000177013 001__ 177013 000177013 005__ 20240229133728.0 000177013 0247_ $$2pmid$$apmid:34636137 000177013 0247_ $$2ISSN$$a0098-1532 000177013 0247_ $$2ISSN$$a1096-911X 000177013 0247_ $$2ISSN$$a1545-5009 000177013 0247_ $$2ISSN$$a1545-5017 000177013 0247_ $$2doi$$aDOI: 10.1002/pbc.29403 000177013 0247_ $$2doi$$aDOI: 10.1002/pbc.29403 000177013 0247_ $$2altmetric$$aaltmetric:115073530 000177013 037__ $$aDKFZ-2021-02243 000177013 041__ $$aEnglish 000177013 082__ $$a610 000177013 1001_ $$00000-0001-9551-2399$$aSparber-Sauer, Monika$$b0 000177013 245__ $$aInfantile myofibromatosis: Excellent prognosis but also rare fatal progressive disease. Treatment results of five Cooperative Weichteilsarkom Studiengruppe (CWS) trials and one registry. 000177013 260__ $$aNew York, NY$$bWiley$$c2022 000177013 3367_ $$2DRIVER$$aarticle 000177013 3367_ $$2DataCite$$aOutput Types/Journal article 000177013 3367_ $$0PUB:(DE-HGF)16$$2PUB:(DE-HGF)$$aJournal Article$$bjournal$$mjournal$$s1644584537_13894 000177013 3367_ $$2BibTeX$$aARTICLE 000177013 3367_ $$2ORCID$$aJOURNAL_ARTICLE 000177013 3367_ $$00$$2EndNote$$aJournal Article 000177013 500__ $$aVolume69, Issue3, March 2022, e29403 000177013 520__ $$aInfantile myofibromatosis (IM) is a rare benign soft tissue tumor and often a self-limiting disease but rarely includes life-threatening complications. Little is known about optimal treatment of primary localized (LD) and multifocal disease (MFD).Treatment and outcome of 95 children with IM registered within five Cooperative Weichteilsarkom Studiengruppe (CWS) trials and one registry (1981-2016) were evaluated.LD was diagnosed in 71 patients at a median age of 0.4 years (range 0.0-17.7). MFD was present in 24 patients. The mainstay of treatment was watch-and-wait strategy (w&w) after initial biopsy or resection. Low-dose chemotherapy (CHT) was administered to 16/71 (23%) patients with LD and eight of 24 (33%) patients with MFD, imatinib was added in two. A delayed resection was possible in eight of 71 (11%) and five of 24 (21%) patients with LD and MFD, respectively. Overall, patients were alive in complete remission (n = 77) and partial remission (n = 10) at a median follow-up time of 3.4 years after diagnosis (range 0.01-19.4); no data available (n = 5). Three patients died of progressive disease (PD) despite CHT. Gender, tumor size, and location correlated with a favorable event-free survival (EFS) in patients with LD. The 5-year EFS and overall survival of patients with LD were 73% (±12, confidence interval [CI] 95%) and 95% (±6, CI 95%), respectively; for MFD 51% (±22, CI 95%) and 95% (±10, CI 95%).Prognosis is excellent in patients with LD and MFD. Targeted treatment needs to be evaluated for rare fatal PD. 000177013 536__ $$0G:(DE-HGF)POF4-313$$a313 - Krebsrisikofaktoren und Prävention (POF4-313)$$cPOF4-313$$fPOF IV$$x0 000177013 588__ $$aDataset connected to DataCite, PubMed, , Journals: inrepo01.inet.dkfz-heidelberg.de 000177013 650_7 $$2Other$$aCWS Group 000177013 650_7 $$2Other$$ainfantile myofibromatosis 000177013 650_7 $$2Other$$ainfants and children 000177013 650_7 $$2Other$$alocalized and multifocal disease 000177013 7001_ $$00000-0002-4138-4536$$aVokuhl, Christian$$b1 000177013 7001_ $$00000-0001-9280-1451$$aSeitz, Guido$$b2 000177013 7001_ $$aSorg, Benjamin$$b3 000177013 7001_ $$0P:(DE-HGF)0$$aTobias, Möllers$$b4 000177013 7001_ $$avon Kalle, Thekla$$b5 000177013 7001_ $$aMünter, Marc$$b6 000177013 7001_ $$aBielack, Stefan S$$b7 000177013 7001_ $$aLadenstein, Ruth$$b8 000177013 7001_ $$aLjungman, Gustaf$$b9 000177013 7001_ $$aNiggli, Felix$$b10 000177013 7001_ $$aFrühwald, Michael$$b11 000177013 7001_ $$aLoff, Stefan$$b12 000177013 7001_ $$aKlingebiel, Thomas$$b13 000177013 7001_ $$00000-0002-1519-7569$$aKoscielniak, Ewa$$b14 000177013 773__ $$0PERI:(DE-600)2130978-4$$a10.1002/pbc.29403$$n3$$pe29403$$tPediatric blood & cancer$$v69$$x1545-5009$$y2022 000177013 909CO $$ooai:inrepo02.dkfz.de:177013$$pVDB 000177013 9101_ $$0I:(DE-588b)2036810-0$$6P:(DE-HGF)0$$aDeutsches Krebsforschungszentrum$$b4$$kDKFZ 000177013 9131_ $$0G:(DE-HGF)POF4-313$$1G:(DE-HGF)POF4-310$$2G:(DE-HGF)POF4-300$$3G:(DE-HGF)POF4$$4G:(DE-HGF)POF$$aDE-HGF$$bGesundheit$$lKrebsforschung$$vKrebsrisikofaktoren und Prävention$$x0 000177013 9141_ $$y2021 000177013 915__ $$0StatID:(DE-HGF)3001$$2StatID$$aDEAL Wiley$$d2021-02-02$$wger 000177013 915__ $$0StatID:(DE-HGF)0160$$2StatID$$aDBCoverage$$bEssential Science Indicators$$d2021-02-02 000177013 915__ $$0StatID:(DE-HGF)1190$$2StatID$$aDBCoverage$$bBiological Abstracts$$d2021-02-02 000177013 915__ $$0StatID:(DE-HGF)0113$$2StatID$$aWoS$$bScience Citation Index Expanded$$d2021-02-02 000177013 915__ $$0StatID:(DE-HGF)0200$$2StatID$$aDBCoverage$$bSCOPUS$$d2022-11-18 000177013 915__ $$0StatID:(DE-HGF)0300$$2StatID$$aDBCoverage$$bMedline$$d2022-11-18 000177013 915__ $$0StatID:(DE-HGF)0199$$2StatID$$aDBCoverage$$bClarivate Analytics Master Journal List$$d2022-11-18 000177013 915__ $$0StatID:(DE-HGF)0150$$2StatID$$aDBCoverage$$bWeb of Science Core Collection$$d2022-11-18 000177013 915__ $$0StatID:(DE-HGF)1050$$2StatID$$aDBCoverage$$bBIOSIS Previews$$d2022-11-18 000177013 915__ $$0StatID:(DE-HGF)1110$$2StatID$$aDBCoverage$$bCurrent Contents - Clinical Medicine$$d2022-11-18 000177013 9201_ $$0I:(DE-He78)C070-20160331$$kC070$$lC070 Klinische Epidemiologie und Alternf.$$x0 000177013 980__ $$ajournal 000177013 980__ $$aVDB 000177013 980__ $$aI:(DE-He78)C070-20160331 000177013 980__ $$aUNRESTRICTED