Table 2.

Reported outcomes related to efficacy and adverse effects.

	Progression-free survival (PFS)Overall survival (PFS)												Adverse effects (Grade ≥3)
Year; Author	ORR (%) [95% CI]	DCR (%) [95% CI]	DOR (months) [95% CI]	Median PFS (months) [95% CI]	PFS6m (%)	PFS1y (%)	HR [95% CI]	Median OS (months) [95% CI]	OS6m (%)	OS1y (%)	OS2y (%)	HR [95% CI]	Treatment related	n (%)
2018; Adams S68	39.4 [22.9-57.9]	51.5 [33.5-69.2]	9.1 [2.9-20.9]	5.5 [5.1-7.7]	45.5	-	-	14.7 [10.1-NE]	-	57.4	40.6	-	All AEs	24(73)
													Neutropenia	15(46)
													Thrombocytopenia	3(9)
													Diarrhea	2(6)
													Anemia	2(6)
													Pneumonia	2(6)
													Leukopenia	2(6)
													Peripheral neuropathy	1(3)
													Myalgia	1(3)
													Bone pain	1(3)
													Colitis	1(3)
													Syncope	1(3)
													Elevated AST	1(3)
													Febrile neutropenia	1(3)
													Type 1 diabetes mellitus	1(3)
													Pneumonia mycoplasma	1(3)
													Paronychia	1(3)
2018; Adams S Cohort A70	2018; Adams S Cohort A70	5.3 [2.7-9.9]	7.6 [4.4-12.7]	2.0 [1.9-2.0]	14.9	8.1	-	9.0 [7.6-11.2]	69.7	39.8	-	-	Any AEs	22(13)
													Diarrhea	3 (1.8)
													Fatigue	1 (0.6)
													Nausea	1 (0.6)
2018; Adams S Cohort B71	21.4 [13.9-31.4]	23.8 [15.9-34.0]	10.4 [4.9-19.2]	2.1 [2.0-2.2]	27.0	-	-	18.0 [12.9-23.0]	81.0	61.7	-	-	Any AEs	8 (9.5)
													Diarrhea	1 (1.2)
													Fatigue	1 (1.2)
													Anemia	1 (1.2)
2018; Schmid P72	56.0 [51.3-60.6]	-	7.4 [6.9-9.0]	7.2 [5.6-7.5]	50.1	23.7	0.62* [0.49-0.78]	21.3 [17.3-23.4]	-	60.09	42.1	0.62* [0.45-0.86]	Neutropenia	37 (8.2)
													Peripheral neuropathy	25 (5.5)
													Nausea	5 (1.1)
													Alopecia	3 (0.7)
													Pyrexia	3 (0.7)
2018; Emens LA69	24.0 [8.0-47.0]	29.0	21.0 [10.0-38.0]	1.4 [1.3-1.6]	-	-	-	17.6 [10.2-NE]	-	41.0	19.0	2.21° 1.17-4.16]	Any AEs	46(40)
													Hyperglycemia	1 (0.9)
													Pneumonitis	1 (0.9)
													Pulmonary hypertension	1 (0.9)
2017; irix LY73	5.2 [1.1-14.4]	31.0	-	5.9 [5.7-6.9]	12.4	-	-	9.2 [4.3-NE]	-	37.9	-	-	Any AEs	23 (13.7)
													Fatigue	3 (1.8)
													Anemia	3 (1.8)
													Autoimmune hepatitis	3 (1.8)
													Elevated GGT	3 (1.8)
													Arthralgia	1 (0.6)
													Acute hepatic failure	1 (0.6)
													Dyspnea exertional	1 (0.6)
													Elevated AST	1 (0.6)
													Elevated ALT	1 (0.6)
													Hypoxia	1 (0.6)
													Hypokalemia	1 (0.6)
													Pneumonitis	1 (0.6)
													Axillary pain	1 (0.6)
													Pleuritic pain	1 (0.6)
													Non-cardiac chest pain	1 (0.6)
													Thrombocytopenia	1 (0.6)
													Cardiac arrest	1 (0.6)
													Hypertriglyceridemia	1 (0.6)
													Neutropenia	1 (0.6)
													Proteinuria	1 (0.6)
													Respiratory failure	1 (0.6)
													Respiratory distress	1 (0.6)
													Pulmonary arterial hypertension1	(0.6)
2016; Nanda R74	18.5 [6.3-38.1]	25.9	-	1.9 1.7-5.5]	24.4	-	-	11.2 [5.3-NE]	66.7	43.0	-	-	Anemia	1 (3.1)
													Aseptic meningitis	1 (3.1)
													Disseminated intravascular	(3.1)
													coagulation
													Headache	1 (3.1)
													Lymphopenia	1 (3.1)
													Pyrexia	1 (3.1)

ORR, overall response rate; DCR, disease control rate; DOR, duration of response; PFS, progression-free survival; PFS6m, 6-months PFS rate; PFS1y, 1-year PFS rate; OS, overall survival; OS6m, 6-months OS rate; OS1y, 1-year OS rate; OS2y, 2-year OS rate; HR, hazard ratio; NE, not estimable; AEs, adverse effects; AST, aspartate aminotransferase; ALT, alanine aminotransferase; GGT, -glutamyl transferase

*HR calculated between intervention arm and placebo arm

°HR calculated between 1st and 2nd line of therapy.