blood cells blood cells close-up of man

Transfusion Therapy for TDT For patients with transfusion-dependent beta (β)-thalassemia (TDT), lifelong transfusions provide symptom management1

Treatment with RBC transfusions
Initiating RBC transfusions
Transfusion therapy management
Treatment with RBC transfusions
Initiating RBC transfusions
Transfusion therapy management

Lifelong supportive care with regular RBC transfusions is necessary in TDT1

Until effective therapy was developed, beta-thalassemia major was considered to be a pediatric condition: in the absence of a regular transfusion regimen, children with the disease usually died within their first few years of life.1


Red blood cell (RBC) transfusions, which are central to the treatment of severe disease, enable survival by helping to correct the anemia characteristic of beta-thalassemia and limit bone marrow expansion. However, many challenges, such as organ damage due to underlying disease and iron overload, still exist.1,2 

Transfusions address Hb levels temporarily1

Hemoglobin (Hb) level is measured as the amount of Hb in grams (g) per deciliter (dL) of whole blood, with a deciliter being 100 milliliters (mL). Normal Hb ranges are as follows3: 

Male

2-9 years 11.5-14.5 g/dL

2-9 years 11.5-14.5 g/dL

10-17 years 12.5-16.1 g/dL

10-17 years 12.5-16.1 g/dL

≥18 years 13.5-18 g/dL

Female

2-9 years 11.5-14.5 g/dL

2-9 years 11.5-14.5 g/dL

10-17 years 12.5-15 g/dL

10-17 years 12.5-15 g/dL

≥18 years 12.5-16 g/dL

The 2021 Guidelines for the Management of TDT, published by the Thalassaemia International Federation (TIF), recommend regular lifelong transfusions, typically every 2 to 5 weeks, in patients who meet the following criteria1:

  • Confirmed diagnosis of beta-thalassemia
  • Laboratory criteria
    • Hb level <7.0 g/dL on 2 occasions, >2 weeks apart (excluding all other contributory causes such as infections)

AND/OR

  • Confirmed diagnosis of beta-thalassemia
  • Laboratory criteria
    • Hb level <7.0 g/dL on 2 occasions,
      >2 weeks apart (excluding all other
      contributory causes such as infections)

AND/OR

  • Clinical criteria irrespective of hemoglobin level:
    • Significant symptoms of anemia
    • Poor growth/failure to thrive
    • Complications from excessive intramedullary hematopoiesis, such as pathological fracture and facial changes
    • Clinically significant extramedullary hematopoiesis

The Thalassaemia International Federation (TIF)1,4

Founded in 1987 by a small group of patients and parents, TIF is a nonprofit, nongovernmental organization dedicated to promoting appropriate clinical management of thalassemia in every affected country.4

Since 1996, TIF has had official relations with the World Health Organization (WHO). The outcome of this partnership is an extensive network of collaboration between scientific and medical professionals from more than 60 countries around the world. To further its mission, TIF has published important clinical documents for thalassemia, including guidelines for managing and caring for patients with both TDT and non–transfusion-dependent beta-thalassemia.1,4

The above content was used with the approval of TIF. bluebird bio does not endorse the nonprofit organization or any of its affiliates.

It is essential to maintain hemoglobin levels in patients with TDT1,5

The TIF guidelines recommend regular blood transfusions, usually administered every 2 to 5 weeks, to maintain a pretransfusion level of 9.5 to 10.5 g/dL, which has been shown to help1:

  • promote normal growth1
  • allow normal physical activities1
  • adequately suppress bone marrow activity1
  • improve anemia6
  • minimize transfusional iron accumulation and ineffective erythropoiesis1,6
  • avoid skeletal abnormalities, organomegaly, and growth retardation in children1,6

Because hemoglobin levels are higher post-transfusion, which increases the risk of stroke, post-transfusion hemoglobin target should be 13 to 15 g/dL.1

TIF Guidelines for Transfusion: Maintaining a Pretransfusion Level of 9.5 to 10.5 g/dL1*

 SWIPE OR ROTATE
Chart showing thalassemia blood transfusion guidelines
Chart showing thalassemia blood transfusion guidelines

*A higher target pretransfusion hemoglobin level of 11 to 12 g/dL may be appropriate for certain patients with heart disease or other comorbidities.1

Hemoglobin levels should be measured intermittently after transfusion to determine how quickly hemoglobin levels drop. This decrease may be valuable for evaluating the effects of changes in the transfusion regimen.1

Understanding your patient’s symptoms between transfusions may
help you optimize their treatment schedule.

As hemoglobin levels wane between transfusions, patients with TDT may experience various symptoms of anemia, such as1,6,7:

  • fatigue
  • weakness

Patients may also experience transfusion-associated complications, such as1:

  • transfusion reactions
  • alloimmunization
  • infections

Over the years, a greater understanding of the pathophysiology of beta-thalassemia has helped improve disease management and drive the investigation of future potential therapies.1 As an example, today adult patients with beta-thalassemia receiving regular transfusions may have the option of adding an erythroid maturation agent to their chronic transfusion schedule to help moderate their transfusions.1

Regular transfusions enable survival but only temporarily address the symptoms of TDT.1

Actor portrayal. Not a real patient.

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 2 to 5 weeks. Which of the following is not true about transfusion therapy?

Actor portrayal. Not a real patient.