Publications by authors named "Grégory Pugnet"

68 Publications

Hematopoietic stem cell transplantation in chronic inflammatory demyelinating polyneuropathy: French experience about four patients, under the behalf of French society for bone marrow transplantation.

J Neurol 2021 Apr 2;268(4):1536-1539. Epub 2021 Mar 2.

Unité de Médecine Interne: Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Centre de Référence des Maladies auto-immunes systémiques Rares d'Ile-de-France MATHEC (FAI2R), Assistance Publique-Hôpitaux de Paris, 75010, Paris, France.

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http://dx.doi.org/10.1007/s00415-021-10452-6DOI Listing
April 2021

French recommendations for the management of systemic necrotizing vasculitides (polyarteritis nodosa and ANCA-associated vasculitides).

Orphanet J Rare Dis 2020 12 29;15(Suppl 2):351. Epub 2020 Dec 29.

Internal Medicine, CHU Cochin, AP-HP, Paris, France.

Systemic necrotizing vasculitis comprises a group of diseases resembling polyarteritis nodosa and anti-neutrophil cytoplasmic antibody-associated vasculitis (ANCA): granulomatosis with polyangiitis, eosinophilic granulomatosis with polyangiitis, and microscopic polyangiitis. The definitive diagnosis is made in cooperation with a reference center for autoimmune diseases and rare systemic diseases or a competency center. The management goals are: to obtain remission and, in the long term, healing; to reduce the risk of relapses; to limit and reduce the sequelae linked to the disease; to limit the side effects and the sequelae linked to the treatments; to improve or at least maintain the best possible quality of life; and to maintain socio-professional integration and/or allow a rapid return to school and/or professional activity. Information and therapeutic education of the patients and those around them are an integral part of the care. All health professionals and patients should be informed of the existence of patient associations. The treatment of vasculitis is based on variable combinations of glucocorticoids and immunosuppressants, chosen and adapted according to the disease concerned, the severity and/or extent of the disease, and the underlying factors (age, kidney function, etc.). Follow-up clinical and paraclinical examinations must be carried out regularly to clarify the progression of the disease, detect and manage treatment failures and possible relapses early on, and limit sequelae and complications (early then late) related to the disease or treatment. A distinction is made between the induction therapy, lasting approximately 3-6 months and aimed at putting the disease into remission, and the maintenance treatment, lasting 12-48 months, or even longer. The role of the increase or testing positive again for ANCA as a predictor of a relapse, which has long been controversial, now seems to have greater consensus: Anti-myeloperoxidase ANCAs are less often associated with a relapse of vasculitis than anti-PR3 ANCA.
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http://dx.doi.org/10.1186/s13023-020-01621-3DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7771069PMC
December 2020

Assessment of the Accuracy of Using Codes to Identify Systemic Sclerosis.

Clin Epidemiol 2020 8;12:1355-1359. Epub 2020 Dec 8.

Department of Internal Medicine, CHU Toulouse, Toulouse, France.

Importance: With the increased use of data from electronic medical records for research, it is important to validate in-patient electronic health records/hospital electronic health records for specific diseases identification using International Classification of Diseases, Tenth Revision () codes.

Objective: To assess the accuracy of using codes to identify systemic sclerosis (SSc) in the French hospital database.

Design Setting And Participants: Electronic health record database analysis. The setting of the study's in-patient database was the Toulouse University Hospital, a tertiary referral center (2880 beds) that serves approximately 2.9 million inhabitants. Participants were patients with discharge diagnosis codes of SSc seen at Toulouse University Hospital between January 1, 2010, and December 31, 2017.

Main Outcomes And Measures: The main outcome was the positive predictive value (PPV) of discharge diagnosis codes for identifying SSc. The PPVs were calculated by determining the ratio of the confirmed cases found by medical record review to the total number of cases identified by code.

Results: Of the 2766 hospital stays, 216 patients were identified by an SSc discharge diagnosis code. Two hundred were confirmed as SSc after medical record review. The overall PPV was 93% (95% CI, 88-95%). The PPV for limited cutaneous SSc was 95% (95% CI, 85-98%).

Conclusions And Relevance: Our results suggest that using codes alone to capture SSc is reliable in The French hospital database.
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http://dx.doi.org/10.2147/CLEP.S260733DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7733391PMC
December 2020

Osteitis in Systemic Sclerosis: a nationwide case-control retrospective study (SCLEROS Study).

Arthritis Care Res (Hoboken) 2020 Dec 5. Epub 2020 Dec 5.

Service de Médecine Interne, Centre de Référence Maladies systémiques auto-immunes rares d'Ile de France, Hôpital Cochin, Université Paris Descartes, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris, France.

Objective: Systemic sclerosis (SSc) is an autoimmune (AI) connective tissue disorder characterized by skin fibrosis, vasculopathy and dysimmunity. Data regarding osteitis in SSc are scarce.

Method: We performed a nationwide multicentre retrospective case-control study including patients with SSc according to the 2013 ACR/EULAR classification, with a diagnosis of osteitis. The objectives of the study were to describe, to characterize, and to identify associated factors for osteitis in patients with SSc.

Results: Forty-eight patients were included. Twenty-six patients (54.1%) had osteitis beneath digital tip ulcers. Physical symptoms included: pain (36/48, 75%), erythema (35/48, 73%), and local warmth (35/48, 73%). Thirty-one (65%) patients had C-reactive protein levels >2 mg/L (8 [2.7 - 44.3] mg/L). On X-ray, CT-scans or MRI, osteitis was characterized by swelling or abscess of soft tissues with acro-osteolysis or lysis in 28 patients (58%). Microbiological sampling was performed in 45 (94%) patients. Most pathogens were Staphylococcus aureus (43.8%); anaerobes and Enterobacteriaceae (29.1%) and Pseudomonas aeruginosa (10.4%). Management comprised antibiotics in 37 (77.1%) patients and/or surgery in 26 (54.2%). Fluoroquinolones were used in 22 (45.8%) patients and amoxicillin + beta-lactamase inhibitor in 7 (14.6%). Six (12.6%) patients relapsed, 6 (12.6%) patients had osteitis recurrence, 15 (32%) sequelae, and 2 patients had septic shock and died.

Conclusion: This study confirmed digital tip ulcers as an associated factor for osteitis, and revealed a high rate of functional sequelae. Antimicrobial therapy with oral fluoroquinolone or intravenous amoxicillin and beta-lactamase inhibitor are used as first-line antibiotherapy in SSc patients with osteitis.
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http://dx.doi.org/10.1002/acr.24530DOI Listing
December 2020

[Crohn's disease and autologous hemapoietic cell transplantation: Guidelines from the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC)].

