Goss Cheryl, Giardina Patricia, Degtyaryova Diana, Kleinert Dorothy, Sheth Sujit, Cushing Melissa
Departments of Transfusion Medicine and Pediatrics, New York Presbyterian Hospital-Weill Cornell Medical College, New York, New York.
Transfusion. 2014 Jul;54(7):1773-81. doi: 10.1111/trf.12571. Epub 2014 Feb 24.
In the absence of curative treatment, such as stem cell transplant, regular transfusions remain the mainstay of therapy for individuals with thalassemia major, a syndrome that results from marked ineffective erythropoiesis and the resultant anemia. The primary objectives of transfusion therapy are twofold: to suppress ineffective erythropoiesis and to ensure appropriate growth and development through childhood. In practice, a number of different transfusion protocols are in use across the developed world, with on-demand transfusion still being the paradigm in most of the developing world with limited resources.
To investigate perceived differences in transfusion practice, a self-reported electronic survey was disseminated to eight US thalassemia treatment centers in February 2011. The survey was divided into sections ranging from laboratory and clinical practices to emerging transfusion-transmitted diseases.
The survey response rate was 100%. The total number of transfused patients was 411. One-hundred percent of institutions used leukoreduced blood. No centers routinely provided cytomegalovirus-seronegative red blood cells (RBCs). Half the centers provided irradiated RBCs; only one routinely provided washed RBCs, and none transfused RBCs of defined storage age. Seventy-five percent of centers routinely phenotyped thalassemia patients' RBC antigens; 50% prophylactically matched for Rh and K antigens. The frequency of antibody investigations varied widely, and 25% of centers routinely medicated patients before transfusion.
Eight thalassemia centers in the United States were surveyed to determine the uniformity of transfusion practice. The variability of the results was surprising. Consequently, we performed a literature review and propose an evidence-based protocol for routine transfusion therapy for patients with thalassemia.
在缺乏如干细胞移植等治愈性治疗的情况下，定期输血仍然是重型地中海贫血患者治疗的主要手段，重型地中海贫血是一种由于显著的无效红细胞生成及由此导致的贫血所引起的综合征。输血治疗的主要目标有两个：抑制无效红细胞生成，并确保患儿在童年期能够正常生长发育。实际上，在发达国家有多种不同的输血方案在使用，而在资源有限的大多数发展中国家，按需输血仍是主要模式。
为了调查输血实践中存在的认知差异，2011年2月向美国8个地中海贫血治疗中心发放了一份自我报告式电子调查问卷。该问卷分为从实验室及临床实践到新兴的输血传播疾病等多个部分。
调查回复率为100%。输血患者总数为411例。所有机构都使用了白细胞滤除血液。没有中心常规提供巨细胞病毒血清阴性红细胞。一半的中心提供辐照红细胞；只有一个中心常规提供洗涤红细胞，且没有中心输注规定储存期的红细胞。75%的中心常规对地中海贫血患者的红细胞抗原进行表型分析；50%进行Rh和K抗原的预防性配型。抗体检测的频率差异很大，25%的中心在输血前常规给患者用药。
对美国8个地中海贫血中心进行了调查，以确定输血实践的一致性。结果的变异性令人惊讶。因此，我们进行了文献综述，并提出了一个基于证据的重型地中海贫血患者常规输血治疗方案。
