Table 1.
Patient characteristics and clinical course.
| No. | Age (months) | ♀♂ | Diagnosis | Site | Biopsy | SIR duration (months) | SIR mean trough level (ng/ml) | VCR duration (months) | Time to response (months) | Other interventions* | Outcome | Follow-up (months) |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 0 | ♀ | LM | Cervicofacial | – | 33 | 5.78 | 0.75 | – | PR | Ongoing | |
| 2 | 2.5 | ♂ | LM | Cervicofacial | – | 11 | 5.53 | 0.5 | – | PR | Ongoing | |
| 3 | 0.75 | ♀ | CCLA | Lung | – | 3.5 | 12.01 | n/a | Multiple | PD | n/a | |
| 4 | 0 | ♀ | CLAPO | Cervicofacial, skull base | – | 14 | 7.53 | 1 | OK432, tracheostomy | PR | 18 | |
| 5 | 0 | ♀ | CLOVES | Thorax | – | 9 | 12.88 | 0.25 | – | PR | Ongoing | |
| 6 | 4.2 | ♂ | GSD | Skull base | + | 58 | 8.60 | 0.5 | Pamidronate | PR | Ongoing | |
| 7 | 127.2 | ♂ | GLA | Lung, pericardium | + | 50 | 9.63 | 2 | Steroids | PR | Ongoing | |
| 8 | 179 | ♀ | GLA | Lung, spleen, retroperit. | + | 9 | 5.38 | 2 | – | PR | Ongoing | |
| 9 | 37.2 | ♂ | LVM | Orbita | – | 36 | 8.00 | 1 | – | PR | 10 | |
| 10 | 218.4 | ♀ | ALVM | Cervicofacial, skull base | + | 30 | 9.44 | 1.5 | 2 × Surgery | PR | Ongoing | |
| 11 | 5 | ♂ | KHE | Neck, mediastinum | –*** | 27 | 9.03 | 9 | 4.5 | Pericardial fenestration | PR | 5 |
| 12 | 14 | ♂ | KHE+KMP | Thigh | + | 16 | 9.81 | 3 | 0.75 | Embolization | CR | 64 |
| 13 | 5 | ♀ | KHE | Liver | + | 24 | 9.82 | 3 | 0.5 | Multiple | PR | 51 |
| 14 | 3 | ♀ | KHE+KMP | Cervical | – | 16 | 6.42 | 6 | 1 | – | CR | 8 |
| 15 | 2 | ♀ | KHE +KMP | Thigh | + | 33 | 9.05 | 6 | 1.5 | – | CR | 15 |
| 16 | 134.4 | ♂ | EHE | Liver, lung | + | 65 | 7.48 | 3 | – | PR | Ongoing | |
| Median | 4.6 | 6 | 1 | |||||||||
| Mean** | 27.2 | 8.52 | ||||||||||
| N (%) | 8/16 (50%) | 8/16 (50%) |
Age, age at the beginning of sirolimus therapy; SIR, sirolimus therapy; VCR, vincristine intensification; LM, lymphatic malformation; CCLA, central conducting lymphatic anomaly; CLAPO, lower lip capillary malformation/face and neck LM asymmetry and partial overgrowth-syndrome; CLOVES, congenital lipomatous overgrowth, vascular malformations, epidermal nevi, skeletal and spinal abnormalities-syndrome; GSD, Gorham–Stout disease; GLA, generalized lymphatic anomaly; LVM, lymphatic-venous malformation; ALVM, arterio-lymphatic-venous malformation; KHE, kaposiform hemangioendothelioma; KMP, Kasabach–Merritt phenomenon; EHE, epithelioid hemangioendothelioma.
Interventions performed prior to or during the course of sirolimus therapy.
Mean was calculated for the parameters with normal distribution.
Biopsy attempted but failed to deliver a tissue sample due to a severe life-threatening intraoperative bleeding.