Practical guidelines on the prevention and management of diabetes-related foot disease (IWGDF 2023 update).

Diabetes-related foot disease results in a major global burden for patients and the healthcare system. The International Working Group on the Diabetic Foot (IWGDF) has been producing evidence-based guidelines on the prevention and management of diabetes-related foot disease since 1999. In 2023, all IWGDF Guidelines have been updated based on systematic reviews of the literature and formulation of recommendations by multidisciplinary experts from all over the world. In addition, a new guideline on acute Charcot neuro-osteoarthropathy was created. In this document, the IWGDF Practical Guidelines, we describe the basic principles of prevention, classification and management of diabetes-related foot disease based on the seven IWGDF Guidelines. We also describe the organisational levels to successfully prevent and treat diabetes-related foot disease according to these principles and provide addenda to assist with foot screening. The information in these practical guidelines is aimed at the global community of healthcare professionals who are involved in the care of persons with diabetes. Many studies around the world support our belief that implementing these prevention and management principles is associated with a decrease in the frequency of diabetes-related lower-extremity amputations. The burden of foot disease and amputations is increasing at a rapid rate, and comparatively more so in middle to lower income countries. These guidelines also assist in defining standards of prevention and care in these countries. In conclusion, we hope that these updated practical guidelines continue to serve as a reference document to aid healthcare providers in reducing the global burden of diabetes-related foot disease.

Dupilumab provides sustained effectiveness on patient-reported outcomes and favorable safety in patients with moderate-to-severe atopic dermatitis: Up to 5-year results from the daily practice BioDay registry.

BACKGROUND: Long-term daily practice data on patient-reported benefits of dupilumab for atopic dermatitis (AD) remains limited. OBJECTIVE: To evaluate patient-reported outcome measures (PROMs) and the safety of dupilumab in patients with moderate-to-severe AD over a follow-up period of up to 5 years. METHODS: Data were extracted from the prospective, multicenter BioDay registry (October 2017-2022) of patients with moderate-to-severe AD treated with dupilumab in daily practice. RESULTS: In total 1223 patients, 1108 adults and 115 pediatric patients were included. After ≥1 year of treatment, mean Patient-Oriented Eczema Measure (POEM), Dermatology Life Quality Index (DLQI), Numeric rating scale (NRS)-pruritus ranged between 7.8 and 8.7, 3.5 and 4.2, and 2.9 and 3.1 in adults, respectively, whilst these patient-reported outcome measures (PROMs) ranged between 8.9 and 10.9, 4.4 and 6.4, and 3.0 and 3.7 in pediatric patients, respectively. At follow-up, overall work impairment decreased from 40.1% to 16.3% to 13.3% in adults. Furthermore, class I obesity and itch-dominant patients generally had less favorable treatment response. Of all patients, 66.8% reported ≥1 adverse event, with conjunctivitis being the most common (33.7%). LIMITATIONS: The overall percentage of missing values for selected PROMs was 26% in adults and 46% in pediatric patients. CONCLUSION: In addition to favorable safety, dupilumab has demonstrated sustained effectiveness across various PROMs, underscoring the treatment benefits from patients' perspectives.

Telemedicine and Remote Management of Patients with Heart Failure: From Theory to Daily Practice.

