Telemonitoring for Cystic fibrosis patients of Bambino Gesù Children's Hospital during COVID-19.

Background: Cystic fibrosis (CF) is the most common autosomal recessive genetic pathology of the Caucasian race and it affects nearly 100,000 people worldwide (many have not been diagnosed) and, in Italy, there are about 6000 patients. In the last few years, telemedicine has proved to be an effective home care tool for patients suffering from chronic pathologies. The advent of the COVID-19 pandemic has caused an increase of communications through mobile devices. Aim: To evaluate the role of telemonitoring during the pandemic phase of Covid-19. Materials and methods: 34 (M 15, F 19) (M 44%, F 56%) Cystic Fi-brosis patients were evaluated; Median age ± SD 30.97±10.59 Median FEV1 2020 74.76; number of trasmission and hospital admissions. Results: It was evident that the absolute number of telemedicine visits increased from 1456 to 1605 in the pandemic year (10% more). Conclusions: Telemedicine became an important tool for home management of patients, in particular about chronic diseases. Telemonitoring, an integral part of telemedicine, underlined its effectiveness in all health emergency phase.

Evaluation of nutritional status through bioimpedance analysis in a group of Cystic Fibrosis patients.

Abstract: Cystic fibrosis (CF) is the most common genetic disease in Caucasian people. Nutritional status represents an important key in the progression of the pulmonary disease in CF. People with better nutritional status, generally, maintain good levels of physical activity. Generally Bioelectrical impedance (BIA) analysis is frequently used as a method of body composition assessment, due to easy of use, safety and low cost of this procedure. The aim of this study was to investigate nutritional parameters in cystic fibrosis patient. We performed a single group cohort study. The study examined change in nutritional values in people with CF who practice sport or not, measured by bio-impedance analysis (BIA). Inclusion criteria were people with CF diagnosis confirmed. Primary outcome was evaluate body composition and the correlation with the rate of physical activity. A total of 32 patients were included in the analysis. The most important data was a correlation between Phangle and Body cellular mass index (BCMI) Pvalue<0.01, expecially in patients who had a good levels of aerobic and anaerobic session-training. Patients who did strong physical activity training had a statistically significant values of correlation with nutritional status. Further study were necessary to find association between exercise capa city and body mass index.

The PRECEDE-PROCEED model as a tool in Public Health screening: a systematic review.

OBJETCTIVE: The aim of this systematic review was to summarize the scientific literature concerning the use of the Precede-Proceed model (PPM) applied to educational programs and health screenings contextsV. STUDY DESIGN: Systematic review. METHODS: The search process was based on a selection of publications listed in Medline and Scopus. The keywords used were "Precede-Proceed" AND ("screening" OR "educational programs"). Studies included in the systematic review were subdivided into those applying the model in a screening context, and those applying it within educational programs. RESULTS: Twenty-seven studies were retrieved, mostly performed in the USA and, generally, the promoting center was the University. In the context of cancer screening, the PPM model was most of all applied to Mammography Screening (5 of 13 studies in cancer screening), and Cervical Cancer Screening (5 of 13). Another three studies within the cancer field investigated Menopause-Inducing Cancer Treatments, Oral cancer prevention, and cancer screening in general. In the remaining studies, the model was applied in various screening areas, particularly chronic and degenerative diseases. There were many different study designs, most of which cross-sectional (8), though several RTCs (8) and focus groups (5) were also found. For the cross-sectional studies the methodological quality varied between 3/10 and 9/10, whilst for the RCTs it ranged from 2/5 to 3/5. CONCLUSIONS: The PPM provides an excellent framework for health intervention programs especially in screening contexts, and could improve the understanding of the relationship between variables such as knowledge and screening. Given the complexity of a behavioral change process, certain important predisposing factors could be measured in future studies, and during health intervention planning.

Fifth Åland Island conference on von Willebrand disease.

The fifth Åland Island meeting on von Willebrand disease (VWD) was held on the Åland Islands, Finland, from 22 to 24 September 2016-90 years after the first case of VWD was diagnosed in a patient from the Åland Islands in 1926. This meeting brought together experts in the field of VWD to share knowledge and expertise on current trends and challenges in VWD. Topics included the storage and release of von Willebrand factor (VWF), epidemiology and diagnostics in VWD, treatment of VWD, angiogenesis and VWF inhibitors.

How to not detonate the bomb: the case of the Italian National Health Service.

