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Eltrombopag is an oral, non-peptide, thrombopoietin-receptor agonist that stimulates thrombopoiesis, leading to increased platelet production. This study assessed the efficacy, safety, and tolerability of once daily eltrombopag 50 mg, and explored the efficacy of a dose increase to 75 mg. In this phase III, randomised, double-blind, placebo-controlled study, adults from 63 sites in 23 countries with chronic idiopathic thrombocytopenic purpura (ITP), platelet counts less than 30 000 per μL of blood, and one or more previous ITP treatment received standard care plus once-daily eltrombopag 50 mg (n=76) or placebo (n=38) for up to 6 weeks. Patients were randomly assigned in a 2:1 ratio of eltrombopag:placebo by a validated randomisation system. After 3 weeks, patients with platelet counts less than 50 000 per μL could increase study drug to 75 mg. The primary endpoint was the proportion of patients achieving platelet counts 50 000 per μL or more at day 43. All participants who received at least one dose of their allocated treatment were included in the analysis. This study is registered with ClinicalTrials.gov, number NCT00102739. 73 patients in the eltrombopag group and 37 in the placebo group were included in the efficacy population and were evaluable for day-43 analyses. 43 (59%) eltrombopag patients and six (16%) placebo patients responded (ie, achieved platelet counts ≥50 000 per μL; odds ratio [OR] 9·61 [95% CI 3·31–27·86]; p<0·0001). Response to eltrombopag compared with placebo was not affected by predefined study stratification variables (baseline platelet counts, concomitant ITP drugs, and splenectomy status) or by the number of previous ITP treatments. Of the 34 patients in the efficacy analysis who increased their dose of eltrombopag, ten (29%) responded. Platelet counts generally returned to baseline values within 2 weeks after the end of treatment. Patients receiving eltrombopag had less bleeding at any time during the study than did those receiving placebo (OR 0·49 [95% CI 0·26–0·89]; p=0·021). The frequency of grade 3–4 adverse events during treatment (eltrombopag, two [3%]; placebo, one [3%]) and adverse events leading to study discontinuation (eltrombopag, three [4%]; placebo, two [5%]), were similar in both groups. Eltrombopag is an effective treatment for managment of thrombocytopenia in chronic ITP. GlaxoSmithKline.

This study aimed to develop and validate a risk prediction model for moderate to severe bleeding in children with immune thrombocytopenia (ITP). Data from 286 ITP patients were prospectively collected and randomly split into training (80%) and test (20%) sets. Least Absolute Shrinkage and Selection Operator (LASSO) regression was used for feature selection. Among seven machine learning algorithms, the eXtreme Gradient Boosting (XGBoost) model demonstrated the best performance (AUC = 0.886, 95% CI: 0.790–0.982) and was selected as the optimal model. Shapley Additive Explanations (SHAP) were used for model interpretation, identifying child age, age at diagnosis, and initial platelet count as key predictors of moderate to severe bleeding risk. Conclusion : The XGBoost-based prediction model shows strong predictive performance and could assist healthcare providers in identifying high-risk ITP patients, supporting appropriate clinical decision-making. Trial registration number : ChiCTR2100054216, December 11, 2021 What is Known: • Current clinical practice relies solely on platelet counts to guide hospitalization and treatment in ITP children, often overlooking bleeding manifestations, leading to delayed or inappropriate treatment. Existing severe bleeding risk prediction models are primarily designed for adults and lack applicability to children. What is New:  • This study prospectively collected data, enhancing accuracy. A novel machine learning-based prediction model was developed to assess moderate to severe bleeding risk in pediatric ITP patients.

