Treatment outcomes and prognostic factors in 47 patients with primary anorectal malignant melanoma in the immune therapy era.
Treatment outcomes and prognostic factors in 47 patients with primary anorectal malignant melanoma in the immune therapy era.
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免疫治疗时代47例原发性肛门直肠恶性黑色素瘤患者的治疗结果和预后因素。
DOI：
10.1007/s00432-022-03933-2
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发表时间：
2022
期刊：
影响因子：
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通讯作者：
Yamazaki N
中科院分区：
文献类型：
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作者：
Ogata D;Tsutsui K;Namikawa K;Moritani K;Nakama K;Jinnai S;Takahashi A;Tsukamoto S;Kanemitsu Y;Yamazaki N
原发性肛门直肠黑色素瘤（ARM）约占所有黑色素瘤的1.2%，占所有粘膜黑色素瘤的16.5%。ARM与疾病进展的最短间隔和最高的转移率相关;然而，ARM的最佳治疗策略仍存在争议。本研究旨在评估ARM的理想手术干预，并确定免疫检查点抑制剂（ICI）的效果。MethodsWe纳入了2011年至2020年在日本国立癌症中心医院接受治疗的47例ARM患者。我们对以下各组进行了生存分析：（i）ARM患者（n= 47）;（ii）初次就诊时可手术的非IV期病例（n= 35）;结果5年总生存率为53.6%，直肠和肛门部位的5年OS差异无统计学意义（50.9%vs.56.7%）。在非IV期亚组中，手术类型（腹会阴切除术或广泛局部切除术）与OS无关（HR 1.85; 95% CI 0.46-7.5;p= 0.39）。在IV期亚组中，ICI治疗组的2年OS为61.4%，而达卡巴嗪方案组为0%（p= 0.048）。我们的研究结果表明，ICI治疗的可用性可能会提高晚期ARM. However，进一步的研究是必要的，以确定反应的临床和分子预测，以改善患者的选择。
PurposePrimary anorectal melanoma (ARM) accounts for approximately 1.2% of all melanomas and 16.5% of all mucosal melanomas. ARM is associated with the shortest interval to disease progression and the highest rate of metastasis; however, optimal therapeutic strategies for ARM remain controversial. This study aimed to assess the ideal surgical intervention for ARM and to determine the effect of immune checkpoint inhibitors (ICI).MethodsWe included 47 patients with ARM treated at the National Cancer Center Hospital in Japan from 2011 to 2020. We performed a survival analysis for each of these groups: (i) patients with ARM (n= 47); (ii) operable non-stage IV cases at initial presentation (n= 35); and (iii) stage IV cases (n= 32).ResultsThe 5-year overall survival (OS) was 53.6%, and the median OS was 78.7 months in patients with ARM. No statistically significant difference in 5-year OS was found between rectal and anal sites (50.9% vs. 56.7%). In the non-stage IV subgroup, the type of surgery (abdominoperineal resection or wide local excision) did not correlate with OS (HR 1.85; 95% CI 0.46–7.5;p= 0.39). In the stage IV subgroup, the 2-year OS of the ICI treatment group was 61.4%, whereas that of the dacarbazine regimen group was 0% (p= 0.048).ConclusionOur ARM prognosis was better than that of previous studies. Our findings suggest that the availability of ICI therapy may improve survival in patients with advanced ARM. However, further research is warranted to identify both the clinical and molecular predictors of response to improve patient selection.