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

Jennifer Hammer; Emmanuel Seront; Steven Duez; Sophie Dupont; An Van Damme; Sandra Schmitz; Claire Hoyoux; Caroline Chopinet; Philippe Clapuyt; Frank Hammer; Miikka Vikkula; Laurence M. Boon

doi:10.1186/s13023-018-0934-z

Orphanet Journal of Rare Diseases

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%)

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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