Efficacy of Low-Dose Rituximab in Primary Immune Thrombocytopenia

Objective:To explore the effect of low-dose rituximab in primary immune thrombocytopenia.Methods:From January 2022 to January 2023,60 patients with primary immune thrombocytopenia were randomly divided into two groups.The control group was treated with standard doses of rituximab,and the observation group was treated with low doses of rituximab.Rituximab was used for treatment,and the clinical curative effect of the two groups was observed.Results:Before treatment,there was no statistically significant difference in platelet count(PLT),anti-GPⅡb/Ⅲa antibody,and anti-GPⅠb/Ⅸantibody between the two groups(P>0.05).After treatment,the PLT of the two groups increased significantly.Antibodies were all decreased,and there was no significant difference between the two groups(P>0.05).The incidence of adverse reactions in the observation group was 13.33%,and that in the control group was 40.00%.The adverse reactions in the observation group were significantly lower than the control group(P<0.05).Conclusion:In the clinical treatment of primary immune thrombocytopenia,low-dose rituximab can control the progression of the disease,improve blood routine indicators,and have fewer adverse reactions.

Study on the Biological Basis of Qi, Blood, and Vessel in Immune Thrombocytopenia Patients With Syndrome of Qi Failing to Govern Blood Based on the Theory of Qi and Blood

Objective: To investigate the biological basis of qi, blood and vessel in immune thrombocytopenia(ITP) patients with syndrome of qi failing to govern blood(SQFGB) based on traditional Chinese medicine.Methods: A total of 52 ITP patients with SQFCB were enrolled and divided into bleeding group(38 cases) and non-bleeding group(14 cases).Bleeding group was further divided into mild qi deficiency group(25 cases) and moderate/severe qi deficiency group(13 cases) based on Chinese Medicine syndrome score.20 healthy volunteer were recruited as control group.The count of platelet(PLT) was taken as the blood related indicator.The expressions of cytokines including IL-1β, IL-17 A, TNF-α, CD40 L, and TGF-β, detected by Aim Plex Multiple Immunoassays for Flow, were taken as the qi related indicators.The expressions of VEGF-A, detected by Aim Plex Multiple Immunoassays for Flow and NO, NOS, and ET-1 detected by ELISA, were taken as the vessel related indicators.Results: As compared to the control group, the count of PLT, taken as the blood related indicator, was significantly lower in ITP group patients with SQFCB(P<0.05).The expression levels of IL-17 A and TNF-α, taken as the qi related indicators, were significantly higher, while those of CD40 L, IL-1β, and TGF-β, also taken as the qi related indicators, were significantly lower in ITP patients with SQFCB, respectively(P<0.05).The expression levels of NO and ET-1, taken as the vessel related indicators, were significantly higher, while the expression levels of NOS and VEGF-A also taken as the vessel related indicators, were significantly lower in ITP patients with SQFCB, respectively(P<0.05).The count of PLT, taken as the blood related indicator, was significantly lower in moderate/severe group than those in mild group(P<0.05).The expression levels of CD40 L and TGF-β, taken as the qi related indicators, were also significantly lower in moderate/severe group than those in mild group, respectively(P<0.05).Conclusion: The count of PLT might be the biological basis of blood.The expressions of NO, NOS, ET-1 and VEGF-A might be the biological basis of vessel.The expressions of IL-1β, IL-17 A, TNF-α, TGF-β, and CD40 L may be the biological basis of qi.The expressions of CD40 L and TGF-β could reflect the degree of qi deficiency in ITP patients based on the theory of qi and blood.

Effectiveness of total therapy in immune thrombocytopenia purpura:case series

Immune Thrombocytopenic purpura(ITP)is a haematologicimmune-mediated disorder in which the amount of platelet in the blood decreases abnormally.Single-agent therapies for ITP have not proven successful in achieving long-term remission,with relapse occurring in about half of the patients(p/t).Treatment options which include Rituximab with Dexamethasone as frontline therapy,have durable response rates ranging from 58%to 76%.In this study,we have used‘Total therapy’as treatment which includes low-dose Rituximab in combination with Thrombopoietin receptor agonist(TPO-RA)(Romiplostim)and high-dose Dexamethasone.In this case series study,each patient received romiplostim(250 mcg weekly s/c,4 doses)in combination with low-dose rituximab(100 mg weekly IV,4 doses)and high-dose dexamethasone(40 mgIV on days 1-4and days 15-18).This treatment combination demonstrated rapid response rates and a low rate of side effects,making it a good alternative for individuals with ITP.

