Pour les patients

The radical improvements in prevention and management of β-thalassaemia major that have been taking place in the last three decades, primarily in the West, have led to significant progress in thalassaemia prevention and treatment. Thanks to these improvements, patients with β-thalassaemia major can now have an increased survival rate and a better quality of life. TIF’s mission is to ensure this progress reaches and benefits all patients across the world.

The Thalassaemia International Federation (TIF) has developed a plethora of educational material and resources that provide concise, up to date information on all aspects of thalassaemia, from prevention to clinical management, and cover almost all age groups. Constantly monitoring international developments, TIF’s objective is to provide strong and solid knowledge about the latest advances in thalassaemia prevention and care to patients, their families, healthcare professionals and the community at large, and thus empower them to advocate for better quality of life for thalassaemia patients worldwide.

This section provides a variety of material and electronic resources of interest to patients, including articles, books, e-courses, clinical trial updates, mobile apps, and opportunities to participate in awareness-raising activities.

Read

Read about thalassaemia, clinical trial updates aiming to provide the final cure, patients’ stories and key publications.

Patients’ Stories Patients’ Rights About Thalassaemia All About Thalassaemia (kids’ edition) Clinical trial updatesSickle Cell Disease TRANSFUSION for thalassaemia: I can help Useful Links Learn through videos

Participate & Learn

Discover exciting opportunities to participate & learn through contests and complete our online courses to become an expert patient. Join the International Thalassaemia Community to share ideas and experiences with patients all over the world.

Thal e-Platform for Patients Thalassaemia Patients Connect International Thalassaemia Day Join the ThaliME community TIF Digital Library

Latest Posts for Patients

TIF Webinar Fridays Are Back With A New Session on « Heart Disease In Thalassaemia »

This Friday, 23 October 2020, at 15:00 EEST/13:00 BST, get ready to join Prof. Malcolm Walker and Dr. Dimitris Farmakis in our webinar on  »Heart Disease In Thalassaemia » for Patients/Parents, powered by TIF’s Thal e-Course, an online educational initiative aiming at the development of thalassaemia patients’ knowledge and skills. Registrations are now open: https://bit.ly/348iy9z Learn …

Thal e-Course Webinar Series: Iron Monitoring in Thalassaemia

The effective management of iron overload and the importance of frequent monitoring of iron status in patients with thalassaemia were the focal points of the Thal e-Course Webinar Series’ 2nd session, which took place online on Friday, 9 October 2020. Dr Paul Telfer explained how survival of patients with transfusion-dependent thalassaemia can be improved with …

Johnson & Johnson Pauses Clinical Trials For Its Covid-19 Vaccine Over Participant’s Illness

Johnson & Johnson has paused its Covid-19 vaccine trial due to an “unexplained illness” in a participant, the company confirmed yesterday evening. The pharmaceutical giant was unclear if the patient was administered a placebo or the experimental vaccine, and it’s not remarkable for studies as large as the one Johnson & Johnson are conducting – …

Hidden Victims Of The Pandemic: Blood Bank Stocks Run Low As India’s Healthcare System Buckles

As public healthcare resources are redirected to halt the spread of coronavirus, victims of other life-threatening diseases pay the ultimate price. This recent news story, published by The Telegraph, depicts the severe implications of the COVID-19 pandemic on several patients with thalassaemia in India, where healthcare services are pushed to capacity due to the exceptional …

New Video Series on Living With Thalassaemia by CDC & CAF

The Centers for Disease Control and Prevention (CDC) and TIF´s Member Cooley´s Anemia Foundation (CAF) have announced the launch of a new video series highlighting the story of Aaron Cheng, a physician in training with thalassaemia. Aaron shares his experience growing up with thalassaemia and provides tips for others on how to manage the condition …

Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease

Fulcrum Therapeutics, Inc.  a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announced that FTX-6058, its investigational treatment for sickle cell disease (SCD) and β-thalassaemia, which has showed efficacy at raising the levels of fetal haemoglobin in cellular and animal models of the diseases, will now enter a a …

TIF Develops Mobile Application To Support Patients With Thalassaemia

As part of its enduring efforts to develop useful tools for individuals with thalassaemia and haemoglobin disorders worldwide, TIF will soon introduce a novel mobile health application, named ‘’THALIA Mobile App’’. This application is primarily designed to constitute a digital assistant for patients which will help them self-manage their disease more effectively on a daily …

Coming Up This Week:  »Iron Monitoring in Thalassaemia » Webinar for Patients

Although chronic blood transfusion regimens have added decades to the lives of patients with thalassaemia, clinicians are now faced with increasingly complicated management challenges. The devastating effect of the accumulated iron from chronic blood transfusions necessitates a more finely tuned approach to limit the complications of the disease, as well as its treatment. Survival in …

