Doxorubicin is a widely used anticancer agent as a first-line treatment for various tumor types, including sarcomas. Its use is hampered by adverse events, among which is the risk of dose dependence. The potential cardiotoxicity, which increases with higher doses, poses a significant challenge to its safe and effective application. To try to overcome these undesired effects, encapsulation of doxorubicin in liposomes has been proposed. Caelyx and Myocet are different formulations of pegylated (PLD) and non-pegylated liposomal doxorubicin (NPLD), respectively. Both PLD and NPLD have shown similar activity compared with free drugs but with reduced cardiotoxicity. While the hand–foot syndrome exhibits a high occurrence among patients treated with PLD, its frequency is notably reduced in those receiving NPLD. In this prospective, multicenter, one-stage, single-arm phase II trial, we assessed the combination of NPLD and ifosfamide as first-line treatment for advanced/metastatic soft tissue sarcoma (STS). Patients received six cycles of NPLD (50 mg/m2) on day 1 along with ifosfamide (3000 mg/m2on days 1, 2, and 3 with equidose MESNA) administered every 3 weeks. The overall response rate, yielding 40% (95% CI: 0.29–0.51), resulted in statistical significance; the disease control rate stood at 81% (95% CI: 0.73—0.90), while only 16% (95% CI: 0.08–0.24) of patients experienced a progressive disease. These findings indicate that the combination of NPLD and ifosfamide yields a statistically significant response rate in advanced/metastatic STS with limited toxicity.
阿霉素作为一线治疗药物，广泛应用于包括肉瘤在内的多种肿瘤治疗。其临床应用受限于不良反应，其中剂量依赖性风险尤为突出。潜在的心脏毒性随剂量增加而加剧，对其安全有效应用构成重大挑战。为克服这些不良效应，研究者提出将阿霉素包载于脂质体的策略。Caelyx与Myocet分别为聚乙二醇化脂质体阿霉素（PLD）与非聚乙二醇化脂质体阿霉素（NPLD）的不同剂型。相较于游离药物，PLD与NPLD在保持相似抗肿瘤活性的同时显著降低了心脏毒性。尽管手足综合征在PLD治疗患者中发生率较高，但NPLD治疗组该症状发生率显著降低。本项前瞻性、多中心、单阶段、单臂II期临床试验评估了NPLD联合异环磷酰胺作为晚期/转移性软组织肉瘤一线治疗的疗效。患者每3周接受一次治疗：第1天静脉注射NPLD（50 mg/m2），并于第1、2、3天联合等剂量美司钠的异环磷酰胺（3000 mg/m2/日），共进行6个周期。总体缓解率达40%（95% CI：0.29–0.51），具有统计学显著性；疾病控制率为81%（95% CI：0.73—0.90），仅16%（95% CI：0.08–0.24）患者出现疾病进展。这些结果表明，NPLD联合异环磷酰胺方案在晚期/转移性软组织肉瘤治疗中能产生具有统计学显著性的缓解率，且毒性反应可控。
肿瘤(癌症)患者之家