大剂量和标准剂量糖皮质激素对慢性特发性血小板减少性紫癜治疗的临床研究

特发性血小板减少性紫癜（ITP）是临床最常见的出血性疾病，其主要原因是体内产生抗自身血小板的抗体，致使血小板寿命缩短，破坏加速。同时有巨核细胞成熟障碍，使血小板生成减少。既往泼尼松常作为治疗ITP的首选药物，     

升血小板胶囊联合半量糖皮质激素治疗难治性特发性血小板减少性紫癜的疗效观察

特发性血小板减少性紫癜(ITP)是一种与自身免疫紊乱有关的疾病。近年来研究发现,本病病因主要是体内产生抗血小板自身抗体或免疫复合物结合于血小板细胞膜表面而导致血小板破坏增多,而脾脏是产生抗自身血小板抗体和破坏血小板的主要器官。因此,目前针对ITP采用的主要治疗措施有:糖皮质激素、静脉注射人免疫球蛋白、脾切除和免疫抑制剂。其中糖皮质激素为常用一线药物,初始治疗剂量多以泼尼松1～2mg.kg-1.     

胸腺肽α1联合大剂量地塞米松治疗慢性特发性血小板减少性紫癜的疗效分析

目的探讨胸腺肽α1（Tα1）联合大剂量地塞米松（Dex）短程冲击治疗初治慢性特发性血小板减少性紫癜（ITP）的疗效及其细胞免疫机制。方法（1）66例初诊ITP患者口服Dex 40mg/d,连用4d;其中39例同时给予Tα1 1．6mg皮下注射,每周3次,连续应用4周。（2）ELISA法检测正常对照组20例及初诊ITP患者治疗前后血浆Tα1、IFNγ、IL-2、IL-4、IL-10及转化生长因子（TGF）-β1水平。结果（1）Tα1＋Dex联合治疗组与Dex单药治疗组,完全缓解（CR）率分别为76．9％（30／39）、44．4％（12／27）（P〈0．05）;长期反应率分别为61．5％（24／39）、34．6％（9／26）,复发率分别为38．5％（15／39）、65．4％（17／26）,差异均有统计学意义（P〈0．05）。（2）Tα1＋Dex治疗后Tα1水平为（1．83±1．22）μg/L较治疗前（2．43±1．47）μg／L明显降低（P〈0．05）。（3）Tα1＋Dex治疗后患者IFNγ和IL-2的血浆水平[（11．57±4．33）、（14．56±10．76）]ng／L均较治疗前[（22．71±7．98）、（28．42±11．27）]ng／L显著降低（P〈0．01）,且与正常对照组比较[（10．23±3．97）、（8．73±8．22）]ng／L,差异无统计学意义（P〉0．05）。IL-4和IL-10的血浆水平[（9．87±4．82）、（7．90±2．71）]ng／L均较治疗前[（5．93±3．85）、（3．24±1．36）]ng／L明显升高（P〈0．05）,且与正常对照组比较,差异无统计学意义（P〉0．05）。（4）Tα1＋Dex治疗后患者血浆TGF-β1水平（4．19±1．80）μg/L较治疗前（1．31±0．71）μg／L明显升高（P〈0．01）。（5）Tα1＋Dex治疗后患者Tα1水平与TGF-β1含量之间呈显著正相关（r=0．6028,P〈0．05）。结论（1）Tα1＋Dex联合治疗可以纠正ITP患者体内Th1／Th2平衡紊乱,减少血小板破坏,其CR率高、复发率较低且耐受性好,可作为ITP治疗的一种新的尝试。（2）Tα1＋Dex治疗后患者Tα1水平与作为Th3特异性标志的TGF-β1之间呈显著正相关,可能与NK细胞的生理性抑制功能上调有关。     

糖皮质激素耐药特发性血小板减少性紫癜治疗进展

特发性血小板减少性紫癜(ITP)是一种自身免疫性疾病,糖皮质激素常作为ITP治疗首选药物,有效率70%~80%,仍有部分耐药ITP。下面将糖皮质激素耐药ITP治疗进展做一综述。1针对脾的治疗脾既是血小板抗体产生的主要器官,又是血小板破坏的主要部位。针对脾的治疗(包括脾切除、脾栓塞、     

122例特发性血小板减少性紫癜血小板输注疗效观察

目的:探讨特发性血小板减少性紫癜(ITP)患者临床血小板输注的指征.方法:比较122例ITP患者输注血小板和未输注血小板的临床转归;比较血小板输注前及输注24 h后血小板计数.参照PAIg,分析抗体与血小板输注疗效的相关性.结果:67例未输注血小板的患者中,有2例PLT＜10×109/L的患者发生严重出血危及生命,55例输注血小板的惠者,有17例(31%)PLT较输注前降低,其中7例输注后PLT＜10×109/L,未发生严重出血;ITP患者血小板输注无效率86%,PAIg升高患者血小板输注无效率升高,有统计学差异.结论:如无明显的出血症状,ITP血小板输注指征建议PLT＜10×109/L,应输注少白细胞同型单采血小板制荆.     

