Are pharmacological treatments for familial amyloid polyneuropathy effective and safe? A Cochrane Review summary with commentary

BACKGROUND: Familial amyloid polyneuropathies (FAPs) are a group of rare autosomal dominant transmitted disorders that can progressively lead to disability from neuropathy, autonomic failure and other system involvement. OBJECTIVE: The aim of this commentary is to discuss Cochrane evidence on the efficacy and safety of disease-modifying drugs (DMDs) for the treatment of FAPs from a rehabilitation perspective. METHODS: To summarize and discuss from a rehabilitation perspective the published Cochrane Review 'Pharmacological treatment for familial amyloid polyneuropathy' by Magrinelli et al. RESULTS: This Cochrane review included 4 randomized controlled trials (RCTs) involving 655 adults with FAP. These four trials compared four different DMDs with placebo. The Cochrane Systematic Review reported that current evidence is limited. CONCLUSIONS: FAPs are a group of chronic disabling conditions in which a multidisciplinary approach, including an adequate rehabilitation programme along with a long-term effective pharmacological therapy, should always be envisaged

I-21 Current therapeutic guidelines in Duchenne Muscular Dystrophy to prolong life

Duchenne's myopathy is an X-linked disease with well defined evolutionary phases, characterized by degradation of the walking function, development of evolutive scoliosis and progressive decline of the respiratory function leading patients to premature death. In 1985 Y. Rideau in France carried out a new global therapeutic strategy for treatment of lower limb deformities, scoliosis deformity and progressive restrictive syndrome. The indication for surgery at the lower limbs is made very early, at the onset of the first signs of disease. The procedures are carried out at the same time and always bilaterally; they include: (i) hip section of superficial flexors; (ii) iliotibial band resection; (iii) subcutaneous tenotomy of semitendineous and gracilis; (iv) subcutaneous lengthening of Achilles tendons. In the post-operative period, the patient begins exercises of active and passive mobility in few days and after three weeks recovers his performances; ambulation will remain almost normal for several years. A comparison of two groups of patients, the first precociously operated on the lower limbs, the other one not operated, shows better performances in the operated group. The indications for surgical treatment of Duchenne scoliosis must be made after the loss of ambulation and not too late, to avoid the concurrent respiratory restrictive syndrome makes the patient inoperable. Over ten years ago, in Poitiers, a specific instrumentation for Duchenne scoliosis was created, providing for cylindrical rods fixed by peduncular screws at the sacro-lumbar level. On the dorso-lumbar level, the rod becomes flat to allow more flexibility of the trunk. The complications observed in a group of 55 patients operated for scoliosis, consisted in 2 cases of breaking of rods and 1 superficial infection. The surgery approach in DMD has the double aim to prolong the time of the autonomous ambulation and to avoid the evolution of scoliosis, limiting the harmful effects of the scoliosis on the respiratory function. However, the surgery alone is unable to prolong the life expectancy in these patients, without treating the restrictive respiratory syndrome, first by nasal ventilation and then by elective tracheotomy, essential for the survival of the patient

Karim Hammou, Une histoire du rap en France

Dans la nuit du 29 au 30 décembre 2012, France 2 diffuse Urban music show, une émission célébrant les 30 ans du rap et des musiques dites urbaines en France. C’est à un voyage dans ces trente années (1981-2010) que nous convie le sociologue Karim Hammou. L’ouvrage repose sur un travail de thèse réécrit auquel l’auteur a adjoint de nouvelles parties, composant un ensemble riche. Il est solidement ancré dans un travail empirique et mobilise le cadre théorique développé par Howard Becker (qui s..

