August 2014 | Volume 16, Issue 1
Dear Friends,
With the summer now officially behind us, and hints of autumn finally beginning to take hold, we are pleased to present our August issue of the TIF Newsletter. This issue highlights some of the many activities and events that have taken place in the August as TIF is now preparing for the busyness of fall. Happy reading!
Editorial Team
TIF Around the World

An important highlight of August was the preparatory meeting which was held on 26 August 2014 with the aim of discussing the direction of a Workshop which is to be organized by TIF with the collaboration of the Cyprus Alliance for Rare Diseases on the topic of the Cyprus General Health Plan.This workshop is anticipated to take place in early October in Nicosia. The aims of this workshop will be to learn about all aspects of the General Health Plan but also to examine its provisions as regards to rare diseases including haemoglobinopathies.

In this context, this meeting was participated by the Organisational Committee of this Conference, among which Dr Androulla Eleftheriou, TIF Executive Director, Dr Andreas Polinikis, of the Medical School of the European University of Cyprus, Dr George Potamitis, President of the Cyprus Society of Gastroenterology, Mrs Katia, Kyriakou, President of the Foundation "Pedika Xamogela", and Mrs Katerina Papadopoulou, President of the Cyprus association of Congenital Heart Diseases in Teenagers and Adults.

The outcome of this meeting is a very fruitful discussion regarding the vision, the direction and the objectives of the General Health Plan in Cyprus.

4th Pan-European Conference on Haemoglobinopathies and Rare Anaemias – the preparations continue! Keep posted!

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

More information about the conference here!

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.

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.

TIF Publications

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 :


      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.

International News

Car enthusiasts, take note! Cooley’s Anaemia Foundation is organising Suffolk Chapter’s 3rd Classic Car Show and Test & Tune! The Suffolk chapter of CAF is holding ithis event 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.

Seen but not heard? The patient voice in drug regulation today

‘Increasing the patient voice in drug regulatory authorities’, was a meeting hosted by Alianza Latina and the International Alliance of Patients’ Organizations, on Saturday 23 August 2014, Rio de Janeiro.

Health experts, patient representatives and groups from across Latin America came 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 included, Dr 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 debated how including patients at all stages of regulation can improve safety and access to treatment. The meeting also focused on national, regional and global activities on biological and biosimilar medicines.

WHO/Europe’s efforts for the prevention and treatment of hepatitis focus on types B and C. These are the most prevalent types in the WHO European Region, where 13.3 million people are estimated to live with hepatitis B, and 15 million with hepatitis C. Of those infected, over 120 000 die every year. Two thirds of the people in the Region with hepatitis B and C live in eastern Europe and central Asia.

Hepatitis is closely connected to Thalassaemia as some patients develop liver complications because they become infected with hepatitis A, B or C. Hepatitis B and C can cause fibrosis and cirrhosis. One route of transmission for all three of these viruses is from blood transfusion from infected donors.

Read more

IAPO members at UN meeting on non communicable diseases (NCDs)

IAPO Governing Board Member Eva Maria Ruiz de Castilla recently attended two UN meetings on non-communicable diseases (NCDs).

An interactive meeting for civil society was held on 19 June 2014 to discuss progress in Non Communicable Diseases (NCDs) work since 2011 and to identify priorities going forward.

Following this, the official UN NCD review was held on 10-11 July 2014. This was an opportunity for governments, the UN system, and civil society to take stock of progress since 2011, identify gaps in action, and gather consensus on scaling up and transforming commitments into action at the national level, where the fight against NCDs must be won.

Around 150 civil society representatives and 37 member states attended the hearing. This was followed by two roundtable panel discussions on enhancing international cooperation, and mobilizing whole-of-society efforts around NCDs.IAPO NCD statement.

Time is running out!

Just one week until the deadline for the thalassaemia video challenge is over! It’s not too late to submit your video! Share your stories, your concerns, and your success!

Already we have received many inspiring videos until this point and we are anticipating even more!

