Thalassemia International Federation Research Articles

SAT545 Erythropoietic Thyroid Nodule in the Setting of Myelofibrosis

Abstract Disclosure: L.M. Vasquez: None. J.M. Bruder: None. M. Koops: None. J. Trejo: None. Background: Extramedullary hematopoiesis is frequently seen in non-osseous locations but not endocrine organs, this is a case of hematopoesis in a thyroid nodule. Clinical Case: A 79-year-old man with myelofibrosis diagnosed at age 66, CAD post CABG, atrial fibrillation on anticoagulation and HFrEF was referred to the endocrinology clinic due to enlarging left lobe thyroid nodule. He was diagnosed with a goiter at age 72 but denied compressive symptoms. He had no history of radiation to his neck or family history of thyroid cancer. On physical exam, the left thyroid lobe was visibly enlarged with slight right tracheal deviation without lymphadenopathy. TSH level was normal. Thyroid ultrasound demonstrated a 6.4 x 4.0 x 4.8 cm, complex cystic-solid, taller than wide nodule in the left lobe and a sub-centimeter cystic nodule in the right lobe. FNA showed hematopoietic elements, including maturing cells in three lineages of hematopoiesis. On repeat ultrasound 5 years later. The left nodule increased in size to 7.3 x 5.4 x 4.4 cm and highly suspicious (TR4 and TR3) by Ti-RADS criteria. Repeat FNA of the nodule showed many red blood cells with few hematopoietic cells, flow cytometry did not show any abnormal populations. No thyroid follicular cells were reported. Myelofibrosis was diagnosed by bone marrow biopsy many years prior. He had maintained a stable CBC without palpable spleen or hepatomegaly. Given this history without thyroid dysfunction or reported symptoms, surgery was not pursued. External beam radiation was a treatment option should the nodule cause symptoms. He continued to have mild anemia not requiring blood transfusions. He passed away 2 years later in hospice care after hospitalization for severe heart failure decompensation. Conclusion: Extramedullary hematopoiesis involving the thyroid is rare but has been reported(1). This type of extramedullary hematopoiesis. can be treated with surgery or low-dose radiation because of the radiosensitivity of hematopoietic tissue (2). This was a potential treatment modality if compressive symptoms arose, but they did not. This case illustrates a situation in which surgical intervention on highly suspicious nodules would not have been the optimal treatment choice. Reference: 1. Jan I., Weng M., Wang C., et al. Extramedullary hematopoiesis involving the thyroid: A rare cytologic finding in otherwise healthy patients and review of literature. Journal of the Formosan Medical Association. (2018) 117, 1108-1114. 2. Taher A, Vichinsky E, Musallam K et al., authors; Weatherall D, editor. Guidelines for the Management of Non-Transfusion Dependent Thalassaemia (NTDT) [Internet]. Nicosia (Cyprus): Thalassaemia International Federation; 2013. Chapter 11, EXTRAMEDULLARY HEMATOPOIESIS. Available from: https://www.ncbi.nlm.nih.gov/books/NBK190455/ Presentation Date: Saturday, June 17, 2023

Juggling between the Cost and Value of New Therapies: Does Science Still Serve Patient Needs?

Thalassaemia International Federation (TIF), representing the united voice of people with thalassaemia and their families globally, has been striving for more than three decades to empower research, by academic communities and industry, to focus on developing a safe and effective curative approach for thalassaemia. Such a cure would lead to new lives with equal opportunities and challenges, as for every other person not suffering from a severe chronic disease. A gene therapy product was finally authorised in May 2019 by the European Medicinal Agency, thus marking a milestone in the history of the disease. However, after this conditional authorization, everyone focused on numbers and opted for cost of illness and cost-effectiveness studies, inadmissibly ignoring patients’ voices and needs. The product was finally withdrawn from Europe, despite the fact that all implicated stakeholders, including governments, academia and industry always knew that an innovative and complex therapy would be expensive but always supported and fought for its development. In this article, TIF expresses its view on this issue, including some thoughts on how to address the high cost of innovative therapies.

2021 Guidelines For The Management of Transfusion Dependent Thalassaemia (TDT), 4th EditionCapelliniMD, FarmakisD, PorterJ and TaherA (Eds). Thalassaemia International Federation, 2021, ISBN‐13 978‐9963‐717‐18‐7.