Why is Italy one of the world's highest ranked for ability and quality of healthcare in relationship to the resources invested? The last decade has been characterized by many Italian Regions with Recovery Plans, whose main focus was on short-term issues with a high impact on healthcare costs. Italy is now leaving this phase and at the regional level there will be an increase of new hirings in the healthcare sectors, as stated by the Ministry of Health. There is a large amount of scientific literature that supports the role of factors such as lifestyles, diet and genetics as the base of population health. The success of the Italian National Health Service (INHS) function is rooted in the ability of a system to adapt to evolving situations, but it is also important to ensure a mechanism of positive feedback correction. In the future, INHS will require a new set of reforms, like the redefinition of structures and mechanisms of governance, the implementation of strategic plans that conjugate better clinical and financial issues. In this context, Health Data Entanglement could be an option to improve the effectiveness of the health governance system in order to develop better quality of care. In Public Health several criticisms could detonate the bomb, and above all the decreasing levels of primary prevention in the fight to obesity (promoting the Mediterranean Diet and physical activity), to smoking and alcohol consumption, as well as to infectious diseases (promoting high vaccination rates). Secondary prevention is also key to this function as a practical experience of re-engineering of the public expense, giving much attention to what works in terms of cost effectiveness, and in particular to cancer screening.

State of the art: von Willebrand disease.

The State of the Art in von Willebrand disease (VWD) has been impacted not only by discoveries in the field of haemostasis, but also by changes in practice in other fields. The development of bleeding assessment tools has led to the clarification of bleeding symptoms and phenotype in VWD. New discoveries in the biology and genetics of von Willebrand factor (VWF) are challenging our existing diagnostics and classification(s). An improved understanding of reproductive physiology and the pathology of VWD along with changing obstetric, gynaecologic and haemostatic therapies necessitate an evolving response to the care of women with VWD. The survival of patients with autoimmune disease, malignancies and congenital heart disease along with increasing use of circulatory support devices and extracorporeal membrane oxygenation is increasing the prevalence of acquired von Willebrand syndrome. In each of these challenges, there are opportunities to improve the care of our patients with VWD.

The cancer screening monitoring system: indicators for organised programmes and possible extension to spontaneous screening.

European Commission recommends the implementation of organized screening programs for cervical cancer based on active invitation of the target population and with a systematic monitoring system and quality assurance. Nevertheless, in many Member States opportunistic screening is still the only or the main way to access Pap test. In Italy, Pap test coverage in women aged 25-64 is close to 80%, about half of them are screened in organized programs and half by opportunistic screening. Organized programs are diffused in the vast majority of the country (78% in 2009) even if in some cases they are not able to actively invite all the target population every three years (actual extension 67%); furthermore, participation rate after invitation is quite low (39%). Organized screening programs showed performance indicators in line with most of the international standards: low referral rates (2.4%), low inadequate cytological results (4.7%), and high positive predictive value for high grade cervical intraepithelial neoplasia (16.2%). Opportunistic screening has no systematic monitoring system. The coexistence of the two screening models, organized programs and opportunistic, can be a source of inappropriate use of secondary prevention duplicating the tests and favoring deviations from recommended protocols. The Italian Ministry of Health recommends re-organizing cervical cancer prevention favoring organized programs or integrating spontaneous Pap testing in an organized system. To implement such integration it is necessary to have monitoring system and quality assurance for all providers and to integrate archives in order to avoid over-testing and deviation from protocols.

Efficacy and safety during formulation switch of a pasteurized VWF/FVIII concentrate: results from an Italian prospective observational study in patients with von Willebrand disease.

Von Willebrand disease (VWD) is an inherited bleeding disorder caused by the quantitative or qualitative deficiency of von Willebrand factor (VWF). Replacement therapy with plasma-derived VWF/factor VIII (FVIII) concentrates is required in patients unresponsive to desmopressin. To assess the efficacy, safety and ease of use of a new, volume-reduced (VR) formulation of VWF/FVIII concentrate Haemate(®) P in patients requiring treatment for bleeding or prophylaxis for recurrent bleeding or for invasive procedures. Pharmacoeconomic variables were also recorded. Data were analysed using descriptive statistics. This was a multicentre, prospective, observational study. Consecutively enrolled patients received Haemate(®) P VR according to their needs, and were followed for 24 months. Of the 121 patients enrolled, 25.6% had type 3 VWD and more than 40% had severe disease. All patients were followed for 2 years, for a total of 521 visits. On-demand treatment was given to 61.9% of patients, secondary long-term prophylaxis to 25.6% and prophylaxis for surgery, dental or invasive procedures to 45.5%. The response to treatment was rated as good to excellent in >93-99% of interventions. The new formulation was well tolerated by all patients with no report of drug-related adverse events. The switch to volume-reduced Haemate(®) P was easy to perform and infusion duration was decreased twofold compared with the previous formulation. Volume-reduced Haemate(®) P was at least as effective and well-tolerated as the previous formulation.

