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.     

立足中国实际的原发免疫性血小板减少症诊治——2020版成人原发免疫性血小板减少症诊断与治疗中国指南解读

原发免疫性血小板减少症(primary immune thrombocytopenia,ITP)是临床最为常见的获得性自身免疫性出血性疾病,以缺乏明确特异病因的孤立性血小板减少为特征.随着国内外对ITP发病机制及治疗领域的不断探索,ITP的诊治受到广泛关注,成为热点领域之一.依据近年来ITP诊治领域最新循证医学证据进展...     

重组血小板生成素联合地塞米松治疗原发免疫性血小板减少症的疗效

目的探讨重组血小板生成素(rh TPO)联合地塞米松治疗原发免疫性血小板减少症(ITP)的临床效果。方法选择2012年2月至2014年3月在该院接受治疗的60例ITP患者。以数字法随机分成观察组和对照组各30例,观察组予地塞米松联合rh TPO治疗,对照组单纯使用地塞米松治疗,1个疗程(28 d)后比较两组临床疗效、血小板水平变化及不良反应情况。结果观察组的总有效率〔93.33%(28/30)〕显著优于对照组〔73.33%(22/30)〕(χ2=7.283,P=0.007)。两组在治疗前、治疗1~7 d的血小板水平差异均无统计学意义(t=0.432,0.588;P=0.682,0.312);治疗后7~14 d、14~28 d观察组血小板水平均显著高于对照组(t=13.981,12.925;均P=0.000)。观察组不良反应率为23.33%(7/30),对照组为16.67%(5/30),两组比较差异无统计学意义(χ2=2.857,P=0.091)。结论 rh TPO联合地塞米松治疗ITP疗效显著,不良反应少,值得推广。     

原发免疫性血小板减少症的诊疗进展

原发免疫性血小板减少症(ITP)既往称为特发性血小板减少性紫癜,是一种以血小板减少为独立特征的自身免疫性疾病,是临床出血性疾病的常见病因之一。患者无明显临床症状、体征或有皮肤黏膜出血,乏力是该病最为常见的非出血表现。近年来ITP的基础及临床研究得到进一步发展,为开发新的治疗靶点提供了理论依据。现对近年来ITP诊断和治疗的进展进行综述。     

慢性成人原发免疫性血小板减少症的治疗进展

原发免疫性血小板减少症(immune thrombo-cytopenia,ITP)是一种获得性自身免疫性疾病,其特征在于血小板破坏过多和(或)血小板生成受损导致血小板计数低;发生率为(2～5)/10万人,可以是孤立的原发疾病,也可以由其他疾病继发产生.ITP按疾病持续时间可分为:新诊断(0～3个 月),持续(3～12个...     

低剂量地西他滨治疗成人难治复发原发免疫性血小板减少症的临床研究

目的:探讨低剂量地西他滨治疗成人难治复发性原发免疫性血小板减少症(ITP)的临床疗效、起效时间、疗效维持时间及不良反应。方法:采用同期非随机对照的前瞻性研究方法,将46例成人难治复发性ITP患者分为试验组和对照组。试验组21例,采用低剂量地西他滨治疗:3.5 mg/(m²·d),连用3 d,每4周用药1次;对照组25例,给予达那唑治疗;治疗后比较2组的疗效及不良反应。结果:试验组21例患者治疗后完全反应5例(23.81%),有效6例(28.57%),总有效率52.38%(11/21);对照组25例患者治疗后完全反应1例(4.00%),有效8例(32.00%),总有效率36.00%(9/25);2组疗效比较差异有统计学意义(P<0.05)。试验组中位起效时间4(2～10)周,较对照组起效时间[10(8～18)周]明显缩短(P<0.05)。2组患者疗效维持时间均多在3～12个月。试验组较对照组有更少的不良反应,耐受性更好。结论:低剂量地西他滨治疗成人难治复发性ITP有一定疗效,不良反应可耐受。     

成人原发免疫性血小板减少症的治疗进展

为不断提高学术水平及推广疾病诊治新理论、新疗法、新技术,进一步增强杂志和广大读者的紧密联系,从本期开始,本刊将不断强化专家笔谈这一栏目。本栏目将在每一期设立1～2个重点,邀请国内外知名专家,特别是工作在临床一线的中青年学者,就相关疾病诊断、治疗中的前沿问题、热点问题、争议问题及困难问题进行讲述、介绍和争鸣,以期引起更多的关注和讨论。本期我们特邀侯明、杨仁池、张广森、郭涛4位中青年专家,就出血性疾病中最为常见的免疫性血小板减少症的诊断、治疗中的一些问题,作了精辟的介绍。编者读后,深感获益匪浅。我们深信,这一栏目将会受到全国临床血液病工作者的欢迎,将有更多同道参与进来。     

