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Objective@#To analyze the gene mutation spectrum, clinical features, and the factors of disease progression and prognosis in patients with essential thrombocytosis (ET) .@*Methods@#A retrospective analysis was conducted on 178 newly diagnosed ET patients admitted from February 1st, 2009 to November 1st, 2018.@*Results@#Of the 178 patients, 89 were male and 89 female, and the median diagnosis age was 49.5 (3-86) years old. JAK2V617F, CALR and MPL mutations frequencies were 16.45% (1.67%-43.90%) , 40.00% (10.00%-49.15%) and 25.10% (25.00%-40.00%) , respectively. Compared with patients with CALR mutations, patients with JAK2V617F mutation had higher diagnosis age (P=0.035) , higher white blood cell count (P=0.040) , higher hemoglobin concentration (P=0.001) , and lower platelet count (P=0.002) , respectively. Of them, 47 patients (27.01%) developed thrombotic events before diagnosis, and 3 ones (1.72%) experienced thrombotic events after diagnosis. Multivariate analysis revealed age >60 years (P=0.013, OR=4.595, 95%CI 1.382-15.282) and cardiovascular risk factors (CVF) (P<0.001, OR=8.873, 95%CI 2.921-26.955) as risk factors for thrombotic events, CALR mutation (P=0.032, OR=0.126, 95%CI 0.019-0.838) as a protective factor for thrombotic events. Age >60 years (P=0.042, OR=4.045, 95%CI 1.053-15.534) was found to be a risk factor for the overall survival (OS) of ET patients. OS of age ≤60 years and age>60 years were calculated by Kaplan-Meier analysis to be (115.231±1.899) months and (83.291±4.991) months (χ2=6.406, P=0.011) , respectively.@*Conclusion@#Age>60 years and CVF were risk factors for thrombotic event. CALR mutation was a protective factor for thrombotic event. Age >60 years was a risk factor for OS in ET patients.
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Objective@#To evaluate the efficacy and safety of eltrombopag in the treatment of pediatric primary immune thrombocytopenia (ITP) .@*Methods@#The clinical characteristics of 23 pediatric ITP patients who received eltrombopag from May 2015 to March 2019 were retrospectively analyzed. Eltrombopag started with an initial dose of 12.5-50.0 mg/d and the maximum dose was 75.0 mg/d.@*Results@#Among 23 children, there were 11 boys and 12 girls with median age 11.0 (2.0-17.0) years. Four cases were newly diagnosed ITP, the other 8 of persistent ITP and 11 of chronic ITP. The duration of eltrombopag application ranged from 4.5 to 95 weeks (8/23 still ongoing) . The median platelet (PLT) counts at 2 weeks, 4 weeks, 3 months and the 6 months after treatment were 40 (4-170) ×109/L, 20 (4-130) ×109/L, 60 (4-110) ×109/L, and 70 (18-160) ×109/L, which were all significantly higher than that before treatment 14 (2-82) ×109/L (z=-3.440, P=0.001; z=-1.964, P=0.049; z=-4.339, P<0.001;z=-5.794, P<0.001 respectively) . The overall response rate was 60.87% (14/23 cases) . The median time to PLT count ≥30×109/L was 10.5 (3-42) days. Seven patients (30.43%) responded within the first week, and 10 cases (43.48%) achieved PLT counts ≥30×109/L within 2 weeks. All patients were divided into three groups according to the age (<6 years old, 6-12 years old, 13-17 years old) . The response rates were similar in three groups, as 33.33%, 60.00%, 85.71%, respectively. WHO bleeding scores as 0, 1, 2 were corresponded to 4, 12 and 7 patients before treatment. Patient numbers changed to 13, 7, 3 with bleeding scores 0, 1, 2 respectively after treatment (χ2=7.558, P=0.006) . Eltrombopag was well tolerated, the common adverse events included elevated transaminase (4 cases) and serum bilirubin (4 cases) ; mild nausea (1 case) , vomiting (1 case) and dizziness (1 case) . No drug withdrawal occurred due to adverse events.@*Conclusion@#Eltrombopag is safe and effective in pediatric patients with primary ITP.