Background: Heart failure (HF) is responsible for a high number of hospitalizations, caused by a progressive worsening quality of life. Telemedicine allows for better management of patients' complex conditions, improving the care released. However, the risk of remaining at a testing stage often limits the integration of remote care in daily pathways for HF patients. The aim of this study is to outline the steps needed to integrate telemedicine activities into ordinary HF clinic practices. This methodology is applied to observe activities and trend improvements over a 12-month routine phase. Method: Three steps have been defined for an efficient introduction of remote care services in ordinary activities, integrating them with traditional in-person care: (i) introduction of temporary telemedicine projects, (ii) systematization of telemedicine pathways, and (iii) evaluation of monitoring phase. Observational data have been collected from structured interviews to show the rate of telemedicine activities achieved in clinical practice over the last year. Results: The methodology has been proposed in the HF clinic of the Italian hospital ASST Bergamo Est. After an initial testing phase, in which usability and user experience have been tested, four different remote activities were added: (i) telemonitoring for patients with an implantable device, (ii) follow-up televisits, (iii) nursing telephone support, and (iv) high-intensity telesurveillance pathways for patients after an HF acute event. During the last year, 218 telemonitoring pathways, 75 televisits, 500 telephone calls, and nine telesurveillance pathways have been performed. Success rates were high, and patients gave positive feedback. Conclusion: By integrating multiple telemedicine activities, it has been possible to better manage complex patients, keep track of disease progression, and improve their participation in care.

Survey on electronic visual field data transfer practices among Japan Glaucoma Society board members.

BACKGROUND: Visual field (VF) testing in combination with a specialized VF analysis software is critical for characterizing and monitoring visual loss in glaucoma. Although performing glaucoma progression analysis requires original VF data rather than printouts or image files, extent of VF data transfer between referring and referred ophthalmologists is unclear. Here, we surveyed glaucoma specialists who belong to the Japan Glaucoma Society (JGS). METHODS: An internet survey of daily practice patterns regarding electronic VF data transfer at the time of glaucoma referrals (referring/referred) was sent to all 50 JGS board members. The survey consisted with 11 questionnaires, and the response rate was 100%. RESULTS: The respondents included 33 university hospital ophthalmologists (66%) (Q1), and those scattered throughout Japan (Q2). All respondents used Humphrey Visual Filed Analyzer (HFA) (Q3) and at least one of a VF progression analysis software (Q4). Ten respondents (20%) actively transferred electronic VF data, while 40 (80%) did not (Q5). The major reasons for not actively transferring data electronically were that there was no support for data transfer by neighboring (n = 26, 65%) and/or own (25, 63%) institutes (Q6). All 40 inactive respondents responded that electronic data transfer is ideal (Q7). All 10 active respondents transferred data using USB flash memory (Q8). Of the 10 active respondents, seven (70%) reported that the percentage of referral letters accompanying electronic VF data in a format that allows for progression analysis from the beginning was less than 25% (Q9). When the referral letters did not accompany the electronic VF data, four (40%) reported that they further requested the data transfer in < 25% of cases (Q10). When the 10 active respondents were requested to transfer data, six (60%) had experienced rejection due to various reasons (Q11). CONCLUSION: An internet survey showed that 80% of the JGS board members were not actively transferring VF data mainly because of the absence of a system in place at institutions for sending and receiving data, although they feel that the electronic VF data transfer is ideal. The results provide basic data for future discussions on the promotion of the VF data transfer.

Patient-centered dupilumab dosing regimen leads to successful dose reduction in persistently controlled atopic dermatitis.

BACKGROUND: At present, no real-world studies are available on different dupilumab dosing regimens in controlled atopic dermatitis (AD). The aim of this study was to clinically evaluate a patient-centered dupilumab dosing regimen in patients with controlled AD and to relate this to serum drug levels and serum biomarkers. METHODS: Ninety adult AD patients from the prospective BioDay registry were included based on their dupilumab administration interval according to a predefined patient-centered dosing regimen. Group A (n = 30) did not fulfill the criteria for interval prolongation and continued using the standard dupilumab dosage (300 mg/2 weeks), group B (n = 30) prolonged dupilumab interval with 50% (300 mg/4 weeks), and group C (n = 30) prolonged dupilumab interval with 66%-75% (300 mg/6-8 weeks). AD severity score, patient-reported outcomes, serum dupilumab levels, and serum biomarkers were analyzed over time. RESULTS: Disease severity scores did not significantly change over time during the tapering period in any of the groups. In groups B and C, the Numeric Rating Scale (NRS)-pruritus temporarily significantly increased after interval prolongation but remained low (median NRS-pruritus≤4). Median dupilumab levels remained stable in group A (standard dosage), but significantly decreased in groups B and C (24.1 mg/L (IQR = 17.1-45.6); 12.5 mg/L (IQR = 1.7-22.3)) compared with the levels during the standard dosage (88.2 mg/L [IQR = 67.1-123.0, p < .001]). Disease severity biomarker levels (CCL17/CCL18) remained low in all study groups during the whole observation period. CONCLUSIONS: This study showed that dose reduction was successful in a subgroup of patients with controlled AD by using a patient-centered dosing regimen. These patients showed stable low disease activity and low severity biomarkers over time.