Bull Cancer 2020 Dec 14;107(12S):S140-S150. Epub 2020 Oct 14.

Centre de référence des maladies auto-immunes systémiques rares d'Île-de-France, hôpital St-Louis (AP-HP), unité de médecine interne : maladies auto-immunes et pathologie vasculaire (UF 04), 1, avenue Claude-Vellefaux, 75010 Paris, France; Université Paris-Denis-Diderot, institut de recherche Saint-Louis, EA 3518, Sorbonne Paris Cité, France; McGill University, department of internal medicine, Montreal, Canada. Electronic address:

Crohn's Disease (CD) is an auto-inflammatory disease, which may involve the entire gastro-intestinal tract. CD is diagnosed on several clinical, biological, endoscopic and histological criteria. First line therapy is based on oral or iv steroids. In case of steroids dependence or resistance, several types of immunosuppressive or immunomodulating therapies are available: classical antimetabolites (thiopurines or methotrexate) or monoclonal antibodies against TNFα, against interleukin 12/23 or against integrin. Nonetheless, Crohn's disease may remain active despite the use of several lines of therapy. In such cases, autologous hematopoietic cell transplantation (AHCT) is an effective therapeutic option in highly selected CD patients with specific criteria. The MATHEC-SFGM-TC Good Clinical Practice Guidelines (GCPG) were developed by a multidisciplinary group of experts including gastroenterologists, hematologists and members of the reference center for stem cell therapy in auto-immune diseases (MATHEC), including members of the French groupe d'étude thérapeutique des affections inflammatoires du tube digestif(GETAID) under the auspices of the French speaking Society of bone marrow transplantation and cellular therapy (SFGM-TC). The aim of the present guidelines is to define the eligibility criteria for CD patients when candidates to AHCT, the procedures for mobilization of hematopoietic stem cell (HSC), conditioning regimen and standardized follow-up after AHCT including monitoring of gastroenterological treatments during AHCT and thereafter throughout all follow-up.
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http://dx.doi.org/10.1016/j.bulcan.2020.08.009DOI Listing
December 2020

Use of Biologics to Treat Relapsing and/or Refractory Eosinophilic Granulomatosis With Polyangiitis: Data From a European Collaborative Study.

Arthritis Rheumatol 2021 03 23;73(3):498-503. Epub 2021 Jan 23.

National Referral Center for Rare Systemic and Autoimmune Diseases, Hôpital Cochin, AP-HP, Université Paris Descartes, Paris, France.

Objective: To describe the efficacy and safety of biologics for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA).

Methods: A retrospective European collaborative study was conducted in patients with EGPA who received treatment with biologics for refractory and/or relapsing disease.

Results: Among the 147 patients with EGPA included in the study, 63 received rituximab (RTX), 51 received mepolizumab (MEPO), and 33 received omalizumab (OMA). At the time of inclusion, the median Birmingham Vasculitis Activity Score (BVAS) was 8.5 (interquartile range [IQR] 5-13) in the RTX group, while the median BVAS in the OMA group was 2 (IQR 1-4.5) and the median BVAS in the MEPO group was 2 (IQR 1-5). In patients receiving RTX, the median BVAS declined both at 6 months (median 1, IQR 0-4.5) and at 12 months (median 0, IQR 0-2), and the frequency of remission, partial response, treatment failure, and stopping treatment due to adverse events was 49%, 24%, 24%, and 3%, respectively. For the treatment of glucocorticoid (GC)-dependent asthma, patients who received MEPO had a much better GC-sparing effect and overall response than did patients who received OMA. The frequency of remission, partial response, treatment failure, and stopping treatment due to adverse events was 15%, 33%, 48%, and 4%, respectively, in the OMA group and 78%, 10%, 8%, and 4%, respectively, in the MEPO group. Remission rates at 12 months were 76% and 82% among patients receiving MEPO at a doses of 100 mg and 300 mg, respectively.

Conclusion: These results suggest that RTX could be effective in treating relapses of EGPA vasculitis. MEPO is highly effective with a good safety profile in patients with GC-dependent asthma. Our data suggest that 100 mg MEPO monthly could be an acceptable dosage for first-line therapy in selected instances of EGPA, recognizing, however, that this has not been compared to the validated dosage of 300 mg monthly.
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http://dx.doi.org/10.1002/art.41534DOI Listing
March 2021

Temporal Arteritis Revealing Antineutrophil Cytoplasmic Antibody-Associated Vasculitides: A Case-Control Study.

Arthritis Rheumatol 2021 02 21;73(2):286-294. Epub 2020 Dec 21.

Centre de Référence des Maladies Auto-Immunes Systémiques Rares d'Ile-de-France, Hôpital Cochin, and Université Paris Descartes, Paris, France.

Objective: Temporal arteritis (TA) is a typical manifestation of giant cell arteritis (GCA). Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitides (AAVs) are rarely revealed by TA manifestations, leading to a risk of misdiagnosis of GCA and inappropriate treatments. This study was undertaken to describe the clinical, biologic, and histologic presentations and outcomes in cases of TA revealing AAV (TA-AAV) compared to controls with classic GCA.

Methods: In this retrospective case-control study, the characteristics of patients with TA-AAV were compared to those of control subjects with classic GCA. Log-rank test, with hazard ratios (HRs) and 95% confidence intervals (95% CIs), was used to assess the risk of treatment failure.

Results: Fifty patients with TA-AAV (median age 70 years) were included. Thirty-three patients (66%) presented with atypical symptoms of GCA (ear, nose, and throat involvement in 32% of patients, and renal, pulmonary, and neurologic involvement in 26%, 20%, and 16% of patients, respectively). Blood samples were screened for ANCAs at the time of disease onset in 33 patients, and results were positive in 88%, leading to a diagnosis of early TA-AAV in 20 patients. The diagnosis of AAV was delayed a median interval of 15 months in 30 patients. Compared to controls with GCA, patients with TA-AAV were younger (median age 70 years versus 74 years), were more frequently men (48% versus 30%), and had high frequencies of atypical manifestations and higher C-reactive protein levels (median 10.8 mg/dl versus 7.0 mg/dl). In patients with TA-AAV, temporal artery biopsy (TAB) showed fibrinoid necrosis and small branch vasculitis in 23% of patients each, whereas neither of these characteristics was evident in controls with GCA. Treatment failure-free survival was comparable between early TA-AAV cases and GCA controls, whereas those with delayed TA-AAV had a significantly higher risk of treatment failure compared to controls (HR 3.85, 95% CI 1.97-7.51; P < 0.0001).

