High-dose dexamethasone (HDD) is widely used as first-line therapy for immune thrombocytopenia (ITP) and is administered at a fixed dose regardless of body weight. The impact of body mass index (BMI) on treatment response to HDD remains unclear. This retrospective, two-center study included 60 adult patients with newly diagnosed ITP who received HDD as first-line therapy. Demographic characteristics, BMI, baseline laboratory values, and treatment responses at1, 6, and 12months were analyzed. BMI was evaluated using cut-off values of 25, 27, and 30. The median number of HDD cycles administered was 1 (range: 1-4), and the median BMI at diagnosis was 27.0kg/m2 (range: 18.0-44.0). No significant differences were observed between BMI categories with regard to treatment responses at months 1 and 6 (p > 0.05 for both). However, at month 12, a complete response (CR) was more likely in patients with BMI < 30, and a partial response (PR) in those with BMI ≥ 30 (p = 0.023). Across all time points, no other demographic or clinical variable emerged as an independent predictor of treatment response (p > 0.05). The results of this study indicate that in newly diagnosed ITP patients receiving HDD as first-line treatment, BMI does not influence early or durable treatment responses, although it may have a modest adverse effect on late response. Larger prospective studies are needed to clarify underlying mechanisms and assess whether obesity-related factors should inform individualized treatment.

Prevalence and associated factors of sexual dysfunction in female patients with immune thrombocytopenia: a longitudinal follow-up study.

Postviral antiplatelet factor 4 (PF4) immunothrombosis is an emerging pediatric entity characterized by thrombocytopenia, thrombosis, and markedly elevated D-dimer levels and anti-PF4 antibodies. It shares immunopathologic features with vaccine-induced immune thrombotic thrombocytopenia but arises after natural infection, most often adenoviral infection. We reviewed pediatric cases and mechanistic studies identified through PubMed and reference screening (latest search: November 20, 2025), with a focus on pathophysiology, differential diagnosis, laboratory evaluation, and treatment. We identified 10 pediatric patients, with a reported mortality rate of 20%. Children typically presented with acute severe headache, focal neurological deficits, and thrombocytopenia 5-14 days after recent viral illness. All reported cases had markedly increased D-dimers. Rapid immunoassays for heparin-induced thrombocytopenia were often negative; PF4-specific enzyme-linked immunosorbent assay and PF4-enhanced functional assays were positive. Treatments in published cases included anticoagulation (9/10 cases), intravenous immunoglobulin (5/10 cases), and plasma exchange therapy (3/10 cases). On the basis of these findings and mechanistic parallels, we propose a diagnostic and therapeutic approach, acknowledging the limited evidence base. In conclusion, postviral anti-PF4 immunothrombosis in children, although rare, is potentially fatal and likely underrecognized. Further research is needed to establish standardized diagnostic criteria and evidence-based treatment protocols.

IRE1α regulates macrophage phagocytosis in immune thrombocytopenia through NR1D1 mRNA decay and lysosomal biogenesis.

The PI3K-AKT pathway mediates the imbalance of bone marrow macrophage polarization in patients with immune thrombocytopenia.

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Short- and long-term outcome of neonates with thrombocytopenia from maternal immune thrombocytopenia.

Background: The objective of this study was to evaluate the efficacy of mycophenolate mofetil (MMF) as a second- or third-line treatment for chronic immune thrombocytopenia (ITP).Methods: A cohort of 13 patients with chronic ITP, who had previously been treated with eltrombopag, corticosteroids, or both, without achieving an effective response, was administered mycophenolate mofetil at a daily dosage of 1000 mg. Platelet counts were monitored at the fourth and twelfth weeks of treatment.Results: In our study, 13 patients were included, consisting of 4 females (30.8%) and 9 males (69.2%). At the 12-week mark following the initiation of MMF therapy, three out of 13 patients achieved a complete response, three out of 13 patients achieved a partial response, and seven patients showed no response. (Fig.1). At the time of transitioning to Mycophenolate Mofetil (MMF), all patients had a platelet count below 30 × 10^9/L. Approximately 40% of patients experienced an increase in platelet counts to levels above 30 × 10^9/L.Conclusions: Mycophenolate mofetil may be considered a potential treatment option for patients with chronic ITP who are refractory to first- and second-line therapies.

