Treatment Of Immune Thrombocytopenia Research Articles

On the horizon: upcoming new agents for the management of ITP.

Treatment of immune thrombocytopenia (ITP) has evolved over the last 20 years in response to our increased understanding of the pathophysiology of this complex immune disorder. New treatments in development have taken advantage of our evolving understanding of the biology of this disease to target new mechanisms and expand the available ways in which to approach patients with this disorder. This review focuses on novel therapeutics in the ITP pipeline and discusses the pathophysiology of ITP that has led to their development.

Insights on treatment of adult ITP: algorithm for management and role of multimodal therapy.

The management of immune thrombocytopenia (ITP) is continuously evolving with the development and introduction of newer therapies and a better understanding of the disease. Corticosteroids still represent the cornerstone of first-line treatment. Patients who fail to achieve remission with a short course of corticosteroids require subsequent therapy. Most guidelines recommend starting with either a thrombopoietin receptor agonist (TPO-RA), rituximab, or fostamatinib since these agents have been investigated in randomized trials and have well-characterized efficacy and safety profiles. Patients' involvement to reach a shared decision regarding choice of therapy is essential as these treatments have different modes of administration and mechanisms of action. Less than 10% will fail to respond to and/or be intolerant of multiple second-line therapeutic options and thus be considered to have refractory ITP and require a third-line therapeutic option. Such patients may require drugs with different targets or a combination of drugs with different mechanisms of action. Combining a TPO-RA and an immunomodulatory agent may be an appropriate approach at this stage. Many studies have been conducted during the last 2 decades investigating the efficacy and safety of combinations strategies for first and later lines of therapies. Yet none of these are recommended by current guidelines or have gained wide acceptance and consensus.

Romiplostim, low-dose rituximab and high-dose dexamethasone for newly diagnosed immune thrombocytopenia: A pilot study.

Therapies such as corticosteroids, thrombopoietin receptor agonists and immunomodulators have been consistently under the spotlight in the search for a better treatment for immune thrombocytopenia (ITP). However, none of them has been widely embraced as a new standard. In this pilot study, we investigated feasibility, safety and preliminary efficacy of romiplostim, low-dose rituximab and high-dose dexamethasone for newly diagnosed ITP. We conducted a single-centre single-arm pilot. One-month treatment adherence was 100%. The 1-month overall response rate was 92.3%. Complete response occurred in 11 (84.6%) patients. Median duration of response was 4.13 monts (range 0.2-25). No serious adverse events presented.

Inhibition of FcRn with rozanolixizumab in adults with immune thrombocytopenia: Two randomised, double‐blind, placebo‐controlled phase 3 studies and their open‐label extension

SummaryPrimary immune thrombocytopenia (ITP) is an antiplatelet‐antibody‐mediated disorder with accelerated platelet clearance and decreased platelet production. Rozanolixizumab, a monoclonal IgG4 anti‐FcRn antibody, blocks IgG recycling and decreases IgG levels. We report efficacy and safety of rozanolixizumab in adults with persistent/chronic ITP in 24‐week phase 3 studies (TP0003; TP0006), and their 52‐week open‐label extension (OLE). Primary end‐point was durable clinically meaningful platelet response (DCMPR) of ≥50 × 109/L for 8/12 weeks during Weeks 13–25 in the double‐blind studies. Operational delays and evolving ITP treatment landscape led the sponsor to terminate these studies early; thus, only 21 and 12 (TP0003) and 20 and 10 (TP0006) patients were randomised to rozanolixizumab or placebo. Forty‐three patients enrolled in the OLE: 42 started on every 2‐week dosing; 21 later switched to weekly dosing. More rozanolixizumab‐treated than placebo‐treated patients achieved DCMPR: 4/21 versus 0 (TP0003) and 1/20 versus 0 (TP0006). Platelet increases to ≥50 × 109/L were observed on Day 8 in 52.4% (TP0003; 2/12 placebo) and 45.0% (TP0006; 1/10 placebo) of rozanolixizumab‐treated patients. OLE platelet increases were maintained while on weekly dosing. The most frequent treatment‐emergent adverse events overall were headache, pyrexia and nausea, as seen previously. Weekly dosing appears more efficacious than every 2‐week dosing.

A novel small molecule phagocytosis inhibitor, KB-208, ameliorates ITP in mouse models with similar efficacy as IVIG.