Conclusion: TA-AAV should be considered diagnostically in cases of atypical manifestations of GCA, refractoriness to glucocorticoid treatment, or early relapse. Analysis of TAB specimens for the detection of small branch vasculitis and/or fibrinoid necrosis could be useful. Detection of ANCAs should be performed in cases of suspected GCA with atypical clinical features and/or evidence of temporal artery abnormalities on TAB.
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http://dx.doi.org/10.1002/art.41527DOI Listing
February 2021

Renal involvement in eosinophilic granulomatosis with polyangiitis (EGPA): a multicentric retrospective study of 63 biopsy-proven cases.

Rheumatology (Oxford) 2021 01;60(1):359-365

Department of Nephrology, Hôpital Européen Georges Pompidou, AP-HP, Paris, France.

Objective: Eosinophilic granulomatosis with polyangiitis (EGPA) is a systemic small-vessel vasculitis characterized by asthma, hypereosinophilia and ANCA positivity in 40% of patients. Renal involvement is rare and poorly described, leading to this renal biopsy-proven based study in a large EGPA cohort.

Methods: We conducted a retrospective multicentre study including patients fulfilling the 1990 ACR criteria and/or the 2012 revised Chapel Hill Consensus Conference criteria for EGPA and/or the modified criteria of the MIRRA trial, with biopsy-proven nephropathy.

Results: Sixty-three patients [27 women, median age 60 years (18-83)] were included. Renal disease was present at vasculitis diagnosis in 54 patients (86%). ANCA were positive in 53 cases (84%) with anti-MPO specificity in 44 (83%). All patients had late-onset asthma. Peripheral neuropathy was present in 29 cases (46%), alveolar haemorrhage in 10 (16%). The most common renal presentation was acute renal failure (75%). Renal biopsy revealed pauci-immune necrotizing GN in 49 cases (78%). Membranous nephropathy (10%) and membranoproliferative GN (3%) were mostly observed in ANCA-negative patients. Pure acute interstitial nephritis was found in six cases (10%); important interstitial inflammation was observed in 28 (44%). All patients received steroids with adjunctive immunosuppression in 54 cases (86%). After a median follow-up of 51 months (1-296), 58 patients (92%) were alive, nine (14%) were on chronic dialysis and two (3%) had undergone kidney transplantation.

Conclusion: Necrotizing pauci-immune GN is the most common renal presentation in ANCA-positive EGPA. ANCA-negative patients had frequent atypical renal presentation with other glomerulopathies such as membranous nephropathy. An important eosinophilic interstitial infiltration was observed in almost 50% of cases.
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http://dx.doi.org/10.1093/rheumatology/keaa416DOI Listing
January 2021

Pulmonary mucormycosis following autologous hematopoietic stem cell transplantation for rapidly progressive diffuse cutaneous systemic sclerosis: A case report.

Medicine (Baltimore) 2020 Jul;99(31):e21431

Department of Internal Medicine.

Rationale: The use of autologous hematopoietic stem cell transplantation (AHSCT) for autoimmune diseases has become the first indication for transplant in nonmalignant disease. Mucormycosis is a rare invasive infection with increasing incidence in patients treated with AHSCT. We report the first case of pulmonary mucormycosis following AHSCT for systemic sclerosis (SSc).

Patient Concerns: A 24-year-old woman with rapidly progressive diffuse cutaneous SSc presented with an acute respiratory distress syndrome 6 days after AHSCT.

Diagnoses: The results of clinical and computed tomography scan were consistent with pulmonary mucormycosis and the diagnosis was confirmed by a positive Mucorales Polymerase Chain Reaction on a peripheral blood sample.

Interventions And Outcomes: Early antifungal therapy by intravenous amphotericin B provided rapid improvement within 4 days and sustained recovery after 2 years of follow-up.

Lessons: With the progressively increasing use of AHSCT and other stem cell therapy for treatment of severe SSc and other autoimmune diseases, the potential onset of rare post-transplant fungal infections, such as mucormycosis, requires careful patient monitoring and better awareness of early initiation of adequate therapy.
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http://dx.doi.org/10.1097/MD.0000000000021431DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7402716PMC
July 2020

Quality of life in patients with uveitis: data from the ULISSE study (Uveitis: cLInical and medico-economic evaluation of a Standardised Strategy for the Etiological diagnosis).

Br J Ophthalmol 2020 Jul 24. Epub 2020 Jul 24.

Internal Medicine, Hospices Civils De Lyon, Lyon, France.

Aims: To assess vision-related (VR-QOL) and health-related quality of life (HR-QOL) in a large series of patients with de novo uveitis at baseline and 6-month follow-up.

Methods: Non-inferiority, prospective, multicentre, cluster randomised controlled trial registered under the Unique Identifier: NCT01162070. VR-QOL and HR-QOL were assessed by the 25-item National Eye Institute Visual Function Questionnaire (VFQ-25) and the Medical Outcomes Study 36-item Short Form Survey (SF-36).

Results: At inclusion, 466 patients completed the VFQ-25. The mean composite score was 80.0 (±16.7). In multivariate analysis, higher age, female sex and insidious onset were significantly associated with lower QOL. At 6 months, 138 patients completed the VFQ-25, with a significantly higher mean composite score of 82.6 (±16.7). SF-36 mental component was 42.9 (±11.3) and physical component was 47.2 (±8.5) at inclusion (n=425). HR-QOL improvement at 6 months was not clinically significant.

Conclusion: QOL seems relatively well preserved in this cohort; only VR-QOL improved significantly at 6 months, especially in patients with low initial visual acuity.
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http://dx.doi.org/10.1136/bjophthalmol-2020-315862DOI Listing
July 2020

ANCA-associated vasculitides: Recommendations of the French Vasculitis Study Group on the use of immunosuppressants and biotherapies for remission induction and maintenance.

Presse Med 2020 Oct 6;49(3):104031. Epub 2020 Jul 6.

Service de médecine interne, hôpital Cochin, université Paris Descartes, Sorbonne Paris Cité, Paris, France; Centre de référence pour les maladies auto-immunes rares, hôpital Cochin, Paris, France.

Treatment of vasculitides associated with anti-neutrophil cytoplasm antibodies (ANCA) (AAVs) has evolved dramatically in recent years, particularly since the demonstration of rituximab efficacy as remission induction and maintenance therapy for granulomatosis with polyangiitis and microscopic polyangiitis. In 2013, the French Vasculitis Study Group (FVSG) published recommendations for its use by clinicians. Since then, new data have made it possible to better specify and codify prescription of rituximab to treat AAVs. Herein, the FVSG Recommendations Committee, an expert panel comprised of physicians with extensive experience in the treatment and management of vasculitides, presents its consensus guidelines based on literature analysis, the results of prospective therapeutic trials and personal experience.
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http://dx.doi.org/10.1016/j.lpm.2020.104031DOI Listing
October 2020

Long-Term Rituximab Use to Maintain Remission of Antineutrophil Cytoplasmic Antibody-Associated Vasculitis: A Randomized Trial.