Why Turner patients with 45, X monosomy should not be excluded from fertility preservation services.

In this case report, we highlight the practical dilemma, i.e. to perform ovarian tissue cryopreservation surgery in a 45, X Turner Syndrome patient or not, by reporting on the presence of follicles in a 13-year-old female diagnosed with 45, X monosomy and an unmeasurable anti-müllerian hormone serum level. We compare our results with previous research, highlight the challenges we faced in this case and provide recommendations for daily practice. Hereby, we demonstrate that excluding certain subgroups of Turner Syndrome patients (e.g. monosomy patients, and/or girls with an anti-müllerian hormone level below 2.0 ng/l) may be premature, especially based on the current state of published research data. This practical example of a challenging dilemma in the counselling of Turner Syndrome patients for fertility preservation is of interest for clinicians involved in fertility counselling and Turner Syndrome care.

Secukinumab is effective and safe in the long-term treatment of plaque psoriasis in a daily practice setting: Multicenter study in 384 Spanish patients.

The aim of the study was to assess the long-term effectiveness and safety of secukinumab in Spanish patients with moderate-to-severe psoriasis in a daily practice setting. Nationwide multicenter, observational, retrospective, non-interventional, single-cohort study including patients who initiated treatment with secukinumab in daily clinical practice conditions. Subjects were followed for a minimum of 3 months and a maximum of 24 months. Psoriasis Area Severity Index (PASI), Body Surface Area and Physician's Global Assessments were collected at baseline and months 3, 6, 12, 18 and 24 during treatment. Adverse events and reasons for secukinumab withdrawal were collected and classified for analyses. A total of 384 patients were enrolled in the study. Median PASI declined rapidly from 14.3 at baseline to 2.7 at month 3, 2.1 at month 12, and remained low (2.8) at month 24. Within the group of patients with PASI ≥10 at baseline (n = 278), 58.3%, 60.4% and 56.5% achieved a PASI90 response at months 3, 12 and 24, respectively. As for absolute PASI, 86.5%, 69.5%, 42.7% and 37% achieved PASI <5, < 3, < 1 and 0, respectively, at month 3. Secukinumab was more effective in biologic-naïve patients and in those with lower Body Mass Index. Secukinumab presented a good long-term safety profile. Secukinumab was effective and safe in a routine clinical setting, in a large cohort of patients with moderate-to-severe plaque psoriasis, in the short-, medium- and long-term (up to 24 months).

Ustekinumab to guselkumab transitions: A series of 54 patients emulating the navigate trial in real life.

NAVIGATE clinical trial demonstrated a higher rate of Psoriasis Assesment Severity Index (PASI)90 response in patients treated with guselkumab when compared to ustekinumab and an improved response in those who switched from ustekinumab to guselkumab due to partial response. The objective of the study is to describe ustekinumab to guselkumab switching in clinical practice. Observational, multicentric, descriptive study including 54 psoriasis patients who switched to guselkumab after treatment with ustekinumab from March 2019 to February 2021. Mean basal PASI with ustekinumab (16.7) was higher than with guselkumab (7.2). Up to 49.01% of patients were able to reach PASI90 with ustekinumab and up to 21.56% had a less frequent dosage regime vs. summary of product characteristics. Main reason to start guselkumab was a loss of ustekinumab cutaneous or articular response (82.36%) but up to 17.64% were switched in order to increase dosage regime efficiency. Six months after starting guselkumab, the absolute PASI was lower than 2 in 72% of patients and 38.5% of them were treated with a reduced dosage regime. Guselkumab doses used by our cohort were 19.5% lower than the expected according to the summary of product characteristics. No adverse events reported. There is no real-world evidence regarding patients who switched from ustekinumab to guselkumab. A short paragraph in the article by Fougerousse et al, reported 63 patients with a mean basal PASI of 5.3 and similar efficacy rate at week 16 to NAVIGATE. Our study adds practical information regarding efficacy, safety and efficiency through dose optimization in a real-world cohort.