Gene Therapy LentiGlobin Granted Priority Medicines Designation for Sickle Cell Disease in the EU

Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD). Given to candidate therapies with promising clinical data for diseases of high unmet medical need, the designation is designed to speed clinical development and review of medicines, helping them to reach patients earlier. “The PRIME designation shows …

Thal e-Course Webinar Series: Liver Disease in Thalassaemia

Meeting your continuing educational needs on thalassaemia has never been easier! The 1st Webinar to inaugurate the TIF Thal e-Course Webinar Series for patients with thalassaemia has been successfully completed. Prof Geoffrey Dusheiko discussed the diagnosis, monitoring and new therapeutic approaches of liver disease in patients with thalassaemia, whereas Prof John Koskinas made extended reference …

Health Canada Approves REBLOZYL® For Adult Patients with β-Thalassaemia

Bristol Myers Squibb Canada (BMS) and Acceleron Pharma Inc. announced today that Health Canada has approved REBLOZYL® (luspatercept) for the treatment of adult patients with red blood cell (RBC) transfusion-dependent anemia associated with β-thalassaemia. REBLOZYL® is the first and only erythroid maturation agent in Canada, representing a new class of treatment for eligible patients. “Canadians living with β-thalassaemia, who …

TIF Takes Active Part At The IAPO 9th Global Patients Congress

The International Alliance of Patients’ Organizations (IAPO) conducted online the 9th Global Patients Congress #GPC2020 on 16-17 September 2020, under the theme  »Co-creation of Innovative Healthcare ». The Congress featured a total of 1200 attendees from 101 countries and 2 days of interactive discussions about healthcare strengthening and preparedness through patient-led innovative solutions, and the impact …

Gene-editing Therapy CTX-001 Granted Priority Medicines Designation for Severe SCD in EU

CTX001, an investigational gene-editing cell therapy, has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of severe sickle cell disease (SCD). The announcement was made by CRISPR Therapeutics and Vertex Pharmaceuticals, which are jointly developing CTX001 for severe SCD and transfusion-dependent β-thalassaemia (TDT). PRIME designation is issued to candidate therapies with promising clinical data that show potential to treat diseases of …

AstraZeneca Resumes Final Stage Trials For Its Covid-19 vaccine After Initial Halt

AstraZeneca Plc and the University of Oxford have resumed the British clinical trials of its Covid-19 vaccine, after the initial suspension over concerns with a participant who fell ill. University of Oxford, in a statement, said that the UK regulator, Medicines Health Regulatory Authority (MHRA), had recommended a restart of the trials, after an independent …

The  »International Blood Diseases Symposium » Is Just Around The Corner

The Turkish Federation of Blood Diseases, Kan Hastalıkları Federasyonu  is joining forces with TIF for the organisation of the  »International Blood Diseases Virtual Symposium », which will be held virtually on September 26-27, 2020. A 5-member team of our Federation will take part in this important event, consisting of Dr Androulla Eleftheriou, TIF Executive Director, Mr …

TIF Has Joined the EASL Patient Organisations Community

TIF is constantly fortifying synergies with influential patient organisations for the benefit of individuals with thalassaemia and other Haemoglobin Disorders. Therefore, we are excited to announce that the Federation has recently joined one of the most dynamic hepatology communities in the world, the European Association for the Study of Liver (EASL) Patient Organisations Community. As a first activity, TIF …

Pfizer Has Reached Over 50% Enrollment for Coronavirus Vaccine Candidate Phase 3 Trial

One of the leading COVID-19 vaccine candidates currently in development is getting closer to a potential finish line. Pfizer said on Wednesday that the recruitment for the crucial U.S. phase 3 clinical trial of its BNT162b2 is more than 50% complete. The company is aiming to recruit 30,000 individuals for the trial. The news was reported by …

Malaysian Health Minister: The Country Needs More Geneticists to Detect Thalassaemia Early

Malaysia needs more geneticists in order to identify thalassaemia patients at an early stage, stated Health Minister Datuk Seri Dr Adham Baba, after launching the 2019 Malaysia Thalassaemia Registry (MTR) report at Tunku Azizah Hospital yesterday. ‘’Thalassaemia can actually be prevented, if we can detect the carriers at an early stage. If these carriers get …

Coronavirus Pandemic: How Close Are We to a Vaccine?

