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2.  A certification/accreditation model for Haemophilia Centres in Italy 
Blood Transfusion  2014;12(Suppl 3):s505-s509.
The Italian Association of Haemophilia Centres has developed a voluntary programme of professional accreditation of Haemophilia Centres, run by its members. Participation in the programme, which aims to foster staff involvement in clinical governance, includes both medical personnel and nurses.
Materials and methods
Accreditation is awarded provided the candidate Haemophilia Centre is able to adhere to a pre-established set of quality standards and meet a number of clinical and organisational requirements, previously defined on the basis of evidence-based medicine. Self-evaluation is the first step in the programme, followed by a site visit by a team of peer professionals experienced in quality auditing.
The programme has so far involved 21 Italian Haemophilia Centres. The comparison between self- and peer-evaluation revealed less discrepancies for disease-related than for organisational requirements, the latter being met to a lesser degree by most Haemophilia Centres.
This programme of professional accreditation developed by the Italian Association of Haemophilia Centres has the potential to describe, monitor and improve clinical and organisational performances in the management of patients with haemophilia and allied inherited coagulation disorders. It should also be seen as a contribution to the implementation of the strategy for improving professional governance in Haemophilia Centres.
PMCID: PMC4044808  PMID: 24922289
professional accreditation; quality of care; standards
3.  Prophylactic efficacy and pharmacokinetically guided dosing of a von Willebrand factor/factor VIII concentrate in adults and children with von Willebrand's disease undergoing elective surgery: a pooled and comparative analysis of data from USA and European Union clinical trials 
Blood Transfusion  2013;11(4):533-540.
Surgical intervention may pose significant risk of life-threatening bleeding in patients with von Willebrand's disease; prophylactic treatment with von Willebrand factor/factor VIII concentrate is generally indicated for von Willebrand's disease characterized by moderate to severe qualitative and quantitative deficiencies of Willebrand factor to raise and maintain both Willebrand factor and FVIII at haemostatic levels for surgical prophylaxis.
Materials and methods
Since prospective clinical data in such situations were lacking, two recent, prospective, multicentre studies evaluated the prophylactic perioperative use of the on Willebrand factor/ factor VIII concentrates, Humate-P® and Haemate P. Despite some differences in the two studies, one conducted in the USA (n =35) and one in the European Union (n =27), the designs were similar enough to allow for a limited pooled analysis of data. In both studies, preoperative loading doses and subsequent maintenance doses were calculated using individual subject-derived incremental in vivo recovery values, although von Willebrand factor:ristocetin cofactor and FVIII:coagulation activity target levels differed between the protocols. Efficacy was rated daily by the investigator as excellent, good, moderate, or poor.
Overall haemostatic efficacy (rating of excellent/good), assessed 24 hours after the last infusion (USA) or taken as the worst rating between surgery and day 14 (EU), was achieved in 95% of the pooled population of 62 adults and children. Efficacy did not appear to be affected by dosing variations. The rate of possibly related adverse events was low (8 subjects; 13%); one of these events was considered serious (pulmonary embolism).
This pooled analysis of a relatively large number of patients for a rare disease confirms the feasibility of pharmacokinetically guided dosing of von Willebrand factor/factor VIII concentrate and highlights its efficacy and safety in the prevention of excessive perioperative bleeding.
PMCID: PMC3827397  PMID: 23736911
von Willebrand's disease; factor VIII; prophylaxis; elective surgery
4.  Recombinant factor VIIa as haemostatic therapy in advanced liver disease 
Blood Transfusion  2013;11(4):487-490.
PMCID: PMC3827390  PMID: 23114524
recombinant factor VIIa; tranexamic acid; liver cirrhosis; liver transplantation; hepatectomy
5.  Present and future challanges in the treatment of haemophilia: a clinician’s perspective 
Blood Transfusion  2013;11(Suppl 4):s77-s81.
PMCID: PMC3853988  PMID: 24333317
haemophilia; factor VIII; factor IX; inhibitors; recombinant technology
6.  Liver disease, coagulopathies and transfusion therapy 
Blood Transfusion  2013;11(1):32-36.
PMCID: PMC3557474  PMID: 23058863
liver disease; coagulopathy; transfusion therapy
7.  Hypercoagulability in congenital haemolytic anaemias 
Blood Transfusion  2012;10(4):423-427.
