Session Type: Poster Session (Monday)
Session Time: 9:00AM-11:00AM
Background/Purpose: Polyarteritis nodosa (PAN) is a rare systemic necrotizing vasculitis of medium- and small-sized arteries, not associated with antineutrophil cytoplasmic antibodies (ANCA). Conventional treatments include glucocorticoids (GCs) for non-severe disease, and combined GCs and immunosuppressants for severe disease. Nevertheless, some patients have refractory and/or relapsing disease. We examined off-label biotherapy use for relapsing/refractory PAN.
Methods: This retrospective European collaborative study included patients with PAN meeting ACR criteria and/or Chapel Hill Consensus Conference 2012 definitions. Treatment efficacy and safety were recorded. Remission was defined as the absence of vasculitis manifestations (BVAS = 0) with ≤5 mg/day of prednisone. Partial response was defined as BVAS = 0, with 6–10 mg/day of prednisone.
Results: Fifty-two patients (24 men and 28 women; median age 51 years) were included. Nineteen (37%) patients received TNF-α blockers, 16 (31%) rituximab (RTX), 9 (17%) tocilizumab (TCZ), and 8 (15%) other biologics (i.e. alemtuzumab for 3, anakinra for 2, interferon-α for 2 and abatacept for 1). Previous treatments were: GCs for all, including methylprednisolone infusions (73%), cyclophosphamide (62%), azathioprine (54%), mycophenolate mofetil (48%) or methotrexate (46%). At inclusion, median BVAS was 5 (range 0-18), including 5 (2-12) for the TNF-α blocker group, 5 (2-12) for the RTX recipients and 4 (0-6) for those given TCZ.
At month (M) 6 and M12, respectively, median BVAS fell to 3 and 0 for TNF-α blockers group, to 3.5 and 0 for those given RTX, and to 0 and 0 for the TCZ group. Median GCs dose declined from baseline 17.5 mg/day to 10 at 6 months and 5 at 12 months for the TNF-α blocker recipient, from 15 to 10 and 5 mg/day for those given RTX, respectively, and from 15 to 7 and 5 mg/day for the TCZ group.
Nine (47%) patients stopped TNF-α blockers: for adverse events in 2 and/or refractory disease in 8. Refractory disease led 6 (38%) to stop RTX. Finally, 4 (44%) stopped TCZ: 2 for adverse events (testicular abscess and worsening renal failure), and 2 for refractory disease. Two additional patients had to decrease the dose because of cytopenias.
After median follow-up of 34.4 [IQR 21.5–59.5] months, remissions, partial responses, treatment failure and stop for adverse event, respectively, occurred in 43%, 5%, 47% and 5% for TNF-α blockers recipients, 31%, 13%, 56% and 0% for RTX recipients, and 56%, 0%, 22% and 22% for TCZ recipients. No remission was noted in patients treated with anakinra, alemtuzumab, interferon-α and abatacept.
Conclusion: These results suggest that TNF-α blockers and TCZ could achieve higher remission and GC-sparing rates for relapsing and/or refractory PAN than other biologics. Our findings warrant further study to confirm or refute them.
To cite this abstract in AMA style:Canzian A, Karadag O, Contis A, Maurier F, Sartorelli S, Denis L, Sanges S, de Moreuil C, Durel C, Durupt S, Jachiet M, Rouzaud D, Salvarani C, Schiavon F, Dagna L, Bonnet F, Jayne D, Guillevin L, Terrier B. Off-Label Use of Biotherapies to Treat Relapsing And/or Refractory Polyarteritis Nodosa [abstract]. Arthritis Rheumatol. 2019; 71 (suppl 10). https://acrabstracts.org/abstract/off-label-use-of-biotherapies-to-treat-relapsing-and-or-refractory-polyarteritis-nodosa/. Accessed April 12, 2021.
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ACR Meeting Abstracts - https://acrabstracts.org/abstract/off-label-use-of-biotherapies-to-treat-relapsing-and-or-refractory-polyarteritis-nodosa/