Transthyretin amyloid polyneuropathy in France: A cross-sectional study with 413 patients and real-world tafamidis meglumine use (2009-2019).

OBJECTIVE: We aimed to describe characteristics of patients with ATTR variant polyneuropathy (ATTRv-PN) and ATTRv-mixed and assess the real-world use and safety profile of tafamidis meglumine 20mg. METHODS: Thirty-eight French hospitals were invited. Patient files were reviewed to identify clinical manifestations, diagnostic methods, and treatment compliance. RESULTS: Four hundred and thirteen patients (296 ATTRv-PN, 117 ATTRv-mixed) were analyzed. Patients were predominantly male (68.0%) with a mean age of 57.2±17.2 years. Interval between first symptom(s) and diagnosis was 3.4±4.3 years. First symptoms included sensory complaints (85.9%), dysautonomia (38.5%), motor deficits (26.4%), carpal tunnel syndrome (31.5%), shortness of breath (13.3%), and unexplained weight loss (16.0%). Mini-invasive accessory salivary gland or punch skin and nerve biopsies were most common, with a performance of 78.8-100%. TTR genetic sequencing, performed in all patients, revealed 31 TTR variants. Tafamidis meglumine was initiated in 156/214 (72.9%) ATTRv-PN patients at an early disease stage. Median treatment duration was 6.00 years in ATTRv-PN and 3.42 years in ATTRv-mixed patients. Tafamidis was well tolerated, with 20 adverse events likely related to study drug among the 336 patients. CONCLUSION: In France, ATTRv patients are usually identified early thanks to the national network and the help of diagnosis combining genetic testing and mini-invasive biopsies.

Surface-guided radiotherapy overview: Technical aspects and clinical applications.

In radiotherapy, patient positioning has long been ensured by ionizing imaging (kV or MV). Over the past ten years, surface-guided radiotherapy has appeared in radiotherapy departments. It is a continuous three-dimensional acquisition of the surface of the patient, based on the use of several optical cameras. The acquired surface is compared to an expected surface (usually taken from the planning scanner). Operators can constantly appreciate poor position, anatomical deformity or patient shift. Thus, the system allows an aid to the positioning of the patient, possibly without tattooing, but also a follow-up of the patient during the duration of the session. The most obvious contribution of the system concerns the treatment of the breast. In fact, for this location, the bone registration is not ideal and the target is visible in surface-guided radiotherapy. These systems also make it possible to treat in deep inspiration breath hold. But several other locations can benefit from it (pelvis, thorax, etc.).

[Source blocking in HDR brachytherapy: Practical situation and experience feedback at the Eugène Marquis-Rennes]. / Blocage de source en curiethérapie de haut débit de dose : mise en situation pratique et retour d'expérience au centre Eugène Marquis- Rennes.

At the Eugene Marquis Center, high dose rate brachytherapy is part of the care offering. The risk analysis and the national experience feedback linked to the use of high activity sources show that blocking the source outside its storage position, during treatment, would be the main risk of exposure of ionizing radiation. In a process of radiation protection of patients and workers, and to limit the consequences of such an accident, the Eugene Marquis Center has set up periodic training with practical experience for all brachytherapy professionals. This article describes the experience feedback from this training by brachytherapy technicians.

[Lupus nephritis associated with common variable immunodeficiency: favourable outcome with intravenous immunoglobulin treatment]. / Néphropathie lupique liée à un déficit immunitaire commun variable d'évolution favorable sous immunoglobulines intraveineuses.

We report a 24-year-old woman who presented with a nephrotic syndrome as the revealing manifestation of systemic lupus erythematosus (SLE) and an associated hypogammaglobulinemia related to a common variable immunodeficiency (CVID). Outcome of SLE was favourable with intravenous immunoglobulin treatment solely. Relationships between SLE and CVID are discussed.

[Inappropriate prescription of heparin at curative doses in the hospital. Can the information to prescribing physicians decrease misuse?]. / Prescriptions inappropriées d'héparine à dose curative en milieu hospitalier, l'information des prescripteurs diminue-t-elle les erreurs?

OBJECTIVES: To control whether prescriptions of curative doses of heparin (non fractioned heparins, enoxaparin, tinzaparin) in the hospital complied with the official recommendations; to provide the physicians with information adapted to the recorded misuse and to evaluate the influence of this information. METHODS: A prospective study was conducted between May and October 1999 on the prescriptions of 20 residents from 6 services in 3 phases: phase P1 with initial evaluation (particularly on the indications for heparin, the molecule administered, initial dose and monitoring), phase P2 with analysis and diffusion of an adapted information and phase P3 with final evaluation. RESULTS: 111 inpatients were included in the phase P1 (66.7% aged over 75 years, 18.9% with creatinin clearance below 30 ml/mn) and 101 inpatients were included in the phase P3 (56.4% aged over 75 years, 10.8% with renal failure). During phase P1: among the prescriptions of low molecular weight heparin (LMWH) 54.3% did not comply with the official recommendations; initial doses were too high in 15.3% of patients; mean initial doses of LMWH were not adapted to age, weight or creatinin clearance. Only 58.5% of patients had their platelets monitored. On the other hand, 15.3% of patients exhibited heparin side effects. During phase P3, the main modifications in prescriptions were a reduction in inappropriate indications for LMWH, reduction in LMWH prescriptions in patients aged over 75 or with excessive body weight or with renal failure, and increased platelet monitoring, but without significant difference. On the other hand, mean initial doses of each heparin were not modified. Heparin complications decreased but not significantly. CONCLUSION: This study highlights a real context of heparin prescription at curative doses, often differing from clinical studies, particularly with regards to age, renal failure and comorbidity; prescriptions often unadapted to official recommendations on indications, dose and monitoring; a real but limited influence of appropriate information for the physicians, which partially depends on the accuracy of official recommendations, particularly in patients with increased hemorrhagic risk.

Cardiac malignant lymphoma successfully treated with chemotherapy.

We report the case of an 83-year-old woman with diffuse large B-cell lymphoma located in the right cardiac ventricle revealed by acute congestive heart failure. Complete and long-lasting disappearance of the cardiac tumor was observed with monochemotherapy (cyclophosphamide).

Surface Complexation of Calcite by Carboxylates in Water.

Small molecules that have two carboxylic functions can adsorb from water onto calcite. The adsorption site is a -Ca+ site. The mechanism of adsorption is a complexation of the -Ca+ site by the two carboxylates, similar to the solution complexation of Ca++ ions. The complex has a ring structure where the two carboxylates are joined on one side by the -Ca+ ion and on the other by the n CH2 groups of the small molecule. Five-bond rings (n = 0) are the most stable, followed by six-bond rings (n = 1) and seven-bond rings (n = 2). Five-bond rings can also be formed with one carboxylate and one hydroxyl group (this is the case for alpha-hydroxycarboxylates) or with one enolate and one hydroxyl group (catechol). The sequence of binding strengths is enolate > carboxylate > hydroxyl; it matches the sequence of complexation efficiencies of these groups in solution and their characters as electron donors toward the metal cation. Copyright 1999 Academic Press.