Ann Intern Med 2020 08 2;173(3):179-187. Epub 2020 Jun 2.

Cochin Hospital, Paris Descartes University, Paris, France (P.C., C.V., X.P., B.T., L.M., L.G.).

Background: Biannual rituximab infusions over 18 months effectively maintain remission after a "standard" remission induction regimen for patients with antineutrophil cytoplasmic antibody-associated vasculitis (AAV).

Objective: To evaluate the efficacy of prolonged rituximab therapy in preventing AAV relapses in patients with granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) who have achieved complete remission after completing an 18-month maintenance regimen.

Design: Randomized controlled trial. (ClinicalTrials.gov: NCT02433522).

Setting: 39 clinical centers in France.

Patients: 68 patients with GPA and 29 with MPA who achieved complete remission after the first phase of maintenance therapy.

Intervention: Rituximab or placebo infusion every 6 months for 18 months (4 infusions).

Measurements: The primary end point was relapse-free survival at month 28. Relapse was defined as new or reappearing symptoms or worsening disease, with a Birmingham Vasculitis Activity Score greater than 0.

Results: From March 2015 to April 2016, 97 patients (mean age, 63.9 years; 35% women) were randomly assigned, 50 to the rituximab and 47 to the placebo group. Relapse-free survival estimates at month 28 were 96% (95% CI, 91% to 100%) and 74% (CI, 63% to 88%) in the rituximab and placebo groups, respectively, an absolute difference of 22% (CI, 9% to 36%) with a hazard ratio of 7.5 (CI, 1.67 to 33.7) ( = 0.008). Major relapse-free survival estimates at month 28 were 100% (CI, 93% to 100%) versus 87% (CI, 78% to 97%) ( = 0.009), respectively. At least 1 serious adverse event developed in 12 patients (24%) in the rituximab group (with 9 infectious serious adverse events occurring among 6 patients [12%]) versus 14 patients (30%) in the placebo group (with 6 infectious serious adverse events developing among 4 patients [9%]). No deaths occurred in either group.

Limitation: Potential selection bias based on previous rituximab response and tolerance.

Conclusion: Extended therapy with biannual rituximab infusions over 18 months was associated with a lower incidence of AAV relapse compared with standard maintenance therapy.

Primary Funding Source: French Ministry of Health and Hoffmann-La Roche.
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http://dx.doi.org/10.7326/M19-3827DOI Listing
August 2020

Hydroxychloroquine in Coronavirus Disease 2019 Patients: What Still Needs to Be Known About the Kinetics.

Clin Infect Dis 2020 12;71(11):2962-2964

UMR INTHERES, INRA-ENVT, Toulouse, France.

Different dosage regimens of hydroxychloroquine are used to manage coronavirus disease 2019 (COVID-19) patients, without information on the pharmacokinetics in this population. Blood samples (n = 101) were collected from 57 COVID-19 patients for 7 days, and concentrations were compared with simulated kinetic profiles. Hydroxychloroquine exposure is low and cannot be predicted by other populations.
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http://dx.doi.org/10.1093/cid/ciaa558DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7239205PMC
December 2020

Reducing the initial number of rituximab maintenance-therapy infusions for ANCA-associated vasculitides: randomized-trial post-hoc analysis.

Rheumatology (Oxford) 2020 10;59(10):2970-2975

Centre de Référence Maladies Systémiques et Auto-Immunes Rares, Université Paris Descartes, APHP, Hôpital Cochin, Paris.

Objective: The randomized, controlled MAINRITSAN2 trial was designed to compare the capacity of an individually tailored therapy [randomization day 0 (D0)], with reinfusion only when CD19+ lymphocytes or ANCA had reappeared, or if the latter's titre rose markedly, with that of five fixed-schedule 500-mg rituximab infusions [D0 + D14, then months (M) 6, 12 and 18] to maintain ANCA-associated vasculitis (AAV) remissions. Relapse rates did not differ at M28. This ancillary study was undertaken to evaluate the effect of omitting the D14 rituximab infusion on AAV relapse rates at M12.

Methods: MAINRITSAN2 trial data were subjected to post-hoc analyses of M3, M6, M9 and M12 relapse-free survival rates in each arm as primary end points. Exploratory subgroup analyses were run according to CYC or rituximab induction and newly diagnosed or relapsing AAV.

Results: At M3, M6, M9 and M12, respectively, among the 161 patients included, 79/80 (98.8%), 76/80 (95%), 74/80 (92.5%) and 73/80 (91.3%) from D0, and 80/81 (98.8%), 78/81 (96.3%), 76/81 (93.8%) and 76/81 (93.8%) from D0+D14 groups were alive and relapse-free. No between-group differences were observed. Results were not affected by CYC or rituximab induction, or newly diagnosed or relapsing AAV.

Conclusions: We were not able to detect a difference between the relapse-free survival rates for up to M12 for the D0 and D0+D14 rituximab-infusion groups, which could suggest that omitting the D14 rituximab remission-maintenance dose did not modify the short-term relapse-free rate. Nevertheless, results at M12 may also have been influenced by the rituximab-infusion strategies for both groups.
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http://dx.doi.org/10.1093/rheumatology/kez621DOI Listing
October 2020

Early trajectories of skin thickening are associated with severity and mortality in systemic sclerosis.

Arthritis Res Ther 2020 02 18;22(1):30. Epub 2020 Feb 18.

Univ. Lille, Institute for Translational Research in Inflammation (INFINITE), F-59000, Lille, France.

Background: Systemic sclerosis (SSc) is a severe and highly heterogeneous disease. The modified Rodnan skin score (mRSS) is a widely used tool for the assessment of the extent and degree of skin thickness. This study aimed to identify the classes of patients with early similar skin thickening trajectories without any a priori assumptions and study their associations with organ involvement and survival.

Methods: From the French SSc national cohort, patients with a disease duration of less than 2 years at inclusion and with at least 2 mRSS available within the first 4 years of follow-up were enrolled. Classes of patients with similar mRSS trajectories were identified based on a latent class mixed model. The clinical characteristics and survival rate were compared between the obtained classes.

