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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

CDC | Many Children With Sickle Cell Anaemia In The US Not Receiving Lifesaving Screening And Treatment

According to a recent CDC study, fewer than half children with the disease in the US get the necessary screening, and only about half or fewer get treatment with hydroxyurea …

CLINICAL NEWS | PYRUKYND® Receives Positive CHMP Opinion For The Treatment of PK Deficiency in Adults

PYRUKYND® is a first-in-class, oral PK activator that was recently approved by the U.S. Food and Drug Administration (FDA). If approved by the European Commission (EC), PYRUKYND® will be the first approved disease-modifying therapy …

SAFER BLOOD | EU Patients Have Begun Receiving Transfusions With Hemanext ONE® RBC Processing & Storage System

Doctors have begun transfusing patients suffering from hematological malignancies using RBCs processed and stored with the Hemanext ONE® system, as part of a post-market clinical study in Bergen, Norway. This is …

WORLD PATIENT SAFETY DAY 2022 | Medication Without Harm: Know. Check. Ask.

 Each one of us, wherever we may live in the world, at some point in our lives, will take medications to prevent or treat illness. Medications sometimes, though, can provoke …

SICKLE CELL DISEASE | African Health Ministers Launch Drive to Curb SCD’s Toll

This effort, backed by the African Regional Office of the World Health Organization (WHO), aims to curb SCD΄s toll in a region that has historically been hit hard by this …

BREAKING NEWS | Bluebird’s Gene Therapy For β-Thalassaemia Wins US Approval

The green light is “monumental” for both patients and the company itself, Tom Klima, Bluebird’s Chief Commercial Officer, said in an interview. Bluebird has endured its share of “bumps and …

EHA2022 | Agios Presents New Data Supporting the Benefits of PYRUKYND®In Adults With PKD

Data were presented at the European Hematology Association (EHA) Annual Congress, hosted virtually and in person in Vienna on June 9-12, 2022. “Following the FDA approval of PYRUKYND® for the treatment …

The International Thalassaemia Day 2021 Official Page is Now Live!

INTERNATIONAL THALASSAEMIA DAY 2021 – 8 MAY  ”Addressing Health Inequalities Across the Global Thalassaemia Community” Visit the International Thalassaemia Day page, now LIVE on TIF website, to find out why …

Hemanext® Inc. Receives CE Mark Certification For Innovative Red Blood Cell (RBC) Processing & Storage System

Hemanext Inc. announced today it has received a CE Certificate of Conformity for the CE Mark for its Hemanext ONE Red Blood Cell (RBC) Processing and Storage System, allowing the …

Blood Donors’ COVID-19 Antibodies: Do They Have A Positive Significance for Blood Recipients?

According to recent data released by the American Red Cross, in the first week of March 2021 more than 20% of blood donations from unvaccinated people in the US had …

COMING UP: New TIF Webinar On ”COVID-19 Vaccinations & Patients With Haemoglobin Disorders”

TIF is delighted to inform you that a Special Webinar on ‘’COVID-19 Vaccinations & Patients with Haemoglobin Disorders’’ will take place on Wednesday, 31 March 2021, at 14:30-16:00 EEST/12:30-14:00 GMT. …

A Head-to-Head Comparison of BMT, Gene Therapy & Gene Editing Through A Brand New TIF Infographic

The expansion of management options and therapies for patients with thalassaemia is nowadays a reality, thanks to the application of new, revolutionary scientific procedures. However, patients’ understanding on the distinct …

TIF’s Point of View: Medicines Regulatory Systems – Continued Strengthening Is Required

Access to quality-assured medical products improves health and saves lives. Nevertheless, one third of the world’s population lacks timely access to quality-assured medicines while estimates indicate that at least 10% …

Out Now: The Latest Issue of TIF’s COVID-19 Vaccinations Update

Everything you need to know about the different COVID-19 vaccines, both already available and developing, in one place! Discover our newly updated Guide ”Vaccinations & Therapeutic Drugs for COVID-19”, containing a …

TIF’s Submission To The UK NICE’s Consultation Document For Zynteglo®

On February 12, in an appraisal consultation document, the UK National Institute for Health and Care Excellence (NICE) recommended against the use of Bluebird Bio’s Zynteglo (betibeglogene autotemcel) gene therapy for the …

Time To Get Ready For Rare Disease Day 2021!

