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1.
N Engl J Med ; 385(17): 1547-1558, 2021 10 21.
Artigo em Inglês | MEDLINE | ID: mdl-34670042

RESUMO

BACKGROUND: Management of nonalcoholic steatohepatitis (NASH) is an unmet clinical need. Lanifibranor is a pan-PPAR (peroxisome proliferator-activated receptor) agonist that modulates key metabolic, inflammatory, and fibrogenic pathways in the pathogenesis of NASH. METHODS: In this phase 2b, double-blind, randomized, placebo-controlled trial, patients with noncirrhotic, highly active NASH were randomly assigned in a 1:1:1 ratio to receive 1200 mg or 800 mg of lanifibranor or placebo once daily for 24 weeks. The primary end point was a decrease of at least 2 points in the SAF-A score (the activity part of the Steatosis, Activity, Fibrosis [SAF] scoring system that incorporates scores for ballooning and inflammation) without worsening of fibrosis; SAF-A scores range from 0 to 4, with higher scores indicating more-severe disease activity. Secondary end points included resolution of NASH and regression of fibrosis. RESULTS: A total of 247 patients underwent randomization, of whom 103 (42%) had type 2 diabetes mellitus and 188 (76%) had significant (moderate) or advanced fibrosis. The percentage of patients who had a decrease of at least 2 points in the SAF-A score without worsening of fibrosis was significantly higher among those who received the 1200-mg dose, but not among those who received the 800-mg dose, of lanifibranor than among those who received placebo (1200-mg dose vs. placebo, 55% vs. 33%, P = 0.007; 800-mg dose vs. placebo, 48% vs. 33%, P = 0.07). The results favored both the 1200-mg and 800-mg doses of lanifibranor over placebo for resolution of NASH without worsening of fibrosis (49% and 39%, respectively, vs. 22%), improvement in fibrosis stage of at least 1 without worsening of NASH (48% and 34%, respectively, vs. 29%), and resolution of NASH plus improvement in fibrosis stage of at least 1 (35% and 25%, respectively, vs. 9%). Liver enzyme levels decreased and the levels of the majority of lipid, inflammatory, and fibrosis biomarkers improved in the lanifibranor groups. The dropout rate for adverse events was less than 5% and was similar across the trial groups. Diarrhea, nausea, peripheral edema, anemia, and weight gain occurred more frequently with lanifibranor than with placebo. CONCLUSIONS: In this phase 2b trial involving patients with active NASH, the percentage of patients who had a decrease of at least 2 points in the SAF-A score without worsening of fibrosis was significantly higher with the 1200-mg dose of lanifibranor than with placebo. These findings support further assessment of lanifibranor in phase 3 trials. (Funded by Inventiva Pharma; NATIVE ClinicalTrials.gov number, NCT03008070.).


Assuntos
Benzotiazóis/uso terapêutico , Cirrose Hepática/tratamento farmacológico , Hepatopatia Gordurosa não Alcoólica/tratamento farmacológico , Receptores Ativados por Proliferador de Peroxissomo/agonistas , Sulfonamidas/uso terapêutico , Benzotiazóis/administração & dosagem , Benzotiazóis/efeitos adversos , Índice de Massa Corporal , Diabetes Mellitus Tipo 2/complicações , Relação Dose-Resposta a Droga , Método Duplo-Cego , Feminino , Humanos , Cirrose Hepática/etiologia , Masculino , Pessoa de Meia-Idade , Hepatopatia Gordurosa não Alcoólica/etiologia , Índice de Gravidade de Doença , Sulfonamidas/administração & dosagem , Sulfonamidas/efeitos adversos
2.
Nat Mater ; 22(6): 754-761, 2023 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-37095227

RESUMO

Surface strain is widely employed in gas phase catalysis and electrocatalysis to control the binding energies of adsorbates on active sites. However, in situ or operando strain measurements are experimentally challenging, especially on nanomaterials. Here we exploit coherent diffraction at the new fourth-generation Extremely Brilliant Source of the European Synchrotron Radiation Facility to map and quantify strain within individual Pt catalyst nanoparticles under electrochemical control. Three-dimensional nanoresolution strain microscopy, together with density functional theory and atomistic simulations, show evidence of heterogeneous and potential-dependent strain distribution between highly coordinated ({100} and {111} facets) and undercoordinated atoms (edges and corners), as well as evidence of strain propagation from the surface to the bulk of the nanoparticle. These dynamic structural relationships directly inform the design of strain-engineered nanocatalysts for energy storage and conversion applications.

