Publications by authors named "Pascal Cathebras"

60 Publications

JAK1/2 Inhibition in Severe TAFRO Syndrome: A Case Report.

Ann Intern Med 2021 Jan 12. Epub 2021 Jan 12.

Centre Hospitalier Universitaire de Saint-Etienne and Université Jean Monnet, Université de Lyon, Saint-Etienne, France (M.K., F.F., S.G., P.B., P.C., S.P.).

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http://dx.doi.org/10.7326/L20-1051DOI Listing
January 2021

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.

Centre de Référence des Maladies Pulmonaires Rares, CHU de Dijon Bourgogne, Université de Bourgogne, INSERM 1231, Dijon, 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

Paraneoplastic subacute sensory neuropathy with triple positive antineuronal antibodies associated with small-cell lung cancer.

BMJ Case Rep 2020 Aug 24;13(8). Epub 2020 Aug 24.

Department of Internal Medicine, Centre Hospitalier Universitaire de Saint-Etienne, Saint-Etienne, Loire, France

A 67-year-old woman with a history of smoking and cardiovascular risk factors was admitted to the emergency room for uncontrolled diabetes, loss of appetite, nausea, significant weight loss and asthenia. The initial investigation, including cerebral and gastrointestinal explorations, were normal. One month later, she started presenting severe asymmetric proprioceptive ataxia of the lower extremities. She also reported paresthesia and neuropathic pain in both feet and ankles. A positron emission tomography (PET)-scanner showed a hypermetabolic nodule in the right lung. The neurological symptoms were attributed to paraneoplastic sensory and dysautonomic neuropathy, even though the bronchoscopic biopsies came back negative at first. Anti-Hu, anti-CV2/CRMP5 and anti-SOX1 antibodies were documented. Due to the severity and rapid progression of symptoms (from the lower to the upper limbs), corticosteroids, intravenous immunoglobulins and immunosuppressants were introduced prior to biopsies revealing a small-cell lung cancer. Despite these treatments and antineoplastic chemotherapy, her status deteriorated rapidly.
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http://dx.doi.org/10.1136/bcr-2020-235668DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7449276PMC
August 2020

[Use and management of proton pump inhibitors: An observational study].

Therapie 2020 Nov - Dec;75(6):649-662. Epub 2020 Jun 4.

Service de médecine interne, CHU de Saint-Étienne, 42055 Saint-Étienne cedex 2, France.

Introduction: Proton pump inhibitors (PPIs) have improved the management and prevention of digestive diseases, leading to a heavy prescription of this therapy. In 2015, nearly one quarter of the French population had consumed a PPI and half of them were long-term users. The main objective of this study was to analyze, in patients hospitalized in several medical departments, the adequacy of long-term PPI prescriptions to recommendations.

Method: The Use and management of proton pump inhibitors: an observational study project (UTOPPIA) is a longitudinal observational study conducted at the University Hospital of Saint-Étienne in the departments of hepato-gastroenterology, infectious and tropical diseases, internal medicine, vascular medicine and nephrology. All patients with PPI treatment on their usual outpatient prescription were interviewed.

Results: Over a 3-month period, 334 of hospitalized patients (30.7%) had received a long-term PPI prescription and 181 patients (54.2%) could be included in the study for a total of 274 indications. Ninety-nine patients (54.7%) had a long-term PPI prescription in accordance with the recommendations. The most frequent indication (70 prescriptions) was the prescription of an antiplatelet drug or anticoagulant for subjects at high risk of bleeding in 70 prescriptions. Fifty-three PPI treatments were amended during the hospital stay, including 9 discontinuations. The justification for the change was documented in the patients' chart in only 17% of cases. Individual interviews of patients revealed that 75.1% of them were in favour of discontinuing their PPI treatment.

Conclusions: About one-third of hospitalized patients in medical wards in France have long-term PPI treatment and half of these prescriptions do not comply with good practice recommendations. A majority of patients report being willing to try to stop PPI therapy.
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http://dx.doi.org/10.1016/j.therap.2020.03.002DOI Listing
June 2020

Analysis of risk factors for complications and adverse obstetrical outcomes in women with Takayasu arteritis: a French retrospective study and literature review.

Clin Rheumatol 2020 Sep 23;39(9):2707-2713. Epub 2020 Mar 23.

Sorbonne Université, Département Hospitalo-Universitaire-Inflammation-Immunopathologie-Biothérapie (DMU i3), Service de Médecine Interne, F-75005, Paris, France.

Objective: Takayasu arteritis (TAK) is a large vessel vasculitis affecting young women of childbearing age. The outcome of pregnancies in TAK patients, factors associated with maternal and foetal complications and adverse outcomes were analysed.

Methods: All pregnancies in women with a TAK diagnosis were retrospectively included from 20 French hospitals providing care for TAK, until August 2015.

