Patient | Sex, age, age at disease onset, body weight | Clinical phenotype and disease severitya, age at disease onset, MSA status | Disease activity at time of fSCIg initiation | Previous or concurrent antirheumatic treatments | Reason for initiation of fSCIg instead of IVIg, CMAS at time of fSCIg initiation | Disease course following initiation of fSCIg |
---|---|---|---|---|---|---|
1 | Male, 11 years, 9 years at disease onset, 35 kg | Classic, severe JDM with prominent skin involvement, joint contractures, Anti-Mi-2 positive | Clinically inactive (however, recent recurrence after attempted IVIg discontiuation) | IVIg (60 g/month), fSCIg (70 g/month), PDN 0.125 mg/kg/day, MTX 15 mg/m2/week, HCQ | IVIg adverse effects (headaches, nausea and vomiting), needle phobia CMAS 51 | Within 3 months mild deterioration of CMAS (49 instead of 51); resolution after switching to fSCIg 5 days apart |
2 | Female, 10 years, 8 years at disease onset, 35 kg | Classic, severe JDM with prominent vaculopathy, dysphagia, cutaneous ulcerations, myocarditis, joint contractures, calcinosis, Anti-TIF-1gamma positive | Residual disease activity, vasculopathy | IVIg (70 g/month), fSCIg (70 g/month), PDN 0.21 mg/kg/day, MMF 1200 mg/m2/d, MTX 15 mg/m2/week, RTX (status post 375 mg/m2 ×4), CYC (status post 6 × 750 mg/m2) | IVIg adverse effects (headaches), difficult peripheral venous access with port-a-cath CMAS not interpretable due to severity of contractures | Stable mild residual disease activity even after switching to fSCIg 5 days apart |
3 | Female, 7 years, 5 years at disease onset, 20 kg | Classic, severe JDM with prominent vasculopathy, cutaneous ulcerations, Anti-SRP and anti-MI-2 positive | Clinically inactive (recent recurrence) | IVIg (40 g/month), fSCIg (40 g/month), PDN 0.125 mg/kg/day, MMF 1200 mg/m2/d, MTX 15 mg/m2/week RTX (status post 375 mg/m2 × 4 [first cycle], 750 mg/m2 × 1 [second cycle]) | IVIg adverse effects (headaches, nausea and vomiting), needle phobia CMAS 52 | Maintenance of clinically inactive disease; CMAS 52; fSCIg continued biweekly |
4 | Male, 12 years, 8 years at disease onset, 35 kg | Classic, moderately severe JDM with nodular dystrophic calcification, anti-NXP-2 positive | Clinically inactive (however, progressive calcinosis) | IVIg (35 g/month), fSCIg (60 g/month) PDN 0.07 mg/kg/day, MMF 1200 mg/m2/d, Colchicin | IVIg adverse effects (headaches, nausea and vomiting), needle phobia, residual disease activity with insufficient IVIg dose CMAS 52 | Maintenance of clinically inactive disease; CMAS 52; calcinosis decreasing; fSCIg continued biweekly |
5 | Female, 8 years, 5 years at disease onset, 20 kg | Classic, severe JDM with rhadomyolysis, failure to thrive and nephrolithiasis, MSA negative | Active skin and muscle disease (IVIg was discontinued 6 months earlier) | IVIG (40 g/month), fSCIg (50 g/month) PDN 0.19 mg/kg/day, MMF 600 mg/m2/d (diarrhea with higher doses), MTX 15 mg/m2/week | IVIg adverse effects (headaches, nausea and vomiting), needle phobia CMAS 46 | Improvement but residual, mild proximal muscle weakness; normal muscle strength (inactive disease) after switching to fSCIg 5 days apart; CMAS 50; further PDN reduction |