3.
Br J Dermatol ; 2024 Jun 20.
Artigo em Inglês | MEDLINE | ID: mdl-38900904

RESUMO

BACKGROUND: Very few studies evaluated the global prevalence of pruritus. OBJECTIVE: To assess its prevalence according to age, gender, ethnicity, and geographic regions. METHODS: An international cross-sectional study was conducted in 20 countries from January to April 2023. Participants were asked to complete a questionnaire on sociodemographics, confirm the presence or absence of a skin disease in the last 12 months and the presence or absence of pruritus in the last 7 days. RESULTS: The studied sample included 50552 individuals. The worldwide prevalence of pruritus was 39.8%. The age group ≥65 had the highest prevalence (43.3%). The prevalence was 40.7% among women and 38.9% among men (p<0.001). There was no significant difference between ethnicities (p=0.14). Compared to North America (41.6%), the prevalence of pruritus was significantly lower in Europe (35.9%, p<0.001), Australia (38.4%, p=0.017), East Asia (40.2%, p=0.04), and Latin America (36.5%, p<0.001), and higher in Africa (45,7%, p=0.007). No significant difference was found with the Middle East (40.2%, p=0.36). The prevalence was 40.3% in developed countries and 38.7% in BRICS countries 40.7% (p<10-3). LIMITATIONS: No information about the severity or type (acute, chronic) of pruritus. CONCLUSION: Global prevalence of pruritus revealed age, gender, and geographic region differences, with no ethnic differences.

4.
Br J Dermatol ; 190(2): 258-265, 2024 Jan 23.
Artigo em Inglês | MEDLINE | ID: mdl-37792727

RESUMO

BACKGROUND: Interest in the use of omalizumab to treat bullous pemphigoid (BP) in the event of resistance or contraindication to conventional therapies is currently based on limited evidence. OBJECTIVES: To assess the effectiveness and safety of omalizumab in BP and to identify predictive factors in response to treatment. METHODS: We conducted a French national multicentre retrospective study including patients with a confirmed diagnosis of BP treated with omalizumab after failure of one or several treatment lines. We excluded patients with clinically atypical BP, as per Vaillant's criteria. The criteria for clinical response to omalizumab were defined according to the 2012 international consensus conference. Anti-BP180-NC16A IgE enzyme-linked immunosorbent assay was performed on sera collected before initiating omalizumab, when available. RESULTS: Between 2014 and 2021, 100 patients treated in 18 expert departments were included. Median age at diagnosis was 77 years (range 20-98). Complete remission (CR) was achieved in 77% of patients, and partial remission in an additional 9%. CR was maintained 'off therapy' in 11.7%, 'on minimal therapy' in 57.1%, and 'on non-minimal therapy' in 31.2%. Median time to CR was 3 months (range 2.2-24.5). Relapse rate was 14%, with a median follow-up time of 12 months (range 6-73). Adverse events occurred in four patients. CR was more frequently observed in patients with an increased serum baseline level of anti-BP180-NC16A IgE (75% vs. 41%; P = 0.011). Conversely, urticarial lesions, blood total IgE concentration or eosinophil count were not predictive of CR. Patients with an omalizumab dosage > 300 mg every 4 weeks showed a similar final outcome to those with a dosage ≤ 300 mg every 4 weeks, but control of disease activity [median 10 days (range 5-30) vs. 15 days (range 10-60); P < 0.001] and CR [median 2.4 months (range 2.2-8.2) vs. 3.9 months (range 2.3-24.5); P < 0.001] were achieved significantly faster. CONCLUSIONS: We report the largest series to date of BP treated by omalizumab and confirm its effectiveness and safety in this indication. Serum baseline level of anti-BP180-NC16A IgE may predict response to treatment.


Assuntos
Penfigoide Bolhoso , Humanos , Adulto Jovem , Adulto , Pessoa de Meia-Idade , Idoso , Idoso de 80 Anos ou mais , Penfigoide Bolhoso/diagnóstico , Colágeno Tipo XVII , Omalizumab/uso terapêutico , Estudos Retrospectivos , Colágenos não Fibrilares , Autoantígenos , Imunoglobulina E , Autoanticorpos
5.
J Eur Acad Dermatol Venereol ; 38(7): 1373-1382, 2024 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-38314863