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! Click here to view the video.

We are also very happy to have received a very inspiring submission by Josephine Billa for the thalassaemia video challenge! We wish her good luck! Click on this link to watch the video

To submit a video click here

To Australia and beyond...

TIF has initiated a new endeavour, an ambitious project called the 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 Newsletter.

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.

We would like inform you on the latest updates from WHO on the Ebola Outbreak. In view of this out-break and for your own safety when travelling, please read the links below of the WHO website to carefully to get informed of how this epidemic is being controlled.
To read about the Disease Outbreak Updates, click here and here

Join ENERCA and ESH at their next Training Course on Haemoglobin Disorders!

On January 23-24 ENERCA and ESH will join their efforts in Barcelona to tackle rare anaemias! The ENERCA project started in 2002 with the purpose of offering an improved public health service to medical practioners, health professionals and  patients, in every aspect of rare anaemias.

One of the key tools to achieve this objective is continuing medical education and  ENERCA has therefore renewed the close collaboration established with the European School of Haematology (ESH) during previous phases of the project. In the on-going e-ENERCA project, the Working Package (WP5) led by Dr. Patricia Aguilar-Martínez, from the CHU de Montpellier (France), will focus on educational aspects of rare anaemias, using both onsite and internet –based learning.


Research & Therapeutic News

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. (AABB 6/8/2014)
Read more

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 Patient Blood Management (AABB 6/8/2014)
Read more

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 (AABB 28/7/2014).
Read more

Blood Cell Genetic Variants That Reduce Sickle Cell And Thalassemia Symptoms Originated In Africa
In this study published in the Annals of Human Genetics, scientists found that certain patients of African and South Asian descent had the ability to produce fetal hemoglobin even in adulthood and this reduced their blood disorder symptoms. These patients carried a genetic variant that controlled the red blood cell regulator gene MYB — MYB enhancer variant — on chromosome (MedicDaily 15/08/2014)
Read more

IthaGenes: an interactive database for haemoglobin variations and epidemiology

We are delighted to announce the publication of an article describing the IthaGenes database and the companion IthaMaps tool in the open-access journal PLoS ONE. IthaGenes is an interactive archive of all sequence variations affecting haemoglobin disorders, including the globin loci and disease modifiers and polymorphisms with relevance for clinical diagnosis. (ITHANET 22/08/2014)

Read more

Comparing Acute Pain Management Protocols for Patients With Sickle Cell Disease

The goal of this pilot study is to improve emergency department (ED) pain management for adults with sickle cell disease (SCD). Management of painful episodes associated with SCD, referred to as vaso-occlusive crises (VOC), is the most common reason for SCD patients to visit the ED. (ITHANET 22/08/2014)

Read more

Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators (SHiP HU)

Multi-phase, patient navigator-based program in the Richmond and Tidewater regions of Virginia to demonstrate: the feasibility of using patient navigators to improve the percentage of adult (age 15 and older) patients with sickle cell disease (SCD) in SCD specialty care the efficacy of using patient navigators to improve hydroxyurea (HU) (re-)initiation and adherence among adult patients with SCD eligible for HU (Patient navigators may also be known as public health workers.) (ITHANET 21/08/2014)

Read more

Patient-Provider Tools to Improve the Transition to Adult Care in Sickle Cell Disease (iTransition)

The purpose of the study is to develop patient-provider clinical support tools to improve clinical practice, patient self-management, and disease outcomes in sickle cell disease during transition to adult care. (ITHANET 21/08/2014)

Read more

Assessment of Tolerance of Mobilizing Peripheral Hematopoietic Stem Cells by Plerixafor in Sickle Cell Patients (DrepaMob)
The purpose of this study is to assess the tolerance and efficacy of mobilizing hematopoietic stem cells after a single injection of plerixafor (0.24mg/kg) in 3 adult patients affected by sickle cell disease. (ITHANET 21/08/2014)

Read more
Delegation Visits
See our next issue for delegation visits that are to take place


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.

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