Long-term safety and efficacy of romiplostim for treatment of immune thrombocytopenia

Figures & data

Table 1 Platelet response-adapted dosing of romiplostim

Starting dose:

• 1 µg/kg

Subsequent dosing:

• 2 µg/kg every week if 10×10^9/L or less

• 2 µg/kg every 2 weeks if 11×10^9/L to 50×10^9/L

Maintenance dosing:

• Increase dose by 1 µg/kg every week if <10×10^9/L

• Increase dose by 1 µg/kg after 2 weeks if 11×10^9/L to 50×10^9/L

• No dose adjustment if 50–200×10^9/L

• Reduce dose by 1 µg/kg after 2 consecutive weeks if 201×10^9/L–400×10^9/L

• Hold dose if >400×10^9/L. Check platelet count weekly; resume at dose reduced by 1 µg/kg after platelet count <200×10^9/L

Maximum allowed dose: 15 µg/kg

Note: Data from Kuter et al.²²

Table 2 Platelet response in placebo-controlled studies²²

Outcomes	Study 1 (nonsplenectomized patients)		Study 2 (splenectomized patients)
	Romiplostim (n=41)	Placebo (n=21)	Romiplostim (n=42)	Placebo (n=21)
Overall platelet response, n (%)	36 (88)	3 (14)	33 (79)	0 (0)
Durable platelet response, n (%)	26 (61)	1 (5)	16 (38)	0 (0)
Requiring rescue therapy, n (%)	8 (20)	13 (62)	11 (26)	12 (57)
Average number of weeks with platelet counts	15	1	12	0
≥50×10^9/L

Notes: Reprinted from The Lancet. Lancet 2008;371(9610), Kuter DJ, Bussel JB, Lyons RM, et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. 395–403. Copyright 2008, with permission from Elsevier.²² All P-values <0.05 for platelet response and rescue therapy.

Table 3 Incidence of adverse events in patients receiving long-term romiplostim treatment for chronic ITP²⁶

	Cohort 1 (n=33)	Cohort 2 (n=88)	Cohort 3 (n=31)	Cohort 4 (n=139)	Total (n=291)
Any AE, n (%)	32 (97)	88 (100)	30 (97)	134 (96)	284 (98)
Serious AE, n (%)	18 (55)	40 (46)	14 (45)	45 (32)	117 (40)
Treatment-related AE, n (%)	18 (55)	37 (42)	10 (32)	38 (27)	103 (35)
Treatment-related serious AE, n (%)	8 (24)	3 (3)	5 (16)	8 (6)	24 (8)
Deaths, n (%)	0 (0)	6 (7)	3 (10)	7 (5)	16 (5)
Thrombotic events, n (%)	5 (15)	5 (6)	8 (26)	7 (5)	19 (7)
Bleeding events, n (%)	30 (91)	63 (72)	23 (74)	50 (36)	166 (57)

Note: Reprinted from Br J Haematol. Kuter DJ, Bussel JB, Newland A, et al. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. 2013;161(3):411–423. Copyright 2013, with permission from John Wiley and Sons.²⁶

Abbreviations: AE, adverse event; ITP, immune thrombocytopenia.

KuterDJBusselJBLyonsRMEfficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trialLancet2008371961039540318242413

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KuterDJBusselJBNewlandALong-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacyBr J Haematol2013161341142323432528

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