Efficacy and Effectiveness of Extracorporeal Shockwave Therapy in Patients with Myofascial Pain or Fibromyalgia: A Scoping Review

Myofascial pain syndrome (MPS) and fibromyalgia (FM) are underestimated painful musculoskeletal conditions that could impact function and quality of life. A consensus about the most appropriate therapeutic approach is still not reached. Considering the long course of the diseases, prolonged assumption of drugs, such as NSAIDs and pain killers, could increase the risk of adverse events, often leading affected patients and physicians to prefer non-pharmacological approaches. Among these, radial and focused extracorporeal shock waves therapies (ESWT) are widely used in the management of painful musculoskeletal conditions, despite the fact that the mechanisms of action in the context of pain modulation should be further clarified. We performed a scoping review on PubMed using Mesh terms for analyzing the current evidence about the efficacy and effectiveness of ESWT for patients with MPS or FM. We included 19 clinical studies (randomized controlled trials and observational studies); 12 used radial ESWT, and 7 used focused ESWT for MPS. Qualitative analysis suggests a beneficial role of ESWT for improving clinical and functional outcomes in people with MPS, whereas no evidence was found for FM. Considering this research gap, we finally suggested a therapeutic protocol for this latter condition according to the most recent diagnostic criteria

The Rationale for the Intra-Articular Administration of Clodronate in Osteoarthritis

Background: Several pharmacological therapeutic approaches have been proposed to manage osteoarthritis (OA), including intra-articular (IA) injections. Although the discovery of clodronate, a bisphosphonate, dates back to the 1960s and the effects of its IA administration have been investigated for decades in animal models, mechanisms of action of this drug are not quite clear, particularly in OA. This scoping review is an overview of the biological as well as the clinical role of clodronic acid in OA. Method: A scoping review based on the PRISMA-ScR (Preferred Reporting Items for Systematic Reviews and Meta-Analyses Extension for Scoping Reviews) model was performed to characterize the mechanisms of action of IA clodronate in OA and to evaluate its efficacy from a clinical point of view. Results: Several effects of clodronate have been observed in animal models of OA, including depletion of synovial lining cells that results in reduced production of chemokines (IL-1, TNF- α), growth factors (TGF-β, BMP 2/4), and metalloproteases (MMP 2/3/9); prevention of cartilage damage, synovial hyperplasia, and proteoglycans loss; reduction in joint inflammation, joint swelling, and osteophyte formation. From a clinical perspective, patients with knee OA treated with IA clodronate experienced improvements in pain and joint mobility. Conclusion: Clodronate appears to have different mechanisms of action interfering with the pathogenic processes contributing to OA development and progression. This intervention demonstrated positive effects for patients affected by knee OA

Post-acute covid-19 rehabilitation network proposal: From intensive to extensive and home-based it supported services

Management of COVID-19 post-acute syndrome is an emerging health issue in rehabilitation. This article aims to present a proposal, based on the principles of clinical governance, health management and information technology (IT), and to respond to the need for a structured organization model for post-acute COVID-19 rehabilitation. The authors present a regional-based model of a network of clinicians and healthcare managers using a dedicated IT platform to achieve both effectiveness and efficiency objectives, to ensure coordination of the available resources and the most appropriate rehabilitative treatment for patients. The proposed post-acute COVID-19 rehabilitation network has been designed according to the model of a clinical management project within the Italian national healthcare system, and its context is an easily adjustable model for the European healthcare systems. The authors base the project on current laws and scientific guidelines in rehabilitation in Italy and in Europe and use the SWOT analysis technique to assess the proposal feasibility. The primary aims of the project are: (1) standardizing the minimum assessment tools of post-COVID-19 patients with disabilities; (2) ensuring an individual rehabilitation project for each patient with international classification of functioning, disability and health (ICF) coding and (3) reporting the activity performance with appropriate indicators. The secondary aims are: (1) developing educational programs for patients and care givers also aimed at acquiring better empowerment and positive behavior; (2) creating a regional database for data collection and (3) improving IT, and specifically tele-rehabilitation, as a suitable approach during the COVID-19 emergency and also in the future. Expected results are: continuum of care; effectiveness, efficacy and appropriateness in the delivery of rehabilitation treatments through a standardized minimum assessment and the wording of the individual rehabilitation project and a precise reporting of performance indicators to measure the effectiveness of clinical activities and the satisfaction of patients and caregivers. The assessment of results will be analyzed at three and six months to implement corrective actions according to the concept of continuous improvement of the Deming cycle. The IT remote approach allows the patient to meet the needs of proximity of care and empowerment, and, at the same time, to contain the spread of infection. This project could have a significant healthcare impact ensuring a more efficient and effective management of the demand of rehabilitation post-acute COVID-19, expanding the professional skills of the rehabilitation team members, improving both clinical and process data, in addition to optimal allocation of available economic resources