Researchers around the world are racing to develop a vaccine against Covid-19, with more than 140 candidate vaccines now tracked by the World Health Organization (WHO). Vaccines normally require years of testing and additional time to produce at scale, but scientists are hoping to develop a coronavirus vaccine within 12 to 18 months. Vaccines mimic the virus …

TIF Publishes its Annual Report for 2019

This Annual Report showcases the Federation’s intensive efforts that took place in 2019 with the aim to ensure equal access to quality healthcare for every patient with thalassaemia and haemoglobin disorders across the world. We invite readers to navigate through the numerous and diverse global activities TIF organised throughout the year, with a sharp focus …

Viral Hepatitis & Thalassaemia: Q&A with Prof. John Koskinas

On the occasion of today´s World Hepatitis Day and with the aim of informing and raising awareness among patients with thalassaemia – and not only – about viral hepatitis that continues to plague tens of millions of people across the world, Prof John Koskinas, Professor of Medicine and Hepatology, Department of Internal Medicine, Medical School …

Novartis sickle cell drug Adakveo put on path to EU approval

Novartis announced last Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending conditional marketing authorization of Adakveo® (crizanlizumab) for the prevention of recurrent vaso-occlusive crises (VOCs), or pain crises, in patients with sickle cell disease (SCD) aged 16 years and older. Adakveo can …

WORLD HEPATITIS DAY 2020: Towards a ”Hepatitis-free Future”

World Hepatitis Day is commemorated each year on 28 July to enhance awareness of viral hepatitis, an inflammation of the liver that causes a range of health problems, including liver cancer. There are five main strains of the hepatitis virus – A, B, C, D and E. Together, Hepatitis B and C are the most …

THOUGHTS ABOUT ME AND MY DAUGHTER by Melissa Basberber

´´Today I was asked to answer a survey and participate in an interview about how is it like to live with Beta Thalassaemia. It is so exciting and fun that my journey with the disease can reach out to people that are interested so they can read it! So, I completed the online survey that …

Oxford vaccine: Early Trials Show Promising Results that Suggest “Double Protection” from Coronavirus

The experimental vaccine being developed by AstraZeneca and Oxford University against the new coronavirus produced an immune response in early-stage clinical trials, data showed on Monday, preserving hopes it could be in use by the end of the year. This vaccine, called ChAdOx1 nCoV-19, has been described by the World Health Organization’s chief scientist as …

TIF.ACCESS: A Global TIF Initiative for the Promotion of the Availability & Accessibility of Innovative Therapies

The swift scientific progress made in the field of thalassaemia research has gained momentum with the market authorisations of both the Zynteglo Gene Therapy by the European Medicines Agency (EMA) and the Reblozyl drug by both the EMA and the US Food and Drug Administration (FDA) for patients with transfusion-dependent β-thalassemia (TDT). As the accessibility …

Early-Stage Trial Data on Oxford University COVID-19 Vaccine to be Published July 20

Early-stage human trial data on a vaccine being developed by Oxford University and AstraZeneca will be published on July 20, The Lancet medical journal announced yesterday. The potential vaccine is already in large-scale Phase III human trials to assess whether it can protect against COVID-19, but its developers have yet to report Phase I results which …

COVID-19 and Thalassaemia: A Position Statement of the Thalassaemia International Federation

Many patients with haemoglobinopathies, including thalassaemia and sickle cell disease, are at increased risk of developing severe complications from the coronavirus disease 2019 (COVID‐19). Although epidemiologic evidence concerning the novel coronavirus (SARS‐CoV‐2) infection in these patients is currently lacking, the COVID‐19 pandemic represents a significant challenge for haemoglobinopathy patients, their families and their attending physicians. …

TIF Postpones its International Conference on Thalassaemia to 2021

Being mindful of the COVID-19 situation and wanting to ensure participants’ safety, TIF has taken the decision to postpone its long-awaited ’15th International Conference on Thalassaemia & other Haemoglobinopathies‘ & the ’17th TIF Conference for Patients & Parents’ from November 2020 to 13-16 May, 2021! The venue of both aforementioned TIF Conferences will be Thessaloniki, Greece, a country where …

Coronavirus Disease among Persons with Sickle Cell Disease

Individuals with sickle cell disease (SCD) are a particularly vulnerable group of patients, with a higher risk of severe complications due to COVID-19 infection than the general public. A recent scientific article entitled ‘Coronavirus Disease among Persons with Sickle Cell Disease, United States, March 20–May 21, 2020‘, published in the Emerging Infectious Diseases Journal (EID) …

TIF Position Statement on the Use of DEHP Plasticisers in Medical Devices

The safety concerns linked to the use of DEHP plasticizers, a manufactured chemical common in blood bags and PVC intravenous fluid sets, has been explored – and even questioned – by many in recent years. ΤΙF has also been preoccupied for possible toxicity of multi-transfused patients with thalassaemia to DEHP, due to their prolonged exposure …

GBT Announces Plans to Seek Regulatory Approval for Oxbryta® (Voxelotor) to Treat Sickle Cell Patients in Europe

Global Blood Therapeutics (GBT) plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Oxbryta (Voxelotor) be approved for treating hemolytic anemia in patients with sickle cell disease (SCD) who are 12 or older. The company plans to submit the MAA by mid-2021. “Currently, there are no approved therapies in Europe to treat …