Expression and Significance of Regulatory B Cells in Patients with Immune Thrombocytopenia

Objective: To detect the expression and significance of regulatory B cells in patients with immune thrombocytopenia. Methods: 73 ITP patients were divided into glucocorticoids treatment group (n = 42) and recombinant human thrombopoietin (rhTPO) treatment group (n = 31). According to the therapeutic effect, it was divided into effective group and ineffective group. The expression of CD19+ CD24hiCD38hi Breg in peripheral blood was detected by flow cytometry before and after treatment. The expression levels of transforming growth factor (TGF)-β1, interleukin (IL-10) and interferon (IFN)-γ were detected by ELISA before and after treatment. 30 volunteers were selected as the control group. Results: The expression of CD19+ CD24hiCD38hi Breg and cytokines IL-10 and TGF-β1 in 73 ITP patients before treatment was lower than that in the control group, while the expression of IFN-γ was higher than that in the control group (p < 0. 05). The expression levels of CD19+ CD24hiCD38hi Breg, IL-10 and TGF-β1 in the effective group were significantly higher than before treatment, while the expression of IFN-γ was significantly lower than before treatment (p < 0. 05). The expression of CD19+ CD24hiCD38hi Breg, IFN-γ, IL-10 and TGF-β1 in the invalid group had no significant change compared with before treatment. Conclusion: Abnormal expression of CD19+ CD24hiCD38hi Breg and related cytokines is involved in the pathogenesis of ITP.

MST4 kinase regulates immune thrombocytopenia by phosphorylating STAT1-mediated M1 polarization of macrophages

Primary immune thrombocytopenia(ITP)is an autoimmune hemorrhagic disorder in which macrophages play a critical role.Mammalian sterile-20-like kinase 4(MST4),a member of the germinal-center kinase STE20 family,has been demonstrated to be a regulator of inflammation.Whether MST4 participates in the macrophage-dependent inflammation of ITP remains elusive.The expression and function of MST4 in macrophages of ITP patients and THP-1 cells,and of a macrophage-specific Mst4−/−(Mst4ΔM/ΔM)ITP mouse model were determined.Macrophage phagocytic assays,RNA sequencing(RNA-seq)analysis,immunofluorescence analysis,coimmunoprecipitation(co-IP),mass spectrometry(MS),bioinformatics analysis,and phosphoproteomics analysis were performed to reveal the underlying mechanisms.The expression levels of the MST4 gene were elevated in the expanded M1-like macrophages of ITP patients,and this elevated expression of MST4 was restored to basal levels in patients with remission after high-dose dexamethasone treatment.The expression of the MST4 gene was significantly elevated in THP-1-derived M1 macrophages.Silencing of MST4 decreased the expression of M1 macrophage markers and cytokines,and impaired phagocytosis,which could be increased by overexpression of MST4.In a passive ITP mouse model,macrophage-specific depletion of Mst4 reduced the numbers of M1 macrophages in the spleen and peritoneal lavage fluid,attenuated the expression of M1 cytokines,and promoted the predominance of FcγRIIb in splenic macrophages,which resulted in amelioration of thrombocytopenia.Downregulation of MST4 directly inhibited STAT1 phosphorylation,which is essential for M1 polarization of macrophages.Our study elucidates a critical role for MST4 kinase in the pathology of ITP and identifies MST4 kinase as a potential therapeutic target for refractory ITP.

Combination of two target agonists of the thrombopoietin and thrombopoietin receptor in the treatment of elderly patients with refractory immune thrombocytopenia

Immune thrombocytopenia(ITP)is common in the elderly.Because of the coexistence of multiple diseases,there are many reservations regarding corticosteroid use in the elderly.Thrombopoietin(TPO)and its analogs can promote platelet production,but it is often difficult to correct TP in a short period.Recombinant human TPO(rh-TPO)acting on the cell membrane and the small-molecule TPO-receptor(MPL)agonist acting on the transmembrane receptor may have synergistic effects and accelerate platelet production because of different sites of action in the signaling pathway.In this study,two elderly patients with refractory ITP were successfully treated with two TPO-MPL signaling pathway agonists:recombinant human thrombopoietin(rh-TPO)and eltrombopag.This combination is safe with rapid and lasting effects.However,in elderly patients with refractory,recurrent,and glucocorticoid contraindications,the combination of different TPO agonists'clinical efficacy and adverse reactions needs to be further evaluated.

