小分子血小板生成素受体激动药Eltrombopag

慢性特发性血小板减少性紫癜（ITP）是一种自身免疫性疾病，由致血小板（Pit）减少的一种自身免疫反应引起，其特征是Pit数降低，患者面临出血高风险，往往导致患者小血管出血，症状为皮肤瘀斑、鼻出血和牙龈出血，严重者可出现致命性胃肠道和脑内出血。目前治疗ITP的主要手段是使用免疫抑制类药物，如皮质类固醇、静注免疫球蛋白、抗D免疫球蛋白和利妥昔单抗（Rituximab），而对严重衰竭性病人的最后治疗手段则是行脾切除术。其中大多数治疗药物未曾经历随机安慰剂对照临床试验的考察，虽对不同患者群均有效，但同时也会产生严重毒副作用，其作用机制是阻止血小板破坏，而非促进Pit产生。

Eltrombopag-related renal vein thromboembolism in a patient with immune thrombocytopenia: A case report

BACKGROUND Eltrombopag is an orally administered thrombopoietin receptor agonist linked to a heightened risk of treatment-related thromboembolism.Both venous and arterial thromboses have been documented in the medical literature.CASE SUMMARY In the absence of nephropathy,a 48-year-old patient receiving eltrombopag for immune thrombocytopenia(ITP)developed renal vein thrombosis and pulmonary embolism.The renal vein thrombus spontaneously resolved during subsequent anticoagulant treatment,restoring venous circulation.CONCLUSION A rapid upsurge in platelets,rather than their absolute number,may trigger thrombotic events in this setting.For patients at high thrombotic risk,individualized eltrombopag dosing and vigilance in platelet monitoring are perhaps needed during treatment of ITP.

Eltrombopag in chronic hepatitis C

Chronic hepatitis C is a public health problem worldwide.Unfortunately,not all patients may benefit from antiviral therapy due to thrombocytopenia.Its causes are represented by portal hypertension and platelet sequestration in the spleen,decreased serum levels or activity of thrombopoietin,the bone marrow suppression induced by hepatitis C virus and a possible adverse effect of interferon.Thrombopoietin receptor analogs may contribute to increase platelet counts in these patients.Eltrombopag binds to another region of the thrombopoietin receptor compared to endogenous thrombopoietin and stimulates the proliferation and maturation of megakaryocytes and the platelet production in a dose-dependent manner.Eltrombopag has proven its effectiveness for the treatment of patients with primary immune thrombocytopenia.Its indication for other hemopathies or situations(like thrombocytopenia secondary to chemo- or radiotherapy,acute leukemia,myelodysplastic syndroms,acquired and hereditary bone marrow failure,and platelet donors) is under study.Eltrombopag may be particularly useful in patients with advanced chronic hepatitis or liver cirrhosis who require antiviral treatment.We present a minireview on the results of treatment with eltrombopag in patients chronically infected with hepatitis C virus,highlighting the benefits and mentioning possible adverse effects.In some studies eltrombopag increased the number of virological responses after clasical antiviral treatment of patients with chronic hepatitis C and reduced the transfusional requirements of those who had to be subjected to invasive surgery.Eltrombopag is a solution for many of these patients,which allows them receiving antiviral therapy and sometimes getting a sustained virological response,but they must be well monitored to prevent possible thromboembolic or bone marrow complications or liver failure occurrence.

Eltrombopag Managed Severe Immune Thrombocytopenic Purpura in Pregnancy: A Case Report, Latifa Hospital, DHA, Dubai, UAE

Immune thrombocytopenic purpura (ITP) is an acquired autoimmune disorder, defined by a platelet count of less than 100 × 109/L, secondary to impaired production and immune destruction of platelets. Bleeding tendency is the main presentation of this condition. Clinical symptoms and investigations will confirm the diagnosis. Steroid is the first line of treatment. Although Rituximab and Thrombopoietin receptor agonists are useful second line agents in non-pregnant adults, the data about their role in pregnancy are still limited. We present the case of a 30 year old primigravida, who was a known case of chronic ITP since childhood;the course of her disease was fluctuating, for which oral steroids were used accordingly. She presented with gum bleeding and petechial rash with very low platelets count. She was sponsored by the Patient Support Program and was given Eltrombopag during the third trimester. She responded well to Eltrombopag with no noticeable side effects, neither to the mother nor to the baby so far. Eltrombopag has been assigned Category C by the Federal Drugs Agency (FDA) nevertheless there are no well controlled data in the literature about its role in pregnancy.

