TABLE 1.
Baseline patient characteristics
| Characteristics | Total (N = 29) N (%) | Malignant PEComa N (%) | Epithelioid AML/AML N (%) | LAM N (%) |
|---|---|---|---|---|
| Sex | ||||
| Female | 21 (72.4) | 13 (76.5) | 6 (66.7) | 2 (66.7) |
| Male | 8 (27.6) | 4 (23.5) | 3 (33.3) | 1 (33.3) |
| Age (years) | ||||
| Median, range | 39 (5–71) | 48 (5–71) | 37 (22–69) | 29 (23–39) |
| Histology | ||||
| Malignant PEComa | 17 (58.6) | |||
| Epithelioid AML | 5 (17.2) | |||
| AML | 4 (13.7) | |||
| LAM | 2 (6.9) | |||
| Primary site | ||||
| Kidney | 10 (34.5) | 2 (11.8) | 8 (88.9) | 0 |
| Uterus | 9 (31.0) | 9 (52.9) | 0 | 0 |
| Retroperitoneum | 5 (17.2) | 1 (5.9) | 1 (11.1) | 3 (100.0) |
| Pelvis | 1 (3.4) | 1 (5.9) | 0 | 0 |
| Ovaries | 1 (3.4) | 1 (5.9) | 0 | 0 |
| Colon | 1 (3.4) | 1 (5.9) | 0 | 0 |
| Bladder | 1 (3.4) | 1 (5.9) | 0 | 0 |
| Trunk | 1 (3.4) | 1 (5.9) | 0 | 0 |
| Primary tumor resected | ||||
| Yes | 22 (75.9) | 17 (100) | 5 (55.6) | 0 |
| No | 7 (24.1) | 0 | 4 (44.4) | 3 (100) |
| Metastatic at diagnosis | ||||
| Yes | 7 (24.1) | 5 (29.4) | 2 (22.2) | 0 |
| No | 22 (75.9) | 12 (70.6) | 7 (77.8) | 3 (100) |
| Locally advanced | ||||
| Yes | 13 (44.8) | 9 (52.9) | 1 (11.1) | 3 (100) |
| No | 16 (55.2) | 8 (47.1) | 8 (88.9) | 0 |
| Metastases site | ||||
| Lung | 8 (27.6) | 7 (36.8) | 1 (20.0) | — |
| Abdominal cavity | 4 (13.8) | 3 (15.8) | 1 (20.0) | — |
| Spine | 4 (13.8) | 3 (15.8) | 1 (20.0) | — |
| Liver | 3 (10.3) | 2 (10.5) | 1 (20.0) | — |
| Pelvis | 3 (10.3) | 3 (15.8) | 0 | — |
| Lymph nodes | 2 (6.9) | 1 (5.3) | 1 (20.0) | — |
| Tuberous sclerosis | ||||
| Yes | 8 (27.6) | 2 (11.8) | 4 (44.4) | 2 (66.7) |
| No | 21 (72.4) | 15 (88.2) | 5 (55.6) | 1 (33.3) |
| Ethnicity | ||||
| Caucasian | 18 (62.1) | 12 (70.6) | 4 (44.4) | 2 (66.7) |
| Hispanic/Latino | 7 (24.1) | 4 (23.5) | 3 (33.3) | 0 |
| Asian | 4 (13.8) | 1 (5.9) | 2 (22.2) | 1 (33.3) |
| ECOG | ||||
| 0 | 20 (69.0) | 11 (64.7) | 7 (77.8) | 3 (100) |
| 1 | 9 (31.0) | 6 (35.3) | 2 (22.2) | 0 |
| Primary tumor size (cm) | ||||
| Median (range) | 12 (4.2–31.0) | 10 (7.5–24.0) | 12.1 (5.5–31.0) | 13 (4.2–13.9) |
| Missing | 4 | 4 | 0 | 0 |
| Lines of therapy | ||||
| 1 | 16 (55.2) | 6 (35.3) | 7 (77.8) | 3 (100) |
| 2 | 6 (20.7) | 5 (29.4) | 1 (11.1) | 0 |
| ≥3 | 7 (24.1) | 6 (35.3) | 1 (11.1) | 0 |
| Follow-up time (years) | ||||
| Median (range) | 4.4 (0.4–18.0) | 4.4 (1.3–17.6) | 4.4 (0.4–18.0) | 7.9 (2.8–11.1) |
| Mutational burden (mut/Mb) | ||||
| Median, range | 2.5 (1.0–17.0) | 2.5 (1.0–17.0) | — | — |
| Missing | 21 | 9 | 9 | 3 |
| Status at last follow-up | ||||
| Alive | 19 (65.5) | 9 (52.9) | 7 (77.8) | 3 (100) |
| Deceased | 10 (34.5) | 8 (47.1) | 2 (22.2) | 0 |
Abbreviations: AML, angiomyolipoma; ECOG, Eastern cooperative oncology group; LAM, lymphangioleiomyomatosis; mut/Mb, mutations per megabase; PEComa, perivascular epithelioid cell tumors.