PMCID: PMC3496237  PMID: 22044960
thalassaemia; sickle cell anaemia; thrombosis; therapy
8.  Prophylaxis for adults with haemophilia: towards a personalised approach? 
Blood Transfusion  2012;10(2):123-124.
PMCID: PMC3320771  PMID: 22337279
9.  Genome-Wide Association Studies in Myocardial Infarction and Coronary Artery Disease 
Myocardial infarction (MI) and its major determinant, coronary artery disease (CAD), are complex diseases arising from the interaction between several genetic and environmental factors. Until recently, the genetic basis of these diseases was poorly understood. Genome-wide genetic association studies have afforded a comprehensive insight into the association between genetic variants and diseases. To date, seven genome-wide association studies have been conducted in CAD/MI, identifying thirteen genomic regions at which common genetic variants influence the predisposition to these diseases. This review article summarizes the progress achieved in the genetic basis of MI and CAD by means of genome-wide association studies and the potential clinical applications of these findings.
PMCID: PMC3466835  PMID: 23074578
Genome-wide association study; Myocardial infarction; Coronary artery disease
10.  Evidence-based recommendations on the treatment of von Willebrand disease in Italy 
Blood Transfusion  2009;7(2):117-126.
von Willebrand disease (VWD) is the most common hereditary bleeding disorder affecting both males and females. It arises from quantitative or qualitative defects of von Willebrand factor (VWF) and causes bleeding of mucous membranes and soft tissues. The aim of treatment is to correct the dual defect of haemostasis caused by the abnormal/reduced VWF and the concomitant deficiency of factor VIII (FVIII).
Material and methods
This document contains evidence-based recommendations for the management of VWD compiled by AICE (the Italian Association of Haemophilia Centres). All the evidence supporting these recommendations are based on non-randomised comparative studies or case series, because randomised controlled clinical trials or meta-analyses are not available for this disease.
Results and conclusions
Desmopressin (DDAVP) is the treatment of choice for patients with type 1 VWD with FVIII and VWF levels of 10 U/dL or more, while VWF/FVIII concentrates are indicated for those who are unresponsive or insufficiently responsive to DDAVP (severe type 1, type 2 and 3 VWD). VWF concentrates devoid of FVIII, not yet licensed in Italy, may be considered for short-term prophylaxis in elective surgery or for long-term secondary prophylaxis.
PMCID: PMC2689066  PMID: 19503633
von Willebrand disease; desmopressin; von Willebrand factor
11.  Prophylaxis in congenital coagulation disorders: past, present and future 
Blood Transfusion  2008;6(Suppl 2):s1-s3.
PMCID: PMC2652216  PMID: 19105502
12.  Advantages and limits of ADAMTS13 testing in thrombotic thrombocytopenic purpura 
Blood Transfusion  2008;6(3):127-135.
PMCID: PMC2626866  PMID: 18705236
ADAMTS13; thrombotic thrombocytopenic purpura; TTP
13.  Autoimmune haemophilia 
Blood Transfusion  2008;6(1):6-7.
PMCID: PMC2626853  PMID: 18661917
15.  The European Haemophilia Network (EUHANET) 
Blood Transfusion  2014;12(Suppl 3):s515-s518.
PMCID: PMC4044796  PMID: 24922291
haemophilia; EUHANET; reference networks; European; certification
16.  The European standards of Haemophilia Centres 
Blood Transfusion  2014;12(Suppl 3):s525-s530.
The European haemophilia community of professionals and patients has agreed on the principles of haemophilia care to address comprehensive optimal delivery of care which is nowadays scattered throughout Europe. Many of the health facilities call themselves Haemophilia Centres despite their variation in size, expertise and services provided. Only a small number of countries have Haemophilia Centre accreditation systems in place.
In the framework of the European Haemophilia Network project, following an inclusive process of stakeholder involvement, the European Guidelines for the certification of haemophilia centres have been developed in order to set quality standards for European Haemophilia Centres and criteria for their certification.
The Guidelines define the standards and criteria for the designation of two levels of care delivery: European Haemophilia Treatment Centres, providing local routine care, and European Haemophilia Comprehensive Care Centres, providing specialised and multi-disciplinary care and functioning as tertiary referral centres. Additionally, they define standards about general requirements, patient care, provision of an advisory service and establishment of network of clinical and specialised services.