MicroRNAs在原发免疫性血小板减少症中作用研究进展

<正>原发免疫性血小板减少症(primary immune thrombocytopenia,ITP)是临床上常见的一种以皮肤黏膜出血为主要表现的获得性出血性疾病,占出血性疾病的1/3,严重者可发生内脏出血、颅内出血等并发症[1]。ITP的发病机制非常复杂,迄今为止具体机制仍不明确[2-3]。目前研究公认的发病机制是患者对自身抗原免疫失耐受,从而导致自身免疫介导的血小板破坏过多及巨核细胞生成血小板不足[4]。MicroRNAs(miRNAs)     

成人原发免疫性血小板减少症诊断与治疗中国指南(2020年版)解读

原发免疫性血小板减少症( ITP)是血液科最常见的出血性疾病,由机体免疫紊乱介导的血小板破坏增加和生成减少所致.随着对ITP发病机制及临床研究的不断深入,该病诊疗进展迅速.时隔十年,美国血液学会发布了关于成人ITP的诊疗新指南[1] ,同时ITP国际工作组也发布了关于ITP诊断治疗的更新版共识报告( ICR) [2]....     

老年原发免疫性血小板减少症诊治的研究进展

原发免疫性血小板减少症(ITP)以皮肤黏膜出血为起病时的主要临床表现,老年人是ITP的高发人群之一。目前尚无关于老年ITP的指南与专家共识。老年ITP作为成人ITP的特殊群体,在诊断、鉴别诊断及治疗等方面,既有与成人ITP的共性,也有其特殊性。老年ITP的治疗需要明确治疗目标并合理掌握启动治疗的时机,使血小板计数达到安全水平而不是正常水平。老年ITP的一线治疗方案包括糖皮质激素和免疫球蛋白,二线治疗方案包括促血小板生成素、利妥昔单抗、脾切除术等。老年ITP的治疗应遵循个体化原则,在最小化治疗措施不良反应的基础上提升血小板计数至安全水平,减少出血事件。     

丙戊酸联合小剂量化疗治疗中高危骨髓增生异常综合征的临床观察

目的 观察丙戊酸(VPA)联合小剂量化疗治疗中高危骨髓增生异常综合征(MDS)的疗效和安全性.方法 回顾性分析2007年6月至2009年6月41例初治MDS患者,19例采用小剂量化疗,22例采用VPA联合小剂量化疗.小剂量化疗方案:中危组采用静脉滴注高三尖杉酯碱1～2mg/d,14～28 d;或皮下注射阿糖胞苷15 mg/m2,每12小时1次,14～21 d;或阿克拉霉素5～7 mg·m-2·d-1,1～8 d,15～23 d;中性粒细胞缺乏时,皮下注射粒细胞集落刺激因子(G-CSF)200μg·m-2·d-1.高危组采用以上化疗药物中任意两种组合化疗.结果 VPA组完全缓解(CR)9例(40.9%),总有效率为77.2%(17/22).对照组CR 6例(31.6%),总有效率为47.4%(9/19).VPA组总有效率高于对照组(P＜0.05),但CR率比较差异无统计学意义(P＞0.05).结论 VPA联合小剂量化疗治疗中高危MDS安全有效,长期疗效仍需进一步观察.

Abstract：

Objective To evaluate the efficacy and adverse effect of valproic acid (VPA) in combination with low dose chemotherapy on intermediate and high-risk myelodysplastic syndrome. Methods A total of 41 patients with intermediate (34) and high-risk (7) myelodysplastic syndrome were retrospectively analyzed. Among them, 19 patients received low dose chemotherapy regimen and 22 received low dose chemotherapy plus VPA.Low dose chemotherapy regimen included: homoharringtonine,1-2 mg·m-2·d-1 intravenously,14-28 d; clarubicin,5-7 mg·m-2·-1 intravenously,1-8 d,15-23 d;cytarabine 15 mg/m2 subcutaneously once every 12 h, 14-21 d; and subcutaneously use of granulocyte colony-stimulating factor 200 μg·m-2·d -1 when neutrophil deficiency.The outcome and adverse effect were recorded after the treatment. Results The overall response rate in the low dose chemotherapy regimen group was 47.4% (9/19), 6 patients (31.6%) achieved complete response (CR). The overall response rate in the VPA group was 77.2% (17/22), 9 patients (40.9%) achieved CR. The overall response rate of the low dose chemotherapy in combination with VPA group was significantly higher than that in the low dose chemotherapy group (P＜0.05) while no difference was found in CR rate. The adverse effect of the low dose chemotherapy in combination with VPA regimen was tolerated. Conclusion With acceptable adverse effect, the low dose chemotherapy in combination with VPA regimen is effective for the treatment of intermediate and high-risk myelodysplastic syndrome. Long-term outcome needs further investigation.     