Efficacy and safety of highly purified, doubly virus-inactivated VWF/FVIII concentrates in inherited von Willebrand's disease: results of an Italian cohort study on 120 patients characterized by bleeding severity score.

The efficacy of highly purified VWF/FVIII concentrates with standardized ristocetin cofactor content (VWF:RCo) has been already proven in patients with von Willebrand's disease (VWD). Aim of this retrospective study is to confirm efficacy and safety of two highly purified, doubly virus-inactivated VWF/FVIII concentrates in a large cohort of patients with VWD who were characterized at enrolment by bleeding severity score. Study drugs Alphanate or Fanhdi were given to 120 cases (51 males, 69 females, median age 50 years, range 6-83 years). Patients had VWD3 (10), VWD2A (19), VWD2B (25), VWD2M (10) and DDAVP-unresponsive VWD1 (56) and a median bleeding severity score of 8 (range 0-27). A total of 114 bleeding episodes in 55 cases and 131 surgical procedures in 85 cases could be analysed. Excellent-good clinical responses were seen in 97% of bleeding episodes and in 99% of surgical procedures. To prevent recurrent gastrointestinal (GI) bleeding, cerebral (CNS) haemorrhage, haemarthroses, urogenital or multisite bleeding in more severe patients, secondary prophylaxis was also carried out in 15 cases with VWD3 (3), VWD2A (3), VWD2B (2), VWD1 (7). A median dose of 42 IU VWF:RCo kg(-1) given every other day or twice a week over a median period of 334 days (range 24-799) prevented bleeding completely in 13 cases and reduced its incidence in the remaining two. These results confirm the efficacy and safety of the study concentrates, not only in the management of bleeding and surgery but also in secondary prophylaxis of severe VWD.

[Mass screening and early detection of cancer: organizational characteristics of preventive services in Italy]. / Prevenzione secondaria dei tumori in Italia: caratteristiche degli interventi di popolazione e individuali.

The commitment to fight against cancer is a part of European Community health strategies and of Italian health services planning. Interesting and useful results have been achieved in Europe and in our country where most of the population can participate to screening programmes. In Italy there are two main organisational models to deliver intervention for secondary prevention: population based and individual access. Several experimental data show population-based prevention is more fair and appropriate and achieves better results in reducing incidence and mortality rates; data are summarized. Because of this differences we go trough the different characteristics of the two models to handle them better planning and managing more efficiently a sustainable secondary-prevention.

Treatment of classic Kaposi sarcoma with a nicotine dermal patch: a phase II clinical trial.

BACKGROUND: Kaposi sarcoma (KS), a malignancy of dermal endothelial cells that is caused by human herpesvirus 8 (HHV8) infection, is sensitive to perturbations of immunity. Nicotine might be effective against KS because of its immunologic and vascular effects and because smoking is associated with a low risk of KS. OBJECTIVE AND STUDY DESIGN: We conducted a masked, randomized phase 2 clinical trial of transdermal nicotine and placebo patches to assess the safety and efficacy of nicotine against classic KS (cKS). SUBJECTS AND METHODS: Three cKS lesions, predominantly nodules, in each of 24 non-smoking patients were randomly assigned to 15 weeks continuous treatment with nicotine patch (escalated to 7 mg), identical masked placebo patch or no patch. Changes in lesion area and elevation from baseline through six follow-up visits, by direct measurement and by two independent readers using digital photographs of the lesions, were compared using non-parametric and regression methods. Changes in longitudinal levels of HHV8 antibodies and DNA in blood cells were similarly assessed. RESULTS: There were no systemic or serious adverse events, and compliance was good. One patient resumed smoking and discontinued patches, and two patients withdrew at week 12 for unrelated indications. Six (29%) of the remaining 21 suspended use of patches to relieve local skin irritation; four of these six completed the trial at reduced dose. Treatment assignment was not associated with significant or consistent changes in cKS lesion area or elevation, HHV8 viral load or antibodies. CONCLUSION: Transdermal nicotine and placebo patches caused no serious toxicities but had no demonstrable effect on nodular cKS lesions or HHV8 levels.

The use of desmopressin in von Willebrand disease: the experience of the first 30 years (1977-2007).

