Therapeutic Strategy in Patients with Mixed Connective Tissue Disease: A Multicenter Retrospective Study in the French MCTD Cohort

Kevin Chevalier¹, Benjamin Thoreau², Marc Michel³, Bertrand Godeau⁴, Christian AGARD⁵, Thomas Papo⁶, Karim Sacré⁶, Raphaele Seror⁷, Xavier Mariette⁸, Patrice Cacoub⁹, Ygal Benhamou¹⁰, Hervé Levesque¹⁰, Cécile Goujard¹¹, Olivier Lambotte¹¹, Bernard Bonnotte¹², Maxime SAMSON¹³, Felix Ackermann¹⁴, Jean Schmidt¹⁵, Pierre Duhaut¹⁶, Jean-Emmanuel Kahn¹⁷, Thomas Hanslik¹⁷, Nathalie Costedoat-chalumeau¹⁸, Benjamin Terrier¹⁹, Alexis Régent²⁰, bertrand Dunogue², Pascal cohen²¹, Veronique LE GUERN²², Eric Hachulla²³, Luc Mouthon² and Benjamin Chaigne²⁴, ¹Department of Internal Medicine, National Reference Center for Rare Systemic Autoimmune Diseases, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris, Université Paris Cité, Paris, France, Paris, France, ²Service de Médecine Interne, Centre de Référence Maladies Systémiques Autoimmunes et Autoinflammatoires Rares d'Ile de France de l’Est et de l’Ouest, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France, ³2. Department of Internal Medicine, Henri-Mondor University Hospital, Assistance Publique-Hôpitaux de Paris, Université Paris Est Créteil (UPEC), Créteil, France., Créteil, France, ⁴Department of Internal Medicine, Henri-Mondor University Hospital, Assistance Publique-Hôpitaux de Paris, Université Paris Est Créteil (UPEC), Créteil, France., Créteil, France, ⁵Nantes University Hospital, Nantes, France, ⁶Université Paris Cité, Paris, France, ⁷Service de Rhumatologie, Hôpital Bicêtre, AP-HP, le Kremlin Bicetre, Ile-de-France, France, ⁸Service de Rhumatologie, Hôpital Bicêtre, AP-HP, Le Kremlin Bicetre, France, ⁹Sorbonne Université, Paris, ¹⁰Department of Internal Medicine, CHU de Rouen, UniRouen, Rouen, France., Rouen, France, ¹¹Université Paris Saclay, Department of Internal Medicine and clinical immunology, Bicêtre hospital, Assistance Publique-Hôpitaux de Paris, UMR1184 Inserm, CEA, Le Kremlin Bicêtre, France, Kremlin-Bicêtre, France, ¹²Department of Internal Medicine and Clinical Immunology, Dijon University Hospital, Dijon, France., Dijon, France, ¹³Dijon University Hospital, Dijon, France, ¹⁴Department of Internal Medicine, Foch Hospital, Suresnes, France., Suresnes, France, ¹⁵Department of Internal Medicine and RECIF, Amiens University Hospital, Université Picardie Jules Verne, Amiens, France., Amiens, France, ¹⁶Department of Internal Medicine and RECIF, Amiens-Picardie University Hospital, AMIENS, France, ¹⁷Department of Internal Medicine, Ambroise Paré Hospital, Assistance Publique - Hôpitaux de Paris, Université de Versailles Saint-Quentin-en-Yvelines, Boulogne-Billancourt, France, Boulogne Billancourt, France, ¹⁸University Paris Cité, Paris, France, ¹⁹Service de Médecine interne, Hôpital Cochin, AP-HP, Paris, Ile-de-France, France, ²⁰National Referral Center For Rare Systemic Autoimmune Diseases, Paris, France, ²¹National Referral Center For Rare Systemic Autoimmune Diseases, Paris, Ile-de-France, France, ²²Cochin hospital, Paris, France, ²³Department of Internal Medicine and clinical immunology, North-West National Reference Center for Rare Systemic Autoimmune Diseases iques et Auto-Immunes Rares du Nord-Ouest, Hôpital Claude Huriez, Université de Lille, France, Lille, France, ²⁴Service de Médecine Interne, Centre de Référence Maladies Systémiques Autoimmunes et Autoinflammatoires Rares d'Ile de France de l’Est et de l’Ouest, Hôpital Cochin, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, Ile-de-France, France

Meeting: ACR Convergence 2024

Keywords: Cohort Study, lupus-like disease, Therapy, complementary

Session Information

Date: Sunday, November 17, 2024

Title: Systemic Sclerosis & Related Disorders – Clinical Poster II

Session Type: Poster Session B

Session Time: 10:30AM-12:30PM

Background/Purpose: Mixed connective tissue disease (MCTD) is a rare systemic disorder that belongs to connective tissue diseases (CTD). Few studies are available on MCTD treatment. Taking advantage of the French MCTD cohort (1), we conducted an observational study to describe the therapeutic strategy used in MCTD.

Methods: We conducted an observational study within the French MCTD cohort (1). This cohort includes patients diagnosed with MCTD according to at least one of the four diagnostic criteria for MCTD and who, at disease onset, did not fulfill classification criteria for systemic sclerosis, systemic lupus erythematosus, Sjögren’s syndrome, idiopathic inflammatory myopathies, nor rheumatoid arthritis. Data regarding treatment were collected at MCTD diagnosis, during follow-up, and at the last follow-up (LFU).