The comparison of gammaglobulin to steroids in treating adult immune thrombocytopenia

Summary Symptomatic immune thrombocytopenia is a life-threatening situation which is conventionally treated in the adult with prednisone, although subsequent splenectomy is frequently unavoidable. Recently, high-dose intravenous gammaglobulin has been reported to be an effective alternative option, particularly in children. To determine the role of this agent in adults a controlled prospective trial has been undertaken. Previously untreated patients with immune thrombocytopenia were randomised to compare oral prednisone (1 mg/kg/day: Group 1: n=13) to high-dose intravenous gammaglobulin (400 mg/kg on days 1 through 5: Group 2: n=7), or a combination of both agents given on the same schedule (Group 3: n=12). The time from diagnosis to commencement of treatment, initial platelet counts, age and sex were comparable in the three groups. At this interim analysis there has been no mortality, but one patient has suffered a cerebrovascular accident. Objective response, defined as a platelet count greater than 50×10⁹/l, was achieved in a median of 5, 5 and 3 days, whereas the time taken to reach peak counts were 9, 5 and 7 days, respectively. The relapse rates, percentage of patients subsequently requiring splenectomy for control of symptomatic bleeding and the postoperative course was comparable between the three groups. These data, although preliminary, re-emphasize differences between the paediatric and adult forms of immune thrombocytopenia and also suggest in the latter patients a need for caution before advocating replacement of prednisone by gammaglobulin as the primary form of treatment.Supported by the University of Cape Town Leukaemia Centre and Staff Research Fund, the Gwendoline Moore Trust, the Medical Research Council, the National Cancer Association, the Michael Chanani and Kaliski Bequests, Cape, South AfricaPresented at the International Workshop on ITP, August 26 and 27, 1988, Lucerne, Switzerland     

Reduced corticosteroid use in adult patients with primary immune thrombocytopenia receiving romiplostim

Abstract

**Adult patients with primary immune thrombocytopenia requiring first-line treatment typically receive corticosteroids, which are associated with low response rates and many potential side effects. In a retrospective analysis of two 6-month, placebo-controlled, phase III trials, corticosteroid use decreased from 30 to 26% among patients treated with the novel thrombopoietin-mimetic romiplostim (n?=?83) and remained above 30% for placebo-treated patients (n?=?42). Moreover, compared to placebo, patients were spared 7 weeks of corticosteroid treatment for every 100 weeks of romiplostim treatment. Thereafter, corticosteroid use continued to decrease significantly, from 35 to 20%, in patients treated with romiplostim for up to 3 years in an open-label extension study (n?=?101), and patients were spared a further 8 weeks of corticosteroid treatment for each additional 100 weeks of romiplostim treatment. Such reductions in corticosteroids may improve health-related quality of life in patients with primary immune thrombocytopenia.     

Low‐dose rituximab in adult patients with primary immune thrombocytopenia

Backgrounds: Rituximab 375 mg/m² weekly for 4 wks has significant activity in adults with primary immune thrombocytopenia (ITP). In this setting, several evidences support the possible use of lower doses of rituximab. Objectives: To investigate the activity of low‐dose rituximab as salvage therapy in previously treated symptomatic ITP. Methods: Forty‐eight adult patients were treated prospectively with rituximab 100 mg weekly for 4 wks. Results: Overall and complete responses (CR) (platelet level ≥ 50 and 100 × 10⁹/L) were 60.5% and 39.5%, respectively. In responders, the median time to response was 35 d (range: 7–112 d). The median time of observation was 18 months (range 3–49 months). Sixteen of 29 responding patients (55%) relapsed and 14 needed further treatments. The 12‐ and 24‐month cumulative relapse‐free survival was 61% and 45%, respectively. In univariate analysis, CR rate was in inverse relation with weight OR = 0.95, CI_95% [0.91; 0.99] (P = 0.019) and age OR=0.96, CI_95% [0.93; 0.99] (P = 0.047). Cox regression model showed that relapse probability increases as weight (HR = 1.06, CI_95% [1.0031; 1.111]) and period between diagnosis and rituximab therapy (HR = 1.01, CI_95% [1.002; 1.017]) increase. One patient developed an interstitial pneumonia 1 month after the end of rituximab treatment. No other infectious, hematologic or extra‐hematologic complications were documented during follow‐up. Conclusions: Low‐dose rituximab is active in ITP but has moderate long‐term effect. A comparative study with standard dose is warranted.     