British Journal of HaematologyVolume 200, Issue 4 p. 532-532 BOOK REVIEW 2021 Guidelines For The Management of Transfusion Dependent Thalassaemia (TDT), 4th Edition , MD Capellini, D Farmakis, J Porter and A Taher (Eds). Thalassaemia International Federation, 2021, ISBN-13 978-9963-717-18-7. Barbara Bain, Corresponding Author Barbara Bain b.bain@imperial.ac.uk orcid.org/0000-0003-3077-4579 Centre for Haematology, St Mary's Hospital Campus of Imperial College London, St. Mary's Hospital, London, UKSearch for more papers by this author Barbara Bain, Corresponding Author Barbara Bain b.bain@imperial.ac.uk orcid.org/0000-0003-3077-4579 Centre for Haematology, St Mary's Hospital Campus of Imperial College London, St. Mary's Hospital, London, UKSearch for more papers by this author First published: 16 January 2023 https://doi.org/10.1111/bjh.18567Read the full textAboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onFacebookTwitterLinkedInRedditWechat No abstract is available for this article. Volume200, Issue4February 2023Pages 532-532 RelatedInformation

TIF Standards for Haemoglobinopathy Reference Centres

Haemoglobin disorders are hereditary, lifelong and characterised by the need for multifaceted management. The question of quality in meeting standards of care that are likely to bring the best possible outcomes for patients is a necessary consideration. The concept of reference centres supporting peripheral treatment centres in a formal networking relationship is a response to the real needs of patients and a practical solution in public health terms. In this report, a team of advisors of Thalassaemia International Federation (TIF) attempts to suggest a set of standards for haemoglobinopathy reference centres, also based on the founding principles of TIF, aiming to act as a guideline for its member associations and professional collaborators. The standards described herein can form the basis of an accreditation process and also serve as a guide for those who would advocate for quality improvement for thalassaemia services.

The Outcomes of Patients with Haemoglobin Disorders in Cyprus: A Joined Report of the Thalassaemia International Federation and the Nicosia and Paphos Thalassaemia Centres (State Health Services Organisation)

Haemoglobinopathies, including thalassaemias and sickle-cell syndromes, are demanding, lifelong conditions that pose a significant burden to patients, families, and healthcare systems. Despite the therapeutic advances and the resulting improvements in prognosis accomplished in past decades, these patients still face important challenges, including suboptimal access to quality care in areas with developing economies, changing epidemiology due to massive migration flows, an evolving clinical spectrum due to ageing in well-treated patients, and limited access to novel high-cost therapies. We herein describe the organization of healthcare services for haemoglobinopathies in Cyprus—with particular focus on beta-thalassaemia, the most prevalent condition in this region—along with selected patient outcomes. This report aims at underscoring the fact that nationally funded and well-coordinated prevention and care programmes for chronic and complex conditions, such as haemoglobinopathies, with active involvement from patient organizations lead to effective disease control and excellent outcomes in survival, quality of life, social adaptation, and public health savings, and allow timely and effective responses to emerging crises, such as the COVID-19 pandemic. The Cyprus paradigm could therefore serve as a blueprint for the organization or adaptation of haemoglobinopathy programs in other countries since these disorders are still widely occurring.

Non-Transfusion-Dependent Thalassemia: A Panoramic Review.

Non-transfusion-dependent thalassemia (NTDT) has been considered less severe than its transfusion-dependent variants. The most common forms of NTDT include β-thalassemia intermedia, hemoglobin E/beta thalassemia, and hemoglobin H disease. Patients with NTDT develop several clinical complications, despite their regular transfusion independence. Ineffective erythropoiesis, iron overload, and hypercoagulability are pathophysiological factors that lead to morbidities in these patients. Therefore, an early and accurate diagnosis of NTDT is essential to ascertaining early interventions. Currently, several conventional management options are available, with guidelines suggested by the Thalassemia International Federation, and novel therapies are being developed in light of the advancement of the understanding of this disease. This review aimed to increase clinicians’ awareness of NTDT, from its basic medical definition and genetics to its pathophysiology. Specific complications to NTDT were reviewed, along with the risk factors for its development. The indications of different therapeutic options were outlined, and recent advancements were reviewed.