Use of eltrombopag in thrombocytopenia of liver disease

Second generation thrombopoietin agonists including eltrombopag and romiplostim act on the thrombopoietin receptor to increase the megakaryocyte production. These agents were needed as use of first generation recombinant products was associated with formation of autoantibodies. Eltrombopag is an oral thrombopoietin agonist found effective in raising platelet counts in patients with immune thrombocytopenia. The drug has now been found to be useful in raising platelet counts in thrombocytopenia related to liver disease including cirrhosis and chronic viral hepatitis. Although the drug may help enable adequate interferon therapy in patients with HCV infection and help carry out invasive procedures in patients with cirrhosis, concerns have been raised of possible thrombotic complications including portal vein thrombosis. Randomized trials have shown that use of eltrombopag concomitant with pegylated interferon and ribavirin increased the chances of sustained virologic response while decreasing the dose reductions of interferon. The data on use of romiplostim in these clinical indications is also emerging. However, in the future, availability of interferon free regimens is likely to decrease the use of eltrombopag for enabling antiviral therapy. The review discusses the role of eltrombopag in management of liver disease related thrombocytopenia in wake of recent data as also the dosage, precautions and adverse effects associated with its use.

Spotlight on eltrombopag concentration in pediatric immune thrombocytopenia:A single-center observational study in China

Importance:Eltrombopag has been recommended for pediatric immune thrombocytopenia(ITP).Response and adverse drug reactions(ADRs)varied widely between individuals,even at the same dose of eltrombopag.The appropriate eltrombopag concentration in ITP has not been reported.Objective:This study aims to explore the appropriate eltrombopag concentration in pediatric ITP.Methods:This was a single-center,prospective cohort study.Children diagnosed with refractory persistent/chronic ITP and platelet count<30×10^(9)/L were treated with eltrombopag and followed up for at least 2 months.Concentration was detected by high-performance liquid chromatography-mass spectrometry at least 2 weeks after eltrombopag.The clinical characteristics-concentration,concentration-response,and concentration-ADRs were analyzed.Results:A total of 30 patients were enrolled,comprising 13 males and 17 females,with a median age of 72(45-94)months.The median dose and concentration were 1.39(1.09-1.56)mg/kg and 2.70(2.25-4.13)mg/L,respectively.Of the enrolled patients,14 responded to treatment,whereas 16 did not.Additionally,five experienced adverse drug reactions.No linear correlation was observed between eltrombopag concentration and clinical characteristics.The concentration was lower in the response group than in the nonresponse group,but there was no significant difference(t=0.755,P=0.457).Patients who experienced ADRs had a higher concentration than those without ADRs(t=2.538,P=0.017).The area under the receiver operating characteristic curve of ADRs was 0.78(95%confidence interval:0.56-1.00).Youden’s index identified the cutoff point as 4.33 mg/L,with a sensitivity of 88%and a specificity of 60%.Logistic regression analysis demonstrated that a higher platelet count before eltrombopag predicted a favorable response.Interpretation:Eltrombopag proves efficacious and well-tolerated for treating pediatric ITP.However,prolonged and high-dose administration may increase the likelihood of ADRs.Thus,examining the appropriate eltrombopag concentration assists in directing individualized management of pediatric ITP.

血小板生成素受体激动剂治疗化疗相关性血小板减少症的研究现状

化疗相关性血小板减少症(chemotherapy-induced thrombocytopenia, CIT)仍是目前肿瘤治疗的严重并发症,既往针对CIT的处理主要为输注血小板、使用重组人白介素-11(recombinanthumaninterleukin-11,rhIL-11)及重组人血小板生成素(recombinanthuman thrombopoietin, rhTPO)等,但对一些难治性CIT,效果有限。近年来,新型血小板生成素受体激动剂(thrombopoietin receptor agonists, TPO-RAs)已上市并用于血小板减少症,但尚未批准用于CIT治疗。本文对TPO-RAs类药物在CIT方面已开展的临床研究进行汇总,结果显示罗米司亭、艾曲泊帕、阿伐曲泊帕研究相对较多,但多为小样本或单中心回顾性分析,而芦曲泊帕和海曲泊帕尚缺乏治疗CIT的临床研究。