The aim of the treatment for von Willebrand disease (VWD) is to correct the dual defect of haemostasis, i.e. the abnormal platelet adhesion as a result of reduced and/or dysfunctional von Willebrand factor (VWF) and the abnormal coagulation expressed by low levels of factor VIII (FVIII). Correction of both deficiencies can be achieved by administering the synthetic peptide desmopressin (DDAVP) or, in cases unresponsive to this agent, the plasma concentrates containing VWF and FVIII (VWF/FVIII). DDAVP is the treatment of choice for type 1 VWD because it can induce release of normal VWF from cellular compartments, but the drug can be clinically useful also in other VWD types, including acquired von Willebrand syndrome (AVWS). A test dose of DDAVP at the time of diagnosis is recommended to establish the individual patterns of biological response and to predict clinical efficacy during bleeding and surgery. DDAVP is not effective in VWD type 3 and in severe forms of VWD 1 and 2. It can induce transient thrombocytopenia in patients with VWD type 2B. The results of several retrospective studies on the use of DDAVP in VWD management have been reported by many authors in different countries for the last 30 years. However, despite the widespread use of DDAVP in the treatment of VWD, there are only a few prospective clinical trials in a large number of cases on DDAVP efficacy and safety aimed at determining benefits and limits of this therapeutic approach. An investigator-driven observational prospective study on clinical efficacy of DDAVP in 200 patients with VWD types 1 and 2 has been recently organized: the effectiveness and safety of DDAVP will be evaluated prospectively for 24 months during bleeding episodes and minor or major surgeries in the VWD patients who were exposed to an infusion trial at enrollment.

[Colorectal cancer screening: survey of endoscopy center's physicians in Lazio region]. / Screening per il cancro del colon retto: indagine sui medici endoscopisti della regione Lazio.

The aim of the present work is to describe the characteristics of digestive endoscopy centers and the physicians that work there, with particular attention to their attitudes and practices in colorectal cancer screening. A questionnaire was sent to all 80 digestive endoscopy centers in the Lazio region, identified by the annual census of Italian Society of Digestive Endoscopy (Società Italiana di Endoscopia Digestiva, SIED). Seventy-one centers (89%), returned the questionnaire. Screening activity on average represents 14% of the centers' colonoscopy workload. Colonoscopy was considered to be a "very effective" screening test by 96% of physicians, the faecal occult blood test "very effective" by 20%, and flexosigmoidoscopy "very effective" by 11%. Ninety-seven percent (97%) of physicians reported recommending any test for screening: 80% colonoscopy, 61% faecal occult blood test, 14% double contrast barium enema and 11% flexosigmoidoscopy. Despite the fact that almost all physicians reported recommending screening, the centres are only marginally involved in screening practice. Endoscopy centers' physicians tend to have an aggressive strategy for colorectal cancer prevention and exclusive trust in colonoscopy; an attitude more consistent with a clinical-diagnostic approach than with real mass screening of a healthy population.

Von Willebrand's disease: clinical management.

The aim of treatment of von Willebrand's disease (VWD) is to correct the dual defect of haemostasis, i.e. the abnormal platelet adhesion due to reduced and/or dysfunctional von Willebrand factor (VWF) and the abnormal coagulation expressed by low levels of factor VIII (FVIII). Desmopressin (DDAVP) is the treatment of choice for type 1 VWD because it can induce release of normal VWF from cellular compartments. Prospective studies on biological response versus clinical efficacy of DDAVP in VWD type 1 and 2 are in progress to further explore its benefits and limits as therapeutic option. In type 3 and in severe forms of type 1 and 2 VWD, DDAVP is not effective and for these patients plasma virally inactivated concentrates containing VWF and FVIII are the mainstay of treatment. Several intermediate- and high-purity VWF/FVIII concentrates are available and have been shown to be effective in clinical practice (bleeding and surgery). New VWF products almost devoid of FVIII are now under evaluation in clinical practice. Although thrombotic events are rare in VWD patients receiving repeated infusions of concentrates, there is some concern that sustained high FVIII levels may increase risk of postoperative venous thromboembolism. Dosage and timing of VWF/FVIII administrations should be planned to keep FVIII level between 50 and 150 U/dL. Appropriate dosage and timing in repeated infusions are also very important in patients exposed to secondary long term prophylaxis for recurrent bleedings.

A quantitative analysis of bleeding symptoms in type 1 von Willebrand disease: results from a multicenter European study (MCMDM-1 VWD).

