Efficacy and safety of thrombopoietin receptor agonists in children with chronic immune thrombocytopenic purpura: meta-analysis

Immune thrombocytopenic purpura (ITP) is an autoimmune disorder presenting with low platelet count <100 × 10⁹/L. The condition affects both adults and children. Thrombopoietin receptor agonists (TPO-RAs) are second-line of therapy that includes Romiplostim and Eltrombopag, which stimulate the production of normally functioning platelets. Although the biological effect of these drugs is well established, there has not been a meta-analysis in children. To estimate the efficacy and safety of Romiplostim and Eltrombopag, we performed a systematic review and meta-analysis in children with chronic ITP. Systematic literature search was conducted in the following database: PubMed, EMBASE, and Cochrane Central Register of Controlled Trials (CENTRAL). Review Manager 5.3 for Windows was used to analyze the data. Five randomized controlled trials with total of 261 pediatric patients from 1–17 years of age were included. The efficacy and safety analysis showed TPO-RA groups were superior over placebo, and there was no difference in adverse event occurrence between TPO-RA (Romiplostim and Eltrombopag) and placebo groups. The efficacy and safety of Eltrombopag did not differ significantly from those of Romiplostim. Both drugs were effective in treatment of children with chronic ITP. Our findings extend the currently available data on ITP treatment and is helpful for pediatric health providers and for the design of future clinical trials.     

加味小柴胡颗粒及其组方对ITP模型小鼠造血系统的影响

目的:观察加味小柴胡颗粒及其组方对免疫性血小板减少性紫癜(ITP)模型小鼠血小板计数及骨髓巨核细胞的影响。方法:建立ITP模型小鼠,将之分为7组:正常组、模型组、维血宁组、强的松组、加味小柴胡颗粒组、小柴胡颗粒组、丹参三七颗粒组,于造模后第14天开始分组给药,给药体积为30mL/kg体重,每日给药1次,共14天,分别于给药前后称重;给药前、给药后4天、7天及14天检测血小板计数;实验结束剥离股骨取骨髓测巨核细胞计数和分类。结果:ITP模型小鼠体重增长率下降,血小板计数持续下降,骨髓巨核细胞增加,产板巨减少、成熟度下降,有统计学意义(P<0.05)。加味小柴胡颗粒组能提高ITP模型小鼠体重增长率(P<0.05);各给药组均使血小板计数上增,其中加味小柴胡颗粒组对血小板提升作用高于维血宁组及其组方小柴胡颗粒组和丹参三七组,有统计学意义(P<0.05),与强的松组无差别(P>0.05);强的松组、加味小柴胡颗粒、小柴胡颗粒组使巨核细胞数减少,产板巨增高,其中加味小柴胡颗粒组优于小柴胡颗粒组,有统计学意义(P<0.05),与强的松组无差别(P>0.05)。结论:加味小柴胡颗粒能提高ITP模型小鼠体重增长率,并明显提升血小板计数,促使骨髓巨核成熟,效果优于维血宁组,且优于其组方小柴胡颗粒和丹参三七颗粒,与强的松组疗效相似。     

加味小柴胡颗粒及其组方对ITP模型小鼠造血系统的影响

目的：观察加味小柴胡颗粒及其组方对免疫性血小板减少性紫癜（ITP）模型小鼠血小板计数及骨髓巨核细胞的影响。方法：建立ITP模型小鼠，将之分为7组：正常组、模型组、维血宁组、强的松组、加味小柴胡颗粒组、小柴胡颗粒组、丹参三七颗粒组，于造模后第14天开始分组给药，给药体积为30mL／kg体重，每日给药1次，共14天，分别于给药前后称重；给药前、给药后4天、7天及14天检测血小板计数；实验结束剥离股骨取骨髓测巨核细胞计数和分类。结果：ITP模型小鼠体重增长率下降，血小板计数持续下降，骨髓巨核细胞增加，产板巨减少、成熟度下降，有统计学意义（P〈0，05）。加味小柴胡颗粒组能提高ITP模型小鼠体重增长率（P〈0．05）；各给药组均使血小板计数上增，其中加味小柴胡颗粒组对血小板提升作用高于维血宁组及其组方小柴胡颗粒组和丹参三七组，有统计学意义（P〈0．05），与强的松组无差别（P〉0．05）；强的松组、加味小柴胡颗粒、小柴胡颗粒组使巨核细胞数减少，产板巨增高，其中加味小柴胡颗粒组优于小柴胡颗粒组，有统计学意义（P〈0．05），与强的松组无差别（P〉0．05）。结论：加味小柴胡颗粒能提高ITP模型小鼠体重增长率，并明显提升血小板计数，促使骨髓巨核成熟，效果优于维血宁组，且优于其组方小柴胡颗粒和丹参三七颗粒，与强的松组疗效相似。     

婴儿急性特发性血小板减少性紫癜与巨细胞病毒感染的相关性探讨

目的探讨婴儿急性特发性血小板减少性紫癜(AITP)与巨细胞病毒(CMV)感染的相关性、临床特点及治疗。方法对于AITP患者,应用ELESA方法测CMVIgG、IgM,对仅CMVIgG阳性者,再予CMVDNAPCR检测,同时予肝功能检测。对于CMVIgG或CMVDNAPCR阳性而无肝功能损害,均予地塞米松(Dex)+静注人血丙球(IVIG)常规治疗,而对同时有肝功能损害者分析予Dex+IVIG+阿托莫兰或Dex+IVIG+阿托莫兰+更昔洛韦(GCV)治疗。结果仅CMV感染而无肝功能损害与非CMV感染的AITP患者经正规治疗后,两组血小板升至正常时间差异无显著性,而肝功能异常时,两组血小板升至正常及肝功能恢复时间有显著性差异。结论婴儿AITP与CMV感染密切相关,若合并有其它脏器损害需联合应用GCV治疗,否则只需常规治疗。     

