Endothelial cell malignancies in infants, children and adolescents: Treatment results of three Cooperative Weichteilsarkom Studiengruppe (CWS) trials and one registry.

Sparber-Sauer, Monika; Koscielniak, Ewa; Vokuhl, Christian; Schmid, Irene; Bien, Ewa; Seitz, Guido; Hallmen, Erika; von Kalle, Thekla; Scheer, Monika; Münter, Marc; Bielack, Stefan S; Niggli, Felix; Ljungman, Gustaf; Fuchs, Joerg; Hettmer, Simone; Rössler, Jochen; Klingebiel, Thomas (2020). Endothelial cell malignancies in infants, children and adolescents: Treatment results of three Cooperative Weichteilsarkom Studiengruppe (CWS) trials and one registry. Pediatric blood & cancer, 67(3), e28095. Wiley-Liss 10.1002/pbc.28095

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BACKGROUND Endothelial cell malignancies are extremely rare in childhood. New identification of genetic abnormalities (WWTR1:CAMTA1 translocation) helps to recognize potential therapeutic targets. Little is known about treatment and outcome of these patients. METHODS Clinical course, treatment, and outcome in patients with endothelial cell malignancies treated within the Cooperative Weichteilsarkom Studiengruppe (CWS) trials CWS-91, -96, -2002P, and the Soft-Tissue Sarcoma Registry (SoTiSaR) were analyzed (1991-2019). RESULTS Patients had angiosarcoma (AS) (n = 12), malignant epithelioid hemangioendothelioma (EHE) (n = 16), and kaposiform hemangioendothelioma (KHE) (n = 13). The median age was 5.39 years (range, 0.8-17.34); 33 patients had localized disease (LD), and 8 patients had metastatic disease. Therapy consisted of chemotherapy (CHT) (AS n = 8, EHE n = 9, KHE n = 5), interferon or new agent therapy (EHE n = 5, 2 KHE n = 2), microscopically or macroscopically complete resection (AS n = 3, EHE n = 6, KHE n = 3), and radiotherapy (AS n = 6, EHE n = 2, KHE n = 1). Two patients (KHE) had watch-and-wait strategy resulting in stable disease. Complete remission (CR) was achieved in AS (10/12; 83%), EHE (10/16; 63%), and KHE (5/13; 38%). The five-year EFS and OS for patients with AS was 64% (± 29 CI 95%) and 80% (± 25, CI 95%), with EHE 62% (± 24, CI 95%) and 78% (± 23, CI 95%), with KHE 33% (± 34, CI 95%) and 92% (± 15, CI 95%), respectively. Complete resection was a significant prognostic factor for AS, LD for EHE. CONCLUSIONS Endothelial cell malignancies in childhood have a fair outcome with multimodal treatment. New treatment options are needed for metastic disease.

Item Type:

Journal Article (Original Article)

Division/Institute:

04 Faculty of Medicine > Department of Gynaecology, Paediatrics and Endocrinology (DFKE) > Clinic of Paediatric Medicine
04 Faculty of Medicine > Department of Gynaecology, Paediatrics and Endocrinology (DFKE) > Clinic of Paediatric Medicine > Paediatric Haematology/Oncology

UniBE Contributor:

Rössler, Jochen Karl

Subjects:

600 Technology > 610 Medicine & health

ISSN:

1545-5009

Publisher:

Wiley-Liss

Language:

English

Submitter:

Jochen Karl Rössler

Date Deposited:

24 Jan 2020 17:10

Last Modified:

25 Jan 2020 01:33

Publisher DOI:

10.1002/pbc.28095

PubMed ID:

31814291

Uncontrolled Keywords:

CWS Group angiosarcoma children endothelial cell malignancies epithelioid hemangioendothelioma kaposiform hemangioendothelioma

BORIS DOI:

10.7892/boris.137589

URI:

https://boris.unibe.ch/id/eprint/137589

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