Results: The study consisted of 43 pregnancies in 33 women, including 29 with a pre-existing TAK diagnosis and 4 diagnosed during pregnancy. Complications were observed in 20 pregnancies (47%), including 35% with arterial hypertension (n = 15), 9% with pre-eclampsia (n = 4), 2% with HELLP syndrome (n = 1) and 14% with intrauterine growth restriction (IUGR, n = 6, leading in one case to a medically indicated termination of pregnancy). There were 42 live births (98%) at a median term of 38 [27-42] weeks gestation including 9 before 37 weeks (21%). The median birth weight was 2940 [610-4310] grams. Five children (12%) required transfer to a neonatal intensive care unit. One premature boy (27 weeks gestation) died after 2 days. Treatment during pregnancy included steroids (n = 25/43; 58%), azathioprine (n = 9/43; 21%) and infliximab (n = 1/43; 2%). The risk of developing arterial hypertension during pregnancy was associated with previous chronic arterial hypertension and with an infra-diaphragmatic vasculitis injury (P = 0.01 and P = 0.04, respectively). No correlation was reported between TAK activity and any of the obstetrical complications described in the study.

Conclusion: This study showed a high rate of adverse obstetrical complications without significant impact on live birth rates. Pregnancy did not appear to influence TAK disease activity. Key Points • We observed a high rate of adverse obstetrical complications in women with Takayasu arteritis; however, the rate of live births was high. Pregnancy did not appear to influence TA disease activity.
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http://dx.doi.org/10.1007/s10067-020-05024-4DOI Listing
September 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

What attitude towards a patient with somatoform disorder?

Rev Prat 2019 02;69(2):209-213

CHU de Saint-Étienne, hôpital Nord, service de médecine interne, Saint-Étienne, France.

The diagnosis of a somatic symptom disorder must be based on both a negative approach - eliminating another psychiatric or non-psychiatric disorder that would better explain the symptoms - and a positive one, which is, based on the search for characteristic thoughts, emotions or behaviours as well as biological or psychological factors that may promote, trigger or sustain the disorder. Additional tests and specialized medical consultations should not be prescribed solely to reassure the patient; they may actually worsen the condition. The management will move away from the outdated notion of "medically unexplained symptoms" to rely on: the acknowledgment of the painful, debilitating and involuntary nature of the symptoms; the proposal of a positive diagnosis acceptable by the patient and an explanatory model compatible with his or her representations, aimed at putting an end to dysfunctional health care utilization; the proposal of therapeutic objectives aimed at functional rather than symptomatic recovery; the negotiation of pharmacological (selective or mixed serotonin reuptake inhibitor if necessary) and non-pharmacological interventions, especially when it comes to limiting the factors that sustain the disorder; the coordination of the various healthcare professionals.
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February 2019

Betaine anhydrous in homocystinuria: results from the RoCH registry.

Orphanet J Rare Dis 2019 03 14;14(1):66. Epub 2019 Mar 14.

CHRU de Tours, Service de Médecine Interne, Université François Rabelais, Tours, France.

Background: The Registry of Adult and Paediatric Patients Treated with Cystadane® - Homocystinuria (RoCH) is a non-interventional, observational, multi-centre, post-authorization safety study that aimed to identify safety of betaine anhydrous (Cystadane®) in the treatment of patients with inborn errors of homocysteine metabolism (homocystinuria) in order to minimise the treatment associated risks and establish better knowledge on its clinical use. The registry included patients of all ages with homocystinuria who were treated with betaine anhydrous in conjunction with other therapies. Clinical data were collected retrospectively from 2007 to 2013, then prospectively up to February 2014. All adverse events (AEs) reported during the study were recorded. The clinical and biological status of patients was monitored at least once a year.

Results: A total of 125 patients with homocystinuria (adults [> 18 years]: 50; paediatric [≤18 years]: 75) were enrolled at 29 centres in France and Spain. Patients were treated with betaine anhydrous for a mean duration of 7.4 ± 4.3 years. The median total daily dose of betaine anhydrous at the first and last study visits was 6 g/day for cystathionine β-synthase (CBS)-deficient vitamin B6 responders and 9 g/day for methylenetetrahydrofolate reductase-deficient patients, while the median daily dose increased in CBS-deficient B6 non-responders (from 6 to 9 g/day) and cobalamin metabolism-defective patients (from 3 to 6 g/day) between the first and last visits. Treatment caused a mean overall reduction of 29% in plasma homocysteine levels in the study population. A total of 277 AEs were reported during the study, of which two non-serious AEs (bad taste and headache) and one serious AE (interstitial lung disease) were considered to be drug related. Overall, betaine anhydrous was well tolerated with no major safety concerns.

Conclusions: Data from the RoCH registry provided real-world evidence on the clinical safety and efficacy of betaine anhydrous in the management of homocystinuria in paediatric and adult patients.
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http://dx.doi.org/10.1186/s13023-019-1036-2DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6419445PMC
March 2019

Clinical and biological features in -hereditary xerocytosis and Gardos channelopathy: a retrospective series of 126 patients.

Haematologica 2019 08 17;104(8):1554-1564. Epub 2019 Jan 17.