Effect of Yiqi Tongyang Decoction(益气通阳方) on Blood T Cell Subsets in Patients with Chronic Immune Thrombocytopenia

Objective： To measure the proportions of blood T cel subsets, Th1, Th2, Th17, Th22, and Treg cel s, and other parameters in patients with chronic immune thrombocytopenia（CITP） before and after treatment with Yiqi Tongyang Decoction（益气通阳方, YTD） to explore T cel status of patients with CITP, and to define the mechanism of action of YTD. Methods： The changes in peripheral blood T lymphocyte subsets, and those of Th1, Th2, Th17, Th22, and Treg cel s in 30 patients with CITP（22 females and 8 males） were analyzed using multiparametric flow cytometry before and after treatment with YTD for 6 months, and 26 healthy volunteers（14 males and 12 females） acted as a control. T-box expressed in T-cel s（T-bet） and GATA binding protein 3（GATA-3） m RNA levels in patients and controls were analyzed using real-time reverse transcription-polymerase chain reaction. Results： The proportions of Th1, Th17, Th22, Th1/Th2, and Th17/Treg cells increased in the peripheral blood of patients with CITP compared to those in controls before YTD therapy（P〈0.05）. Th1 cel numbers and the Th1/Th2 ratio fel in the treated patients with CITP to approximate the values of the control group（P〉0.05）. Th17 cel numbers and the Th17/Treg ratio also decreased in the treatment group（P〈0.05）, but not to the levels of the controls. The number of Treg cel s in the peripheral blood of patients with CITP before treatment was lower than that in the control group（P〈0.05）, but increased after YTD treatment（P〈0.05）, but not to the level of controls. T-bet and GATA-3 m RNA levels in peripheral blood were initially higher in patients before treatment than controls（P〈0.05）, but decreased after YTD therapy（P〈0.05）. Conclusions： Imbalances in T lymphocyte levels, particularly those of Th1/Th2 and Th17/Treg cel s, play important roles in the pathogenesis of CITP. YTD efficiently regulated the dynamics of Th1/Th2 and Th17/Treg equilibria.

Recombinant human thrombopoietin in combination with cyclosporin A as a novel therapy in corticosteroid-resistant primary immune thrombocytopenia

Background The management of patients with refractory immune thrombocytopenia （ITP） is challenging, as there is no standard treatment option. The aim of this study was to investigate the efficacy of recombinant human thrombopoietin （rhTPO） in combination with cyclosporin A （CsA） for the management of patients with corticosteroid-resistant primary ITP.

Gut microbiome alterations and its link to corticosteroid resistance in immune thrombocytopenia

Quantitative metagenomic studies have linked the gut microbiota to autoimmune disorders.Here,we performed deep shotgun metagenomic sequencing of fecal samples from 99 immune thrombocytopenia(ITP)patients and 52 healthy controls.Dysbiosis in the gut microbiome of ITP was detected phylogenetically and functionally,and classifier based on species markers distinguished individuals with ITP from healthy controls.In particular,the abundance of Ruminococcus gnavus,Bifidobacterium longum and Akkermansia muciniphila was markedly increased in treatment-na?ve ITP patients,and the alterations of microbial species were correlated with clinical indices.Functionally,the secondary bile acid biosynthesis and flagellar assembly were depleted in the gut microbiota of ITP,which may contribute to the onset of ITP by affecting the immune system.Furthermore,we found that corticosteroid treatment affected the gut microbiome of ITP.Compared with corticosteroid-sensitive ITP patients,we identified that the corticosteroid-resistant ITP patients displayed a distinct gut microbiome,which was different from that of the treatment-na?ve ITP patients.Together,we provided support for the critical role of gut microbiota in the development of ITP and established a foundation for further research characterizing gut microbiota in relation to corticosteroid resistance of ITP.