丙种球蛋白联合地塞米松在小儿特发性血小板减少性紫癜中的疗效观察

目的观察和分析对小儿特发性血小板减少紫癜采取丙种球蛋白联合地塞米松治疗的临床效果及价值。方法选取于2012年7月-2013年11月在我院接受治疗的特发性血小板减少紫癜患儿34例为研究对象,采取数字标记法随机将上述患儿分成对照组和观察组,观察组患儿采取丙种球蛋白联合地塞米松进行治疗,静脉注射3d后,口服泼尼松进行治疗;对照组患儿不给于丙种球蛋白治疗,其余治疗药物与观察组相同;观察和对比两组患儿出血症状消失时间、血小板上升及恢复时间、住院时间以及临床疗效。结果观察组患儿出血症状消失时间、血小板上升时间、血小板恢复时间及住院时间较对照组而言,均明显缩短,两组间上述指标对比差异均有统计学意义(P0.05);观察组34例患者治疗疗效明显好于对照组,两组间该指标对比差异有统计学意义(P0.05)。结论对于特发性血小板减少紫癜患儿采取丙种球蛋白联合地塞米松进行治疗,不仅疗效明确,而且有助于患儿症状的快速改善,这对提升该病的治疗效果,保证治疗的顺利进行有着积极的意义,值得在临床上推广。     

地塞米松与强的松治疗小儿特发性血小板减少性紫癜临床疗效对比观察

不少作者已报道采用地塞米松冲击治疗儿童特发性血小板减少性紫癜(ITP),但其副作用较大。本研究目的在于探讨治疗本病时地塞米松的合适剂量及用法并与强的松对比。1 临床资料1.1 一般资料28例初发患儿,男20例,女8例,年龄5月～9岁,病程1～37天,均符合全国制定的ITP诊断标准。28例中均有粘膜、皮肤瘀斑,伴鼻血者7例,牙龈出血者5例。分为两组:地塞米松治疗组(12例),强的松治疗组(16例)。两组一般资料比较经精确概率法X~2检验均无显著性差异。     

利妥昔单抗治疗糖皮质激素无效的特发性血小板减少性紫癜疗效观察

目的 探讨利妥昔单抗治疗特发性血小板减少性紫癜(ITP)的疗效、安全性及治疗前后B细胞、血小板膜糖蛋白(GP)特异性自身抗体的变化.方法 利妥昔单抗(375 mg/m2,每周1次,连用4周)治疗12例糖皮质激素治疗无效的ITP患者,监测治疗前后的血常规、血清免疫球蛋白定量(IgG、IgM、IgA)、血小板GPⅡb/Ⅲa和(或)GP Ⅰ b/Ⅸ特异性自身抗体、CD+3、CD+4、CD+8、CD+19、CD+20细胞数.结果 4例完全有效,3例部分有效,2例微效,3例无效.随访中位时间5(0.5～12)个月,疗效均维持较好.有效患者治疗后血小板自身抗体均转阴.治疗前后血清IgG、IgM、IgA无明显变化,CD+3、CD+4、CD+8细胞数无明显变化.治疗后CD+19/CD+20细胞数(4.1±2.2)×106/L与治疗前(295.0±86.4)×106/L比较明显下降(P＜0.01).无严重不良反应.结论 利妥昔单抗治疗糖皮质激素无效的ITP患者安全、有效.     

