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Table 5 Pediatric Studies in AAV

From: Hallmark trials in ANCA-associated vasculitis (AAV) for the pediatric rheumatologist

Reference, Country

Study Design

Patient Selection

Induction therapy

Maintenance therapy

Therapy-related outcomes

Adverse Events

Akikusa et al., 2007, Canada

Retrospective 1984–2005

n = 25

GPA 100%

GC 100%

CYC 76%

AZA 40%

MTX 32%

n = 7*

AZA (n = 3)

MTX (n = 4)

100% achieved remission (median 5 months, range 3–6 months)

75% relapse rate (median 10 months)

12 hospitalizations in 5 patients for infections

0 malignancies

0 deaths

Iudici et al., 2015, France

Retrospective

n = 35

GPA 71.4%

EGPA 17.1%

MPA 11.4%

GC 91.4%

GC + IS 77.1%

CYC IV 54.3%

CYC PO 5.7%

AZA 8.6%

MTX 5.7%

SZ 2.9%

PLEX 2.9%

–

Inactive disease at last follow-up (n = 33, median 96 months):

GPA 68.2% on treatment

EGPA 100% on treatment (50% on GC + IS)

MPA 100% on treatment (66.7% on GC + IS)

Relapse rates: 76% overall (GPA 83%, EGPA 50%, MPA 33%)

9 infections

1 pancreatitis (GC)

2 cataracts (GC)

1 transaminitis (AZA)

1 hematologic (CYC)

1 hypertension (GC)

4 deaths

Sacri et al., 2015, France

Retrospective 1986–2011

n = 66

GPA 42%

MPA 58%

GC 100%

IV 86%, PO 14%

CYC IV 47%

CYC PO 20%

RTX 13.6%

MMF 4.5%

PLEX 16.7%

GC alone 28.8%

GC + AZA/ MTX/ MMF 63.6%

AZA alone 1.5%

None 6%

92.4% achieved remission

Post-induction: 70% achieved remission

24.2% (n = 16) had refractory disease; 15/16 achieved secondary remission with addition of CYC (n = 8), IVIG (n = 2), RTX (n = 3) PLEX (n = 3)

40.9% relapse rate (median 29 mos)

4 deaths

ARChiVe, Morishita et al., 2017, International

2004–2007 retrospective, 2007–2008 prospective

n = 105

GPA 81%

MPA 13%

EGPA 6%

GC 100% (IV pulse 70%)

CYC 70.5%

MTX 16.2%

RTX 13.3%

AZA 1.9%

MMF 1.0%

PLEX 23.8%

AZA 42.3%

MTX 22.8%

MMF 13.3%

CYC 9.5%

RTX 9.5%

None 4.7%

44/105 (42%) achieved remission by 12 months

21/44 (48%) discontinued GC

41/44 (93%) remained on maintenance

24% relapse rate

80 hospitalizations in 43 patients:

46% flares

16% infection

5% treatment-related

15% other disease-related

18% unrelated to vasculitis

0 deaths

  1. GPA Granulomatosis with Polyangiitis, EGPA Eosinophilic granulomatosis with polyangiitis, MPA Microscopic polyangiitis, GN Glomerulonephritis, GC Glucocorticoids, CYC Cyclophosphamide, AZA Azathioprine, MTX Methotrexate, RTX Rituximab, PLEX Plasmapheresis, IS Immunosuppressant, SZ Sulfasalazine, MMF Mycophenolate mofetil, IVIG Intravenous immunoglobulin
  2. *7 patients were treated with a remission induction/maintenance regimen