2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia.

Beta-thalassemia and particularly its transfusion-dependent form (TDT) is a demanding clinical condition, requiring life-long care and follow-up, ideally in specialized centers and by multidisciplinary teams of experts. Despite the significant progress in TDT diagnosis and treatment over the past decades that has dramatically improved patients’ prognosis, its management remains challenging. On one hand, diagnostic and therapeutic advances are not equally applied to all patients across the world, particularly in several high-prevalence eastern regions. On the other, healthcare systems in low-prevalence western countries that have recently received large numbers of migrant thalassemia patients, were not ready to address patients’ special needs. Thalassaemia International Federation (TIF), a global patient-driven umbrella federation with 232 member-associations in 62 countries, strives for equal access to quality care for all patients suffering from thalassemia or other hemoglobinopathies in every part of the world by promoting education, research, awareness, and advocacy. One of TIF’s main actions is the development and dissemination of clinical practice guidelines for the management of these patients. In 2021, the fourth edition of TIF’s guidelines for the management of TDT was published. The full text provides detailed information on the management of TDT patients and the clinical presentation, pathophysiology, diagnostic approach, and treatment of disease complications or other clinical entities that may occur in these patients, while also covering relevant psychosocial and organizational issues. The present document is a summary of the 2021 TIF guidelines for TDT that focuses mainly on clinical practice issues and recommendations.

An urgent need for improving thalassemia care due to the wide gap in current real-life practice and clinical practice guidelines

Based on Thalassemia International Federation clinical practice guidelines (CPG) for non-transfusion dependent and transfusion dependent thalassemia, several measures should be routinely implemented such as monitoring and surveillance of thalassemia related complications for early detection and proper clinical management. To evaluate the prevalence and the performance of routine surveillance for thalassemia related complications during 2 periods; before and after published CPGs (2012–2014 vs 2015–2017), data from 524 adult thalassemia patients attended at Siriraj hospital were compared among different treating physician groups; thalassemia, private hematology, and internal medicine clinics. Three most common complications were osteopenia/osteoporosis (69.8%), gallstones (67.6%) and abnormal vitamin D level (67.6%). Iron overload has been widely evaluated (93.1%) followed by liver function test (82.3%). However, the rate of evaluation for other complications were significantly reduced and < 25% of patients were evaluated in several complications. Comparing among clinics, the surveillance rate has increased significantly for several endocrine complications only in patients treated at thalassemia clinic but not in others. This study was the first study that evaluated real-world practical management of thalassemia patient in terms of complication surveillance. This different clinical practice has called for an immediate policy change to improve and standardize a care for thalassemia patients in Thailand.

Hemoglobin Disorders in Europe: A Systematic Effort of Identifying and Addressing Unmet Needs and Challenges by the Thalassemia International Federation

Hemoglobin disorders (thalassemia and sickle cell disease) are a group of hereditary anemias that today occur across the world. The recent population movement has led to a steady increase of carriers and patients in all countries of the European Union. Requiring complex monitoring and treatment and, as a consequence, well-organized and nationally coordinated, supported and funded services, these lifelong conditions are now visible to healthcare services in the EU. The purpose of this study is to provide an overview of the current situation pertaining to these disorders, as perceived by the patient/parent community that the Thalassemia International Federation (TIF) represents. The aim is to establish a comprehensive understanding of the situation and unmet needs faced by migrants with thalassemia. The implementation of activities by TIF in 2018–2020 to identify and address these challenges, paves the way to increased awareness, education and policy changes building on international expertise and knowledge that will enable the provision of state-of-art clinical management services thus guaranteeing an improved quality of life. A bird’s eye view of the prevalence of these disorders is presented contributing to the further understanding of challenges met by both patients and healthcare professionals in the receipt and provision of quality healthcare respectively.

COVID-19 and thalassaemia: A position statement of the Thalassaemia International Federation.