Laboratoire d'Hématologie, Center Hospitalier Universitaire (CHU) Bicêtre, Assistance publique - Hôpitaux de Paris (AP-HP), Le Kremlin-Bicêtre

We describe the clinical, hematologic and genetic characteristics of a retrospective series of 126 subjects from 64 families with hereditary xerocytosis. Twelve patients from six families carried a mutation, five had the recurrent p.Arg352His mutation and one had a new deletion at the exon 7-intron 7 junction. Forty-nine families carried a mutation, which was a known recurrent mutation in only one-third of the cases and private sequence variation in others; 12 new probably pathogenic missense mutations were identified. The two dominant features leading to diagnosis were hemolysis that persisted after splenectomy and hyperferritinemia, with an inconstant correlation with liver iron content assessed by magnetic resonance imaging. -hereditary xerocytosis was characterized by compensated hemolysis in most cases, perinatal edema of heterogeneous severity in more than 20% of families and a major risk of post-splenectomy thrombotic events, including a high frequency of portal thrombosis. In -related disease, the main symptoms were more severe anemia, hemolysis and iron overload, with no clear sign of red cell dehydration; therefore, this disorder would be better described as a 'Gardos channelopathy'. These data on the largest series to date indicate that -hereditary xerocytosis and Gardos channelopathy are not the same disease although they share hemolysis, a high rate of iron overload and inefficient splenectomy. They demonstrate the high variability in clinical expression as well as genetic bases of -hereditary xerocytosis. These results will help to improve the diagnosis of hereditary xerocytosis and to provide recommendations on the clinical management in terms of splenectomy, iron overload and pregnancy follow-up.
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http://dx.doi.org/10.3324/haematol.2018.205328DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6669138PMC
August 2019

Preemptive rituximab prevents long-term relapses in immune-mediated thrombotic thrombocytopenic purpura.

Blood 2018 11 10;132(20):2143-2153. Epub 2018 Sep 10.

Centre de Référence des Microangiopathies Thrombotiques, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris, Paris, France.

Preemptive rituximab infusions prevent relapses in immune thrombotic thrombocytopenic purpura (iTTP) by maintaining normal ADAMTS13 activity. However, the long-term outcome of these patients and the potential adverse events of this strategy need to be determined. We report the long-term outcome of 92 patients with iTTP in clinical remission who received preemptive rituximab after identification of severe ADAMTS13 deficiency (activity <10%) during the follow-up. Thirty-seven patients had >1 iTTP episode, and the median cumulative relapse incidence before preemptive rituximab was 0.33 episode per year (interquartile range [IQR], 0.23-0.66). After preemptive rituximab, the median cumulative relapse incidence in the whole population decreased to 0 episodes per year (IQR, 0-1.32; < .001). After preemptive rituximab, ADAMTS13 activity recovery was sustained in 34 patients (37%) during a follow-up of 31.5 months (IQR, 18-65), and severe ADAMTS13 deficiency recurred in 45 patients (49%) after the initial improvement. ADAMTS13 activity usually improved with additional courses of preemptive rituximab. In 13 patients (14%), ADAMTS13 activity remained undetectable after the first rituximab course, but retreatment was efficient in 6 of 10 cases. In total, 14 patients (15%) clinically relapsed, and 19 patients (20.7%) experienced benign adverse effects. Preemptive rituximab treatment was associated with a change in ADAMTS13 conformation in respondent patients. Finally, in the group of 23 historical patients with iTTP and persistently undetectable ADAMTS13 activity, 74% clinically relapsed after a 7-year follow-up (IQR, 5-11). In conclusion, persistently undetectable ADAMTS13 activity in iTTP during remission is associated with a higher relapse rate. Preemptive rituximab reduces clinical relapses by maintaining a detectable ADAMTS13 activity with an advantageous risk-benefit balance.
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http://dx.doi.org/10.1182/blood-2018-04-840090DOI Listing
November 2018

[A pernicious cause of infant hypotonia].

Presse Med 2018 Jul - Aug;47(7-8 Pt 1):697-700. Epub 2018 Jun 11.

CHU de Saint-Étienne, hôpital Nord, service de médecine interne, 40255 Saint-Étienne cedex 2, France.

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http://dx.doi.org/10.1016/j.lpm.2018.05.003DOI Listing
November 2018

Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-Kinase δ Syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syndrome Registry.

Front Immunol 2018 16;9:543. Epub 2018 Mar 16.

Paris Descartes-Sorbonne Paris Cité University, Paris, France.

Activated phosphoinositide 3-kinase (PI3K) δ Syndrome (APDS), caused by autosomal dominant mutations in (APDS1) or (APDS2), is a heterogeneous primary immunodeficiency. While initial cohort-descriptions summarized the spectrum of clinical and immunological manifestations, questions about long-term disease evolution and response to therapy remain. The prospective European Society for Immunodeficiencies (ESID)-APDS registry aims to characterize the disease course, identify outcome predictors, and evaluate treatment responses. So far, 77 patients have been recruited (51 APDS1, 26 APDS2). Analysis of disease evolution in the first 68 patients pinpoints the early occurrence of recurrent respiratory infections followed by chronic lymphoproliferation, gastrointestinal manifestations, and cytopenias. Although most manifestations occur by age 15, adult-onset and asymptomatic courses were documented. Bronchiectasis was observed in 24/40 APDS1 patients who received a CT-scan compared with 4/15 APDS2 patients. By age 20, half of the patients had received at least one immunosuppressant, but 2-3 lines of immunosuppressive therapy were not unusual before age 10. Response to rapamycin was rated by physician visual analog scale as good in 10, moderate in 9, and poor in 7. Lymphoproliferation showed the best response (8 complete, 11 partial, 6 no remission), while bowel inflammation (3 complete, 3 partial, 9 no remission) and cytopenia (3 complete, 2 partial, 9 no remission) responded less well. Hence, non-lymphoproliferative manifestations should be a key target for novel therapies. This report from the ESID-APDS registry provides comprehensive baseline documentation for a growing cohort that will be followed prospectively to establish prognostic factors and identify patients for treatment studies.
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http://dx.doi.org/10.3389/fimmu.2018.00543DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5863269PMC
May 2019

Efficacy and safety of biologics in relapsing polychondritis: a French national multicentre study.

