Kroiss Matthias, Deutschbein Timo, Schlötelburg Wiebke, Ronchi Cristina Lucia, Hescot Ségolène, Körbl Daniela, Megerle Felix, Beuschlein Felix, Neu Bruno, Quinkler Marcus, Baudin Eric, Hahner Stefanie, Heidemeier Anke, Fassnacht Martin
Department of Internal Medicine I, Endocrine and Diabetes Unit, University Hospital Würzburg, University of Würzburg, Germany.
Department of Radiology, University Hospital Würzburg, University of Würzburg, Germany.
Exp Clin Endocrinol Diabetes. 2019 Oct;127(9):578-584. doi: 10.1055/a-0747-5571. Epub 2018 Nov 14.
Adrenocortical carcinoma (ACC) is a rare malignancy with a dismal prognosis. In advanced stages, tumour control by mitotane and cytotoxic chemotherapy is often temporary and salvage treatments are warranted.
Retrospective cohort study of participants in the prospective European Networks for the Study of Adrenal Tumours (ENSAT) registry. Main outcome measures were best response during treatment, progression-free survival (PFS), both measured according to RECIST 1.1 by two blinded radiologists, and overall survival (OS).
Twenty-seven patients (13 males; median age 44.1 years) progressing after mitotane and a median of 4 further systemic treatments were included. Thalidomide was administered as tolerated with a starting dose of 50 mg and target dose of 200 mg /d. The median interval between treatment initiation and first imaging was 10.5 (4.4-17.5) weeks. The best response to treatment was stable disease (SD, n=2) and progressive disease (n=25), with a median PFS of 11.2 weeks and a median OS of 36.4 weeks. The first patient with SD discontinued treatment due to mild epistaxis and diarrhea after 22.3 weeks. The second patient had SD at the second treatment evaluation after 25.2 weeks and continued thalidomide but then had clinical progression and deceased after 54.3 weeks. In general, thalidomide induced only mild or moderate adverse effects (mainly fatigue and gastrointestinal complaints).
Thalidomide was overall well tolerated but resulted in disease control in only 2/27 (7.4%) patients. In the absence of predictive response markers, thalidomide should only be considered in exceptional cases as a salvage therapy in ACC.
肾上腺皮质癌（ACC）是一种罕见的恶性肿瘤，预后较差。在晚期，米托坦和细胞毒性化疗对肿瘤的控制通常是暂时的，因此需要进行挽救性治疗。
对前瞻性欧洲肾上腺肿瘤研究网络（ENSAT）登记处的参与者进行回顾性队列研究。主要结局指标为治疗期间的最佳反应、无进展生存期（PFS），均由两名盲法放射科医生根据RECIST 1.1进行测量，以及总生存期（OS）。
纳入了27例患者（13例男性；中位年龄44.1岁），这些患者在接受米托坦治疗后病情进展，且中位接受了另外4次全身治疗。沙利度胺根据耐受情况给药，起始剂量为50mg，目标剂量为200mg/d。从开始治疗到首次成像的中位间隔时间为10.5（4.4 - 17.5）周。治疗的最佳反应为疾病稳定（SD，n = 2）和疾病进展（n = 25），中位PFS为11.2周，中位OS为36.4周。首例出现疾病稳定的患者在22.3周后因轻度鼻出血和腹泻停止治疗。第二例患者在25.2周后的第二次治疗评估时出现疾病稳定，并继续使用沙利度胺，但随后出现临床进展，在54.3周后死亡。总体而言，沙利度胺仅引起轻度或中度不良反应（主要是疲劳和胃肠道不适）。
沙利度胺总体耐受性良好，但仅在2/27（7.4%）的患者中实现了疾病控制。在缺乏预测反应标志物的情况下，沙利度胺仅应在特殊情况下作为ACC的挽救性治疗考虑。
