儿童难治性特发性血小板减少性紫癜的治疗进展

特发性血小板减少性紫癜(ITP)是儿童最常见的出血性疾病,发病率高,对儿童健康造成很大威胁.传统治疗以糖皮质激素、静脉免疫球蛋白、免疫抑制剂等非特异性手段为主,但约有1/3患儿对治疗无效,成为难治性ITP.近年来随着对ITP自身免疫发病机制的深人探讨,许多定向免疫于预措施进入临床研究,该文就儿童难治性ITP分级治疗原则、传统疗法、定向免疫干预治疗等方面的进展作一综述.     

特发性血小板减少性紫癜发病机制研究进展

特发性血小板减少性紫癜(ITP)是一种自身免疫性疾病,是临床最常见的出血性疾病,其发病机制目前仍未完全明确.慢性ITP病情易反复,部分难治性ITP预后差.近年来,对ITP发病机制的研究取得了一系列重要进展.在体液免疫机制方面,对自身抗体产生的研究有了明显进展,并提出了由自身抗体介导引起的巨核细胞数量和质量异常;在细胞免疫机制方面,除了共刺激信号理论和Th细胞失衡及一系列细胞因子异常外,又提出了调节性T细胞数量减少及细胞毒T细胞直接溶解血小板的新理论.现就ITP发病机制的研究进展作一综述.     

儿童难治性特发性血小板减少性紫癜的诊疗进展

特发性血小板减少性紫癜(idiopathic thrombocytopenic purpura,ITP)又称为免疫性血小板减少症,是临床最常见的获得性出血性疾病.以血小板减少、血清中出现抗血小板抗体、骨髓巨核细胞数增加或正常伴成熟障碍为特征.约70％患者在诊断后的6个月内痊愈,但仍有部分患者转为持续性、慢性甚至难治性ITP.ITP的发病机制仍不完全清楚,目前认为主要有遗传易患性、血小板破坏过多及生成不足.因此,新的免疫抑制剂及促血小板生成剂不断被应用于临床中.该文对近年来在难治性ITP的诊断标准、严重度分级和治疗等方面取得的进展作一综述.     

免疫性血小板减少症的细胞免疫致病机制研究最新进展

<正>免疫性血小板减少症(primary immune thrombocytopenia,ITP)是儿童期常见血液病之一,约占出血性疾病的25%～30%~([1])。ITP是因多种已知或未知病因作用下,体内产生血小板自身抗体导致血小板破坏,以及骨髓巨核细胞分化成熟障碍所致血小板生成减少,从而出现外周血小板计数下降并伴有不同程度出血倾向~([2])。既往认为ITP与其他自身免疫性疾病相似,属于体液免疫异常疾病~([3-5])。但随着研究的不断深入,目前发现ITP的免疫异常发病机制,在始动和进展阶段,以及免疫调节过程中,均有细胞免疫机制参与~([6-8])。因此,有关ITP与     

小儿难治性特发性血小板减少性紫癜的现代治疗

特发性血小板减少性紫癜(ITP)是儿科常见的出血性疾病之一。儿童患者绝大部分为急性型,病程多属自限性。虽未经特殊治疗,亦可在4～6周自然康复。但约有10%的患儿病势迁延,治疗困难。ITP的免疫发病机制,主要是循环中经血小板相关抗体(PA-IgG)致敏血小板,被巨     

小儿特发性血小板减少性紫癜与免疫

特发性血小板减少性紫癜(ITP)是小儿常见的出血性疾病之一,其病因未明,发病机制亦尚未完全明了。研究证明该病由免疫介导,急性ITP与病毒感染有关,而慢性ITP可能由抗血小板自身抗体所引起。PAIg/C_3的检测为ITP的诊断提供了依据;血小板相关抗原的研究表明自身抗原主要是血小板膜糖蛋白成分;ITP时有T淋巴细胞亚群的异常,提示存在免疫调节功能缺陷。本文还简要综述了ITP治疗的某些进展。     

