Pancreatic INI1-deficient undifferentiated rhabdoid carcinoma achieves complete clinical response on gemcitabine and nab-paclitaxel following immediate progression on FOLFIRINOX: a case report
Pancreatic INI1-deficient undifferentiated rhabdoid carcinoma achieves complete clinical response on gemcitabine and nab-paclitaxel following immediate progression on FOLFIRINOX: a case report
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DOI：
10.21037/jgo-20-478
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发表时间：
2021-04-01
影响因子：
2.1
通讯作者：
Fisher, George A.
中科院分区：
文献类型：
--
作者：
King, Daniel A.;Rahalkar, Smruti;Fisher, George A.
引言：INI 1缺陷型未分化横纹肌样癌是一种罕见的胰腺癌，其最佳治疗方法尚不清楚。胰腺导管腺癌是胰腺癌最常见的组织学类型，在晚期情况下采用联合化疗进行治疗，这一策略得到了有力研究的有力证据支持。在体能状态良好的患者中，一线治疗通常包括三药方案FOLFIRINOX，以及吉西他滨与nabpaclitaxel的组合，通常毒性低于三药方案，保留用于二线治疗。鉴于缺乏描述罕见胰腺癌患者治疗结局的已发表报告，通常采用与胰腺导管腺癌相同的治疗方法。观察结果：该病例描述了1例转移性胰腺INI 1缺陷型未分化横纹肌样癌患者，该患者主要对FOLFIRINOX治疗耐药，但随后对吉西他滨联合nab-紫杉醇实现了立即、显著和持续的缓解。结论：鉴于缺乏关于INI 1缺陷型胰腺未分化横纹肌样癌最佳治疗的数据，以及吉西他滨与白蛋白结合型紫杉醇联合治疗的特殊疗效，本病例报告突出了一个令人惊讶且可能提供信息的轶事。需要进一步的研究来证实本报告中观察到的反应，这些反应综合在一起可能会强烈影响这种罕见恶性肿瘤的一线治疗选择。鉴于在任何一个机构中难以获得足够数量的这些罕见组织学，在研究罕见胰腺恶性肿瘤的结局方面，多机构合作可能是必不可少的。
Introduction: INI1-deficient undifferentiated rhabdoid carcinoma is a rare pancreatic carcinoma for which the optimal treatment is unknown. Pancreatic ductal adenocarcinoma, the most common histology of pancreas cancer, is treated with combination chemotherapy in the advanced setting, a strategy supported by strong evidence in well powered studies. In patients with excellent performance status, first-line treatment usually consists of the three-drug regimen FOLFIRINOX, with the combination of gemcitabine with nabpaclitaxel, typically less toxic than the three-drug regimen, reserved for second-line therapy. Given the lack of published reports describing treatment outcomes for patients with rare forms of pancreatic cancer, the same treatment approach used for pancreatic ductal adenocarcinoma is typically employed. Observation: This case describes a patient with metastatic pancreatic INI1-deficient undifferentiated rhabdoid carcinoma who was primarily resistant to FOLFIRINOX therapy but who then achieved an immediate, marked and sustained response to gemcitabine with nab-paclitaxel. Conclusion: Given the lack of data informing on optimal management of INI1-deficient pancreatic undifferentiated rhabdoid carcinoma, and the exceptional response achieved by gemcitabine with nab-paclitaxel, this case report highlights a surprising and potentially informative anecdote. Additional studies are needed to confirm responses observed in this report which when taken together may strongly influence first-line therapy choice for this rare malignancy. Given the difficult in acquiring sufficient numbers of these rare histologies in any one institution, multi-institution collaboration in studying outcomes of rare pancreatic malignancies is likely essential.