TABLE 3.
Cox PH analysis for PFS from first-line treatment
| Univariable | Multivariable | |||||
|---|---|---|---|---|---|---|
| Variables | N Episodes (%) | HR (95% CI) | P (Cox-Wald) | P (log-rank) | HR (95% CI) | P (Cox-Wald) |
| Sex | ||||||
| Male | 8 (27.6) | 0.5 (0.1–2.5) | 0.4 | 0.4 | ||
| Female | 21 (72.4) | — | — | |||
| Primary site | ||||||
| Uterine | 9 (31.0) | 2.5 (0.7–8.5) | 0.1 | 0.1 | ||
| Extrauterine | 20 (68.9) | — | — | |||
| Age, years (median, range) | 39 (5–71) | 1.1 (1.0–1.2) | 0.001 | 0.0001 | 1.1 (1.0–1.1) | 0.006 |
| Metastatic at diagnosis | ||||||
| Yes | 7 (24.1) | 2.7 (0.7–10.3) | 0.1 | 0.1 | ||
| No | 22 (75.9) | |||||
| TSC mutation | ||||||
| TSC2 mutated | 5 (17.2) | 0.5 (0.08–3.6) | 0.5 | 0.1 | ||
| TSC1/TSC2 Wild type | 20 (68.9) | 0.2 (0.04–1.2) | 0.07 | |||
| TSC1 mutated | 4 (13.8) | — | — | |||
| TP53 | ||||||
| Mutated | 5 (17.2) | 2.8 (0.8–10.1) | 0.1 | 0.09 | ||
| Wild type | 24 (82.8) | — | — | |||
| Lines of therapy | ||||||
| 1 | 17 (58.6) | — | — | — | — | |
| 2 | 5 (17.2) | 18.4 (3.1–109.7) | 0.001 | 0.0004 | 3.7 (0.4–31.3) | 0.2 |
| ≥3 | 7 (24.1) | 4.6 (1.0–20.7) | 0.04 | 5.2 (0.3–89.8) | 0.2 | |
| TFE3 | ||||||
| Negative | 23 (79.3) | — | — | |||
| Positive | 6 (20.7) | 2.0 (0.5–8.0) | 0.3 | 0.3 | ||
| Histology | ||||||
| Malignant PEComa | 17 (58.6) | 9.6 (1.2–76.8) | 0.03 | 0.01 | 4.9 (0.4–61.2) | 0.2 |
| LAM/AML/Epithelioid AML | 12 (41.4) | — | — | — | — | |
| History of tuberous sclerosis | ||||||
| Yes | 8 (27.6) | 0.3 (0.04–2.7) | 0.3 | 0.3 | ||
| No | 21 (72.4) | — | — | |||
| Treatment (first line) | ||||||
| mTOR inhibitors | 24 (82.8) | 0.7 (0.2–2.9) | 0.7 | 0.7 | 0.5 (0.03–8.9) | 0.6 |
| Chemotherapy | 5 (17.2) | — | — | — | — | |
| Adjuvant treatment | ||||||
| Yes | 5 (17.2) | 1.6 (0.4–6.1) | 0.5 | 0.5 | 0.2 (0.01–4.2) | 0.3 |
| No | 24 (82.8) | — | ||||
Abbreviations: AML, angiomyolipoma; LAM, lymphangioleiomyomatosis; mTOR, mammalian target of rapamycin; PEComa, perivascular epithelioid cell tumors.