TABLE 2.

Comparison between time to first response (TTFR), time to disease progression (TTP), and median PFS time in Phase 2 clinical trials.

	Trial registration number	Treatment arm(s)	Timing of scans	Number of patients (TTFR)	TTFR per LI (months, 95% CI)	TTFR per BICR (months, 95% CI)	Number of patients (PFS)	Time to progression (TTP) per LI (months, 95% CI)	Time to progression (TTP) per BICR (months, 95% CI)	Median PFS per LI (months, 95% CI)	Median PFS per BICR (months, 95% CI)
1	NCT00875667	E: lenalidomide	Every two cycles for 6 months; every 90 days thereafter until disease progression a	E: 170	E: 5.5 (3.9 – 5.9)	E: 4.3 (3.9 – 11.5)	E: 170	E: 9.1 (5.8 − 14.1)	E: 9.1 (5.6 – 12.2)	E: 8.6 (5.6 – 12.1)	E: 8.7 (5.5 – 12.1)
		AC: investigator’s choice single agent		AC: 84	AC: 9.2 (5.9 – NR)	AC: NR	AC: 84	AC: 5.7 (3.7 − 8.5)	AC: 5.7 (3.7 – 6.9)	AC: 5.4 (3.6 – 7.7)	AC: 5.2 (3.7 – 6.9)
2	NCT01685060 - EudraCT 2012-003432-24	Ceritinib (LDK378)	Every 8 weeks	LI: 57	3.0 (SD, 3.54)	2.2 (SD, 1.44)	140	NA	NA	5.8 (5.4–7.6)	7.4 (5.6–10.9)
				BICR: 50
3	NCT01685138	Ceritinib (LDK378)	Every 8 weeks	LI: 84	2.5 (SD, 2.66)	2.2 (SD, 1.22)	124	NA	NA	16.6 (11.0–23.2)	19.4 (10.9–29.3)
				BICR: 79
7	NCT02040870	Ceritinib (LDK378)	Every 8 weeks	LI: 43	1.90 (1.6–12.9)	1.80 (1.6–3.7)	103	NA	NA	7.2 (4.1–7.5)	3.8 (3.6–5.6)
				BICR: 33
8	NCT02336451	Ceritinib (LDK378)	At week 8; every 8 weeks thereafter	LI	Arm 1: 1.87 (1.7–9.3)	Arm 1: 2.00 (1.7 – 12.9)	LI	NA	NA	Arm 1: 7.2 (3.3–10.9)	Arm 1: 5.0 (3.3–9.1)
				Arm 1: 15	Arm 2: 2.00 (1.7–9.3)	Arm 2: 1.76 (1.6–1.9)	Arm 1: 32			Arm 2: 5.6 (3.6–9.2)	Arm 2: 5.5 (3.6–7.3)
				Arm 2: 12	Arm 3: 1.82 (1.2–30.1)	Arm 3: 1.82 (1.7–26.5)	Arm 2: 35			Arm 3: NR (1.0 - NR)	Arm 3: 15.5 (1.0 - NR)
				Arm 3: 6	Arm 4: 1.81 (1.3–3.7)	Arm 4: 1.81 (1.3–22.0)	Arm 3: 6			Arm 4: 7.9 (5.5–9.4)	Arm 4: 7.7 (5.5–9.7)
				Arm 4: 26	Arm 5: 1.91 (1.8–3.6)	Arm 5: 1.86 (1.8–1.9)	Arm 4: 33			Arm 5: 5.2 (1.6–7.2)	Arm 5: 3.6 (1.6–5.4)
				Arm 5: 3			Arm 5: 14
				BICR			BICR
				Arm 1: 10			Arm 1: 34
				Arm 2: 6			Arm 2: 36
				Arm 3: 4			Arm 3: 8
				Arm 4: 27			Arm 4: 33
				Arm 5: 2			Arm 5:14
9	NCT00117637	Sorafenib (BAY43-9006) + interferon	Every 8 weeks	LI	E: 3.5 (1.6–11.1)	E: 1.8 (1.7–3.7)	E: 97	NA	NA	E: 5.6 (5.4–7.5)	E: 5.7 (5.0–7.4)
				E: 21
				AC: 14	AC: 5.4 (1.2–18.3)	AC: 5.4 (3.7–11)	AC: 92			AC: 7.0 (5.4–8.8)	AC: 5.6 (3.7–7.4)
				BICR
				E: 5
				AC: 8
10	NCT01943864	Trametinib	Every 8 weeks	LI: 0	NA	20.1 weeks	20	NA	NA	10.6 weeks (4.6–12.1)	10.6 weeks (4.6–12.7)
				BICR: 1
13	NCT03525678 (EudraCT 2017-004810-25)	Belantamab mafodotin (GSK2857916)	Every 3 weeks (radiography for skeletal lytic lesions, but it is not specified in the timing)	LI	Arm 1: 1.4 (0.8–2.1)	Arm 1: 1.4 (0.8–2.1)	Arm 1: 97	Arm 1: 2.3 (0.8 - NR)	Arm 1: 3.0 (0.9 - NR)	Arm 1: 2.2 (0.8 - NR)	Arm 1: 2.9 (0.9 - NR)
				Arm 1: 29	Arm 2: 1.5 (0.9–3.0)	Arm 2: 1.4 (0.8–2.8)	Arm 2: 99	Arm 2: 4.2 (1.3 - NR)	Arm 2: 5.8 (0.9 - NA)	Arm 2: 3.8 (1.1 - NR)	Arm 2: 4.9 (0.9 - NR)
				Arm 2: 31	Arm 3: 0.9 (0.8–1.0)	Arm 3: 0.9 (0.8–1.6)	Arm 3: 25	Arm 3: 4.3 (2.1 - NR)	Arm 3: NA (2.2 - NA)	Arm 3: 4.3 (2.1 - NR)	Arm 3: NA (2.2 - NR)
				Arm 3: 13
				BICR
				Arm 1: 30
				Arm 2: 34
				Arm 3: 12

Trials that were included in our initial analysis (Dello Russo et al., 2021) are highlighted in gray. ITT, intention to treat population; NA, not available; NR, not reached; ORR, objective response rate; PFS, progression-free survival; BICR, blind independent central review.

^aData included in the table refer to the analysis at data cutoff date, 7 March 2014 (Trněný et al., 2016).