RESUMO

BACKGROUND: Dermatoses represent a significant burden. Patients and their caregivers can turn to social media and digital communities to exchange with each other. These public exchanges constitute real-life data that can be analysed to better understand the patients' feelings and expectations, and the daily difficulties encountered. OBJECTIVE: An infodemiologic study of public testimonies of patients and caregivers related to five dermatoses: eczema, rosacea, vitiligo, acne and psoriasis, over a 3-year time frame (September 2018 to September 2021) in France. To identify main topics of discussion, encountered difficulties and unmet medical needs. METHODS: Data extraction was performed based on a list of pertinent keywords. Web-users' profiles were determined by a specifically trained machine learning algorithm. Encountered difficulties were identified by manual annotation based on a standardized search grid. Co-occurrence analysis of difficulties allowed contextualization of challenges and unmet needs for each dermatosis. RESULTS: A total of 20,282 messages coming from 16,800 web users was extracted. The main topics of discussion were 'Impact on self- image and self-confidence' (23.6%), 'Generic discussion about therapeutics' (23.3%) and 'Burden of others' gaze' (12.8%). The top three mentioned difficulties for the five targeted dermatoses were similar and focused on 'Fear of/and management of symptoms', 'Impact on/and mood management' and 'Damaged self-image'. CONCLUSION: This infodemiologic study highlighted the real-life management of five skin diseases by patients and their caregivers, who turned to social networks to openly express their suffering and seek solutions. The joined analysis of the five diseases enabled a common comprehension of what it is to live with a skin disease, from a patient-centric point of view. The specific analysis of each patient group objectified specific challenges, and main unmet medical needs.


Assuntos
Emoções , Dermatopatias , Mídias Sociais , Humanos , França , Dermatopatias/psicologia , Dermatopatias/terapia , Feminino , Masculino , Acne Vulgar/psicologia , Acne Vulgar/complicações , Rosácea/psicologia , Rosácea/terapia , Psoríase/psicologia , Eczema/psicologia , Eczema/terapia , Vitiligo/psicologia , Vitiligo/terapia , Adulto
6.
Artigo em Inglês | MEDLINE | ID: mdl-38738687

RESUMO

BACKGROUND: As exposure to UV radiation is the primary modifiable environmental risk factor associated with skin cancer, it remains the principal focus of most prevention strategies. Numerous sun protection campaigns have been implemented worldwide; however, their impact on the actual incidence and mortality rates of skin cancer seems to be limited. To create successful skin cancer prevention campaigns, it is important to have a comprehensive understanding of individuals' attitudes and behaviours regarding sun protection. The aim of the current study was to determine and report on the prevalence of self-reported attitudes, knowledge and behaviours regarding two of the major sun protection recommendations-avoidance of sun exposure and use of sunscreens-in an international representative sample across five continents. METHODS: This cross-sectional study was conducted in 20 countries using a web-based online survey. FINDINGS: A total of 50,552 individuals, comprising 25,388 men (50.22%) and 25,164 women (49.78%), participated in the survey. Among them, 83.2% reported having been voluntarily exposed to the sun (for sun-basking reasons) at least once in the last 12 months, and 47.96% acknowledged being exposed to the sun between the hours of 10 AM and 4 PM. The primary reason for non-adherence was that these hours were the most convenient times (32.28%). Only 24.05% reported applying sunscreen every 2 h when outdoors. Forgetfulness was the primary reason as provided by 27.79% of participants. Males and older age groups were less likely to adopt sun-protective behaviours around the world. Forgetfulness and the challenges posed by time constraints seem to be the biggest barriers to proper adherence. INTERPRETATION: These findings should prompt the collaboration with health authorities and the manufacturers to enhance adherence by setting reasonable sunscreen prices and creating formulations that make their application less burdensome.

7.
Acta Derm Venereol ; 103: adv5285, 2023 Jun 26.
Artigo em Inglês | MEDLINE | ID: mdl-37358393

RESUMO

Atopic dermatitis is a chronic, relapsing and inflammatory skin disease. The impact of atopic dermatitis on the partners living with patients has been poorly investigated. The objective of this study was to evaluate the impact of atopic dermatitis in the daily lives of adult patients and to assess the burden of the disease on their partners. A population-based study was conducted on a representative sample of the general population of French adults aged 18 years of age using stratified, proportional sampling with a replacement design. Data were collected on 1,266 atopic dermatitis patient-partner dyads (mean age of patients 41.6 years, 723 (57.1%) women). The mean age of partners was 41.8 years. Patient burden, measured by the Atopic Dermatitis Burden Scale for Adults (ABS-A) score, was closely related to the objective atopic dermatitis severity: the mean score in the mild group (29.5) was significantly lower than in the moderate (43.9) and severe groups (48.6) (p < 0.0001). Partner burden, measured by the EczemaPartner score, was highly related to atopic dermatitis severity (p < 0.0001). Daytime sleepiness, measured by the Epworth Sleepiness Scale, showed a mean score of 9.24 in patients and 9.01 in their partners, indicating impaired sleep. Atopic dermatitis was found to decrease sexual desire in 39% and 26% of partners and patients respectively.


