Spotlight on romiplostim in the treatment of children with chronic immune thrombocytopenia: design, development, and potential place in therapy

Figures & data

Table 1 Romiplostim in the treatment of children with chronic immune thrombocytopenia

Study	Design	N	Median age (years)	Duration of ITP prior to romiplostim (median years)	Treatments prior to romiplostim	Dose (median µg/kg)	Duration treatment/time to response	Outcome	Treatment-related SAE
Tarantino et al^Citation42	Randomized	42	10	1.9	38% received ≥3 treatments	3.9	24 weeks/4.5 weeks	22/42 (52%) with platelets ≥50 k/µL for 6 weeks with no rescue	1 SAE (headache and
Bussel et al^Citation40	Randomized	17	9	2.4	88% received ≥3 treatments	5	12 weeks/7 weeks	15/17 (88%) with platelets >50 k/µL for 2 weeks and increase 20 k/µL above baseline for 2 weeks	None
Elalfy et al^Citation39	Randomized	12	8.5	2.3	44% received ≥3 treatments	5	12 weeks/NR	10/12 (83%) with platelets >50 k/µL	None
Bussel et al^Citation41	2nd extension	12	10	2.4	NR	5.4	167 weeks/NR	After 1st 12 weeks – median platelet counts were >50 k/µL	None
Pasquet et a^Citation35	Retrospective, observational	10	NR	1.9	90% received ≥3 treatments	4–10 (at the time of clinical/lab response)	9 months/NR	1/10 (10%) with platelets >100 k/µL, 4/10 (40%) with platelet 30–100 k/µL and 2× baseline without bleeding	None
Mokhtar et al^Citation33	Retrospective, observational	7	5.5	2.4	100% received ≥3 treatments	5.7	Median 12 weeks/7 weeks	1/7 (14%) with platelets >50 k/µL for 6 weeks, 4/7 (57%) with 2× baseline platelet count	None
Marquínez-Alonso et al^Citation32	Retrospective, observational	4	10.3	2.7	75% received ≥3 treatments	4	Median 50 weeks/NR	3/4 (75%) with platelets >50 k/µL for 6 or more of last 8 weeks	None
Escudero Vilaplana et al^Citation31	Retrospective, observational	3	9	2.8	100% received ≥3 treatments	3.3	Mean 33 weeks/NR	1/3 (33%) with platelets >100 k/µL	None
Ramaswamy et al^Citation36	Retrospective, observational	21	11.4	NR	Mean number of 3.6 treatments		Duration: 6–44 months – 11/18 ongoing	18/21 (85%) with platelets >50 k/µL and >20 k/µL above baseline for 2 weeks	None
Seidel et al^Citation34	Single-center cohort	7	11	NR	100% received ≥3 treatments	5–10	Median 14 months/NR	6/7 (85%) with platelets >50 k/µL	None
Neunert et al^Citation38	Retrospective, observational	43	Mean 10.9	Mean 1.7	42% received steroids at start of therapy	5	6.4 weeks (response time)	71% with platelets ≥20 k/µL above baseline ×2 consecutive weeks, 84% with platelets ≥50 k/µL without rescue therapy in 7 days	1 neutralizing

Abbreviations: ITP, immune thrombocytopenia; NR, not recorded; SAE, serious adverse event.

Table 2 Romiplostim in the treatment of children with chronic immune thrombocytopenia and health-related quality of life

Study	Design	N	Median age (years)	Duration of ITP prior to romiplostim	Median number of treatments	Length of treatment (weeks)	Outcome (KIT scores)
Tarantino et al^Citation42	Randomized	42	10	1.9 years	12% other concommitant therapy at baseline	24	No difference in KIT scores – child self, parent proxy, or parent impact. Parent impact scores improved in mixed effect
Mathias et al^Citation46	Randomized	42	Mean 9.7 years	Mean 3 years	38% received ≥3 treatments	24	KIT scores – self/proxy and parent impact – greater reduction in parent burden from baseline w/romiplostim (compared with placebo) – P=0.015
Klaassen et al^Citation45	Randomized	17	9	2.4 years	94% with prior treatment	12	KIT scores – self/proxy and parent impact – greater reduction in parent burden from baseline w/romiplostim (compared with placebo) – P=0.008

Abbreviations: ITP, immune thrombocytopenia; K IT, kids’ ITP tools.

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