A Retrospective Review of the Use of Sirolimus for Pediatric Patients With Epithelioid Hemangioendothelioma

被引：23

作者：

Engel, Elissa R. ^{[1
]}

Cournoyer, Eily ^{[4
]}

Adams, Denise M. ^{[5
]}

Stapleton, Stacie ^{[2
,3
]}

机构：

[1] Univ S Florida, Dept Pediat, Tampa, FL 33620 USA

[2] Johns Hopkins All Childrens Hosp Canc, St Petersburg, FL USA

[3] Blood Disorders Inst, St Petersburg, FL USA

[4] Boston Univ, Sch Med, Boston, MA 02118 USA

[5] Boston Childrens Hosp, Vasc Anomalies Ctr, Div Hematol & Oncol, Boston, MA USA

来源：

JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY | 2020年 / 42卷 / 08期

关键词：

epithelioid hemangioendothelioma; treatment; sirolimus; pediatrics; children; VASCULAR ANOMALIES;

D O I：

10.1097/MPH.0000000000001643

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Epithelioid hemangioendothelioma (EHE) is a rare malignant vascular tumor with no standardized treatment. The mammalian target of rapamycin inhibitor, sirolimus, has been used successfully in adult EHE and other vascular tumors in children but has not been studied in pediatric EHE. The aim of this retrospective case series is to discuss the results of sirolimus for treatment in 6 pediatric patients with EHE. Four of 6 patients demonstrated partial response or disease stabilization with sirolimus treatment. No treatment dosing, trough goals, or duration of treatment recommendations can be made. Prospective studies are warranted to further investigate the use of sirolimus in treatment of EHE.

引用

收藏

页码：E826 / E829

页数：4

相关论文

共 13 条

[1] Efficacy and Safety of Sirolimus in the Treatment of Complicated Vascular Anomalies [J].

Adams, Denise M. ;

Trenor, Cameron C., III ;

Hammill, Adrienne M. ;

Vinks, Alexander A. ;

Patel, Manish N. ;

Chaudry, Gulraiz ;

Wentzel, Mary Sue ;

Mobberley-Schuman, Paula S. ;

Campbell, Lisa M. ;

Brookbank, Christine ;

Gupta, Anita ;

Chute, Carol ;

Eile, Jennifer ;

McKenna, Jesse ;

Merrow, Arnold C. ;

Fei, Lin ;

Hornung, Lindsey ;

Seid, Michael ;

Dasgupta, A. Roshni ;

Dickie, Belinda H. ;

Elluru, Ravindhra G. ;

Lucky, Anne W. ;

Weiss, Brian ;

Azizkhan, Richard G. .

PEDIATRICS, 2016, 137 (02)

[2] An open-label, multicenter, phase II study of bevacizumab for the treatment of angiosarcoma and epithelioid hemangioendotheliomas† [J].

Agulnik, M. ;

Yarber, J. L. ;

Okuno, S. H. ;

von Mehren, M. ;

Jovanovic, B. D. ;

Brockstein, B. E. ;

Evens, A. M. ;

Benjamin, R. S. .

ANNALS OF ONCOLOGY, 2013, 24 (01) :257-263

[3] Sorafenib in patients with progressive epithelioid hemangioendothelioma [J].

Chevreau, Christine ;

Le Cesne, Axel ;

Ray-Coquard, Isabelle ;

Italiano, Antoine ;

Cioffi, Angela ;

Isambert, Nicolas ;

Robin, Yves Marie ;

Fournier, Charles ;

Clisant, Stephanie ;

Chaigneau, Loic ;

Bay, Jacques-Olivier ;

Bompas, Emmanuelle ;

Gauthier, Eric ;

Blay, Jean Y. ;

Penel, Nicolas .

CANCER, 2013, 119 (14) :2639-2644

[4] Sirolimus for the Treatment of Complicated Vascular Anomalies in Children [J].

Hammill, Adrienne M. ;

Wentzel, MarySue ;

Gupta, Anita ;

Nelson, Stephen ;

Lucky, Anne ;

Elluru, Ravi ;

Dasgupta, Roshni ;

Azizkhan, Richard G. ;

Adams, Denise M. .

PEDIATRIC BLOOD & CANCER, 2011, 57 (06) :1018-1024

[5] Epithelioid hemangioendotheliomas of the liver and lung in children and adolescents [J].

Hettmer, Simone ;

Andrieux, Geoffroy ;

Hochrein, Jochen ;

Kurz, Philipp ;

Roessler, Jochen ;

Lassmann, Silke ;

Werner, Martin ;

von Bubnoff, Nikolas ;

Peters, Christoph ;

Koscielniak, Ewa ;

Sparber-Sauer, Monika ;

Niemeyer, Charlotte ;

Mentzel, Thomas ;

Busch, Hauke ;

Boerries, Melanie .

PEDIATRIC BLOOD & CANCER, 2017, 64 (12)

[6] Sirolimus for the treatment of children with various complicated vascular anomalies [J].

Lackner, Herwig ;

Karastaneva, Anna ;

Schwinger, Wolfgang ;

Benesch, Martin ;

Sovinz, Petra ;

Seidel, Markus ;

Sperl, Daniela ;

Lanz, Sofia ;

Haxhija, Emir ;

Reiterer, Friedrich ;

Sorantin, Erich ;

Urban, Christian E. .

EUROPEAN JOURNAL OF PEDIATRICS, 2015, 174 (12) :1579-1584

[7] Efficacy and Safety of Mammalian Target of Rapamycin Inhibitors in Vascular Anomalies: A Systematic Review [J].

Nadal, Marion ;

Giraudeau, Bruno ;

Tavernier, Elsa ;

Jonville-Bera, Annie-Pierre ;

Lorette, Gerard ;

Maruani, Annabel .

ACTA DERMATO-VENEREOLOGICA, 2016, 96 (04) :448-452

[8] Epithelioid Hemangioendothelioma: Update on Diagnosis and Treatment [J].

Rosenberg, Ari ;

Agulnik, Mark .

CURRENT TREATMENT OPTIONS IN ONCOLOGY, 2018, 19 (04)

[9] Epithelioid hemangioendothelioma: an overview and update on a rare vascular tumor [J].

Sardaro, Angela ;

Bardoscia, Lilia ;

Petruzzelli, Maria Fonte ;

Portaluri, Maurizio .

ONCOLOGY REVIEWS, 2014, 8 (02) :82-91

[10] CAMTA1 is a useful immunohistochemical marker for diagnosing epithelioid haemangioendothelioma [J].

Shibuya, Ryo ;

Matsuyama, Atsuji ;

Shiba, Eisuke ;

Harada, Hiroshi ;

Yabuki, Kei ;

Hisaoka, Masanori .

HISTOPATHOLOGY, 2015, 67 (06) :827-835