小剂量奥曲肽治疗肝硬化顽固性腹水33例临床疗效及安全性分析

探讨小剂量奥曲肽治疗肝硬化顽固性腹水的疗效及安全性。方法将符合肝硬化顽固性腹水诊断的住院患者随机分为两组,给予对照组利尿、补充白蛋白及支持等常规治疗,治疗组在常规治疗基础上加用奥曲肽注射液0.1 mg 皮下注射,1次/8 h,疗程为2 w。分别观察两组治疗前后患者的症状,记录其体质量、腹围、24 h尿量及血清钠、钾、空腹血糖、尿素氮及血肌酐（serum creatinine,sCr）的变化。结果在治疗结束时,33例治疗组患者总有效率达63.6%,28例对照组患者为46.4%（P〈0.05）;治疗组患者体质量、腹围和24 h尿量分别为（62.1±5.7） kg、（86.0±7.4） cm和（2478±512） ml,而对照组则分别为（68.3±5.6）kg、（92.6±5.3） cm和（1860±230） ml （P〈0.05）;两组空腹血糖、电解质及肾功能比较无显著性差异（P〉0.05）。结论小剂量奥曲肽可提高肝硬化顽固性腹水治疗的疗效,且副反应少,安全性高。     

Dapsone salvage therapy for adult patients with immune thrombocytopenia relapsed or refractory to steroid and rituximab

Dapsone is an antibacterial sulfonamide with anti-inflammatory property, which showed therapeutic activity in patients with immune thrombocytopenia (ITP); the activity in patients who showed refractoriness to rituximab is unknown. We evaluated the effect of dapsone in 20 consecutive adult patients, median age 51 years, with primary ITP previously treated at least with steroids and rituximab. Median baseline platelet count was 19 × 10?/L, and the median interval between diagnosis of ITP and dapsone therapy was 46 months. Response (platelet count ≥ 30 × 10?/L) and complete response (CR; platelet count ≥ 100 × 10?/L) were 55 and 20%, respectively; median time to response (TTR) was 1 month. All responders were able to interrupt any other specific anti-ITP treatment. The median duration of dapsone therapy in responders and the median response duration were 31 and 42 months, respectively. None of responders lost response during treatment. One patient in CR interrupted dapsone after 9 months and still maintained the response after 48 months. None of the patients interrupted the treatment for toxicity. All the patients were screened for normal glucose-6-phosphate-dehydrogenase (G6PD); two patients showed mild increase of methemoglobin (MHb). These results highlight the therapeutic activity and good safety profile of dapsone in patients with ITP who previously failed rituximab treatment.     

Long-term safety and efficacy of rituximab in 7 Japanese patients with ANCA-associated vasculitis

Abstract

Objectives. The safety and efficacy of rituximab were examined in a multicenter open-label pilot study in patients with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) in Japan.

Methods. Patients with refractory AAV were administered a rituximab infusion at a weekly dose of 375 mg/m² for 4 weeks. All patients also received oral daily prednisolone. The primary outcome was complete remission, which was defined as a Birmingham Vasculitis Activity Score (BVAS) of 0 or 1.

Results. The mean age of the 7 patients was 57 (range, 34–71) years. The mean follow-up period after rituximab treatment was 62.9 (range, 4.8–81) months. The mean BVAS at entry was 16.7 (range, 2–34). Complete remission occurred in all cases, except in 1 case in which the patient died, with a significant decline in BVAS from baseline at 12 months after initiation of rituximab. Rituximab reduced granulomatous orbital involvement in a patient with granulomatosis with polyangiitis. Relapse occurred in five patients. Adverse events included de novo hepatitis B in one patient, cancer (hepatocellular carcinoma and prostate cancer) in two patients, and transient visual disturbance, atypical mycobacterial infection, urinary tract infection, sepsis, and cytomegalovirus infection. Two patients died due to recurrent infections and airway obstruction, caused by an AAV lesion.

Conclusions. Rituximab had a beneficial effect on refractory AAV in Japanese patients, but several adverse effects occurred during rituximab treatment.     

The efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia

正Objective To investigate the safety and efficacy of eltrombopag for adult patients with chronic immune thrombocytopenia(cITP).Methods It was a randomised,single-centre,6 weeks,placebo-controlled study.Beginning on January 29~(th),2013,35 patients were enrolled,and the trial was completed on May 16~(th),