BACKGROUND: A quantitative description of bleeding symptoms in type 1 von Willebrand disease (VWD) has never been reported. OBJECTIVES: The aim was to quantitatively evaluate the severity of bleeding symptoms in type 1 VWD and its correlation with clinical and laboratory features. PATIENTS AND METHODS: Bleeding symptoms were retrospectively recorded in a European cohort of VWD type 1 families, and for each subject a quantitative bleeding score (BS) was obtained together with phenotypic tests. RESULTS: A total of 712 subjects belonging to 144 families and 195 controls were available for analysis. The BS was higher in index cases than in affected family members (BS 9 vs. 5, P < 0.0001) and in unaffected family members than in controls (BS 0 vs. -1, P < 0.0001). There was no effect of ABO blood group. BS showed a strong significant inverse relation with either von Willebrand ristocetin cofactor (VWF:RCo), von Willebrand antigen (VWF:Ag) or factor VIII procoagulant activity (FVIII:C) measured at time of enrollment, even after adjustment for age, sex and blood group (P < 0.001 for all the four upper quintiles of BS vs. the first quintile, for either VWF:RCo, VWF:Ag or FVIII:C). Higher BS was related with increasing likelihood of VWD, and a mucocutaneous BS (computed from spontaneous, mucocutaneous symptoms) was strongly associated with bleeding after surgery or tooth extraction. CONCLUSIONS: Quantitative analysis of bleeding symptoms is potentially useful for a more accurate diagnosis of type 1 VWD and to develop guidelines for its optimal treatment.

[Risk management in a regional screening program for breast cancer in the region of Lazio, Italy]. / Garantire la sicurezza dei pazienti in un intero profilo assistenziale: il programma di gestione del rischio clinico nello screening mammografico della Regione Lazio.

In the Lazio Region, it has been put into effect a plan of clinical Risk Management for the Breast Cancer Screening Regional Program (BCSP), involving all of the 12 Local Health Units and the Public Health Agency of Lazio (ASP). Being the BCSP a health care service, it consists of a perfect integration of health care structures, professionals and skills working for the citizens. This program originates from an unexpressed health need and leads to a evidence-based health benefit. The BCSP provides free breast screening for 700,000 women aged between 50 and 69 in the Lazio region; the Public Health Agency carries out the clinical governance of the BCSP The prevention of errors and incidents represents a fundamental basis of governance: it is a contribution to the achievement of efficacy in breast cancer screening. The BCSP deals with screening incidents from a systemic point of view and actively involves several Local Health Units staff going through each step of the whole patient's clinical path, from the identification of the target population, to the oncological treatment of positive cases. The programme is an integration of different tools: literature research, process analysis using the HFMEA methodology and reporting system. The results reached so far are the following: the regional severity rating scale, the regional Master-List of possible adverse events, occurrence and detection rating scale.

[Evidence-based planning: the case of mass screening for colorectal cancer in the Lazio region of Italy]. / Programmazione basata sulle prove di efficacia: il caso dello screening del cancro colorettale nel Lazio.

Several population-based trials have shown the efficacy of colorectal cancer mass-screening based on guaiac faecal blood testing in a generic risk population. SCCR represents a very complex and resource-demanding public health intervention and, for this reason, its planning requires actual efficacy as a main goal. Since evidence of efficacy demonstrated by population-based trials may not actually generate effectiveness, the Regional Government of Latium Region decided to implement some experimental studies before introducing a screening programme, in order to define an evidence-based organisational model of SCCR and a feasibility evaluation of the real needs for screening. The aims of the pilot studies were to define an evidence-based organisational model, to evaluate the necessary resources and the actual quality standard of clinical examination, treatment and surgery. The aim of the feasibility study is to test the organisational model for SCCR for about 300,000 citizens residing in the Latium region. The present article illustrates the scheduling path set out, which is based on the involvement of experts, GP representatives and specialists from scientific societies and it is planned by the following actions: Definition of evidence-based recommendations; identification of further investigations; realization of experimental studies; definition of an evidence-based organisational model. The main research areas have been dealt with using randomised trials, in order to evaluate the efficacy of the involvement of GPs and the kind of test for RSOF Our work has produced evidences which were sometimes in contrast with information in the literature, demonstrating that guaiac RSOF testing is less reproducible and determines lower uptake than immunochemical testing. Our work also shows that the involvement of GPs should be based on their personal skills rather than on their role. Such evidences are fundamental to the definition of the organisational model and confirm the need of basing an evidence-based planning not only on evidences found in the literature. The necessity of this approach is strengthened by the level of organisational complexity and by the amount of resources needed to put a public health intervention into practice.