Belgian rheumatologists' preferences regarding measures of disease activity in patients with rheumatoid arthritis: results from a mixed-methods study.

The reliability and clinical usefulness of the different composite disease activity scores and their individual components in Rheumatoid Arthritis (RA) are still debated. This study investigated which measures of disease activity were preferred by rheumatologists. A mixed-method study was performed. First, ten Belgian rheumatologists were invited for individual interviews on their current practice and preferences for measurement of RA disease activity. Results of this qualitative study and evidence from literature served as input for developing a survey. This survey asked rheumatologists to rate preferred standard disease activity score(s), their individual components, ultrasound and related patient-reported outcomes (PROs), by maximum difference scaling. The relative importance score (RIS) for each indicator was calculated using hierarchical Bayes modeling. The qualitative study included 6/10 invited rheumatologists. Composite scores and components were perceived as useful, while PROs were found subjective. Interestingly, ultrasound was used to mediate discrepancies between physician and patient. The survey based on this was sent to 244 Belgian rheumatologists, 83/244 (34%) responded, including 66/83 (80%) complete and 17/83 (20%) incomplete surveys (two missing essential information). Most rheumatologists (75/81, 93%) used a disease activity score and 68/81 (84%) preferred the DAS28-CRP. Swollen joint count obtained the highest mean ± SD RIS (22.54 ± 2.64), followed by DAS28 ESR/CRP (20.61 ± 4.06), ultrasound (16.47 ± 7.97), CRP (13.34 ± 6.11) and physician's global assessment (12.59 ± 7.83). PROs including fatigue, pain, and patient's global assessment, and Health Assessment Questionnaire, obtained the lowest mean RIS (0.34-2.54). Rheumatologists place more faith in self-assessed disease activity components or in laboratory tests. Trust in PROs to evaluate disease activity is low in clinical practice.

Dupilumab shows long-term effectiveness in a large cohort of treatment-refractory atopic dermatitis patients in daily practice: 52-Week results from the Dutch BioDay registry.

BACKGROUND: Real-life data on long-term effectiveness and safety of dupilumab in atopic dermatitis patients are limited. OBJECTIVE: To study 52-week effectiveness and safety of dupilumab in a prospective multicenter cohort of adult patients with treatment-refractory atopic dermatitis. METHODS: Patients treated with dupilumab and participating in the Dutch BioDay registry were included. Clinical effectiveness and safety were evaluated. RESULTS: Two hundred ten atopic dermatitis patients were included. Mean percentage change in Eczema Area and Severity Index score after 16 weeks was -70.0% (standard deviation 33.2%) and further decreased to -76.6% (standard deviation 30.6%) by week 52. A greater than or equal to 75% improvement in the score was achieved by 59.9% of individuals by week 16 and by 70.3% by week 52. The most reported adverse effect was conjunctivitis (34%). Limited patients (17; 8.1%) discontinued dupilumab treatment. LIMITATIONS: Because of the lack of a control group and observational design, factors of bias may have been induced. CONCLUSION: Treatment with dupilumab resulted in a rapid improvement in clinical outcome measures, and effectiveness further improved during the 52-week follow-up period.

Evaluation of a One-step Dose Reduction Strategy of Adalimumab, Etanercept and Ustekinumab in Patients with Psoriasis in Daily Practice.

