July was landmarked by a key meeting in which TIF participated representing the Cyprus Alliance for Rare Diseases (CARD), of which TIF is a founding member. This meeting was held at the TIF headquarters in Nicosia, Cyprus on the 10th July 2014 with the aim to discuss future action on the direction of rare diseases in Cyprus. It was attended by representatives of individual Patient Organisations for Rare Diseases across Cyprus.

Among the topics discussed were the promotion of joint projects involving common policies on rare diseases, the detailed lobbying of all national, regional, and international governmental and non- governmental Rare Disease Patient Organisations, Bodies and Agencies, the initiation of a collaboration with rare disease specialists, the compilation of guidelines indexing social and medical services in Cyprus relating to chronic diseases, and specifically to rare diseases, and the establishment, and implementation of EU and national programs.

The discussion was fruitful, giving new life and impetus for the materialisation of a joint action plan on rare diseases, which also include haemoglobinopathies, as they are amongst the family of rare diseases in Europe and globally through the EC Communication on Action in the field of Rare Diseases, 2009/C 151/02.

TIF video sharing platform for patients!
Video sharing platform
Thank you Ken, from Dallas, Texas, for sharing your story with us and your thoughts on living with Thalassaemia!! To watch this motivational video click on the link provided below! Share your stories, your concerns, and your success! The deadline, 5 September, is slowly approaching so hurry!  Click here to view the video

Good Luck! To submit a video, please click here

Shere Project

Screening and Prevention Programmes for Haemoglobinopathies Assessment (SPHerA)
We are excited to inform you of the proposal entitled “Screening and Prevention Programmes for Haemoglobinopathies Assessment (SPHerA)”.
This project has made it to Phase II of the EU’s Horizon 2020 Funding Scheme, under the Research and Innovation Actions. The consortium for this project is comprised with partners from 15 different countries in Europe and abroad and TIF is participating as a Work Package Leader.

SPHerA Project Objectives
The main objective of the proposed project is to assess the effectiveness of the existing screening and prevention programmes for haemoglobinopathies in all European countries. More specifically, this project aims to: • Assess and compare existing screening and prevention programmes to produce a full Health Technology Assessment (HTA) report investigating available evidence on the effectiveness and cost-effectiveness of the assessed programmes, and including the evaluation of relevant health outcomes, quality of life (QoL), and their ethical considerations. • Monitor the current and future burden of haemoglobinopathies in Europe and neighbouring non-EU affected countries which are also impacted by migration, in order to support and implement the most appropriate EUwide and national public health policies.
• Provide a roadmap towards the alignment of surveyed programmes with comparable socioeconomic parameters and towards a minimisation of the combined haemoglobinopathy-related health burden in EU.

Expected Impact
1. Gather evidence for the diffusion, or discontinuation of existing screening and prevention programmes allowing informed decisions by policymakers.
2. Assess capacity building through such screening and prevention programmes.
3. Assess improved health outcomes, greater health equity and cost effectiveness based on the implementation of effective screening and prevention programmes
This way, the best prevention strategies will lead to the reduction of the impact of haemoglobinopathies and additional inappropriate costs for the European Health Systems will be avoided.

Coming Soon!

• Guidelines for the Clinical Management of Transfusion Dependent Thalassaemias (2014)
  
  

Important News!

• TIF has established a new collaboration with the European Society of Emergency Medicine (EuSEM) in endorsing the book "Emergency Management of thalassaemia" and in distributing it to its members.

• Translated Books - Coming Soon!
• • “A Guide for the Haemoglobinopathy Nurse (2013) in Greek and Farsi.
  • Emergency Management of Thalassaemia (2012) - Porter J, Taher A, Mufarij A, Gavalas M in Greek
• About thalassaemia Cartoon in Bahasa Indonesian
• • Standards for the Clinical Care of Sickle Cell Disease of in adults (2008) Greek
  •  

    Scientific papers

    In addition, there are 4 scientific papers prepared by the TIF scientific medical panel in collaboration with WHO Regional Directors and WHO Directors of the Non Communicable Disease (NCD) Units of the 4 different Regional Offices of WHO, including :
    
    -EMRO
    -SEARO
    -WPRO
    -EURO
    
    These scientific papers are focused on the current status of control of haemoglobin disorders and in particular thalassaemia,  in terms of their prevention and clinical management within and in between each country of each region. We have secured collaboration and interest of the director of each WHO Regional Office for the preparation of these papers.
    
