Objectives To assess the efficacy and the safety of biologics in a cohort of patients with relapsing polychondritis (RP).
Methods We conducted a French multicentre retrospective cohort study including patients treated with biologics for RP. Efficacy outcomes were clinical response (partial or complete) and complete response during the first 6 months of exposure, plus daily corticosteroid dose at 6 months. Other outcomes were adverse drug reactions (ADRs), persistence of biologics and factors associated with a response.
Results This study included 41 patients exposed to 105 biologics (tumour-necrosis factor (TNF) inhibitors, n=60; tocilizumab, n=17; anakinra, n=15; rituximab, n=7; abatacept, n=6). Overall response rate during the first 6 months of exposure was 62.9%. Complete response rate was 19.0%. Reduced corticosteroid doses were highly variable among patients. ADRs were mostly infections (n=42). Reasons for biologic withdrawal (73.3%) were insufficient efficacy (34.3%; ranging from 23.5% for tocilizumab to 72.7% for etanercept), loss of efficacy (18.1%) and ADRs (20.9%; mostly for anakinra: 46.7%). Persistence was comparable among biologic classes. Among TNF inhibitors, the highest persistence was observed with adalimumab. Differences in clinical response rates were observed depending on biologics and organ involvement. There were trends towards a lower response rate in cases with associated myelodysplastic syndrome and for a higher response rate for nasal/auricular chondritis, sternal chondritis and concomitant exposure to non-biologic disease-modifying antirheumatic drugs.
Conclusions This study describes the efficacy of biologics for refractory RP. However, the number of complete responses was low and there were concerns about the risk of ADRs, particularly infections.
- relapsing polychondritis
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Handling editor Josef S Smolen
Contributors GM, GP, ML-M and LS designed the study. All other authors included patients into the study. GM and AP conducted the statistical analyses. GM, GP, ML-M and LS interpreted the results and drafted the manuscript. All authors reviewed the manuscript and gave their approval for submission.
Funding This study was funded by Toulouse University Hospital.
Competing interests GM received a travel grant from Abbvie in 2013 and Amgen in 2017 and received research grants from Novartis, CSL Behring and the Institut Servier in 2016 and 2017. GP received travel support and lecture fees from Abbvie. DW received speaking fees and membership on the advisory boards of the following societies: AbbVie, BMS, MSD, Pfizer, Roche Chugai, Amgen, Nordic Pharma, UCB, SOBI, Sanofi Aventis, Novartis, Janssen, Celgene, Hospira, Lilly and Sandoz; he received grants/hospitality from Abbvie, Pfizer, Roche Chugai, MSD and UCB. BT received travel support from Roche and LFB, and received consulting fees from Roche, GSK, LFB and Grifols. PC received consulting and lecturing fees from Abbvie, Astra Zeneca, Bristol-Myers Squibb, Gilead, Glaxo Smith Kline, Janssen, Merck Sharp Dohme, Roche, Servier and Vifor.
Patient consent Not required.
Ethics approval This study received approval from the Toulouse University Ethics Committee in 2013, and according to French law from the Comité Consultatif du Traitement de l’Information et de la Recherche en Santé (n°13.251) in 2013 and then authorisation from the Commission Nationale de l’Informatique et des Libertés (n°DR-2013-378) in 2013.
Provenance and peer review Not commissioned; externally peer reviewed.
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