Assuntos
Dermatite Atópica , Adulto , Humanos , Feminino , Adolescente , Masculino , Dermatite Atópica/diagnóstico , Dermatite Atópica/epidemiologia , Qualidade de Vida , Índice de Gravidade de Doença , Libido , Pacientes
8.
Acta Derm Venereol ; 103: adv5382, 2023 Apr 21.
Artigo em Inglês | MEDLINE | ID: mdl-37083095

RESUMO

Atopic dermatitis is a chronic skin condition for which a range of systemic treatments have recently been approved. A treat-to-target strategy has been developed previously alongside an algorithm to guide the management of patients with atopic dermatitis. Here, we review the strategy and algorithm in the context of the evolving therapeutic landscape, and identify areas for further refinement and development.


Assuntos
Dermatite Atópica , Humanos , Administração Cutânea , Algoritmos , Dermatite Atópica/diagnóstico , Dermatite Atópica/tratamento farmacológico
9.
Clin Exp Dermatol ; 48(11): 1238-1246, 2023 Oct 25.
Artigo em Inglês | MEDLINE | ID: mdl-37409606

RESUMO

BACKGROUND: Little is known about phototype and the response to systemic treatment in psoriasis. OBJECTIVES: To assess the characteristics of psoriasis, the therapeutic choice and its efficacy according to phototype. METHODS: We included patients from the PsoBioTeq cohort initiating a first biologic. Patients were classified according to their phototype. The evaluation included disease characteristics, choice of the initial biologic and therapeutic response at 12 months based on 90% improvement from baseline in Psoriasis Area and Severity Index (PASI 90) and Dermatology Life Quality Index (DLQI) 0/1. RESULTS: Of the 1400 patients included, 423 (30.2%), 904 (64.6%) and 73 (5.2%) were in the phototype I-II, III-IV and V-VI groups, respectively. The V-VI group had a higher initial DLQI, and more frequently initiated ustekinumab. Patients in the V-VI group maintained the initial biologic prescribed as did the other phototype groups, even though the proportion of patients reaching PASI 90 and DLQI 0/1 at 12 months was lower in this group than the other groups. CONCLUSIONS: Patient phototype seems associated with quality of life and choice of the initial biologic in psoriasis. The phototype V-VI group less frequently switched treatments than did the other groups when the response was not efficient.


Assuntos
Produtos Biológicos , Psoríase , Humanos , Qualidade de Vida , Ustekinumab/uso terapêutico , Psoríase/tratamento farmacológico , Produtos Biológicos/uso terapêutico , Índice de Gravidade de Doença , Resultado do Tratamento
10.
J Eur Acad Dermatol Venereol ; 37(2): 443-450, 2023 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-36201355

RESUMO

BACKGROUND: The skin plays an important role in establishing interpersonal relationships, and thus visible skin disorders, which have a significant impact on physical appearance, influence other people's behaviours and attitudes. OBJECTIVE: To develop and validate a dermatologic-specific questionnaire to evaluate stigmatization in individuals with visible skin conditions. METHODS: Items were generated by a verbatim report based on qualitative interviews with patients with various dermatologic conditions. Subsequently, a study was implemented for psychometric analysis. A dermatology-specific stigmatization questionnaire (PUSH-D) was refined via item reduction according to inter-question correlations, consensus among experts and exploratory factor analysis. Internal consistency was determined by calculating Cronbach's α. Concurrent validity was determined by calculating the correlation between PUSH-D and the Dermatology Life Quality Index (DLQI) and the Rosenberg Self-Esteem Scale (RSES). RESULTS: From a primary list of 22 items, PUSH-D was reduced to a 17-item questionnaire, covering two pertinent dimensions based on the exploratory factor analysis. Construct validity was demonstrated, and PUSH-D showed good internal consistency (Cronbach's α = 0.9). PUSH-D correlated strongly with the DLQI 0.72 (p < 0.001) and moderately with the RSES 0.49 (p < 0.001). CONCLUSION: PUSH-D allows a comprehensive view of the degree of stigmatization in visible skin disorders, as well as the comparability of stigmatization levels across various skin conditions.


Assuntos
Dermatologia , Estereotipagem , Humanos , Qualidade de Vida , Dermatologia/métodos , Inquéritos e Questionários , Psicometria , Reprodutibilidade dos Testes
11.
J Eur Acad Dermatol Venereol ; 37 Suppl 7: 6-14, 2023 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-37806002

