Thalassemia is a hereditary hemolytic disease characterized by abnormal hemoglobin (Hb) production; the severity of the disease is based on the specific globin chain mutation or deletion.  Blood transfusions can help to treat the symptoms of several forms of thalassemia, and many patients with thalassemia can expect to have normal life expectancies.

The severe forms of thalassemia are the most common inherited anemias managed with regular blood transfusion therapy. Transfusion policies and complications are critical to the quality of life and survival, but there is a lack of standardized care.

A survey of 58 items was completed in 2016 by 11 centers in California, Washington, Oregon, Nevada, and Arizona providing long-term care for thalassemia. The questionnaire addressed demographic information, transfusion practices and complications, and educational needs.

Although transfusion guidelines exist for patients with β-thalassemia, a recent survey conducted of 11 thalassemia treatment centers in California, Washington, Nevada, and Arizona found that over half of the patients had α-thalassemia (394/717, 55%), while 44% (314/717) had β-thalassemia (314/717). 

Almost 35% of patients were transfusion dependent, requiring 8 or more transfusions in the past year; 56% did not receive transfusions, and 9% were intermittently transfused.  Based on the survey, 83% of patients were Asian. 

Transfusion policies varied by the center, as did the prescribed Hb goal; seven centers transfused patients when Hb dropped below 7 g/dL. Almost 17% of patients were alloimmunized despite policies to match for Rh and Kell antigens; some centers extended matching to Kidd, Duffy, and S/s antigens. Iron overload was identified as the biggest challenge for treatment centers, followed by a lack of clear clinical practice guidelines.

The centers followed 717 patients with β-thalassemia (314, 43.8%) or α-thalassemia (394, 55%). One-third (34.7%) of patients were transfusion-dependent. Indications and goals of transfusion therapy differed between centers.

Prestorage leukoreduction was universal, while routine irradiation of units was limited to one site. Red blood cell antigen phenotype was determined before the first transfusion and patients received Rh/Kell-matched units.

However, more than half of the transfused patients had received blood at multiple hospitals within or outside the United States. Alloantibodies were seen in 16.9% of the transfused group, but the management of such patients was variable.

Unusual or emerging transfusion-transmitted pathogens were not observed. Multiple educational needs were recognized, with iron overload as the biggest challenge; the approach to iron chelation varied within the group.

This study identified many patients not included in earlier surveys limited to major national centers, suggesting that the thalassemia population in the United States is vastly underestimated. Lack of evidence-based guidelines is a barrier to optimal care, which should be addressed through regional consortia of thalassemia centers.