Results: A total of 198 patients fulfilled the inclusion criteria, with a total of 641 mRSS available. The median disease duration and follow-up were 0.8 (interquartile range 0.4; 1.2) and 6.3 (3.8; 8.9) years, respectively. Individual trajectories of mRSS were highly heterogeneous between patients. Models with 1-6 latent classes of trajectories were sequentially assessed, and the 5-class model represented the best fit to data. Each class was characterized by a unique global trajectory of mRSS. The median disease duration did not differ significantly between classes. Baseline organ involvement was more frequent in classes with significant change over time (classes 2-5) than in class 1 (low baseline mRSS without significant change over time). Using Cox regression, we observed a progressively increasing risk of death from classes 1 to 5.

Conclusions: Early identification of clinical phenotype based on skin thickening trajectories could predict morbi-mortality in SSc. This study suggested that mRSS trajectories characterization might be pivotal for clinical practice and future trial designs.
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http://dx.doi.org/10.1186/s13075-020-2113-6DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7029583PMC
February 2020

Evaluating the cost-consequence of a standardized strategy for the etiological diagnosis of uveitis (ULISSE study).

PLoS One 2020 14;15(2):e0228918. Epub 2020 Feb 14.

Department of Internal Medicine, Hospices Civils de Lyon, Croix-Rousse Hospital, Lyon, France.

Main Objective: To prospectively assess the cost-consequence of a standardized diagnostic strategy as to compared to an open one for the etiological diagnosis of uveitis.

Design: This was a prospective, non-inferiority, multicentre, randomized controlled trial.

Methods: We included all consecutive patients with uveitis who had visited at least one of the Departments of Ophthalmology. In the standardized group, patients had a minimal work-up regardless of the type of uveitis (including evaluation of the CBC, ESR, C-reactive protein, tuberculin skin test, syphilis serology and chest X-ray). Depending on ophthalmological findings, further investigations could be performed. In the open strategy, ophthalmologists were free to order any kind of investigation. The main outcome was the mean cost per patient of each strategy.

Results: 903 uveitis patients were included from January, 2010 to May, 2013. The mean cost per patient of the standardized strategy was 182.97 euros [CI 95% (173.14; 192.80)], and the mean cost per patient of the open strategy was 251.75 euros [CI 95% (229.24; 274.25)]. Therefore, the mean cost per patient of the standardized strategy was significantly lower than the mean cost per patient of the open strategy (p<0.001). There were significantly fewer visits (p<0.001), fewer radiological procedures (p<0.004) and fewer laboratory investigations (p<0.001) in the standardized group.

Conclusion: A standardized strategy is a cost-saving approach for the etiological diagnosis of uveitis.
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http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0228918PLOS
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7021300PMC
May 2020

Autologous stem cell transplantation for progressive systemic sclerosis: a prospective non-interventional study from the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party.

Haematologica 2021 Feb 1;106(2):375-383. Epub 2021 Feb 1.

Assistance Publique-Hopitaux de Paris, Saint-Louis Hospital, Paris, France.

Three randomized controlled trials in early severe systemic sclerosis demonstrated that autologous hematopoietic stem cell transplantation was superior to standard cyclophosphamide therapy. This European Society for Blood and Marrow Transplantation multi-center prospective non-interventional study was designed to further decipher efficacy and safety of this procedure for severe systemic sclerosis patients in real-life practice and to search for prognostic factors. All consecutive adult systemic sclerosis patients undergoing a first autologous hematopoietic stem cell transplantation between December 2012 and February 2016 were prospectively included in the study. Primary endpoint was progression free survival. Secondary endpoints were overall survival, non-relapse mortality, response and incidence of progression. Eighty systemic sclerosis patients were included. Median follow-up duration was 24 (6-57) months after stem cell transplantation using cyclophosphamide plus antithymocyte globulins conditioning for all, with CD34+ selection in 35 patients. At 2 years, progression free survival was 81.8%, overall survival was 90%, response was 88.7% and incidence of progression was 11.9%. The 100 days non-relapse mortality was 6.25% (n=5) with four deaths from cardiac event, including three due to cyclophosphamide toxicity. Modified Rodnan skin score and forced vital capacity improved with time (p< 0.001). By multivariate analysis, baseline skin score >24 and older age at transplant were associated with lower progression free survival (Hazard ration 3.32) and 1.77, respectively). CD34+-selection was associated with better response (Hazard ration: 0.46). This study confirms the efficacy of autologous stem cell transplantation in real-life practice for severe systemic sclerosis using non myeloablative conditioning. Careful cardio-pulmonary assessment to identify organ involvement at patient referral, reduced cyclophosphamide doses and CD34+ selection may improve outcomes. The study was registered at ClinicalTrials.gov: NCT02516124.
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http://dx.doi.org/10.3324/haematol.2019.230128DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7849556PMC
February 2021

Survival Improved in Patients Aged ≤ 70 Years With Systemic Sclerosis-Associated Pulmonary Arterial Hypertension During the Period 2006 to 2017 in France.

Chest 2020 04 19;157(4):945-954. Epub 2019 Nov 19.

Université de Lille, Inserm, CHU Lille, U995-LIRIC-Lille Inflammation Research International Center, F-59000 Lille, France.

Background: To date, nothing is known about the evolution of survival in systemic sclerosis-associated pulmonary arterial hypertension (PAH) over the last decade.

Methods: This study used a multivariate Cox regression model adjusted for clinically relevant baseline confounders to assess the association between the occurrence of death and date of PAH diagnosis comparing two periods of the same duration (2006-2011 vs 2012-2017). Interactions between the two diagnosis periods and baseline variables were tested.

Results: A total of 306 incident patients were included, 167 (54.6%) with a PAH diagnosis occurring in 2006 to 2011 and 139 (45.4%) in 2012 to 2017. No significant difference in survival was observed between patients diagnosed with PAH in 2012 to 2017 compared with those diagnosed in 2006 to 2011 (hazard ratio [HR], 0.76 [0.46-1.26]; P = .29). A significant interaction was observed between PAH diagnosis periods and age (P = .05). When stratifying according to age (based on the median age of 70 years), a significant increase was observed in survival in patients aged ≤ 70 years between the 2006 to 2011 period and the 2012 to 2017 period (HR, 0.40 [0.17-0.99]; P = .046) but not in older patients (HR, 1.29 [0.67-2.51]; P = .44). A significantly higher proportion of initial (ie, within the first 4 months) endothelin receptor antagonist/phosphodiesterase type 5 inhibitor combination therapy was observed in younger patients diagnosed from 2012 to 2017 vs those diagnosed from 2006 to 2011 (42.9% vs 19.5%; P = .002) but not in older patients.

Conclusions: Over the period 2006 to 2017, survival in systemic sclerosis-associated PAH improved over time in patients aged ≤ 70 years but not in older patients. Further investigations are needed to confirm this relation, as general improvement in medical care and management may also be a possible explanation.
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http://dx.doi.org/10.1016/j.chest.2019.10.045DOI Listing
April 2020

Cardiac magnetic resonance imaging with late gadolinium enhancement in acute myocarditis: Towards differentiation between immune-mediated and viral-related aetiologies.