特发性血小板减少性紫癜分期论治

特发性血小板减少性紫癜 (ＩＴＰ)是临床常见出血性疾病 ,笔者近年来致力于该病的研究 ,临床将本病分为两期论治 ,并按各期不同特点 ,单纯采用中药治疗 ,取得了较好效果 ,现介绍如下。1　急性期血热炽盛 ,清热凉血止血为首法　　急性期多发于 2岁～ 9岁儿童 ,起病前 1周～ 3周常有上呼吸道感染史 ,起病急 ,出血重 ,以皮肤粘膜突然出现紫斑 ,斑色鲜红而密集 ,伴有齿鼻衄血为突出表现 ,实验室检查除血小板减少外 ,患儿血循环中抗血小板抗体明显升高 ,骨髓中巨核细胞数正常或增多。证属风热疫毒入血 ,灼伤血络 ,迫血妄行 ,治当清热解毒 ,凉血止…     

短周期大剂量地塞米松治疗特发性血小板减少性紫癜的疗效分析

组的同期复发率.结论 短周期3个疗程HD-DXM方案是治疗成人初诊ITP的有效方法,HD-DXM使用间歇期小剂量地塞米松维持治疗有助于提高患者的远期疗效.     

Elevated levels of T-cell immune response cDNA 7 in patients with immune thrombocytopenia

Objectives

This study aimed to investigate the expression levels of T-cell immune response cDNA 7 (TIRC7) in immune thrombocytopenia (ITP) patients before and after high-dose dexamethasone (HD-DXM) treatment.

Methods

Forty-four patients with ITP were enrolled and received dexamethasone (40 mg/day) for 4 consecutive days. Patients who had platelet counts more than 50 × 10⁹/l or less were defined as responders or non-responders, respectively. Quantitative polymerase chain reaction and enzyme-linked immunosorbent assay were used to measure RNA level and plasma level of TIRC7, respectively.

Results

TIRC7 levels (RNA and plasma level) were significantly higher in ITP than that in control (P < 0.0001). However, after treatment, TIRC7 levels were significantly decreased in responders (P < 0.0001) but not in non-responders (P > 0.05).

Discussions

TIRC7 might be associated with the pathogenesis of ITP, and TIRC7 levels could be used as an indicator to evaluate patients’ response to HD-DXM treatment.     

Obesity is associated with poor outcomes of corticosteroid treatment in patients with primary immune thrombocytopenia

Emerging evidence has demonstrated that obesity impacts multiple immune-related diseases. It remains unclear whether and how obesity alters treatment outcomes in patients with primary immune thrombocytopenia (ITP). Thus, we retrospectively investigated 214 treatment-naïve patients who received standard high-dose dexamethasone therapy in Qilu Hospital. Patients with obesity showed significantly lower overall initial response (underweight vs. normal vs. overweight vs. obese: 85.7% vs. 85.2% vs. 72.0% vs. 52.3%, p = 0.001) and initial complete response ([CR], 71.4% vs. 70.4% vs. 53.3% vs. 27.3%, p < 0.001) rates. The same trend was observed in the 6-month sustained response (63.6% vs. 52.3% vs. 35.6% vs. 22.7%, p = 0.03) and sustained CR (36.4% vs. 44.6% vs. 24.4% vs. 9.1%, p = 0.01). The Kaplan–Meier analysis revealed a shortened duration of remission in the obese group (median duration of remission, not reached vs. 16 months vs. 2 months vs. 1 month, p = 0.002). In multivariate regression analysis, obesity was independently associated with poor initial and sustained responses, and an increased risk for relapse. In conclusion, obesity is a negative predictor for outcomes of corticosteroid treatment. A stratified strategy according to body mass index status may facilitate the precision management of ITP.     

Acute exacerbation of pulmonary toxoplasmosis during corticosteroid therapy for immune thrombocytopenia: A case report and literature review

Rationale:Pulmonary toxoplasmosis (PT) is an infectious disease that can be fatal if reactivation occurs in the recipients of hematopoietic stem cell transplantation (HSCT) who were previously infected with Toxoplasma gondii. However, whether the toxoplasmosis reactivation is an actual risk factor for patients receiving immunosuppressive therapies without HSCT remains unclear. Therefore, reactivated PT is not typically considered as a differential diagnosis for pneumonia other than in patients with HSCT or human immunodeficiency virus (HIV).Patient concerns:A 77-year-old man presented with fever and nonproductive cough for several days. He was hospitalized due to atypical pneumonia that worsened immediately despite antibiotic therapy. Before 4 months, he was diagnosed with immune thrombocytopenia (ITP) and received corticosteroid therapy. Trimethoprim–sulfamethoxazole (ST) was administered to prevent pneumocystis pneumonia resulting from corticosteroid therapy.Diagnosis:The serological and culture test results were negative for all pathogens except T. gondii immunoglobulin G antibody. Polymerase chain reaction, which can detect T. gondii from frozen bronchoalveolar lavage fluid, showed positive results. Therefore, he was diagnosed with PT.Intervention:ST, clindamycin, and azithromycin were administered. Pyrimethamine and sulfadiazine could not be administered because his general condition significantly worsened at the time of polymerase chain reaction (PCR) examination.Outcomes:The patient died of acute respiratory distress syndrome despite anti-T. gondii treatment. An autopsy revealed a severe organizing pneumonia and a small area of bronchopneumonia.Lessons:PT should be considered as a differential diagnosis in patients with pneumonia, particularly in seropositive patients who receive immunosuppressive therapies even for other than HSCT or HIV.     