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Objective@#To probe the incidence and risk factors for thrombosis in Chinese immune thrombocytopenia through a retrospective analysis of the inpatients referred to the Blood Disease Hospital, CAMS & PUMC.@*Methods@#A retrospective survey of 3 225 patients with ITP from October 2005 to December 2017 was performed, the clinical data of the patients with thrombosis were collected to analyze the causes, diagnosis, treatment and prognosis.@*Results@#A total of 46 patients experienced a thrombotic event with a prevalence of 1.43%(46/3 225 cases) with the median age of thrombosis as 54 years (26-83) years, the prevalence of thrombosis was 3.37% (40/1 187 cases) in>40 years old, which was significantly higher than 0.58% (6/1 030 cases) in those under 40 years old, in adults (P=0.00). There were 20 males and 26 females, there was no statistical difference in the incidence of thrombosis between males and females[1.53% (20/1 309) vs 1.36% (26/1 916), P=0.187]; The prevalence of arterial thrombosis was 1.12% (36/3 225) higher than venous thrombosis[0.22% (7/3 225), P=0.00]when 82.61%(38/46 cases) of patients with PLT<100×109/L. Post-splenectomy are risk factors for thrombosis in ITP patients, P values was 0.022, There was no statistical difference in the presence or absence of thrombotic events whether received glucocorticoid or TPO/TPO-Ra treatment, the P values were 0.075 and 0.531, respectively.@*Conclusions@#In Chinese population, ITP disease maybe with a higher risk of thrombosis, there was no positive correlation between thrombosis and platelet level; and had obvious age distribution characteristics. The history of tobacco, hypertension, diabetes and post-splenectomy are risk factors for thrombosis in ITP patients.
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Objective@#To evaluate the efficacy and safety of a domestic human plasma derived coagulation Factor Ⅸ concentrate (pd-FⅨ) in patients with hemophilia B.@*Methods@#The study was a multicenter, open-label and single-arm study. The efficacy of pd-F Ⅸ was evaluated by objective performance criteria. The doses of pd-FⅨ were calculated according to the bleeding symptom and disease severity. The infusion efficiency of pd-FⅨ and improvement of bleeding symptoms were measured at 30 minutes and (24±4) h after the first infusion, respectively. Adverse events were recorded. Viral infection and FⅨ inhibitor were detected 90 d after the first infusion.@*Results@#All 36 subjects with hemophilia B were enrolled in the study. The median age of these patients was 31 years old and the median injection doses were 4 (1-17) times. The hemostatic effect of 27/36 (75.00%) and 9/36 (25.00%) acute bleeding events were rated as "excellent" and "better" , respectively. The recovery rate was 111.92% (65.55%-194.28%) at 30 minutes after infusion of FⅨ. There was no adverse event related to FⅨ. No reactivation of HBV, HCV or HIV and FⅨ inhibitor was detected at 90-104 d after the first FⅨ infusion.@*Conclusion@#This domestically made human plasma derived FⅨ concentrate is safe and effective in the treatment of acute bleeding in patients with hemophilia B.@*Clinical trial registration@#China food and Durg Administration, 2016L08027.
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Objective@#To evaluate the applicability of Chinese disseminated intravascular coagulation scoring system (CDSS) in the diagnose of DIC in patients with acute promyelocytic leukemia (APL) patients. @*Methods@#Medical records of 220 APL patients diagnosed and receiving induction therapy in Blood Disease Hospital, CAMS & PUMC from January 2004 to February 2018 were retrospectively analyzed. Each patient was evaluated by CDSS, the International Society of Thrombosis and Haemostais (ISTH) scoring system for overt DIC and Japanese Ministry of Health and Welfare (JMHW) scoring system for overt DIC, respectively. @*Results@#A total of 220 APL patients were enrolled in the study, with a median age of 38.5 (12-70) years, 114 male and 106 female. Among them, 173 were in the low-medium risk group, 47 high-risk group; 11 patients died during induction treatment. The positive rates of DIC diagnosed by CDSS criteria, ISHT criteria, JMHW criteria was 62.27%, 54.09%, 69.09%, respectively. The consistency rate of CDSS and ISTH in diagnosing DIC was 78.10%; the consistency rate of CDSS and JMHW was 88.32%. There was significant difference in PT, APTT, FIB, D-Dimer and FDP in DIC(+) and DIC(-) group by CDSS (all P<0.05), but patients in the DIC(+) group had lower level of D-Dimer than in the DIC(-) group [21.9(1.2-477.1) mg/L vs 26.3(0.6-488.7) mg/L, χ2=1.871, P=0.002] by ISTH, and there was not significant difference in APTT by JMHW [27.05(18.0-181.0) s vs 26.15(18.2-35.5) s, χ2=1.162, P=0.134]. In this study, both of the gender and age had no difference in the DIC (+) and DIC (-) group by CDSS. Univariate analysis showed that the level of WBC and the percent of abnormal promyelocytic cells in bone marrow when diagnosed were different in DIC (+) and DIC (-) group by CDSS (P<0.05). Multiple analysis showed the level of WBC (OR=3.525, 95% CI 1.875-6.629, P<0.001) was the only independent predictor in DIC diagnosis by CDSS. @*Conclusion@#The sensitivity of diagnosing DIC by CDSS was higher than the ISTH; and the specificity was superior to JMHW. Using CDSS can help to make the DIC diagnosis and treatment in time for APL patients who with the coagulation abnormalities.
