, XIAO Peifang, HU Shaoyan. Construction of risk prediction model for primary graft failure after umbilical cord blood transplantation in pediatric leukemia [J]. Journal of Clinical Pediatrics, 2025, 43(7): 511-518.
[13]	JIANG Wenwen, LI Sitong, XU Yongsheng. Analysis on risk factors associated with false-negative results of interferon-gamma release assay in children with pulmonary tuberculosis [J]. Journal of Clinical Pediatrics, 2025, 43(7): 519-524.
[14]	LI Fan, HUANG Xianjie, FAN Yazhen, ZHAO Jianchuang, CUI Chenhang, GUO Qiliang, QIAO Junying. Tocilizumab treatment for febrile infection-related epilepsy syndrome in children: 2 cases report and literature review [J]. Journal of Clinical Pediatrics, 2025, 43(7): 532-538.
[15]	DUAN Haolin, ZHANG Ciliu, XIONG Juan, PANG Nan, YIN Fei, PENG Jing. Clinical efficacy analysis of disease-modifying therapies for spinal muscular atrophy with SMN1 gene compound heterozygous variants [J]. Journal of Clinical Pediatrics, 2025, 43(7): 543-548.