CORR Insights®:在接受更积极局部治疗的硬纤维瘤患者中,PROMIS功能评分较低。

J. Healey
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摘要

在过去的几十年里,硬纤维瘤的治疗已经从手术发展到越来越少的侵入性手术。我认为,这一运动始于一项关于加德纳综合征(家族性腺瘤性息肉病)的研究,该研究发现,舒林酸和吲哚美辛加大剂量维生素C可使肠息肉消退,减少结直肠癌的发生,并减少硬样瘤的发生[10]。虽然选择性雌激素受体抑制剂或低剂量化疗(甲氨蝶呤和vinblastine)的应用越来越广泛[11],但当一项研究发现阴性切缘不能预测剩余的无复发,阳性切缘也不能常规地与局部复发相关联时,手术的影响就更大了[2]。更复杂的靶向治疗已经取得了很高的应答率[4]。一项研究发现,使用索拉非尼治疗组的进展或死亡风险比安慰剂组低87%,尽管在使用活性药物时仍有12%的进展[4]。可以通过评估肿瘤的相对细胞性来监测反应,因为细胞成分的生长和收缩远比相对稳定的纤维成分更为剧烈[4]。该方法已成为硬纤维瘤的一线治疗方法。然而,对靶向药物的反应是有时间依赖性的,可能需要几个月;因此,患者通常需要治疗1至2年。尽管延长了治疗时间,但按照RESIST 1.1标准,患者的部分缓解率为33%。好消息是,在接受这些靶向治疗时,疾病很少进展。然而,治疗的毒性可能是严重的。约20%的患者出现掌足底红觉不良(疼痛的红肿,有时起水泡,通常称为手足综合征),9.4% ~ 18.9%的患者出现高血压[8]。在目前的研究中,Newman等[7]使用患者报告的预后测量信息系统(PROMIS)来评估硬纤维瘤患者的生活质量(QOL)。由于硬纤维瘤是一种局部疾病,治疗可能比疾病更糟糕,因此生活质量和患者满意度是非常重要的考虑结果。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
CORR Insights®: PROMIS Function Scores Are Lower in Patients Who Underwent More Aggressive Local Treatment for Desmoid Tumors.
During the last several decades, treatment for desmoid tumors has evolved away from surgery and toward fewer and lessinvasive operations. I believe this movement started when a study on Gardner’s syndrome (familial adenomatous polyposis) found that sulindac and indomethacin plus high-dose vitamin C caused regression of intestinal polyps, resulting in fewer colorectal cancers, as well as a decrease in desmoid tumors [10]. While selective estrogen-receptor inhibitors or of lowdose chemotherapy (methotrexate and vinblastine) are seeing wider use [11], the pendulum swung further away from surgery when a study found that negative margins did not predict remaining recurrence free, nor were positive margins routinely associated with local recurrence [2]. More-sophisticated, targeted therapies have achieved high response rates [4]. One study found an 87% lower risk of progression or death in a group treated with sorafenib than in the placebo group, although 12% still progressed while on the active drug [4]. Responses can be monitored by assessing the relative cellularity of the tumor, since it is the cellular component that can grow and shrink far more dramatically than the relatively stable fibrous component [4]. This approach has become the first-line treatment for desmoid tumors. However, the responses to targeted agents are timedependent, and can take many months; as a result, patients often are treated for 1 to 2 years. Despite prolonged therapy, patients had partial response rates of 33% by RESIST 1.1 criteria. The favorable news is that disease rarely progressed while on these targeted therapies. However, the toxicity of treatment can be severe. Palmar-plantar erythrodysesthesia (painful redness, swelling and sometimes blistering, often referred to as hand-foot syndrome) occurs in about 20% of patients and hypertension in 9.4% to 18.9% of patients [8]. In the current study, Newman and colleagues [7] use the Patient-Reported Outcomes Measurement Information System (PROMIS) to assess the quality of life (QOL) of patients treated for desmoid tumors. Because desmoid tumors are a local disease, where the treatment can be worse than the disease, QOL and patient satisfaction are very important outcomes to consider.
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