Transfusion practices and complications in thalassemia

Abstract

BACKGROUND: The severe forms of thalassemia are the most common inherited anemias managed with regular blood transfusion therapy. Transfusion policies and complications are critical to quality of life and survival, but there is a lack of standardized care. STUDY DESIGN AND METHODS: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. RESULTS:The centers followed 717 patients with β-thalassemia (314, 43.8%) or α-thalassemia (394, 55%). One-third (34.7%) of patients were transfusiondependent. 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 transfused group, but 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.CONCLUSION: 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.ABBREVIATIONS: LIC = liver iron concentration; NTD = non-transfusion dependent. From the Human subjects protectionCompletion of the surveys was acknowledged as a quality improvement activity exempt from the need for approval by institutional review boards. Each center provided only summary data and did not share patient-level information or identifiers. Statistical analysisResponses from all centers were summarized as tables and the proportion of individual responses were calculated.

Challenges in chronic transfusion for patients with thalassemia

“…The development of antibodies to red cell antigens is a significant threat to the long-term success of transfusion therapy. [38][39][40][41][42][43] In the United States, the proportion of patients with thalassemia with red cell alloimmunization is 17% to 22%, only slightly lower than sickle cell disease (19% to 31%). 38,44,45 Older age at initiation of transfusions and splenectomy are identified as major risk factors for developing alloimmunization in thalassemia.…”

“…47 In countries where thalassemia predominantly affects immigrant communities, the greater degree of donorrecipient red cell antigen mismatch can elevate the risk of alloimmunization, as shown in Table 2. 39,44,46,[48][49][50] In particular, the low frequency of Kell and c antigens in patients of Asian background facilitates development of alloimmunization. 39,46 One-half of the patients with 1 alloantibody will develop further antibodies against 1 or more additional antigens.…”

Thalassemia in the emergency department: special considerations for a rare disease

“…However, the presentation of immune-mediated hemolytic anemia may be acute or delayed up to 14 days. Alloimmunization is primarily caused by red blood cell (RBC) antigen incompatibility between recipient and donor [16,17]. Alloimmunization has been reported to be more prevalent in patients with thalassemia when compared to patients with hematologic malignancies and the general population [18,19].…”

Cost of chronic red blood cell transfusion in the Brazilian private healthcare sector from a payer perspective

“…Also, despite being acknowledged as a possible cost reference, the CBHPM is not officially adopted by the National Regulatory Agency for Private Health Insurance and Plans (ANS), and reimbursement values are still negotiated directly between health insurance/ health care providers. Nevertheless, inaccurate information regarding transfusion cost may compromise the adequate management of conditions requiring long-term blood transfusion, such as sickle cell disease (Detterich et al, 2015), thalassemia or MDS, which often involve transfusion dependence (Lal et al, 2018;Leitch et al, 2017).…”