Ann Rheum Dis 2018 08 13;77(8):1172-1178. Epub 2018 Mar 13.

UMR 1027, INSERM, University of Toulouse, Toulouse, France.

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.
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http://dx.doi.org/10.1136/annrheumdis-2017-212705DOI Listing
August 2018

Contribution of diagnostic tests for the etiological assessment of uveitis, data from the ULISSE study (Uveitis: Clinical and medicoeconomic evaluation of a standardized strategy of the etiological diagnosis).

Autoimmun Rev 2018 Apr 7;17(4):331-343. Epub 2018 Feb 7.

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

Purpose: ULISSE is the only study that prospectively assessed the efficiency of a standardized strategy, compared to an open strategy for the etiologic diagnosis of uveitis. Our aim was to evaluate the diagnostic yield of the tests prescribed in the ULISSE study to clarify their relevance.

Methods: ULISSE is a non-inferiority, prospective, multicenter and cluster randomized study. The standardized strategy is a two-steps strategy: in the first step, common standard tests were performed, and in the second step, tests were guided by the clinical and anatomic type of uveitis. We reported the relevance of the diagnostic tests used in the standardized strategy, as well as the profitability of the tests that were prescribed to more than twenty patients in each group. Based on diagnostic criteria, either an ophthalmologist, or an internist, established the profitability of a test by considering whether the test lead to a diagnosis or not.

Results: Among the 676 patients included (standardized 303; open 373), a diagnosis was made for 152 (50.4%) in the standardized group and 203 (54.4%) in the open group. The most common entities were HLA-B27 associated uveitis (22%), spondyloarthritis (11%), sarcoidosis (18%), tuberculosis (10.7%) and herpes virus infections (8.5%). Among the first step's systematic tests, tuberculin skin test was the most contributive investigation (17.1%), followed by chest X-ray (8.4%), C reactive protein and ESR (6.6% and 5.1%), complete blood count (2.2%) and VDRL (2.0%). The second step's most often contributive tests were: HLA B27 (56.3%), chest-CT (30.3%) and angiotensin converting enzyme (ACE) (16.5%). HLA B27 and ACE were significantly more contributive in the standardized group than in the open group. Immunological tests were never contributive. Among the free investigations, or among the investigations guided by clinical or paraclinical findings, the most often contributive tests were: Quantiferon® (24%), electrophoresis of serum protein (7.8%) and sacroiliac imagery (46.4%). Intracellular serologies (1.7%), serum calcium (2.1%) and hepatic tests (3.3%) were exceptionally contributive. Among the third intention tests, labial salivary gland biopsies were contributive in 17.9% of cases, but the profitability of other invasive investigations (anterior chamber tap, vitrectomy, bronchoscopy and lumbar puncture) or specialized imagery (18F-FDG PET, Brain MRI) could not be determined since these test were rarely performed.

Conclusion: Only a few diagnostic tests are useful for the etiological assessment of uveitis. They are often cheap, simple, more often guided by the clinical findings, and lead to an etiological diagnosis in most patients. On the other hand, some tests are never or exceptionally contributive, such as immunological tests or intracellular serologies. Further studies are required to evaluate the profitability of third intention imagery and invasive investigations.
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http://dx.doi.org/10.1016/j.autrev.2017.10.018DOI Listing
April 2018

Lithium-induced downbeat nystagmus.

Am J Ophthalmol Case Rep 2017 Sep 21;7:74-75. Epub 2017 Jun 21.

Internal Medicine, University Hospital of Saint-Etienne, France.

We report the case of a 76-year old lady under lithium carbonate for a bipolar disorder who presented with a suspected optic neuritis. A typical lithium-induced downbeat nystagmus was observed. Discontinuation of lithium therapy resulted in frank improvement in visual acuity and disappearance of the nystagmus.
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http://dx.doi.org/10.1016/j.ajoc.2017.06.012DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5722167PMC
September 2017

Muscle Damage Due to Fusidic Acid-Statin Interaction: Review of 75 Cases From the French Pharmacovigilance Database and Literature Reports.

Am J Ther 2019 May/Jun;26(3):e375-e379

Internal Medicine Department, University Hospital of Saint-Etienne, Saint-Etienne, France.

Background/area Of Uncertainty: Statins, which reduce cardiovascular risk in both primary and secondary prevention, are one of the most widely prescribed therapeutic classes in the world. Usually well-tolerated, statin-associated muscle symptoms are a well-known adverse effect. Fusidic acid (FA) is a bacteriostatic antibiotic of interest in the treatment of methicillin-resistant Staphylococcus aureus infections. Cases of rhabdomyolysis, sometimes fatal, have been reported after coprescription of FA and a statin.

Data Sources/area Of Uncertainty: We studied 75 cases of muscle damage related to interaction between FA and a statin reported in the French national pharmacovigilance database (43 cases) and from a literature review (32 cases).

Results: Cases were mostly men (72.5%), often overweight (mean body mass index: 29.4). The most commonly reported statins were atorvastatin (60%), simvastatin (22.7%), and rosuvastatin (8.0%). Muscle disorders appeared on average 30 days after initiation of FA. Symptoms were muscle weakness (82%), dark urine (71%), and myalgia (61%). Mean creatine kinase level at diagnosis was 43,890 UI/mL, and acute renal injury occurred more than half of the cases. Outcome was fatal in 22% of cases and 28% kept sequelae at the end of the follow-up (54 days).