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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 in January 29th, 2013, 35 patients were enrolled, and the trial was completed on May 16th, 2014. 17 patients were assigned to receive eltrombopag (starting dose 25 mg/d) and 18 were assigned to receive placebo.@*Results@#A total of 35 cases of adult cITP, 6 males and 29 females with a median age of 42(22-66) years were enrolled. One patient withdrew from eltrombopag treatment group for the adverse event (AE) and discontinued treatment. In first two weeks, 27.78% (5/18) of placebo-treated compared with 64.71%(11/17) of eltrombopag-treated patients achieved platelet counts ≥ 30×109/L(P=0.031); Treatment 6 weeks, the proportion of platelet counts reached ≥50×109/L and ≥ 30×109/L in eltrombopag-treated were higher than placebo-treated ones with statistically significant differences in both groups [64.71%(11/17) vs 11.11% (2/18), P=0.001; 76.47% (13/17) vs 38.89% (7/18), P=0.028]; The study also indicated a statistically significant difference in favour of eltrombopag compared with placebo in the odds of achieving the outcome of a platelet count ≥ 50×109/L at least once during 6-week treatment (94.11% vs 33.33%, P<0.001), and 70.59%(12/17) of patients with the platelet count continuously ≥ 50×109/L in 50% of treatment time in eltrombopag-treated group was more than placebo-treated one [11.11%(2/18), P<0.001]. Proportions of patients who required rescue treatment were 44.44% in placebo group and none in eltrombopag-treated one, respectively (P=0.002); The odds of bleeding symptoms with the WHO bleeding scale had no difference in both groups after 6 weeks (P=0.066). Adverse events that occurred more frequently due to eltrombopag than placebo included increased transaminase (3/17) and blood bilirubin (5/17), cerebral infarction(1/17).@*Conclusions@#The thrombopoietin receptor agonist eltrombopag was a suitable therapeutic option for Chinese adults with cITP.
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Objective@#To explore the immune tolerance induction (ITI) in a case of severe hemophilia B patient with inhibitor.@*Methods@#The F Ⅸ∶C was detected using a one-stage method and FIX inhibitor was assayed using Bethesda method. ITI was performed with prothrombin complex concentrates (PCC) in combination with rituximab.@*Results@#His past exposure days (ED) with PCC were 20 ED and his peak FⅨ inhibitor titer was 56 BU/ml. When his FIX inhibitor titer decreased to 10.4 BU/ml in Nov. 2015 and after receiving the informed consent from his parents, ITI was started. PCC with low dose rituximab successfully eradicated the high titer inhibitor within 17 months. There was no anaphylaxis, thrombotic event and infection.@*Conclusion@#This is the first case report for successful immune tolerance induction therapy in Chinese hemophilia B patient. ITI using PCC combined with rituximab is an effective choice to induce immune tolerance of hemophilia B with inhibitor.
