FIRST AUTHOR | DESIGN | YEAR | POPULATION | INTERVENTION | COMPARISON | PRIMARY OUTCOME | |
---|---|---|---|---|---|---|---|
RANDOMIZED | Mükke | Cross-over [48] | 2020 | Venous malformation Adults and children | Physiotherapy (compression stockings) | No treatment | Change in volume of the malformation measured non-invasively by MRI. |
Novartis | Delayed-start [49] | 2020 | PROS Adults and children | Enteral drug (alpelisib) | Placebo | Change in the target lesion volumes assessed by MRI. | |
Maruani | Randomized placebo phase [43] | 2019 | Lymphatic malformations Adults and children | Topical drug (sirolimus) | No treatment | Evaluation of global severity of the lingual microcystic lymphatic malformation using Physical Global Assessment (PGA). | |
Leducq | Within-person [71] | 2019 | Lymphatic malformations Adults and children | Topical drug (sirolimus) | Placebo | Evaluation of cutaneous microcystic lymphatic malformation versus topical vehicle using PGA. | |
Maruani | Randomized placebo phase [85] | 2018 | Vascular malformation Children | Enteral drug (sirolimus) | No treatment | Change in the volume of the vascular malformation seen on MRI. | |
Smith | Delayed-start [103] | 2009 | Lymphatic malformations Children | Parenteral drug (picibanil) | No treatment | Change in volume/size of the malformation evaluated by CT or MRI. | |
NON-RANDOMIZED | te Loo | Challenge–dechallenge–rechallenge [74] | 2019 | Vascular malformation Adults and children | Enteral drug (sirolimus) | No treatment | Evaluation of the quality of life measured by TAPQOL, PedsQL and Research and development Rand-36. Evaluation of the pain using VAS score and NRS score. |
Parker | Observational run-in period [81] | 2019 | PROS Adults and children | Enteral drug (sirolimus) | No treatment | Percent change in unaffected and affected fibrofatty tissue measured by DXA and MRI scan. | |
Hammer | Cross-overa [65] | 2018 | Vascular malformation Adults and children | Enteral drug (sirolimus) | Patient’s long term history (Surgery, interventional radiology etc.) | Physical, functional, biological, radiological and quality of life evaluations. | |
Weitz-Tuoretmaa | Historical control group [73] | 2017 | Venous malformation Adults and children | Sclerotherapy (polidocanol) | Sclerotherapy (ethanol) | Severity evaluation of symptoms by a self-evaluation questionnaire. | |
James | Historical control group [66] | 2011 | Venous malformation Adults and children | Sclerotherapy | Surgery | Evaluation of lesions dimensions by MRI, surgical parameters, and duration of hospital stay. |