Patients with TDT require lifelong supportive care with regular red blood cell transfusions. Transfusion and iron chelation therapy have significantly improved the survival of TDT patients over the last few decades.1,2,3
Patients with transfusion‑dependent beta‑thalassemia typically require transfusions every two to five weeks, with the goal, according to TIF guidelines, to correct anemia, suppress ineffective erythropoiesis, and enable survival.1
Current TIF guidelines for TDT recommend lifelong transfusions, typically every 2 to 5 weeks, in patients who meet the following criteria:1
MANY PATIENTS WITH TRANSFUSION-DEPENDENT BETA-THALASSEMIA (TDT) EXPERIENCE COMPLICATIONS AND ORGAN DAMAGE DUE TO UNDERLYING DISEASE AND IRON OVERLOAD.1
Maintaining a Pretransfusion Level of 9-10.5 g/dL1
In patients with transfusion-dependent beta-thalassemia, red blood cell transfusions are the main driver for iron overload, which can subsequently lead to multi-organ damage.1,2
In iron overload, transferrin becomes saturated, and iron that is not bound to transferrin (non-transferrin bound iron, or NTBI) accumulates in the plasma. This free iron is highly reactive and generates harmful free radicals, which can damage lipid membranes, organelles, and DNA, causing cell death and fibrosis. The distribution of NTBI and the pattern of tissue iron uptake determine the pattern of organ damage, with myocardial muscle, endocrine tissue, and hepatocytes taking up NTBI rapidly.1
Adapted from Guidelines for the Management of Transfusion Dependent Thalassaemia (TDT).
3rd ed. Thalassaemia International Federation. 2014.
Transfusions temporarily relieve symptoms of anemia, but do not correct the underlying globin chain imbalance or restore normal erythropoiesis. They also introduce excess iron into the body, necessitating iron chelation therapy.1,4,6,7
Well treated thalassaemia will lead the patient beyond childhood, to an age where there is multiple organ involvement. The consequences, mainly of iron overload, cannot be totally prevented even by present day iron chelation treatment.
—Thalassaemia International Federation GuidelinesLearn how lifelong transfusion therapy may impact quality of life for patients with beta‑thalassemia
The management of iron overload is centered around1
Iron monitoring is key to determining the extent of iron overload and establishing an effective iron chelation regimen in accordance with a patient’s individual needs. Current TIF guidelines recommend several methods to monitor iron levels:1,2,8,9
Survival rates and quality of life in patients with beta-thalassemia have drastically improved with iron chelation therapy. However, complications still remain:
While advances in the last two decades have dramatically reduced the frequency of cardiac complications, the risk of heart failure, left ventricular dysfunction, and arrhythmias remains.1,9
Iron deposition in the liver can lead to increased ALT and AST, fibrosis, and cirrhosis.1,9 Cirrhosis predisposes patients to a higher risk of hepatocellular carcinoma.1,2 Hepatic disease is becoming a leading cause of mortality as cardiac-related mortality declines due to advances in monitoring and chelation treatment.
These are common and may be difficult to manage. They include hypogonadism (50-60% of patients with thalassemia major); diabetes (14% of transfused thalassemia major patients in North America); hypothyroidism (about 8-10% overall prevalence); hypoparathyroidism; and calcium metabolism abnormalities independent of hypothyroidism, like hypercalciuria (up to 50%) and nephrolithiasis (about 10%).2
Other complications include pain, growth failure, and bone disease.2
While effective iron chelation therapy has dramatically improved survival and quality of life in patients with beta‑thalassemia,13,14 complications can still occur, including in the cardiovascular, hepatobiliary, and endocrine systems.12
While chronic transfusion therapy addresses Hb levels transiently, this addresses only the symptoms of TDT. By correcting the genetic defect, there’s potential to eliminate the need for chronic transfusions and reduce the risk of long-term complications.1,2,3
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Consider Asking Your Patients:
"In what ways do RBC transfusions and iron chelation therapy affect your lifestyle today?"
Actor portrayals throughout. Not real patients.
Take the Beta-Thalassemia Challenge
Patients with transfusion‑dependent beta‑thalassemia (TDT) require lifelong supportive care with regular red blood cell transfusions—typically given every two to five weeks. Which of the following is not true about transfusion therapy?
Chronic transfusion and chelation therapy can help bring hemoglobin levels within the normal range and have improved survival in patients with beta‑thalassemia. However, many patients with TDT experience complications and organ damage due to underlying disease and iron overload, including cirrhosis of the liver.1 Transfusion therapy is life‑long and does not correct the genetic basis of TDT.1,2 Patients with TDT may experience various symptoms of anemia as hemoglobin levels wane between transfusions.1,5