特发性血小板减少性紫癜研究进展

特发性血小板减少性紫癜(idiopathic thrombocytopenic purpura,ITP)是儿童常见的出血性疾病,临床表现为皮肤、粘膜的自发性出血,实验室检查血小板数量减少,骨髓巨核细胞数量正常或增多,伴成熟障碍.目前ITP确切的发病机制尚不清楚,早期研究发现ITP的发生与自身抗体有关,进而又证实ITP存在细胞免疫功能的紊乱,近年来,有学者提出ITP患者血小板和巨核细胞也存在异常,提示ITP不仅表现为血小板数量减少,同时存在功能障碍,井且与免疫功能紊乱有一定关系.本文就近年来ITP血小板的相关研究进展作一综述.     

重症免疫性血小板减少性紫癜的治疗进展

免疫性血小板减少性紫癜(ITP)是由于自身产生血小板抗体,导致血小板在单核-巨噬细胞系统破坏过多的自身免疫性疾病,是儿童常见的出血性疾病之一.与成人不同,儿童的ITP大部分呈急性、自限性过程.据美国血液学会统计,不予治疗的患儿血小板计数升高占83%,在6个月内恢复的患儿占70%～80% [1].然而有一小部分患儿血小板持续<20×109/L,伴有一个以上部位出血,称之为重症ITP,这部分患儿往往对治疗的反应不佳.     

儿童免疫性血小板减少症发病机制研究进展

免疫性血小板减少症(immune thrombocytopenia,ITP)是一种由自身免疫介导的出血性疾病,其特点为血小板破坏增多和生成不足.ITP发病机制复杂多样,具有异质性,至今尚未完全明确,在儿童中尤其突出.现有研究表明,免疫失耐受引起各种免疫细胞或分子异常,进而导致血小板破坏增多或生成不足,是ITP的核心发病...     

儿童慢性原发性免疫性血小板减少症

原发性免疫性血小板减少症(ITP)是一种自身免疫性疾病,在儿童中发病率约为(4～5)/10万,多数(80%～90%)ITP患儿在确诊后12个月内血小板数可恢复正常,但少数(10%～20%)患儿血小板减少会超过1年,成为慢性ITP。慢性ITP的发病机制尚未清楚,现有研究认为是在患儿易感基因背景下,由于感染和免疫紊乱产生的自身免疫抗体对单核-巨噬细胞系统的破坏造成血小板减少。文章综述慢性ITP的诊断,预测因素及相关治疗方法。     

Low-dose cyclosporin A therapy in children with refractory immune thrombocytopenic purpura

Although splenectomy is the most effective treatment for chronic idiopathic thrombocytopenic purpura (ITP), many post-splenectomy patients have recurrent thrombocytopenia refractory to multiple medical therapies. Three consecutive patients with relapsed ITP after splenectomy and who were refractory to multiple medical therapies were treated with low dose cyclosporin A (CsA). In all 3 patients, the platelet count increased dramatically within 1 month from the onset of CsA therapy. The only detectable toxicity was hypomagnesemia and mild hypertension in 1 patient. CsA may be efficacious in treating patients with chronic ITP, which is refractory to all medical and surgical therapies currently being used.     

儿童特发性血小板减少性紫癜病毒感染的临床分析

目的探讨儿童ITP病毒感染情况及其治疗与病情转归。方法(1)统计患儿发病的诱因,部分患儿检测EB病毒、巨细胞病毒、微小病毒B19、单纯疱疹病毒、支原体、自身抗体。分析病毒感染与ITP预后关系。(2)分析难治性与慢性TIP预后与治疗的关系。结果(1)病情严重程度与预后无关。(2)有特殊病毒感染诱因者共22例,其中12例(54.5%)为难治性或转为慢性;无特殊病毒感染诱因者共77例,其中19例(24.7%)为难治性或慢性,差异有显著性(P<0.05)。(3)难治性ITP转慢性的12例治疗效果均欠佳。(4)慢性ITP有特殊病毒感染者6例,其中好转的4例均多次短期大剂量应用丙种球蛋白,此4例中的3例同时或之前应用抗病毒药物,2例应用肾上腺皮质激素冲击。无特殊病毒感染史者13例,治疗好转6例,无效6例,均未应用大剂量丙种球蛋白。结论由特殊病毒感染引起的儿童ITP预后欠佳,加强联合大剂量丙种球蛋白、抗病毒的力度可改善预后。     

