Patterns of care and outcomes of patients with METAstatic soft tissue SARComa in a real-life setting: the METASARC observational study.
Patterns of care and outcomes of patients with METAstatic soft tissue SARComa in a real-life setting: the METASARC observational study.
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DOI：
10.1186/s12916-017-0831-7
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发表时间：
2017-04-10
期刊：
影响因子：
9.3
通讯作者：
Italiano A
中科院分区：
文献类型：
--
作者：
Savina M;Le Cesne A;Blay JY;Ray-Coquard I;Mir O;Toulmonde M;Cousin S;Terrier P;Ranchere-Vince D;Meeus P;Stoeckle E;Honoré C;Sargos P;Sunyach MP;Le Péchoux C;Giraud A;Bellera C;Le Loarer F;Italiano A
需要对罕见肿瘤患者进行精心设计的观察性研究，以改善患者护理，临床研究和医疗保健专业人员的教育。分析了1990年至2013年间诊断并记录在法国肉瘤组前瞻性维护数据库中的2225例转移性软组织肉瘤患者的护理模式、结局和预后因素。全身治疗的中位次数为3次（范围，1-6次）; 27%的患者未接受任何全身治疗，1054例（49%）患者接受了转移灶的局部治疗。接受化疗的患者中有一半（n = 810）接受了标签外药物。平滑肌肉瘤的预后明显好于其他组织学亚型。除平滑肌瘤外，大于三线方案的获益非常有限，至下次治疗的中位时间（TNT）和总生存期（OS）分别为2.3 - 3.7个月和5.4 - 8.5个月。TNT与OS高度相关。在多变量分析中，女性性别、平滑肌肉瘤组织学、转移灶的局部治疗、临床试验入选和一线综合化疗治疗与OS改善显著相关。多柔比星与第二种药物（如异环磷酰胺）的联合治疗是一种有效的选择，特别是当肿瘤缩小有望提供临床获益时。二线治疗失败后，应考虑给予最佳支持治疗，特别是对于无资格参加临床试验的非平滑肌肉瘤组织学患者。在可行的情况下，转移灶的局部治疗应始终包括在治疗策略中。TNT可能代表临床研究中OS的有用替代终点。本文的在线版本（doi：10.1186/s12916-017-0831-7）包含补充材料，可供授权用户使用。
Well-designed observational studies of individuals with rare tumors are needed to improve patient care, clinical investigations, and the education of healthcare professionals. The patterns of care, outcomes, and prognostic factors of a cohort of 2225 patients with metastatic soft tissue sarcomas who were diagnosed between 1990 and 2013 and documented in the prospectively maintained database of the French Sarcoma Group were analyzed. The median number of systemic treatments was 3 (range, 1–6); 27% of the patients did not receive any systemic treatment and 1054 (49%) patients underwent locoregional treatment of the metastasis. Half of the patients who underwent chemotherapy (n = 810) received an off-label drug. Leiomyosarcoma was associated with a significantly better outcome than the other histological subtypes. With the exception of leiomyosarcomas, the benefit of a greater than third-line regimen was very limited, with a median time to next treatment (TNT) and overall survival (OS) ranging between 2.3 and 3.7 months and 5.4 and 8.5 months, respectively. The TNT was highly correlated with OS. Female sex, leiomyosarcoma histology, locoregional treatment of metastases, inclusion in a clinical trial, and treatment with first-line polychemotherapy were significantly associated with improved OS in the multivariate analysis. The combination of doxorubicin with a second drug, such as ifosfamide, represents a valid option, particularly when tumor shrinkage is expected to provide clinical benefits. After failure of the second-line therapy, best supportive care should be considered, particularly in patients with non-leiomyosarcoma histology who are not eligible to participate in a clinical trial. Locoregional treatment of metastasis should always be included in the therapeutic strategy when feasible. TNT may represent a useful surrogate endpoint for OS in clinical studies. The online version of this article (doi:10.1186/s12916-017-0831-7) contains supplementary material, which is available to authorized users.