Open research questions in Platelet Disorders and Treatments
91 unresolved questions extracted from the limitations and future-work sections of 392 Platelet Disorders and Treatments papers in our library. Each links back to the study that raised it.
What the literature leaves open
lack of knowledge on the exact relationship between the degree of thrombocytopenia and bleeding symptoms, - lack of knowledge of predictive factors of response to treatments, - significant variability regarding the therapeutic approach, - no clinical trials to demonstrate the superiority of any treatment option
Current Understanding of Immune Thrombocytopenia: A Review of Pathogenesis and Treatment Options · 2024 · DOIidentification of new molecules that target different pathways involved in the pathogenesis of ITP, - evaluation of the efficacy and safety of new therapeutic targets in ITP, - investigation of the role of FCγR signaling in the pathogenesis of ITP, - study of the inhibition of the classical complement pathway in ITP
Current Understanding of Immune Thrombocytopenia: A Review of Pathogenesis and Treatment Options · 2024 · DOIObjectives: Their impact on hepatic thrombopoietin (TPO) regulation after AMI remains poorly understood to date.
The heart-liver axis controls platelet turnover by hepatic STAT3 phosphorylation and TPO regulation after acute myocardial infarction · 2026 · DOIAbstract Current antithrombotic therapies are effective in reducing thrombotic events but are limited by their associated risk of bleeding.
Selective degradation of platelet BTK by PROTAC NX-5948 provides antithrombotic benefits without affecting hemostasis · 2026 · DOIAbstract Introduction Drug-induced immune thrombocytopenia (DITP) is a rare, antibody-mediated adverse effect that typically occurs within days to weeks of drug exposure and is rarely reported in infants receiving prolonged diuretic therapy.
Severe, delayed-onset thrombocytopenia after prolonged chlorothiazide use in a former preterm infant with bronchopulmonary dysplasia: a case report · 2026 · DOIHowever, it remains unknown whether CXCR5+γδ T cells are involved in the pathogenesis of ITP.
The expression pattern and role of circulating CXCR5+γδ T cells in children with newly diagnosed immune thrombocytopenia · 2025 · DOIUnlike previous cases, this patient exhibited IgA deposition in the mesangial region, suggesting need for further investigation into prognostic and therapeutic significance of this finding.
Novel variant in MYH9 in a child with proteinuria and thrombocytopenia: a case report and literature review · 2025 · DOIVancomycin-induced thrombocytopenia (VIT) is infrequently and often under-recognized.
Incidence and risk factors of Vancomycin-induced thrombocytopenia: a six-year real-world study · 2025 · DOIDue to limited knowledge and heterogenous manifestations, clinical diagnosis of the disease is challenging.
A novel missense mutation in the MECOM gene in a Chinese boy with radioulnar synostosis with amegakaryocytic thrombocytopenia · 2024 · DOIThere is a lack of understanding of the biology of von Willebrand factor and its functions. There is a need for effective treatments for von Willebrand disease.
The paper suggests that future research could focus on improving the generation of intersection hypotheses. The paper mentions that there are multiple ways to implement Algorithm 1 in Bretz et al. (2011).
The paper identifies a gap in the use of graphical multiple comparison procedures. The paper aims to provide a way to reject hypotheses using these procedures.
Conflict of interest Platelets sit at the immune-coagulation-vascular interface and provide a distinctive entry point for nanomedicine engineering. Over the past decade, platelet functions have been modularized into synthetic platforms. The central value of platelet biomimicry is selective, controllable reconstruction that decouples efficacy from systemic risk within the structure-membrane-function-gating design space. Across indications, performance gains most often arise from interface state recognition and programmable control at immune- coagulation coupling nodes rather than unconditional procoagulant or aggregation enhancement. Platelet-inspired systems recurrently The author(s) declared that this work was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
Engineering design of platelet-mimicking therapeutic systems: multilevel biomimicry, gating strategies, and translational boundaries · 2026 · DOIThrombopoietin receptor agonists (TPO-RAs) such as eltrombopag have emerged as promising agents in refractory SAA, though evidence of their safety in pregnancy remains scarce.
Use of Thrombopoietin Receptor Agonists in Severe Aplastic Anemia During Pregnancy: A Case Report · 2026 · DOIThe study suggests that future research should focus on developing more accurate predictive models for CTIT. The study suggests that future research should explore the application of machine learning models in other areas of cancer treatment. The study suggests that future research should investigate the potential biases in the dataset.
Predicting the efficacy of recombinant human thrombopoietin in treating cancer therapy-related thrombocytopenia: based on stacking ensemble methods · 2026 · DOISeveral limitations of this study should be acknowledged. First, the clinical cohort comprised a relatively small num- ber of subjects (24 CAD patients and 24 controls), and there were some incomparable confounding factors between the groups (e.g. hypertension, hyperlipidemia, and smoking). These factors reduced statistical power and increased the risk of both type I and type II errors. The cross-sectional design precluded any causal inference regarding the relationship between FVII levels, monocytes activation, and inflamma- tory factors. The high correlations between coagulation and inflammation, observed across the combined cohort, might partly reflect a clustering effect (separation of control group vs. CAD group) rather than a genuine mechanistic link. There- fore, our clinical findings should be considered exploratory, and independent validation in larger, well-matched cohorts is required. Third, the in vitro experiments used a suprapatho- logical concentration of FVIIa (100 nM), which was consid- erably higher than the levels observed in patients with CAD. This raised concerns about the pathophysiological relevance of the observed effects, as supraphysiological stimuli might exaggerate cellular responses. Moreover, NF-κB activation was evaluated only by total protein expression, we did not assess phosphorylation status or nuclear translocation, which would provide more direct evidence of pathway activation. Future studies should use disease-relevant concentrations of FVIIa and incorporate more rigorous experimental valida- tion to confirm the mechanistic link. Collectively, despite these limitations, the study provides interesting and poten- tially impactful insights into the coagulation-inflammatory mechanisms contributing to residual inflammation in CAD.