    Already the scientific paper which was compiled with the collaboration of the EMRO Regional Office has been finalised and is being reviewed at the moment by EMRO focal point, Dr Samer Jabbour and other medical experts of the region to give it a completion. By the end of 2014 the other scientific papers will be finalised.
    

     

    Position Papers

    Two important  position papers are underway:
    - Position paper on Hepatitis B and C
    - Position paper on Blood Safety.

Cooley’s Anaemia Foundation: Suffolk Chapter’s 3rd Classic Car Show and Test & Tune is 13 September
Car enthusiasts, take note: the Suffolk chapter of CAF is holding its’ Third Classic Car Show and Test & Tune on Saturday, September 13 from 9:00 a.m. – 4:00 p.m.!
Held in conjunction with Long Island Drag Racing, this special event takes place at the Hempstead F.D. Training Facility , 370 Milburn Avenue in Hempstead, NY. (The rain date, should that be necessary, is the next Saturday, September 20.)
Full information can be downloaded by clicking here.

Monday 18 August 2014
Media alert for immediate release
Spanish/ Portuguese version

Seen but not heard? The patient voice in drug regulation today
‘Increasing the patient voice in drug regulatory authorities’, a meeting hosted by Alianza Latina and the International Alliance of Patients’ Organizations, Saturday 23 August 2014, Rio de Janeiro.
Health experts, patient representatives and groups from across Latin America are coming together to discuss how to achieve universal health coverage – where every person has access to the health services they need – and how drugs regulatory authorities must work with patients to achieve this.Jo Groves, Chief Executive Officer of the International Alliance of Patients' Organizations, said: "This issue cannot be understated. It is one of the most pressing matters facing patients in Latin America today."
Experts will include Aline Silveira, from the Brazilian Ministry of Health, Jose Luis Castro, from the Pan American Health Organization and World Health Organization, and patient advocate Cesar Garrido, from the National Centre for Hemophilia, Venezuela, along with many other high profile guests.
Patients’ organizations, drug regulatory authorities, academia, industry, policy-makers and healthcare providers will debate how including patients at all stages of regulation can improve safety and access to treatment. The meeting will also focus on national, regional and global activities on biological and biosimilar medicines.
The meeting will be held on Saturday 23 August 2014 from 14.00-18.30 at the Hotel Windsor Florida, Rio de Janeiro. This will be followed by an evening reception at 18.30. The event will be held in English, Spanish and Portuguese. The official invitation can be found here.

Take care while travelling! Read the WHO Health Regulation on vaccinations in the different parts of the world offering advice to people regarding vaccination regulations they need to know when they travel. Read more on this link.

The recent European elections have provided a good opportunity to review the way viral hepatitis is addressed in the EU. Currently, the sheer number of reports and recommendations available to MEPs and the European Commission is failing to spur action; there is simply too much information from too many angles.

In response, the European Liver Patients Association, together with the World Hepatitis Alliance, called an EU Partners Meeting in Brussels 15 July to begin the process of synthesising all this information into one report to send to Members of the European Parliament (MEPs). Having all advocates speaking from one policy document is likely to significantly increase engagement.

Progress has been achieved in the prevention and control of non-communicable diseases! IAPO Governing Board Member, Eva Maria Ruiz de Castilla, has represented IAPO at a high-level meeting convened by the UN General Assembly on the 10th - 11th July 2014. The meeting has comprehensively reviewed and assessed the progress achieved in the prevention and control of non-communicable diseases.

The overall theme of the meeting was "Taking stock of progress in implementing the Political Declaration on the Prevention and Control of Non-Communicable Diseases and scaling up of multi-stakeholder and national multi-sectoral responses to the prevention and control of non-communicable diseases including in the context of the post-2015 development agenda".

Featured Article

Access to HCV/HBV screening and treatment in Eastern and Southeastern Europe

The burden of viral hepatitis in Eastern and Southeastern Europe (EE/SEE)
Recent epidemiological estimates clearly show that countries in Eastern Europe and Central Asia carry the highest burden of HCV and HBV infection in Europe.

