Systemic treatments and outcomes in CIC-rearranged Sarcoma: A national multi-centre clinicopathological series and literature review.
Systemic treatments and outcomes in CIC-rearranged Sarcoma: A national multi-centre clinicopathological series and literature review.
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DOI：
10.1002/cam4.4580
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发表时间：
2022-04
期刊：
影响因子：
4
通讯作者：
Hong, Angela
中科院分区：
文献类型：
--
作者：
Connolly, Elizabeth A.;Bhadri, Vivek A.;Wake, Johnathon;Ingley, Katrina M.;Lewin, Jeremy;Bae, Susie;Wong, Daniel D.;Long, Anne P.;Pryor, David;Thompson, Stephen R.;Strach, Madeleine C.;Grimison, Peter S.;Mahar, Annabelle;Bonar, Fiona;Maclean, Fiona;Hong, Angela
CIC重排肉瘤是最近建立的一种超罕见、分子定义的肉瘤亚型。我们的目标是进一步描述CIC重排肉瘤的临床特征，并探索包括系统治疗和结果在内的临床治疗方法。一项针对2014-2019年间确诊患者的多中心回溯性队列研究。共鉴定了18名患者。中位年龄27岁(13-56岁)，男性10例(56%)，有局限性疾病11例(61%)，确诊时有7例晚期(转移或无法切除)疾病。在11例确诊为局限性疾病的患者中，中位总生存期(OS)为40.6个月，1、2和5年OS估计分别为82%、%和34%。9名患者(82%)接受了手术(均接受了R0切除)，8名患者(73%)接受了原发部位的放射治疗(28次中位剂量57Gy)，8名患者(73%)接受了化疗(主要是尤因方案)。发生转移的比例为55%，中位复发时间为10.5个月。在确诊为晚期疾病的患者中，中位OS为12.6个月(95%可信区间5.1~20.1)，1年OS为57%。中位无进展生存期为5.8个月(95%可信区间为4.5~7.2)。持久的系统治疗反应很少发生，系统治疗反应的中位数持续时间为2.1个月。转移性疾病对VDC/IE化疗有一个持久的完全缓解。未见对帕佐帕尼(n=1)和培溴利珠单抗(n=1)的反应。在本系列中，CIC重排的肉瘤影响年轻人，并具有较高的转移性疾病的发生率。总体来说，预后很差。在晚期疾病中，持久的系统治疗反应很少见。最近建立的超罕见肉瘤亚型的系统治疗和结果的多中心回顾性系列研究。CIC重排的肉瘤似乎对化疗相对不敏感，这从局部疾病的高复发率和晚期的短期治疗反应中可见一斑。在晚期疾病中，CIC重排肉瘤的持久系统治疗反应是有限的，在本系列中，系统治疗反应的中位持续时间为2.1个月。
CIC‐rearranged sarcoma is a recently established, ultra‐rare, molecularly defined sarcoma subtype. We aimed to further characterise clinical features of CIC‐rearranged sarcomas and explore clinical management including systemic treatments and outcomes. A multi‐centre retrospective cohort study of patients diagnosed between 2014–2019. Eighteen patients were identified. The median age was 27 years (range 13–56), 10 patients were male (56%), 11 patients (61%) had localised disease and 7 patients had advanced (metastatic or unresectable) disease at diagnosis. Of 11 patients with localised disease at diagnosis, median overall survival (OS) was 40.6 months and the 1‐, 2‐ and 5‐year OS estimates were 82%, 64% and 34% respectively. Nine patients (82%) underwent surgery (all had R0 resections), 8 (73%) patients received radiotherapy to the primary site (median dose 57Gy in 28 fractions), and 8 (73%) patients received chemotherapy (predominantly Ewing‐based regimens). Metastases developed in 55% with a median time to recurrence of 10.5 months. In patients with advanced disease at diagnosis, median OS was 12.6 months (95% CI 5.1–20.1), 1‐year OS was 57%. Median progression‐free survival was 5.8 months (95% CI 4.5–7.2). Durable systemic therapy responses occurred infrequently with a median duration of systemic treatment response of 2.1 months. One durable complete response of metastatic disease to VDC/IE chemotherapy was seen. Responses to pazopanib (n = 1) and pembrolizumab (n = 1) were not seen. In this series, CIC‐rearranged sarcomas affected young adults and had a high incidence of presenting with, or developing, metastatic disease. The prognosis overall was poor. In advanced disease, durable systemic therapy responses were infrequent. Multi‐centre retrospective series of systemic therapy and outcomes for recently established ultra‐rare sarcoma subtype. CIC‐rearranged sarcomas appear to be relatively chemo‐insensitive as illustrated by a high frequency of relapse in localised disease and short durations of treatment response in the advanced setting. In advanced disease, durable systemic therapy responses in CIC‐rearranged sarcoma are limited with a median duration of systemic treatment response of 2.1 months in this series.
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影响因子：
5.6
作者：
Choi, Eun-Young Karen;Thomas, Dafydd G.;Lucas, David R.
通讯作者：
Lucas, David R.
影响因子：
51.1
作者：
Gounder, Mrinal;Schoffski, Patrick;Stacchiotti, Silvia
通讯作者：
Stacchiotti, Silvia
影响因子：
51.1
作者：
Judson, Ian;Morden, James P.;Bliss, Judith M.
通讯作者：
Bliss, Judith M.
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：
3.7
作者：
Italiano, Antoine;Sung, Yun Shao;Antonescu, Cristina R.
通讯作者：
Antonescu, Cristina R.