RESUMO

BACKGROUND: Several large studies on the burden of skin diseases have been performed in patients recruited in hospitals or clinical centres, thus missing people with skin diseases who do not undergo a clinical consultation. OBJECTIVES: To evaluate the burden of the most common dermatological diseases in adult patients across Europe, in terms of quality of life, work life, and stigmatization. METHODS: Population-based survey on a representative sample of the European general population aged 18 years or older. Participants who declared to have had one or more skin problem or disease during the previous 12 months completed the Dermatology Life Quality Index questionnaire, and answered questions regarding the impact of their skin disease on daily and work life, anxiety/depression, and stigmatization. RESULTS: The study population included 19,915 individuals, 44.7% of whom were men. Quality of life was particularly impaired in people with hidradenitis suppurativa (HS), and sexually transmitted diseases. About a half of participants with acne, alopecia, or chronic urticaria, and about 40% of those with atopic dermatitis (AD), skin cancers, or psoriasis reported a modest to extremely large effect of the disease on their quality of life. Overall, 88.1% of participants considered their skin disease as embarrassing in personal life and 83% in work life. About half of the respondents reported sleeping difficulty, feeling tired, and impact of the disease on taking care of themselves. Concerning stigmatization, 14.5% felt to have been rejected by others because of the skin disease, and 19.2% to have been looked at with disgust. Anxiety and depression were frequently reported by patients with all the diseases. CONCLUSIONS: Skin diseases may heavily affect patients' daily and work life, and cause feelings of stigmatization. An early intervention is needed to avoid consequences on the patients' life course.


Assuntos
Psoríase , Dermatopatias , Adulto , Masculino , Humanos , Feminino , Qualidade de Vida , Estereotipagem , Europa (Continente) , Inquéritos e Questionários , Dermatopatias/epidemiologia
12.
J Eur Acad Dermatol Venereol ; 37 Suppl 7: 27-37, 2023 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-37806001

RESUMO

BACKGROUND: The public perception of dermatology has been poorly investigated in Europe. OBJECTIVE: To determine the general public's perception of dermatologists in Europe. METHODS: This multinational, cross-sectional study was conducted within the framework of the EADV population-based survey on the 'Burden of skin diseases in Europe'. Data were collected using a web-based online survey on a representative sample of the general populations aged 18 years or more of 27 European countries. Proportional quota sampling with replacement design was used for each country. RESULTS: A total of 44,689 adult participants responded to the questionnaire, of whom 18,004 visited a dermatologist in the preceding 3 years. The dermatologist was the second most often visited specialist among all medical specialties, with 69.7%, 72.1%, 42.1% and 78.1% of participants in Western Europe (WE), Eastern Europe (EE), Northern Europe (NE) and Southern Europe (SE), respectively, having consulted a dermatologist over the past 24 months. Most participants across all regions agreed that the dermatologist was the first healthcare provider for chronic skin diseases (61.9% in WE, 69.8% in EE, 45.7% in NE and 60.4% in SE) and for skin cancers (65.5% in WE, 67.6% in EE, 42.4% in NE and 63.0% in SE). The five most common reasons for visiting a dermatologist among all participants were: naevi check-up or skin cancer screening (20.2%), chronic skin diseases (16.5%), acute skin diseases (12.4%), cosmetic advice or procedure (10.2%), hyperpigmentation or congenital lesions (9.1%) and hair or nail disorder (7.7%). Most participants (84.6% in WE, 82.5% in EE, 78.3% in NE and 82.8% in SE) were very swatisfied/somewhat satisfied with the service provided. CONCLUSION: Our study findings underscore the central role of dermatologists in skin health and highlights them as valued and trusted care providers across Europe. Understanding the perceived position of the dermatologist is the first step in improving health policy development and implementation. Notably, access to a dermatologist was lowest in NE, probably reflecting differences in healthcare organizational structures or possibly cultural differences in healthcare seeking behaviour.


Assuntos
Dermatopatias , Neoplasias Cutâneas , Adulto , Humanos , Dermatologistas , Estudos Transversais , Opinião Pública , Europa (Continente) , Dermatopatias/epidemiologia
13.
J Eur Acad Dermatol Venereol ; 37 Suppl 7: 17-24, 2023 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-37806003

RESUMO

BACKGROUND: The journey of patients with skin diseases through healthcare has been scarcely investigated. OBJECTIVE: To analyse the journey of people with skin diseases in the different healthcare environment in Europe. METHODS: This multinational, cross-sectional, European study was conducted on a representative sample of the adult general population of 27 European countries. The prevalence of the most frequent skin diseases was determined. Information was collected on the patient journey from the first medical consultation to the diagnosis, and the reasons for not consulting a healthcare professional. RESULTS: On a total of 44,689 individuals, 30.3% reported to have consulted a dermatologist during the previous 2 years. Participants consulted mainly for mole control or skin cancer screening (22.3%), followed by chronic skin diseases (16.2%). The diagnoses of acne, atopic dermatitis, psoriasis and rosacea were made most frequently by a dermatologist, while fungal skin infections were diagnosed more often by a general practitioner (GP), and sexually transmitted diseases (STD) by other specialists. The diagnosis was not always definitive at the first consultation, in particular for STD. The percentage of people who did not consult a healthcare professional for their skin disease was particularly high for acne (36.4%), alopecia (44.7%) and fungal infection (30.0%). Moreover, 17.7% of respondents with psoriasis did not consult. A high percentage of participants with alopecia thought that the disease was not worrying, while patients with psoriasis often answered that they were able to manage the disease since they had already consulted a doctor. Many patients with acne (41.1%) and fungal infection (48.2%) thought that they were able to handle the disease by themselves. CONCLUSION: The analysis of the self-reported medical journey of patients with common skin diseases may allow to understand the unmet needs of patients, thus improving outcomes and reducing expenses.