Dr Evangelia Yannaki on Gene Therapy _ Interview for Greek TV Station Mega Channel

Thalassaemia Prior and Consequent to COVID-19 Pandemic: The Perspective of Thalassaemia International Federation (TIF)

Patients with haemoglobin disorders, particularly β-thalassaemia or sickle cell disease (SCD) or combined forms, on account of their underlying disease pathology and associated (iron load mainly in the case of thalassaemia) co-morbidities, are defined as high-risk individuals, prone to develop more severe complications from SARS-COV-2. Despite the fact that epidemiological evidence concerning severe acute respiratory …

Advances in the Care & Cure of Thalassaemia: New Hopes & Challenges

TIF, in collaboration with Thalassemics India and Thalassemia Patients Advovacy Group – India, conducted on June 12, 2020 a Webinar on the novel treatments emerging for thalassaemia and the hopes and concerns of patients affected by the disease. The webinar was directed to audience (patients/parents) from the SEARO region.

IMR-687 Receives FDA Orphan Drug Designation for the Treatment of β-Τhalassaemia

IMARA Inc., a clinical-stage biopharmaceutical company operating in the haemoglobin disorders field, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for IMR-687 for the treatment of patients with beta-thalassaemia. The FDA had previously granted Orphan Drug Designation for IMR-687 for the treatment of patients with sickle cell disease (SCD). …

Gene Therapy & Thalassaemia

 Millions of people worldwide suffer from inherited non-malignant blood disorders like thalassaemia and sickle cell disease. The culprit: genetic defects in their blood stem cells.   One of the novel therapies for thalassaemia that has stood out recently has undoubtedly been the first and only, one-time, groundbreaking gene therapy for transfusion dependent β-thalassaemia ZYNTEGLO by …

25th EHA Annual Virtual Congress: TIF Brings you All the Latest Developments on Thalassaemia

TIF was excited to participate in the 25th European Hematology Association (EHA) Annual  Virtual Congress, held this year virtually on 11 – 21 June 2020. Dr Androulla Eleftheriou (TIF Executive Director) and Ms Lily Cannon (TIF Operations Manager) have attended the following satellite symposia on 11 June: MDS AND β-THALASSEMIA: MINIMIZING TRANSFUSIONS AND IRON OVERLOAD …

COVID-19 & Haemoglobin Disorders: A TIF Webinar with Patients from America

On June 1st, TIF representatives, Dr Androulla Eleftheriou and Dr Michael Angastiniotis and Mr Loris Brunetta, Board Member, had a fruitful discussion with patient advocates from the AMRO region about the pandemic’s situation reports and challenges related to individuals with thalassaemia in these areas.

COVID-19 & Haemoglobin Disorders: The Impact of Coronavirus on Sickle Cell Disease Patients

An Expert Panel of Six Medical Professionals from various countries, such as India, the UK, Italy and Saudi Arabia, shared their experiences with TIF on the impact of the COVID-19 pandemic on Sickle Cell Disease (SCD) patients. Among other issues, the increased danger for complications of the coronavirus in this group of patients, as well …

TIF Thal e-Course: Testimonials from Online Learning Thalassaemia Patients

See what patients from various countries across the world who successfully took TIF‘s Thal e-Course, the ultimate self-paced, interactive, online Εducational Course for individuals with thalassaemia and their families, have to say about their experience with the course in their own language! Well-informed patients are able to gain in-depth understanding of their condition and make purposeful decisions …

FDA Grants Orphan Drug Designation to Mitapivat for Treatment of Thalassaemia

Agios Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company’s first-in-class pyruvate kinase-R (PKR) activator Mitapivat for the treatment of patients with NTDT α- and β-thalassaemia. Mitapivat is an investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes. It …

Explaining Gene Therapy in thalassaemia (2020)

The second educational brochure from the TIF’s ‘Gene Therapy’ booklet series, providing an in-depth view of how this revolutionary therapy works in patients with β-thalassaemia major.

TIF Issues Position Statement in Honour of the World Blood Donor Day 2020

There is one thing that definitely makes us heroes: giving blood.  It is a life-saving gesture that any of us in good health should very seriously consider making. Every couple of seconds someone around the world needs blood. Blood transfusions save millions of lives each year, both in emergency and non-emergency situations, and based on …

Prevention of Inherited Diseases – The Example of β-Thalassaemia (2020)

An educational brochure explaining the inheritance patterns of β-thalassaemia and the choices available for an  »at-risk » couple to have a child without the disease.

