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醛氧柔红霉素治疗转移性孤立性纤维性肿瘤的疗效及心脏安全性

Efficacy and cardiac safety of aldoxorubicin in metastatic solitary fibrous tumour.

作者信息

Vosylius Karolina, Price Gareth, Napolitano Andrea, Benson Charlotte, Fotiadis Nicos, Thway Khin, Christien Li Ka Hou, Jones Robin L

机构信息

Sarcoma Trials Unit, Royal Marsden NHS Foundation Trust, London, UK.

Department of Oncology, Oxford University, Oxford, UK.

出版信息

Rare Tumors. 2025 Aug 7;17:20363613251353649. doi: 10.1177/20363613251353649. eCollection 2025.

Abstract

Solitary fibrous tumours (SFT) are very rare mesenchymal neoplasms. While surgery remains a standard treatment for localised disease, effective and long term treatment options for metastatic disease are lacking, making the use of aldoxorubicin a novel and promising systemic treatment in SFTs. We present a 30-year-old male who underwent surgical resection for a solitary fibrous tumour of the right leg. Postoperative imaging revealed metastatic disease in the liver and left upper quadrant. He was initially treated with pazopanib but experienced disease progression after 24 weeks. The patient was then enrolled on a phase III trial evaluating aldoxorubicin for advanced soft tissue sarcomas and received 350 mg/m (260 mg/m doxorubicin equivalent) intravenously every 21 days, cumulative dose being 9100 mg/m. Treatment was well tolerated, with manageable toxicities including alopecia, leukopenia, mucositis, and grade 3 neutropenia requiring G-CSF support. Notably, serial echocardiograms showed no evidence of cardiotoxicity, with a preserved ejection fraction (56-65%). He completed 26 cycles with stable disease, followed by a 7-month treatment break before receiving compassionate-use aldoxorubicin. Disease stability persisted for 6 months until progression, which was treated with radiotherapy. Three months later, systemic progression led to treatment discontinuation. This case illustrates the favourable cardiac safety profile of aldoxorubicin and efficacy in solitary fibrous tumours.

摘要

孤立性纤维瘤(SFT)是非常罕见的间叶性肿瘤。虽然手术仍然是局限性疾病的标准治疗方法,但对于转移性疾病缺乏有效且长期的治疗选择,这使得阿霉素成为SFT中一种新颖且有前景的全身治疗药物。我们报告一名30岁男性,因右腿孤立性纤维瘤接受了手术切除。术后影像学检查显示肝脏和左上象限有转移性疾病。他最初接受帕唑帕尼治疗,但24周后病情进展。然后该患者参加了一项评估阿霉素治疗晚期软组织肉瘤的III期试验,每21天静脉注射350mg/m²(相当于阿霉素260mg/m²),累积剂量为9100mg/m²。治疗耐受性良好,毒性可控,包括脱发、白细胞减少、粘膜炎以及需要粒细胞集落刺激因子支持的3级中性粒细胞减少。值得注意的是,系列超声心动图显示无心脏毒性证据,射血分数保持在56 - 65%。他完成了26个周期,病情稳定,随后在接受同情用药阿霉素之前有7个月的治疗间歇期。疾病稳定持续了6个月直至进展,进展后接受了放射治疗。三个月后,全身进展导致治疗中断。该病例说明了阿霉素在孤立性纤维瘤中的良好心脏安全性和疗效。

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