This review summarizes current understanding of platelet-endothelial contributions to thrombosis, emphasizing molecular crosstalk [von Willebrand factor (VWF)/ADAMTS13 balance, P-selectin, platelet glycoprotein VI (GPVI), integrins, extracellular vesicles, neutrophil extracellular traps (NETs)], high-risk clinical settings, and translational advances. Highlighting GPVI-directed therapeutics, the VWF/ADAMTS13 axis in COVID-19, and opportunities and challenges for targeting the platelet-endothelial interface. Clinical and translational studies support the safety and potential efficacy of targeting platelet-endothelial interfaces. GPVI inhibitors (Glenzocimab, Revacept) have advanced through phase I/II studies with reassuring bleeding profiles and suggest benefit in ischemic stroke and lesion-directed settings. Direct interruption of platelet-VWF interactions (Caplacizumab) is established in immune thrombotic thrombocytopenic purpura (TTP), while studies show a persistent VWF/ADAMTS13 imbalance in severe COVID-19 and inflammatory states linked to microthrombosis and worse outcomes. Antiadhesion strategies (P-selectin blockade) and modulators of immunothrombosis (NET inhibitors, targeting extracellular vesicle) are also in evaluation. Targeting platelet-endothelial crosstalk has potential to reduce pathologic thrombosis while preserving hemostasis. Clinical proof of principle exists for focused approaches (anti-VWF in TTP; P-selectin blockade in vaso-occlusion; emerging GPVI inhibitors). Priorities are: defining disease contexts and timing where interface targeting is effective; validating biomarkers (VWF/ADAMTS13 ratio, soluble P-selectin, platelet activation signatures) for patient selection; and conducting adequately powered trials with rigorous bleeding endpoints.

Despite several studies reporting the effectiveness of dapsone in immune thrombocytopenic purpura (ITP), information on its mechanism of action, dose-response relationship and response predictors remains limited. To address this knowledge gap, we analysed 433 healthcare visit data of 58 ITP patients (29 males and 29 females; 39 adults and 19 paediatric) treated with dapsone (mean: 1.19 mg/kg/day) after 0-4 prior treatments. Among them, 14 had newly diagnosed, 23 had persistent and 21 had chronic ITP. The median ages for paediatric and adult patients were 6.4 and 40.9 years respectively. Median response time, response duration and follow-up period were 40, 151 and 142 days respectively. The overall response rate was 58.6%, of which 94.1% of patients responded within 6 months and 44.8% (n = 26) maintained a response at the last follow-up. Mediation analysis showed that the association between dapsone and responses was mediated via haemoglobin reduction. Response rates were 5.38 times higher in patients who experienced a reduction in haemoglobin than in those who did not experience a reduction in haemoglobin (p = 0.008). Age, sex, previous treatment numbers and response, ITP types, baseline platelets and concurrent treatments were not associated with a response. Two patients needed dapsone discontinuation due to adverse events.

Lack of association between vaccine-induced immune thrombocytopenia and thrombosis and HLA loci in a large cohort.

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Background: Immune thrombocytopenia (ITP) is a rare autoimmune disorder resulting in reduced platelet count. The annual incidence is 3.3 per 100,000 adults and 8.8 per 100,000 person-years in children. Immune thrombocytopenia impacts the quality of life, productivity, and psychosocial well-being of patients and caregivers. This quality improvement study aims to characterize needs of patients, caregivers, and health-care providers (HCPs) managing chronic ITP to improve management in the future. Methods: We conducted a two-session Learning Exchange program: one with HCPs and adult patients, and one with HCPs and caregivers of pediatric patients. Discussions encompassed clinical and personal experiences with chronic ITP, the health-care system, and support resources. Session transcripts identified key themes, which participants subsequently prioritized in a post-meeting survey. Results: A total of 7 patients, 6 caregivers, and 14 HCPs participated in the Learning Exchange sessions, discussing challenges in chronic ITP management and areas for improvement. Post-meeting surveys identified shared priorities, ranked as follows: (1) empowering patients to self-advocate; (2) strengthening shared decision-making; (3) improving resources and support; (4) following the evolving treatment landscape; and (5) mental health support. Conclusions: The sessions identified key needs involving improved education for patients, caregivers, and HCPs; improved communication between patients, caregivers, and HCPs; the greater need for self-advocacy and shared decision-making; and the desire for better support resources and networks. This knowledge will enable the development of improved educational resources, support programs, and future research to benefit the chronic ITP community and improve the quality of management of chronic ITP for patients, caregivers, and HCPs.

Severe thrombocytopenia is a frequent but diagnostically challenging complication in people living with HIV (PLWH). We report the case of a 43-year-old woman with HIV who developed life-threatening hematochezia and profound thrombocytopenia after the interruption of antiretroviral therapy. Although bone marrow aspiration showed hypocellular fatty marrow, preserved leukocyte counts and the overall clinical context supported immune thrombocytopenia secondary to uncontrolled HIV replication. Treatment with intravenous immunoglobulin and high-dose corticosteroids led to independent transfusion and platelet recovery. Helicobacter pylori infection was identified and eradicated. This case highlights the fact that hypocellular marrow findings mimicking bone marrow failure do not exclude ITP in PLWH.

Novel use of telitacicept in primary immune thrombocytopenia: a case report.

CDK8/CDK19 inhibition restores T-cell homeostasis in primary immune thrombocytopenia.

