Biologic disease modifying antirheumatic drugs and Janus kinase inhibitors in paediatric rheumatology – what we know and what we do not know from randomized controlled trials

Table 5 Overview and general characteristics of identified reviewed randomized controlled trials performed in non-JIA patients (July 2020)

Drug class	Drug	Dose	Study (phase)	Studytime^c	Population	N	Age criteria	Age^a	Primary outcome/endpoint	Main conclusion
IL-12/23 inhibitor	ustekinumab	0.75 mg/kg, 22.5/45/ 90 mg	^dCADMUS, NCT01090427 (III) [93]^b	60	Plaque psoriasis	110	12 to 17	15.2	PGA ≤1	Effective
IL-17 inhibitor	ixekizumab	20 mg BW < 25 kg q4w, 40 mg BW 25–50 kg q4w, 80 mg BW > 50 kg q4w	^eIXORA-PEDS, NCT03073200 (III) [94] ^b	12	Plaque psoriasis	201	6 to 17	13.5	PASI75, PGA ≤1	Effective
TNF inhibitor	adalimumab	0.4 mg/kg q2w, 0.8 mg/kg q2w	^fM04–717, NCT01251614 [95,96,97,98] ^b	52	Plaque psoriasis	114	4 to 17	13	PASI75, PGA ≤1	Effective
	etanercept	0.8 mg/kg/qw	EATAK, NCT00841789 (II) [99] ^b	6	KD	205	0 to 18	3.7	Fever	Not effective
	etanercept	0.8 mg/kg/qw	20030211, NCT00078819 (III) [100,101,102,103,104] ^b	48	Plaque psoriasis	211	4 to 17	13^a	PASI75	Effective
	infliximab	5 mg/kg single dose	Han 2018 (NA) [105] ^b	0.571	KD	154	0 to 4	2.2^a	Unclear	Effective
		5 mg/kg single dose	TA-650-22, NCT01596335 (III) [106] ^b	8	KD	31	1 to 10	3^a	Defervescence	Effective
		5 mg/kg single dose	Tremoulet 2014, NCT00760435 (III) [107, 108] ^b	5	KD	196	0 to 17	3^a	Fever	Effective
		5 mg/kg, 10 mg/kg q4w	Pro00000057, NCT00589628 (IV) [109] ^b	39	Non-infectious uveitis	13	4 to 18	NA	Uveitis disease activity	NA
BAFF inhibitor	belimumab	10 mg/kg qm	^gPLUTO, NCT01649765 (II) [110, 111] ^b	52	SLE	93	5 to 17	14	SRI4	Effective

Abbreviations: Drug class: IL interleukin, TNF tumour necrosis factor; Dose: mg milligram, kg kilogram, qw once per week, q2w once per every 2 weeks, q4w once per every 4 weeks, qm once every month; Population: KD Kawasaki disease, SLE systemic lupus erythematosus; Outcome: PASI psoriasis area and severity index, PGA Physician global assessment, SRI4 systemic lupus erythematosus response index 4, NA not available
^amedian age, otherwise mean age across all arms of the study, ^bparallel study design, ^cduration in weeks; ^dAlso registered under EudraCT 2009–014368-20, ^eAlso registered under EudraCT 2016–003331-38;^fAlso registered under EudraCT 2009–013072-52; ^gAlso registered under EudraCT 2011–000368-88

ISSN: 1546-0096