Understanding the Impact of Belzutifan on Treatment Strategies for Patients with VHL.
Understanding the Impact of Belzutifan on Treatment Strategies for Patients with VHL.
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DOI：
10.15586/jkcvhl.v9i3.245
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发表时间：
2022
影响因子：
1.6
通讯作者：
中科院分区：
文献类型：
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Belzutifan最近被批准用于治疗Von Hippel-Lindau病(VHL)。考虑到复发治疗的发病率，减少或消除手术需求的系统治疗一直是人们期待已久的。在这里，我们试图通过调查美国的VHL肾癌专家来了解未来的使用情况。VHL联盟(VHL)临床咨询委员会成员开发的一份调查报告被分发给VHL联盟和国家综合癌症网络(NCCN)中心的肾癌提供者。调查是在安全的网络平台上进行的。共有来自29家机构的60名受访者参与。泌尿科医生(50%)和内科肿瘤学家(43%)代表了大多数参与者。大多数受访者(98%)预计Belzutifan的批准将显著改变目前的治疗格局。大多数人报告说治疗应该是持续的(76%)。不同专业的医生对处方贝珠替凡的意愿存在差异(38%的泌尿科医生与91%的内科肿瘤科医生(P=0.02))。在患有3厘米以上肾脏肿瘤的患者中，36%的人仍然建议进行监测，而36%的人会建议使用贝珠替芬来防止生长。在那些多灶性肾脏病变并在Belzutifan上有孤立肿瘤生长的患者中，50%的患者将继续仅对该部位进行治疗。总而言之，VHL肾癌专家预计，在Belzutifan的批准下，治疗模式将发生转变。提供者的角色可能会随着远离手术管理的移动而改变。关于治疗适应症，如何时开始治疗和如何最好地抢救，意见分歧很大，因此专家之间的合作努力可能有助于制定新的临床指南。
Belzutifan was recently approved for the management of Von Hippel–Lindau disease (VHL). Given the morbidity of recurrent treatment, systemic therapy to reduce or eliminate the need for surgery has been long-awaited. Herein, we sought to gain insight about future utilization by surveying VHL kidney cancer experts in the United States. A survey developed by members of the VHL Alliance (VHLA) Clinical Advisory Council was distributed to kidney cancer providers at VHLA and National Comprehensive Cancer Network (NCCN) centers. Surveys were administered on a secure web-based platform. A total of 60 respondents from 29 institutions participated. Urologists (50%) and medical oncologists (43%) represented the majority of participants. The majority (98%) of respondents anticipated that belzutifan’s approval would significantly change the current treatment landscape. Most reported that therapy should be continuous (76%). There was a difference in willingness to prescribe belzutifan by specialty (38% of urologists vs 91% of medical oncologists (P = 0.02)). In individuals with renal tumors <3 cm, 36% would still recommend surveillance, while 36% would initiate belzutifan to prevent growth. In those with multifocal renal lesions and growth of a solitary tumor on belzutifan, 50% would proceed with only treatment of that site. In conclusion, VHL kidney cancer specialists anticipate a paradigm shift with the approval of belzutifan. Provider roles may change with movement away from surgical management. Opinions on treatment indications, such as when to initiate therapy and how to best salvage, vary widely and therefore collaborative efforts among experts may assist in the development of new clinical guidelines.