Assuntos
Acne Vulgar , Micoses , Psoríase , Infecções Sexualmente Transmissíveis , Dermatopatias , Adulto , Humanos , Estudos Transversais , Dermatopatias/diagnóstico , Dermatopatias/epidemiologia , Psoríase/diagnóstico , Psoríase/epidemiologia , Encaminhamento e Consulta , Alopecia , Acne Vulgar/diagnóstico , Acne Vulgar/epidemiologia
14.
J Eur Acad Dermatol Venereol ; 37(6): 1207-1214, 2023 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-36802102

RESUMO

BACKGROUND: Adverse pregnancy outcomes (APO) occur in 35% of patients with pemphigoid gestationis (PG). No biological predictor of APO has been established yet. OBJECTIVES: To assess a potential relationship between the occurrence of APO and the serum value of anti-BP180 antibodies at the time of PG diagnosis. METHODS: Multicentre retrospective study conducted from January 2009 to December 2019 in 35 secondary and tertiary care centres. INCLUSION CRITERIA: (i) diagnosis of PG according to clinical, histological and immunological criteria, (ii) ELISA measurement of anti-BP180 IgG antibodies determined at the time of PG diagnosis with the same commercial kit and (iii) obstetrical data available. RESULTS: Of the 95 patients with PG included, 42 had one or more APO, which mainly corresponded to preterm birth (n = 26), intrauterine growth restriction (IUGR) (n = 18) and small weight for gestational age at birth (n = 16). From a ROC curve, we identified a threshold of 150 IU ELISA value as the most discriminating to differentiate between patients with or without IUGR, with 78% sensitivity, 55% specificity, 30% positive and 91% negative predictive value. The threshold >150 IU was confirmed using a cross-validation based on bootstrap resampling, which showed that the median threshold was 159 IU. Upon adjusting for oral corticosteroid intake and main clinical predictors of APO, an ELISA value of >150 IU was associated with the occurrence of IUGR (OR = 5.11; 95% CI: 1.48-22.30; p = 0.016) but not with any other APO. The combination of blisters and ELISA values higher than 150 IU led to a 2.4-fold higher risk of all-cause APO (OR: 10.90; 95% CI: 2.33-82.3) relative to patients with blisters but lower values of anti-BP180 antibodies (OR of 4.54; 95% CI 0.92-34.2). CONCLUSION: These findings suggest that anti-BP180 antibody ELISA value in combination with clinical markers is helpful in managing the risk of APO, in particular IUGR, in patients with PG.


Assuntos
Penfigoide Gestacional , Penfigoide Bolhoso , Nascimento Prematuro , Gravidez , Feminino , Humanos , Recém-Nascido , Penfigoide Gestacional/diagnóstico , Estudos Retrospectivos , Penfigoide Bolhoso/diagnóstico , Vesícula , Resultado da Gravidez , Colágenos não Fibrilares , Ensaio de Imunoadsorção Enzimática , Imunoglobulina G , Autoantígenos , Autoanticorpos
15.
J Inherit Metab Dis ; 45(2): 340-352, 2022 03.
Artigo em Inglês | MEDLINE | ID: mdl-34910312

RESUMO

Mucopolysaccharidosis (MPS) disorders are a group of rare, progressive lysosomal storage diseases characterized by the accumulation of glycosaminoglycans (GAGs) and classified according to the deficient enzyme. Enzyme replacement therapy (ERT) of MPS VI has limited effects on ophthalmic, cardiovascular, and skeletal systems. Odiparcil is an orally available small molecule that results in the synthesis of odiparcil-linked GAGs facilitating their excretion and reducing cellular and tissue GAG accumulation. Improve MPS treatment was a Phase 2a study of the safety, pharmacokinetics/pharmacodynamics, and efficacy of two doses of odiparcil in patients with MPS VI. The core study was a 26-week, randomized, double-blind, placebo-controlled trial in patients receiving ERT and an open-label, noncomparative, single-dose cohort not receiving ERT. Patients aged ≥ 16 years receiving ERT were randomized to odiparcil 250 or 500 mg twice daily or placebo. Patients without ERT received odiparcil 500 mg twice daily. Of 20 patients enrolled, 13 (65.0%) completed the study. Odiparcil increased total urine GAGs (uGAGs), chondroitin sulfate, and dermatan sulfate concentrations. A linear increase in uGAG levels and odiparcil exposure occurred with increased odiparcil dose. Odiparcil demonstrated a good safety and tolerability profile. Individual analyses found more improvements in pain, corneal clouding, cardiac, vascular, and respiratory functions in the odiparcil groups vs placebo. This study confirmed the mechanism of action and established the safety of odiparcil with clinical beneficial effects after only a short treatment duration in an advanced stage of disease. Further assessment of odiparcil in younger patients is needed.