JUST LANDED: The  »Prevention of Inherited Diseases – The Example of β-Thalassaemi » Educational Brochure by TIF

Prevention is the only measure that can drastically reduce the incidencies of severe haemoglobin disorders, such as β-thalassaemia. It is also what allowed countries like Greece, Italy and Cyprus to go from historically high rates of thalassaemia to a substantial decrease of thalassaemia major cases in the last decades. This TIF educational brochure focuses on the inheritance …

COVID-19 & Haemoglobin Disorders: Compilation of Information for Sickle Cell Disease (2020)

This document, prepared by the Thalassaemia International Federation , is a compilation of information exclusively for Sickle Cell Disease patients with regard to the coronavirus pandemic.

WORLD BLOOD DONOR DAY 2020: Give blood and make the world a healthier place

On 14 June 2020 WHO and all countries worldwide will celebrate World Blood Donor Day!   The campaign theme for this year’s World Blood Donor Day is “Safe blood saves lives” with the slogan “Give blood and make the world a healthier place”. The idea is to focus on the contribution an individual giver can …

IGNORANCE ISN’T BLISS by Jensen Chan

 »Growing up as an avid member of the The Boys’ Brigade, my younger brother, Neilsen, was physically active and never had any fitness or health issues. However, at age 16, he began to develop symptoms of anaemia, such as easy fatigue and shortness of breath during exercise. Our concerned mother took him for a blood …

”Why I decided to increase awareness about thalassaemia in Singapore”: The story of Jensen Chan

”I was shocked at the huge discrepancy between how common thalassaemia is and how few people actually know about it.” Jensen Chan, a University student from Singapore, explains how his brother, a thalassaemia carrier, inspired him to learn more about this blood disorder and start a campaign to increase public awareness and promote thalassaemia screening. …

FDA Approves New Formulation for Ferriprox (Deferiprone) Twice-a-Day Tablets

Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., today announced that the U.S. Food and Drug Administration (FDA) has approved Ferriprox® (deferiprone) twice-a-day tablets for the treatment of patients with transfusional iron overload due to thalassaemia syndromes, when current chelation therapy is inadequate. The new formulation of twice-a-day Ferriprox 1000 mg oral …

The Patients’ Perspective on the Accessibility & Affordability of Novel Therapies for Thalassaemia

9 patients with thalassaemia, members of TIF’s Patient Advocacy Group for Thalassaemia and Sickle Cell Disease (T-PAG), are discussing with the Thalassaemia International Federation the accessibility and affordability of the new and innovative Therapies for the disease, as well as the COVID-19 pandemic challenges in their respective countries. With the participation of: Mr Seymour Baghbanli, …

COVID-19 & Haemoglobin Disorders: A TIF Webinar with Patient Advocates from South East Asia

COVID-19 & Haemoglobin Disorders: A TIF Webinar with Patient Advocates from Αfrica & the Eastern Mediterranean

COVID-19 & Haemoglobin Disorders: A TIF Webinar with Patient Advocates from Europe

Patient Advocates from Greece, Germany, the UK, the Netherlands, Romania and other countries of the WHO European Region exchange thoughts and opinions with TIF about the daunting challenges and concerns the pandemic has brought about for thalassaemia and sickle cell disease patients in these areas.

TIF Newsletter – May 2020

COVID-19 & Haemoglobin Disorders: A Classification of Risk Groups & Other Considerations (2020)

A TIF-compiled document aiming to provide a division of risk levels for thalassaemia and SCD patients with regards to the ongoing COVID-19 pandemic.

WEEKLY UPDATE: Developing Vaccinations & Therapeutic Drugs for COVID-19

All the latest updates of TIF’s Vaccinations & Therapeutic Drugs Guide for COVID-19 gathered in one single document.

FDA Grants RMAT Designation to CTX-001™ for the Treatment of Severe TDT β-Thalassaemia & SCD

CRISPR Therapeutics and Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX-001, an investigational, autologous, Gene Editing hematopoietic stem cell therapy, for the treatment of severe Transfusion-Dependent β-Thalassaemia (TDT) and Sickle Cell Disease (SCD). As with the FDA’s breakthrough therapy designation, cell or gene therapies that receive RMAT designation may benefit from early interactions with the FDA and be …

TIF’s Latest Guide for the COVID-19 Pandemic is Going Public on the International Thalassaemia Day 2020

As the online celebrations for the International Thalassaemia Day 2020 are at their peak, TIF decided to publish today as a gift to its worldwide patient communities a new guide entitled ”COVID-19 & Haemoglobin Disorders: A Classification of Risk Groups & Other Considerations”. Patients with thalassaemia and sickle cell disease (SCD) are likely to be at increased …

Reblozyl (luspatercept-aamt) Receives Positive CHMP Opinion for the Treatment of Adults with β-Thalassaemia

On 30 April 2020, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product REBLOZYL, intended for the treatment of adults with beta-thalassaemia or transfusion-dependent anaemia associated with myelodysplastic syndromes (MDS). The applicant for this medicinal product is Celgene Europe BV. This CHMP recommendation will now be reviewed …