The characteristic feature of immune cytopenias involves the process of extravascular phagocytosis, wherein macrophages in the spleen and/or liver engage in the destruction of blood cells that have been opsonized by auto- or alloantibodies. Therefore, new treatments that prevent phagocytosis will be advantageous, especially for short-term usage along with alternative options. KB-208, a small molecule drug, previously shown to be efficacious for the in vitro inhibition of phagocytosis was synthesized. A passive antibody mouse model of immune thrombocytopenia (ITP) was used. Three different mouse strains (BALB/c, C57BL/6, CD1) were used to determine the efficacy of KB-208 compared with IVIG to ameliorate the ITP. Toxicity was investigated after 60-day chronic administration of KB-208 by a biochemistry panel, gross necroscopy and histopathology. KB-208 showed similar efficacy to ameliorate the thrombocytopenia compared with IVIG in all three mouse strains. This small molecule drug was effective at 1 mg/kg in ameliorating ITP, in comparison with IVIG at 1000-2500 mg/kg. KB-208 did not affect other blood parameters or elevate serum biochemistry markers of toxicity nor were any abnormal histopathological findings found. KB-208 is similar to IVIG for the amelioration of ITP in multiple mouse strains. Chronic administration of KB-208 for 60 days did not demonstrate in vivo toxicity. These findings indicate that KB-208 is efficacious, without significant in vivo toxicities in mice, and is a potential small molecule candidate for further evaluation to be used in the treatment of ITP and possibly all immune cytopenias where phagocytosis is responsible for the pathophysiology.

Health-related quality of life and complications of corticosteroid treatment in patients with immune thrombocytopenia in two teaching hospitals in Ethiopia: a cross-sectional study.

The treatment of immune thrombocytopenia (ITP) is a major clinical challenge and has a significant impact on health-related quality of life (HRQoL), and prolonged use of corticosteroids may have a negative impact on HRQoL. To evaluate the impact of ITP on HRQoL and complications of corticosteroid treatment in patients with ITP in two teaching hospitals in Ethiopia. The institution-based cross-sectional study was conducted from November 15, 2022, to March 15, 2023, to recruit 214 study participants during the study period (4 months). The ITP Life Quality Index (ILQI) in the Amharic version was used to assess the impact of ITP. Linear regression analysis models were also used, and a p-value of less than 0.05 was generally considered statistically significant. Regarding treatment, the majority 172 (80.4%) of study participants were taking prednisolone only, and 143 (66.8%) of study participants had at least one side effect of corticosteroids during the entire treatment period. Predictive factors for a higher impact of ITP on HRQoL; all correlated variables explained 36.5% (adjusted R-squared = 0.365, p < 0.0001) of the variance and had a moderate impact on HRQoL. Furthermore, predictive factors for an increasingly higher impact of ITP on HRQoL were the development of emotionally related corticosteroid side effects (β = 0.392, 95% CI: 5.160-9.961, p < 0.001), the presence of fatigue during the assessment (β = 0.326, 95% CI: 4.394-9.475, p < 0.001), patients not taking cotrimoxazole prophylaxis treatment (β = 0.236, 95% CI: 2.236-6.570, p < 0.001), living far from the hematology clinic (outside Addis Ababa) (β = 0166, 95% CI: 1.107-5.114 p = 0.003), having epistaxis and/or wet purpura (mucosal bleeding) (β = 0.191, 95% CI: 0.091-4.259, p = 0.001), and skin symptoms (petechiae and ecchymosis) (β = 0.041, 95% CI: 0.091-4.259 p = 0.041) during diagnosis. The impact of ITP on their energy levels and work life was high compared to the impact of ITP on daily life. The side effects of corticosteroids also affect the HRQoL of ITP patients.

Management of Adult Patients with Newly Diagnosed or Relapsed Primary Immune Thrombocytopenia in Eastern Austria.