Conclusions: Muscle damage induced by interaction between FA and statin is a potentially life-threatening complication, leading to contraindication of this association in France. This is to be reminded especially because FA is about to get FDA approval and should soon be available in the United States.
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http://dx.doi.org/10.1097/MJT.0000000000000679DOI Listing
November 2019

Immunomodulatory treatments for persistent and chronic immune thrombocytopenic purpura: A PRISMA-compliant systematic review and meta-analysis of 28 studies.

Medicine (Baltimore) 2017 Sep;96(37):e7534

Department of Internal and Vascular Medicine, Centre Hospitalier Lyon-Sud, Pierre-Bénite Equipe d'Accueil HESPER 7425, Claude Bernard University Lyon 1, Villeurbanne Department of Internal Medicine, Hôpital Nord, Centre Hospitalier Universitaire de Saint-Étienne, Saint-Priest-en-Jarez UMR 5558, Laboratoire de Biométrie et Biologie Evolutive, CNRS, Claude Bernard University Lyon 1, Villeurbanne, France.

Background: Corticosteroid sparing is required in 15% to 40% of adults with persistent or chronic primary immune thrombocytopenic purpura (ITP). Herein, the efficacy of immunomodulatory drugs (dapsone, interferon alpha, danazol, and hydroxychloroquine as second-third-line therapies in ITP is investigated.

Methods: MEDLINE was searched for studies that included patients with persistent or chronic primary ITP and published before the end of December 2014. Two investigators independently extracted data regarding study design, patient characteristics, dosage schedule, time to response, and occurrence of adverse events. The pooled overall response rate (ORR; platelet count >30 × 10 L) and the complete response rate (CRR; platelet count >100 × 10 L) were evaluated to determine drug efficacy by calculating weighted mean proportion using a fixed or random-effects model according to heterogeneity (I > 50%). The study was performed following the MOOSE and PRISMA guidelines.

Results: A total of 28 studies (415 patients) were included (dapsone: k = 7 studies, n = 80; danazol: k = 12, n = 224; interferon alpha: k = 8, n = 83; hydroxychloroquine: k = 1, n = 28). The mean patient age was 50 years (female sex 70%, splenectomy 47%). The ORR and CRR were 55% (95% CI: 44%-66%, I = 0%) and 21% (95% CI: 13%-31%, I = 0%), respectively, for dapsone; 42% (95% CI: 22%-65%, I = 63%) and 18% (95% CI: 10%-29%, I = 9%), respectively, for interferon alpha; and 58% (95% CI: 42%-72%, I = 67%) and 29% (95% CI: 19%-42%, I = 63%), respectively, for danazol. The ORR was 50% (95% CI: 32%-67%) for hydroxychloroquine (data not available for CRR). Meta-regression analysis found a correlation between the ORR for interferon alpha and the splenectomized status of the patient (P = .02) and between the CRR for danazol and disease duration (P < .001). In total, 73%, 51%, 30%, and 0% of patients who received danazol, dapsone, interferon alpha, and hydroxychloroquine experienced side effects, respectively.

Conclusion: The ORR was equivalent for hydroxychloroquine, danazol, and dapsone in ITP. Regarding their low CRR, patients at high risk of infection or at low risk of bleeding should benefit from these treatments. Thanks to their best efficacy and safety profiles, dapsone and hydroxychloroquine in patients with antinuclear antibodies should be preferred over danazol and interferon alpha.
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http://dx.doi.org/10.1097/MD.0000000000007534DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5604622PMC
September 2017

Central nervous system involvement in eosinophilic granulomatosis with polyangiitis (Churg-Strauss): Report of 26 patients and review of the literature.

Autoimmun Rev 2017 Sep 12;16(9):963-969. Epub 2017 Jul 12.

Department of Internal Medicine, Hôpital Cochin, F-75014 Paris, France; National Referral Center for Systemic and Autoimmune Diseases, Hôpital Cochin, F-75014 Paris, France; Université Paris Descartes, Faculté de Médecine Paris Descartes, F-75014 Paris, France; Université Paris Descartes, Paris 5, Paris, France; Inserm, U1016, Institut Cochin, F-75014 Paris, France. Electronic address:

Background: Although peripheral nervous system involvement is common in eosinophilic granulomatosis with polyangiitis (EGPA), central nervous system (CNS) manifestations are poorly described. This study aimed to describe CNS involvement in EGPA.

Patients And Methods: This retrospective, observational, multicenter study included patients with EGPA and CNS involvement affecting cranial nerves, brain and/or spinal cord. We also undertook a systematic literature review.

