Tran Minh-Ha, Jones Jennifer, Qiao Jesse
University of California, Irvine, Department of Pathology & Laboratory Medicine, Orange, CA, United States.
University of Michigan Medicine, Department of Pathology and Internal Medicine, United States.
Transfus Apher Sci. 2025 Aug;64(4):104174. doi: 10.1016/j.transci.2025.104174. Epub 2025 Jun 7.
Immune Thrombotic thrombocytopenic purpura (iTTP) is a rare but life-threatening thrombotic microangiopathy characterized by an autoantibody against ADAMTS13, leading to accumulation of ultra-large von Willebrand multimers, systemic platelet microthrombi, end-organ damage, and mortality if untreated. Therapeutic plasma exchange and corticosteroids have been the mainstay therapy for decades, but there exists significant relapse potential after the initial acute episode. While more recent advances in the use of immunotherapy (e.g. rituximab and caplacizumab) have significantly improved acute survival and short-term exacerbation/relapse prevention, long-term complications of the disease remain a concern for survivors. This narrative review discusses challenges of optimizing post-remission care after iTTP, highlighting disease impact on neurological, cardiovascular, and psychological health. Chronic cognitive impairment, increased risk of hypertension and ischemic events, and mental health challenges such as anxiety and depression are reported in iTTP survivors. Moreover, recurrence risk and persistent ADAMTS13 deficiency may define the need for long-term monitoring and individualized treatment of potential relapse. We emphasize the importance of multidisciplinary, patient-centered management, not only in the management and prevention of iTTP relapse episodes, but to improve quality of life and reduce morbidity in survivors of this rare disease. Providers should possess heightened awareness of long-term complications and atypical manifestations of relapse in survivors. We advocate for further research and observational cohort studies to formulate standardized guidelines for surveillance and intervention to mitigate the chronic burden of iTTP.
免疫性血栓性血小板减少性紫癜(iTTP)是一种罕见但危及生命的血栓性微血管病,其特征是存在针对ADAMTS13的自身抗体,导致超大血管性血友病因子多聚体蓄积、全身性血小板微血栓形成、终末器官损伤,若不治疗可导致死亡。几十年来,治疗性血浆置换和皮质类固醇一直是主要治疗方法,但在初始急性发作后仍有显著的复发可能性。虽然免疫疗法(如利妥昔单抗和卡帕单抗)的最新进展显著提高了急性生存期并预防了短期病情加重/复发,但该疾病的长期并发症仍是幸存者的一大担忧。本叙述性综述讨论了优化iTTP缓解后护理的挑战,强调了该疾病对神经、心血管和心理健康的影响。据报道,iTTP幸存者存在慢性认知障碍、高血压和缺血性事件风险增加以及焦虑和抑郁等心理健康问题。此外,复发风险和持续性ADAMTS13缺乏可能决定了对潜在复发进行长期监测和个体化治疗的必要性。我们强调多学科、以患者为中心管理的重要性,这不仅有助于管理和预防iTTP复发,还能提高这种罕见疾病幸存者的生活质量并降低发病率。医疗服务提供者应提高对幸存者长期并发症和复发非典型表现的认识。我们主张开展进一步研究和观察性队列研究,以制定标准化的监测和干预指南,减轻iTTP的慢性负担。