Dose reduction of biologics for psoriasis could contribute to lower drug exposure. This study evaluated a one-step, tightly controlled, biologic dose reduction strategy in a prospective daily practice cohort. In patients with psoriasis with low disease activity using adalimumab, etanercept or ustekinumab for at least 6 months, the dosing interval was prolonged with 33%. Patients could return to their normal dosing interval in case of disease flare. Of 108 eligible patients, 80 started dose reduction and were analysed. In total, 36/80 patients (45.0%) discontinued dose reduction after 19 months (95% confidence interval 14.9-23.1 months). Of 67 patients with 1-year follow-up, 45 (67.2%) still used the lower dose after 1 year. No serious adverse events related to dose reduction occurred. Cumulative dose and costs decreased by 22.7% during 1 year. In conclusion, a one-step tightly controlled dose reduction strategy for adalimumab, etanercept and ustekinumab has considerable potential to safely decrease biologic dosages in patients with psoriasis in daily practice.

RAPP-children: A new tool for assessing quality of life in patients with asthma and rhinitis.

BACKGROUND: RhinAsthma Patient Perspective (RAPP) is a short, validated questionnaire for assessing health-related quality of life (HRQoL) in adult patients with comorbid asthma and rhinitis, while a paediatric version is still not available. OBJECTIVE: The current study aimed to develop and validate the RAPP-children questionnaire. METHODS: RAPP-children was derived by combining RhinAsthma-children subscales into five unique items. At baseline (T0) and after 30 days (T1), 150 children (6-11 years) with comorbid asthma (predominantly intermittent or mild persistent) and rhinitis were given the following: RAPP-children, RhinAsthma-children, Paediatric Asthma Quality of Life Questionnaire (PAQLQ, age >6 years), Paediatric Rhinoconjunctivitis Quality of Life Questionnaire (PRQLQ), Childhood Asthma Control Test (CACT), KiddyKindl® (age 6 years) or KidKindl® (age >6 years), and a Visual Analogue Scale for nasal symptoms (VAS). At the final visit, a Global Rating of Change (GRC) scale was administered. The approved study was registered on the central registration system ClinicalTrials.gov (ID: NCT03276416). RESULTS: RAPP-children fairly reproduced RhinAsthma-children scores (concordance correlation coefficients between 0.91 and 0.95). RAPP-children showed adequate convergent validity (absolute Spearman's rho larger than 0.5 with PAQLQ, PRQLQ, CACT, KiddyKindl/KidKindl, and VAS), internal consistency (Cronbach's alpha > 0.70), repeatability (intra-cluster correlation coefficient between 0.61 and 0.8) in the presence of clinical stability (GRC = 0), discriminant validity (sensitivity to asthma control status and rhinitis severity), and sensitivity to symptom improvements (GRC > 1). The minimal important difference (MID) was -20. Floor and ceiling effects were minimal. RAPP-children showed fair usability also in younger children (6-8 years). CONCLUSION & CLINICAL RELEVANCE: RAPP-children is a valid, five-item questionnaire for assessing HRQoL in children aged 6 to 11 years with concomitant asthma and rhinitis. Although further investigation is required in moderate and severe asthmatics, this tool can be useful in clinical trials and in routine medical practice for improving the management of respiratory allergy in children.

Dupilumab is very effective in a large cohort of difficult-to-treat adult atopic dermatitis patients: First clinical and biomarker results from the BioDay registry.

