Orphan drugs

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The Birth of Endowiki, An Italian Online Platform For Continuous Medical Education in Endocrinology

Background Conventional tools for medical education are burdened by many drawbacks. Textbooks become rapidly outdated, meeting attendance is expensive, and results reported in journals are not easily suitable for clinical practice. Uptodate and Endotext are excellent online tools, but they have been developed for a clinical context far different from that of our country. Methodology Italian Association of Clinical Endocrinologists (AME) charged a task force aiming to develop an educational tool specifically tailored for Italian clinical endocrinologists. Required characteristics were clinical approach, modularity, continuous updating, full online availability (even by mobile devices), open sections and sections for registered members only, opportunity for individualization, indexing and search engine to facilitate browsing. Starting from an open-source platform, Joomla, several changes were implemented. Results Three editors, 2 central editorial secretaries, 30 section coordinators and over 350 authors have been involved in the writing of 21 different sections covering all fields of endocrine and metabolic diseases. The access to Endowiki is free for AME members and is fully open in sections for patients. All readers are requested to serve as referee, pointing to mistakes and need for revision. The system is attended daily by a mean of 250 individuals. Conclusions Endowiki stands alone as an opportunity for medical education in Italy. The big challenges will be the continuous updating and the link to the national certified system for CME

lanreotide 60 mg a new long acting formulation effectiveness in the chronic treatment of acromegaly

Lanreotide (LAN) 60 mg (LAN60), a new long-acting formulation of LAN alleged to suppress GH/IGF-I hypersecretion for 28 d in acromegalic patients, was administered in a prospective open multicenter study to 92 patients with active acromegaly (61 women and 31 men, aged 20–79 yr). LAN60 was given as adjuvant treatment (AT) in 62 patients; the other 30 patients [primary treatment (PT)] were de novo (n = 20) or previously treated only by pharmacotherapy (n = 10). After wash-out from previous treatments, LAN60 was started im every 28 d for 3 injections; the dose was then individually tailored, aiming at lowering GH to less than 2.5 μg/liter and IGF-I to the normal range. After a median follow-up of 24 months (range, 6–48 months), IGF-I normalized in 65% of patients, decreasing from 199 ± 8% (expressed as a percentage of the upper limit of normal range; mean ± se) to 87 ± 4% (P < 0.0001). GH fell to less than 2.5 μg/liter in 63% of patients and to less than 1 μg/liter in 25%, decreasing from 20 ± 3 to 3 ± 0.4 μ..

Does the addition of a second daily session of hyperbaric oxygen therapy to intratympanic steroid influence the outcomes of sudden hearing loss? [L’aggiunta di una seconda sessione giornaliera di camera iperbarica al trattamento steroideo intratimpanico influenza i risultati terapeutici nella sordità improvvisa?]

The aim of this study is to investigate whether, in addition to intratympanic steroid therapy, additional hyperbaric oxygen therapy (HBOT) sessions per day (twice a day for 5 days) is more useful than one session per day for 10 days in patients affected by severe and profound idiopathic sudden sensorineural hearing loss (ISSNHL). A total of 55 patients affected by unilateral severe and profound ISSNHL were recruited. Two protocols were adopted. In the first, 27 patients (13 with profound and 14 with severe hearing loss) underwent one session of HBOT per day for 10 days, 6 days a week. An HBOT session comprised a period of 14 minutes air compression followed by 90 min at 2.4 atm absolute (ATA) followed by a decompression period of 15 min in oxygen. Patients breathed 100% oxygen through an appropriate mask checked for leaks. Patients were given 0.4 ml of 62.5 mg/ml of intratympanic prednisolone during the first three days of the protocol. In the second protocol, 28 patients (10 with profound and 18 with severe hearing loss) received 10 sessions of HBOT, twice a day for five days, 2.4 ATA 90 min 100% oxygen. The intratympanic injections of prednisolone were given between the two sessions of HBOT during the first three days of the protocol. Since there were no significant differences in hearing outcomes between the two protocols, the present study shows that the protocol of two sessions of HBOT per day is a valid treatment and equally effective as the one HBOT session per day, but with shorter treatment time

Use of thyroid hormones in hypothyroid and euthyroid patients: a THESIS* questionnaire survey of Polish physicians. *THESIS: Treatment of hypothyroidism in Europe by specialists: an international survey

