A phase 1/2 safety and efficacy study of TAK-754 gene therapy: The challenge of achieving durable factor VIII expression in haemophilia A clinical trials.

Introduction: Haemophilia A is an X-linked bleeding disorder resulting from a deficiency of factor VIII (FVIII). To date, multiple gene therapies have entered clinical trials with the goal of providing durable haemostatic protection from a single dose. TAK 754 (BAX 888) is an investigational AAV8-based gene therapy containing a FVIII transgene.

The role of body composition in neurological and hematologic toxicity in a retrospective analysis of 120 breast cancer patients undergoing neoadjuvant chemotherapy: the COMBOTOX study.

Purpose: Neoadjuvant chemotherapy (NAC) has a well-established role in locally advanced or chemoresponsive breast cancers (BC). Chemotherapic regimens are effective when patients receive the optimal doses. Toxicities are common in overweight/obese patients but may occur also in normal weight counterparts.

Adequacy of Pain Management in Patients Referred for Radiation Therapy: A Subanalysis of the Multicenter ARISE-1 Study.

Background: Pain is a prevalent symptom among cancer patients, and its management is crucial for improving their quality of life. However, pain management in cancer patients referred to radiotherapy (RT) departments is often inadequate, and limited research has been conducted on this specific population. This study aimed to assess the adequacy and effectiveness of pain management when patients are referred for RT.

Immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients with severe hemophilia A: interim results from a phase 3, prospective, multicenter, open-label study.

Aim: To determine the immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients (PUPs) with severe hemophilia A (HA).

Methods: This prospective, phase 3 study (NCT02615691) was conducted in PUPs, or patients with ≤2 exposure days (EDs) prior to screening, aged <6 years with severe HA. The primary endpoint was incidence of factor VIII (FVIII) inhibitor development.

Endocrine Disruptors in Food, Estrobolome and Breast Cancer.

The microbiota is now recognized as one of the major players in human health and diseases, including cancer. Regarding breast cancer (BC), a clear link between microbiota and oncogenesis still needs to be confirmed. Yet, part of the bacterial gene mass inside the gut, constituting the so called "estrobolome", influences sexual hormonal balance and, since the increased exposure to estrogens is associated with an increased risk, may impact on the onset, progression, and treatment of hormonal dependent cancers (which account for more than 70% of all BCs).

[Improvement of the cognitive-behavioral pathway by setting up a multidisciplinary city-hospital staff].

In order to improve the cognitive-behavioral pathway, and based on a well-established history of city-hospital collaboration, a monthly staff meeting bringing together hospital and out-of-hospital professionals and a users' association makes it possible to analyze and respond in a coordinated and graduated manner to complex situations. After nine months of operation, the story of three complex situations illustrates the benefits and limits of such a system.

Identification of a minimum number of genes to predict triple-negative breast cancer subgroups from gene expression profiles.

Background: Triple-negative breast cancer (TNBC) is a very heterogeneous disease. Several gene expression and mutation profiling approaches were used to classify it, and all converged to the identification of distinct molecular subtypes, with some overlapping across different approaches. However, a standardised tool to routinely classify TNBC in the clinics and guide personalised treatment is lacking.

Screening for Physical Activity Levels in Non-Metastatic Breast Cancer Patients Undergoing Surgery: An Observational Study.

Background: Physical activity (PA) can play a role in lowering the risk of breast cancer (BC), but also in reducing perioperative complications and treatments related side effects, improving the quality of life and decreasing mortality in BC survivors. PA and nutritional screening are not offered to patients after cancer diagnosis as standard of care, even in high quality breast units.

Methods: From February 2019 to March 2020, we performed a preoperative physical and nutritional screening in 504 consecutive BC patients waiting for surgery.

Sexuality in breast cancer survivors: sexual experiences, emotions, and cognitions in a group of women under hormonal therapy.

Background: Earlier diagnosis and improved treatments have led to better outcomes in breast cancer, making quality of life a key issue. Sexuality represents a pillar of quality of life, although it is often neglected by both healthcare providers and patients when it comes to cancer. This study aims to explore the differences in sexual functioning, distress, psychopathology, emotions, and cognitions between breast cancer patients under hormonal treatment and controls.

A Model of an Integrative Approach to Breast Cancer Patients.

Background: Breast cancer (BC) survivors have physical and psychological needs that require convincing responses by health care providers. The quality of life issue and clinical unmet needs are among the main reasons pushing a number of patients toward "natural" therapies that are often misleading and alternative to mainstream cancer care. Integrative Oncology (IO) tries to respond to many of those needs, by combining lifestyle counseling, body-mind activities, and complementary evidence-informed therapies with anticancer standard treatments.

Neoadjuvant Chemotherapy in Breast Cancer: An Advanced Personalized Multidisciplinary Prehabilitation Model (APMP-M) to Optimize Outcomes.

Neoadjuvant chemotherapy is increasingly being employed in the management of breast cancer patients. Efforts and resources have been devoted over the years to the search for an optimal strategy that can improve outcomes in the neoadjuvant setting. Today, a multidisciplinary approach with the application of evidence-based medicine is considered the gold standard for the improvement of oncological results and patient satisfaction.

Recombinant ADAMTS-13: first-in-human pharmacokinetics and safety in congenital thrombotic thrombocytopenic purpura.