A diagnostic approach that may help to discriminate inherited thrombocytopenia from chronic immune thrombocytopenia in adult patients

Inherited thrombocytopenia (IT) is a heterogeneous group of rare diseases that are often confused with immune thrombocytopenia (ITP). The objective of this study was to supply clinicobiological elements that allow a distinction to be drawn between IT and chronic ITP. We then compared 23 adult patients with IT and 9 patients with chronic ITP. Our study revealed six discriminating criteria: (i) an age of discovery <34 years: positive predictive value (PPV) = 88.2% [63.6; 98.5], (ii) a family history of thrombocytopenia: PPV = 100.0% [82.4; 100.0], (iii) a personal history of bleeding: PPV = 100% [76.8; 100.0], (iv) a mean platelet volume >11 fL: PPV = 93.3% [68.1; 99.8], (v) an excess of giant platelets on blood smear: 100.0% [76.8; 100.0], and (vi) a percentage >44% of platelets with a surface area >4 µm² in electron microscopy: PPV = 83.3% [58.6; 96.4]. If at least three of these criteria were combined, it was possible to distinguish IT from chronic ITP with 91.3% [72.0; 98.9] sensitivity and PPV = 100.0% [66.4; 100.0] specificity. The secondary objective of this study was to assess the prevalence of potential IT diagnosis in patients with chronic thrombocytopenia of uncertain origin. Applying our diagnostic approach to a series of 20 cases allowed us to estimate that 40% of them could be suffering from IT. Finally, our diagnostic approach may help to correctly distinguish IT from chronic ITP, particularly in the context of macrothrombocytopenia.     

Targeted-immunosuppression with vincristine infusion in the treatment of immune thrombocytopenia

Abstract Twenty-four patients with immune thrombocytopenia (ITP) were treated with vincristine (VCR) 1.0–2.0mg given as 4-hr I.V. infusions at weekly intervals for four-six weeks; four patients received further infusions, as maintenance therapy, at increasing intervals for up to 12 months. Eight often patients with recent-onset (< 6 months) ITP showed an excellent and sustained response, 7/8 without maintenance therapy. Among the 14 patients with ITP of >6 months' duration, seven showed a good or excellent but only transient response; sustained responses (two good, one partial) were seen only in the three patients who received maintenance therapy. The collective global experience with this novel therapeutic approach of targeted-immunosuppression for ITP is still small, but results to date suggest a promising role for this approach, especially in patients with recent-onset ITP. (Aust NZ J Med 1991; 21: 405–407.)     

The role of genetics in refractory immune thrombocytopenia

Patients with refractory immune thrombocytopenia (rITP) have increased morbidity and mortality. Currently, there is limited understanding of the cause of refractoriness and no markers to help direct novel treatment options. Understanding the reason(s) for refractoriness is crucial to determining novel treatment options. The pathogenesis underlying rITP has generally been thought to be an underlying genetic predisposition with an environmental trigger. Familial ITP remains rare, and there are few twin studies, suggesting that a simple genetic cause is unlikely. However, genetic mutations provide the background for several autoimmune diseases. In this review, we explore the evidence of either an inherited genetic cause of rITP or an acquired mutation, in particular one resulting in clonal expansion of cytotoxic T cells.     

Intramuscular Anti-D treatment for immune thrombocytopenia: A single centre experience

Intravenous Anti-Rhesus-D immunoglobulin (Anti-D) is a first-line treatment option for immune thrombocytopenia in non-splenectomised and RhD-positive patients. In this report, we retrospectively review our experience with intramuscular (IM) Anti-D treatment in 74 adult patients between 1990 and 2018. We found that 73% of patients showed a response; almost all of them had complete responses (68.9%), and 26% achieved complete responses sustained at least 6 months after treatment discontinuation. [Correction added on 02 December 2022, after first online publication: In the preceding sentence, ‘(68.89%)’ has been corrected to ‘(68.9%)’ in this version.] No significant side effects were observed with no cases of acute haemolysis or anaemia reported. We conclude from this study that IM Anti-D is an effective and safe treatment for immune thrombocytopenia.     