Simple estimates of the number of current HBV and HCV infections in the WHO European Region showed that of the 427 615 000 adults aged ≥18 years living in
EU/EFTA countries, 4 487 000 (1%) have HBsAg and 5 467 000 (1.3%) have HCV RNA.

However, of the 304 522 000 adults living in the WHO European Area countries outside the EU/EFTA, 8 821 000 (2.9%) have HBsAg and 9 536 000 (3.1%) have HCV RNA.
1
Countries in the East and Southeast of Europe (EE/SEE) face particular challenges in relation to viral hepatitis due to a legacy of war, forced migrations and civil unrest as well as huge social and economic changes associated with the transition from communist to civil societies in the last decades.

Screening policies in EE/SEE A WHO report on viral hepatitis focusing on 44 member states from the WHO European region showed that 21 states (47.7%) have a national policy relating to screening and referral to care for hepatitis C.

Testing for hepatitis C was compulsory for  members of specific groups including blood donors, health-care workers, pregnant women, and patients on haemodialysis in 50% of the countries. Furthermore, 7 of 13 member states have a written national strategy or plan that focuses exclusively or primarily on the prevention and control of viral hepatitis and includes screening policies come from EE region (Czech Republic, Kyrgyzstan, Republic of Moldova, Armenia, Uzbekistan, Turkey, Russian Federation).

Analysis of HCV screening programs in SEE/EE countries shows different approaches to the choice of target populations for screening in individual countries (except for compulsory screening of blood and organ donors in all countries). Target populations with variable inclusion in screening programs in SEE/EE countries include: pregnant women (screened in Armenia, Ukraine, Montenegro, Serbia), health- care workers (Armenia, Moldova, Russia) as well as occupational exposure groups (Bosnia and Herzegovina, Croatia, Serbia) 4,5,6,7, hemodialysis patients (Bosnia and Herzegovina, Croatia, Moldova, Serbia, Russia) and persons with a history of shared injecting equipment (Bosnia and Herzegovina, Croatia, Serbia).

Target populations with very limited inclusion in SEE/EE screening programs are: military recruits (Ukraine), family contacts (Moldova, Croatia), candidates for chemotherapy or immunosuppressive treatment (Croatia), persons with a history of long-term imprisonment (Croatia, Serbia), HIV-infected persons (Croatia, Serbia) as well as IVF candidates (Croatia).

Noteworthy, surveillance of migrant populations is currently not a part of HCV screening programs in SEE/EE countries. The abovementioned data show that considerable efforts will be needed to ensure the systematic approach to the screening of high-risk populations such as migrants, intravenous-drug users, prisoners, STI clinic attendees and MSM in all SEE/EE countries for hepatitis C.

National policy related to screening and referral to care for hepatitis B is available in 50% of countries from the WHO European region.

Mandatory testing for hepatitis B for specific groups (blood donors, health-care workers, pregnant women and patients on haemodialysis) is reported for 54.5% of the countries.
Three quarters of the WHO European Region countries reported that a national policy that specifically targets mother-to-child transmission of hepatitis B includes screening of all pregnant women for hepatitis B.
Access to treatment in EE/SEE According to the 2013 WHO report on the European Region, national clinical guidelines for the management of viral hepatitis are available in 29 countries (65.9%).

Countries with clinical guidelines focusing on viral hepatitis from the SEE/EE include Croatia, Bosnia and Herzegovina, Georgia, Lithuania, Poland, Slovenia, Slovakia, Moldova, Latvia, Hungary, Estonia and Bulgaria. Thirty-nine (88.6%) countries from the European region reported that at least one available drug for treating hepatitis C is included on the national essential medicines list or subsidized by the government.

The drugs most commonly reported were interferon alpha, pegylated interferon and ribavirin whereas inclusion of first-generation of direct acting antivirals (DAA) boceprevir and telaprevir was observed only in 38.6% of reporting countries (n=12).

Restricted access to the first-generation DAAs in EE/SEE compared to Western and Central Europe is mainly associated with high treatment costs. The majority of patients from SEE/EE was treated with first-generation DAAs as a part of expanded access programs funded by the pharmaceutical companies in Croatia, Poland, Romania, Slovenia, Slovakia, Hungary, Estonia, Serbia and Bulgaria. Additionally, significant delays are observed between EU registrations of DAAs and national reimbursement decisions in SEE/EE.