INTRODUCTION: Dupilumab has recently been approved for the treatment of moderate to severe atopic dermatitis (AD) in adults. Daily practice data on dupilumab treatment are scarce. OBJECTIVE: To study the effect of 16-week treatment with dupilumab on clinical response and serum biomarkers in adult patients with moderate-severe AD in daily practice. METHODS: Data were extracted from the BioDay registry, a prospective multicenter registry. Sixteen-week clinical effectiveness of dupilumab was expressed as number of patients achieving EASI-50 (Eczema Area and Severity Index) or EASI-75, as well as patient-reported outcomes measures (Patient-Oriented Eczema Measure, Dermatology Life Quality Index, Numeric Rating Scale pruritus). Twenty-one biomarkers were measured in patients treated with dupilumab without concomitant use of oral immunosuppressive drugs at five different time points (baseline, 4, 8, 12, and 16 weeks). RESULTS: In total, 138 patients treated with dupilumab in daily practice were included. This cohort consisted of patients with very difficult-to-treat AD, including 84 (61%) patients who failed treatment on ≥2 immunosuppressive drugs. At week 16, the mean percent change in EASI score was 73%. The EASI-50 and EASI-75 were achieved by 114 (86%) and 82 (62%) patients after 16 weeks of treatment. The most reported side effect was conjunctivitis, occurring in 47 (34%) patients. During dupilumab treatment, disease severity-related serum biomarkers (TARC, PARC, periostin, and IL-22), eotaxin-1, and eotaxin-3 significantly decreased. CONCLUSION: Treatment with dupilumab significantly improved disease severity and decreased severity-related serum biomarkers in patients with very difficult-to-treat AD in a daily practice setting.

Practical Guidelines on the prevention and management of diabetic foot disease (IWGDF 2019 update).

Diabetic foot disease results in a major global burden for patients and the health care system. The International Working Group on the Diabetic Foot (IWGDF) has been producing evidence-based guidelines on the prevention and management of diabetic foot disease since 1999. In 2019, all IWGDF Guidelines have been updated based on systematic reviews of the literature and formulation of recommendations by multidisciplinary experts from all over the world. In this document, the IWGDF Practical Guidelines, we describe the basic principles of prevention, classification, and treatment of diabetic foot disease, based on the six IWGDF Guideline chapters. We also describe the organizational levels to successfully prevent and treat diabetic foot disease according to these principles and provide addenda to assist with foot screening. The information in these practical guidelines is aimed at the global community of health care professionals who are involved in the care of persons with diabetes. Many studies around the world support our belief that implementing these prevention and management principles is associated with a decrease in the frequency of diabetes-related lower extremity amputations. We hope that these updated practical guidelines continue to serve as reference document to aid health care providers in reducing the global burden of diabetic foot disease.

Long-term effectiveness and safety of treatment with dupilumab in patients with atopic dermatitis: Results of the TREAT NL (TREatment of ATopic eczema, the Netherlands) registry.

BACKGROUND: Evidence on long-term dupilumab treatment for atopic dermatitis in daily practice is lacking. OBJECTIVE: To investigate patient characteristics, treatment aspects, effectiveness, and safety of up to 84 weeks of dupilumab treatment. METHODS: An observational prospective cohort study was conducted of patients with atopic dermatitis starting dupilumab in routine clinical care. RESULTS: Of the 221 included patients, 103 used systemic therapy at baseline. At 84 weeks, we found a change of -15.2 (SE, 1.7) for the Eczema Area and Severity Index, -16.9 (SE, 1.4) for the Patient-Oriented Eczema Measure, and -17.2 (SE, 1.6) for the Dermatology Life Quality Index. We found a trend for improvement over time for the Investigator Global Assessment and Numerical Rating Scale for pruritus. Severe (n = 79) including serious (n = 11) adverse events were observed in 69 patients. Eye complaints were most frequently reported (n = 46). Twenty-one patients adjusted the regular dosing schedule, and 14 patients discontinued treatment, mainly due to ineffectiveness (n = 7). LIMITATIONS: Only adverse events of severe and serious nature were registered for feasibility reasons. CONCLUSION: Daily practice dupilumab treatment of up to 84 weeks is generally well-tolerated, apart from the reporting of eye complaints. It can be considered a long-term effective treatment for atopic dermatitis in combination with topical and initial concomitant systemic treatment, showing a sustained improvement of signs, symptoms, and quality of life.