Glucocorticoid receptor modulates myeloid-derived suppressor cell function via mitochondrial metabolism in immune thrombocytopenia

Myeloid-derived suppressor cells(MDSCs)are a heterogeneous population of immature cells and natural inhibitors of adaptive immunity.Intracellular metabolic changes in MDSCs exert a direct immunological influence on their suppressive activity.Our previous study demonstrated that high-dose dexamethasone(HD-DXM)corrected the functional impairment of MDSCs in immune thrombocytopenia(ITP);however,the MDSC population was not restored in nonresponders,and the mechanism remained unclear.In this study,altered mitochondrial physiology and reduced mitochondrial gene transcription were detected in MDSCs from HD-DXM nonresponders,accompanied by decreased levels of carnitine palmitoyltransferase-1(CPT-1),a rate-limiting enzyme in fatty acid oxidation(FAO).Blockade of FAO with a CPT-1 inhibitor abolished the immunosuppressive function of MDSCs in HD-DXM responders.We also report that MDSCs from ITP patients had lower expression of the glucocorticoid receptor(GR),which can translocate into mitochondria to regulate the transcription of mitochondrial DNA(mtDNA)as well as the level of oxidative phosphorylation.It was confirmed that the expression of CPT-1 and mtDNA-encoded genes was downregulated in GR-siRNA-treated murine MDSCs.Finally,by establishing murine models of active and passive ITP via adoptive transfer of DXM-modulated MDSCs,we confirmed that GR-silenced MDSCs failed to alleviate thrombocytopenia in mice with ITP.In conclusion,our study indicated that impaired aerobic metabolism in MDSCs participates in the pathogenesis of glucocorticoid resistance in ITP and that intact control of MDSC metabolism by GR contributes to the homeostatic regulation of immunosuppressive cell function.

Advances in immunopathogenesis of adult immune thrombocytopenia

Primary immune thrombocytopenia(ITP)is an autoimmune disorder characterized by immune-mediated accelerated platelet destruction and/or suppressed platelet production.Although the development of autoantibodies against platelet glycoproteins remains central in the pathophysiology of ITP,several abnormalities involving the cellular mechanisms of immune modulation have been identified,and the pathways behind the immune-mediated destruction of platelets have opened new avenues for the design of specific immunotherapies in an attempt to reduce the platelet destruction.This review is primarily focused on the recent literature with respect to immunopathological mechanisms in patients with ITP.

Switching between eltrombopag and recombinant human thrombopoietin in patients with immune thrombocytopenia: an observational study

Background:Recombinant human thrombopoietin(rh-TPO)and eltrombopag are two distinct TPO receptor agonists(TPO-RAs)with different mechanisms.During the pandemic,when immunosuppressive medications are controversial,switching to another TPO-RA may be worth exploring in patients who do not benefit from their first TPO-RA.We investigated the outcomes of switching from rh-TPO to eltrombopag or vice versa in immune thrombocytopenia(ITP)patients.Methods:This prospective,open-label,observational investigation included 96 adult ITP patients who needed to switch between rh-TPO and eltrombopag between January 2020 and January 2021 at Peking University People’s Hospital in China.The study evaluated response rates and platelet counts at different time points after the switch,bleeding events,time to response,duration of response,and adverse events.Results:At 6 weeks after switching,response was observed in 21/49 patients(43%)who switched for inefficacy and 34/47 patients(72%)who switched for non-efficacy-related issues.In the inefficacy group,9/27 patients(33%)responded to eltrombopag,and 12/22 patients(55%)responded to rh-TPO.In the non-efficacy-related group,21/26(81%)and 13/21(62%)patients in the eltrombopag and rh-TPO groups maintained their response rates at 6 weeks after switching,respectively.Response at 6 months was achieved in 24/49 patients(49%)switching for inefficacy and 37/47 patients(79%)switching for non-efficacy issues.In the inefficacy group,13/27 patients(48%)responded to eltrombopag,and 11/22 patients(50%)responded to rh-TPO.In the non-efficacy-related group,22/26 patients(85%)and 15/21 patients(71%)in the eltrombopag and rh-TPO groups maintained their response rates at 6 months after switching,respectively.Both eltrombopag and rh-TPO were well tolerated.Conclusions:Our study confirmed the safety and effectiveness of switching between rh-TPO and eltrombopag for ITP patients who had no response to or experienced adverse events with their first TPO-RA.When the switch was motivated by other reasons,including patient preference and platelet count fluctuations,the probability of response was high.Registration:ClinicalTrials.gov,NCT04214951.