用于治疗慢性特发性血小板减少性紫癜药物——Eltrombopag

Eltrombopag是由葛兰素史克公司开发的一个小分子、非肽类血小板生成素（TPO）受体激动剂。商品名为Revolade^POM和Promacta化学名为33’-{2-[1-（3，4-dimethylphenyl）-3-methyl-5-oxo-4，5-dihydro-1H—pyrazol-4-ylidene]hydrazino}-2’-hydroxybipheny1-3-carboxylic acid，分子式为C25H22N4O4，相对分子质量442．47，CAS号496775-61-2，结构见图1。

艾曲泊帕乙醇胺片对特发性血小板减少性紫癜患儿CD40/CD40L的影响

目的探讨艾曲泊帕乙醇胺片对特发性血小板减少性紫癜(ITP)患儿CD40/CD40L轴的影响机制。方法选取80例ITP患儿作为研究对象,根据数字随机表法将其分为对照组(n=40)和研究组(n=40)。对照组给予环孢素治疗,研究组给予艾曲泊帕乙醇胺片治疗,两组均连续治疗3个月。对比两组患者的临床疗效、T淋巴细胞亚群水平(CD3^(+)、CD4^(+)和CD4^(+)/CD8^(+))以及不良反应。检测两组患者淋巴细胞膜表面和血小板膜表面的CD40、CD40L水平,使用ELISA检测血浆sCD40和sCD40L水平。结果研究组的总有效率(92.50%)明显高于对照组的总有效率(75.00%)(P<0.05)。两组治疗后的CD3^(+)、CD4^(+)、CD4^(+)/CD8^(+)、血浆s CD40表达水平均高于治疗前,CD8^(+)以及外周血CD19^(+)CD40^(+)、CD3^(+)CD40L^(+)细胞和血浆sCD40L表达水平低于治疗前;研究组CD3^(+)、CD4^(+)、CD4^(+)/CD8^(+)、血浆sCD40表达水平高于对照组,CD8^(+)以及外周血CD19^(+)CD40^(+)、CD3^(+)CD40L^(+)细胞和血浆sCD40L表达水平低于对照组(P<0.05)。两组治疗后的外周血血小板膜表面的CD40L^(+)细胞低于治疗前;研究组的外周血血小板膜表面的CD40^(+)高于对照组,CD40L^(+)细胞低于对照组(P<0.05)。研究组的总不良率(15.00%)低于对照组的总不良率(22.50%),但是两组比较差异无统计学意义(P>0.05)。结论艾曲泊帕乙醇胺片治疗ITP患儿具有良好的临床疗效,调节T淋巴细胞亚群水平,安全性良好。

艾曲泊帕治疗原发免疫性血小板减少症患者的回顾性研究

目的探究艾曲泊帕治疗原发免疫性血小板减少症(primary immune thrombocytopenia,ITP)患者的回顾性研究。方法回顾性分析2018年10月至2022年2月于笔者医院接受治疗的90例ITP患者的病历资料,根据不同治疗方式,分为地塞米松组和艾曲泊帕组,地塞米松组采用地塞米松治疗,艾曲泊帕组在地塞米松组基础上联合艾曲泊帕治疗(均n=45)。比较免疫功能、治疗效果及不良反应。结果治疗后4周,白细胞介素[(interleukin)-2,IL-2]、干扰素-γ(interferon-gamma,INF-γ)水平比较,艾曲泊帕组及地塞米松组均较治疗前降低,且艾曲泊帕组低于地塞米松组,IL-4水平比较,艾曲泊帕组及地塞米松组较治疗前提高,且艾曲泊帕组高于地塞米松组(P<0.05)。治疗后4周,艾曲泊帕组及地塞米松组血小板(platelet,PLT)计数≥30×10^(9)/L比例分别为68.89%、40.00%,PLT≥50×10^(9)/L比例分别为55.55%、20.00%,艾曲泊帕组各比例均高于地塞米松组(P<0.05),治疗4周内,艾曲泊帕组及地塞米松组1/2时间PLT≥50×10^(9)/L比例分别为66.67%、33.33%,至少1次PLT≥50×10^(9)/L比例分别为93.33%、44.44%,治疗9个月至少1次PLT≥50×10^(9)/L比例分别为60.00%、26.67%,艾曲泊帕组高于地塞米松组(P<0.05)。艾曲泊帕组及地塞米松组不良反应率比较(P>0.05)。结论艾曲泊帕可明显降低ITP患者出血风险,改善免疫功能,提升治疗效果,同时治疗安全性较高。