For example, reimbursement for first-generation DAAs in Romania, Estonia, Russia and Bulgaria is expected in 2014. Second-generation DAAs, most importantly sofosbuvir and simeprevir, are currently unavailable in the SEE/EE. Due to the high predicted costs of the treatment regimens that include second-generation DAAs as well as future IFN-free regimens (>85 000 USD), it is reasonable to assume that the new treatment options will be available to patients in the SEE/EE exclusively via expanded access programs for some time to come thereby increasing the treatment gap between Eastern and Western Europe.

Major issues in hepatitis C treatment funding policies in SEE/EE: implications for the availability of triple therapy and 2nd generation DAAs and opportunities for the future National health insurance funds cover, at least in part, the price of hepatitis C and hepatitis B treatment (including diagnostic monitoring) in the majority of countries in SEE/EE. In reality, access to treatment is restricted due to the financial limitations of the national health insurance funds.

Patient’s financial contribution and/or additional state or private healthcare insurance is required to provide hepatitis treatment in many countries in the region.
Importantly, insurance schemes in some countries do not cover unemployed individuals, thereby excluding a large portion of intravenous drug users that carry the largest burden of infection in the region.

Some countries requested funding from international donors (Global fund, World Bank) but this covered only a small portion of overall treatment need (some programs were aimed at HIV/HCV coinfected persons and needle exchange programs only).

Removal of obstacles to excellence in viral hepatitis clinical care in SEE/EE will require close collaboration between the government, physicians, patients and pharmaceutical companies. Governments are expected to develop comprehensive and coordinated national strategies on hepatitis C that integrate prevention, surveillance, testing, treatment, care, support, training and awareness-raising efforts (legislative framework), play a more active role in harmonizing efforts on governmental and non-governmental levels and activate government mechanisms aimed at the reduction of pharmaceutical spending in the field of hepatitis C. Physicians are required to provide leadership in the creation and implementation of national prevention, testing and treatment policies on hepatitis C. They should develop national guidelines on the clinical management of hepatitis C in order to ensure that the most effective treatment regiments are included in the list of subsidized/reimbursed medications. They are expected to play an active role in the training of specialists, family doctors and other medical professionals involved in hepatitis C treatment.

Patients’ advocate groups can contribute via collaboration with government and medical professionals to ensure the patient’s perspective in national hepatitis C policies. They can also provide leadership in public awareness campaigns on hepatitis C (particularly in the context of stigmatization) and in efforts to promote point-of-care testing as well as by collaboration with pharma companies in efforts to promote compassionate use of DAAs.

Conclusions:
• Continued EU and WHO coordinated efforts will be needed to harmonize the access to treatment in Western and Eastern Europe.
• Good national plans that include  clever screening programs for  populations at risk are needed.
• Scaling-up of HCV screening  programs is required for healthcare planning in Eastern Europe, particularly in providing economic estimates during negotiations with various stakeholders and assisting patients’ organizations to gain the public awareness of chronic hepatitis C as a curable disease.
• Despite the clear benefits of DAAs in providing a cure, treatment costs are likely to remain an important obstacle to achieving excellence in HCV clinical care in Eastern Europe.

Click here for the article.

4th Pan-European Conference
Visit the News Section of the 4th Pan-European Conference website which was created with the intention of providing an outlook on all the latest updates and news regarding this conference. Regular updates will be provided on a weekly basis. More news are on the way!http://www.tif2014.org/resources/news

Visit the Conference website for further information at www.tif2014.org

Australian Blood Project
Australia has a significant number of Thalassaemia affected people, which is increasing as migration patterns change. However, the exact number of affected people is unknown as well as the severity of each case. Little is known about their long-term complications and outcomes. Although different centres currently manage the same disease, they do not follow a common protocol, and there is a need to determine which strategies work best for patients.
In this context, we are delighted to share with you that TIF has been invited to join a grant application which is to be submitted to the National Health and Medical Research Council (NHMRC) with the aim of creating a national haemoglobinopathy electronic registry in Australia. The invitation was sent by a group of haematologists led by the Head of the Transfusion Research Unit at Department of Epidemiology and Preventive Medicine (DEPM) in Australia, Dr Erica Wood.
The aim of the national Haemoglobinopathies electronic registry will: • Provide an important framework for future research to improve patient care
• Be used to help to plan health care services for the future
• Bring together a network of Australian health care professionals with a special interest in Haemoglobinopathies, for sharing ideas.
The Registry is the first step in a major project to ensure that all Australians with a haemoglobinopathy continue to receive the best possible care. Further details about this project will follow in our next Board Member Update.

