September 2015 | Volume 26, Issue 1
Dear Friends,

Summer is slowly drifting away, but activity is flowing at the TIF headquarters! We hope everyone's summer break was both restful and productive! We are anticipating a new start and look forward to sharing new challenges, opportunities and accomplishments with you!

Editorial Team
TIF Around the World

TIF conducted a delegation visit to Iran, between 16-18 September 2015 after receiving an invitation by medical specialists of the region to participate in its national haematology conference, thus providing its expertise and knowledge for improving the quality of health and life of thalassaemia patients in Iran.

The TIF delegation consisted of Mr George Constantinou, TIF Board Member and Dr Michael Angastiniotis, TIF Medical Advisor.
The aim of this visit was to meet with Patients’ Associations for Thalassaemia to  address and assess their various concerns, which revolved mainly around the extensive use of counterfeit drugs in their country. As part of this visit, many meetings were organized with state health officials and relevant stakeholders.

Iran is a country in the Middle East which constitutes a model in the region but in which many gaps still need to bridged with regards to its infrastructures for thalassaemia. There is still an urgent need for the implementation of national programs to ensure proper treatment of the disease in all areas of the country.

University of Nicosia Medical School signs MOU with TIF
The Medical School of the University of Nicosia and TIF have signed a memorandum of understanding (MOU) to promote academic and research collaboration in the field of haemoglobin disorders.   Among other actions and activities, the Medical School with the support of TIF, will develop and offer an elective module on anaemias and rare disorders and will incorporate it within the structure of the MSc in Family Medicine offered by the School. 

Furthermore, the Medical School and TIF will award, on a biannual basis, the ‘Panos Englezos Prize’, to an individual who has made outstanding contributions to the field of haemoglobin disorders or public health. The Prize was established in 1997 by the TIF Board of Directors and its International Advisory Panel, in honour and recognition of the vast, lifelong, volunteer contribution of Mr Panos Englezos in promoting and safeguarding the interest and rights of patients across countries, religions, gender, social and cultural differences for equal access to quality health and other care.

The Prize – a solid silver drop of blood (TIF’s logo) – is accompanied by a monetary award of $10,000, and it will be awarded during the graduation ceremony of the Medical School.

TIF has also completed a visit to Germany between 25-27 September 2015. TIF was represented by Mr George Constantinou, TIF Board Member.

The objective of his visit was to consolidate the patient groups in Germany and increase their capacity to advocate for equal patient care.

A more detailed report about this visit is in the process of being compiled by Mr George Constantinou and we will share it with you in our upcoming Newsletter.

TIF Publications

Out Now!

  • The Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT), 3rd edition  has been released on the NLM Bookshelf’s public site. Please click here to view it. The book’s contents are accessible by search query

  • Translations of Publications
      • Standards for the Clinical Care of Sickle Cell Disease in Adults (2008) in Greek
    • A Guide for the Haemoglobinopathy Nurse (2013) in Greek
      • Emergency Management of Thalassaemia (2012) in Greek
  • Coming Soon!
    Translations of Publications
  • About thalassaemia Cartoon (2010) in Bahasa Indonesian
  • Sickle Cell Disease (2008) in Arabic
  • Emergency Management of Thalassaemia (2012) in French, Arabic, Turkish
  • A Guide for the Haemoglobinopathy Nurse (2013) in Burmese and in Farsi
  • Guidelines for the Clinical Management of Transfusion Dependent Thalassaemia (3rd ed.) (2014), Chinese, French, Arabic, Turkish, Greek.
  • Guidelines for the Clinical Management of Non Transfusion Dependent Thalassaemia, French, Arabic, Turkish, and Greek.
  • Translations of Videos
    • All about Thalassaemia Cartoon Animation (2013) is being translated into Bahasa Indonesian, Malaysian, Chinese, Hindi, Urdu, and Turkish
      • Papers for publication

        • A paper has been submitted for publication named “Thalassaemia Reviews"
      • Another paper about TIF and its role has been prepared by the TIF office to be published in commonly read journals in every region of the world
International News

CAF Speaks at FDA Pediatric Advisory Committee Meeting

On September 17, 2015, the FDA (Food and Drug Administration) Pediatric Advisory Committee (PAC) advises and makes recommendations to the Commissioner of Food and Drugs regarding a range of issues related to prescription drugs intended for use in the pediatric population. A panel of doctors and other medical specialists sit on this panel, with appropriate FDA staff providing support. CAF’s National Vice President Amy Celento currently serves as its patient/family advocate, a role that is vital in providing the point of view of parents and family members and for which she has expert skills. As a mandate for when the FDA expanded the indication of Exjade for use among non-transfusion-dependent thalassemia patients (including those that fall within the pediatric population), the FDA reviewed all Exjade-related Adverse Events reported through the FDA Adverse Events Reporting System (FAERS) from January 23, 2013 through February 28, 2015.