Introduction: Over the past several years new evidence on the management of hypothyroidism has emerged, which has influenced recommendations from professional bodies. The presentation of hypothyroid patients has also changed, and new cases are increasingly diagnosed by indiscriminate screening, often identifying cases with minor biochemical disturbances. Little is known about the physician responses and attitudes to this changing landscape. THESIS (Treatment of Hypothyroidism in Europe by Specialists: an International Survey) is a large-scale survey of European physicians who treat patients with hypothyroidism. Here we document current practices of Polish physicians relating to the use of thyroid hormones in hypothyroid and euthyroid patients.  Material and methods: Members of the Polish Society of Endocrinology were invited to participate in the web-based THESIS survey. Results: In total 423 (54.6% of the 774 invited) physicians completed the survey. The majority of respondents (74.2%) would prescribe thyroid hormones for euthyroid patients for certain indications, such as female infertility with elevated thyroid antibodies (63.4%), simple goitre (40.9%), unexplained fatigue (12.1%), obesity (9.7%), hypercholesterolaemia (9.0%), and depression (9.2%). Nearly all physicians (96.0%) declared that the treatment of choice for hypothyroidism is levothyroxine (LT4). However, around one-third (30.3%) were also using LT4 and liothyronine (LT3) combination treatment; LT3 alone was rarely prescribed (1.7%), and none prescribed desiccated thyroid extract. The majority of respondents preferred LT4 tablets. Among alternative formulations, liquid LT4 was most commonly recommended for patients unable to take LT4 in the fasting state (26.0%) and patients with malabsorption (19.9%). Respondents considered prescribing dietary supplements (such as selenium and iodine) in hypothyroid patients with coexisting autoimmune thyroiditis (29.6%) or at the patients’ request (32.2%). LT4 + LT3 combination therapy was used by 32.2% when symptoms persisted notwithstanding normal serum TSH concentration. Psychosocial factors, comorbidities, and the burden of chronic disease were considered as the most likely causes of persistent symptoms. Conclusions: Apart from clinical practice recommendations, other factors influence the thyroid hormone therapy patterns. Moreover, certain areas of clinical practice were identified (the use of thyroid hormones in euthyroid subjects and the use of dietary supplements), which are not in accordance with the current evidence.

Right ventricular failure in left heart disease: from pathophysiology to clinical manifestations and prognosis

Right heart failure (RHF) is a clinical syndrome in which symptoms and signs are caused by dysfunction and/or overload of the right heart structures, predominantly the right ventricle (RV), resulting in systemic venous hypertension, peripheral oedema and finally, the impaired ability of the right heart to provide tissue perfusion. Pathogenesis of RHF includes the incompetence of the right heart to maintain systemic venous pressure sufficiently low to guarantee an optimal venous return and to preserve renal function. Virtually, all myocardial diseases involving the left heart may be responsible for RHF. This may result from coronary artery disease, hypertension, valvular heart disease, cardiomyopathies and myocarditis. The most prominent clinical signs of RHF comprise swelling of the neck veins with an elevation of jugular venous pressure and ankle oedema. As the situation worsens, fluid accumulation becomes generalised with extensive oedema of the legs, congestive hepatomegaly and eventually ascites. Diagnosis of RHF requires the presence of signs of elevated right atrial and venous pressures, including dilation of neck veins, with at least one of the following criteria: (1) compromised RV function; (2) pulmonary hypertension; (3) peripheral oedema and congestive hepatomegaly. Early recognition of RHF and identifying the underlying aetiology as well as triggering factors are crucial to treating patients and possibly reversing the clinical manifestations effectively and improving prognosis

An Unusual Case of Primary Extranodal Lymphoma of the Gallbladder.

Primary gallbladder lymphoma is an extremely rare disease. We report a case of a 63 year-old woman who has been admitted with gradual onset abdominal pain in the upper right quadrant and in the suprapubic region, nausea and malaise. According to the computed tomography scan of the abdomen, which was suggestive of chronic cholecystitis, she was treated conservatively. A laparoscopic cholecystectomy was performed 5 months later and the histological examination of the gallbladder showed a low grade small lymphocytic lymphoma. The patient has been taken over by the hematology team who kept her under surveillance as no further treatment was deemed as necessary. The purpose of this paper is to report a rare case of primary gallbladder lymphoma and to demonstrate that a laparoscopic cholecystectomy may be a valid treatment for this disease.This article is available via Open Access. Click on the Additional Link above to access the full-text via the publisher's site

State-of-the-Art Review on Diabetes Care in Italy.

Background: Diabetes is a significant health problem in Italy as in other western countries. Objective: To review available epidemiological data and the legislative framework for diabetes care in Italy. Methods: Review of Italian Health Ministry's official documents and analysis of epidemiological data published by Italian Scientific Societies. Findings: Diabetes affects more than 5% of the Italian population. The expenditures for the care of people with diabetes are about €10 billion ($US 11 billion) a year and are increasing over time. Italian law regulates the clinical care of people with diabetes and creates a clinical framework involving medical organizations, prevention programs, personnel training, and legal protection. The National Health Program is structured in essential levels of assistance that can be defined differently in the various regions. In 2013, the "National Diabetes Plan," defining priority areas for intervention, was approved and represents the main regulatory tool for the management of diabetes within the Italian National Health Service. In Italy, the status of diabetes care is being monitored using the data from 2 permanent observatories: the ARNO Observatory Diabetes and the Associazione Medici Diabetologi Annals. Conclusions: A comprehensive approach to diabetes is offered to all citizens, consonant with the constitutionally guaranteed right to health. However, this important effort translates into a relevant financial burden for the National Health Service