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Objective@#To study the fatigue symptoms of adult patients with primary immune thrombocytopenia (ITP) and to analyze the possible factors that affect the severity of fatigue.@*Methods@#Eligible adult patients with ITP who admitted to Institute of Hematology & Blood Diseases Hospital were enrolled in this study and the questionnaires including a Chinese version of the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) , the Pittsburgh sleep quality index (PSQI) , the Hospital Anxiety and Depression scale (HADS) and demographic information were completed. The predictors of fatigue were determined with multiple linear regression analyses.@*Results@#A total of 207 patients with ITP were enrolled, including 70 males (33.8%) and 137 females (66.2%) , the median age was 42 (18-72) years old. The FACIT-F score in ITP patients was (37.50±9.05) . The FACIT-F severity of ITP patients was positively correlated with the platelet count (r=0.307, P<0.001) . The FACIT-F severity was negatively correlated with bleeding severity (r=-0.276, P<0.001) , sleep quality (r=-0.654, P<0.001) , depression (r=-0.598, P<0.001) and anxiety (r=-0.616, P<0.001) . A multiple linear regression analysis revealed that the severity of ITP-related fatigue was significantly correlated with platelet count (P<0.001) , bleeding severity (P=0.004) , sleep quality (P<0.001) and depression (P<0.001) .@*Conclusion@#Fatigue was determined by complicated factors in adult ITP patients. Interventions addressing depressive symptoms, sleep quality, bleeding symptoms and platelet count could be potential avenues for treatment of fatigue in patients with ITP.
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Objective@#To evaluate the role of the revised International Prognostic Score of Thrombosis (IPSET-thrombosis) in predicting the occurrence of thrombotic events in Chinese patients with essential thrombocythemia (ET) and to develop a thrombosis predicting model more applicable to Chinese ET patients.@*Methods@#Medical records of 746 adult patients with an initial diagnosis of ET were retrospectively analyzed.@*Results@#The median age at diagnosis was 52 (18-87) years, with 305 males and 441 females. According to the revised IPSET-thrombosis model, the number of very low-, low-, intermediate-, and high-risk patients were 271 (36.3%) , 223 (29.9%) , 63 (8.4%) and 189 (25.3%) , respectively. The four groups exhibited significantly different thrombosis-free survival (χ2=72.301, P<0.001) . Thirty-six patients were reclassified as intermediate-risk according to the revised IPSET-thrombosis instead of low-risk as per the original IPSET-thrombosis. Nineteen intermediate-risk patients as per the original IPSET-thrombosis were upgraded to high-risk according to the revised IPSET-thrombosis. Fifty-one high-risk patients as per the original IPSET-thrombosis were reclassified as low-risk in the revised IPSET-thrombosis. It suggests that the revised IPSET-thrombosis potentially avoids over- or under-treatment. In low-risk patients as per the revised IPSET-thrombosis, the rate of thrombosis in patients with cardiovascular risk factors (CVF) was higher than that in those without (16.3% vs 5.2%, χ2=5.264, P=0.022) , and comparable with intermediate-risk patients as per the revised IPSET-thrombosis (16.3% vs 14.3%, χ2=0.089, P=0.765) . As a result, a new revised IPSET-thrombosis model more applicable to Chinese ET patients was developed in which patients with CVF in the low-risk group as per the revised IPSET-thrombosis were reclassified as intermediate-risk group.@*Conclusion@#For predicting the occurrence of thrombotic events, the revised IPSET-thrombosis model was better than the original IPSET-thrombosis model. The revised IPSET-thrombosis was optimized and a new revised IPSET-thrombosis model more applicable to Chinese ET patients was developed, and the new evidence for risk stratification and treatment of ET in Chinese was provided.
