Ashutosh Lal1, Trisha E Wong2, Jennifer Andrews3, Vinod V Balasa4, Jong H Chung5, Craig M Forester1,6, Alan K Ikeda7, Siobán B Keel8, Monica B Pagano9, Geetha Puthenveetil10, Sanjay J Shah11, Jennifer C Yu12, Elliott P Vichinsky1. 1. Hematology/Oncology, University of California San Francisco Benioff Children's Hospital, Oakland, California. 2. Pathology and Pediatric Hematology Oncology, Oregon Health and Science University, Portland, Oregon. 3. Pathology and Pediatric Hematology/Oncology, Stanford University, Palo Alto, California. 4. Pediatric Hematology/Oncology, Valley Children's Hospital, Madera, California. 5. Pediatric Hematology/Oncology, University of California at Davis, Sacramento, California. 6. Bone Marrow Transplantation, University of California San Francisco Benioff Children's Hospital, San Francisco, California. 7. Hematology/Oncology, Children's Specialty Center of Nevada, Las Vegas, Nevada. 8. Department of Laboratory Medicine, University of Washington, Seattle, Washington. 9. Division of Hematology, University of Washington, Seattle, Washington. 10. Hematology, Children's Hospital of Orange County, Orange, California. 11. Hematology/Oncology, Phoenix Children's Hospital, Phoenix, Arizona. 12. Pediatric Hematology/Oncology, University of California at San Diego, La Jolla, California.
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 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 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.
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 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 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.