Immune thrombocytopenia (ITP) is a common complication of connective tissue diseases (CTD). However, refractory and recurrent cases are frequent, who often need intensive immunotherapy. In the real world to compare the efficacy and safety of two common options, rituximab (RTX) and cyclosporine (CsA), in patients with refractory CTD-ITP, we conducted this retrospective study. Inpatients diagnosed with CTD-ITP who experienced treatment failure with initial prednisone or other immunosuppressants and who subsequently received either RTX or CsA between 2013 and 2018 were identified. All the patients were followed up for at least 6 months. Remission was defined as sustained platelet count ≥ 50 × 10^9/L, where ≥ 100 × 10^9/L was considered complete remission and 50–100 × 10^9/L was considered partial remission. Propensity score weighting analysis was performed to balance the confounders as indication. A total of 83 patients with CTD-ITP were identified, of whom 43 had systemic lupus erythematosus, 24 had undifferentiated CTD, and 16 had primary Sjogren syndrome. The RTX group (n = 53) had a much higher remission rate than the CsA group (n = 30) after 3 months and throughout the following 3 months (3 m, 86.8% vs 63.6%, p = 0.025; 6 m, 81.8% vs 53.5%, p = 0.011). Binary logistic regression analysis confirmed that treatment with RTX predicted better outcome (OR 4.09, 1.42 ~ 11.79), while age > 50 (OR 0.31, 0.11 ~ 0.93) was a risk factor. Furthermore, we reinforced the conclusions by propensity score weighting analysis (RTX OR 4.89, 1.64 ~ 14.58; age > 50 OR 0.31, 0.12 ~ 0.83). In our real-world retrospective study, for patients with refractory CTD-ITP, RTX was superior to CsA in terms of the durable remission rate.Key Points:• Refractory cases are common in patients with immune thrombocytopenia secondary to connective tissue diseases (CTD-ITP), requiring intensive immunotherapy.• Randomized controlled trials comparing rituximab and a traditional immunosuppressive agents (IS), such as cyclosporin, are lacking in these patients.• Our real-word retrospective study indicated that rituximab was superior to cyclosporin in patients with refractory CTD-ITP.

Eradication of Helicobacter pylori was reported to increase the platelet counts in some H. pylori-positive patients with chronic idiopathic thrombocytopenic purpura (cITP). However, the efficacy of the eradication was quite different according to the previous reports. To determine whether H. pylori infection can contribute to cITP, we performed a randomized controlled trial for the first time. In addition, to investigate the possible pathogenic mechanisms and to predict the platelet response after eradication of H. pylori in each cITP patient, several H. pylori virulence factors, the urease activities of the infected H. pylori strains, and the titers of anti-CagA IgG antibodies were analyzed. Patients with cITP underwent gastroscopy and gastric H. pylori infection was confirmed by culture. H. pylori-positive cITP patients were randomly assigned to either the eradication or the non-eradication group. The eradication group received a standard antibiotic therapy for H. pylori. Response to treatment was defined as complete (CR) if the platelet count was above 150x10(3)/microl and partial (PR) if the platelet count increased by more than 50x10(3)/microl above the pretreatment count. The virulence factors were investigated by PCR and PCR-based direct sequencing. Anti-CagA IgG antibody titer of each patient's serum was measured by ELISA. Of the 36 ITP patients, 25 (69.4%) were positive for H. pylori and eradication was achieved in 84.6% of these patients. The platelet response was significantly different between the eradication group (46.2%) and the non-eradication group (0%). No significant differences were found in clinical factors between the responders and the nonresponders. H. pylori virulence factors and the urease activity were not associated with the response. The titers of anti-CagA antibodies in the responders were significantly higher than those in the nonresponders (p=0.04). H. pylori eradication treatment is a favorable therapeutic option for H. pylori-positive patients with cITP. Moreover, an ELISA titer of serum anti-CagA antibody may be a good predictor of platelet recovery, and immunological reaction between platelet and anti-CagA antibodies may have some relation to the pathogenesis of H. pylori-positive patients with cITP.

In a multicenter, open-label, randomized trial in patients with immune thrombocytopenia, 91.5% of patients who received mycophenolate mofetil plus glucocorticoids had platelet levels above 100,000 per microliter, as compared with 63.9% of patients who received glucocorticoids only. Quality-of-life measures of physical function and fatigue were worse in the mycophenolate mofetil group.