Treatment sequence in primary immune thrombocytopenia (ITP) is based on national and international recommendations, treatment availability, and physician expertise. This article aimed to provide real-world data on treatment sequence and responses to first- and second-line treatments in newly diagnosed and relapsed adult ITP patients. We analyzed a cohort of 46 adult ITP patients from the Vienna ITP Biobank, who started first-line therapy within 1 week before their first study visit between February 2016 and March 2023. We investigated clinical patient characteristics and patient management in our specialized center and examined the impact of the international ASH guidelines on ITP treatment. Forty-six primary ITP patients, 27 (58.7%) with newly diagnosed ITP and 19 (41.3%) with relapsed ITP, were investigated. Most patients were female (65.2%) with a median platelet count of 9 × 109/L, and 31 patients (67.4%) had bleeding symptoms. All patients received first-line treatment with oral prednisolone; 15 patients received oral prednisolone combined with intravenous immunoglobulins (IVIGs), which were more commonly administered in newly diagnosed than in relapsed ITP patients. First-line therapy resulted an overall response in 82.6% of patients after a median (interquartile range [IQR]) time of 10 (5-25) days. There was no difference in treatment responses between newly diagnosed and relapsed ITP patients, but newly diagnosed patients had a shorter time to response (median [IQR]: 8 [5-14] and 14 [8-27], p = 0.02). Twenty-three (50%) of the patients (11/27 newly diagnosed [40.7%], 12/19 relapsed [63.2%]) required second-line ITP therapy. Thrombopoietin-receptor agonists (TPO-RAs) were the most commonly used second-line therapy with a response rate of 73.7%, and a median (IQR) time to treatment response of 15 (12-20) days. Overall response rates to TPO-RA treatment did not differ between newly diagnosed and relapsed ITP. Following the publication of novel guidelines in 2019, the median (IQR) duration of corticosteroid treatment shortened (100-52 days, p = 0.01), as did the time to second-line treatment (160-47 days, p = 0.01), and the median number of first-line therapies decreased from 2 (1-3) to 1 (1-2). Initial treatment with corticosteroids was effective in the majority of newly diagnosed and relapsed ITP. Response rates to initial corticosteroid treatment in ITP patients are consistent with previous data, but only 50% achieve sustained remission. TPO-RAs, which are well tolerated and effective, are the most commonly used second-line therapy in our study population. International guidelines have led to faster treatment transitions and reduced splenectomy rates. Integration of real-life experience, expert consensus, and guidelines optimizes ITP patient management.

Early Treatment Use of Fostamatinib for Immune Thrombocytopenia (ITP): Efficacy and Safety As Second/Third ITP Treatment Lines in Clinical Practice in Spain

Early Treatment Use of Fostamatinib for Immune Thrombocytopenia (ITP): Efficacy and Safety As Second/Third ITP Treatment Lines in Clinical Practice in Spain

Case report:Clinical manifestations and therapeutic options following rhTPO-induced neutralizing antibody production in a child with immune thrombocytopenia.

Recombinant human thrombopoietin (rhTPO) is commonly used to improve low platelet status in immune thrombocytopenia (ITP), as one protein product, even with a very low rate, there is still the possibility to produce neutralizing antibodies of thrombopoietin (TPO). We described a 7-year-old boy with ITP and normal TPO levels who had previously received rhTPO for 2 weeks but showed persistent thrombocytopenia and was misdiagnosed as acquired amegakaryocytic thrombocytopenia (AATP) and ineffectively treated with cyclosporine A (CsA) in combination with avatrombopag (AVA). As suspicious the TPO neutralizing antibody development as re-test of TPO level is 0, the CD20 + deletion antibody drug rituximab (RTX) was prescribed and received efficacy. rhTPO serves as one bio-protein drug and should be cautious with developing neutralizing antibodies if the drug effect is lost. The tests for antibody and/or TPO level should be done for the diagnosis, and the antibody eradication medication as an anti-CD20 antibody, RTX, should be prescribed to delete thes e neutralizing antibodies to recover the TPO level to reattain the response of ITP treatment.

The Establishment of a Novel Murine Model of Immune Thrombocytopenia in Pregnancy and the Impacts of Thrombopoietin Receptor Agonist on Platelet Production.

