TABLE 4.
Cox PH analysis for PFS for all treatment episodes combined, regardless of line of therapy
| Univariable | Multivariable | |||||
|---|---|---|---|---|---|---|
| Variables | N Episodes (%) | HR (95% CI) | P (Cox-Wald) | P (log-rank) | HR (95%CI) | P (Cox-Wald) |
| Sex | ||||||
| Male | 11 (18.6) | 0.5 (0.1–1.6) | 0.2 | 0.3 | ||
| Female | 48 (81.4) | — | — | |||
| Primary site | ||||||
| Uterine | 25 (42.4) | 1.9 (0.9–3.9) | 0.1 | 0.09 | ||
| Extrauterine | 34 (57.6) | — | — | |||
| Age, years (median, range) | 39 (5–71) | 1.1 (1.0–1.1) | 0.004 | 0.003 | 1.1 (1.0–1.1) | 0.02 |
| Metastatic at diagnosis | ||||||
| Yes | 13 (22.0) | 1.9 (0.7–5.4) | 0.2 | 0.2 | ||
| No | 46 (78.0) | |||||
| TSC mutation | ||||||
| TSC2 mutated | 10 (16.9) | 0.7 (0.2–2.9) | 0.6 | 0.4 | ||
| TSC1/TSC2 wild type | 38 (64.5) | 0.5 (0.1–1.5) | 0.2 | |||
| TSC1 mutated | 11 (18.6) | — | — | |||
| TP53 | ||||||
| Mutated | 10 (16.9) | 2.3 (0.8–6.1) | 0.1 | 0.09 | ||
| Wild type | 49 (83.1) | — | — | |||
| Lines of therapy | ||||||
| 1 | 18 (30.6) | — | — | — | — | |
| 2 | 10 (16.9) | 13.8 (3.4–56.4) | 0.0003 | 0.00007 | 2.9 (0.3–25.0) | 0.3 |
| ≥3 | 31 (52.5) | 7.9 (2.3–27.3) | 0.0009 | 7.0 (1.1–44.2) | 0.04 | |
| TFE3 | ||||||
| Negative | 46 (77.9) | — | — | |||
| Positive | 13 (22.1) | 2.7 (0.9–7.7) | 0.06 | 0.05 | ||
| Frailty (Patient ID) | — | — | — | — | — | |
| Histology | ||||||
| Malignant PEComa | 42 (71.2) | 5.6 (1.7–18.7) | 0.005 | 0.002 | 11.4 (1.4–96.1) | 0.02 |
| LAM/AML/Epithelioid AML | 17 (28.8) | — | — | — | — | |
| History of tuberous sclerosis | ||||||
| Yes | 11 (18.6) | 0.3 (0.04–2.7) | 0.3 | 0.3 | ||
| No | 48 (81.4) | — | — | |||
| Treatment (all lines) | ||||||
| mTOR inhibitors | 40 (67.8) | 0.8 (0.3– 2.3) | 0.7 | 0.6 | 0.2 (0.04–1.2) | 0.07 |
| Othera | 5 (8.4) | 1.9 (0.4–8.6) | 0.4 | 0.3 (0.04–2.9) | 0.3 | |
| ICI | 5 (8.4) | 1.5 (0.2–13.7) | 0.7 | 0.2 (0.02–3.3) | 0.3 | |
| Chemotherapy | 9 (15.4) | — | — | — | — | |
| Adjuvant treatment | ||||||
| Yes | 7 (11.9) | 0.3 (0.07–1.0) | 0.051 | 0.6 | 0.04 (0.007–0.3) | 0.0007 |
| No | 52 (88.1) | — | ||||
Abbreviations: AML, angiomyolipoma; LAM, lymphangioleiomyomatosis; mTOR, mammalian target of rapamycin; ICI: Immune check-point inhibitors; PEComa, perivascular epithelioid cell tumors.
a Other: olaparib (n = 1), pazopanib (n = 1), pazopanib-everolimus (n = 1), anastrozole (n = 1), and levantinib-everolimus (n = 1).