Directions for research in autoimmune thrombocytopenic purpura (ITP)

All attendees participated in a round-table discussion regarding directions for research in autoimmune thrombocytopenic purpura (ITP). Suggested areas for study were grouped into five main areas: (i) improved classification of ITP identifying subsets of patients with differing clinical syndromes and response to treatment, and those more likely to have serious bleeding manifestations; identification of patients with reduced thrombopoiesis was emphasized; (ii) studies aimed at elucidating the aetiology and pathophysiology of ITP, with emphasis on distinctions between acute and chronic ITP and between patients responsive or refractory to therapy; these studies focused on measures of humoral and cellular immune dysregulation; (iii) studies of platelet function in ITP, with the intent of defining these abnormalities and correlating them with the clinical manifestations of the disease; (iv) new approaches to treatment, particularly of refractory patients; and (v) a miscellaneous group, which included development of an ITP registry, evaluation of the "burden" of disease, investigation of mood changes in ITP, etc. The discussion was not intended to be all-inclusive, but focused on the content of other talks in this symposium. It is hoped that some of thesesuggestions will be further developed for investigation in multicentre co-operative studies to improve the diagnosis, understanding and treatment of ITP.     

High-dose dexamethasone therapy for a 14-month-old boy with refractory idiopathic thrombocytopenic purpura

A 14-month-old boy with refractory idiopathic thrombocytopenic purpura (ITP), who was successfully treated with pulsed high-dose oral dexamethasone therapy is reported. The platelet count increased after six scheduled courses of treatment (10 mg/day × 4 days, six courses). Twenty-four months later, the platelet count remained over 10.0 × 10⁴/μL. No obvious side effects were observed during or after the therapy. This treatment could be taken into consideration not only for adults but also for young children with refractory ITP. It is effective, safe, easy to administer, patient comfort is taken into consideration, and hospitalization duration and costs are minimized.     

Use of Romiplostim for Primary Immune Thrombocytopenia in Children

Very little has been published on the use of romiplostim to treat primary immune thrombocytopenia (ITP), refractory to previous treatments, in children. The objective of this study was to determine its efficacy and safety in pediatric patients in a university general hospital. Retrospective, longitudinal observational study of pediatric patients on treatment with romiplostim. The principal efficacy variable was platelet count. Safety was evaluated by recording possible adverse reactions to the medication, monitoring the appearance of thrombosis, thrombocytopenia during dose reduction, hemorrhage, and myelodysplastic syndromes. Three patients in the authors’ center have been treated with romiplostim (subcutaneous [SC], initial dose: 1 μg/kg/week) for ITP refractory to various treatments: 1 with newly diagnosed ITP and 2 with chronic ITP. Patients were followed up for 27 to 39 weeks after starting treatment. Responses were achieved in 7 to 28 days, and complete responses were maintained for 37% to 91% of the follow-up period, with median platelet counts between 40 × 10³/μL and 215 × 10³/μL. The adverse reactions observed during follow-up were headache and asthenia in one patient and mucocutaneous bleeding after dose suspension in another one. With regard to effectiveness, the response in the 3 patients was varied. The drug was considered to be safe, as there were only mild adverse reactions. Although further studies and long-term follow-up are required, these results show that romiplostim could be considered an alternative to immunosuppressive therapies, such as rituximab, or splenectomy in refractory chronic ITP.     

