To describe the effectiveness and safety of biologics for the treatment of relapsing and/or refractory polyarteritis nodosa (PAN).
A retrospective European collaborative study was conducted in ...patients with PAN who received biologics for relapsing and/or refractory disease.
Forty-two patients with PAN received a total of 53 biologic courses, including TNF-α blockers in 15 cases, rituximab (RTX) in 18 cases, tocilizumab (TCZ) in 10 cases and other biologics in 10 cases. TNF-α blockers and TCZ were mainly used for refractory diseases whereas RTX was mainly initiated for relapsing disease. After a median follow-up of 29 (8-50) months, remission, partial response, treatment failure and treatment discontinuation due to severe adverse events occurred in, respectively, 40%, 13%, 40% and 7% of patients receiving TNF-α blockers, 50%, none, 30% and 20% of TCZ recipients, and 33%, 11%, 56% and none of the RTX recipients. No remission was noted in patients treated with other biologics. Severe adverse events were observed in 14 (28%) patients without significant differences between the three biologics, leading to early biologics discontinuation in only three cases.
These results suggest that TCZ may be effective in relapsing and/or refractory PAN. Our data warrant further study to confirm these findings.
BackgroundVascular phenotype is associated with a poor prognosis in systemic sclerosis (SSc). The identification of its risk factors could facilitate its early detection.ObjectivesTo explore risk ...factors for a vascular phenotype of SSc, among them a history of pre-eclampsia.MethodsThis observational multicentre case–control study enrolled adult women fulfilling European Alliance of Associations for Rheumatology 2013 diagnosis criteria for SSc and having a pregnancy history≥6 months before SSc diagnosis in 14 French hospital-based recruiting centres from July 2020 to July 2022. Cases had specific vascular complications of SSc defined as history of digital ischaemic ulcers, pulmonary arterial hypertension, specific cardiac involvement or renal crisis. Women with SSc were included during their annual follow-up visit and filled in a self-administered questionnaire about pregnancy. A case report form was completed by their physician, reporting data on medical history, physical examination, clinical investigations and current medication. The main outcome was the presence/absence of a personal history of pre-eclampsia before SSc diagnosis, according to the validated pre-eclampsia questionnaire.Results378 women were included: 129 cases with a vascular phenotype and 249 matched controls. A history of pre-eclampsia was reported in 5 (3.9%) cases and 12 (4.8%) controls and was not associated with a vascular phenotype (OR=0.96, 95% CI 0.28 to 3.34, p=0.9). Besides, Rodnan skin score and disease duration≥5 years were risk factors for vascular phenotype.ConclusionsIn women with SSc and a pregnancy history≥6 months before SSc, a history of pre-eclampsia is not associated with a vascular phenotype.
Cancer can occur in patients with inflammatory myopathies. This association is mainly observed in dermatomyositis, and myositis-specific antibodies have allowed us to delineate patients at an ...increased risk. Malignancy is also reported in patients with necrotizing autoimmune myopathies, but the risk remains elusive. Anti-signal recognition particle or anti-HMGCR antibodies have been specifically associated with necrotizing autoimmune myopathies. We aimed at screening the incidence of cancer in necrotizing autoimmune myopathies. A group of patients (n = 115) with necrotizing autoimmune myopathies with or without myositis-specific antibodies was analysed. Malignancy occurred more frequently in seronegative necrotizing autoimmune myopathies patients and in HMGCR-positive patients compared to anti-signal recognition particle positive patients. Synchronous malignancy was diagnosed in 21.4% and 11.5% of cases, respectively, and incidence of cancer was higher compared to the general population in both groups. No specific type of cancer was predominant. Patients suffering from a synchronous cancer had a decreased median survival time. Cancer screening is necessary in seronegative necrotizing autoimmune myopathies and in HMGCR-positive patients but not in anti-signal recognition particle-positive patients.