The implementation of the European Guidelines for the certification of Haemophilia Centres will contribute to the reduction of health inequalities through the standardisation of quality of care in European Union Member States and could represent a model to be taken into consideration for other rare disease groups.
PMCID: PMC4044800  PMID: 24922293
standards and criteria; certification system; haemophilia care; EUHANET
17.  The methodology for defining the European Standards for the certification of Haemophilia Centres in Europe 
Blood Transfusion  2014;12(Suppl 3):s519-s524.
Work Package 4 Development of the standardisation criteria of the European Haemophilia Network project has the main objective of implementing a common and shared European strategy for a certification system for two levels of Haemophilia Centres: European Haemophilia Treatment Centres and European Haemophilia Comprehensive Care Centres in the Member States of the European Union.
Materials and methods
An inclusive and participatory process for developing shared standards and criteria for the management of patients with inherited bleeding disorders has been carried out. The process has been implemented through four different consultation events involving the entire European community of stakeholders that significantly contributed in the drafting of the European Guidelines for the certification of Haemophilia Centres.
The Guidelines set the standards for the designation of centres that provide specialised and multidisciplinary care (Haemophilia Comprehensive Care Centres) as well as local routine care (Haemophilia Treatment Centres). Standards cover several issues such as: general requirements; patient care; advisory services; laboratory; networking of clinical and specialised services.
The drafting of the European Guidelines for the certification of Haemophilia Centres was performed adopting a rigorous methodological approach. In order to build the widest possible consensus to the quality standards, the main institutional and scientific stakeholders have been involved. The resulting document will significantly contribute in promoting standardisation in the quality of diagnosis and treatment in European Haemophilia Centres.
PMCID: PMC4044802  PMID: 24922292
standards; criteria; certification system; haemophilia care; EUHANET
18.  Fc-fusion technology and recombinant FVIII and FIX in the management of the hemophilias 
Prophylaxis with regular infusions of factor VIII (FVIII)- or factor IX (FIX)- containing products is the mainstay of modern hemophilia care. However, this therapeutic regimen is inconvenient, requiring repeated intravenous injections from childhood. Approaches meant to prolong the half-life of FVIII and FIX in plasma have been developed in order to improve the feasibility and acceptability of replacement therapy, extending protection from bleeding, reducing infusion frequency and hence the need for venous access devices in young children. Several strategies have been implemented to enhance the pharmacokinetics of clotting factors, including conjugation with polyethylene glycol and the production by genetic engineering of fusion proteins containing the coagulation factors linked to a long-lived plasma protein such as albumin or the Fc fragment of immunoglobulin (Ig)G. The latter technology is one of the most promising, since the prolongation of FVIII and FIX half-life is obtained by exploiting the physiological binding of the Fc domain to the neonatal Fc receptor. Fc fusion monomers have been obtained with both recombinant FVIII (rFVIIIFc) and FIX (rFIXFc), and data from preclinical and clinical studies showed improved pharmacokinetics for both factors, which are produced in human embryonic kidney (HEK) 293 cells, thus ensuring full human post-translational modifications. In Phase I/IIa studies, rFVIIIFc and rFIXFc showed 1.5–1.7 fold and 3.0–4.0 fold longer elimination half-life, respectively. Similar data have been obtained in the Phase III clinical studies with rFVIIIFc and rFIX-Fc published recently. Both drugs were satisfactorily safe, particularly with respect to immunogenicity, and no serious adverse event was observed.
PMCID: PMC3974692  PMID: 24729686
factor VIII; factor IX; long-acting molecules
19.  Plasma-derived medicinal products: demand and clinical use 
Blood Transfusion  2013;11(Suppl 4):s2-s5.
PMCID: PMC3853983  PMID: 24333309
20.  The demand for factor VIII and for factor IX and the toll fractionation product surplus management 
Blood Transfusion  2013;11(Suppl 4):s64-s76.