MRI Information Material

The MRI information material has already been prepared by the members of the MRI consulting group of TIF that have met in Abu Dhabi in October 2013 in the context of the TIF International Congress. Dr Androulla Eleftheriou with her own expert group at the TIF headquarters are now reviewing the material and are in the process of finalising three types of educational/ informational material:
1. For the Patients/parents
2. For the medical specialists, haematologists, and paediatricians treating Thalassaemia
3. For users of the MRI

We are also in the process of seeing in which most possible way we can encourage the members of every country  to ensure that the quality of their MRI results are up to a certain standard.

28 of July is World Hepatitis Day! Resolution WHA63.18, adopted in 2010 by the World Health Assembly, recognized viral hepatitis as a global public health problem and saw the need for governments and populations to take action to prevent, diagnose and treat viral hepatitis (To view the resolution visit on this link).

Millions of people across the world now take part in World Hepatitis Day, to raise awareness about viral hepatitis, and to call for access to treatment, better prevention programs and government action. Take a look at the World Hepatitis Day Website to find posters, and many more materials and watch the World Hepatitis Day video by visiting this link!

New Committee at UKTS
The UK Thalassaemia Society held its AGM on the 25th February 2014. We were delighted to welcome four new Trustees to the UKTS Management Committee - Tina Bhagirath, Chris Fassis, Anand Singh Ghattaura and Raj Klair. It is always refreshing for any organisation to have new people with new ideas and perspectives; and we look forward to a very productive year!

The Trustees of the Society are (in alphabetical order):
 - Tina Bhagirath 
- George Constantinou (Secretary) 
- Chris Fassis (Assistant Secretary) 
- Pany Garibaldinos (Assistant Treasurer) 
- Anand Singh Ghattaura (Vice President) 
- Raj Klair 
- Romaine Maharaj (Treasurer) 
- Gabriel Theophanous (President)

New guidelines for managing thalassaemia in pregnancy announced at the Royal College of Obstetricians and Gynaecologists (RCOG) World Congress in India

New advice for clinicians on the management of women with thalassaemia in pregnancy, was published by the Royal College of Obstetricians and Gynaecologists (RCOG) and launched at the annual RCOG World Congress in Hyderabad, India. To read the full guideline please go this link.

Article : Double Baby - Congratulations to Rita and Andrew
By Rita Rodricks
Andrew and Rita Rodricks are proud to announce the birth of our twins, Dylan and Ellie, born on the 19th June 2013 at North Middlesex Hospital. I am 44 years old, with thalassemia major; and we had been trying for 7 years to have a baby. It was hard living with this disease (both emotionally and financially) with the repeated knock backs, but we kept on with it and now we are blessed with two beautiful children. I would just like to say don’t give up just because you have thalassemia. It didn’t beat us! I was so shocked when told we were having twins. I had a good pregnancy until the eighth month when I started having difficulties; and my babies were born at 34 weeks. As they were premature the twins remained in hospital for 2 weeks, until their weights reached the correct levels. Other than that, they are perfectly healthy babies!

We know that all our readers will be thrilled for the proud parents. Rita is under the care of Consultant Haematologist Dr Anne Yardumian at the North Middlesex Hospital. Our grateful thanks to Dr Yardumian, Consultant Obstetrician Miss Frances Evans, Transfusion Practitioner Karen Madgwick and Clinical Nurse Specialist Francis Mate-Kole - says Rita “I had wonderful support from my doctors; they were absolutely brilliant - and Karen was there for me morning, noon and night. My nurse Francis even came in on his day off to cannulate me while I was on the labour ward! I thank them all from the bottom of my heart”.