The FDA PAC met on September 16 to consider the review submitted by the FDA. This meeting opened an opportunity for CAF to ask the FDA its opinion on whether data available supported the need to amend the Exjade label and advise users to temporarily stop its use in the case of febrile illness. With this in mind, CAF testified during the public presentation portion of the meeting, as did Sarah Baqueri-Connolly, who detailed for the committee circumstances surrounding her daughter’s tragic death. Due to CAF and Sarah’s testimony, the Committee requested that the FDA further review case histories and all other information available through manufacturer’s reports and provide more data so that the committee can make a determination if a label change should be added clarifying whether Exjade use should be halted temporarily when fever is present.


New Project to Improve Timely Access to Medicines!

EURORDIS is partnering in the recently launched ADAPT SMART project, which will establish a platform that enables the coordination of Medicines Adaptive Pathways to Patients (MAPPs) activities. MAPPs can help to ensure access to new treatments for patients with unmet medical needs, such as rare diseases, at the earliest appropriate time in the entire life cycle of a new treatment.

9th Annual Sickle Cell and Thalassaemia Advanced Conference

Date: 7– 9 October 2015
Time: 09.00 – 17.00
Venue: The Hilton Tower Bridge, 5 More London Place, Tooley Street, London, SE1 2BY

Now in it's ninth year, this global conference is aimed at those with a common interest in sickle cell disease (SCD) and thalassaemia, including specialist registrars, consultant haematologists, paediatricians and clinical nurse specialists.

It is designed to update professionals working with patients with SCD and thalassaemia to consider the most efficient and safe way to manage such vulnerable patients.

Participants from around the world will gain insight on a range of areas, including new therapies, managing acute and chronic complications, iron chelation therapy, managing the transition from paediatric to adulthood, and how to close the gap between the management of SCD in developing  and developed countries.

International Liver CongressTM 2015!



Interested in viral hepatitis, co-infections, liver cirrhosis, abnormal liver tests, metabolics and cholestasis?

Four years later, we are returning to Barcelona for yet another incredible International Liver CongressTM.

The application has been extended to 28 September 2015 giving you a little extra time to apply! 

Please note the following dates: 

Abstract submission
Opens 01.10.2015
Closes 24.11.2015

Congress & registration website
Available 01.10.2015

Late-breaker abstract submission
Opens 24.11.2015
Closes 24.02.2015
Have a look at the conference programme and apply now!

World Health Organization (WHO)’s Access to new medicines in Europe: technical review of policy initiatives and opportunities for collaboration and research

This report by WHO, with a focus on sustainable access to new medicines, reviews policies that affect medicines throughout their lifecycle (from research and development to disinvestment), examining the current evidence base across Europe.

While many European countries have not traditionally required active priority-setting for access to medicines, appraising new medicines using pharmacoeconomics is increasingly seen as critical in order to improve efficiency in spending while maintaining an appropriate balance between access and cost–effectiveness.

The study features findings from 27 countries and explores different ways that health authorities in European countries are dealing with high spending on new medicines, including methods such as restrictive treatment guidelines, target levels for use of generics, and limitations on the use of particularly expensive drugs. It also outlines possible policy directions and choices that may help governments to reduce high prices when introducing new drugs.

Only available online.

We are delighted to share with you a Patients' Journey, Dr Chandan Das' biography entitled "The Road Taken" (English Edition).

The plot revolves around a child born with thalassemia in a little town by the sea among hills and forests. Poetry, the lords and ladies of Byzantium, wolves, firs, and aspens bending in the wind and the search across the world for life-saving treatment for a child with thalassaemia are the main ingredients of this story.

For the e-book, please click here.

Delegation Visits


The 2nd Pan-Asian Conference on Haemoglobinopathies , Hanoi, Vietnam, 26-27 September  2015
An educational event in a unique region of the world: South-East Asia and the Western Pacific!

We are very proud to share with you that the 2nd Pan-Asian Conference has been conducted between 26-27 September 2015, in Hanoi, Vietnam with immense success!

 This educational event is organized in the context of TIF’s educational programme and in particular in its focus on regional needs and expectations.

This conference gathered together over 700 health professionals and patients/parents from 19 countries of the targeted regions and the importance attached to this educational event is reflected through the participation of His Excellency the Minister of Health of Vietnam, Representative of ASEAN and SEARO WHO, Her Highness Sheikha Sheikha Bint Seif Al-Nahyan, a distinguished member of TIF’s Board of Directors.

As for the Programme of the conference, it was divided into two parts running simultaneously:
1) The Scientific Programme which focused on scientific medical advances as well as reviews on existing progress in addressing the prevention and management of haemoglobin disorders, and
2) The Patients’/Parents’ Programme which addressed the needs for education, awareness and capacity building of patients in the region.
The significance of the Patients’/Parents’ Programme is today more and more important, as we now live in the era of patient-centered healthcare reforms and patient involvement in decision-making in the health sector is very important. Patients however, need to be knowledgeable and competent in order to have a stronger and more reliable voice, as well as an important role in advocating and for achieving changes.
The topics were covered by 40 national, regional and international experts and at the end of the conference a joint declaration and an outcome of the Conference to move forward and continue this effort was signed by the participants and delivered to the Ministry of Health of Vietnam and the WHO of the SEAR and WPR regions.

