Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia

28 Jan 2025

5th Edition, 2025
GUIDELINES FOR THE MANAGEMENT OF TRANSFUSION-DEPENDENT β-THALASSAEMIA (TDT)
Publishers: THALASSAEMIA INTERNATIONAL FEDERATION (TIF)
Editors: Taher, A.T., Farmakis, D., Porter, J.B., Cappellini, M.D., Musallam, K.M.

FORWARD

Dear Colleagues,

On behalf of the Board of Directors of the Thalassaemia International Federation (TIF) and the Editors of this 5th Edition of the ‘Guidelines for The Management of Transfusion-Dependent βThalassaemia (TDT)’, it is both an honour and a privilege to present this introductory foreword.
We are fortunate to live in an era where haemoglobin disorders, once considered fatal childhood afflictions, are now preventable, manageable, and even curable. This remarkable transformation has shifted the narrative of thalassaemia from despair to hope, ushering in an age of unprecedented scientific and medical advancements. Compelling evidence, including increased survival rates, decreased mortality and morbidity, and improved quality of life and social integration, unequivocally illustrates that the battle against thalassaemia is nearing victory. The advent of groundbreaking therapies in the past decade – including two gene therapies and one disease-modifying treatment approved for clinical use – alongside numerous others in various stages of development, heralds a brighter future for patients worldwide.
Regrettably, these advancements remain largely confined to nations with robust economies, where comprehensive health and social care systems provide universal access to treatment. In stark contrast, the vast majority of patients – estimated by TIF to be less than 10% of the global patient population – are unable to benefit from these developments. This disparity underscores the urgent need to empower countries, particularly those with high disease prevalence, to prioritise diseasespecific policies for prevention and care. The World Health Organization (WHO), through and not confined to its resolutions on disease management, blood safety, patient safety, congenital anomalies and birth defects, human genomics in global health, and access to essential medicines and health products, offers a robust framework for national efforts. Similarly, the European Union (EU)’s exemplary initiatives on rare diseases since the 1990s provide invaluable guidance that transcends regional boundaries. These initiatives provide a foundation and model for other countries beyond the EU to build upon,
highlighting their significant benefits – first and foremost for patients as well as for the sustainability and resilience of health and social care systems. It is our hope that, as nations strive to achieve the United Nations’ 2030 Sustainable Development Goals, meaningful progress will be realised, albeit unevenly and with great inequalities, across the globe.
TIF remains steadfast in its commitment to supporting national patient and parent organisations, the healthcare professionals’ community, and policymakers. The preparation, publication, translation, and free distribution of Guidelines such as this one is a cornerstone of our educational
mission. Widely recognised for their significant impact on the care of patients with these disorders, these Guidelines serve as critical resources for healthcare providers and a foundation upon which national policies and practices can be built.
We are profoundly grateful for the tireless efforts of national thalassaemia associations across the world – members of TIF – whose voluntary contributions have complemented and, in some cases, exceeded governmental initiatives. We humbly urge governments worldwide to lend their support in addressing the many and multiple unmet needs of patients with these disorders, especially those in underdeveloped regions. This is particularly crucial given the immense geopolitical, environmental, and public health challenges that the world is facing today, which severely threaten health and social care systems and disproportionately affect patients with such disorders, who rely
daily on highly specialised health and social services to manage their lifelong dependencies. The role of patient advocates in shaping national, regional, and international strategies cannot be overstated. Since its inception in 1986, TIF has been fighting for the inclusion of patients in decisionmaking processes. This 5th Edition of the TDT Guidelines proudly introduces a new chapter dedicated to the value of patient engagement, emphasising the transformative power of empowering patients as informed and effective advocates for their needs. Additionally, patients have contributed to the review of various other chapters in this book, ensuring that issues such as lifestyle and mental health are comprehensively addressed through the understanding and recognition of the patients’ perspective. TIF remains committed to continuing and expanding its support for the patients it represents, through strengthening its active and meaningful collaboration with national associations, decision-making bodies, healthcare professionals, industry leaders, and other key stakeholders.
We strongly encourage healthcare professionals who study these Guidelines to advocate for their adoption and implementation within their centres and countries by supporting and guiding national competent health authorities in their country about their immense value. Evidence-based practices outlined in these Guidelines are vital for enabling early diagnosis, effective management, and timely intervention. They also serve as a roadmap for integrating novel therapies that are under development or already approved into care frameworks, ensuring that all TDT patients, regardless of location, culture, religion, and language have access to optimal standards of care – a basic and
fundamental human right.

This updated 5th Edition features seventeen meticulously crafted chapters, authored by leading experts with decades of experience in TDT management. It offers insights into cutting-edge therapies and highlights considerations for care in resource-limited settings, providing a lifeline for underserved regions. In closing, allow us to extend our heartfelt gratitude to the key Editors and each and every Chapter Author, whose unwavering dedication and expertise in their respective fields have been instrumental in advancing the field of thalassaemia care and in making possible the development and completion of this upgraded edition of Guidelines. This publication, and indeed most of TIF’s educational endeavours, stand as a testament to their exceptional commitment.

On behalf of the Thalassaemia International Federation
Dr. Panos Englezos, hon. phD President, TIF
Dr. Androulla Eleftheriou BSC, MSc, PhD Executive Director, TIF
On behalf of the Editors prof. Maria Domenica Cappellini, MD, FRCp, FACp Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy

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