Arch Cardiovasc Dis 2019 Oct 21;112(10):559-566. Epub 2019 Oct 21.

Department of Cardiology, University Hospital of Rangueil, 31059 Toulouse, France; Cardiac Imaging Centre, Toulouse University Hospital, 31300 Toulouse, France; Department of Nuclear Medicine, University Hospital of Rangueil, 31059 Toulouse, France; Medical School, Toulouse III Paul-Sabatier University, 31400 Toulouse, France. Electronic address:

Background: Diagnosing immune-mediated myocarditis is challenging because of non-specific clinical signs and symptoms. Cardiac magnetic resonance imaging (CMR) provides subepicardial late gadolinium enhancement (LGE) in the setting of acute myocarditis, but the diagnostic value of LGE pattern for differentiating between immune-mediated and viral-related aetiologies remains unknown.

Aims: To determine the value of LGE pattern for differentiating between immune-mediated and viral-related aetiologies in patients with acute myocarditis.

Methods: One hundred and five patients with acute myocarditis who underwent CMR, including LGE variables, were included retrospectively. Viral-related aetiology was retained with a negative autoimmune and autoinflammatory assessment at diagnosis and 6-month follow-up.

Results: Aetiology was immune-mediated in 31 patients and viral-related in 74 patients. Patients with immune-mediated myocarditis were older (55±16 vs. 31±12years; P<0.001) and more likely to be female (52% vs. 14%; P<0.001) than those with viral-related myocarditis. There was no difference in left ventricular ejection fraction between the immune-mediated and viral-related myocarditis groups (53±15% vs. 57±8%; P=0.61). Regarding LGE, patients with viral-related myocarditis were more likely to have basal anteroseptal, mid anteroseptal, mid anterior and basal anterolateral location. Patients with immune-mediated myocarditis were more likely to have apical septal, apical inferior, apical lateral, mid anterolateral and basal inferior location. Segments with difference in prevalence of LGE between aetiologies were summed to build a score where positive significant association with immune-mediated myocarditis was quoted 1 and positive significant association with viral-related myocarditis was quoted -1. A score≥0 differentiated immune-mediated from viral-related myocarditis with 94% sensitivity and 77% specificity (area under the receiver operating characteristic curve 0.88; P<0.001).

Conclusion: CMR provides arguments for differentiating immune-mediated from viral-related acute myocarditis by showing preferential LGE localization in apical septal, apical inferior, apical lateral and basal inferior segments.
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http://dx.doi.org/10.1016/j.acvd.2019.09.001DOI Listing
October 2019

Usual interstitial pneumonia in ANCA-associated vasculitis: A poor prognostic factor.

J Autoimmun 2020 01 27;106:102338. Epub 2019 Sep 27.

Department of Internal Medicine and Clinical Immunology, CHU Dijon Bourgogne, Dijon, France.

Background: Progressive fibrosing interstitial lung disease (ILD) is rarely associated with antineutrophil cytoplasm antibody (ANCA)-associated vasculitis (AAV). This study focused on the outcomes of ILD patients with associated AAV (AAV-ILD).

Methods: AAV-ILD (cases: microscopic polyangiitis (MPA) or granulomatosis with polyangiitis (GPA) with ILD) were compared to AAV patients without ILD (controls). ILD was defined as a usual interstitial pneumonia (UIP) or non-specific interstitial pneumonia (NSIP) pattern. Two controls were matched to each case for age (>or ≤65 years), ANCA status (PR3-or MPO-positive) and creatininemia (≥or <150 μmol/L).

Results: Sixty-two cases (89% MPO-ANCA+) were included. Median age at AAV diagnosis was 66 years. ILD (63% UIP), was diagnosed before (52%) or simultaneously (39%) with AAV. Cases versus 124 controls less frequently had systemic vasculitis symptoms. One-, 3- and 5-year overall survival rates, respectively, were: 96.7%, 80% and 66% for cases versus 93.5%, 89.6% and 83.8% for controls (p = 0.008). Multivariate analyses retained age >65 years (hazard ratio (HR) 4.54; p < 0.001), alveolar haemorrhage (HR 2.25; p = 0.019) and UIP (HR 2.73; p = 0.002), but not immunosuppressant use, as factors independently associated with shorter survival.

Conclusion: For AAV-ILD patients, only UIP was associated with poorer prognosis. Immunosuppressants did not improve the AAV-ILD prognosis. But in analogy to idiopathic pulmonary fibrosis, anti-fibrosing agents might be useful and should be assessed in AAV-ILD patients with a UIP pattern.
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http://dx.doi.org/10.1016/j.jaut.2019.102338DOI Listing
January 2020

High rate of indeterminate results of the QuantiFERON-TB Gold in-tube test, third generation, in patients with systemic vasculitis.

Rheumatology (Oxford) 2020 05;59(5):1006-1010

Department of Internal Medicine.

Objectives: To describe the frequency of QuantiFERON-TB Gold in-tube test® (QFT-GIT) indeterminate results due to no response to phytohaemagglutinin A stimulation in the control tube in vasculitis patients prior to immunosuppressant therapy; and to compare it with other groups of patients.

Methods: This was a single-centre, retrospective study. Patients and controls were included between 1 January 2008 and 31 December 2015. We assessed the rate of indeterminate results of the QFT-GIT in 38 patients with systemic vasculitis prior to any corticosteroid or immunosuppressant therapy, compared with 40 non-vasculitis patients with biological inflammatory syndrome, and 310 non-immunosuppressed patients matched for gender and age.

Results: Indeterminate results due to no response to phytohaemagglutinin A were more frequent in vasculitis patients (21.1%) compared with non-vasculitis patients with biological inflammatory syndrome (7.5%) (Fisher's exact test: P = 0.11) and to anonymized controls (7%) (P = 0.009). Responses to phytohaemagglutinin A were significantly lower in vasculitis patients compared with other groups (Kruskal-Wallis test: P < 0.0001) and compared with non-vasculitis patients with biological inflammatory syndrome (P = 0.0015). The multivariable analysis identified as independent predictors of an indeterminate result of the QFT-GIT: the presence of systemic vasculitis (odds ratio 9.64 [1.14-81.3], P = 0.037) and a high neutrophil-to-lymphocyte ratio (odds ratio 1.70 [1.21-2.37], P = 0.002). One patient with an indeterminate result of QFT-GIT developed active tuberculosis after one year of corticosteroid therapy for giant cell arteritis.