More information is available on our website!

We are delighted to announce that TIF's is in the process of finalizing its new application, the Digital Library app!

This app provides users with a powerful platform containing all of TIF’s publications and informing them regarding all of TIF’s events and news.

The TIF Digital Library will be available soon on both the Google Market and the Apple Store!

Share our ThaliMe page on Facebook! We have some exciting news coming very soon!

Conference on European Reference Networks
We are delighted to share with you that TIF, represented by Dr Androulla Eleftheriou, will participate in  the “2nd conference on European Reference Networks”, organised by the  DG Health and Food Safety between 8-9 October 2015 in Lisbon, Portugal. 
This 2nd conference, hosted by the Ministry of Health of Portugal, is an event of the Luxembourgish EU Presidency.   The event builds on the success of the 1st conference on European Reference Networks which took place in Brussels June 23rd 2014 (see the presentations and outcomes).

The aim of the conference is to discuss and raise awareness on the state of the art on the organisation of highly specialised networks and centres of expertise across the EU, to present the draft assessment manual and toolbox to be used for assess the Network proposals and to help prepare Health Care Providers for the call for proposals of European Reference Networks that the Commission will launch.

The conference aims to bring together, highly specialised healthcare providers, experts, national authorities, decision-makers, patient and professional organisations and other interested stakeholders.

TIF’s MSc in Inherited Haemoglobin Disorders: Thalassaemia and Sickle Cell Syndromes (Blended)
TIF is delighted to announce that the work for establishing its MSc course in Inherited Haemoglobin Disorders - Thalassaemia and Sickle Cell Syndromes (Blended mode) in collaboration with the University of Nicosia, Cyprus is gradually advancing and is in the process of being finalised.

More details about this programme here.

Theme: Patient Voice
Slogan: Join us in making the voice of rare diseases heard

2016 marks the ninth year that the international rare disease community celebrates Rare Disease Day.
On 29 February 2016, people living with or affected by a rare disease, patient organisations, politicians, carers, medical professionals, researchers and industry will come together in solidarity to raise awareness of rare diseases.

The Rare Disease Day 2016 theme ‘Patient Voice’ recognises the crucial role that patients play in voicing their needs and in instigating change that improves their lives and the lives of their families and carers.

The Rare Disease Day 2016 slogan ‘Join us in making the voice of rare diseases heard’ appeals to a wider audience, those that are not living with or directly affected by a rare disease, to join the rare disease community in making known the impact of rare diseases. People living with a rare disease and their families are often isolated. The wider community can help to bring them out of this isolation.


September - Sickle Cell
Awareness Month!

SCTI has run information sessions on 13 September in various areas of Dublin to raise awareness for Sickle Cell anaemia as part of their annual September Sickle Cell awareness month celebrations/activities.

If you are interested in being volunteers to act as missionaries for Sickle Cell awareness, please contact: info@sicklecellireland.ie or Telephone; 0870656807

Read more

Research & Therapeutic News

National Science Foundation gives grant to developer of sickle cell disease monitor
The National Science Foundation gave a $166,935 grant to Florida Atlantic University researcher Sarah Du to design a disposable testing system that can be connected to a smartphone to track the activity of red blood cells in sickle cell disease patients. Data gathered by the system could be used by patients to implement steps for early (AABB 11/9/2015).
Read more

La Jolla Pharmaceutical's iron overload treatment receives EMA panel backing
The European Medicines Agency's Committee for Orphan Medicinal Products (COMP) adopts a positive opinion recommending La Jolla Pharmaceutical's (NASDAQ:LJPC) LJPC-401 an orphan drug for the treatment of chronic iron overload requiring chelation therapy. Chelating agents bind to and help clear excessive iron from the body, but they are toxic. La Jolla's LJPC-401 is a novel formulation of hepcidin, a naturally occurring peptide hormone that prevents the accumulation of iron in organs.Abnormal iron accumulation occurs in disorders such as sickle cell disease, beta thalassemia and hereditary hemochromatosis (AABB 11/9/2015) .
Read more

Gene Therapy for Transfusion Dependent Beta-thalassemia (TIGET-BTHAL)
A research is being conducted - currently in phase I/II study - evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by transfusion dependent beta-thalassemia (ITHANET 17/6/2015)
Read more

Scientists develop novel biosensor for hepatitis treatment
Researchers with the Moscow Institute of Physics and Technology's Laboratory of Nanooptics and Plasmonics have developed a biosensor-based graphene oxide. The study team reported in the journal ACS Applied Materials & Interfaces that they have found a new carbon allotrope that increases the sensitivity of biosensors (28/9/2015).
Read more

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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