Platelet surface-associated immunoglobulin (PSAIG) occurs in thrombocytopenic dogs with vector-borne diseases and immune thrombocytopenia (ITP) and may be associated with thrombocytopenia severity and inflammatory markers, including neutrophil-to-lymphocyte and platelet-to-lymphocyte ratios (NLR, PLR). Assess associations between PSAIG positivity, hematologic parameters, thrombocytopenia severity, and vector-borne status in thrombocytopenic dogs. Sixty-nine client-owned thrombocytopenic dogs (<200×103/μL) were enrolled between June 2022 and June 2023. Dogs were prospectively enrolled. Platelet surface-associated immunoglobulin was measured using flow cytometry. Vector-borne pathogens were assessed by serology (Ehrlichia spp., Anaplasma spp., Borrelia burgdorferi, Dirofilaria immitis) and PCR for Ehrlichia canis. Hematologic parameters were compared between PSAIG groups (Mann-Whitney U), and associations tested by univariable logistic regression. Dogs positive for PSAIG (n=16) had lower median automated platelet counts (16.5×103/μL; interquartile range [IQR]: 8.25-40.75) than PSAIG-negative dogs (n=53; 64×103/μL; IQR: 25.0-92.5; P=.001), with similarly lower manual platelet counts (48×103/μL; IQR: 20-86 vs 96×103/μL; IQR: 55-138; P=.01) and automated PLR (7.14; IQR: 3.30-15.28 vs 21.82; IQR: 9.42-38.99; P=.01). In logistic regression, PSAIG positivity was associated with lower platelet counts and automated PLR, E. canis PCR positivity, and Anaplasma seropositivity, with the strongest association for concurrent E. canis PCR and Anaplasma seropositivity (odds ratio [OR]; 15.3; 95% confidence interval [CI]: 2.69-86.99; P=.002). Lower platelet counts and automated PLR were associated with PSAIG positivity in thrombocytopenic dogs. Associations between PSAIG, E. canis infection, and co-exposure to Anaplasma spp. support immune-mediated platelet destruction in infected dogs.

Adult patients with immune thrombocytopenia (ITP) have an increased risk of venous thrombosis as compared to the general population. The management of ITP in the context of anticoagulation is challenging. We conducted an observational study in the prospective, multicenter, national CARMEN-France registry. Adult patients with newly diagnosed ITP between June 2013 and May 2022 were selected. We assessed the cumulative incidence of venous thrombosis during follow-up with death as a competing event, described these events, and assessed patient outcomes depending on management strategies, with a focus on thromboses that occurred during treatment with thrombopoietin receptor agonists (TPO-RA). Among the 1303 patients selected for this study, 53 experienced venous thrombosis. The cumulative incidence of venous thrombosis was 2.6% (95% CI: 1.8-3.7) at 1 year and 8.6% (95% CI: 5.8-12.0) at 5 years. In patients exposed to TPO-RA, the cumulative incidence was 9.3% (95% CI: 6.2-13.2) and 13.4% (95% CI: 8.6-19.2) at 1 and 5 years of exposure, respectively. Patients who experienced thrombosis were older, had more frequently a history of venous thrombosis and secondary ITP, a more severe ITP, and were more frequently treated with TPO-RAs. Twenty (37.7%) of the 53 events were atypical, including five cerebral venous thromboses. Four patients died, and seven experienced major bleeding. The analysis of different managements of ITP after the thrombotic event suggested that the safest strategy was to promptly control ITP to enable early anticoagulation, including using TPO-RAs. Long-term anticoagulation therapy should be considered in patients treated with TPO-RAs and persistent risk factors for thrombosis.

BackgroundEltrombopag (ELT) is an established thrombopoietin receptor agonist (TPO-RA) for chronic immune thrombocytopenia (ITP), yet accumulating translational evidence indicates clinically relevant iron-chelating activity. Adult primary ITP–focused data characterizing longitudinal iron trajectories during ELT remain limited. We assessed whether ELT exposure is independently associated with iron deficiency (ID) in routine practice.MethodsIn this multicenter retrospective study, adults with ITP were evaluated with longitudinal monitoring of platelet count, ferritin, transferrin saturation (Tsat), hemoglobin (Hb), and mean platelet volume (MPV). Within-patient change was defined as the difference between baseline and follow-up (Δ). Outcomes were compared by ELT exposure and dose strata. Multivariable linear regression was used to identify independent determinants of Δ-ferritin, adjusting for age, gender, relapse status, and iron replacement therapy (IRT).ResultsThe cohort included 283 adults with ITP; 110 received ELT (median 25 months). ELT was associated with greater declines in ferritin and Tsat (p<0.001), with a dose-graded effect across 25–75 mg and earlier iron depletion at higher dose intensity. In relapsed patients not receiving ELT, the mean Δ-ferritin was positive and did not differ by bleeding status. In multivariable linear regression, ELT was the dominant independent predictor of lower Δ-ferritin (B≈−79.8 μg/L, p<0.001), whereas age, gender, and relapse were not significant; IRT attenuated ferritin decline but did not negate ELT effects.ConclusionELT exposure was independently associated with ID, supporting a clinically meaningful ELT-related iron chelation phenotype in routine practice. Monitoring and timely correction of ID during ELT therapy may mitigate a modifiable contributor to fatigue during follow-up.