ObjectivesMany 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.MethodsThe present statement summarizes the key challenges concerning the management of haemoglobinopathies, with particular focus on patients with either transfusion‐dependent or non‐transfusion‐dependent thalassaemia, identifies the gaps in knowledge and suggests measures and strategies to deal with the pandemic, based on available evidence and expert opinions. Key areas covered include patients’ risk level, adaptation of haemoglobinopathy care, safety of blood transfusions, blood supply challenges, and lifestyle and nutritional considerations.ConclusionsThe proposed measures and strategies may be useful as a blueprint for other disorders which require regular hospital visits, as well as for the timely adaptation of patient care during similar future pandemics.

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 coronavirus disease-2019 (COVID-19). Despite the fact that epidemiological evidence concerning severe acute respiratory syndrome-coronavirus-2 (SARS-CoV-2) infection in these patients is currently limited across the world, it is expected that COVID-19 pandemic will have a very serious, negative impact on national economies, healthcare and social systems and consequently significant respective repercussions on the patients particularly chronic ones, and their families. Although this may be a temporary challenge in some countries of high HDI and robust health, public health and social infrastructures, this can be a long term challenge with serious to tragic consequences in countries particularly devoid of universally covered heath care systems. Thalassaemia International Federation (TIF) in this present paper summarises the key challenges as expressed by the patients, their families and involved health care professionals themselves prior and consequent to COVID-19 pandemic, describes its response during the pandemic and expresses its position in support of its global patient community.

The changing epidemiology of the ageing thalassaemia populations: A position statement of the Thalassaemia International Federation

Therapeutic advances in β-thalassaemia have gradually lead to a significant improvement in prognosis over the past few decades. As a result, patients living in areas where disease-specific programmes offering access to modern therapy are in place experience a new era of prolonged survival that tends to reach that of the normal population. This ageing thalassaemia population, however, faces a new spectrum of comorbidities resulting from increasing age that may jeopardise the advances in prognosis provided by current therapy and thus poses new challenges in diagnosis, monitoring and treatment. In this position paper of the Thalassaemia International Federation, we review the changing epidemiology and clinical spectrum of patients with β-thalassaemia and propose actions to be undertaken in order to address the emerging spectrum of comorbidities resulting from ageing.

Now I Am The Voice: "a journey of self-discovery through the eyes of a Thalassemic".

Now I Am The Voice: "a journey of self-discovery through the eyes of a Thalassemic".

TIF 2.0: The Thal e-Course and TIF Expert Patients’ Programme for Disease-Related Education and Self-Management Skills in Thalassaemia

In response to the fundamental shift that has been taking place in the way chronic diseases are perceived and managed and the increasingly established role of patients as equal partners in the management of their condition, the Thalassaemia International Federation (TIF) has undertaken the design and development of a comprehensive online Expert Patients’ Programme (EPP) for patients with thalassaemia. Focusing particularly on β-thalassaemia, the most severe form of thalassaemia, the goal of the programme is to develop patients’ disease-related knowledge and self-care skills and enable them to co-manage their disease in a meaningful partnership with their treating physicians. An important goal of this ecourse is to empower patients to advocate for the improvement of national treatment services in every affected country. The aim of this article is threefold: (1) Relate TIF’s EPP with the goals and outcomes of other EPPs, as they are made available in the literature. (2) Describe the rationale and distinguishing features of TIF’s EPP on the basis of learning theories of knowledge acquisition and attrition, and best practices from the scientific disciplines of Human Computer Interaction (HCI) and Technology-Assisted Learning (TEL). (3) Relay the objectives of TIF’s EPP and the intended international impact in relation to TIF’s mission.

Global Initiatives for Improving Quality Healthcare by the Thalassaemia International Federation

In today’s health care arena, a number of issues are being raised that have received more attention either from the health care consumers or the media [...]

An Urgent Need for Improving Thalassemia Care in Adult Patients Due to a Wide Gap on Current Real-Life Practice and Recommended Clinical Practice Guidelines