Results: We analyzed 26 personal cases and 62 previously reported cases. At EGPA diagnosis, asthma was noted in 97%, eosinophilia in 98%, peripheral neuropathy in 55% and cardiac involvement in 41%. 38/71 (54%) were ANCA-positive, with a perinuclear-labeling pattern and/or anti-MPO specificity. CNS was involved in 86% at EGPA diagnosis, preceded EGPA in 2%, and occurred during follow-up in 12% after a median of 24months. Main neurological manifestations were ischemic cerebrovascular lesions in 46 (52%), intracerebral hemorrhage and/or subarachnoid hemorrhage in 21 (24%), loss of visual acuity in 28 (33%) (15 with optic neuritis, 9 with central retinal artery occlusion, 4 with cortical blindness), and cranial nerves palsies in 18 (21%), with 25 patients having ≥1 of these clinical CNS manifestations. Among the 81 patients with assessable neurological responses, 43% had complete responses without sequelae, 43% had partial responses with long-term sequelae and 14% refractory disease. After a mean follow-up of 36months, 11 patients died including 5 from intracerebral hemorrhages.

Conclusion: EGPA-related CNS manifestations form 4 distinct neurological pictures: ischemic lesions, intracerebral hemorrhages, cranial nerve palsies and loss of visual acuity. Such manifestation should prompt practitioners to consider EGPA in such conditions. Long-term neurological sequelae were common, and intracerebral hemorrhages had the worst prognostic impact.
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http://dx.doi.org/10.1016/j.autrev.2017.07.007DOI Listing
September 2017

Long-Term Follow-up Consultation After Childhood Cancer in the Rhône-Alpes Region of France: Feedback From Adult Survivors and Their General Practitioners.

J Adolesc Young Adult Oncol 2017 Dec 25;6(4):524-534. Epub 2017 May 25.

9 Department of Internal Medicine Unit, University Hospital of Saint-Etienne , Saint-Etienne Cedex 02, France .

Purpose: We evaluated the satisfaction of adult survivors of childhood cancers and their general practitioners (GP) after a long-term consultation.

Methods: The first Long-term Follow-up Study in Oncology (SALTO1) is a prospective cohort study of survivors of childhood cancers (except leukemia) diagnosed between 1987 and 1992 in the Rhône-Alpes and Auvergne regions of France. Of the 481 patients eligible for the study, 150 participated in a long-term consultation with a pediatric oncologist and an internist, after which survivors and their GPs received long-term plans and recommendations based on consultation findings. A year after the consultation, survivors and their GPs assessed their satisfaction with the process.

Results: Of the 150 survivor participants in the long-term follow-up, 120 (80%) completed the satisfaction form, with 107 (89%) reporting satisfaction. Forty-eight (32%) expressed strengthening their follow-up as a consequence of the consultation. Of the 79 survivors sent recommendations, 76 (96%) reported reading them, most (n = 68; 86%) found them useful, and 56 (71%) followed recommendations. Of the 107 GPs of the survivors, 82 (77%) conceded having been poorly informed about long-term complications for their patients after chemotherapy, and 93 (88%) appreciated having a hospital contact available for these patients.

Conclusion: The long-term consultations ultimately enhanced medical follow-up of survivor participants, improving knowledge of both patients and family physicians regarding the patients' early disease, its treatments, and possible concerns, and offering consultative resources of medical specialists. The levels of participation of survivors and their physicians and reported satisfaction encourage the adoption of such consultations throughout France.
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http://dx.doi.org/10.1089/jayao.2017.0019DOI Listing
December 2017

Randomized Controlled Trial Evaluating a Standardized Strategy for Uveitis Etiologic Diagnosis (ULISSE).

Am J Ophthalmol 2017 Jun 31;178:176-185. Epub 2017 Mar 31.

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

Purpose: To prospectively assess the efficiency of a standardized diagnostic approach, compared to an open strategy, for the etiologic diagnosis of uveitis.

Design: Noninferiority, prospective, multicenter, clustered randomized controlled trial.

Methods: Consecutive patients with uveitis, who visited 1 of the participating departments of ophthalmology, were included. In the standardized group, all patients had a minimal evaluation regardless of the type of uveitis (complete blood count, erythrocyte sedimentation rate, C-reactive protein, tuberculin skin test, syphilis serology, and chest radiograph) followed by more complex investigations according to ophthalmologic findings. In the open group, the ophthalmologist could order any type of investigation. Main outcome was the percentage of etiologic diagnoses at 6 months.

Results: Nine hundred and three patients with uveitis were included from January 2010 to May 2013 and the per-protocol population comprised 676 patients (open 373; standardized 303). Mean age at diagnosis was 46 years. Anatomic distribution of uveitis was as follows: anterior (60.8% and 72.3%, P = .0017), intermediate (11.7% and 12.3%, P = .8028), posterior (17.8% and 8.2%, P = .0004), and panuveitis (15.3% and 15.2%, P = .9596). An etiologic diagnosis was established in 54.4% of cases in the open group and 49.5% in the standardized group (P = .2029). The difference between both strategies (standardized minus open) was -4.9% (95% CI [-12.5%; 2.6%]). There were more investigations in the open group than in the standardized group (5371 vs 3759, P < .0001).

Conclusion: The standardized strategy appears to be an efficient diagnostic approach for the etiologic diagnosis of uveitis, although its noninferiority cannot be proved.
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http://dx.doi.org/10.1016/j.ajo.2017.03.029DOI Listing
June 2017

Kawasaki disease in adults: Observations in France and literature review.

Autoimmun Rev 2016 Mar 26;15(3):242-9. Epub 2015 Nov 26.

Service de Pneumologie, Centre Hospitalier Universitaire de Rennes, France.

Objective: Kawasaki disease (KD) is a vasculitis that mostly occurs in young children and rarely in adults. We analyzed the characteristics of adult-onset KD (AKD) in France.