Conclusion: Our results question the reliability of QFT-GIT to rule out latent tuberculosis in vasculitis patients at diagnosis, prior to immunosuppressant therapy.
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http://dx.doi.org/10.1093/rheumatology/kez390DOI Listing
May 2020

Health-related quality of life in systemic sclerosis before and after autologous haematopoietic stem cell transplant-a systematic review.

Rheumatology (Oxford) 2020 04;59(4):779-789

Unité de Médecine Interne: Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Centre de Référence des Maladies auto-immunes systémiques Rares d'Ile-de-France, AP-HP, Hôpital St-Louis, Saint Louis.

Objectives: In severe rapidly progressive SSc, autologous haematopoietic stem cell transplantation (AHSCT) allows significant improvements in overall and event-free survival. We undertook this study to identify, appraise and synthesize the evidence on health-related quality of life (HRQoL) before and after AHSCT for SSc.

Methods: We performed a systematic review of the literature, following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines, in PubMed and ScienceDirect from database inception to 1 February 2019. All articles with original HRQoL data were selected.

Results: The search identified 1080 articles, of which 8 were selected: 3 unblinded randomized controlled trials [American Scleroderma Stem Cell versus Immune Suppression Trial (ASSIST), Autologous Stem Cell Transplantation International Scleroderma, Scleroderma: Cyclophosphamide or Transplantation), 3 uncontrolled phase I or II trials and 2 cohort studies. HRQoL data from 289 SSc patients treated with AHSCT and 125 treated with intravenous CYC as a comparator with median 1.25-4.5 years follow-up were included. HRQoL was evaluated with the HAQ Disability Index (HAQ-DI; 275 patients), the 36-item Short Form Health Survey (SF-36; 249 patients) and the European Quality of Life 5-Dimensions questionnaire (EQ-5D; 138 patients). The quality of the studies was moderate to low. AHSCT was associated with significant improvement in the HAQ-DI (P = 0.02-<0.001), SF-36 Physical Component Summary score (P = 0.02-<0.0001) and EQ-5D index-based utility score (P < 0.001). The SF-36 Mental Component Summary score improved in the ASSIST (n = 19) and one small retrospective cohort (n = 30 patients, P = 0.005) but did not improve significantly in 2 randomized controlled trials (n = 200 patients, P = 0.1-0.91).

Conclusion: AHSCT in severe SSc patients is associated with significant and durable improvement in physical HRQoL.
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http://dx.doi.org/10.1093/rheumatology/kez300DOI Listing
April 2020

Clinical Characteristics and Outcomes of Ocular Cicatricial Pemphigoid: A Cohort Study and Literature Review.

Cornea 2019 Nov;38(11):1406-1411

Department of Internal Medicine, Centre Hospitalier Universitaire, Toulouse, France.

Purpose: Ocular cicatricial pemphigoid (OCP) is a rare systemic autoimmune disease and a potentially blinding subepithelial blistering disorder. The purpose of this study was to describe the clinical spectrum of the disease and to assess the efficacy and safety of immunosuppressive agents in a cohort of patients with OCP.

Methods: We conducted a monocentric retrospective cross-sectional cohort study of all unselected consecutive patients diagnosed with progressive OCP. Ocular and extra ophthalmological involvement as well as histological findings were gathered. Other outcomes were exposures to immunosuppressive agents defined by the use of a particular treatment. For each exposure, success in controlling ocular inflammation was graded as a complete response, response, or failure. Relapses and adverse events (AE) were also recorded.

Results: Seventeen patients (34 affected eyes), 35% of whom were women, were included, with an age at diagnosis of 75 ± 11 years. Corneal involvement was diagnosed in 30 of 34 eyes, and 22 of 34 eyes had progressive fibrosing conjunctival involvement. Sixty-two exposures to immunosuppressive agents or biologics were recorded: dapsone, n = 26; mycophenolate mofetil, n = 6; azathioprine, n = 4; cyclophosphamide, n = 10; rituximab, n = 14; and intravenous immunoglobulin, n = 2. Rates of response and of complete response achievement during the first 3 months were 84% and 45%, respectively. Response rates were 100%, 100%, 86%, 85%, and 80% for intravenous immunoglobulin, mycophenolate mofetil, rituximab, dapsone, and cyclophosphamide, respectively. Thirteen percent of those drugs were discontinued because of an adverse event in 4 patients.

Conclusions: This study describes the efficacy of immunosuppressants or biologics with an acceptable safety profile for OCP.
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http://dx.doi.org/10.1097/ICO.0000000000002080DOI Listing
November 2019

Large-vessel vasculitis diagnosed between 50 and 60 years: Case-control study based on 183 cases and 183 controls aged over 60 years.

Autoimmun Rev 2019 Jul 4;18(7):714-720. Epub 2019 May 4.

National Referral Center for Rare Autoimmune and Systemic Diseases, Hopital Cochin, AP-HP, Université Paris Descartes, Paris, France. Electronic address:

Background: Age at onset of large-vessel vasculitis (LVV) is commonly used to distinguish giant cell arteritis (GCA) and Takayasu arteritis (TA). However, LVV between age 50 and 60 years may be difficult to classify.

Methods: We conducted a retrospective study including LVV aged between 50 and 60 years at onset (LVV, cases) and compared them to LVV aged over 60 years (LVV, controls). LVV was defined histologically and/or morphologically. Controls fulfilled ACR 1990 criteria for GCA or presented isolated aortitis.

Results: We included 183 LVV and 183 gender-matched LVV. LVV had more frequent peripheral limb manifestations (23 vs. 5%), and less frequent cephalic (73 vs. 90%) and ocular signs (17 vs. 27%) than LVV. Compared to LVV, CT angiography and PET/CT scan were more frequently abnormal in LVV (74 vs. 38%, and 90 vs. 72%, respectively), with aorta being more frequently involved (78 vs. 47%). By multivariate analysis, absence of cephalic symptoms, presence of peripheral limb ischemia and aorta involvement, and increased CRP level were significantly associated with LVV presentation compared to LVV. At last follow-up, compared to LVV, LVV received significantly more lines of treatment (2 vs. 1), more frequent biologics (12 vs. 3%), had more surgery (10 vs. 0%), and had higher prednisone dose (8.8 vs. 6.5 mg/d) at last follow-up, CONCLUSION: LVV onset between 50 and 60 years identifies a subset of patients with more frequent aorta and peripheral vascular involvement and more refractory disease compared to patients with LVV onset after 60.
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http://dx.doi.org/10.1016/j.autrev.2019.05.008DOI Listing
July 2019

Orbital mass in ANCA-associated vasculitides: data on clinical, biological, radiological and histological presentation, therapeutic management, and outcome from 59 patients.