Background Immune thrombocytopenia (ITP) is an immune-mediated acquired disease that is characterized by a decrease in the platelet count and an increased risk of bleeding. There is little information in the literature about the results of major joint replacement surgery in patients with ITP. The aim of this study was to report on the results of total hip arthroplasty (THA) in patients with primary ITP. Methods We retrospectively identified 15 THAs performed in 11 patients with primary ITP. The study group was matched (1:2) to a non-ITP control group of 30 THAs in 22 patients. According to the perioperative hematologic evaluation, blood management interventions were performed. All procedures were performed by a single surgeon and all patients received cementless components with ceramic-on-ceramic bearing. Mean duration of follow-up was 7.1 years (range, 2–13). Results No significant differences were found between the two groups with regard to mean operative time, intraoperative blood loss, amount of closed suction drainage, length of hospital stay, and readmission rate. However, the proportion of patients requiring transfusion of packed red blood cells and/or platelet concentrate was higher in the ITP group when compared to the non-ITP group. Mean Harris hip score improved from 49.5 points preoperatively to 93.4 points at the final follow-up and no hips were revised for loosening or osteolysis in the ITP group. No significant differences were found between the two groups with respect to mean postoperative Harris hip scores and complication rates. Conclusions Our study showed encouraging clinical and radiographic results of THA in patients with ITP without increased risk of adverse events compared to those in patients without ITP. On the basis of these findings, we suggest that modern cementless THA might be a viable treatment for achieving functional improvement in patients with ITP and end-stage hip disease.

The modern Gastroenterology have witnessed an essential stride since Helicobacter pylori was first found in the stomach and then its pathogenic effect was discovered. According to the researches conducted during the nearly 40 years, it has been found that this bacterium is associated with a natural history of many upper gastrointestinal diseases. Epidemiological data show an increased incidence of autoimmune disorders with or after infection with specific microorganisms. The researches have revealed that H. pylori is a potential trigger of gastric autoimmunity, and it may be associated with other autoimmune diseases, both innate and acquired. This paper reviews the current support or opposition about H. pylori as the role of potential triggers of autoimmune diseases, including inflammatory bowel disease, autoimmune thyroiditis, type 1 diabetes mellitus, autoimmune liver diseases, rheumatoid arthritis, idiopathic thrombocytopenic purpura, systemic lupus erythematosus, as well as Sjogren’s syndrome, chronic urticaria and psoriasis, and tried to explain the possible mechanisms.

Thrombocytopenia in Pregnant WomenPlatelet counts often fall during pregnancy; decreases below 50×103 per cubic millimeter may be due to an autoimmune cause. Detection and management of immune thrombocytopenia may be lifesaving.

Romiplostim increases platelet counts and reduces the risk of bleeding in patients with immune thrombocytopenia (ITP). This post hoc analysis compared the effect of romiplostim versus medical standard of care (SOC) on clinically relevant bleeding-related episodes (BREs) in a 52-week open-label study of patients with ITP. BREs were defined as actual bleeding events and/or use of rescue medication. Nonsplenectomized adult patients with ITP were randomized to receive weekly subcutaneous injections of romiplostim ( n  = 157) or SOC ( n  = 77). The rate of all BREs (per 100 patient-weeks) was lower in patients treated with romiplostim (3.1) than in those treated with SOC (9.4); the relative rate (romiplostim/SOC) was 0.33 (95 % CI 0.27–0.40). The rate of BREs associated with immunoglobulin (Ig) rescue medication was also lower for romiplostim (0.2) than SOC (4.8); the relative rate (romiplostim/SOC) was 0.05 (95 % CI 0.03–0.08). BRE rates were lower in patients with platelet counts ≥50 × 10 9 /L, and patients treated with romiplostim spent more time with platelet counts ≥50 × 10 9 /L than did patients treated with SOC. Bleeding-related hospitalizations were rare in both groups. Thus, romiplostim treatment provided greater reductions in all BREs, as well as BREs involving Ig rescue medications, than did SOC.

VITT is caused by a somatic hypermutation of an anti–adenovirus pVII antibody that generates more avid binding of platelet factor 4 than of adenovirus pVII, its original target, which results in platelet activation.