Optimization of Diabetic Macular Edema Management in the Real World: A Model of Excellence in Retina Units: The EMUREX Initiative.

PURPOSE: To define and develop a model of excellence for the clinical management of diabetic macular edema (DME) patients in a real-world setting. METHODS: A multidisciplinary joint commission (5 ophthalmologists, 1 nurse, and 1 pharmaceutic) established a series of preliminary recommendations based on clinical guidelines and DME activity results from 8 Pilot Hospitals (PH). These were validated by members of each PH and a group of DME patients in discussion workshops. Thus, the validated guideline (VG) took into consideration different aspects, namely, main core points (ranging 0-100), criteria, and indicators. Finally, each PH own setting was compared to the VG in order to settle down a starting point to clinical excellence. RESULTS: Mean PH score was 51.5 (range 30-65). As compared to their maximum, main points that showed best scores were Clinical Guidelines and Protocols (78%) and Portfolio of Services (73%). Topics reaching close to 50% scoring included Resources (55%), Innovation (54%), Care Process (53%), Organization (52%), and Leadership (50%). Lowest scores were observed in the Strategic Alliances (46%) and Staff (37%) points. CONCLUSIONS: Analysis of each PH by the VG delivered a global vision of the starting situation, especially focused in the identification of the different improvement areas. In order to further extend this model into the Public Health System, the effect of implementing it in different hospitals should be assessed to analyze its impact on daily clinical practice and health economics.

Palliative care in its own discourse: a focused ethnography of professional messaging in palliative care.

BACKGROUND: Despite 50 years of modern palliative care (PC), a misunderstanding of its purpose persists. The original message that PC is focused on total care, helping to live until the person dies, is being replaced and linked to feelings of fear, anxiety and death, instead of compassion, support or appropriate care. Society is still afraid to speak its name, and specialized units are identified as "places of death" as opposed to "places of life" meant to treat suffering. This issue is prohibitive to the implementation and development of PC policies worldwide. It is imperative to identify what message PC professionals are relaying to patients and other health care specialists and how that message may condition understandings of the right to access PC. METHODS: A qualitative study, employing focused ethnography and participant observation (PO) of the daily interaction of PC professionals with patients and family members in three different PC services. Two researchers independently conducted a thematic analysis, followed by member checking with participants. RESULTS: A total of 242 h of participant observation revealed the following messages sent by PC professionals in their daily interaction with patients and families: i) We are focused on your wellbeing; ii) You matter: we want to get to know you; iii) Your family is important to us. CONCLUSION: The complexity of PC discourses contributes to the difficulty of identifying a clear universal message between PC professionals, patients and families. The PC professionals observed transmit a simple message focused on their actions rather than their identity, which may perpetuate some social/cultural misunderstandings of PC. It seems there is a common culture, based on the same values and attitudes, within the messages that PC professionals transmit to patients and their families. PC teams are characterised by their availability.

Assessment of Muscle Function and Physical Performance in Daily Clinical Practice : A position paper endorsed by the European Society for Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases (ESCEO).

It is well recognized that poor muscle function and poor physical performance are strong predictors of clinically relevant adverse events in older people. Given the large number of approaches to measure muscle function and physical performance, clinicians often struggle to choose a tool that is appropriate and validated for the population of older people they deal with. In this paper, an overview of different methods available and applicable in clinical settings is proposed. This paper is based on literature reviews performed by members of the European Society for Clinical and Economic Aspects of Osteoporosis and Osteoarthritis (ESCEO) working group on frailty and sarcopenia. Face-to-face meetings were organized afterwards where the whole group could amend and discuss the recommendations further. Several characteristics should be considered when choosing a tool: (1) purpose of the assessment (intervention, screening, diagnosis); (2) patient characteristics (population, settings, functional ability, etc.); (3) psychometric properties of the tool (test-retest reliability, inter-rater reliability, responsiveness, floor and ceiling effects, etc.); (4) applicability of the tool in clinical settings (overall cost, time required for the examination, level of training, equipment, patient acceptance, etc.); (5) prognostic reliability for relevant clinical outcomes. Based on these criteria and the available evidence, the expert group advises the use of grip strength to measure muscle strength and the use of 4-m gait speed or the Short Physical Performance Battery test to measure physical performance in daily practice. The tools proposed are relevant for the assessment of muscle weakness and physical performance. Subjects with low values should receive additional diagnostic workups to achieve a full diagnosis of the underlying condition responsible (sarcopenia, frailty or other).