新型口服抗血小板减少药物——eltrombopag

Eltrombopag（商品名为PROMACTA）足一种血小板生成索（thrombopoietin，TPO）受体激动剂，由葛兰素史克公司研发，2008年11月20日获美国FDA批准上市，用于治疗对糖皮质激素、免疫球蛋白或脾切除术反应欠佳的慢性特发性血小板减少性紫癜（idiopathic thrombocytopenic purpura，ITP）。分了式为C25H22N4O4·2（C2H7NO），相对分子质量564．65。结构式见图1。

艾曲泊帕治疗儿童免疫性血小板减少症的临床综合评价

目的对艾曲泊帕治疗儿童免疫性血小板减少症(Immune thrombocytopenia,ITP)进行临床综合评价,为药品说明书修订、临床合理用药提供循证依据。方法以中文关键词“艾曲泊帕/艾曲波帕/瑞弗兰”“免疫性血小板减少症/特发性血小板减少性紫癜”“儿童”和对应的英文关键词“eltrombopag”“immune thrombocytopenia/idiopathic thrombocytopenic purpura”“child/children”在中国知网、万方、PubMed、Cochrane library等数据库检索从建库至2023年5月的文献。采用文献研究、成本效果研究等方法,比较艾曲泊帕与罗米司亭、海曲泊帕以及重组人血小板生成素(rhTPO)治疗儿童ITP的有效性、安全性、经济性。结果本研究共检索到相关文献613篇,最终纳入符合标准的有效文献14篇。艾曲泊帕在减少出血事件和改善生活质量方面显著优于罗米司亭,并且艾曲泊帕的增量成本效果相较于罗米司亭具有优势。此外,艾曲泊帕在治疗儿童ITP方面并未出现明显有别于罗米司亭的安全性事件。结论艾曲泊帕具有良好的有效性、安全性、经济性。应开展更多的真实世界相关研究,为艾曲泊帕治疗儿童ITP提供更多临床循证依据。

艾曲泊帕致儿童不良反应文献分析

目的分析艾曲泊帕(Eltrombopag)在儿童患者中致不良反应(Adverse drug reaction,ADR)的临床特点和一般规律,为其临床合理、安全使用提供参考。方法检索中国知网(CNKI)、万方、维普(VIP)、PubMed等数据库收录的艾曲泊帕致儿童不良反应相关病例报道,检索时限为建库至2023年5月,并对纳入文献进行统计与分析。结果共纳入文献22篇,应用艾曲泊帕治疗的患儿共800例,ADR共计246例,主要累及消化系统和血液系统,绝大多数ADR为轻症,可自行缓解,或经减停药、对症处理后缓解。结论儿童群体应用艾曲泊帕整体耐受性良好,但仍需加强监测,保障用药安全。

艾曲波帕与利妥昔单抗二线治疗成人原发性免疫性血小板减少症的疗效研究

目的探讨艾曲波帕与利妥昔单抗二线治疗成人原发性免疫性血小板减少症(ITP)的疗效。方法前瞻性选取2021年5月至2024年3月亳州市人民医院血液内科收治的80例成人ITP患者作为研究对象,按照随机数字表法将其分为对照组和试验组,每组各40例。对照组给予利妥昔单抗治疗,剂量为100 mg,每周静脉滴注1次,分别在第1、8、15、22天进行,持续4周。试验组在对照组基础上给予艾曲波帕治疗,最初剂量为每日25~50 mg的空腹口服给药,根据患者的病情、血小板计数以及药物说明书调整给药剂量,最大剂量为每日75 mg,持续12周。比较两组治疗疗效,比较两组患者治疗前、治疗后7 d、治疗后14 d、治疗后1个月、治疗后3个月的血小板计数,比较两组患者治疗前、治疗后3个月的外周血调节性T细胞(Treg)、辅助性T细胞17(Th17)细胞百分比及Treg/Th17水平,两组的不良反应发生率。结果试验组治疗总有效率为72.50%,高于对照组(50.00%),差异有统计学意义(P<0.05)。治疗后7 d、14 d、1个月、3个月,两组患者的血小板计数均高于治疗前,且试验组治疗后3个月的血小板计数为(133.26±40.23)×10^(9)/L,高于对照组[(85.73±22.24)×10^(9)/L],差异均有统计学意义(P<0.05);试验组与对照组治疗后7 d、14 d、1个月的血小板计数比较,差异均无统计学意义(P>0.05)。治疗后3个月,两组患者的外周血Treg及Treg/Th17均高于治疗前,Th17细胞百分比低于治疗前,差异均有统计学意义(P<0.05);且试验组患者治疗后3个月的外周血Treg及Treg/Th17分别为(5.47±0.50)%、(3.42±0.36),均高于对照组[(5.06±0.49)%、2.89±0.40],Th17细胞百分比为(1.60±0.11)%,低于对照组[(1.75±0.16)%],差异均有统计学意义(P<0.05)。对照组的总不良反应发生率(17.50%)与试验组(27.50%)比较,差异无统计学意义(P>0.05)。结论艾曲波帕与利妥昔单抗二线治疗成人ITP疗效显著,可提高患者的血小板计数,改善患者的免疫调节状态,安全性良好。

