Biologics in Relapsing Polychondritis

Relapsing polychondritis is a rare, often severe, and untreated fatal connective tissue disease. The inflammation of the ear cartilage is often the initial symptom, at least in the patients, who had been referred to our center. We’re still lacking a specific lab test for relapsing polychondritis. We have different diagnostic criteria by McAdam, Damiani and Michet. I’ve already written about the disease on this blog, less than I should have considering the fact that I treat patients with this disease. There have been four abstracts at the 2016 ACR Annual Meeting. I’ll also talk about the abstracts/studies, which doesn’t concern biologics.

Guillaume Moulis and his more than 20 co-authors presented [#1329]: “Efficacy and Safety of Biologics in Relapsing Polychondritis: A National Multicenter Study in France”. Conclusion: “Overall, biologics are an interesting option for RP treatment. “ You might call this statement laconic, but there’s lots of work included. The authors saw 41 patients and had to try several drugs until one worked. The rate of partial and complete remission is about 60-70%, with anakinra and abatacept being lowest at 50-53%. The authors looked at all TNF alpha inhibitor, anakinra, abatacept, tocilizumab, and rituximab.

Marcela Ferrada and colleagues presented [#1330]: “Clinical Presentations of Relapsing Polychondritis: More Than a Swollen Ear”. The authors acquired data via an internet-based questionnaire to catalogue the variety of possible clinical presentations of relapsing polychondritis. They could evaluate 180 questionnaires. In results the authors point out: “Common initial symptoms included dizziness, eye inflammation, constochondritis, and shortness of breath, nose pain, and voice changes. Some patients also reported fatigue, flu-like symptoms, fever and difficulty swallowing as initial symptoms. Complications of RP included disability (25%), tracheomalacia (16%) and intubation related to RP (12%).”  The study has limitations as you can’t validate the diagnosis and there’s a recall bias. But still, I think the study is very valuable.

Chee Ken Cheah and collegues presented [#1331]: “Disease Patterns and Long Term Outcome Amongst Patients with Relapsing Polychondritis – Single Centre Experience”. Conclusions: “Our study revealed higher number of initial organ presentation, and younger age of disease onset correlated with potential diagnosis delay. Male gender with airway involvements correlated with higher number of organ damages and poorer outcome. Multicenter registries may lead to a better understanding of this disease.”

Toshiki Nakajima and colleagues looked at 33 patients with relapsing polychondritis in this study [#1332]: “Severe Complications and Immunosuppressive Treatments in 33 Patients with Relapsing Polychondritis”. In results the authors inform us about HLA haplotypes, which are associated with relapsing polychondritis: “Positivity of HLA-DQB1 05:02 was 17.6% (3/17),　higher than 2.6% in healthy Japanese.” [The authors recently reported an association of disease onset and 3 HLA haplotypes (DQB1*05:02, B*67:01 and DRB1*16:02 in linkage disequilibrium) in C. Terao et al.: Rheumatology (Oxford) doi: 10.1093/rheumatology/kew233]. “Methotrexate (MTX), azathioprine (AZP), intravenous cyclophosphamide (IVCY), infliximab (IFX) and tocilizumab (TCZ) were used along with glucocorticoid ….” Conclusion: “The linkage with certain haplotypes of HLA and the positivity of autoantibodies (~30%) consolidate that RP is an autoimmune disease. IVCY showed a good response in patients with TB lesions in the present study. The prognosis of patients with CNS lesions was poor. Further collection of cases is required to elucidate pathophysiology and improve treatments.”

So we have different approaches according to disease severeness, organ involvement, and activity. Immunosuppressants as well as biologics are used. As the disease is rare there won’t be any randomized controlled trials and any drug will be off label. Anyway, a partial remission of 50-70% is feasible with biologics. Hopefully one can speed up approval proceedings with insurance companies because of the off label use.

Links:

https://en.wikipedia.org/wiki/Relapsing_polychondritis

http://rheumatologe.blogspot.de/2011/12/biologics-in-relapsing-polychondritis.html

Moulis G, Pugnet G, Costedoat-Chalumeau N, Mathian A, Leroux G, Boutemy J, Bouillet L, Berthier S, Gaultier JB, Jeandel PY, Konaté A, Mékinian A, Solau-Gervais E, Terrier B, Wendling D, Garnier C, Cathebras P, Arnaud L, Cacoub P, Amoura Z, Piette JC, Arlet P, Palmaro A, Lapeyre-Mestre M, Sailler L. Efficacy and Safety of Biologics in Relapsing Polychondritis: A National Multicenter Study in France [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). http://acrabstracts.org/abstract/efficacy-and-safety-of-biologics-in-relapsing-polychondritis-a-national-multicenter-study-in-france/. Accessed December 15, 2016.

Ferrada M, Choudhury SD, Newman K, Sinaii N, Guma M, Christie T, Katz JD. Clinical Presentations of Relapsing Polychondritis: More Than a Swollen Ear [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). http://acrabstracts.org/abstract/clinical-presentations-of-relapsing-polychondritis-more-than-a-swollen-ear/. Accessed December 15, 2016.

Cheah CK, Sangle (Joint First Author) S, D'Cruz D. Disease Patterns and Long Term Outcome Amongst Patients with Relapsing Polychondritis – Single Centre Experience [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). http://acrabstracts.org/abstract/disease-patterns-and-long-term-outcome-amongst-patients-with-relapsing-polychondritis-single-centre-experience/. Accessed December 15, 2016.

Nakajima T, Yoshifuji H, Terao C, Murakami K, Kuramoto N, Nakashima R, Imura Y, Tanaka M, Ohmura K, Mimori T. Severe Complications and Immunosuppressive Treatments in 33 Patients with Relapsing Polychondritis [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). http://acrabstracts.org/abstract/severe-complications-and-immunosuppressive-treatments-in-33-patients-with-relapsing-polychondritis/. Accessed December 15, 2016.