Methods: We collected retrospective and prospective data for patients with a diagnosis of KD occurring after the age of 18 years. Cases were obtained via various French medical networks and identified from the international literature.

Results: We included 43 patients of AKD at 26 institution from 1992 to 2015, with mean (SD) age 30 (11) years (range 18-68) and sex ratio (M/F) 1.2; 34 patients met the American Heart Association criteria and 9 were incomplete AKD. The median time to diagnosis was 13 days (interquartile range 8-21). The main symptoms were fever (100%), exanthema (98%), changes in the extremities (91%), conjunctivitis (77%), oral cavity changes (89%), cervical adenitis (55%) and cardiac abnormalities (45%). Overall, 35% of patients showed large-vessel vasculitis: coronary vasculitis (26%) and coronary aneurysm (19%). Treatment was mostly intravenous immunoglobulins (79%) and aspirin (81%). Four patients showed myocardial infarction due to coronary vasculitis, but none were treated with IVIg because of late diagnosis. After a median follow-up of 5 months (range 1-117), persistent aneurysm was noted in 9% of cases. Damage was significantly lower with early treatment than late or no treatment (p=0.01).

Conclusion: Given the high frequency of cardiac involvement and complications in this series of AKD, diagnosis and treatment should not be delayed, and early IVIg treatment seems to improve the outcome.
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http://dx.doi.org/10.1016/j.autrev.2015.11.010DOI Listing
March 2016

A French National Survey on Clotting Disorders in Mastocytosis.

Medicine (Baltimore) 2015 Oct;94(40):e1414

From the French Reference Centre for Mastocytosis (CEREMAST), Necker Children's Hospital, Assistance Publique - Hôpitaux de Paris (APHP), France (ABC, GD, DC, CB, SB, LF, OL, OH, M-OC); Haemostasis and Thrombosis Unit, Haemophilia Clinic, Division of Haematology, St-Luc University Hospital, Brussels, Belgium (ABC, CH); Department of Internal Medicine, Caen University Hospital, France (AA); Department of Haematology, Caen University Hospital, France (GD); Department of Pathology, Necker Children's Hospital, APHP, France (DC); Department of Biological Haematology, Necker Children's Hospital, APHP, France (CB); Department of Haematology and Cell Therapy, Tours University Hospital, France (EG); Department of Internal and Vascular Medicine, Lyon Sud University Hospital, France (SD, ID); Department of Internal Medicine, Saint-Etienne University Hospital, France (PC); Department of Internal Medicine, Cochin Hospital, APHP, France (NC-C); Department of Internal Medicine, Lille University Hospital, France (DL); Infectious Diseases Department, Necker Children's Hospital, APHP, France (BP, OL); Department of Dermatology, Tenon University Hospital, APHP, France (SB); Department of Haematology, Necker Children's Hospital, APHP, France (LF, OH, M-OC); and Sorbonne Paris Cité, Paris Descartes University, Imagine Institute, Paris, France (LF, OL, OH, M-OC).

Mastocytosis is characterized by a clonal mast cell proliferation with organ infiltration and uncontrolled degranulation. Although not characteristic and poorly explained, some patients develop clotting abnormalities. We retrospectively identified patients with established diagnosis of mastocytosis and related clotting abnormalities (clinical and/or biological) using the national French Reference Centre for Mastocytosis database. From our cohort of 14 adult patients with clotting abnormalities (median age 46 years [range 26-75]), 4 had a presentation suggestive of a primary hemostasis disorder alone (by their symptoms and/or abnormal clotting tests [PFA, von Willebrand's disease [vWD] screening]) and 10 had a laboratory impairment of secondary hemostasis. Among these, 7 had bleeds characteristic of a coagulation cascade disorder (severe/life-threatening in 5 and mild in 2 patients). Clotting abnormalities were of variable severity, typically related to intense crisis of degranulation, such as anaphylactic reactions, and/or to severe organ infiltration by mast cells. Importantly, classical hemostatic management with platelet transfusion, fresh frozen plasma, or vitamin K infusions was unsuccessful, as opposed to the use of agents inhibiting mast cell activity, particularly steroids. This illustrates the crucial role of mast cell mediators such as tryptase and heparin, which interfere both with primary (mainly via inhibition of von Willebrand factor) and secondary hemostasis. There was interestingly an unusually high number of aggressive mastocytosis (particularly mast cell leukemia) and increased mortality in the group with secondary hemostasis disorders (n = 5, 36% of the whole cohort). Mast cell degranulation and/or high tumoral burden induce both specific biologic antiaggregant and anticoagulant states with a wide clinical spectrum ranging from asymptomatic to life-threatening bleeds. Hemostatic control is achieved by mast cell inhibitors such as steroids.
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http://dx.doi.org/10.1097/MD.0000000000001414DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4616764PMC
October 2015

Systemic inflammatory and autoimmune manifestations associated with myelodysplastic syndromes and chronic myelomonocytic leukaemia: a French multicentre retrospective study.

Rheumatology (Oxford) 2016 Feb 8;55(2):291-300. Epub 2015 Sep 8.

Service de rhumatologie, CH Croix Saint Simon.

Objective: We describe myelodysplastic syndrome (MDS)-associated systemic inflammatory and autoimmune diseases (SIADs), their treatments and outcomes and the impact of SIADs on overall survival in a French multicentre retrospective study.