TIF joins forces with 300 million people around the world for the global observance of Rare Disease Day, coming up on February 28th. Rare Disease Day is an annual awareness …

TIF’s Public Statement on Current Developments in Gene Therapy SCD Clinical Trials and Thalassaemia Gene Therapy Zynteglo

The Thalassaemia International Federation (TIF) has been informed through official communication by both the European Medicines Agency (EMA) and bluebird bio that the latter has placed Phase 1/2 and Phase …

EMA Accepts MAA For Oxbryta To Treat Haemolytic Anaemia In Sickle Cell Disease

The European Medicines Agency (EMA) has accepted Global Blood Therapeutics (GBT) marketing authorisation application (MAA) for Oxbryta® (voxelotor) for the treatment of haemolytic anaemia in sickle cell disease (SCD). According to the company, …

TIF Updated Vaccinations & Therapeutic Drugs COVID-19 Guide Has Landed

A comprehensive Guide for both existing and developing vaccines and treatments against COVID-19, exclusively developed by TIF for its global thalassaemia community. Featuring: Pfizer, Moderna, AstraZeneca: What are the differencies …

Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy

The U.S. Food & Drug Administration (FDA) has cleared an investigational new drug (IND) application for the experimental gene editing therapy GPH101 by Graphite Bio to initiate a Phase 1/2 …

Blood Supply Challenges & Transfusion Care of Patients With Haemoglobinopathies During COVID-19 Pandemic

The COVID-19 pandemic has had an impact on blood supply in several countries, jeopardizing the already demanding and often suboptimal care for haemoglobinopathies patients, mainly in developing and low-income countries …

EdiGene Announces Approval in China of its IND Application for CRISPR/Cas 9 Gene Editing Therapy ET-01 in β-thalassaemia

The Center for Drug Evaluation (CDE) of China‘s National Medical Products Administration (NMPA) has recently approved EdiGene’s application for ET-01, an investigational CRISPR-Cas9 gene editing therapy for patients with transfusion dependent β-thalassaemia. This marks the first …

Just Updated: TIF’s Vaccinations & Therapeutic Drugs for COVID-19 Guide

Everything you need to know about the different COVID-19 vaccines, both already available and developing, in one place! Discover our newly updated Guide ”Vaccinations & Therapeutic Drugs for COVID-19”, containing …

COVID-19 Vaccines and Haemoglobin Disorders: The Latest Position Statement of the Thalassaemia International Federation

In view of the plans made by national health authorities in every country across the world for the widespread vaccination of individuals against the SARS-CoV-2 virus, the Thalassaemia International Federation …

Leave No Patient Behind: A TIF Declaration For Access To Health At The Time of The COVID-19 Pandemic

Download the TIF Declaration HERE

TIF European Thalassaemia & Sickle Cell Disease Symposium To Take Place On 11-13 December 2020

TIF is proudly organising the European Thalassaemia & Sickle Cell Disease Symposium, a joint patients’ and healthcare professionals’ three-day educational event which will take place online on 11 – 13 December …

Virtual Conference on Alpha Thalassaemia Major By UCSF on 8-9 January 2021

The Center for Maternal-Fetal Precision Medicine (UCSF) will host a 2-day online international conference on Alpha Thalassaemia Major, which was once considered universally fatal in utero, on 8-9 January 2021.  …

PK Deficiency Resources Now Available On TIF’s Website

Recognizing the similarities of thalassaemia with other rare anaemias, particularly those affecting red blood cells, (e.g. PKD, SCD), and noting the absence of disease-specific united internationally coordinated patient organizations to …

Roche and Atea Pharmaceuticals Link Up To Co-Develop Remdesivir Rival Drug For COVID-19

The two companies will jointly develop, manufacture and distribute AT-527, Atea’s investigational oral antiviral for the potential treatment of COVID-19. According to Roche, AT-527 blocks the viral RNA polymerase enzyme …

What’s New In Clinical Trials for Thalassaemia and Sickle Cell Disease?

There is a lot of hustle and bustle in the scientific fields of thalassemia and sickle cell disease during the past few months, with many companies making remarkable progress in …

TIF Participates in EMA’s Annual Training Day 2020

TIF has participated in the Annual Training Day of the European Medicines Agency (EMA), held on 23 October 2020. TIF participants, Ms Lily Cannon (TIF Operations Manager) and Ms Eleni …

EU Approves Adakveo to Treat Vaso Occlusive Crises in Sickle Cell Disease Patients

The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent Vaso-Occlusive Crises (VOCs) in patients, 16 and older, with sickle cell disease (SCD). This approval follows a recommendation for conditional approval issued in July …

TIF At The 6th Annual CEETII Patient Advocacy Group Forum 2020

The Thalassaemia International Federation has participated in the 6th Annual CEETII Patient Advocacy Group Forum 2020 held on 3 November 2020, organised by Bristol Myers Squibb (BMS). The Forum brought …