Objective Immune thrombocytopenia (ITP) is frequently associated with pregnancy. However, treatment options for ITP in pregnancy are limited, and there are few animal models available for the establishment of treatments. Here, we aimed to establish a novel murine pregnant model of ITP and to investigate the impacts of thrombopoietin receptor agonist (TPO-RA) on platelet production and reproductive outcomes. Methods Anti-glycoprotein Ib-alpha (GPIbα) antibody, which binds to megakaryocytes and platelets, was subcutaneously administered to pregnant mice in order to develop an ITP model (ITP group). TPO-RA was given in doses of 1 µg/kg, 10 µg/kg, and 100 µg/kg (low-dose group, mid-dose group, and high-dose group, respectively) for the treatment of ITP in pregnancy. Results The ITP group showed a significant reduction of platelet counts of less than 15% of healthy pregnant mice (control group) and also showed a significant increase in miscarriage rate (control group, 3.8%; ITP group, 44.4%; p < 0.05). Striking increases in platelet counts were observed in every TPO-RA group without any negative effects on fetal growth and placental pathology. No abnormality was noted in the external examination of fetal mice. Interestingly, a significant recovery of miscarriage rate was observed in the mid-dose group (23.5%) compared with the ITP group (p < 0.05). Conclusion A novel ITP model in pregnant mice was induced by injection of an anti-GPIbα antibody, and sufficient effects of TPO-RA on platelet production were observed in the present study. Furthermore, the positive impacts of TPO-RA on reproductive outcomes were revealed in the ITP model.

A Predictive Model for Treatment Effectiveness in Severe Primary Immune Thrombocytopenia during Pregnancy: A Retrospective Study in a Tertiary Critical Maternity Referral Center

Objectives: The study aimed to identify factors influencing the severity of primary immune thrombocytopenia (ITP) during pregnancy, develop a predictive model for treatment response, and report maternal and neonatal outcomes associated with severe ITP. Design: A retrospective analysis was conducted on 155 pregnancies with severe ITP between January 2018 and April 2023 at a tertiary critical maternity referral center in Shanghai, China. Participants/Materials: The study included 155 pregnancies diagnosed with severe ITP, divided into groups based on the lowest platelet count (<30 × 109/L vs. 30–50 × 109/L) and first-line treatment response (non-response vs. response). Setting: The study was conducted at Renji Hospital, Shanghai Jiao Tong University School of Medicine, a tertiary critical maternity rescue referral center. Methods: Clinical characteristics and outcomes were compared between groups. A multivariable logistic regression model was used to identify factors associated with the severity of ITP. A prediction model for treatment response was established using LASSO-logistic regression and internally validated. Results: ITP severity was found to be correlated with low maximum amplitude of thromboelastography (OR 5.43, 95% CI: 1.48–16.00, p = 0.002), bleeding events (OR 4.91, 95% CI: 1.62–14.86, p = 0.005), and low reticulocytes (OR 2.40 × 10−7, 95% CI: 1.06 × 10−13 to 0.55, p = 0.04). Of the 118 patients who received first-line therapy, 52 (44%) responded. The dataset was randomly split into a training (N = 99) and test (N = 23) set with a ratio of 8:2. A predictive nomogram was created and internally validated showing good discrimination. The model yielded an area under receiver operating characteristic curve of 0.78 (0.69–0.87) and 0.85 (0.67–1.00) in the training and validation cohort, respectively. Earlier delivery and high rate of neonatal intensive care unit admission occurred with severe ITP and treatment failure. Limitations: The study was limited by a relatively small sample size and the retrospective observational design, which imposed limitations on the assessment of treatment efficacy. Conclusions: We identified clinical predictors of ITP severity and treatment resistance during pregnancy. A nomogram predicting first-line response was validated. These findings can facilitate clinical decision-making and counseling regarding this challenging pregnancy complication.

Clinical characteristics, treatment, and outcomes of nivolumab induced immune thrombocytopenia.

Immune thrombocytopenia (ITP) represents an uncommon hematological side effect associated with nivolumab, and its distinct clinical attributes remain poorly defined. This research aimed to explore the clinical manifestations and outcomes of ITP induced by nivolumab. Reports on nivolumab induced ITP up to April 30, 2024, were collected for retrospective analysis. The study involved 34 patients with a median age of 67 years (range 32, 82). The onset of ITP varied from 10 to 100 days post initial dosage, with a median onset at 70 days. The majority of patients exhibited no symptoms, with only 23.5% experiencing clinically significant bleeding and 11.8% facing non-clinically significant bleeding. The median platelet count was 12 × 109/L (range 0, 115), with 67.6% of patients having platelet levels below 25 × 109/L. Bone marrow biopsy revealed mainly elevated megakaryocytes. Platelet-associated IgG levels were elevated with a median of 210 ng/107 cells (range 73, 1130). Subsequent interventions, which included cessation of nivolumab, administration of systemic corticosteroids, intravenous immunoglobulin therapy, a thrombopoietin receptor agonist, platelet transfusion, and rituximab treatment, resulted in 82.4% of subjects achieving normalized platelet counts, whereas 5.9% passed away due to ITP. ITP is a rare life-threatening immune-related adverse event and necessitates close monitoring. Systemic steroids are the primary treatment for ITP, while intravenous immunoglobulin, thrombopoietin receptor agonist and rituximab are other options.

