Systemic treatment of advanced clear cell sarcoma: results from a retrospective international series from the World Sarcoma Network.
Systemic treatment of advanced clear cell sarcoma: results from a retrospective international series from the World Sarcoma Network.
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DOI：
10.1016/j.esmoop.2022.100522
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发表时间：
2022-06
期刊：
影响因子：
7.3
通讯作者：
Jones, R. L.
中科院分区：
文献类型：
--
作者：
Smrke, A.;Frezza, A. M.;Giani, C.;Somaiah, N.;Brahmi, M.;Czarnecka, A. M.;Rutkowski, P.;Van der Graaf, W.;Baldi, G. G.;Connolly, E.;Duffaud, F.;Huang, P. H.;Gelderblom, H.;Bhadri, V;Grimison, P.;Mahar, A.;Stacchiotti, S.;Jones, R. L.
透明细胞肉瘤（CCS）是一种易位性侵袭性恶性肿瘤，转移发生率高，预后差。很少有研究描述全身治疗在CCS中的活性。我们报告了一项多机构回顾性研究，研究了世界肉瘤网络（WSN）中接受全身治疗的晚期CCS患者的结果。1985年6月至2021年5月，分子确诊的局部晚期或转移性CCS患者接受了全身治疗。基线人口统计和治疗信息，包括实体肿瘤反应评价标准（RECIST） 1.1的反应，由当地调查人员回顾性收集。进行描述性统计。纳入来自10家机构的55名患者。诊断时，中位年龄为30岁（15-73岁），24% （n = 13/55）有转移性疾病。诊断时的中位年龄为30岁（15-73岁）。大多数原发肿瘤位于腱膜（n = 9/55, 16%）或非腱膜肢体部位（n = 17/55, 31%）。最常见的融合是EWSR1-ATF1 （n = 24/ 55,44 %）。全身治疗的中位数为1次（范围1-7次）。接受舒尼替尼治疗的患者反应率最高（30%,n = 3/10），中位无进展生存期为4个月[95%置信区间（CI） 1-7]个月。晚期/转移性疾病患者的中位总生存期为15个月（95% CI 3-27个月）。软组织肉瘤型全身治疗在晚期CCS中获益有限，反应率较差。需要国际多中心前瞻性转化研究来确定这种超罕见亚型的新治疗方法，并且获得早期临床试验登记仍然是CCS患者的关键。这是报道的最大规模的接受全身治疗的晚期CCS患者系列。肉瘤型全身治疗的活性很差，只有舒尼替尼的反应一般。需要有效的治疗方法来改善这种超罕见肉瘤类型患者的预后。
Clear cell sarcoma (CCS) is a translocated aggressive malignancy with a high incidence of metastases and poor prognosis. There are few studies describing the activity of systemic therapy in CCS. We report a multi-institutional retrospective study of the outcomes of patients with advanced CCS treated with systemic therapy within the World Sarcoma Network (WSN). Patients with molecularly confirmed locally advanced or metastatic CCS treated with systemic therapy from June 1985 to May 2021 were included. Baseline demographic and treatment information, including response by Response Evaluation Criteria in Solid Tumours (RECIST) 1.1, was retrospectively collected by local investigators. Descriptive statistics were carried out. Fifty-five patients from 10 institutions were included. At diagnosis, the median age was 30 (15-73) years and 24% (n = 13/55) had metastatic disease. The median age at diagnosis was 30 (15-73) years. Most primary tumours were at aponeurosis (n = 9/55, 16%) or non-aponeurosis limb sites (n = 17/55, 31%). The most common fusion was EWSR1–ATF1 (n = 24/55, 44%). The median number of systemic therapies was 1 (range 1-7). The best response rate was seen for patients treated with sunitinib (30%, n = 3/10), with a median progression-free survival of 4 [95% confidence interval (CI) 1-7] months. The median overall survival for patients with advanced/metastatic disease was 15 months (95% CI 3-27 months). Soft tissue sarcoma-type systemic therapies have limited benefit in advanced CCS and response rate was poor. International, multicentre prospective translational studies are required to identify new treatments for this ultra-rare subtype, and access to early clinical trial enrolment remains key for patients with CCS. This is the largest reported series of advanced CCS patients treated with systemic therapy. The activity of sarcoma-type systemic therapy is poor and modest responses were seen only with sunitinib. Effective therapies are needed to improve outcomes for patients with this ultra-rare sarcoma type.
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影响因子：
51.1
作者：
Gounder, Mrinal;Schoffski, Patrick;Stacchiotti, Silvia
通讯作者：
Stacchiotti, Silvia
影响因子：
6.2
作者：
Stacchiotti S;Frezza AM;Blay JY;Baldini EH;Bonvalot S;Bovée JVMG;Callegaro D;Casali PG;Chiang RC;Demetri GD;Demicco EG;Desai J;Eriksson M;Gelderblom H;George S;Gounder MM;Gronchi A;Gupta A;Haas RL;Hayes-Jardon A;Hohenberger P;Jones KB;Jones RL;Kasper B;Kawai A;Kirsch DG;Kleinerman ES;Le Cesne A;Lim J;Chirlaque López MD;Maestro R;Marcos-Gragera R;Martin Broto J;Matsuda T;Mir O;Patel SR;Raut CP;Razak ARA;Reed DR;Rutkowski P;Sanfilippo RG;Sbaraglia M;Schaefer IM;Strauss DC;Sundby Hall K;Tap WD;Thomas DM;van der Graaf WTA;van Houdt WJ;Visser O;von Mehren M;Wagner AJ;Wilky BA;Won YJ;Fletcher CDM;Dei Tos AP;Trama A
通讯作者：
Trama A
影响因子：
3.4
作者：
Jones, Robin L.;Constantinidou, Anastasia;Judson, Ian R.
通讯作者：
Judson, Ian R.
影响因子：
50.5
作者：
Schoffski, P.;Wozniak, A.;Bauer, S.
通讯作者：
Bauer, S.
影响因子：
1.5
作者：
Hocar O;Le Cesne A;Berissi S;Terrier P;Bonvalot S;Vanel D;Auperin A;Le Pechoux C;Bui B;Coindre JM;Robert C
通讯作者：
Robert C