Crosstalk between the monocytes and coagulation factor VⅡa aggravates the inflammation in patients with CAD · 2026 · DOISex-related bleeding such as heavy menstrual bleeding and post-partum hemorrhage. The rarity of these coagulation defects poses a challenge in the management of pregnancy and delivery. Limited evidence on the management of these disorders during pregnancy and delivery.
Rare inherited autosomal bleeding disorders in women: sex-related bleeding, pregnancy and delivery. A narrative review. · 2026 · DOIThere is limited evidence on the management of rare inherited autosomal bleeding disorders during pregnancy and delivery. The current clinical practice is often based on the current local policy.
Rare inherited autosomal bleeding disorders in women: sex-related bleeding, pregnancy and delivery. A narrative review. · 2026 · DOIThe role of platelets in TB is not well understood. The mechanisms of platelet-leucocyte interactions in TB are not well characterized.
ITP is a heterogeneous disease with varying responses to treatment. The diagnosis of ITP can be challenging due to the lack of a standard diagnostic test. The treatment of ITP can be complex due to the need to balance the risks and benefits of different therapies.
Predictors of Remission following IVIG in Newly Diagnosed Childhood Immune Thrombocytopenia · 2026 · DOITo extend the model to a wider range of shear rates - To develop new treatments for bleeding disorders using the model - To optimize blood flow in medical devices using the model
The lack of a shear-dependent model of platelet aggregation - The limited understanding of the role of shear-dependent interactions in platelet aggregation
There is a gap in understanding the mechanisms underlying the immune dysfunction in patients with ELF4 deficiency. The clinical spectrum of Deficiency in ELF4, X-linked is not fully understood.
A novel ELF4 gene variant disrupts T and NK cell function in a patient with immune thrombocytopenia (ITP) · 2026 · DOIThe complex relationship between autophagy, NET formation, and inflammation in thrombotic autoimmune diseases. The limited understanding of the role of autophagy in thrombotic autoimmune diseases. The need for further research to elucidate the mechanisms of autoimmune responses in these diseases.
The precise mechanisms of autoimmune responses in thrombotic autoimmune diseases remain unclear. The role of autophagy in thrombotic autoimmune diseases is not well understood. The effect of autophagy modulation on NET formation has yet to be elucidated.
Most-cited papers in Platelet Disorders and Treatments
- Aggregation of Blood Platelets by Adenosine Diphosphate and its Reversal · Nature · 1962 · 3,458 citations
- Platelet Activation and Atherothrombosis · New England Journal of Medicine · 2007 · 1,820 citations
- Platelet-rich plasma: evidence to support its use · Journal of Oral and Maxillofacial Surgery · 2004 · 1,524 citations
- Platelet-rich fibrin (PRF): A second-generation platelet concentrate. Part I: Technological concepts and evolution · Oral Surgery Oral Medicine Oral Pathology Oral Radiology and Endodontology · 2006 · 1,295 citations
- Platelet-rich fibrin (PRF): A second-generation platelet concentrate. Part II: Platelet-related biologic features · Oral Surgery Oral Medicine Oral Pathology Oral Radiology and Endodontology · 2006 · 858 citations
- Platelet-rich fibrin (PRF): A second-generation platelet concentrate. Part III: Leucocyte activation: A new feature for platelet concentrates? · Oral Surgery Oral Medicine Oral Pathology Oral Radiology and Endodontology · 2006 · 559 citations
- Growth Factor and Catabolic Cytokine Concentrations Are Influenced by the Cellular Composition of Platelet-Rich Plasma · The American Journal of Sports Medicine · 2011 · 486 citations
- Growth factor levels in platelet-rich plasma and correlations with donor age, sex, and platelet count · Journal of Cranio-Maxillofacial Surgery · 2002 · 477 citations
- Exaggerated platelet reactivity in major depression · American Journal of Psychiatry · 1996 · 417 citations
- The Bleeding Risk and Natural History of Idiopathic Thrombocytopenic Purpura in Patients With Persistent Low Platelet Counts · Archives of Internal Medicine · 2000 · 343 citations
Most recent work
- Adenoviral Inciting Antigen and Somatic Hypermutation in VITT · New England Journal of Medicine · 2026
- Platelet-derived integrin- and tetraspanin-enriched tethers exacerbate severe inflammation · Science · 2026
- Romiplostim versus Placebo for Chemotherapy-Induced Thrombocytopenia · New England Journal of Medicine · 2026
- BCMA-CD3 T-cell engager as salvage therapy for multirefractory primary immune thrombocytopenia · The Lancet · 2026
- Blinatumomab in Combined Immune Thrombocytopenia and Antiphospholipid Syndrome · New England Journal of Medicine · 2026
- Anti–HPA-1a fetal-neonatal alloimmune thrombocytopenia: reframing diagnostics, pathophysiology, and management · Blood · 2026
- Predicting development of pediatric chronic immune thrombocytopenia at disease onset using a statistical risk model · Blood · 2026
- CDK8/CDK19 inhibition restores T-cell homeostasis in primary immune thrombocytopenia · Blood · 2026
- Von Willebrand factor deficiency impairs angiogenesis via angiopoietin-2: relevance for gut angiodysplasia · Blood · 2026
- A Phase 2 Randomized Trial of Mezagitamab in Primary Immune Thrombocytopenia · New England Journal of Medicine · 2026
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