Ocular immunotherapy-related adverse events (IRAEs), although rare, can be sight-threatening. Our objective was to analyze ocular IRAEs diagnosed in France from the marketing of immune checkpoint ...inhibitors (ICPIs) until June 2021 and to review the literature. We collected the cases of 28 patients (36 ocular IRAEs), occurring after an average of 17 weeks (±19). Forty-six percent of patients were treated for metastatic melanoma. Anti-PD1 agents were responsible for 57% of the IRAEs. Anterior uveitis was the most common (44%), followed by panuveitis (28%). Of 25 uveitis cases, 80% were bilateral and 60% were granulomatous. We found one case with complete Vogt-Koyanagi–Harada syndrome and one case of birdshot retinochoroidopathy. The other IRAEs were eight ocular surface disorders, one optic neuropathy, and one inflammatory orbitopathy. Seventy percent of the IRAEs were grade 3 according to the common terminology of AEs. ICPIs were discontinued in 60% of patients and 50% received local corticosteroids alone. The literature review included 230 uveitis cases, of which 7% were granulomatous. The distributions of ICPIs, cancer, and type of uveitis were similar to our cohort. Ocular IRAEs appeared to be easily controlled by local or systemic corticosteroids and did not require routine discontinuation of ICPIs. Further work is still warranted to define the optimal management of ocular IRAEs.
Objective
The usefulness of positron emission tomography–computed tomography (PET‐CT) with 18F‐labeled fluorodeoxyglucose (18F‐FDG) for the diagnosis of lymphoma in patients with primary Sjögren's ...syndrome (SS) is unclear, since the abnormalities it reveals may be due to systemic manifestations of SS. This study was undertaken to compare 18F‐FDG–PET‐CT in patients with primary SS with lymphoma and those without lymphoma in order to identify patterns associated with lymphoma.
Methods
A retrospective study was conducted in 2 centers and included patients who met the American College of Rheumatology/European League Against Rheumatism 2016 criteria for primary SS and had undergone PET‐CT. Two independent readers who were blinded with regard to lymphoma diagnosis analyzed PET‐CT scans. Abnormalities were compared between patients with and those without lymphoma.
Results
Of the 45 patients included, 15 had lymphoma. Compared to patients without lymphoma, the mean size (P = 0.048) and maximum standardized uptake value (SUVmax) (P = 0.001) of the parotid glands were higher in patients with lymphoma. FDG uptake in the lymph nodes was observed in 53.3% of the patients with lymphoma and 43.3% of the patients without lymphoma, with no difference in the number of sites, uptake pattern, or mean SUVmax. Focal pulmonary uptake (nodules or condensations) was observed in 5 of the patients with lymphoma (33.3%) but only 1 patient without lymphoma (3.3%) (P = 0.01). Having an SUVmax in the parotid gland of ≥4.7 and/or the presence of focal pulmonary lesions was highly suggestive of lymphoma (sensitivity 80%, specificity 83.3%).
Conclusion
Some systemic manifestations of primary SS (lymphadenopathy, pulmonary involvement, and salivary gland involvement) can be visualized by PET‐CT. Involvement of the lymph nodes and parotid glands is commonly observed with a similar frequency in SS patients with and those without lymphoma. An SUVmax in the parotid glands of ≥4.7 and/or the presence of focal lung lesions are associated with the diagnosis of lymphoma.
The usefulness of positron emission tomography-computed tomography (PET-CT) with
F-labeled fluorodeoxyglucose (
F-FDG) for the diagnosis of lymphoma in patients with primary Sjögren's syndrome (SS) ...is unclear, since the abnormalities it reveals may be due to systemic manifestations of SS. This study was undertaken to compare
F-FDG-PET-CT in patients with primary SS with lymphoma and those without lymphoma in order to identify patterns associated with lymphoma.
A retrospective study was conducted in 2 centers and included patients who met the American College of Rheumatology/European League Against Rheumatism 2016 criteria for primary SS and had undergone PET-CT. Two independent readers who were blinded with regard to lymphoma diagnosis analyzed PET-CT scans. Abnormalities were compared between patients with and those without lymphoma.
Of the 45 patients included, 15 had lymphoma. Compared to patients without lymphoma, the mean size (P = 0.048) and maximum standardized uptake value (SUVmax) (P = 0.001) of the parotid glands were higher in patients with lymphoma. FDG uptake in the lymph nodes was observed in 53.3% of the patients with lymphoma and 43.3% of the patients without lymphoma, with no difference in the number of sites, uptake pattern, or mean SUVmax. Focal pulmonary uptake (nodules or condensations) was observed in 5 of the patients with lymphoma (33.3%) but only 1 patient without lymphoma (3.3%) (P = 0.01). Having an SUVmax in the parotid gland of ≥4.7 and/or the presence of focal pulmonary lesions was highly suggestive of lymphoma (sensitivity 80%, specificity 83.3%).