Methods: In this study, 123 patients with MDS and SIADs were analysed.

Results: Mean age was 70 years (s.d. 13) and the male:female ratio was 2. The SIADs were systemic vasculitis in 39 (32%) cases, CTD in 31 (25%) cases, inflammatory arthritis in 28 (23%) cases, a neutrophilic disorder in 12 (10%) cases and unclassified in 13 cases (11%). The SIADs fulfilled the usual classification criteria in 75 (66%) cases, while complete criteria were not reached in 21 (19%) cases. A significant association was shown between chronic myelomonocytic leukaemia (CMML) and systemic vasculitis (P = 0.0024). One hundred and eighteen (96%) SIAD patients were treated (91% with steroids), with an 83% response to first-line treatment, including 80% for steroids alone. A second-line treatment for SIADs was required for steroid dependence or relapse in 48% of cases. The effect of MDS treatment on SIADs could be assessed in 11 patients treated with azacytidine and SIAD response was achieved in 9/11 (80%) and 6/11 (55%) patients at 3 and 6 months, respectively. Compared with 665 MDS/CMML patients without SIADs, MDS/CMML patients with SIADs were younger (P < 0.01), male (P = 0.03), less often had refractory anaemia with ring sideroblasts (P < 0.01), more often had a poor karyotype (16% vs 11%, P = 0.04) and less frequently belonged to low and intermediate-1 International Prognostic Scoring System categories, but no survival difference was seen between patients with MDS-associated SIADs and without SIADs (P = 0.5).

Conclusion: The spectrum of SIADs associated to MDS is heterogeneous, steroid sensitive, but often steroid dependent.
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http://dx.doi.org/10.1093/rheumatology/kev294DOI Listing
February 2016

Musculoskeletal symptoms in patients with cryopyrin-associated periodic syndromes: a large database study.

Arthritis Rheumatol 2015 Nov;67(11):3027-36

Saint Etienne University Hospital, Saint-Etienne, France.

Objective: To determine the type and frequency of musculoskeletal symptoms at onset and during followup of cryopyrin-associated periodic syndromes (CAPS).

Methods: We retrospectively recorded the articular and muscular symptoms of patients with CAPS followed up in French hospitals. Data were presented as frequencies or the median (range), and patient groups were compared using chi-square test, Fisher's exact test, and Mann-Whitney test.

Results: The study included 133 patients (33 children), 20 with familial cold autoinflammatory syndrome, 88 with Muckle-Wells syndrome, 22 with chronic infantile neurologic, cutaneous, articular syndrome, and 3 with unclassified CAPS. The median age was 35 years (range 0-78 years) at the time of the study, 1 year (range 0-41 years) at symptom onset, and 23 years (range 0-58 years) at diagnosis. The disease was sporadic in 17% of the patients. Cutaneous symptoms predominated at onset (77%), followed by articular symptoms (30%). The p.Thr348Met and p.Arg260Trp NLRP3 mutations were significantly associated with the presence and absence of articular symptoms at onset, respectively. During followup, 86% of the patients had musculoskeletal symptoms, 88% had arthralgia, and 58% had arthritis, but only 9% had joint destruction. Tendinopathies occurred in 21.5% of the patients, tender points in 16.5%, and myalgia in 33%. Only 3 patients had typical knee deformities. Radiographs were rarely obtained. Except for bone deformities, osteoarticular symptoms occurred at similar frequencies in the different CAPS phenotypes.

Conclusion: Joint manifestations were frequent in all CAPS phenotypes. Bone deformities were rare. Musculoskeletal manifestations varied within given families but tended to worsen over time.
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http://dx.doi.org/10.1002/art.39292DOI Listing
November 2015

Aplastic anemia as a feature of systemic lupus erythematosus: a case report and literature review.

Rheumatol Int 2015 Jun 30;35(6):1073-82. Epub 2014 Oct 30.

Service de Médecine Interne, Hôpital Nord, CHU de Saint-Étienne, 42055, Saint-Etienne Cedex 2, France,

Peripheral cytopenias are common in systemic lupus erythematosus, but bone marrow involvement is rarely reported. Aplastic anemia is the result of immune-mediated destruction of hematopoietic stem cells causing pancytopenia and characterized by an empty bone marrow. This rare but serious disease has been described as an unusual manifestation of systemic lupus erythematosus. We reviewed the 25 cases published in the English language literature and discuss the clinical presentation, outcome, treatment, and pathophysiology of aplastic anemia as a complication of systemic lupus erythematosus. We report here the first case of aplastic anemia associated with systemic lupus erythematosus treated with an allogeneic hematopoietic stem cell transplant. Over one half of patients received concomitantly the diagnoses of systemic lupus erythematosus and aplastic anemia. No clinical or histological features can distinguish primary aplastic anemia from aplastic anemia occurring in systemic lupus erythematosus patients. The overall mortality is about 15% and corticosteroid-based therapy alone or in combination with other immunomodulatory drugs can restore bone marrow function. Systemic lupus erythematosus may be complicated by bone marrow involvement. The diagnosis of peripheral cytopenias should be confirmed by bone marrow aspiration. All these patients should receive cortisone as a first treatment. Plasma exchanges seem to have some efficacy. Other different immunomodulatory therapies were used with variable results.
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http://dx.doi.org/10.1007/s00296-014-3162-4DOI Listing
June 2015