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<p><b>OBJECTIVE</b>To deepen the understanding of clinical manifestations and treatment of patients with positive lupus anticoagulant (LAC).</p><p><b>METHODS</b>The clinical data of 2 patients were analyzed and related literature were reviewed.</p><p><b>RESULTS</b>Case 1, a 31-year-old female, diagnosed as lupus anticoagulant positive, secondary to undifferentiated connective tissue disease, was presented with menorrhagia and thrombocytopenia. Anti-nuclear antibody (ANA) was positive 1:1000 (homogeneous type) with anti-double stranded DNA positive, and dRVVT LA1/LA2 was 3.4. Coagulation function was alleviated after treatment with glucocorticoid and total glucosides of paeony. Case 2, a 59-year-old female was presented with gingival bleeding, hematuria with the level of F II:C 13%. dRVVT LA1/LA2 was 2.0. Anti-nuclear antibody (ANA) was positive 1:1000 (type of cytoplasmic granule), anti-double stranded DNA was positive. The patient was diagnosed as hypoprothrombinemia-lupus anticoagulant syndrome (LAHS) and acquired coagulation factor deficiency. The signs of hemorrhage were alleviated after treatment with methylprednisolone 40 mg/day and cyclophosphamide, while the level of F II:C was below normal.</p><p><b>CONCLUSION</b>Symptoms of patients with positive LAC are variable. The diagnosis relies on history of disease and laboratory test. Currently, there is no standardized treatment. Cases of LAHS should be thoroughly investigated for any known causes and related disorder.</p>
Subject(s)
Adult , Female , Humans , Middle Aged , Blood Coagulation , Cyclophosphamide , Therapeutic Uses , Glucocorticoids , Therapeutic Uses , Hematologic Tests , Hemorrhage , Hypoprothrombinemias , Diagnosis , Lupus Coagulation Inhibitor , Blood , Methylprednisolone , Therapeutic UsesABSTRACT
Objective To investigate the value of initial absolute lymphocyte counts (ALC) in predicting the prognosis of children with primary immune thrombocytopenia(ITP).Methods The initial clinical data of 214 children with incipient ITP were retrospectively analyzed and followed up for more than one year.Statistical analysis was made to find out the influence factors for the prognosis of patients with ITP in children to evaluate the predictive value of ALC on assessing the prognosis of ITP in children,and to analyze the association between ALC and outcomes and the clinical value of prognostic stratification.Results The remission rate of children with ITP was 71.5％ in 12 months.Multivariate unconditioned Logistic analysis showed that disease duration,the infection history,response to treatment and ALC were independent risk factors for chronic ITP(all P ＜ 0.05).Cutoff value of initial ALC was 3.005 × 109/L,sensitivity and specificity of value were 71.9％,83.6％.Three months and 12 months no remission rate of ITP in children between ALC ＞ 3.005 × 109/L and ≤ 3.005 × 109/L had statistical significant difference (24.2％ vs 64.9％,8.3％ vs 54.2％,x2 =42.13,P ＜0.001).There was no statistical difference in therapy efficacy between 2 groups(x2 =5.098,P ＞ 0.05).Remission rate between the ≤5 years old group and ＞ 5 years old group was statistical difference based on age stratification(x2 =22.371,22.177,all P ＜ 0.01).ITP in children were stratified into low risk group,intermediate risk group,high risk group based on the initial ALC and infection history,with remission rates in 3 month respectively 75.8％,54.1％,22.8％,respectively,and 91.6％,68.4％,31.6％ in 12 months,respectively;there were statistically significant differences (x2 =44.867,68.802,all P ＜ 0.001).Conclusions Initial ALC is an independent effecting factor in the prognosis of children with ITP.Initial ALC could be used for stratifying patients with the infection history before 6 weeks and supposed to be a predictive index for the prognosis of ITP in children.
