背景:腔内UGN-101被批准用于低度非侵袭性上呼吸道尿路上皮癌(UTUC)的治疗。商业化后的研究强调了UGN-101对有必要适应症的患者的益处,这些患者的根治性肾输尿管切除术(RNU)不是可行的选择。
目的:为了描述用途,功效,UGN-101在具有必要的肾脏保存指征的UTUC患者中的安全性,包括高级疾病。
方法:使用来自15个大批量学术和社区中心的多中心集中式注册表,对接受UGN-101并有命令性适应症的患者进行回顾性分析。
方法:我们将必要指征定义为孤立肾患者,慢性肾脏病(CKD)的存在与肾小球滤过率<30毫升/分钟,双边UTUC,不适合或不愿意接受手术摘除的患者。肿瘤特征,疾病进展/复发,不良事件按每个肾脏单位进行记录.
结论:将UGN-101滴入48例适应症患者的52个肾单位(38%),包括29例(56%)孤立肾患者,双侧UTUC的11个肾脏(21%),6例CKD患者(12%),以及6例(12%)不适合或不愿意接受RNU的患者。12个肾脏单位有活检证实的高级乳头状疾病。34%的病例在诱导治疗前肿瘤完全消融,而66%有肿瘤存在。诱导治疗后,17例患者(40%)在输尿管镜检查中没有疾病(NED)的证据,88%的人在中位随访10.8个月时保持了这一状态。在患有高级别疾病的队列中,5例患者(45%)在初始诱导后原发疾病评估时出现NED.不良事件包括肾盂肾炎(8%),输尿管狭窄(8%),贫血(6%),急性肾功能衰竭(4%)。局限性包括回顾性研究设计,缺乏长期随访,和患者选择偏差。
结论:UGN-101在UTUC和紧急适应症患者中的腔内治疗有望成为一种保留肾脏的治疗方式。虽然需要长期随访,这种腔内治疗可能有助于延长RNU的时间,并延缓该共病人群的血液透析发病率.
结果:我们回顾了上尿路癌症患者的结果,以及接受称为UGN-101的凝胶治疗的不允许肾脏切除的其他疾病。我们的结果表明,UGN-101显示出作为保留肾脏治疗的希望。它可能会延迟这些患者需要切除肾脏的时间,并避免与透析相关的负面影响。
Intracavitary UGN-101 is approved for the treatment of low-grade noninvasive upper tract urothelial carcinoma (UTUC). Post-commercialization studies underscore the benefit of UGN-101 administration for patients with imperative indications for whom radical nephroureterectomy (RNU) is not a viable option.
To describe the use, efficacy, and safety of UGN-101 in patients with UTUC with imperative indications for renal preservation, including high-grade disease.
Patients receiving UGN-101 with imperative indications were retrospectively analyzed using a multicenter centralized registry from 15 high-volume academic and community centers.
We defined imperative indications as patients with a solitary kidney, the presence of chronic kidney disease (CKD) with a glomerular filtration rate <30 ml/min, bilateral UTUC, and patients unfit for or unwilling to undergo surgical extirpation. Tumor characteristics, disease progression/recurrence, and adverse events were recorded on a per-renal-unit basis.
UGN-101 was instilled into 52 renal units (38%) in 48 patients for imperative indications, including 29 patients (56%) with a solitary kidney, 11 kidneys (21%) in the setting of bilateral UTUC, six patients (12%) with CKD, and six patients (12%) who were unfit for or unwilling to undergo RNU. Twelve renal units had biopsy-proven high-grade papillary disease. Tumors were completely ablated before induction therapy in 34% of cases, while 66% had tumor present. Following induction therapy, 17 patients (40%) had no evidence of disease (NED) on ureteroscopy, 88% of whom maintained this status at median follow-up of 10.8 mo. In the cohort with high-grade disease, five patients (45%) had NED at initial post-induction primary disease evaluation. Adverse events included pyelonephritis (8%), ureteral stenosis (8%), anemia (6%), and acute renal failure (4%). Limitations include the retrospective study design, the lack of long-term follow up, and patient selection bias.
Intracavitary therapy with UGN-101 in patients with UTUC and imperative indications shows promise as a kidney-sparing treatment modality. While long-term follow-up is needed, this intracavitary treatment may help in prolonging time to RNU and delaying the morbidity of hemodialysis in this comorbid population.
We reviewed results for patients with cancer in the upper urinary tract and an additional condition that would not allow kidney removal who received treatment with a gel called UGN-101. Our results suggest that UGN-101 shows promise as a kidney-sparing treatment. It may delay the time until kidney removal is needed in these patients and avoid the negative effects associated with dialysis.