PMCID: PMC3853991  PMID: 24333316
plasma-derived medicinal products; factor VIII; factor IX; demand; surplus management
21.  Past, present and future of hemophilia: a narrative review 
Over the past forty years the availability of coagulation factor replacement therapy has greatly contributed to the improved care of people with hemophilia. Following the blood-borne viral infections in the late 1970s and early 1980, caused by coagulation factor concentrates manufactured using non-virally inactivated pooled plasma, the need for safer treatment became crucial to the hemophilia community. The introduction of virus inactivated plasma-derived coagulation factors and then of recombinant products has revolutionized the care of these people. These therapeutic weapons have improved their quality of life and that of their families and permitted home treatment, i.e., factor replacement therapy at regular intervals in order to prevent both bleeding and the resultant joint damage (i.e. primary prophylaxis). Accordingly, a near normal lifestyle and life-expectancy have been achieved. The main current problem in hemophilia is the onset of alloantibodies inactivating the infused coagulation factor, even though immune tolerance regimens based on long-term daily injections of large dosages of coagulation factors are able to eradicate inhibitors in approximately two-thirds of affected patients. In addition availability of products that bypass the intrinsic coagulation defects have dramatically improved the management of this complication. The major challenges of current treatment regimens, such the short half life of hemophilia therapeutics with need for frequent intravenous injections, encourage the current efforts to produce coagulation factors with more prolonged bioavailability. Finally, intensive research is devoted to gene transfer therapy, the only way to ultimately obtain cure in hemophilia.
PMCID: PMC3502605  PMID: 22551339
FVIII; FIX; Plasma-derived factor concentrates; Recombinant factor concentrates; Gene therapy
22.  The management of hemophilia in elderly patients 
Clinical Interventions in Aging  2007;2(3):361-368.
After the increasing rate of deaths observed during the 1980s due to human immunodeficiency virus (HIV) infection, the health-related quality of life and life expectancy of persons with hemophilia have improved, mainly due to the progresses of replacement therapy and antiviral drugs and to the improvement of the global comprehensive care provided by specialized centers. As a consequence, an increasing number of hemophiliacs have reached an older age and nowadays physicians in hemophilia centers find that they must handle age-related clinical problems never previously observed in this population. The management of elderly persons with congenital hemophilia is discussed in the first part of this review. The second part describes the general aspects of acquired hemophilia due to anti-factor VIII autoantibodies, focusing on the clinical management of elderly patients, one of the groups most frequently affected by this acquired bleeding disorder.
PMCID: PMC2685275  PMID: 18044186
hemophilia; factor VIII; elderly; aging; inhibitors; bleeding
23.  Air Pollution, Smoking, and Plasma Homocysteine 
Environmental Health Perspectives  2006;115(2):176-181.
Mild hyperhomocysteinemia is independently associated with an increased risk of cardiovascular disease. Air pollution exposure induces short-term inflammatory changes that may determine hyperhomocysteinemia, particularly in the presence of a preexisting proinflammatory status such as that found in cigarette smokers.
We examined the relation of air pollution levels with fasting and postmethionine-load total homocysteine (tHcy) in 1,213 normal subjects from Lombardia, Italy.
We obtained hourly concentrations of particulate matter < 10 μm in aerodynamic diameter (PM10) and gaseous pollutants (carbon monoxide, nitrogen dioxide, sulfur dioxide, ozone) from 53 monitoring sites covering the study area. We applied generalized additive models to compute standardized regression coefficients controlled for age, sex, body mass index, smoking, alcohol, hormone use, temperature, day of the year, and long-term trends.
The estimated difference in tHcy associated with an interquartile increase in average PM10 concentrations in the 24 hr before the study was nonsignificant [0.4%; 95% confidence interval (CI), −2.4 to 3.3 for fasting; and 1.1%, 95% CI, −1.5 to 3.7 for postmethionine-load tHcy]. In smokers, 24-hr PM10 levels were associated with 6.3% (95% CI, 1.3 to 11.6; p < 0.05) and 4.9% (95% CI, 0.5 to 9.6; p < 0.05) increases in fasting and postmethionine-load tHcy, respectively, but no association was seen in nonsmokers (p-interaction = 0.005 for fasting and 0.039 for postmethionine-load tHcy). Average 24-hr O3 concentrations were associated with significant differences in fasting tHcy (6.7%; 95% CI, 0.9 to 12.8; p < 0.05), but no consistent associations were found when postmethionine-load tHcy and/or 7-day average O3 concentrations were considered.
Air particles may interact with cigarette smoking and increase plasma homocysteine in healthy subjects.
PMCID: PMC1831519  PMID: 17384761
air pollution; cardiovascular risk; generalized additive models; homocysteine; particulate matter; smoking

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