Rheumatology (Oxford) 2019 09;58(9):1565-1573

Department of Internal Medicine, National Referral Center for Systemic and Autoimmune Diseases, Hôpital de la Pitié-Salpêtrière, Paris.

Objective: Orbital mass is a rare and sight-threatening manifestation of ANCA-associated vasculitides, which remains a therapeutic challenge. We aimed to describe the presentation, therapeutic management and outcome of ANCA-associated vasculitides-related orbital mass.

Methods: We conducted a French nationwide retrospective study of patients with orbital mass in the setting of ANCA-associated vasculitides according to ACR criteria and/or Chapel Hill Consensus Conference definitions.

Results: Fifty-nine patients [33 women, median age 46 (range 7-90) years] were included. Fifty-six (95%) patients had granulomatosis with polyangiitis, two eosinophilic granulomatosis with polyangiitis and one microscopic polyangiitis. Orbital mass was unilateral in 47 (80%) cases, and seemed to develop from ENT involvement in most cases. Orbital mass biopsy was available in 32 (54%) patients, showing lymphoplasmacytic infiltration in 65%, fibrosis in 55%, granulomas in 48% and vasculitis in 36%. All patients but one received glucocorticoids as first-line therapy associated with immunosuppressive agents in 82%, mainly cyclophosphamide. Response to therapy was noted in 52% of patients treated with cyclophosphamide compared with 91% of those treated with rituximab. Twenty-seven (46%) patients required a second-line therapy because of relapse (59%) or refractory course (41%). Sequelae included visual impairment in 28%, with definitive blindness in 17%. Refractory course was associated with PR3-ANCA positivity, visual loss and contiguous pachymeningitis.

Conclusion: Orbital mass is associated with refractory course and high frequency of sequelae, especially blindness. Refractory course is associated with PR3-ANCA positivity, visual loss and contiguous pachymeningitis.
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http://dx.doi.org/10.1093/rheumatology/kez071DOI Listing
September 2019

The Use of Transthoracic Echocardiogram to Quantify Pulmonary Vascular Resistance in Patients with Systemic Sclerosis.

J Rheumatol 2019 11 1;46(11):1495-1501. Epub 2019 Mar 1.

From the Department of Cardiology, Rangueil University Hospital; Cardiac Imaging Centre, Toulouse University Hospital; UMR 1027, INSERM, Faculté de Médecine, Université de Toulouse III; Medical School of Rangueil, University Paul Sabatier; Department of Internal Medicine, University Hospital of Purpan; Department of Pneumology, Larrey University Hospital, Toulouse; Department of Cardiology, La Réunion University Hospital, Saint-Pierre; INSERM, UMR 1188, Sainte-Clothilde; Département d'Information Médicale, Centre Hospitalier Universitaire de Toulouse; Medical School of Purpan, University Paul Sabatier; Department of Nuclear Medicine, Toulouse University Hospital, Toulouse, France.

Objective: To explore the accuracy of tricuspid regurgitation velocity (TRV) to right ventricular outflow tract time-velocity integral (TVI) ratio by Doppler to determine pulmonary vascular resistance (PVR) in patients with systemic sclerosis (SSc).

Methods: Thirty-five consecutive adult patients with SSc, fulfilling the 2013 European League Against Rheumatism/American College of Rheumatology classification criteria, with sinus rhythm referred for right heart catheterization (RHC), were retrospectively included. All patients underwent a transthoracic echocardiogram (TTE) performed within 24 h of RHC. Patients with SSc were recruited regardless of disease activity, cardiac symptoms, and treatment regimen. Doppler measurements were compared to RHC measurements. A linear regression equation was generated to predict PVR by echocardiogram based on the TRV/TVI ratio. The accuracy of Doppler measurements for predicting PVR > 3 Wood units was assessed by computing the areas under the receiver-operating characteristic curves.

Results: There were 20 (57%) females in the study. The mean age was 65 ± 12 years. Mean and systolic pulmonary arterial pressures were 31 ± 8 and 53 ± 15 mmHg, respectively. There was a good correlation between TRV/TVI ratio assessed by Doppler and PVR measured by RHC (R = 0.743, p < 0.001). The equation generated by this analysis was the following: PVR by Doppler = 11.3 × (TRV/TVI) + 1.7. A cutoff value of 0.21 for TRV/TVI ratio provided the best sensitivity (86%) and specificity (86%) to determine PVR > 3 Wood units.

Conclusion: Our study suggests that TTE using Doppler could be a useful and noninvasive tool for estimating PVR in patients with SSc.
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http://dx.doi.org/10.3899/jrheum.180571DOI Listing
November 2019

Autologous haematopoietic stem cell transplantation (AHSCT) in autoimmune disease adult patients in France: analysis of the long-term outcome from the French Society for Bone Marrow Transplantation and Cellular Therapy (SFGM-TC).

Clin Rheumatol 2019 May 21;38(5):1501-1511. Epub 2019 Jan 21.

Université Denis Diderot, Paris, France.

Introduction: The use of autologous haematopoietic stem cell transplantation (AHSCT) in autoimmune disease (AD) patients has increased progressively worldwide. We retrospectively analysed the long-term outcome of AHSCT for AD reported to the French Society for Bone Marrow Transplantation and Cellular Therapy (SFGM-TC).

Method: All French AD patients (≥ 18 years at transplant) with a first AHSCT between 1997 and 2013 were included. Primary data were derived from the European Society for Blood and Marrow Transplantation (EBMT) registry, and additional data were obtained through a specific questionnaire designed for the study. Primary end-point was overall survival (OS). Secondary end points were progression-free survival (PFS) and non-relapse mortality (NRM).

Results: Ninety-four AD patients were included, of whom 71% suffered from rheumatologic diseases (n = 67, including 56 systemic sclerosis (SSc)), 16% from neurological disease (n = 15, including 14 multiple sclerosis (MS)) and 13% from various other AD (n = 12). After a median (interquartile range, IQR) follow-up of 83 months (38-130), OS at 5 and 10 years were 77% (95% CI 68.5-86.2) and 64% (95% CI 51.7-76.3), and for PFS 51% (95% CI 40.4-61.6) and 44% (95% CI 32.8-55.3), respectively. Overall, NRM was 8.7% (95% CI 4.0-15.5) at day 100, 9.8% (95% CI 4.8-16.9) at 5 years and 13.6% (95% CI 6.9-22.5) at 10 years.

Conclusions: This first SFGM-TC retrospective report shows long-term benefit of AHSCT in AD patients with acceptable toxicity.
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http://dx.doi.org/10.1007/s10067-019-04435-2DOI Listing
May 2019