Development of site-specific biomechanical indices for estimating injury risk in cycling

In this paper we present novel biomechanical indices for site-specific assessment of injury risk in cycling. The indices are built from a multifactorial analysis based on the kinematics and kinetics of the cyclist from the biomechanical side, and muscle excitations and muscle synergies from the neurophysiological side. The indices are specifics for three body regions (back, knee, ankle) which are strongly affected by overuse injuries in cycling. We use these indices for injury risks analysis of a recreational cyclist, who offered to participate in the experiments. The preliminary results are promising towards the use of such indices for planning and/or evaluating training schedule with the final goal of reducing non-traumatic injuries in cycling

Efficacy and Effectiveness of Physical Agent Modalities in Complex Regional Pain Syndrome Type I: A Scoping Review

Complex regional pain syndrome type I (CRPS-I) is a rare condition with huge variability in triggering factors and clinical scenarios. The complexity of the pathophysiology of this condition fosters the proposal of several therapeutic options with different mechanisms of action in both research and clinical practice. An interdisciplinary and multimodal approach, including pharmacological and non-pharmacological interventions, particularly physical therapy, is recommended by international guidelines, but the benefits and harms of available interventions are poorly known. In this scoping review, the clinical rationale for use of physical agent modalities for patients with CRPS-I will be presented. We found 10 studies addressing the role of electromagnetic field therapy, electrotherapy, and laser therapy. Our findings suggest that physical therapy modalities, in particular transcutaneous electrical nerve stimulation (TENS) and pulsed electromagnetic field therapy (PEMF), may contribute to reduce pain and improve function in patients with CRPS-1

Physical Agent Modalities in Early Osteoarthritis: A Scoping Review

Early osteoarthritis (EOA) still represents a challenge for clinicians. Although there is no consensus on its definition and diagnosis, a prompt therapeutic intervention in the early stages can have a significant impact on function and quality of life. Exercise remains a core treatment for EOA; however, several physical modalities are commonly used in this population. The purpose of this paper is to investigate the role of physical agents in the treatment of EOA. A technical expert panel (TEP) of 8 medical specialists with expertise in physical agent modalities and musculoskeletal conditions performed the review following the PRISMA-ScR (Preferred Reporting Items for Systematic Reviews and Meta-Analyses Extension for Scoping Reviews) model. The TEP searched for evidence of the following physical modalities in the management of EOA: “Electric Stimulation Therapy”, “Pulsed Electromagnetic field”, “Low-Level Light Therapy”, “Laser Therapy”, “Magnetic Field Therapy”, “Extracorporeal Shockwave Therapy”, “Hyperthermia, Induced”, “Cryotherapy”, “Vibration therapy”, “Whole Body Vibration”, “Physical Therapy Modalities”. We found preclinical and clinical data on transcutaneous electrical nerve stimulation (TENS), extracorporeal shockwave therapy (ESWT), low-intensity pulsed ultrasound (LIPUS), pulsed electromagnetic fields stimulation (PEMF), and whole-body vibration (WBV) for the treatment of knee EOA. We found two clinical studies about TENS and PEMF and six preclinical studies—three about ESWT, one about WBV, one about PEMF, and one about LIPUS. The preclinical studies demonstrated several biological effects on EOA of physical modalities, suggesting potential disease-modifying effects. However, this role should be better investigated in further clinical studies, considering the limited data on the use of these interventions for EOA patients