TIF’s Global Call for Solidarity & Cooperation in support of WHO

  TIF expresses major concerns over the announcement of the United States to suspend financial support for the World Health Organisation (WHO) until completion of a review of the organisation’s activities related to the COVID-19 pandemic. Being a Federation that represents the wolrdwide thalassaemia community and works in official relations with WHO since 1996, we are issuing …

COVID-19: TIF Releases Position Statement on the Inclusion of Thalassaemia & SCD Patients in the High-Risk Population

The SARS-CoV-2 infection presents particular challenges and great risks to patients with thalassaemia and sickle cell disease (SCD) and, if their appropriate clinical management as described in International Guidelines is not safeguarded in the context of national policies developed for fighting COVID-19, their health and quality of life will be tragically impacted while in addition …

TIF and EHA Explore How COVID-19 Is Affecting Thalassemia & SCD Patients

On 16 April, TIF and the European Hematology Association (EHA) successfully organised a highly interactive webinar entitled  »Thalassaemia/SCD and COVID-19: Possible risks and a proposal for a patient pathway during the pandemic », which registered more than 700 participants from all over the world! Experts from Italy & Cyprus, including TIF’s Executive Director Dr Androulla Eleftheriou, …

NOW ONLINE: TIF’s latest Informational Guide on Developing Vaccinations & Therapeutic Drugs for COVID-19

TIF has made an effort to put together a list of studies/clinical trials for treatments and vaccines about COVID-19, which is by no means exhaustive as this situation is ever-changing and research in the area is dramatically intensified. It must be noted that there are currently no FDA or EMA-approved or even recommended agents for …

Developing Vaccinations & Therapeutic Drugs for COVID-19 (2020)

TIF has compiled a list of studies/clinical trials for developing drugs and vaccines for the COVID-19 Pandemic, which is by no means exhaustive as research in this area is dramatically intensified.

Blood & COVID-19 (2020)

An informational guide about blood adequacy and safety during the COVID-19 Pandemic, compiled on the basis of official recommendations from internationally recognized bodies.

IMPORTANT ANNOUNCEMENT: TIF reschedules the International Thalassaemia Day 2020 Celebrations for the 8th of September due to coronavirus

  Given the unprecedented global public health crisis brought on by the COVID-19 pandemic, TIF announces that the celebrations of the International Thalassaemia Day 2020 on the 8th of May, including all the Federation’s activities that were initially programmed, are rescheduled for Monday, 8 September 2020. TIF strongly urges its members and the global thalassaemia patient …

TIF publishes new material on the COVID-10 Pandemic

TIF continues the efforts to address the questions and concerns of its global patients’ and healthcare professionals’ community about the COVID-19 Pandemic and, in doing so, it has issued 2 brand new informational documents. The TIF-proposed Haemoglobinopathy Patient Pathway for Day Care Centres, Units and Clinics treating patients with thalassaemia and sickle cell disease and …

A TIF-proposed Haemoglobinopathy Patient Pathway during the COVID-19 Pandemic (2020)

A  Haemoglobinopathy Patient Care Pathway suggesting changes to be made during the outbreak of COVID-19 in the admittance of patients to haemoglobinopathy day care centres /units /clinics.

A consortium of life sciences companies jointly commit expertise & assets to fight COVID-19 alongside the Bill & Melinda Gates Foundation

An unprecedented collaboration across government, academia, private sector, and philanthropy is taking place as a response to the coronavirus global crisis. Today, a consortium of life sciences companies announced an important collaboration to accelerate the development, manufacture, and delivery of vaccines, diagnostics, and treatments for COVID-19. The life sciences industry brings a range of assets, …

FDA and EMA Collaborate to Facilitate COVID-19 Vaccine Development

  Global regulators have published a report today presenting the outcomes of a workshop on COVID-19 vaccine development that was convened under the umbrella of the International Coalition of Medicines Regulatory Authorities (ICMRA). This report was the outcome of March 18th first global regulators meeting, chaired jointly by the the U.S Food and Drug Administration …

The changing epidemiology of the ageing thalassaemia populations: A new position statement by TIF

Therapeutic advances in β-thalassaemia have gradually led to a significant improvement in prognosis over the past few decades. As a result, patients living in areas where disease specific programmes offer access to modern therapies, experience a new era of prolonged survival that tends to reach that of the normal population. Clinical management of ageing thalassaemia …

The COVID-19 Pandemic & Haemoglobin Disorders (2020)

An informational leaflet about the new COVID-19 with a special focus to the challenges it presents for patients affected by thalassaemia and sickle cell disease (SCD).

