Division of Hematology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
Mayo Clinic Evidence-Based Practice Research Program, Mayo Clinic, Rochester, MN.
Blood Adv. 2020 Jan 28;4(2):327-355. doi: 10.1182/bloodadvances.2019001143.
Red cell transfusions remain a mainstay of therapy for patients with sickle cell disease (SCD), but pose significant clinical challenges. Guidance for specific indications and administration of transfusion, as well as screening, prevention, and management of alloimmunization, delayed hemolytic transfusion reactions (DHTRs), and iron overload may improve outcomes.
Our objective was to develop evidence-based guidelines to support patients, clinicians, and other healthcare professionals in their decisions about transfusion support for SCD and the management of transfusion-related complications.
The American Society of Hematology formed a multidisciplinary panel that was balanced to minimize bias from conflicts of interest and that included a patient representative. The panel prioritized clinical questions and outcomes. The Mayo Clinic Evidence-Based Practice Research Program supported the guideline development process. The Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach was used to form recommendations, which were subject to public comment.
The panel developed 10 recommendations focused on red cell antigen typing and matching, indications, and mode of administration (simple vs red cell exchange), as well as screening, prevention, and management of alloimmunization, DHTRs, and iron overload.
The majority of panel recommendations were conditional due to the paucity of direct, high-certainty evidence for outcomes of interest. Research priorities were identified, including prospective studies to understand the role of serologic vs genotypic red cell matching, the mechanism of HTRs resulting from specific alloantigens to inform therapy, the role and timing of regular transfusions during pregnancy for women, and the optimal treatment of transfusional iron overload in SCD.
红细胞输注仍然是治疗镰状细胞病（SCD）患者的主要治疗方法，但也带来了重大的临床挑战。特定适应证的指导以及输血管理，以及同种免疫、迟发性溶血性输血反应（DHTR）、铁过载的筛查、预防和管理，可能会改善预后。
我们的目标是制定循证指南，以支持患者、临床医生和其他医疗保健专业人员做出关于 SCD 输血支持和输血相关并发症管理的决策。
美国血液学会成立了一个多学科小组，该小组的组成平衡，以最大限度地减少利益冲突带来的偏见，并包括一名患者代表。该小组确定了临床问题和结果的优先级。梅奥诊所循证实践研究计划支持指南制定过程。采用推荐评估、制定与评价（GRADE）方法制定建议，并接受公众意见。
该小组制定了 10 项建议，重点关注红细胞抗原分型和匹配、适应证和输注方式（简单输血与红细胞交换），以及同种免疫、DHTR 和铁过载的筛查、预防和管理。
由于缺乏对感兴趣结局的直接、高确定性证据，大多数小组建议都是有条件的。确定了研究优先事项，包括前瞻性研究以了解血清学与基因型红细胞匹配的作用、由特定同种抗原引起的 HTR 机制、女性妊娠期间定期输血的作用和时机，以及 SCD 中输血性铁过载的最佳治疗方法。
