Cost-effectiveness of transcatheter aortic valve implantation in patients at low surgical risk in France: a model-based analysis of the Evolut LR trial.

Background: In the recent Evolut Low Risk randomized trial, transcatheter aortic valve implantation (TAVI) was shown to be non-inferior to surgery (SAVR) regarding the composite end point of all-cause mortality or disabling stroke at 24 months.

Aims: To evaluate the cost-effectiveness of self-expandable TAVI in low-risk patients, using the French healthcare system as the basis for analysis.

Methods: Mortality, health-related quality of life, and clinical event rates through two-year follow-up were derived from trial data (N = 725 TAVI and N = 678 SAVR; mean age: 73.

A global antiB cell strategy combining obinutuzumab and daratumumab in severe pediatric nephrotic syndrome.

Background: Steroid-sensitive nephrotic syndrome (SSNS) is, in most patients, a chronic disease with 80% experiencing at least one relapse after first flare. B cell depletion using rituximab is effective in preventing relapse in steroid-dependent (SDNS) patients but fails to maintain long-term remission following B cell recovery, possibly due to development of autoreactive long-lived plasma cells. We investigated sequential combination of antiCD20 antibody targeting all B cell subsets, and antiCD38 antibody with high plasma cell cytotoxicity in patients with uncontrolled SDNS after failure of one or several attempts at B cell depletion.

Systemic lupus erythematosus associated with thymoma: A fifteen-year observational study in France.

Objective: To describe the clinical, biological and pathological characteristics of patients with the association of SLE and thymic epithelial tumors (TET) in a retrospective multicenter series.

Methods: Cases diagnosed in France between 2000 and 2015 were collected after a call for observations from the French network for thymic epithelial tumors (RYTHMIC database) and the French National Society of Internal Medicine (SNFMI).

Results: Fourteen patients were identified, the majority were women (93%).

Mastocytosis among elderly patients: A multicenter retrospective French study on 53 patients.

Mastocytosis is a heterogeneous group of diseases with a young median age at diagnosis. Usually indolent and self-limited in childhood, the disease can exhibit aggressive progression in mid-adulthood. Our objectives were to describe the characteristics of the disease when diagnosed among elderly patients, for which rare data are available.

Hypertrophic Osteoarthropathy and Follicular Thyroid Cancer: A Rare Paraneoplastic Syndrome.

Background: Hypertrophic osteoarthropathy (HOA) is a rare condition characterized by bone and joint pain and digital clubbing usually associated with bronchopulmonary diseases. Primary HOA is rare and the pathogenesis remains unclear.

Objectives: Cases of HOA as a paraneoplastic syndrome associated with thyroid carcinoma are very rare - only 2 cases have been described in the literature.

[Socio-demographic characteristics and health status of patients at a free-of-charge outpatient clinic in Paris].

Purpose: In the context of the French National Health Service, a free access to healthcare facilities (the PASS: "permanence d'accès aux soins de santé") has been implanted in 2000 for patients without health insurance or those dealing with financial hardship. There is few data about socio-demographic characteristics of the patients using these services. The objective of this study was to provide descriptive data about socio-demographic characteristics and motivation of those patients who use these clinics.

Leukocyte telomere length in mastocytosis: correlations with depression and perceived stress.

Background: Mastocytosisis a rare disease associated with chronic symptoms related to mast cell mediator release. Patients with mastocytosis display high level of negative emotionality such as depression and stress sensibility. Brain mast cells are mainly localized in the diencephalon, which is linked to emotion regulatory systems.

Hydroxychloroquine-induced pigmentation in patients with systemic lupus erythematosus: a case-control study.

Importance: Hydroxychloroquine-induced pigmentation is not a rare adverse effect. Our data support the hypothesis that hydroxychloroquine-induced pigmentation is secondary to ecchymosis or bruising.

Objective: To describe the clinical features and outcome of hydroxychloroquine (HCQ)-induced pigmentation in patients with systemic lupus erythematosus (SLE).

The association of systemic lupus erythematosus and myasthenia gravis: a series of 17 cases, with a special focus on hydroxychloroquine use and a review of the literature.

The coexistence of systemic lupus erythematosus (SLE) and myasthenia gravis (MG) is rarely reported, and most of the published studies are case reports. Hydroxychloroquine, an antimalarial agent, is an essential treatment in patients with SLE but special caution is recommended when used in MG patients. We retrospectively analyzed the clinical features, laboratory findings, and outcome of 17 patients with both diseases with a special focus regarding hydroxychloroquine use and with a review of the literature.