Treatment of an infant with severe acute refractory immune thrombocytopenic purpura using combination therapy including rituximab

Some infants with acute immune thrombocytopenic purpura (ITP) do not respond to first‐line therapy, and currently there is no consensus on therapy for these refractory cases. We describe a 12‐week‐old infant with acute ITP who was unresponsive to intravenous immunoglobulin and corticosteroid, and developed gastrointestinal bleeding. Several combination therapies were unsuccessful. After four doses of rituximab followed by intravenous immunoglobulin and corticosteroid, his platelet counts gradually increased. Combined therapy which includes rituximab may be a promising treatment for severe acute refractory ITP. Pediatr Blood Cancer 2009;53:203–205. © 2009 Wiley‐Liss, Inc.     

Use of romiplostim for primary immune thrombocytopenia in children

Very little has been published on the use of romiplostim to treat primary immune thrombocytopenia (ITP), refractory to previous treatments, in children. The objective of this study was to determine its efficacy and safety in pediatric patients in a university general hospital. Retrospective, longitudinal observational study of pediatric patients on treatment with romiplostim. The principal efficacy variable was platelet count. Safety was evaluated by recording possible adverse reactions to the medication, monitoring the appearance of thrombosis, thrombocytopenia during dose reduction, hemorrhage, and myelodysplastic syndromes. Three patients in the authors' center have been treated with romiplostim (subcutaneous [SC], initial dose: 1 μg/kg/week) for ITP refractory to various treatments: 1 with newly diagnosed ITP and 2 with chronic ITP. Patients were followed up for 27 to 39 weeks after starting treatment. Responses were achieved in 7 to 28 days, and complete responses were maintained for 37% to 91% of the follow-up period, with median platelet counts between 40 × 10(3)/μL and 215 × 10(3)/μL. The adverse reactions observed during follow-up were headache and asthenia in one patient and mucocutaneous bleeding after dose suspension in another one. With regard to effectiveness, the response in the 3 patients was varied. The drug was considered to be safe, as there were only mild adverse reactions. Although further studies and long-term follow-up are required, these results show that romiplostim could be considered an alternative to immunosuppressive therapies, such as rituximab, or splenectomy in refractory chronic ITP.     

Successful α-interferon therapy in a child with chronic refractory idiopathic thrombocytopenic purpura: A case report

We treated a sixteen month old male with chronic refractory idiopathic thrombocytopenic purpura (ITP) in whom α-interferon (IFN) therapy was effective. He developed ITP which did not respond to various treatments. Six months after admission, we began to treat him with IFN. The patient's platelet count rapidly responded to the therapy and rose above normal range. Serum levels of platelet associated immunoglobulin G (PA-IgG) showed a tendency to decrease with the administration of IFN. After stopping the IFN therapy for a duration of 3 months, the platelet count remained normal. No serious adverse side effects, except transient fever, were observed. From the experience of this case we propose that IFN is one of the therapeutic options for treatment of refractory ITP not only in adults but also in children.     

利妥昔单抗治疗儿童难治性血小板减少性紫癜合并乙肝病毒感染1例报告及文献复习

目的探讨利妥昔单抗治疗儿童难治性血小板减少性紫癜(rITP)合并乙肝病毒感染的疗效与安全性。方法利妥昔单抗(375 mg/m2,每周1次,连用4周)治疗1例儿童rITP合并既往乙肝病毒感染(Anti-HBs+、Anti-HBe+、Anti-HBc+),同时口服拉米夫定(100 mg/d,1次/d,6个月),监测患儿血小板、免疫球蛋白和乙肝病毒DNA定量的变化,并进行相关文献复习。结果随诊时间6个月,血小板始终>100.0×109/L,乙肝病毒无异常复制。结论在预防应用抑制乙肝病毒复制药物的情况下,利妥昔单抗治疗儿童rITP合并乙肝病毒感染是安全、有效的。