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, …

Emerging Challenges & Future Therapies in Haemoglobinopathies The Focus of the ASCAT 2020 Conference

Mr Panos Englezos, TIF President, and Dr Androulla Eleftheriou, TIF Executive Director, represented the Thalassaemia International Federation at the opening day of the 15th Annual Sickle Cell and Thalassaemia (ASCAT) …

THAL e-COURSE: Iron Monitoring in Thalassaemia by Dr. Paul Telfer

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 …

EdiGene Application of CRISPR/Cas 9 Gene-editing Therapy ET-01 For β-Thalassaemia Has Been Accepted For Review

Τhe Chinese biotechnology company EdiGene announced yesterday that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has accepted for review the Company’s Investigational New Drug …

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 …

TIF At The NORBS Online Regional Conference For Rare Diseases – 23 0ctober 2020

Supporting Serbia and other Eastern European countries in their efforts to upgrade holistic care services for patients with rare diseases, including thalassemia, TIF’s Executive Director, Dr Androulla Eleftheriou, participated  on …

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 …

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, …

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 …

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 …

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 …

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 …

THAL e-COURSE: Liver Disease in Thalassaemia by Prof. John Koskinas

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. …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

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) …

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 …

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 …

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 …

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) …

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 …

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 …

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 …

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) …

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 …

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 …

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 …

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 …

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 – …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

TIF and Hemanext® Inc. Announce New Strategic Alliance

TIF, a worldwide organization dedicated to ensuring equal access to quality healthcare for every patient with thalassaemia and other haemoglobin disorders across the world, and Hemanext Inc., a privately held medical …

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 …

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. English French German Italian

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 …

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. English Bengali

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 …

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. English  

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 …

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, …

”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 …

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 …

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

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 …

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 …

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. English Greek  

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 …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

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. English Arabic Greek Italian  

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 …

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 …

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 daycare centres /units /clinics. English Greek  

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 …

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 …

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 …

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). English Αrabic Farsi French …

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 …

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, …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

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. English Bulgarian …

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 …

Oxbryta Increased Haemoglobin And Reduced Haemolysis in Adults and Adolescents With SCD

Global Blood Therapeutics, Inc. announced The Lancet Haematology has published the complete analysis of 72-week data from the Phase 3 HOPE Study of Oxbryta® (voxelotor) tablets in patients with sickle cell disease …

Take Action For The International Thalassaemia Day 2021

INTERNATIONAL THALASSAEMIA DAY 2021 – 8 MAY  ”Addressing Health Inequalities Across the Global Thalassaemia Community” Each year, individuals around the globe band together for one day of pure and empowering …

INTERNATIONAL THALASSAEMIA DAY | 8 May 2022

Every single one of us has the ability to make a difference, large or small, and together we can make real progress in raising valuable awareness about thalassaemia and its …

NEW WEBINAR | Exploring The Value Of Patient Advocacy For Better Thalassaemia Care

The objective of this meeting, which brings together eminent haemoglobinopathies specialists and expert patient advocates from Italy and other countries, is to highlight the pivotal value of advocacy in enabling …

IRON BOY: SURVIVING β-THALASSAEMIA MAJOR by Arthur Bozikas

Where was my wife? I found myself sitting alone in front of the obstetrician, who my wife, Helen, and I had met for the first time only minutes earlier. But …

TIF’s Statement on Accessibility of Gene Therapy for Thalassaemia

The Thalassaemia International Federation (TIF) expresses its disappointment about the outcome of the reimbursement negotiation process concerning gene therapy (Zynteglo™) for transfusion-dependent thalassaemia patients, announced last week. The Federation’s distress …

العقاقير وسلامتها

ما المقصود بالعقاقير المبتكرة؟ تُعرف الأدوية المبتكرة أيضًا باسم المخلق أو المرجع أو العلامة التجارية وهي عقاقير مرت بثلاث مراحل أو أكثر من التجارب الإكلينكية الصارمة على مدار عقدين من …

INTERNATIONAL THALASSAEMIA DAY | The Theme For 2022 Has Been Announced!