Blood CD34-c-Kit+ cell rate correlates with aggressive forms of systemic mastocytosis and behaves like a mast cell precursor.

Mastocytosis is a heterogeneous disease characterized by the accumulation of mast cells in one or more organs. Our objective was to identify a peripheral mast cell precursor and assess its variation rate in mastocytosis. A peripheral blood phenotypic analysis was performed among 50 patients with mastocytosis who were enrolled in a prospective multicentric French study, and the phenotypic analysis results of the patients were compared with those of healthy donors.

Long-term outcome of 32 patients with chorea and systemic lupus erythematosus or antiphospholipid antibodies.

Objective: The aim of this work was to describe chorea during systemic lupus erythematosus or antiphospholipid antibodies and its long-term outcome.

Methods: We retrospectively analyzed clinical features, laboratory findings, imaging characteristics, and outcome in a series of 32 patients.

Results: Most patients were women (28 of 32), and mean age at onset of chorea was 20.

Systemic mastocytosis and bone involvement in a cohort of 75 patients.

Objectives: To investigate bone involvement in a large cohort of systemic mastocytosis (SM) patients, and evaluate the efficacy of bisphosphonate therapy.

Patients And Methods: From 2000 to 2004, 75 patients with SM according to WHO criteria underwent skeletal x-rays and bone mineral density (BMD) assessment. Sequential BMD assessments were performed in nine patients treated with bisphosphonate (mean follow-up 65 months).

Hemochromatosis and femoral head aseptic osteonecrosis: a nonfortuitous association?

Chondrocalcinosis, chronic pseudo-osteoarthritis arthropathy, and osteoporosis are classic osteoarticular complications of hemochromatosis (HC). Within HC, femoral head aseptic osteonecrosis (FHAO) is not notified in textbooks. We describe 3 cases of FHAO occurring in this setting in 3 patients homozygous for the C282Y mutation on HFE gene who had no other risk factors for FHAO.

Cardiac sarcoidosis: a retrospective study of 41 cases.

This retrospective study concerned 18 female and 23 male patients with cardiac sarcoidosis (CS). The average age at CS diagnosis was 38 years. CS was observed in white (73% of cases) and in black or Caribbean patients (27% of cases).

[Loss of consciousness in a child due to loperamide].

Loperamide is an antidiarrheal peripheral opiate agonist, with rare neurological secondary effects. We report the case of a 26-month-old child who had impaired consciousness under treatment by loperamide, and was treated successfully with naloxone. Limitations of the use of loperamide in young children are underlined.

[Treatment of systemic mastocytosis].

Background: Systemic mastocytosis is a rare disease, characterized by mast cells proliferation in various organs. Two types of clinical manifestations can be distinguished: those related to mast cells mediators release and those related to tumoral proliferation involving different organs, these later defining aggressive systemic mastocytosis. Until recently, treatment was mainly symptomatic, without anti tumoral effect.

[Unusual good prognosis for X-linked myotubular myopathy].

Recessive X-linked myotubular myopathy has recently been shown to be linked to the mutation of a gene located in the Xq28 region. Evolution is used to be considered as fatal but mild forms or forms with a better prognosis have been recorded since. We report a case in a patient, whose parents were warned of fatal outcome once the diagnosis was made during the neonatal period.

Different inactivating mutations of the mineralocorticoid receptor in fourteen families affected by type I pseudohypoaldosteronism.

We have analyzed the human mineralocorticoid receptor (hMR) gene in 14 families with autosomal dominant or sporadic pseudohypoaldosteronism (PHA1), a rare form of mineralocorticoid resistance characterized by neonatal renal salt wasting and failure to thrive. Six heterozygous mutations were detected. Two frameshift mutations in exon 2 (insT1354, del8bp537) and one nonsense mutation in exon 4 (C2157A, Cys645stop) generate truncated proteins due to premature stop codons.

[Organization of patient management in level II centers in the Paris area: a prospective survey].

Perinatal asphyxia is a common emergency for both obstetricians and pediatricians. A prospective study was conducted in 14 maternity hospitals (type II centres) in the Paris suburbs in order to assess pediatric activity and neonatal morbidity associated with supposed perinatal asphyxia in term newborns. Pediatricians were called in at birth very frequently: 1/20 deliveries.

Deflazacort versus prednisone in patients with giant cell arteritis: effects on bone mass loss.

Objective: To compare bone mass loss due to deflazacort versus prednisone in longterm treatment of patients with giant cell arteritis (GCA) in a randomized double blind comparative trial.

Methods: Seventy-four patients were included in a prospective multicenter study. Half received deflazacort (DFZ) and the other half prednisone (PR) for a minimum of 12 months.