艾曲波帕联合重组人血小板生成素治疗糖皮质激素无效型免疫性血小板减少症患者的临床疗效

目的探讨艾曲波帕联合重组人血小板生成素(rhTPO)治疗激素无效型免疫性血小板减少症(ITP)患者的临床效果。方法选取浙江大学医学院附属金华医院2020年7月至2023年7月收治的糖皮质激素无效型ITP患者100例,将患者按治疗方法分为rhTPO组以及联合治疗组,各50例,2组患者均采取rhTPO治疗,联合治疗组患者在此基础上联合艾曲波帕治疗,比较2组患者的疗效、治疗后T细胞亚群分布情况、治疗期间血小板计数(PLT)的变化情况、首次PLT≥50×10^(9)/L的时间、治疗前后B细胞的百分比、出血评分、复发情况以及不良反应的发生情况。结果联合治疗组患者的治疗有效率100%(50/50)高于rhTPO组患者有效率[88%(44/50),χ^(2)=6.383,P<0.05];治疗后,联合治疗组患者CD3^(+)T细胞、CD4^(+)T细胞、CD8+T细胞均高于rhTPO组患者,CD4/CD8比值低于rhTPO组患者(P<0.05),治疗1、2、3个月联合治疗组患者的PLT均高于rhTPO组患者(P<0.05),治疗后联合治疗组患者PLT首次≥50×10^(9)/L时间、B细胞的百分比以及出血评分均低于rhTPO组患者(P<0.05),2组患者复发率以及不良反应发生的比较,差异无统计学意义(P>0.05)。结论艾曲波帕联合rhTPO治疗糖皮质激素无效型ITP患者有较好的临床疗效,可以促进患者血小板的提升情况,减少患者的不良反应。

Discovery of the anti-angiogenesis effect of eltrombopag in breast cancer through targeting of HuR protein

HuR(human antigen R), an mRNA-binding protein responsible for poor prognosis in nearly all kinds of malignancies, is a potential anti-tumor target for drug development. While screening HuR inhibitors with a fluorescence polarization(FP) based high-throughput screening(HTS) system, the clinically used drug eltrombopag was identified. Activity of eltrombopag on molecular level was verified with FP, electrophoretic mobility shift assay(EMSA), simulation docking and surface plasmon resonance(SPR). Further, we showed that eltrombopag inhibited in vitro cell proliferation of multiple cancer cell lines and macrophages, and the in vivo anti-tumor activity was also demonstrated in a 4T1 tumor-bearing mouse model. The in vivo data showed that eltrombopag was efficient in reducing microvessels in tumor tissues. We then confirmed the HuR-dependent anti-angiogenesis effect of eltrombopag in 4T1 cells and RAW264.7 macrophages with qRT-PCR, HuR-overexpression and HuR-silencing assays, RNA stability assays, RNA immunoprecipitation and luciferase assays. Finally, we analyzed the in vitro anti-angiogenesis effect of eltrombopag on human umbilical vein endothelial cells(HUVECs) mediated by macrophages with cell scratch assay and in vitro Matrigel angiogenesis assay. With these data, we revealed the HuR-dependent anti-angiogenesis effect of eltrombopag in breast tumor, suggesting that the existing drug eltrombopag may be used as an anti-cancer drug.