ADAP restraint of STAT1 signaling regulates macrophage phagocytosis in immune thrombocytopenia

Heightened platelet phagocytosis by macrophages accompanied by an increase in IFN-γplay key roles in the etiology of immune thrombocytopenia(ITP);however,it remains elusive how macrophage-mediated platelet clearance is regulated in ITP.Here,we report that adhesion and degranulation-protein adaptor protein(ADAP)restrains platelet phagocytosis by macrophages in ITP via modulation of signal transducer and activator of transcription 1(STAT1)-FcγR signaling.We show that ITP was associated with the underexpression of ADAP in splenic macrophages.Furthermore,macrophages from Adap^(−/−)mice exhibited elevated platelet phagocytosis and upregulated proinflammatory signaling,and thrombocytopenia in Adap^(−/−)mice was mitigated by the depletion of macrophages.Mechanistically,ADAP interacted and competed with STAT1 binding to importinα5.ADAP deficiency potentiated STAT1 nuclear entry,leading to a selective enhancement of FcγRI/IV transcription in macrophages.Moreover,pharmacological inhibition of STAT1 or disruption of the STAT1-importinα5 interaction relieved thrombocytopenia in Adap^(−/−)mice.Thus,our findings not only reveal a critical role for ADAP as an intracellular immune checkpoint for shaping macrophage phagocytosis in ITP but also identify the ADAP-STAT1-importinα5 module as a promising therapeutic target in the treatment of ITP.

Chinese contribution to immune thrombocytopenia： the pathogenesis-oriented treatment

Primary immune thrombocytopenia （ITP） is the most common hemorrhagic disorder characterized byisolated thrombocytopenia in the absence of conditions known to cause thrombocytopenia. The incidence rate of ITP is similar in males and females; the incidence was significantly higher among patients older than 60 years） The clinical manifestation of bleeding varies with patient. The severity of bleeding ranges from mild mucocutanous bleeding （petechiae and ecchymoses） to life-threatening hemorrhages （intracranial or gastrointestinal bleeding）; some patients have no bleeding manifestations. Fatal and major nonfatal hemorrhages were extremely higher in patients older than 60 years old.2 The thrombocytopenia in ITP has been traditionally thought to be caused by the production of autoantibodyies targeting platelet antigens. Autoantibodies attach to the platelets and are destructed by macrophages in the reticuloendothelial system. Understanding of the mechanisms of thrombocytopenia in ITP has greatly expanded in recent years. The pathogenesis of ITP is heterogeneous and complex. The thrombocytopenia in ITP is caused not only by increased platelet destruction but also by impaired platelet production. The advances in the understanding of ITP have led to the development of a novel nonimmunologic therapeutic approach by stimulating platelet production and even revised guidelines in the diagnosis and treatment of ITE3 In the process of exploring the pathogenesis of ITP, the researchers from all over the world have done a lot of studies. This review will provide an insight into the pathogenesis-oriented treatment of ITP, mainly focusing on the studies of Chinese researchers.

Rapid progressive vaccine-induced immune thrombotic thrombocytopenia with cerebral venous thrombosis after ChAdOx1 nCoV-19(AZD1222)vaccination:A case report

BACKGROUND Vaccine-induced immune thrombotic thrombocytopenia(VITT)is a rare and potentially life-threatening condition after receiving coronavirus disease vaccines.It is characterized by symptom onset at 5 to 30 d postvaccination,thrombocytopenia,thrombosis,high D-dimer level,and antiplatelet factor 4(anti-PF4)antibody positivity.VITT can progress very fast,requiring urgent management.Only few studies have described its detailed clinical course and imaging changes.We report a typical VITT case in a patient who underwent regular repeated brain imaging examinations.CASE SUMMARY A young woman presented with headaches at 7 d after the ChAdOx1 nCoV-19vaccine(AZD1222)injection.She then showed progressive symptoms of left upper limb clumsiness.Brain computed tomography revealed venous infarction at the right parietal lobe with a hyperacute thrombus in the cortical vein.Two hours later,brain magnetic resonance imaging revealed hemorrhage at the same area.Magnetic resonance venography showed an irregular contour of the right transverse sinus.Laboratory examination revealed a high D-dimer level,thrombocytopenia,and a high titer for anti-PF4 antibodies.She was treated with anticoagulants,intravenous immunoglobulin,and steroids and analgesic agents were administered for pain control.She had a marked improvement on headaches and clumsiness after treatment along with radiological thrombus resolution.During follow-up at the outpatient department,her modified Rankin scale at 90 d was 1.CONCLUSION Clinicians should be alerted whenever patients present with persistent and progressive headaches or focal motor/sensory deficits postvaccination.

