Publications by authors named "Pascal Cathebras"

Article Synopsis
  • MGUS-associated angioedema due to acquired C1 inhibitor deficiency (AAE-C1-INH) hasn't been specifically characterized before, prompting this study to explore its biological and clinical features over 30 years in France.
  • In a study of 41 patients, most had anti-C1INH antibodies, and treatments included acute management and long-term prophylaxis, with a significant number developing malignant blood disorders like lymphoma or myeloma.
  • The study found a link between the remission of angioedema and the underlying hematological malignancy, emphasizing the need for regular hematological evaluations in patients with MGUS-AE-C1-INH.
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Article Synopsis
  • The study examines the blood-related characteristics and overall prognosis of 127 patients with telomere biology disorders (TBD) who were diagnosed after age 15, highlighting a lack of data on this topic.
  • At diagnosis, significant haematological issues were present in nearly 76% of patients, with bone marrow failure (BMF) being the most common, affecting 46.5% of the cases, while some patients also developed additional complications over time.
  • The findings suggest that BMF patients tend to be younger and have a better survival rate compared to those with higher-risk blood cancers, indicating TBD as a complex multi-organ disease needing further research on its evolutionary nature and outcomes.
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Introduction: Severe acute respiratory syndrome coronavirus 2 is a viral respiratory infection that can cause systemic disorders and lead to death, particularly in older people. Proton pump inhibitors (PPIs) increase the risk of enteric and lung infections. Considering the broad use of PPIs in older people, the potential role of PPIs in COVID-19 could be of dramatic significance.

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Article Synopsis
  • This study evaluated the effectiveness and safety of the IL-5 receptor inhibitor benralizumab for treating patients with eosinophilic granulomatosis with polyangiitis (EGPA) across 28 European centers.
  • Out of 121 patients treated, complete responses increased from 12.4% at 3 months to 46.4% at 12 months, while partial responses decreased over the same period.
  • Notable improvements were seen in disease activity, as measured by the Birmingham Vasculitis Activity Score (BVAS), and a reduction in various disease manifestations, alongside better lung function.
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Background: Benralizumab is effective in the treatment of eosinophilic asthma and is being investigated for the treatment of other eosinophil-associated diseases. Reports on the use of benralizumab for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA) are limited to case reports and small case series.

Methods: We conducted a multicentre, retrospective study including EGPA patients treated with off-label benralizumab.

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Background: Activated phosphoinositide-3-kinase δ syndrome (APDS) is an inborn error of immunity (IEI) with infection susceptibility and immune dysregulation, clinically overlapping with other conditions. Management depends on disease evolution, but predictors of severe disease are lacking.

Objectives: This study sought to report the extended spectrum of disease manifestations in APDS1 versus APDS2; compare these to CTLA4 deficiency, NFKB1 deficiency, and STAT3 gain-of-function (GOF) disease; and identify predictors of severity in APDS.

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Aims: This study aimed to report the association of focal myositis (FM) and Behçet's disease (BD) and to analyse the main characteristics of such an association.

Methods: This is a retrospective multicentre study of patients with BD and FM (BD + FM+ group) and those without FM (BD - FM+ group). Clinical, laboratory, radiological, pathological, treatment and outcome data were analysed.

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Article Synopsis
  • Population differences significantly impact disease severity and treatment effects in biomedical research, yet there is a lack of rigorous practices in labeling subpopulations, especially for complex diseases like cancer.
  • Current classification terms like "Black" and "Caucasian" are overly broad and outdated, leading to inconsistencies in clinical research and potentially obscuring other critical factors influencing health.
  • To improve biomedical practices, a collaborative effort among various disciplines is needed to create clearer, more scientifically accurate labeling and to address the complexities of human diversity in medical research.
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Objective: While myocardial impairment is a predictor of poor prognosis in antineutrophil cytoplasm antibody (ANCA)-associated vasculitides (AAV), little is known about valvular involvement. This study aims at describing the clinical presentation, management, and outcome of endocarditis associated with AAV.

Methods: We conducted a multicenter retrospective study in centers affiliated with the French Vasculitis Study Group.

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Objectives: To describe the characteristics, treatment and outcome of patients with immune thrombocytopenia with clinical significance (ITPCS) associated with SLE.

Methods: This retrospective multicentre study included SLE patients who experienced ≥1 ITPCS (defined as ITP with attributable bleeding disorders and/or a platelet count <30×109/l). Other causes of secondary thrombocytopenia were excluded.

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Sjögren's syndrome (SjS) is a frequent systemic autoimmune disease responsible for a major decrease in patients' quality of life, potentially leading to life-threatening conditions while facing an unmet therapeutic need. Hence, we assessed the immunogenicity, efficacy, and tolerance of IFN-Kinoid (IFN-K), an anti-IFNα vaccination strategy, in a well-known mouse model of systemic autoimmunity with SjS-like features: MRL/MpJ-Faslpr/lpr (MRL/lpr) mice. Two cohorts (with ISA51 or SWE01 as adjuvants) of 26 female MRL/lpr were divided in parallel groups, "controls" (not treated, PBS and Keyhole Limpet Hemocyanin [KLH] groups) or "IFN-K" and followed up for 122 days.

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Unlabelled: Type 1 Gaucher disease (GD) is a rare autosomal recessive lysosomal storage disorder caused by deficient activity of beta-glucocerebrosidase, leading to accumulation of its substrate (glucosylceramide) in macrophages of the reticuloendothelial system, which are then referred to as Gaucher cells. The most frequent symptoms are asthenia, spleen and liver enlargement, bone abnormalities and cytopenia due to bone marrow infiltration. Lung involvement in GD is a rare finding, and it is unclear whether it may regress under enzyme replacement therapy (ERT) or substrate reduction therapy (SRT).

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Fibromyalgia (FM) is characterized by multiple symptoms including pain, fatigue, and sleep disorders, altering patient's quality of life. In the absence of effective pharmacological therapy, the last European guidelines recommend a multidisciplinary management based on exercise and education. Thus, our main objective was to measure the effectiveness of a healthcare organization offering a specific program of adapted physical activity combined with a therapeutic education program for FM patients.

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This paper argues that "functional," "medically unexplained," or "somatoform" symptoms and disorders necessarily require a patient-centered approach from the clinicians. In the first part, I address the multiple causes of the patients' suffering and I analyze the unease of the doctors faced with these disorders. I emphasize the iatrogenic role of medical investigations and the frequent failure in attempting to reassure the patients.

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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).

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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.

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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.

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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.

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