Assuntos
Mucopolissacaridoses , Mucopolissacaridose VI , N-Acetilgalactosamina-4-Sulfatase , Terapia de Reposição de Enzimas/métodos , Glicosaminoglicanos , Glicosídeos/uso terapêutico , Humanos , Mucopolissacaridose VI/tratamento farmacológico , N-Acetilgalactosamina-4-Sulfatase/uso terapêutico
16.
Int J Legal Med ; 136(3): 833-839, 2022 May.
Artigo em Inglês | MEDLINE | ID: mdl-35230486

RESUMO

Age estimation is a key factor for identification procedure in forensic context. Based on anthropological findings, degenerative changes of the sternal extremity of the 4th rib are currently used for age estimation. These have been adapted to post-mortem computed tomography (PMCT). The aim of this study was to validate a post-mortem computed tomography method based on a revision of the Iscan's method on a French sample. A total of 250 PMCT (aged from 18-98 years (IQR 36-68 years, median 51 years); 68 (27%) females) from the Medicolegal Institute of Paris (MLIP) were analyzed by two radiologists. The sternal extremity of 4th right rib was scored using method adapted from Iscan et al. Weighted κ was used to evaluate intra- and inter-observer reliability and Spearman correlation was performed to evaluate relationship between age and score. Confidence intervals for individual prediction of age based on 4th rib score and sex were computed with bootstrapping. The intra-observer reliability and inter-observer reliability were almost perfect (weighted κ = 0.85 [95%CI: 0.78-0.93] and 0.82 [95%CI 0.70-0.96] respectively). We confirmed a high correlation between the 4th rib score and subject age (rho = 0.72, p < 0.001), although the confidence intervals for individual age prediction were large, spanning over several decades. This study confirms the high reliability of Iscan method applied to PMCT for age estimation, although future multimodal age prediction techniques may help reducing the span of confidence intervals for individual age estimation.Trial registration: INDS 0,509,211,020, October 2020, retrospectively registered.


Assuntos
Determinação da Idade pelo Esqueleto , Costelas , Determinação da Idade pelo Esqueleto/métodos , Feminino , Humanos , Reprodutibilidade dos Testes , Costelas/diagnóstico por imagem , Tomografia
17.
Dermatology ; 238(3): 448-453, 2022.
Artigo em Inglês | MEDLINE | ID: mdl-34515100

RESUMO

BACKGROUND: The prevalence and impact of pruritus, pain, and other sensory symptoms in skin diseases are poorly known. OBJECTIVE: To assess the frequency of these symptoms with dermatoses and their association with depression using data from the "Objectifs Peau" survey. METHODS: A representative sample of 20,012 French individuals was created using the usual quota method. RESULTS: When patients suffered from both pruritus and skin pain, they had a higher relative risk of psychological suffering (2.9) than those who suffered only from pruritus (1.4) or skin pain (1.2). Pruritus was reported in 48.55% of patients with acne, 43.24% with mycoses, 44.35% with warts, and 36.51% with rosacea. For skin pain, the results were 11.22%, 27.59%, and 16.13% for atopic dermatitis, acne, and warts, respectively. Other unpleasant sensations, such as tingling or burning, were also frequently reported. CONCLUSION: Pruritus, pain, or other sensory symptoms were found to be common not only in classic pruritic skin diseases but also in acne, rosacea, or warts. The association of pruritus and pain dramatically increased psychological suffering. These symptoms must be systematically searched for in patients, especially since new therapeutic possibilities are emerging for the symptomatic treatment of pruritus.


Assuntos
Acne Vulgar , Rosácea , Dermatopatias , Verrugas , Depressão/epidemiologia , Humanos , Dor/epidemiologia , Prurido/tratamento farmacológico , Prurido/epidemiologia , Prurido/etiologia , Rosácea/complicações , Rosácea/epidemiologia , Dermatopatias/diagnóstico , Dermatopatias/epidemiologia
18.
Dermatology ; 238(4): 630-639, 2022.
Artigo em Inglês | MEDLINE | ID: mdl-34883480