Genome-editing method repairs mutations that cause beta-thalassemia A genome-editing technique has been used to correct mutations that cause beta-thalassemia in human cell lines. Researchers transformed skin cells from a patient into induced pluripotent stem cells and then fixed the mutations before prompting the cells to mature into hematopoietic progenitors and erythroblasts. More work is needed before cells are developed that could be used in transplants. The findings were reported in the journal Genome Research.The Scientist online (AABB 6/8/2014)

App to detect anemia earns top prize at Microsoft tech challenge A mobile health application that can be used to detect anemia in patients has nabbed the top prize at the 2014 Microsoft Imagine Cup challenge. Eyenaemia, designed by medical students Jarrel Seah and Jennifer Tang, allows users to take pictures of their eyes, have the images analyzed and find out... Share This news summary appeared in AABB SmartBrief on 08/06/2014 Click here to view the full issue.

AABB offers preconvention workshop on Patient Blood Management 
AABB will offer a Patient Blood Management Workshop on Friday, Oct. 24, at the Pennsylvania Convention Center prior to the start of the 2014 AABB Annual Meeting. The workshop will address operational and clinical aspects of PBM. Speakers at this interdisciplinary workshop will discuss, among other issues, the use of data and benchmarking in PBM, the challenge of educating practitioners through credentialing or certification, and platelet function in endothelial cell repair. The workshop will be followed by a vendor showcase reception. Those who wish to register for this workshop can do so through the AABB Annual Meeting registration site. For moreinformation, click here

Study: 1 in 3,000 blood donors in England may have hepatitis E 
About 1 in 3,000 donors in England could be infected with the hepatitis E virus, according to a study in The Lancet that retroactively screened 225,000 blood donations in southeast England. The overall risk of hepatitis E is slight, said lead researcher Dr. Richard Tedder of the Blood Borne Virus Unit at Public Health England. But the virus can cause chronic liver disease in immunocompromised patients, and "a policy is needed to identify these persistently infected patients and provide them with appropriate antiviral treatment," Tedder said. Nursing Times (U.K.) (free registration)/The Press Association (U.K.) (7/28) click here for more

Blood Cell Genetic Variants That Reduce Sickle Cell And Thalassemia Symptoms Originated In Africa

scientists have found that certain people, who experience milder symptoms of these conditions, carry two variants of this gene that enables them to make fetal hemoglobin in their bodies even in adulthood. Using worldwide genomic studies, the scientists from King's College London, found that these beneficial genetic variants have spread from Africa into nearly all human populations across the globe.  

Attitudes and behaviours of Greeks concerning blood donation: recruitment and retention campaigns should be focused on need rather than altruism.More Information here (NCBI 12/7/2014)

The risk of transfusion-transmitted viral infections at the Gabonese National Blood Transfusion Centre. More Information here (NCBI 12/7/2014)

Sickle cell disease in areas of immigration of high-risk populations: a low cost and reproducible method of screening in northern Italy. More Information here (NCBI 12/7/2014)

Storing red blood cells with vitamin C and N-acetylcysteine prevents oxidative stress-related lesions: a metabolomics overview More Information here (Research Gate 14/7/2014)

Scientists modify blood cells to deliver therapeutic proteins. More Information here (PBR 4/7/2014)

Baxter buys developer of experimental sickle cell drug. More Information here (Genetic Engineering & Biotechnology News 9/7/2014)

Study will examine immune systems of patients whose hepatitis C is cured. More Information here (AABB 16/7/2014)

Hepatitis C patients might need vitamin D screening, study finds. More Information here (Healio 9/7/2014)

Genetic pathways used to convert stem cells into blood cells. More Information here (AABB 15/7/2014)

Study: Half with hepatitis C in U.S. are unaware of their infection. More Information here (AABB 3/7/2014)

Genotype 1 HCV–Infected Cirrhotic Patients: Still Hard to Treat? More Information here (Clinical Options 27/6/2014)

Appraising the 2014 AASLD/IDSA Guidelines on HCV Testing and Treatment. More Information here (Clinical Options 22/4/2014)

Treating HBV Patients With Cirrhosis: The Need for Vigilant HCC Screening. More Information here (Clinical Options 14/5/2014)

Keep Us Updated!
TIF encourages all its members to update their websites! We plan to upload your news and activities regularly and we will project important information from your Associations' websites.