Department of Biostatistics and Epidemiology, Hudson College of Public Health, University of Oklahoma Health Sciences Center, Oklahoma City, OK 73104, USA.
Department of Pediatrics, Columbia University Irving Medical Center, New York, NY 10032, USA.
Medicina (Kaunas). 2020 Nov 30;56(12):667. doi: 10.3390/medicina56120667.
Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia caused by increased platelet destruction and impaired platelet production. First-line therapies include corticosteroids, intravenous immunoglobulin, and anti-D immunoglobulin. For patients who are refractory to these therapies, those who become corticosteroid dependent, or relapse following treatment with corticosteroid, options include splenectomy, rituximab, and thrombopoietin-receptor agonists, alongside a variety of additional immunosuppressive and experimental therapies. Despite recent advances in the management of ITP, many areas need further research. Although it is recognized that an assessment of patient-reported outcomes in ITP is valuable to understand and guide treatment, these measures are not routinely measured in the clinical setting. Consequently, although corticosteroids are first-line therapies for both children and adults, there are no data to suggest that corticosteroids improve health-related quality of life or other patient-related outcomes in either children or adults. In fact, long courses of corticosteroids, in either children or adults, may have a negative impact on a patient's health-related quality of life, secondary to the impact on sleep disturbance, weight gain, and mental health. In adults, additional therapies may be needed to treat overt hemorrhage, but unfortunately the results are transient for the majority of patients. Therefore, there is a need to recognize the limitations of current existing therapies and evaluate new approaches, such as individualized treatment based on the probability of response and the size of effect on the patient's most bothersome symptoms and risk of adverse effects or complications. Finally, a validated screening tool that identifies clinically significant patient-reported outcomes in routine clinical practice would help both patients and physicians to effectively follow a patient's health beyond simply treating the laboratory findings and physical symptoms of ITP. The goal of this narrative review is to discuss management of newly diagnosed and refractory patients with ITP, with a focus on the limitations of current therapies from the patient's perspective.
原发性免疫性血小板减少症（ITP）是一种获得性自身免疫性疾病，其特征为孤立性血小板减少症，由血小板破坏增加和血小板生成受损引起。一线治疗包括皮质类固醇、静脉注射免疫球蛋白和抗-D 免疫球蛋白。对于这些治疗方法无效的患者、对皮质类固醇依赖的患者或皮质类固醇治疗后复发的患者，选择包括脾切除术、利妥昔单抗和血小板生成素受体激动剂，以及各种其他免疫抑制和实验性治疗。尽管 ITP 的治疗取得了最近的进展，但仍有许多领域需要进一步研究。尽管人们认识到评估 ITP 患者报告的结局对于理解和指导治疗很有价值，但这些措施在临床环境中并未常规测量。因此，尽管皮质类固醇是儿童和成人 ITP 的一线治疗方法，但没有数据表明皮质类固醇可以改善儿童或成人的健康相关生活质量或其他患者相关结局。事实上，长期使用皮质类固醇，无论是在儿童还是成人中，都可能对患者的健康相关生活质量产生负面影响，这是由于皮质类固醇对睡眠障碍、体重增加和心理健康的影响。在成年人中，可能需要额外的治疗来治疗明显的出血，但不幸的是，大多数患者的结果是短暂的。因此，有必要认识到现有治疗方法的局限性，并评估新的方法，例如基于反应概率和对患者最困扰症状和不良反应或并发症风险的影响大小的个体化治疗。最后，一个有效的筛查工具可以在常规临床实践中识别出具有临床意义的患者报告的结局，这将有助于患者和医生不仅要治疗 ITP 的实验室发现和身体症状，还要有效地关注患者的健康。本叙述性综述的目的是讨论新诊断和难治性 ITP 患者的管理，重点是从患者的角度探讨现有治疗方法的局限性。
