The beta thalassemias are common inherited blood disorders characterized by ineffective erythropoiesis, often leading to severe anemia. If left untreated, serious complications, such as heart failure, bone malformations, and hepatosplenomegaly, can occur. Hence, there is a demand for safe and effective treatment strategies.1
In a review article published in Blood Reviews, Farrukh T Shah, MD, of the Whittington Health NHS Trust in the United Kingdom, and colleagues summarized current literature surrounding the challenges of chronic blood transfusions in patients with beta thalassemia. In addition, they reviewed current unmet needs in the delivery of blood transfusion services around the globe.
Classification of Beta Thalassemia
Beta thalassemia is classified into three categories based on patient-specific genetic and clinical characteristics: minor (carriers), intermedia, and major. Lifelong blood transfusions are a necessity in patients with beta thalassemia major and usually begin in early childhood. Without effective blood transfusion services, mortality rates are high, particularly in the first and second decades of life.
The transfusion requirements for beta thalassemia intermedia can vary depending on the specific needs of the patient. In some cases, periodic transfusions may be required, while in others, no transfusions are necessary, but these requirements may change later in life. Along these lines, beta thalassemia can be classified as either transfusion-dependent or nontransfusion-dependent.
Global Transfusion Burden
At present, there remains a large degree of disparity in blood donation levels worldwide. Australia, Europe, and North America are the regions with the highest donation rates, while Southeast Asia and Africa have the lowest. In recent years, there has been a rise in donation rates, but the demand for transfusions in low-income countries exceeds the current supply. Around the globe, more than 22,500 patients with transfusion-dependent beta thalassemia die each year due to insufficient blood supply. Because of this, service providers are often required to seek out alternative blood sources, such as paid donors or family members.
In regions where beta thalassemia is endemic, successful strategies have been enacted to improve transfusion services. For instance, the National Thalassemia Center in the Maldives offers a blood bank exclusive to patients with thalassemia, with the majority of donations provided by family members. Public educational campaigns, created with the goal of expanding unpaid donorship, have also demonstrated success. Other strategies include changing national policies to improve practices and provide fresher blood to patients with beta thalassemia.