Novartis To Donate Up To 130 Million Doses Of Hydroxychloroquine To Treat Covid-19

  On Friday, 20 March, Novartis announced its commitment to donate up to 130 million doses of generic hydroxychloroquine to support the global COVID-19 pandemic response. Hydroxychloroquine and a related drug, chloroquine, are currently under evaluation in clinical trials for the treatment of COVID-19. When supported for use in COVID-19 infected patients by regulatory authorities, …

TIF’s enriched Leaflet  »The COVID-19 Pandemic & Haemoglobin Disorders » is available today!

During these difficult and challenging times the coronavirus (COVID-19) pandemic is creating at a global level, individuals with underlying medical conditions, such as blood disorders, heart disease, chronic liver disease, endocrine disorders or patients with a compromised immune system, may face an increased risk of  COVID-19 infection, according to the US Centres for Disease Control …

A Useful Health & Nutrition Short Guide for the COVID-19 Pandemic by TIF

The coronavirus presents many uncertainties, and none of us can completely eliminate the risk of getting infected with COVID-19. Nevertheless, one thing we can do is eat as healthy as possible! In addition to the well-known personal hygiene and preventive measures against the new coronavirus (COVID-19), we can also follow some simple recommendations regarding our …

AABB’s Develops Toolkit with Information Regarding Blood Donation & the COVID-19 Outbreak

  As the new coronavirus continues to spread affecting an increasing number of countries around the globe and in response to FDA’s Important Information for Blood Establishments Regarding the Novel Coronavirus (COVID-19) Outbreak (FDA Communication), AABB has recently developed a useful toolkit with important information about coronavirus and blood donation, in which examples of optional documents …

A DECENT LIFE WITH THALASSAEMIA by Dimitri Azzopardi

  “My family is from Greece originally but I was born and grew up in Wales, a country which even today has very few thalassaemia patients. When I was born 38 years ago, nobody – including my family – had any idea what was in store. I was eventually diagnosed with beta thalassaemia major at …

FT-4202 Granted FDA Fast Track, Rare Pediatric Disease Designations for SCD

FT-4202, a potential disease-modifying oral treatment for sickle cell disease being developed by Forma Therapeutics, has been given fast track and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). A fast track status is meant to accelerate the development and review of the treatment, facilitating discussions with the FDA and enabling the therapy to qualify for priority review …

Rare Disease Day 2020: Abundance of events & vast participation are expected in this year’s RDD

  Observed every year on the last day of February and in the 29th of February in 2020, Rare Disease Day seeks to raise awareness among the general public, as well as policymakers, public authorities, industry representatives, scientists, and health professionals about the more than 7,000 rare disorders that affect as many as 400 million people worldwide …

FAiTh Society Pakistan organised a series of special events for patients with thalassaemia

FAiTh - Fight Against Thalassemia Patients & Parents Society and its President, Ms Ayesha Mehmood, prepared several special surprises for 17 of their members, patients suffering from thalassaemia, in January and February 2020, at the FAiTh offices in Karachi, Pakistan, where their birthday celebrations were held. Ms Mehmood stated that FAitH, not only seeks to …

Panos Englezos Prize 2020

The application period for the ‘Panos Englezos Prize 2020‘ has now closed

FDA grants Orphan Drug Designation for ARU-1801 for the Treatment of Sickle Cell Disease & β-thalassaemia

  An investigational gene therapy for Sickle Cell Disease (SCD) and β-thalassaemia, known as ARU-1801, has been given Orphan Drug Designation an orphan drug by the U.S. Food and Drug Administration (FDA), a status that helps to advance and support its development. The therapy is expected to increase functioning red blood cells through proprietary technology that inserts a …

Deferasirox Accord: A new generic of Exjade recommended for approval by the EMA

The Committee for Medicinal Products for Human Use (CHMP), the European Medicines Agency‘ (EMA) committee responsible for human medicines, has recently adopted a positive opinion recommending the granting of a marketing authorisation for the medicinal product Deferasirox Accord, by Accord Healthcare S.L.U.   Deferasirox Accord is a generic of Exjade, which has been authorised in …

Thalassaemia Month of Banyumas 2019 -« Banyumas Goes to Zero Growth of Thalassaemia 2023 »

Written by Dinar Faiza (edited)   The Indonesian Thalassemia Foundation of Banyumas and the Banyumas District Government organised and launched, from November 1 to November 30, 2019, a massive pilot project of Thalassaemia Prevention Movement 2019, called  »Thalassaemia Month of Banyumas ». Its theme was entitled « Banyumas Goes To Zero Growth of Thalassemia in 2023 ». The …

Thalassaemia from ‘A’ to ‘Z’: A comprehensive e-glossary for patients with thalassaemia (2019)

The “Thalassaemia from A to Z” electronic glossary is a comprehensive, easy to use, educational tool for thalassaemia, including definitions of all important concepts on the prevention, management, treatment, organ complications, and plenty of other aspects related to the disease.