We are elated to announce that the theme for the International Thalassaemia Day (ITD) 2022 is “Be Aware. Share. Care: Working with the global community as one to improve thalassaemia …

RARE DISEASE DAY 2022 | Raising Awareness For People With Rare Diseases

Rare Disease Day is the movement on rare diseases, working towards equity in social opportunity, healthcare, and access to diagnosis and therapies for people living with a rare disease everywhere …

PYRUVATE KINASE DEFICIENCY | FDA Approves First Drug For The Rare Inherited Anemia

The US Food and Drug Administration (FDA) has announced the approval of Mitapivat (PYRUKYND®) tablets as treatment for haemolytic anemia in adults with pyruvate kinase (PK) deficiency. The oral PK …

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 …

SCD Drug Oxbryta Receives CHMP Positive Opinion For Approval In The EU

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for Oxbryta® (voxelotor) tablets for the treatment of hemolytic …

THALIA2018-2021 | Europe Is Seeing A Surge In Thalassaemia, Project Results Show

“Although thalassemia has always been endemic in Europe, especially in southern European countries such as Cyprus, Greece and Italy, the disease now knows no borders and constitutes an increasing health, …

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 …

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. …

TIF Article: COVID-19 Pandemic Exacerbated Existing Problems With Thalassaemia Care

The article discusses how the COVID-19 pandemic has magnified challenges in thalassaemia care worldwide and underscores TIF’s activities throughout the coronavirus outbreak in support of patients with haemoglobinopathies. Hematology Advisor …

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). English Bulgarian Farsi  

FDA Grants Priority Review to Luspatercept-aamt for Anemia in Adults With NTD β-Thalassaemia

The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2022. In addition, the European Medicines Agency (EMA) has validated the Type II variation …

TIF Signs Agreement With Resonance Health To Deploy FerriSmart®

FerriSmart is an AI-driven system for automated real-time measurement of liver-iron concentrate (LIC) in patients, using non-invasive MRI-based technology. Under the Letter of Agreement, TIF will engage with National Thalassaemia …

La Repubblica: Gene Editing Cured Two Siblings With Thalassaemia In Italy

Cured of thalassemia, thanks to gene editing. According to a recent news report published in the Italian newspaper La Repubblica, Erika and Emanuele Guarini, two siblings from Pistoia, Tuscany, aged …

Oxbryta Gets Marketing Authorisation In The UK For Patients With Sickle Cell Disease

Voxelotor, an oral treatment taken once daily, is the first medicine authorized in Great Britain that directly inhibits sickle haemoglobin (HbS) polymerization, the molecular basis of sickling and destruction of …

Guidelines for the Management of Non-Transfusion Dependent Thalassaemia (NTDT) (2nd Edition – 2017)

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

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

Farmakis, D. Angastiniotis, M. Eleftheriou, A. Based on: Cappellini, MD., Cohen, A., Porter, J., Taher, A., Viprakasit, V. (Eds.), Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT). Thalassaemia International Federation: TIF Publication …

TIF 2021: The World’s Largest Conference On Haemoglobin Disorders Is Back! 19-21 November 2021

The Thalassaemia International Federation is excited to announce the 15th International Conference on Thalassaemia & Haemoglobinopathies & the 17th TIF International Conference for Patients and Parents which will take place …

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 …

COMING UP: 1st Global Forum On Access To Safe Blood Products For Patients In Need Of Regular Transfusion – 5 November 2021

Expected Outcomes An understanding of the strategic vision and goals for blood safety and the protection of the blood supply, especially in low-income settings; A better understanding of the key …

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 …

THALASSAEMIA CHAMPIONS PODCAST | The 3rd And Final Episode Is Now Available

With open dialogue-formatted episodes, featuring thalassaemia patient advocates, mental health professionals, and other special guests, this show explores everything from psychosocial anxiety and relationship issues to treatment-related fears, while striving …

TIF Supports Administration Of COVID-19 Vaccine Booster Dose To Patients With Haemoglobin Disorders

 

Thalassemia Foundation of Canada to Host its National Thalassemia Conference on October 23-24

The Thalassemia Foundation of Canada, a long-time member of TIF, will be holding its 2-day Virtual National Conference on Thalassaemia this weekend, October 23-24, at 12:00-16:00 EDT. The Conference aims at …

POLICY NEWS | TIF Issues Statement On The New EU Regulation For Blood, Tissues And Cells

This single Regulation, once approved by Member States, will replace all existing EU Directives on Blood, Tissues and Cells and will be equally applicable in the EU territory. The proposal …

Sickle Cell Disease (2008)

Inati-Khoriaty, A English Arabic German  

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

Eleftheriou A, Angastiniotis M English Greek  

BLOOD TRANSFUSION | Hemanext Begins Rollout of Hemanext ONE® RBC Processing And Storage System In Europe

Physicians in Norway have initiated recruitment of chronically transfused patients with haematological malignancies, including myelodysplastic syndrome (MDS), to receive transfusions using RBCs processed and stored with the Hemanext ONE® system. …