Results: Of the 330 MCTD patients enrolled in the French MCTD cohort, 315 (96%) were included and followed up during 96 [40-156] months. Patient’s main characteristics and treatments are described in Table 1 and Table 2. At disease onset, 52 (17%) patients were treated with calcium channel blockers and/or nonsteroidal anti-inflammatory drugs only and/or no treatment at all, while 36 (11%) were treated with corticosteroids (CS) alone, 188 (60%) with hydroxychloroquine (HCQ) without disease-modifying antirheumatic drugs (DMARDs) or immunosuppressive treatment (IS), and 39 (12%) with DMARDs and/or IS. At LFU, patients received a lower median dose of CS compared to baseline (5 [5-10] vs 20 [10-40] mg/day; p< 0.001) and more frequently received DMARDs and/or IS than at baseline (p< 0.001). Overall, 10 (3%) patients remained treatment-free, 77 (24%) CS-free, and 148 (47%) were only treated with HCQ and/or low-dose CS at LFU. Among IS, anti-B cell agents were used in 39 (12%) patients during follow-up.

At baseline, patients with sclerodactyly (p=0.002), synovitis (p=0.002), dyspnea (p=0.02), pulmonary arterial hypertension (PAH) (p< 0.0001), interstitial lung disease (ILD) (p=0.01), pulmonary fibrosis (p< 0.0001), and elevated CK (p< 0.01), received significantly more frequently DMARDs and/or IS than others. When comparing MCTD patients who evolved to a differentiated CTD (MCTD-dCTD, n=86; 27%) to patients who remained classified as MCTD, MCTD-dCTD patients were less frequently treated with HCQ (± CS) (23 (27%) vs 144 (46%), p< 0.0001) and were more often treated with a DMARD and/or IS (61 (71%) vs 100 (44%), p< 0.0001). At LFU, they were less frequently treatment-free (7 (8%) vs 51 (22%), p< 0.01) and received higher doses of CS (median dose of 7.5 [5-10] vs. 5 [5-10] mg/day, p=0.004) than MCTD patients. Development of ILD and/or PAH, as well as ILD alone, was associated with a less frequent use of HCQ at baseline (p< 0.05) (Table 3).

Conclusion: Treatments prescribed in patients with MCTD reflect the wide spectrum of the disease. HCQ and CS are cornerstones of the treatment and are sufficient in nearly half of MCTD patients, reflecting the good prognosis of this disease. MCTD-dCTD patients more often require DMARDs or IS. This work suggests that HCQ could prevent the development of life-threatening complications in MCTD.

1. Chevalier K, et al. J Intern Med. 2023

Table 1 – Comparison of baseline characteristics of patients with MCTD according to treatment

Table 2 – Baseline, last follow-up and cumulative treatments used in patients with MCTD

Table 3 – Correlation of hydroxychloroquine with onset of interstitial lung disease and/or pulmonary arterial hypertension in patients with MCTD

Disclosures: K. Chevalier: None; B. Thoreau: None; M. Michel: None; B. Godeau: None; C. AGARD: None; T. Papo: None; K. Sacré: None; R. Seror: Amgen, 6, Boehringer-Ingelheim, 2, Bristol-Myers Squibb(BMS), 2, GlaxoSmithKlein(GSK), 2, Janssen, 2, Pfizer, 6, Roche, 6; X. Mariette: Bristol-Myers Squibb(BMS), 2, Galapagos, 2, GlaxoSmithKlein(GSK), 2, Novartis, 2, Pfizer, 2; P. Cacoub: None; Y. Benhamou: None; H. Levesque: None; C. Goujard: None; O. Lambotte: None; B. Bonnotte: None; M. SAMSON: AstraZeneca; Novartis; CSL Vifor; GlasgowSmithKline; Chugai; BOEHRINGER; FRESENIUS KABI, 2; F. Ackermann: None; J. Schmidt: None; P. Duhaut: None; J. Kahn: None; T. Hanslik: None; N. Costedoat-chalumeau: Bristol-Myers Squibb(BMS), 1; B. Terrier: AstraZeneca, 2, GlaxoSmithKline, 2, Novartis, 2, Vifor Pharma, 2; A. Régent: None; b. Dunogue: None; P. cohen: None; V. LE GUERN: None; E. Hachulla: None; L. Mouthon: None; B. Chaigne: None.

To cite this abstract in AMA style:

Chevalier K, Thoreau B, Michel M, Godeau B, AGARD C, Papo T, Sacré K, Seror R, Mariette X, Cacoub P, Benhamou Y, Levesque H, Goujard C, Lambotte O, Bonnotte B, SAMSON M, Ackermann F, Schmidt J, Duhaut P, Kahn J, Hanslik T, Costedoat-chalumeau N, Terrier B, Régent A, Dunogue b, cohen P, LE GUERN V, Hachulla E, Mouthon L, Chaigne B. Therapeutic Strategy in Patients with Mixed Connective Tissue Disease: A Multicenter Retrospective Study in the French MCTD Cohort [abstract]. Arthritis Rheumatol. 2024; 76 (suppl 9). https://acrabstracts.org/abstract/therapeutic-strategy-in-patients-with-mixed-connective-tissue-disease-a-multicenter-retrospective-study-in-the-french-mctd-cohort/. Accessed .

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