Savina Marion, Le Cesne Axel, Blay Jean-Yves, Ray-Coquard Isabelle, Mir Olivier, Toulmonde Maud, Cousin Sophie, Terrier Philippe, Ranchere-Vince Dominique, Meeus Pierre, Stoeckle Eberhard, Honoré Charles, Sargos Paul, Sunyach Marie-Pierre, Le Péchoux Cécile, Giraud Antoine, Bellera Carine, Le Loarer François, Italiano Antoine
Clinical and Epidemiological Research Unit, Institut Bergonié, Bordeaux, France.
ISPED, INSERM U1219 Bordeaux Population Health Center, Epicene Team, Bordeaux, France.
BMC Med. 2017 Apr 10;15(1):78. doi: 10.1186/s12916-017-0831-7.
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.
需要开展针对罕见肿瘤患者的精心设计的观察性研究，以改善患者护理、临床研究以及对医疗专业人员的教育。
分析了1990年至2013年间确诊的2225例转移性软组织肉瘤患者的治疗模式、结局和预后因素，这些数据记录在法国肉瘤组织前瞻性维护的数据库中。
全身治疗的中位数为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区域治疗。接受化疗的患者中有一半（n = 810）使用了超说明书用药。平滑肌肉瘤的预后明显优于其他组织学亚型。除平滑肌肉瘤外，三线以上治疗方案的获益非常有限，下次治疗的中位时间（TNT）和总生存期（OS）分别在2.3至3.7个月和5.4至8.5个月之间。TNT与OS高度相关。在多变量分析中，女性、平滑肌肉瘤组织学类型、转移灶的局部区域治疗、纳入临床试验以及一线多药化疗与OS改善显著相关。
阿霉素与第二种药物（如异环磷酰胺）联合使用是一种有效的选择，特别是当预期肿瘤缩小能带来临床获益时。二线治疗失败后，应考虑最佳支持治疗，尤其是对于不符合参加临床试验条件的非平滑肌肉瘤组织学类型的患者。在可行的情况下，转移灶的局部区域治疗应始终纳入治疗策略。在临床研究中，TNT可能是OS的一个有用替代终点。
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