Table 3. Treatment-related adverse events overall and in more than 1 pediatric patient treated with ridaforolimus.

	Total N = 20		Ridaforolimus 22 mg/m2 n = 4		Ridaforolimus 28 mg/m2 n = 3		Ridaforolimus 33 mg/m2 n = 13
	Any grade	Grades 3–4	Any grade	Grades 3–4	Any grade	Grades 3–4	Any grade	Grades 3–4
Patients with ≥1 treatment-related adverse event	19 (95)	9 (45)	3 (75)	2 (50)	3 (100)	2 (67)	13 (100)	5 (38)
Nonhematologic
Stomatitis	15 (75)	0	2 (50)	0	3 (100)	0	10 (77)	0
Fatigue	9 (45)	0	2 (50)	0	1 (33)	0	6 (46)	0
Nausea	6 (30)	0	1 (25)	0	1 (33)	0	4 (31)	0
Dysgeusia	3 (15)	0	0	0	0	0	3 (23)	0
Headache	3 (15)	0	0	0	0	0	3 (23)	0
Photophobia	3 (15)	0	0	0	1 (33)	0	2 (15)	0
Vomiting	3 (15)	0	1 (25)	0	0	0	2 (15)	0
Decreased appetite	2 (10)	0	0	0	0	0	2 (15)	0
Decreased weight	2 (10)	0	0	0	0	0	2 (15)	0
Diarrhea	2 (10)	0	0	0	0	0	2 (15)	0
Dry mouth	2 (10)	0	2 (50)	0	0	0	0	0
Epistaxis	2 (10)	0	0	0	0	0	2 (15)	0
Oral herpes	2 (10)	1 (5)	1 (25)	1 (25)	0	0	1 (8)	0
Rash	2 (10)	0	0	0	1 (33)	0	1 (8)	0
Hematologic
Thrombocytopeniaa	13 (65)	2 (10)	2 (50)	0	1 (33)	1 (33)	10 (77)	1 (8)
Anemiaa	8 (40)	0	2 (50)	0	1 (33)	0	5 (38)	0
Leukopeniab	7 (35)	0	1 (25)	0	2 (67)	0	4 (31)	0
Neutropeniaa	6 (30)	1 (5)	2 (50)	0	0	0	4 (31)	1 (8)
Lymphopeniaa	2 (10)	1 (5)	1 (25)	0	0	0	1 (8)	1 (8)
Biochemical
Hypertriglyceridemiaa	10 (50)	1 (5)	2 (50)	0	1 (33)	0	7 (54)	1 (8)
Increased ALT	10 (50)	5 (25)	1 (25)	1 (25)	1 (33)	1 (33)	8 (62)	3 (23)
Hypercholesterolemiaa	9 (45)	1 (5)	2 (50)	0	1 (33)	1 (33)	6 (46)	0
Increased AST	8 (40)	1 (5)	1 (25)	0	1 (33)	0	6 (46)	1 (8)
Hypophosphatemiaa	5 (25)	1 (5)	2 (50)	1 (25)	1 (33)	0	2 (15)	0
Increased γ-glutamyltransferase	5 (25)	1 (5)	1 (25)	0	1 (33)	0	3 (23)	1 (8)
Hyperglycemiaa	2 (10)	0	0	0	1 (33)	0	1 (8)	0
Hypocalcemiaa	2 (10)	0	1 (25)	0	0	0	1 (8)	0
Hypokalemiaa	2 (10)	0	1 (25)	0	1 (33)	0	0	0
Increased alkaline phosphatase	2 (10)	0	1 (25)	0	0	0	1 (8)	0
Increased bilirubin	2 (10)	0	1 (25)	0	0	0	1 (8)	0

Numbers are n (%).

Note: Only the highest reported grade of a given adverse event is counted for an individual patient. Three grade 5 adverse events occurred during the study (neurologic symptoms in a patient with central nervous system metastases, neoplasm progression, and gastric perforation related to underlying disease); none were related to ridaforolimus, according to the investigator. The number of cycles evaluated is shown in Table 2.

^aReported as an adverse event by the investigator or based on laboratory values.

^bBased on laboratory values only; leukopenia was not reported separately by investigators as a treatment-related adverse event.