Seçkin Esra, Ciftciler Rafiye
Selcuk University Faculty of Medicine, Department of Internal Medicine, Konya, Turkey.
Selcuk University, Faculty of Medicine, Department of Hematology, Konya, Turkey.
Hematol Transfus Cell Ther. 2024 Nov;46 Suppl 5(Suppl 5):S208-S213. doi: 10.1016/j.htct.2024.03.008. Epub 2024 Jul 23.
Immune thrombocytopenic purpura, a recurrent autoimmune disease, is characterized by thrombocytopenia, purpura and hemorrhagic episodes with the main factor in the pathogenesis of this disease being autoantibodies against platelets. Since the 1950s, first-line treatment has been glucocorticoids that have indirect and direct effects on thrombocytopenia. Although the characteristics associated with the chronicization of immune thrombocytopenic purpura at the time of diagnosis have been investigated in previous studies, no study was found in the literature investigating the relationship between the response to first-line steroid treatment and the course of the disease, the aim of this study.
This retrospective, single center study revisited electronic files of patients with a diagnosis of immune thrombocytopenic purpura between September 2012 and September at the Department of Clinical Hematology, Selcuk University Faculty of Medicine 2022. The platelet count had been confirmed by peripheral blood smears of patients with a platelet count ≤30 × 10/L. The bleeding status of patients at the time of diagnosis was evaluated according to the immune thrombocytopenic purpura bleeding score. Patient responses to treatment were categorized in three groups: a platelet count ≤30 × 10/L was defined as no-response, a platelet count of 30-100 × 10/L was defined as partial response, and a platelet count >100 × 10/L was defined as complete response. Subsequently, patients in the partial or complete response groups were divided into two subgroups: patients who remained in remission for less than or more than six months.
A total of 100 patients were included in the study; 73 % were in the young (19-65 years old) and 27 % in the old (>65 years old) age group. Most of the patients were female (69 %). Forty-one patients were hospitalized without bleeding. The complete response rate to first-line treatment was 61 %. There was no significant difference between the agents given in first-line treatment in terms of response and length of remission.
The main purpose of immune thrombocytopenic purpura treatment is to prevent severe bleeding rather than bringing the platelet count to normal values. Glucocorticoids, the first step of treatment, provide high response rates. There is no significant difference between glucocorticoid agents in terms of response to treatment and long-term remission. The points to be considered in the selection of glucocorticoid agents are the side effect profiles, ease of administration and individualization of treatment.
免疫性血小板减少性紫癜是一种复发性自身免疫性疾病,其特征为血小板减少、紫癜和出血发作,该疾病发病机制的主要因素是针对血小板的自身抗体。自20世纪50年代以来,一线治疗药物一直是糖皮质激素,其对血小板减少有间接和直接作用。尽管先前的研究已经探讨了免疫性血小板减少性紫癜诊断时与慢性化相关的特征,但文献中未发现有研究调查一线类固醇治疗反应与疾病进程之间的关系,而这正是本研究的目的。
这项回顾性单中心研究重新查阅了2012年9月至2022年9月在塞尔丘克大学医学院临床血液科诊断为免疫性血小板减少性紫癜患者的电子病历。血小板计数≤30×10⁹/L的患者通过外周血涂片确认血小板计数。根据免疫性血小板减少性紫癜出血评分评估患者诊断时的出血状态。患者对治疗的反应分为三组:血小板计数≤30×10⁹/L定义为无反应,血小板计数为30 - 100×10⁹/L定义为部分反应,血小板计数>100×10⁹/L定义为完全反应。随后,部分反应或完全反应组的患者分为两个亚组:缓解持续时间少于或多于六个月的患者。
本研究共纳入100例患者;73%为年轻人(19 - 65岁),27%为老年人(>65岁)。大多数患者为女性(69%)。41例患者住院时无出血。一线治疗的完全反应率为61%。一线治疗所用药物在反应和缓解持续时间方面无显著差异。
免疫性血小板减少性紫癜治疗的主要目的是预防严重出血,而非使血小板计数恢复正常。糖皮质激素作为治疗的第一步,具有较高的反应率。糖皮质激素药物在治疗反应和长期缓解方面无显著差异。选择糖皮质激素药物时应考虑的要点是副作用、给药便利性和治疗个体化。