BackgroundThalassemia syndromes are the most common hereditary hemolytic anemia worldwide. Since 2013, Thalassemia International Federation (TIF) has launched new standard clinical practice guidelines (CPG) for non-transfusion dependent thalassemia (NTDT) and transfusion dependent thalassemia (TDT). Based on these guidelines, several measures should be routinely implemented such as monitoring and surveillance of thalassemia related complications in order to early detect such complications for a proper clinical management. At present, there is no data in Thailand to show that how physicians are treating thalassemia patients especially on early detection of all complications and whether their real life practice has changed to follow those of both guidelines.ObjectivesTo evaluate the performance of routine care, in terms of surveillance for thalassemia related complications and their prevalence in adult thalassemia patients during 2 periods; before and after 2 CPG were published.MethodsIn this retrospective study, we analyzed data from 3,233 adult thalassemia patients who were diagnosed and treated at our clinics at Department of Medicine, Siriraj hospital during 1994-2017. We divided them into 2 groups; those who have been treated and followed-up for 3 consecutive years during 2012-2014 and 2015-2017 (a period before and after the implement of CPG, respectively). Clinical data and laboratory results were collected. Complications were recorded including iron overload (IOL) (ferritin), transaminitis (ALT >3x upper normal limit), diabetes mellitus (DM) or impaired fasting glucose (IFG)(fasting blood sugar), hypothyroidism (thyroid function test), low morning cortisol, vitamin D abnormality, viral infection (hepatitis B and C), osteopenia/osteoporosis (bone mineral density, BMD) and gallstones (ultrasonography or CT scan). To compare the performance of routine care from different treating physician groups, patients were categorized into 3 groups; those who attended thalassemia clinic (Thal) treated by mainly staffs and residents in hematology, those in private hematology clinic (Private) treated by mainly attending hematology staffs and residents in hematology, and those in internal medicine clinic (Non-hem) treated by internists and GP. Prevalence and surveillance rates for each complication between groups were analyzed by Independent t test (Chi-square or Fisher's exact test). This study was approved by local ethical committee.ResultsTotal available 459 NTDT and 65 TDT adult patients were studied. Baseline characteristics were shown in Table 1. Three most common complications were osteopenia/osteoporosis (69.8%), gallstones (67.6%) and abnormal vitamin D level (67.6%). IOL is a complication that has been widely evaluated in all treatment groups (93.1%) followed by evaluation of liver function test (82.3%). However the rate of evaluation for other complications were significantly reduced and <25% of patients were evaluated in several complications. This result suggests that the prevalence of thalassemia related complications might be underestimate the true prevalence and early detection of thalassemia related complications is still lacking.To test whether the implement of CPG has any impact on real-life clinical practice, we found that the rate of complication surveillance has increased significantly for several endocrine complications (DM/IFG, hypothyroid, adrenal insufficiency and low vitamin D) only in the group of patients treated at thalassemia clinic but not in others (Figure1). This suggested that CPG have yet to be implemented by those physicians and further endorsement is highly required.ConclusionThis study was the first study that evaluated the real-world practical management of thalassemia patient in terms of complication surveillance. In our adult thalassemia population, thalassemia related complications were not uncommon and some occurred early in adulthood. Surveillance rates of these complications were low in all clinics. After implement of thalassemia CPG, the surveillance rates for complications were increased only in patients treated at thalassemia clinic but not in other two clinics. A two different standard of clinical practice even within the same tertiary care hospital such as Siriraj hospital has called for an immediate policy change to improve and standardize a care for thalassemia patients in Thailand. [Display omitted] DisclosuresNo relevant conflicts of interest to declare.

Inadequacy of Ferritin Trends for Predicting Changes in LIC Risk Category in Transfusion Depedent and Well Chelated Patients with Haemoglobinopathies

