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Table 2 Efficacy after 1 year of sirolimus treatment

From: Sirolimus is efficacious in treatment for extensive and/or complex slow-flow vascular malformations: a monocentric prospective phase II study

Efficacy at 12 months FU (n = 16) LM (n = 6) GLA (n = 2) VM (n = 4) CVM (n = 1) KTS (n = 2) PHTS (n = 1) Total
Physical examination (n = 14) n = 4/5 (80%) n = 2/2 (100%) n = 3/3 (100%) n = 0 n = 1/2 (50%) n = 1/1 (100%) n = 11/14 (78%)
Organ or mobility dysfunction (n = 15) n = 6/6 (100%) n = 2/2 (100%) n = 4/4 (100%) n = 1/1 (100%) n = 1/1 (100%) n = 1/1 (100%) n = 15/15 (100%)
Pain (n = 10) n = 3/3 (100%) n = 1/1 (100%) n = 4/4 (100%) n = 0 n = 1/1 (100%) n = 1/1 (100%) n = 10/10 (100%)
Bleeding (n = 4) n = 1/1 (100%) n = 0 n = 1/1 (100%) n = 1/1 (100%) n = 1/1 (100%) n = 0 n = 4/4 (100%)
Oozing (n = 4) n = 1/1 (100%) n = 1/1 (100%) n = 0 n = 0 n = 2/2 (100%) n = 0 n = 4/4 (100%)
Repetitive infections (n = 10) n = 6/6 (100%) n = 2/2 (100%) n = 0 n = 0 n = 2/2 (100%) n = 0 n = 10/10 (100%)
Quality of life (n = 16)
- No amelioration n = 0 n = 0 n = 0 n = 0 n = 0 n = 0 n = 0 (0%)
- Moderate amelioration n = 3/6 (50%) n = 0 N = 1/4 (25%) n = 0 n = 2/2 (100%) n = 0 n = 6/16 (37.5%)
- Strong amelioration n = 3/6 (50%) n = 2/2 (100%) n = 3/4 (75%) n = 1/1 (100%) n = 0 n = 1/1 (100%) n = 10/16 (62.5%)
Reduced coagulopathy (n = 8) n/a n/a n = 4 /4 (100%) n = 1/1 (100%) n = 2 /2 (100%) N = 1/1 (100%) n = 8/8 (100%)
MRI (T1,T2 Fat Sat,STIR) (n = 16)
- Progression n = 1/6 (17%) n = 1/2 (50%) n = 1/4 (25%) n = 0 n = 0 n = 0 n = 3/16 (19%)
- Status quo n = 2/6 (33%) n = 0 n = 2/4 (50%) n = 0 n = 1/2 (50%) n = 1/1 (100%) n = 6/16 (37%)
- Amelioration n = 3/6 (50%) n = 1/2 (50%) n = 1/4 (25%) n = 1/1 (100%) n = 1/2 (50%) n = 0 n = 7/16 (44%)
ITK-SNAP 3–0 (n = 9)
- Volume augmentation n = 0 n = 0 n = 0 n = 0 n = 0 n = 0 n = 0/9 (0%)
- No volume modification n = 0 n = 0 n = 1/4 (25%) n = 0 n = 0 n = 0 n = 1/9 (11%)
- Volume reduction n = 2/2 (100%) n = 1/1 (100%) n = 3/4 (75%) n = 1/1 (100%) n = 1/1 (100%) n = 0 n = 8/9 (89%)
  1. FU Follow-Up, LM Lymphatic Malformation, GLA Generalized Lymphatic Anomaly, VM Venous Malformation, CVM Capillary Venous Malformation, KTS Klippel-Trenaunay Syndrome, PHTS PTEN Hamartoma Tumour Syndrome, n/a not applicable
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172