Switching between eltrombopag and recombinant human thrombopoietin in patients with immune thrombocytopenia: an observational study

Background:Recombinant human thrombopoietin(rh-TPO)and eltrombopag are two distinct TPO receptor agonists(TPO-RAs)with different mechanisms.During the pandemic,when immunosuppressive medications are controversial,switching to another TPO-RA may be worth exploring in patients who do not benefit from their first TPO-RA.We investigated the outcomes of switching from rh-TPO to eltrombopag or vice versa in immune thrombocytopenia(ITP)patients.Methods:This prospective,open-label,observational investigation included 96 adult ITP patients who needed to switch between rh-TPO and eltrombopag between January 2020 and January 2021 at Peking University People’s Hospital in China.The study evaluated response rates and platelet counts at different time points after the switch,bleeding events,time to response,duration of response,and adverse events.Results:At 6 weeks after switching,response was observed in 21/49 patients(43%)who switched for inefficacy and 34/47 patients(72%)who switched for non-efficacy-related issues.In the inefficacy group,9/27 patients(33%)responded to eltrombopag,and 12/22 patients(55%)responded to rh-TPO.In the non-efficacy-related group,21/26(81%)and 13/21(62%)patients in the eltrombopag and rh-TPO groups maintained their response rates at 6 weeks after switching,respectively.Response at 6 months was achieved in 24/49 patients(49%)switching for inefficacy and 37/47 patients(79%)switching for non-efficacy issues.In the inefficacy group,13/27 patients(48%)responded to eltrombopag,and 11/22 patients(50%)responded to rh-TPO.In the non-efficacy-related group,22/26 patients(85%)and 15/21 patients(71%)in the eltrombopag and rh-TPO groups maintained their response rates at 6 months after switching,respectively.Both eltrombopag and rh-TPO were well tolerated.Conclusions:Our study confirmed the safety and effectiveness of switching between rh-TPO and eltrombopag for ITP patients who had no response to or experienced adverse events with their first TPO-RA.When the switch was motivated by other reasons,including patient preference and platelet count fluctuations,the probability of response was high.Registration:ClinicalTrials.gov,NCT04214951.

艾曲泊帕治疗成人原发免疫性血小板减少症的最新研究进展

原发免疫性血小板减少症(ITP)是一种以血小板减少为特征的自身免疫性疾病,T细胞免疫失调在ITP的形成过程中发挥了重要作用。艾曲泊帕是一种血小板生成素受体激动剂,除直接刺激巨核细胞生成血小板以外,还可通过诱导调节T细胞生成、降低促炎因子发挥免疫调节作用。艾曲泊帕作为成人ITP的二线治疗药物,在临床上的应用日益广泛。本文对艾曲泊帕在ITP中的作用机制、疗效、安全性及如何减量等最新研究进展作一综述。

艾曲泊帕联合大剂量地塞米松治疗原发免疫性血小板减少症患者的临床疗效及安全性分析

目的:研究艾曲泊帕与大剂量地塞米松对原发免疫性血小板减少症患者的治疗效果。方法:选取2022年1—12月平度市人民医院原发免疫性血小板减少症患者56例,采用随机数字表法分为对照组和观察组,每组28例。对照组采取艾曲泊帕联合常规剂量地塞米松治疗,观察组采取艾曲泊帕联合大剂量地塞米松治疗,观察两组治疗总有效率、T淋巴细胞亚群水平、血小板计数达峰时间、血小板增幅、完全反应持续时间、不良反应发生率等差异。结果:观察组治疗总有效率高于对照组,差异有统计学意义(P<0.05)。治疗前,两组T淋巴细胞亚群中,CD3^(+)、CD4^(+)、CD8^(+)比例及CD4^(+)/CD8^(+)比较,差异无统计学意义(P>0.05);治疗后,两组CD3^(+)、CD4^(+)比例及CD4^(+)/CD8^(+)均高于治疗前,CD8^(+)比例低于治疗前,观察组CD3^(+)、CD4^(+)比例及CD4^(+)/CD8^(+)均高于对照组,CD8^(+)比例低于对照组,差异有统计学意义(P<0.05)。观察组血小板增幅大于对照组,血小板计数达峰时间、完全反应持续时间均短于对照组,差异有统计学意义(P<0.05)。两组不良反应总发生率比较,差异无统计学意义(P>0.05)。结论:艾曲泊帕联合大剂量地塞米松联合治疗原发免疫性血小板减少症效果显著,能在较短时间内使患者血小板计数增加,免疫功能得到改善,且具有较高的安全性。