Introduction. Chronic iron overload may represent a serious complication of potentially lifesaving blood transfusions in different haematological diseases. Excess iron deposits in various tissues of the body, particularly the liver, heart, and endocrine organs (Cappellini. Thalassaemia International Federation, 2014). This process leads to tissue damage and ultimately to significant morbidity and mortality (Musallam. Haematologica 2013). Indeed, organ failure due to chronic iron overload may represent the major cause of death in patients with different haematological diseases who receive blood transfusions regularly without appropriate chelation therapy (Inati. Pediatr Blood Cancer 2011; N Engl J Med 2000; Cleve Clin J Med 2005). The main aim of this study was to look for the relationships between changes in LIC and changes in serum ferritin (SF) level, during real life experience with larger setting of patients with haematological diseases and different chelation regimens, previously described at baseline, according to the LICNET protocol (Vitrano et al., Eur J Haematol 2016).Methods. This was a cross-sectional study of patients with haematological disorders attending 9 Italian centres participating in the LICNET. The LICNET protocol was approved on December 4, 2012 by our Ethical Committee. The underlying diagnoses were regularly transfused Thalassemia Major (TM), Thalassemia Intermedia (TI), Sickle Cell Disease (SCD), Myelodysplastic Syndrome (MDS), Diamond-Blackfan Anemia (DBA). Transfused status was defined as receipt of ≥7 mL/kg/month of packed red blood cells. The inclusion criteria for the cross-sectional analysis were: 1) underlying diagnosis above described; 2) determination of two R2-MRI scans performed as part of the network, for those patients presenting between February 2013 and December 2016; 3) transfusion dependence; 4) same chelation treatment at T0 (MRI1) and T1 (MRI2) . The settled R2-MRI protocol at the Centre follows that of St Pierre et al. (St Pierre et al. Magn Reson Med 2014). Descriptive analysis was provided as means, standard deviations, medians or percentages. Three classes of risk (low, intermediate and high), on the basis of LIC values (<7mg Fe/g dry weight (dw), 7-15 mg Fe/g dw, >15 mg Fe/g dw ) were considered to evaluate the control of iron body burden during the study period. LIC comparisons at MRI1 and MRI2 were made using t -test and/or Wilcoxon test. All p -values are two sided with the level of significance set at <0.05.Results. A total of 130 patients were evaluated, with a median age of 35 years (range: 6-78). The median duration (range) between MRI1 and MRI2 days was 483 (184-1076). Patients' characteristics for the considered chelation regimens are summarized in Table 1. Overall patients, across all chelation regimens, showed a statistically significant difference in variation of LIC between MRI1 and MRI2 (p=0.011, Table 2). Overall variation of LIC, during a period of 483 (184-1076) days, was -0.8 (-29.0-33.0) mg Fe/g dw. Median changes in LIC (range), mg Fe/g dw for single chelation regimen are reported on Tab. 2. Changes in LIC determinations, during the period of the study, according to the baseline values, are shown on Fig. 1: Overall 7.7% of patients, across different chelation regimens and during a period of 483 (184-1076) days, moved from high risk group (LIC >15 mg Fe/g dw) to intermediate risk group (LIC 7-15 mg Fe/g dw) with stabilization of iron overloading in patients in low risk group at baseline; all chelation regimens were able to move LIC from high risk group to intermediate risk group. In the other combination group, the patients moved from the intermediate risk group to the low risk group. Median changes in SF level (range), ng/ml for overall patients and for single chelation regimen are reported on Tab. 2: Overall patients, across all chelation regimens, did not show any statistically significant difference in variation of SF between MRI1 and MRI2 (p= 0.566, Table 2).Conclusions. In conclusion, SF level trends are unable to predict changes in LIC, even in well chelated patients with haematological disorders. Therefore, variations of SF level must be interpreted with caution and confirmed, when it is possible, by direct measurement of LIC for more correct management of chelation treatment. [Display omitted] DisclosuresOliva:Celgene: Consultancy; Amgen Inc.: Consultancy; La Jolla: Consultancy; Novartis: Consultancy; Janssen: Consultancy.

Thalassemia: Yesterday, Today, Tomorrow.

Thalassemia: Yesterday, Today, Tomorrow.

The Thalassemia International Federation: A Global Public Health Paradigm

Many international organizations are struggling today to coordinate limited economic and human resources in support of governments’ efforts to advance public health around the world. The United Nations and the World Health Organization, along with others play a pivotal role in this global effort. Furthermore, during the past few decades an increasingly higher percentage of global efforts on public health are carried out by specific health initiatives, international projects and non-governmental patient-oriented organizations. The Thalassemia International Federation (TIF) is one such organization focusing on the control of thalassemia around the world. The current paper aims at presenting a comprehensive overview of the mission, goals, objectives and activities of this organization. Our ultimate goal is to highlight TIF’s public health paradigm and diffuse its success at an international levels for others to follow. TIF is devoted to disseminating information, knowledge, experience and best practices around the world to empower patients with thalassemia and their relatives, support health professionals providing care to such patients and promote national and international policies, which secure equal access to quality care for all patients with thalassemia.

The Thalassemia International Federation: a global public health paradigm

The Thalassemia International Federation: a global public health paradigm