Tocilizumab for the Treatment of Mevalonate Kinase Deficiency

Table 1

Previous reports of tocilizumab for the treatment of MKD.

	Shendi et al. (n = 1) [19]	Stoffels et al. (n = 2) [15]		Lane et al. (n = 2) [20]		Muster et al. (n = 1) [21]
Patient	1	2	3	4	5	6
TOC dose (mg/kg) and route of administration	7 IV	8 IV	8 IV	8 IV	8 IV	8 IV
Frequency of administration (weeks)	4	4	4	4	4	4
Age (range), years at onset of TOC	13	Not described	Not described	24	13	36
Treatment prior to TOC	COL	ANA	ANA	ANA	ETA	NSAID
	PRED			ETA		SIMVA
	ETA					ANA
	ANA
Duration of treatment (months)	20	5	5	24	13	48–60
Outcome
Clinical	CR	CR	CR	CR		PR
Serological	CR	Not described	PR	CR		—
Adverse events	URTI	Not described	Not described	Not described		Not described
Comments	CR at dose of 8 mg/kg but due to adverse events dose reduced, ultimately with stable clinical and serological status on 7 mg/kg IV every 4 weeks	—	—	MKD complicated by AA amyloidosis. Remained on therapy with PRED 0.5 mg/kg/day	Stabilized on monotherapy with TOC	After starting TOC, average hospital admissions dropped 11/yr to 3/yr
						TOC given in combination with IVMP for first 3 yrs then as monotherapy

IV: intravenous; COL: colchicine; PRED: prednisolone; ETA: etanercept; ANA: anakinra; NSAID: nonsteroidal anti-inflammatory drug; SIMVA: simvastatin; CR: complete response; PR: partial response; URTI: upper respiratory tract infection; TOC: tocilizumab; yrs: years.