Intravenous immunoglobulin for treatment of chronic inflammatory demyelinating polyneuropathy and multifocal motor neuropathy in France: are daily practices in accordance with guidelines?

BACKGROUND AND PURPOSE: Chronic inflammatory demyelinating polyneuropathy (CIDP) and multifocal motor neuropathy (MMN) are rare autoimmune diseases. Guidelines were published in 2010 for their diagnosis and treatment. In France, intravenous immunoglobulins (IVIGs) are mainly used for the first-line treatment. The burden of healthcare costs is often underlined but rarely studied. The aim of this survey was to compare to guidelines, the daily practice of French neurologists with IVIGs for CIDP and MMN treatment. METHODS: This was a retrospective observational study consisting of an online questionnaire performed between March and May 2014. A total of 49 questionnaires were included, a quarter of which were from neurologists working in neuromuscular reference centers (NRCs). RESULTS: A total of 182 patient case reports were studied. Patients were referred to an NRC for initial diagnosis in approximately 30% of cases in CIDP and 50% of cases in MMN. The initial management of IVIG (frequency, dose and duration) was not different between NRCs and non-NRCs. Guidelines were followed and neurologists were relatively at ease in diagnosing and treating patients. CONCLUSIONS: This was the first national study to describe the implementation of the European Federation of Neurological Sciences/Peripheral Nerve Society guidelines in the daily management of IVIGs in patients with MMN and CIDP in France. Efforts are needed to improve long-term tailored treatment and home treatment to reduce economic costs.

A 2-Year Observational Study in Patients Suffering from Idiopathic Pulmonary Fibrosis and Treated with Pirfenidone: A French Ancillary Study of PASSPORT.

BACKGROUND: The European observational, prospective PASSPORT study evaluated the long-term safety of pirfenidone under real-world conditions in idiopathic pulmonary fibrosis (IPF), over up to 2 years following its initiation. OBJECTIVES: The FAS (French Ancillary Study) assessed the clinical outcomes of IPF patients participating in PASSPORT (n = 192). METHODS: Efficacy data were collected retrospectively and prospectively. The primary efficacy endpoints were: change in percent predicted forced vital capacity (FVC) and change in the distance travelled during the 6-min walk test (6MWD). RESULTS: The mean baseline FVC was 71.7% of predicted value. The mean absolute change in the percentage of predicted FVC was -2.4% and -3.8% at months 12 and 24. The mean change in 6MWD was 8.6 and 3.1 m at months 12 and 24, with a range of 23.4-51.7 m. Acute IPF exacerbation and pulmonary hypertension occurred in 20.0 and 8.4% of patients, respectively. The most common reasons for prematurely discontinuing PASSPORT were adverse drug reactions (ADRs) related to pirfenidone (31.3%), death (11.5%), and disease progression (10.9%). The median progression-free survival was 18.4 months (95% CI 12.9, not estimable). The median exposure was 16.3 months (0.5-28.5). The most frequently reported ADRs leading to pirfenidone discontinuation were decreased weight (4.2%), rash (4.2%), and photosensitivity reactions (3.1%). CONCLUSIONS: The efficacy data of FAS are consistent with the efficacy results of published phase III clinical trials in IPF. Approximately one third of IPF patients treated with pirfenidone in real-life settings were still under treatment 2 years after initiation. Safety data are consistent with the known safety profile of pirfenidone.