Translational Hematology and Oncology Research Department, Taussig Cancer Center, Cleveland Clinic, Cleveland, OH; and.
Department of Biomedicine and Prevention, Molecular Medicine and Applied Biotechnology, University of Rome Tor Vergata, Rome, Italy.
Blood. 2021 Apr 15;137(15):2001-2009. doi: 10.1182/blood.2021010898.
Pure red cell aplasia (PRCA) is a rare hematological disorder with multiple etiologies. The multifaceted nature of this disease is emphasized by the variety of concomitant clinical features. Classic idiopathic presentation aside, prompt recognition of pathogenetic clues is important because of their diagnostic and therapeutic implications. As a consequence, treatment of PRCA is diverse and strictly dependent on the presented clinical scenario. Here, we propose a series of clinical vignettes that showcase instructive representative situations derived from our routine clinical practice. Using these illustrative clinical cases, we review the diagnostic workup needed for a precise diagnosis and the currently available therapeutic options, discussing their applications in regard to the various PRCA-associated conditions and individual patients' characteristics. Finally, we propose a treatment algorithm that may offer guidance for personalized therapeutic recommendations.
纯红细胞再生障碍性贫血（PRCA）是一种罕见的血液系统疾病，具有多种病因。由于其具有多种伴随的临床特征，因此这种疾病的多面性更为明显。除了经典的特发性表现外，由于其具有诊断和治疗意义，因此及时识别发病机制线索非常重要。因此，PRCA 的治疗方法多种多样，严格取决于所呈现的临床情况。在这里，我们提出了一系列临床病例，这些病例展示了源自我们常规临床实践的有启发性的代表性情况。使用这些说明性临床病例，我们回顾了精确诊断所需的诊断性检查以及当前可用的治疗选择，并讨论了它们在各种与 PRCA 相关的情况和个体患者特征中的应用。最后，我们提出了一种治疗算法，可为个性化治疗建议提供指导。