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<p><b>OBJECTIVE</b>To explore incidence, risk factors and prognosis of the first 6 months infectious events in adults with newly diagnosed primary immune thrombocytopenia (ITP), and evaluate the value of initial absolute lymphocyte count (ALC) in predicting infection.</p><p><b>METHODS</b>The initial clinical records and infectious events during 6 months of 217 adult with newly diagnosed ITP were retrospectively analyzed. Statistical methods were used to analyze risk factors of the 6 months infections in adults ITP, the prediction of ALC in risk of infection, and the association of ALC and prognosis.</p><p><b>RESULTS</b>Infection rate of ITP patients accepting therapy within 6 months after the initial diagnosis was 13.8% (30/217), and infection rate of patients ≥ 60 years of age 25% (14/56). Multivariate unconditioned Logistic analysis showed that gender and ALC were independent risk factors for the 6 months infection of ITP patients (P<0.05, 95% CI 1.150-7.298, OR 2.722 and P<0.001, 95% CI 6.802-80.749, OR 23.436). Cutoff value of ALC was 1.225 × 10⁹/L, sensitivity and specificity of its value were 0.866 and 0.700 respectively. Infection rate of ALC>1.225 × 10⁹/L in adult ITP was lower than of ALC ≤ 1.225 × 10⁹/L (5.3% vs 45.7%, χ² = 49.151, P<0.001). Furthermore, persistent recovery and the 1-year mortality rate after diagnosis had no difference among patients of different ALC (28.0% vs 26.0%, χ² = 0.071, P>0.05, and 98.6% vs 97.8%, χ² = 0.095, P>0.05). There were no significant differences in persistent recovery in patients with and without infection (30.0% vs 27.3%, χ² = 0.096, P>0.05). The 1-year mortality rate after diagnosis was significantly lower in those patients who developed an infection (93.3% vs 99.3%, χ² = 4.607, P<0.05).</p><p><b>CONCLUSION</b>Initial ALC was an independent risk factor of 6 months infection in adult ITP. It could be a predictive index of infection within 6 months of the initial diagnosis in ITP patients. Infection as an important factor affected the survival of ITP patients.</p>
Subject(s)
Adult , Humans , Middle Aged , Lymphocyte Count , Multivariate Analysis , Prognosis , Purpura, Thrombocytopenic, Idiopathic , Retrospective Studies , Risk FactorsABSTRACT
<p><b>OBJECTIVE</b>To explore the clinical value of immature platelet fraction (IPF), absolute immature platelet fraction (A- IPF) and thrombelastograph (TEG) on assessment of bleeding risk of immune thrombocytopenia (ITP).</p><p><b>METHODS</b>two hundred and seventy- one patients with ITP were assessed based on ITP-BAT bleeding grading system. IPF, A-IPF were determined in 271 patients ,TEG in 125 patients. The correlations between bleeding grades and IPF, A-IPF, variables of TEG in subgroups were analyzed by statistical method. The predictive value of IPF, A-IPF, and variables of TEG on bleeding risk of ITP patients was evaluated.</p><p><b>RESULTS</b>There were no significant differences in bleeding degree in all patients with different gender and disease stage (P>0.05). Mild bleeding rate in children was higher than that in adult (P<0.05). PLT inversely correlated with bleeding grade for the entire cohort (P<0.001). In all subjects, PLT< 30 × 10⁹/L and pediatric cohorts with PLT< 30 × 10⁹/L, PLT were negatively correlated with IPF (P<0.05), positive correlated with A-IPF (P<0.001) and the maximum amplitude (MA (P<0.05). Bleeding grades were significantly correlated with IPF, A-IPF, MA in all subjects and patients with PLT< 30 × 10⁹/L (P<0.001). IPF, A-IPF and MA did not correlate with bleeding grades in children with PLT< 30 × 10⁹/L (P>0.05). ROC curve analysis revealed IPF, A-IPF and MA had better predictive value (AUC 0.745, 0.744, 0.813, P<0.001). Multivariate analysis showed that IPF and MA were independence factors for predicting bleeding risk in ITP patients and comprehensive predictive value was higher (AUC 0.846, P<0.001) than single variable.</p><p><b>CONCLUSION</b>IPF, A-IPF and MA could accurately evaluate bleeding risk in ITP patients. It may be considered as reference index of the treatment and observation index of curative effect.</p>
Subject(s)
Adult , Child , Humans , Blood Platelets , Hemorrhage , Multivariate Analysis , Platelet Count , Purpura, Thrombocytopenic, Idiopathic , ROC CurveABSTRACT
<p><b>OBJECTIVE</b>To evaluate the efficacy and safety of recombinant human thrombopoietin (rhTPO) in treatment of pediatric primary immune thrombocytopenia (ITP).</p><p><b>METHODS</b>The clinical characteristics of 41 pediatric ITP patients who received rhTPO therapy from December 2006 to September 2014 were retrospectively analyzed (as rhTPO group). During the same time another 26 pediatric ITP patients who received vindesine combined with human immunoglobulin therapy were selected as control group. The treatment outcomes were evaluated.