Some systemic manifestations of primary SS (lymphadenopathy, pulmonary involvement, and salivary gland involvement) can be visualized by PET-CT. Involvement of the lymph nodes and parotid glands is commonly observed with a similar frequency in SS patients with and those without lymphoma. An SUVmax in the parotid glands of ≥4.7 and/or the presence of focal lung lesions are associated with the diagnosis of lymphoma.
Acid sphingomyelinase deficiency (ASMD) or Niemann-Pick disease types A, A/B, and B is a progressive, life-limiting, autosomal recessive disorder caused by sphingomyelin phosphodiesterase 1 (SMPD1) ...gene mutations. There is a need to increase the understanding of morbidity and mortality across children to adults diagnosed with ASMD. This observational retrospective survey analysed medical records of patients with ASMD with retrievable data from 27 hospitals in France, diagnosed/followed up between 1.sup.st January 1990 and 31.sup.st December 2020. Eligible records were abstracted to collect demographic, medical/developmental history, and mortality data. Survival outcomes were estimated from birth until death using Kaplan-Meier survival analyses; standardised mortality ratio (SMR) was also explored. A total of 118 medical records of patients with ASMD (type B n = 94, type A n = 15, and type A/B n = 9) were assessed. The majority of patients were males (63.6%); the median range age at diagnosis was 8.0 1.0-18.0 months (type A), 1.0 0-3 year (type A/B), and 5.5 0-73 years (type B). Overall, 30 patients were deceased at the study completion date; the median range age at death for patients with ASMD type A (n = 14) was 1 0-3.6 year, type A/B (n = 6) was 8.5 3.0-30.9 years, and type B (n = 10) was 57.6 3.4-74.1 years. The median 95% confidence interval (CI) survival age from birth in patients with ASMD type A and type A/B was 2.0 1.8-2.7 years and 11.4 5.5-18.5 years, respectively. Survival analysis in ASMD type B was explored using SMR 95% CI analysis (3.5 1.6-5.9), which showed that age-specific deaths in the ASMD type B population were 3.5 times more frequent than those in the general French population. The causes of death were mostly severe progressive neurodegeneration (type A: 16.7%), cancer (type B: 16.7%), or unspecified (across groups: 33.3%). This study illustrated a substantial burden of illness with high mortality rates in patients with ASMD, including adults with ASMD type B, in France.
We conducted a national in-depth analysis including pharmacovigilance reports and clinical study to assess the reporting rate (RR) and to determine the clinical profile of polymyalgia rheumatica ...(PMR) and giant cell arteritis (GCA) in COVID-19-vaccinated individuals. First, based on the French pharmacovigilance database, we estimated the RR of PMR and GCA cases in individuals aged over 50 who developed their initial symptoms within one month of receiving the BNT162b2 mRNA, mRNA-1273, ChAdOx1 nCoV-19, and Ad26.COV2.S vaccines. We then conducted a nationwide survey to gather clinical profiles, therapeutic management, and follow-up data from individuals registered in the pharmacovigilance study. A total of 70 854 684 COVID-19 vaccine doses were administered to 25 260 485 adults, among which, 179 cases of PMR (RR 7. 1 cases/1 000 000 persons) and 54 cases of GCA (RR 2. 1 cases/1 000 000 persons) have been reported. The nationwide survey allowed the characterization of 60 PMR and 35 GCA cases. Median time to the onset of first symptoms was 10 (range 2-30) and 7 (range 2-25) days for PMR and GCA, respectively. Phenotype, GCA-related ischemic complications and -large vessel vasculitis as well as therapeutic management and follow-up seemed similar according to the number of vaccine shots received and when compared to the literature data of unvaccinated population. Although rare, the short time between immunization and the onset of first symptoms of PMR and GCA suggests a temporal association. Physician should be aware of this potential vaccine-related phenomenon.
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