Abstract：
Aim: Real-world treatment patterns in tenosynovial giant cell tumor (TGCT) patients remain unknown. Pexidartinib is the only US FDA-approved treatment for TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Objective: To characterize drug utilization and treatment patterns in TGCT patients. Methods: In a retrospective observational study using IQVIA\'s linked prescription and medical claims databases (2018-2021), TGCT patients were stratified by their earliest systemic therapy claim (pexidartinib [N = 82] or non-FDA-approved systemic therapy [N = 263]). Results: TGCT patients treated with pexidartinib versus non-FDA-approved systemic therapies were predominantly female (61 vs 50.6%) and their median age was 47 and 54 years, respectively. Pexidartinib-treated patients had the highest 12-month probability of remaining on treatment (54%); 34.1% of pexidartinib users had dose reduction after their first claim. Conclusion: This study provides new insights into the unmet need, utilization and treatment patterns of systemic therapies for the treatment of TGCT patients.
Treatment patterns in patients with tenosynovial giant cell tumors in the USAThis database study is the first investigation of how drugs are used to treat patients with tenosynovial giant cell tumor (TGCT) in the real world. We researched adult TGCT patients from IQVIA\'s prescription and medical claims databases who started treatment with pexidartinib (N = 82) or other non-US FDA-approved systemic therapies (N = 263). The patients included in this analysis were mostly women (61.0 and 50.6%) and their median age was 47 and 54 years for pexidartinib and other non-FDA-approved systemic therapies, respectively. The patients treated with pexidartinib were most likely to remain on treatment (54.0%) at the end of the first year. Most patients (79.3%) started pexidartinib treatment at a total daily dose of 800 mg/day, as per the product label. Only 34.1% of patients had reduced medication dose during follow-up. Of note, this study found that TGCT patients were treated with other systemic therapies which remain unproven to be safe and effective in medical studies of TGCT. Given the unmet need, and with pexidartinib being the only approved systemic treatment in USA, there is an opportunity for the larger population of adult TGCT patients to benefit from its use. Further research is needed to identify barriers for access to pexidartinib and treatment of TGCT patients.
Treatment patterns in patients with tenosynovial giant cell tumors in the USAThis database study is the first investigation of how drugs are used to treat patients with tenosynovial giant cell tumor (TGCT) in the real world. We researched adult TGCT patients from IQVIA\'s prescription and medical claims databases who started treatment with pexidartinib (N = 82) or other non-US FDA-approved systemic therapies (N = 263). The patients included in this analysis were mostly women (61.0 and 50.6%) and their median age was 47 and 54 years for pexidartinib and other non-FDA-approved systemic therapies, respectively. The patients treated with pexidartinib were most likely to remain on treatment (54.0%) at the end of the first year. Most patients (79.3%) started pexidartinib treatment at a total daily dose of 800 mg/day, as per the product label. Only 34.1% of patients had reduced medication dose during follow-up. Of note, this study found that TGCT patients were treated with other systemic therapies which remain unproven to be safe and effective in medical studies of TGCT. Given the unmet need, and with pexidartinib being the only approved systemic treatment in USA, there is an opportunity for the larger population of adult TGCT patients to benefit from its use. Further research is needed to identify barriers for access to pexidartinib and treatment of TGCT patients.
摘要：
目的：腱鞘膜巨细胞瘤（TGCT）患者的真实世界治疗模式仍然未知。Pexidartinib是唯一的美国FDA批准的TGCT治疗与严重的发病率或功能限制相关，不适合通过手术改善。目的：探讨TGCT患者的药物利用和治疗方式。方法：在一项回顾性观察研究中，使用IQVIA的关联处方和医疗索赔数据库(2018-2021年)，TGCT患者根据其最早的全身治疗要求（帕西达替尼[N=82]或非FDA批准的全身治疗[N=263]）进行分层。结果：接受帕西达替尼治疗的TGCT患者主要为女性（61vs50.6%），中位年龄为47岁和54岁。分别。接受帕西达替尼治疗的患者12个月的剩余治疗概率最高（54％）；34.1％的帕西达替尼使用者在首次申请后剂量减少。结论：这项研究为未满足的需求提供了新的见解，TGCT患者全身治疗的利用和治疗模式。
该数据库研究是关于如何在现实世界中使用药物治疗腱鞘膜巨细胞瘤（TGCT）患者的首次调查。我们从IQVIA的处方和医疗索赔数据库中研究了成年TGCT患者，这些患者开始接受帕西达替尼（N=82）或其他非美国FDA批准的全身治疗（N=263）。这项分析中包括的患者大多是女性（61.0％和50.6％），他们的中位年龄为47岁和54岁，接受帕西达替尼和其他非FDA批准的全身治疗。分别。接受帕西达替尼治疗的患者在第一年结束时最有可能继续治疗(54.0%)。大多数患者（79.3%）开始帕西达替尼治疗的总剂量为800毫克/天，根据产品标签。只有34.1%的患者在随访期间药物剂量减少。值得注意的是，这项研究发现,TGCT患者接受了其他全身性治疗,这些治疗在TGCT的医学研究中仍未被证明是安全有效的.鉴于未满足的需求，帕西达替尼是美国唯一被批准的全身治疗药物，更多的成人TGCT患者有机会从其使用中获益.需要进一步的研究来确定获得帕西达替尼和TGCT患者治疗的障碍。
该数据库研究是关于如何在现实世界中使用药物治疗腱鞘膜巨细胞瘤（TGCT）患者的首次调查。我们从IQVIA的处方和医疗索赔数据库中研究了成年TGCT患者，这些患者开始接受帕西达替尼（N=82）或其他非美国FDA批准的全身治疗（N=263）。这项分析中包括的患者大多是女性（61.0％和50.6％），他们的中位年龄为47岁和54岁，接受帕西达替尼和其他非FDA批准的全身治疗。分别。接受帕西达替尼治疗的患者在第一年结束时最有可能继续治疗(54.0%)。大多数患者（79.3%）开始帕西达替尼治疗的总剂量为800毫克/天，根据产品标签。只有34.1%的患者在随访期间药物剂量减少。值得注意的是，这项研究发现,TGCT患者接受了其他全身性治疗,这些治疗在TGCT的医学研究中仍未被证明是安全有效的.鉴于未满足的需求，帕西达替尼是美国唯一被批准的全身治疗药物，更多的成人TGCT患者有机会从其使用中获益.需要进一步的研究来确定获得帕西达替尼和TGCT患者治疗的障碍。