连续2个周期大剂量地塞米松治疗成人原发免疫性血小板减少症的疗效和安全性

目的 观察连续2个周期大剂量地塞米松对成人新诊断的原发免疫性血小板减少症(ITP)患者的疗效及安全性.方法 将59例新诊断的ITP患者随机分为两组,地塞米松治疗组30例,地塞米松40 mg/d,连用4 d,7 d后再重复1个周期,以后不进行维持治疗;泼尼松治疗组29例,1.0～1.5 mg·kg-1·d-1口服,连用4周后逐渐减量.观察二组间的近、远期疗效和安全性.结果 近期疗效:治疗后第1、2周地塞米松组有效率明显高于泼尼松组(50.0%比24.1%,73.3%比55.2%,P值分别＜0.01和0.05),治疗后第3周有效率仍高于泼尼松组,但差异无统计学意义(83.3%比68.9%,P＞0.05).远期疗效:随访3个月,除第1个月地塞米松组复发率与泼尼松组差异无统计学意义(16.0%比20.0%,P＞0.05),第2、3个月地塞米松组复发率均明显低于泼尼松组(24.0%比40.0%,32.0%比65.0%,P值分别＜0.05和0.01).地塞米松组不良反应轻微,无1例并发感染及出现库欣综合征.结论 大剂量地塞米松治疗ITP的近、远期疗效均优于常规制量泼尼松且安全性好.

Abstract：

Objective To investigate the efficacy and safety of a schedule of 2 cycles' high-dose dexamethasone (HD-DXM) as an initial therapy in adults immune thrombocytopenia (ITP), and compare with conventional dose prednisone therapy. Method A total of 59 newly diagnosed ITP patients were divided into 2 groups randomly. In 30 patients ( Dexamethasone group), oral HD-DXM was administered at 40 mg/d for 4 consecutive days, repeated one week later, and then failed to maintain. In the remaining 29and then gradually tapered. Results For short-term efficacy, after 1 and 2 weeks of treatment, the response rate in Dexamethasone group was significantly higher than that in Prednisone group (50. 0% vs 24. 1%, P ＜0. 01; 73.3% vs 55.2%, P ＜0. 05 ), while 3 weeks later, there was no remarkable difference between the two groups(83.3% vs 68.9%, P ＞ 0. 05 ), though the response rate in Dexamethasone group remained higher. For long-term effect, at the end of the 2nd and 3rd months of follow-up, the relapse rate in Dexamethasone group was significantly lower than that in Prednisone group(24. 0% vs 40. 0%, P ＜ 0. 05;32.0% vs 65. 0%, P ＜ 0. 01 ), while at the end of the 1st month of follow-up, there was no significant difference( 16. 0% vs 20. 0%, P ＞0.05 ). In addition, it's well tolerated and no complications such as severe infection or Cushing syndrome were complained in Dexamethasone group. Conclusion HD-DXM possesses an advantage over conventional dose prednisone therapy in efficacy and safety.     

益生菌治疗难治性原发免疫性血小板减少症的疗效及对肠道菌群变化的影响

目的 观察益生菌治疗难治性原发免疫性血小板减少症(ITP)的疗效及对肠道菌群变化的影响.方法 将20例难治性ITP患者随机分为益生菌组和对照组,每组10例.益生菌组给予达那唑+环孢素+双歧杆菌乳杆菌三联活菌片口服.对照组给予达那唑+环孢素+维生素C片口服.治疗前及治疗8周后采血用定量酶联免疫吸附试验(ELISA)法检测...     

Resolution of paraneoplastic immune thrombocytopenia following everolimus treatment for metastatic renal cell carcinoma

Autoimmune thrombocytopenia is an uncommon but reported paraneoplastic manifestation of renal cell carcinoma (RCC). Treatment usually involves management of the underlying malignancy; however, steroids have shown a benefit in published case reports. Here, we describe a patient with profound thrombocytopenia secondary to metastatic RCC. It was refractory to steroid and intravenous immunoglobulin, but the platelet count improved markedly following initiation of everolimus. The possible explanation includes immunomodulation, tumour lysis or a combination of both effects. This is the first reported case of everolimus used in paraneoplastic thrombocytopenia from RCC. More studies are needed for further investigation of its potential use in secondary immune thrombocytopenia from RCC and perhaps other malignancies.