RESUMO

BACKGROUND: Psoriasis is a chronic inflammatory skin disease that has a profound effect on health-related quality of life (HRQoL). Patient education programmes may help patients to gain life-long control over their chronic disease. OBJECTIVE: This multicentre randomised controlled study evaluated whether a standardised multidisciplinary education programme was beneficial to psoriasis patients. METHODS: Adults with moderate-to-severe psoriasis were randomly assigned (1:1) to an intervention group to receive an educational programme or to a control group to receive usual care. Randomization was stratified by previous treatment history. The primary outcome was HRQoL, assessed by scoring the Skindex-29 domains emotion, symptom, and functioning. Psoriasis severity was assessed using the psoriasis area severity index (PASI). Levels of perceived stress, patient knowledge about psoriasis, and patient satisfaction were also assessed. Follow-up evaluations were performed at 3, 6, and 12 months. RESULTS: A total 142 patients formed the intention-to-treat population: 70 in the control group and 72 in the intervention group. Skindex component scores and the PASI were significantly lower at 3, 6, and 12 months as compared to baseline in both groups, but no significant differences were found between the groups. Knowledge about psoriasis improved significantly during follow-up amongst patients from the intervention group compared to controls (68% of correct answers vs. 56%; p < 0.01). Patient satisfaction with psoriasis management and treatment was also better in the intervention group. CONCLUSIONS: The standardised education programme did not improve HRQoL and disease severity in psoriasis, but led to a significant improvement in patient knowledge about the disease and increased patient satisfaction.


Assuntos
Educação de Pacientes como Assunto , Psoríase , Adulto , Conhecimentos, Atitudes e Prática em Saúde , Humanos , Avaliação de Programas e Projetos de Saúde , Psoríase/psicologia , Psoríase/terapia , Qualidade de Vida , Índice de Gravidade de Doença
19.
Acta Derm Venereol ; 102: adv00717, 2022 May 19.
Artigo em Inglês | MEDLINE | ID: mdl-35470404

RESUMO

An improved understanding of the impact of skin cancer on patients' daily life may optimize disease management. This questionnaire survey of adult patients in real-world settings aimed to assess patient perception of the diagnosis announcement, and the impact of the diagnosis on the patients' professional and personal life. Data from 355 patients with melanoma and 320 patients with basal cell carcinoma (BCC) were analysed. Melanoma significantly impacted the couple, and sexual relationships, as well as family and social life, and reduced the patient's libido more significantly than did BCC (all p < 0.05). Melanoma and BCC significantly impacted the patients' professional and personal lives. The word "cancer" used for a BCC announcement has a high anxiety-producing meaning for the patient. The announcement of each skin cancer should be made during a dedicated consultation, with more time devoted to the patient and with specific empathy, in order to improve reassurance of the patient.


Assuntos
Carcinoma Basocelular , Carcinoma de Células Escamosas , Melanoma , Neoplasias Cutâneas , Adulto , Carcinoma Basocelular/diagnóstico , Carcinoma Basocelular/patologia , Carcinoma de Células Escamosas/patologia , Humanos , Melanoma/diagnóstico , Melanoma/patologia , Percepção , Qualidade de Vida , Neoplasias Cutâneas/diagnóstico , Neoplasias Cutâneas/patologia
20.
Acta Derm Venereol ; 102: adv00733, 2022 Jun 09.
Artigo em Inglês | MEDLINE | ID: mdl-35506359

RESUMO

Paediatric psoriasis has been associated with school absenteeism, limitation of physical activities, psychiatric disorders and, in the longer term, with sexual dysfunction and addictions. This raises the hypothesis that childhood onset psoriasis may affect patients' educational development, and further social and professional outcomes. This study evaluated the relationship between childhood onset psoriasis and patients' educational and socioeconomic characteristics, and the development of addictions in adulthood. This cross-sectional ancillary study captured patients' characteristics at baseline in the French PSOBIOTEQ registry. Data in adulthood included: educational (baccalaureate) and socioeconomic (working activity) groups, smoking status (self-reporting of being a current smoker vs past smoker or non-smoker), alcohol consumption (defined as at least 1 glass of alcoholic beverage per day), and living conditions (alone/family/social institutions; child at home). A total of 1,960 patients were included, of whom 26.2% had childhood onset psoriasis. In multivariate analyses, childhood onset psoriasis was associated with smoker status (p = 0.02). No association was observed with educational level, working activity, living conditions, or alcohol consumption. This study provides reassuring data overall with regard to the impact of childhood onset psoriasis on major social outcomes. Evidence for some association with addictive behaviours paves the way for larger prospective studies assessing in depth the social and educational impact of this disease.


Assuntos
Comportamento Aditivo , Psoríase , Adulto , Comportamento Aditivo/epidemiologia , Criança , Estudos Transversais , Escolaridade , Humanos , Estudos Prospectivos , Psoríase/diagnóstico , Psoríase/epidemiologia , Fatores Socioeconômicos
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