Deferasirox Mylan: A new generic drug for the treatment of iron overload in β-thalassaemia major obtained approval by the EMA

  The European Medicines Agency’s (EMA) has recently granted marketing authorisation for the medicinal product  »Deferasirox Mylan », intended for the treatment of chronic iron overload due to blood transfusions in patients with β-thalassaemia major, non-transfusion-dependent thalassaemia syndromes and other anaemias. The manufacturer of this medicinal product is Mylan S.A.S.  »Deferasirox Mylan » will be available as 90 mg, 180 …

Gene Therapy in β-thalassaemia and other haemoglobin disorders (2019)

An educational leaflet aiming to strengthen the knowledge and understanding of patients with thalassaemia and other haemoglobin disorders across the world on most aspects relevant to gene therapy.

34 year old Victoria Gray will be the first American to get controversial gene-editing treatment for sickle cell disease

  Victoria Gray, 34-year-old mother-of-four, has volunteered to be the first American to have their genes edited with the controversial CRISPR technology to treat her SickleCell disease (SCD). For the first time, doctors in the U.S. have used the powerful gene-editing technique CRISPR to try to treat a patient with a genetic disorder. Gray volunteered for …

FIGHTING WITH HOPE by Bidhan Sarkar

Winner Story #3 – International Thalassaemia Day 2019    »I was born in a small village in Bangladesh. Four years later, one renowned child specialist diagnosed me as E-beta thalassaemia and gave a prognosis that my life span would be 15 to 20 years old. My parents at that time expected another child and soon …

Bone Marrow Transplantation in β-thalassaemia (2018)

An educational leaflet with important information for β-thalassaemia patients on hematopoietic stem cell transplantation (HSCT), more commonly known as bone marrow transplantation (BMT).

Médicaments et pharmacovigilance

En quoi consistent les médicaments innovants ? Les médicaments innovants sont également connus comme médicament princeps, de référence ou de marque. Les médicaments innovants : ont été soumis à trois phases, ou davantage, d'essais cliniques rigoureux pendant presque deux décennies, et sont autorisés par des agences réglementaires officielles (EMA, FDA) en vue de commercialisation et …

Alpha thalassaemia

Alpha thalassaemia (or α-thalassaemia) is a general term for a group of inherited blood disorders, characterised by a reduced production of the α-globin chains of the haemoglobin molecule, while the β-globin chains are normally produced. This means that there will be an accumulation of the β-(unpaired) globin chains, within the developing red cell. The production …

BLOOD RED by Dr. Namitha A Kumar (PhD) – Thalassemia Intermedia (38 years)

Winner Story #3 - International Thalassaemia Day 2017 If someone were to ask me which color defines you best? I would instantly say “blood red!” One might think why this color? Isn’t it a violent color? Doesn’t it remind of you blood spilt? You would change your mind if you ask a child/adult with Thalassemia …

STORY OF A BEAUTIFUL SOUL by Shaheen Rao

Winner Story #2  - International Thalassaemia Day 2018 It’s a universal truth all typical mothers, who lost their beloved ones, love to talk about or share memories or write something. I feel myself exclusively privileged that TIF has provided an opportunity to express my inner feeling for this forum. There are no proper words to …

Guidelines for the Management of Non-Transfusion Dependent Thalassaemias (2nd edition – 2017)

EDITORS OF THE 2nd EDITION Ali Taher Khaled Musallam Maria Domenica Cappellini

A Short Guide for the Management of Transfusion Dependent Thalassaemia (2017)

Farmakis, D. Angastiniotis, M. Eleftheriou, A.

Delegation Visit to Egypt – 12-16 September 2017

A TIF delegation visited Cairo, Egypt between September 12 and September 16, 2017. The delegation consisted of Dr Michael Angastiniotis, TIF Medical Advisor and Dr Dimitrios Farmakis, Cardiologist. The visit had two main objectives: Discuss with the stakeholders involved in thalassaemia patient care and prevention in Egypt and assess the current situation; Participate in the …

HOPE: MY CURE IS IN OTHER PEOPLE by Rojin Demirkiran (9 years old, Turkey)

About the author Her name is ROJİN DEMİRKIRAN and she is from Diyarbakır, Turkey. Rojin was born in 2008, she has 5 more siblings and she is a thalassaemia major patient. Rojin is currently in grade 5 and she has written a poem through which she wishes to spread awareness about life with thalassaemia and the hope for …

Sickle Cell Disease (2008)

Inati-Khoriaty, A

Guidelines for the Management of Transfusion Dependent Thalassaemia, 3rd Edition (2014)

Cappellini MD, Cohen A, Porter J, Taher A, Viprakasit V

Beta Thalassaemia, Alpha Thalassaemia and Sickle Cell Disease Educational Community booklet (2014)

Eleftheriou A, Angastiniotis M

Bouton retour en haut de la page