EdiGene Announces First Patient Enrolment In Phase I Clinical Trial Of Gene-editing Therapy ET-01 For Patients With β-Thalassaemia

The Chinese biotechnology company EdiGene announced yesterday that the first patient was enrolled in the multicentre, Phase I clinical study of its investigational gene editing hematopoietic stem cell therapy ET-01 …

TIF Position Statement: Demanding Access To Existing And Upcoming Gene Therapies For β-Thalassaemia

The Thalassaemia International Federation (TIF) is expressing its grave disappointment over the recent announcement of Bluebird Bio regarding the “wind-down” of its operations in Europe and the exclusive disposal of …

PYRUVATE KINASE DEFICIENCY | TIF Issues Charter of Priorities For The Global PKD Patient Community

This newly launched document aspires to bring together available information about PKD in an effort to identify gaps, and to make useful imperative suggestions so as to understand what changes …

MITAPIVAT | FDA Grants Priority Review To New Drug For Pyruvate Kinase Deficiency

The NDA was granted a Priority Review designation and has been given a Prescription Drug User Fee Act (PDUFA) action date of February 17, 2022, accelerating the review time from …

TIF Presents The Federation’s Annual Report For 2020

“Our commitment to unite the patient voice and to advocate at the highest possible levels for equal access to all treatment options remains our number one priority.“ TIF President – …

CDC, FDA Authorize Third Dose Of COVID-19 Vaccines For The Immunocompromised In The US

The U.S. Food and Drug Administration (FDA) and the U.S. Centers for Disease Control and Prevention (CDC)  have both recommended on August 16 that certain immunocompromised people receive a third …

TIF e-Academy: Reimagining Thalassaemia and Sickle Cell Disease Education

Read. Learn. Empower. The Thalassaemia International Federation´s internationally acclaimed Educational Programme is a fundamental component of TIF´s work and is specifically designed for and addressed to the ever-growing learning needs …

NEW EVENT | Register Now For The 1st PIBA Members’ Meeting!

The Thalassaemia International Federation (TIF) firmly supports that joint and coordinated efforts are required to create positive changes in the field of blood, given the limited access of patients to …

Global Blood Submits US Application For Expanded Use Of SCD Drug, Oxbryta, In Patients Aged 4-11 Years

Children with sickle cell disease (SCD) as young as age 4 could be prescribed Oxbryta (Voxelotor) if the U.S. Food and Drug Administration (FDA) approves a supplemental new drug application (sNDA) submitted by …

Bluebird Bio Resumes Marketing Of Gene Therapy Zynteglo For β-Thalassaemia In Europe

The European Medicines Agency (EMA)’s safety committee (PRAC) has concluded that there is no evidence Zynteglo causes a blood cancer known as acute myeloid leukaemia (AML). Zynteglo, a gene therapy …

Clinical Trials Update: The June 2021 Edition

TIF provides you with comprehensive and up-to-date information on developing drugs and therapies for thalassaemia and sickle cell disease (SCD) currently in clinical trials. Our most recent June 2021 update …

The June Update: TIF’s Guide On COVID-19 Vaccinations & Therapeutic Drugs

As vaccination rates are slowing in the US and Europe, the spread of the Delta coronavirus variants causes growing concerns worldwide with health experts predicting the highly infectious variant to …

PYRUVATE KINASE DEFICIENCY | Agios Submits New Drug Application To FDA For Mitapivat

“With this NDA filing, we are poised to deliver the first potentially disease-modifying therapy for people with PK deficiency, a chronic, lifelong haemolytic anaemia characterized by serious complications affecting multiple …

TIF Position Statement: Addressing Blood Supply Issues Linked To COVID-19 Vaccination Deferral Times

TIF Position Statement - COVID-19 Vaccination and Blood Donation

New Paper From Blood and Beyond Outlines How To Learn From The Impact Of The COVID-19 Crisis On Blood Use

The multi-stakeholder Blood and Beyond initiative recently published a policy briefing shedding light on how the COVID-19 pandemic affects blood use, blood transfusion services, and patients in whom transfusions are …

UPDATE: COVID-19 Developing Vaccinations & Therapeutic Drugs

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

Ali Sattarpour: ”Thalassaemia did not stop me from living a normal life”

In occasion of the International Thalassaemia Day 2021, Ali Sattarpour, 45, France, Fédération SOS GLOBI Vice President and TIF’s Thalassaemia Patient Advocacy Group (T-PAG) Member, recounts a life of struggles, …

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