</p><p><b>RESULTS</b>A total of 67 cases of pediatric ITP, 31 males and 36 females with a median age 10.0(1.6-17.0) years were enrolled, including 19 cases of newly disgnosed ITP, 18 cases of persistent ITP and 30 cases of chronic ITP. Of them, 43 cases of whom were severe ITP (PLT<10×10⁹/L). The total response rate had no statistically significant difference between the rhTPO group and the control group (68.29% vs 65.38%, P=0.806), neither in newly ITP, persistent and chronic ITP (P=0.320, P=0.763). In severe ITP patients, 17 of 30 cases (56.67%) achieved response with rhTPO therapy, while the control group was 61.54% (8/13) (P=0.766). The median maximum peak of platelet counts and the time of the platelet counts >30×10⁹/L and > 50×10⁹/L had no statistically significant differences in rhTPO group compared with the control group [52(7-608)×10⁹/L vs 40(3-152)×10⁹/L, P=0.05; 7(3-13) d vs 4(2-24) d, P=0.202; 7.5(4-15) d vs 5.5(4-23) d, P=0.557]. The mean platelet counts were 43(3-605)×10⁹/L in the rhTPO group, which were higher than the control group [32(-14-149)×10⁹/L, P=0.042]. No severe adverse effects were observed in both groups.</p><p><b>CONCLUSION</b>For pediatric ITP, rhTPO has a similar outcomes with vindesine combined with human immunoglobulin, and it is an effective and safe treatment option.</p>
Subject(s)
Adolescent , Child , Child, Preschool , Female , Humans , Infant , Male , Platelet Count , Purpura, Thrombocytopenic, Idiopathic , Recombinant Proteins , Retrospective Studies , Thrombopoietin , Treatment Outcome , VindesineABSTRACT
<p><b>OBJECTIVE</b>To analyze clinical and molecular characteristics of low-risk essential thrombocythemia (ET) in a large cohort of Chinese patients and to explore risk factors for major thrombosis and treatment strategies.</p><p><b>METHODS</b>Medical records of patients with an initial diagnosis of ET from March 1982 to May 2012 in our hospital were retrospectively analyzed.</p><p><b>RESULTS</b>A total of 604 low-risk ET patients were enrolled with a median follow-up of 49 months (range:0-338). 43(7.1%) patients experienced major thrombotic events. Cox proportional hazards regression revealed JAK2 V617F mutation (HR=2.279; P=0.035) and cardiovascular risk factors (CVF) (HR=2.541; P=0.006) to be risk factors for total thrombotic events, while only CVF (HR=2.633; P=0.008) was risk factor for arterial thrombosis. None of the evaluated factors was related to venous thrombosis. Patients with both JAK2 V617F mutation and CVF had a worse thrombosis- free survival than those with only one risk factor (P<0.05). In patients with JAK2 V617F or CVF alone, antiplatelet treatment (P=0.016) significantly decreased the risk of thrombosis, while those with both JAK2 V617F and CVF could benefit from cytoreductive agents (P=0.018).</p><p><b>CONCLUSION</b>Chinese low-risk ET patients have a lower risk of thrombosis than Caucasian low-risk ET patients. JAK2 V617F mutation and CVF are the most significant risk factors for thrombosis. Existence of both risk factors further increases the thrombotic risk. Treatment strategies on low-risk ET patients should be made based on presence or absence of risk factors.</p>
Subject(s)
Humans , Asian People , Janus Kinase 2 , Mutation , Retrospective Studies , Risk Factors , Thrombocytopenia , Thrombosis , GeneticsABSTRACT
<p><b>OBJECTIVE</b>To explore the clinical significance, reliability and responsiveness of ITPBAT bleeding grading system for patients with immune thrombocytopenia (ITP).</p><p><b>METHODS</b>One hundred and eighty-three patients with ITP were assessed by using of ITP-BAT bleeding grading system. Test-retest reliability, responsiveness of ITP-BAT bleeding grading system and association between bleeding grades and platelet counts, age, gender, disease stage were analyzed.</p><p><b>RESULTS</b>Bleeding degree of ITP patients and the platelet count were negatively correlated (r=- 0.744, P<0.01) and bleeding degree increased significantly with platelet counts below 20×10⁹/L (χ²=82.40，P<0.01). Mild bleeding rate in children was 68.5%, higher than that in adult(χ²=8.839，P<0.01), and severe bleeding rate in the elderly was 14.3%, higher than that in non-elderly(χ²=7.056，P<0.01). There were no significant differences in bleeding degree in patients with different gender and disease stage (χ²=4.922, P>0.05 and χ²=3.411, P>0.05). Bleeding grades before and after treatment had more significant difference(Z=-6.61, P<0.01). Scoring consistency of two doctors was 66.1% (κ=0.561), and scoring consistency of the same doctor was 94.7% (κ=0.874).</p><p><b>CONCLUSION</b>ITP-BAT bleeding grading system in China has good validity and responsiveness, closely related to clinical indicators. It is sensitive to the variation of the hemorrhage in patients. ITP-BAT could be used as a reference index of the treatment, and also be used as an observation index of curative effect.</p>