Sirolimus for the treatment of vascular tumors and malformations in children: A 10-year single-institution retrospective study

Table 1 Clinical characteristics, treatment, and outcomes of pediatric vascular anomalies treated with sirolimus

Pt	Sex	Age at dx (mo)	Age at SRL tx (mo)	VA type	Location	Clinical presentation	Prior tx (s)	Response to prior tx	SRL regimen	Duration (mo)	Best response	SRL discontinued	AEs (CTCAE v6.0)	FU (mo)
Vascular tumors
1	M	4	60	KHE	Iliopsoas	KMP, flexion contracture	Surgery, CMT	PD	+ PSL	31	PR	Yes	Hypercholesterolemia (G2)	68
2	F	3	8	KHE	Shoulder	Mass, KMP, pain	CMT	PD	+ PSL	62	PR	Yes	None	87
3	M	154	168	CHE	Paranasal sinus	Proptosis, epistaxis	CMT, radiotherapy	PR	Mono	20	PR	Yes	Proteinuria (G1), creatinine increased (G1), hypokalemia (G1)	42
4	M	1	8	IH	Face	Mass	Propranolol	SD	Mono	9	PR	No	Proteinuria (G2)	9
5	M	0	120	NICH	Face, neck	Mass	Propranolol	SD	Mono	5	PR	No	None	5
Vascular malformations
6	M	0	2	LM	Leg	Mass	None	-	Mono	30	SD	Yes	None	30
7	M	0	26	VM	Hand	Mass	Propranolol	SD	Mono	11	PR	Yes	None	33
8	M	0	4	LM	Neck	Mass	Surgery, sclerotherapy	PD	Mono	30	PR	No	Proteinuria (G3)	32
9	F	0	36	LM	Tongue	Mass, pain	None	-	Mono	2	PR	No	None	2
10	M	24	72	LM	Chest	Chylothorax, osteolytic lesion	Surgery	PR	Mono	3	PR	No	None	3
11	F	2	11	LM	Buttock	Mass with lymphatic leakage	Surgery, sclerotherapy	PR	Mono	1	PR	No	None	1

AEs: Adverse events; CHE: Composite hemangioendothelioma; CMT: Chemotherapy; CTCAE: Common Terminology Criteria for Adverse Events; dx: Diagnosis; F: Female; FU: Follow-up; G: Grade; IH: Infantile hemangioma; KHE: Kaposiform hemangioendothelioma; KMP: Kasabach-Merritt phenomenon; LM: Lymphatic malformation; M: Male; mo: Months; NICH: Non-involuting congenital hemangioma; PD: Progressive disease; PR: Partial response; PSL: Prednisolone; Pt: Patient; SD: Stable disease; SRL: Sirolimus; tx: Treatment; VA: Vascular anomaly; VM: Venous malformation.