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للمرضي

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.

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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.

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Latest Posts for Patients

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 …

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 …

Gene Therapy & Thalassaemia Version 2.3: A TIF Survey

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 Bluebird …

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 …

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 …

TIF Launches Online Petition to Promote ”Equitable Access to Safe and Adequate Blood”

A few days before the celebration of the World Blood Donor Day 2020, TIF has launched an online petition, co-signed by the European Blood Alliance (EBA), to promote ”Equitable Access to Safe and Adequate Blood” and voluntary, non-remunerated Blood Donation! Transfusion of blood and blood products help save millions of lives everyday. This statement is …

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 …

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 …

”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. …

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 …

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 …

TIF ITD2020 Webinar: 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 …

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 …

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 …

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.

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 …

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 …

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.

TIF & EHA’s Full Webinar Video on Thalassaemia/SCD & COVID-19 Is Now Available Online!

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, inclufing 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 …

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 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 …

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 ...

‘FAiTh – Fight Against Thalassemia Association’ presents its activities for January 2020

  The ‘FAiTh – Fight Against Thalassemia’ group,  a longtime member of TIF operating in Pakistan, is dedicated to support, serve and love people with thalassaemia and their families, intending to create a general, massive awareness about the illness and motivate the public to further support thalassaemia patients. These are the group’s major activities for …

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 …

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 …

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 …

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 الزمن تقريبًا وهي عقاقيرمرخص لها من قبل الهيئات الرسمية المنشئة للتنظيم (إدارة الغذاء والدواء الفدرالية FDA, وكالة الأدوية الأوروبية (EMA، لتسويقها واستخدامها من قبل متخصصي ...

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 ...

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 ...

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 ...