Treatment of voluminous and complicated superficial slow-flow vascular malformations with sirolimus (PERFORMUS): protocol for a multicenter phase 2 trial with a randomized observational-phase design

Table 1 Schedule of study interventions, outcome data collection and follow-up visits

	Screening	Inclusion	Switch (start sirolimus)	Switch + M1 ± 15 days	Follow-up during sirolimus treatment: every 2 months ±15 days	End of study
	J-30/J0*	J0	Between month 4 and 8**	Between month 5 and 9**	Between month 7 and 11**	Month 12***
Information	x	x
Inclusion/exclusion criteria	x	x	x	x	x
Medical history and demographics	x	x
Consent		x
Randomization		x
MRI	x¹		x			x
Physical examination		x	x	x	x	x
General laboratory tests		x²	x³	x⁴	x⁴
Coagulation markers, serum VEGF, tissue factor		x		x	x	x
Serum level of sirolimus adjustments⁵				x	x
Blood and skin samples for genetic analysis and collection (ancillary study)		x
Photographs		x	x			x
Self-assessment for patients and proxy by visual analog scale			x	x	x	x
Dermatological quality of life scale		x	x	x	x	x
Adverse events/serious adverse events⁶				x	x	x

*possibly by call phone
**depending on the date of the treatment switch from observational to treatment stage
***sirolimus might be maintained at the discretion of investigators after the protocol is finished
¹included in routine care
²complete blood count, ionogram, creatinine, urea, liver enzymes (gamma-GT, SGOT, SGPT), cholesterol, triglycerides, glucose, infection with HIV and hepatitis B and C, serum β-hCG or urine pregnancy test for women of childbearing age
³complete blood count, ionogram, creatinine, urea, liver enzymes, cholesterol, triglycerides, glucose, urinary pregnancy test on women of childbearing age
⁴every month during sirolimus: complete blood count, ionogram, creatinine, urea, liver enzymes, cholesterol, triglycerides, glucose
⁵performed after 15 days of sirolimus and then once a month
⁶adverse events will be recorded from when the informed consent form is signed
VEGF vascular endothelial growth factor

ISSN: 1745-6215