Real-world survival outcomes and MDM2 prevalence in US patients with metastatic dedifferentiated liposarcoma

美国转移性去分化脂肪肉瘤患者的真实世界生存结果和MDM2患病率

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Abstract

BACKGROUND: Limited data exist regarding dedifferentiated liposarcoma (DDLPS) treatment, biomarker frequency, and clinical outcomes. Additional epidemiological data are needed to inform clinical trial design for testing novel therapeutics. MATERIALS AND METHODS: Retrospective data from a US-based deidentified clinico-genomic database were analyzed for patients treated for metastatic DDLPS between 2011 and 2021. RESULTS: Overall survival (OS), real-world progression-free survival (rwPFS), and time to next treatment (TTNT) were described in the overall cohort (n = 51) and in a subgroup of patients with murine double minute 2 (MDM2) amplification and wild-type tumor protein p53 (TP53 WT) (n = 38, 74.5%). Patients had a median age of 64.8 years, and 62.7% were male. The most common first-line treatment was doxorubicin with olaratumab (23.5%). From time of first-line (1 L) treatment, median OS for the entire cohort and MDM2-amplified, TP53 WT subgroup was 12.6 and 11.7 months, respectively; median rwPFS was 2.5 months for both. Median TTNT was 3.9 months for the full cohort and 4.8 months for the MDM2-amplified, TP53 WT subgroup. CONCLUSIONS: The descriptive analysis here contributes real-world data describing treatment patterns, biomarker status, and clinical outcomes for patients with DDLPS, an aggressive and poorly characterized form of LPS with limited treatment options.

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