难治性特发性血小板减少性紫癜治疗的探讨

目的观察脾动脉栓塞和骁悉（MMF）治疗难治性特发性血小板减少性紫癜（R-ITP）的临床意义。方法32例R—ITP患者中23例采用介入方法进行大部分脾动脉栓塞术，9例服用骁悉连服3个月，并与环孢霉素A（CsA）治疗34例R—ITP疗效对照。结果脾栓塞术组所有患者血小板都较术前有不同程度上升，12例显效，4例良效，5例进步，2例无效，有效率为69．6％。骁悉组3例显效，2例良效，1例进步，2例无效，有效率为66．7％。CsA组17例显效，8例良效，6例进步，3例无效，有效率为73．5％。3种治疗方法有效率无显著差异。结论脾动脉栓塞、口服骁悉治疗R—ITP的疗效与CsA同样满意，且副作用小，值得临床推广应用。     

人细小病毒B19感染与小儿特发性血小板减少性紫癜

目的探讨小儿特发性血小板减少性紫癜(ITP)与人细小病毒B19(HPVB19)感染的关系.方法用ELISA技术对102例ITP患儿进行人HPVB19特异性IgM的检测.结果 102例ITP患儿中HPVB19-IgM阳性41例(40.20%),30例正常对照HPVB19-IgM阳性2例(6.67%),ITP组HPVB19感染率明显高于正常对照组,两组比较有显著性差异(P＜0.05).结论人细小病毒B19(HPVB19)与小儿 ITP 的发生密切相关.     

Duration and morbidity of newly diagnosed idiopathic thrombocytopenic purpura in children: A prospective Nordic study of an unselected cohort

OBJECTIVE: To determine the duration of the risk period with platelet counts <20 x 10(9)/L and the frequency of bleeding episodes in unselected children with idiopathic thrombocytopenic purpura (ITP). STUDY DESIGN: We established a registry for patients with newly diagnosed ITP in the five Nordic countries, enrolling children aged 0 to 14 years with platelet counts <30 x 10(9)/L. Treatment centers prospectively reported presenting features, management details, and disease-related events during the first six months after diagnosis. RESULTS: At presentation (n=501), more than half of the children had a platelet count <10 x 10(9)/L, but only 15 (3.0%) had a hemorrhage requiring blood transfusion. During follow-up of 409 patients, thrombocytopenia resolved uneventfully in 277. A risk period was present in 376 cases. Among 283 with self-limiting ITP, 26 were at risk >1 month and 25 had 30 events. Among 93 patients with chronic ITP, 73 were at risk >1 month and 44 had 111 events. Events occurred with an average frequency of 0.39 per month at risk. Life-threatening hemorrhages did not occur in the first six months after diagnosis. CONCLUSION: Most children with ITP are at risk for serious bleeding for less than one month. Continuing severe thrombocytopenia is associated with little morbidity, bleeding episodes being infrequent and very rarely serious.     

Serum Immunoglobulin Levels at Onset: Association with the Prognosis of Childhood Idiopathic Thrombocytopenic Purpura

Between 1981 and 2000, 87 patients with new diagnoses of idiopathic thrombocytopenic purpura (ITP) were admitted to the pediatric department of Kobe City General Hospital or Nishi-Kobe Medical Center. The patients' clinical records were analyzed for the relationships of disease outcome to serum immunoglobulin levels and other factors, including sex, onset age, and initial platelet counts. The disease of 22 patients became chronic, and of the 65 patients with an acute form of the disease, 27 exhibited levels of immunoglobulin G (IgG), IgA, or IgM above the 97.5% confidence limits of the age-matched control subjects. However, only 2 patients with the chronic form of the disease showed elevated serum immunoglobulin levels. The presence of antecedent specific viral infections was also associated with the acute disease form. In predicting the prognosis of childhood ITP, high serum immunoglobulin levels at initial presentation can be considered a good prognostic marker for the acute form of the disease.     

Initial management of immune thrombocytopenic purpura in adults: a randomized controlled trial comparing intermittent anti-D with routine care

We conducted a randomized clinical trial in adults with a new diagnosis of ITP and a platelet count <30000/muL to test the hypothesis that initial intermittent treatment with anti-D may avoid or defer the need for splenectomy when compared to current routine care (glucocorticoid treatment, followed by splenectomy). Splenectomy was to be performed in the anti-D group if patients failed to respond to three consecutive anti-D treatments given within 10 days. The incidences of splenectomy were 14 of 37 (38%) in the routine care group and 14 of 33 (42%) in the anti-D group (absolute risk reduction = 4.6% in favor of the routine care group, 95% CI, -18.4 to 27.6%). However, splenectomy was performed prematurely, not according to the protocol, in 11 of 14 patients in the anti-D group. The median time to splenectomy was 36 days (range, 9-78) in the routine care group and 112 days (range, 19-558) in the anti-D group (P = 0.045 at 100 days after randomization, P = 0.840 at 1 year after randomization, using log-rank analysis). Patients in the anti-D group were treated with prednisone for fewer days (70 days) compared to the routine care group (112 days, P = 0.01). No major bleeding events occurred. In this study, initial treatment of patients with intermittent anti-D initially deferred splenectomy. Whether our aggressive regimen of anti-D could have prevented splenectomy if it had been adhered to in all patients remains uncertain. However, compliance with this anti-D regimen was not feasible for many patients and/or their physicians.