General Anesthesia for Cesarean Section in a Pregnant Woman with Immune Thrombocytopenic Purpura (ITP): A Case Report and Review of the Literature

Background: Management of immune thrombocytopenia (ITP) during pre- gnancy can be challenging, particularly by identifying a threshold for safe administration of neuraxial/general anesthesia and minimizing postpartum hemorrhage. There is controversy over the safety of cesarean section (CS) in ITP patients. In this case report, we discuss general anesthesia management in a patient with ITP with severe thrombocytopenia. Case Presentation: A 28-year-old female with relapsed/refractory ITP and severe thrombocytopenia underwent general anesthesia and emergent cesarean section with successful outcomes and minimal bleeding. Platelet counts before CS were 5000 × 109 L, the patient received 1 unit of platelets before the procedure and 1 unit of platelet and tranexamic acid 500 mg was injected slowly during the procedure. No evidence of bleeding and no complications were observed in the patient or newborn. Conclusions: In an emergent circumstance, general anesthesia and cesarean section procedure were performed safely in a patient with severe thrombocytopenia, no hemorrhagic complications were seen for this patient or neonate. Objective of This Manuscript: To share our experience of a safe emergent CS procedure and general anesthesia in a patient with severe thrombocytopenia. Our experience may guide the management of ITP patients in emergent delivery circumstances.

Association between Immunological Thrombocytopenia and Thrombosis, Is It an Exotic Phenomenon?—A Case Report

Immunologic thrombocytopenia (ITP) is an autoimmune disease associated with the production of autoantibodies against specific platelet membrane glycoproteins. A thrombotic event as an unusual occurrence during ITP is becoming more and more frequent. In fact, several recent studies have shown an increased thrombotic risk in this situation. The case presented here is that of a fifty-one-year-old woman with extensive cerebral venous thrombosis 2 years after her ITP diagnosis. During IT, thrombosis may be triggered by the release of pro-thrombotic platelet micro-particles and by platelet activation due to the interaction between autoantibodies and platelet glycoproteins. Immunosuppressive therapy has also been linked in several studies to the thrombotic phenomenon. Increased thromboembolic risk should be taken into account in all ITP patients.

Diagnostic Approach of Thrombocytopenia in Pregnancy: A Review

Thrombocytopenia (defined as platelet count 9/L) is present in 7% - 12% of pregnant women at delivery. Although there are mild etiologies of this condition that are often diagnosed incidentally, there are more severe causes that can be life threating. Thrombocytopenia also has a great implication in surgical risk and regional anesthesia. A structured evaluation of thrombocytopenia is necessary to allow an adequate diagnostic approach. Here we summarized the current knowledge of thrombocytopenia in pregnancy.

The Treatment of Chinese Herbal Prescription in a 43 Year-old Woman with Sjogren Syndrome: A Case Report

Background:Sjögren syndrome(SS)is a long-term autoimmune disease that affects the body’s moisture-producing glands.It can occur independently of other health problems(primary disease)or as a result of another connective tissue disorder(secondary disease).Medication is directed at the person’s symptoms.Recently,it is reported that prescription of Chinese medicine has a great effect on SS and reduce adverse drugs reaction,which can provide a more safer treatment to SS.Case presentation:We here report a 43 year-old Chinese patient with continuous immune thrombocytopenia diagnosed as Sjögren syndrome,who was not response to routine medication such as Methylprednisolone and acyclovir.After that,the patient was treated by prescription of Chinese medicine,and the symptoms were improved apparently.We suppose that prescription of Chinese medicine has an unexpected effect on SS,which can alleviate patient’s symptoms.Conclusion:The clinical efficacy of Sjogren syndrome(SS)is effective in the treatment of prescription of Chinese medicine.It can improve patient’s symptoms and reduce adverse drug reactions.

基于个体化机器学习的原发性免疫性血小板减少症危重出血预测模型:一项全国前瞻性队列研究

原发性免疫性血小板减少症(ITP)中少见但至关重要的危重出血事件,给患者的预后、生活质量和治疗决策带来严重影响。尽管有一些研究探讨了ITP中与危重出血相关的风险因素,但目前尚缺乏大样本数据、大规模多中心研究结果以及针对ITP患者致命出血事件的预测模型。本研究首次采用国际血栓与止血学会新提出的ITP致命出血标准,利用大样本数据开发了首个基于机器学习的在线应用,用于预测ITP患者的致命出血.研究中,我们使用中国各地大型多中心数据进行开发,并对全国39家医疗中心进行为期一年的外部测试,得到了较好的训练、验证和测试数据集预测能力该基于新算法的便捷网络工具能够快速识别ITP患者的出血风险,辅助临床决策,有望未来降低不良事件的发生。