Safety, tolerability, and pharmacokinetics of recombinant ADAMTS-13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13; BAX 930; SHP655) were investigated in 15 patients diagnosed with severe congenital ADAMTS-13 deficiency (plasma ADAMTS-13 activity <6%) in a prospective phase 1, first-in-human, multicenter dose escalation study. BAX 930 was well tolerated, no serious adverse events occurred, and no anti-ADAMTS-13 antibodies were observed. After single-dose administration of BAX 930 at 5, 20, or 40 U/kg body weight to adolescents and adults, there was approximate dose proportionality with respect to maximum plasma concentration (C [U/mL]) and area under the concentration-time curve (AUC [h∙U/mL]).

The relative biological effectiveness of antiprotons.

Background And Purpose: Aside from the enhancement of physical dose deposited by antiprotons annihilating in tissue-like material compared to protons of the same range a further increase of biological effective dose has been demonstrated. This enhancement can be expressed in an increase of the relative biological effectiveness (RBE) of antiprotons near the end of range. We have performed the first-ever direct measurement of the RBE of antiprotons both at rest and in flight.

Ceftaroline fosamil for the treatment of community-acquired bacterial pneumonia in the intensive care unit.

The Clinical Assessment Program and Teflaro(®) Utilization Registry (CAPTURE) is a multicenter study evaluating the clinical use of ceftaroline fosamil in patients with community-acquired bacterial pneumonia (CABP) or acute bacterial skin and skin structure infection. Data were collected between August 2011 and February 2013, from 398 evaluable patients receiving treatment at 33 sites in the USA. This manuscript presents data collected from patients with CABP who received care in an intensive care unit (ICU) or in general medical wards (35% and 64% of evaluable patients, respectively).

Ceftaroline Fosamil for the Treatment of Bacteremia Secondary to Acute Bacterial Skin and Skin Structure Infections or Community-Acquired Bacterial Pneumonia.

Background: The Clinical Assessment Program and Teflaro® Utilization Registry is designed to collect information on the clinical use of ceftaroline fosamil in the Unites States. This report presents data on the treatment of patients with bacteremia (SAB) secondary to acute bacterial skin and skin structure infections (ABSSSIs) or community-acquired bacterial pneumonia (CABP).

Methods: Patients diagnosed with ABSSSI or CABP were identified through sequential review of randomly ordered charts generated from pharmacy listings from August 2011 to February 2013.

Ceftaroline fosamil for treating skin and skin structure infections or community-acquired pneumonia in patients with renal insufficiency.

The Clinical Assessment Program and Teflaro(®) Utilization Registry (CAPTURE) is a multicenter retrospective study, conducted in the USA, describing the contemporary use of ceftaroline fosamil. Ceftaroline is primarily excreted by the kidneys and the dose should be reduced in patients with moderate to severe renal insufficiency. This article describes the clinical effectiveness of ceftaroline fosamil in the treatment of acute bacterial skin and skin structure infection (ABSSSI) or community-acquired bacterial pneumonia (CABP) patients with renal insufficiency.

Pulmonary embolism due to popliteal vein aneurysm: approach and surgical treatment - a case report.

Pulmonary embolism is the first presentation in 70-80% in patients with aneurysm of popliteal vein. We present a case of young man admitted in our hospital with respiratory distress, tachycardia and D-dimer >3000 mg/dL. The observation of pulmonary embolism required the placement of a caval temporary filter before the surgical treatment.

Double pathological fracture of mandibula caused by actinomycotic osteomyelitis: a case report.

Actinomycosis is an uncommon chronic granulomatous infection that cause formation of abscesses and cutaneous fistula. In mandibular actinomycosis the alveolar bone and mandibular body are usually not involved and the pathogenetic mechanisms of the actinomycotic infiltration is unknown. The patients usually report pain at the alveolar arch with development of a purplish-red swelling firmly attached to the mandibula; the fibrous tissue produces the continued development of new cutaneous fistulas with oncoming pus-secretion.

Viral pathophysiology of multiple sclerosis: A role for Epstein-Barr virus infection?

Multiple sclerosis (MS) is the most common inflammatory demyelinating and degenerative disease of the CNS. The cause of MS is unknown but environmental risk factors are implicated in MS. Several viruses have been proposed as a trigger for MS, and lately Epstein-Barr virus (EBV) has become the leading candidate.

The low level laser therapy in the management of neurological burning mouth syndrome. A pilot study.

Background And Objective: Burning Mouth Syndrome (BMS) is a common disease but still a diagnostic and therapeutic challenge for clinicians. Despite many studies its nature remains obscure and controversial; nowadays there is no consensus about definition, diagnosis and classification. BMS is characterized clinically by burning sensations in the tongue or other oral sites, often without clinical and laboratory findings.

Neuromyelitis optica-IgG (aquaporin-4) autoantibodies in immune mediated optic neuritis.

The clinical course of immune mediated optic neuritis (ON) will depend on the specific underlying inflammatory disease. These disorders have traditionally been classified according to clinical and MRI findings. Aquaporin-4 (AQP4) autoantibodies (neuromyelitis optica-IgG (NMO-IgG)) may have diagnostic and prognostic value in patients who present with isolated ON.

Bone damage induced by different cutting instruments--an in vitro study.

The aim of this study was to compare the peripheral bone damage induced by different cutting systems. Four devices were tested: Er:YAG laser (2.94 mm), Piezosurgery, high-speed drill and low-speed drill.

Doppler ultrasonography and exercise testing in diagnosing a popliteal artery adventitial cyst.

We describe popliteal arterial adventitial cystic disease which causes intermittent claudication in a young athletic man, with atypical manifestation, without loss of foot pulses on knee flexion nor murmur in the popliteal fossa. The findings obtained from Magnetic Resonance Imaging were non-diagnostic. The diagnosis resulted from Echo-Doppler ultrasonography along with peak exercise testing.