Exploratory study on the individualized application of eltrombopag in paediatric immune thrombocytopenia guided by therapeutic drug monitoring.

There are variations in individual eltrombopag concentrations that may impact efficacy and adverse drug reactions (ADRs) in paediatric immune thrombocytopenia (ITP). To solve this problem, we tailored the eltrombopag dosage refer to concentration, then followed up to assess concentration value in paediatric ITP. This is a single-centre, prospective, observational study. The eltrombopag dosage was adjusted, and children were divided into three groups: the maintenance, discontinuation, and increase groups. Concentration and other data were compared to explore concentration value in guiding the individualized treatment of paediatric ITP. Thirty-nine patients were enrolled, including 23 in the maintenance group, 3 in the discontinued group and 13 in the increase group. 3 patients discontinued eltrombopag due to ADRs, which was significantly higher than patients in the maintenance group (t = 3.606, p = 0.001). In all, 13 patients increased their dosage due to poor response, whose concentration and platelet count were significantly lower than patients in the maintenance group (t = 2.461, p = 0.019; t = 4.633, p < 0.001). Two months after the increase, the number of patients reaching CR and R respectively increased by 2 and 3, while the median platelet count was significantly raised (Z = -2.411, p = 0.016). Concentration could be used as a reference index for the individualized treatment of eltrombopag in paediatric ITP.

2025 update on clinical trials in immune thrombocytopenia.

Although the development and regulatory approval of the thrombopoietin receptor agonists revolutionized aspects of the immune thrombocytopenia (ITP) treatment landscape over the past two decades, there remain many areas of high unmet need. Therefore, a number of investigational and repurposed agents are currently undergoing clinical development in ITP. In a departure from historical trials, which largely focused on the indefinite treatment of persistent or chronic ITP, ongoing trials run the gamut of disease phases, and include novel agents being evaluated in early phases of the disease to attempt to modify the disease course. Many agents in development target disease pathophysiologic mechanisms not previously targeted by agents in current use, including platelet autoantibody recycling, B-cell maturation and differentiation, long-lived plasma cells, and the complement system, among others. These agents represent promising treatment options for patients with otherwise refractory disease or who are intolerant of currently available therapies. Additionally, with our increasing understanding of the diverse immune mechanisms at play in ITP, the expansion of the therapeutic armamentarium to include agents targeting diverse pathophysiologic mechanisms may allow a more personalized therapeutic selection in the future. This manuscript provides an up-to-date, in-depth overview of recently completed and ongoing clinical trials in ITP.

Could machine learning revolutionize how we treat immune thrombocytopenia?

The absence of reliable biomarkers in immune thrombocytopenia (ITP) complicates treatment choice, necessitating a trial-and-error approach. Machine learning (ML) holds promise for transforming ITP treatment by analysing complex data to identify predictive factors, as demonstrated by Xu etal.'s study which developed ML-based models to predict responses to corticosteroids, rituximab and thrombopoietin receptor agonists. However, these models require external validation before can be adopted in clinical practice. Commentary on: Xu etal. A novel scoring model for predicting efficacy and guiding individualised treatment in immune thrombocytopenia. Br J Haematol 2024; 205:1108-1120.

Retrospective Evaluation of Survival and Prognostic Factors in Immune Thrombocytopenia: A Single-Center and Cross-Sectional Study.

Background and Objectives: Immune thrombocytopenia (ITP) is an autoimmune disease characterized by the autoantibody-mediated destruction of platelets. The treatment of ITP aims to maintain a sufficient platelet count to prevent bleeding. First-line treatment options include corticosteroids and intravenous immunoglobulin (IVIg), while second-line treatments include splenectomy, rituximab and other immunosuppressive agents, and thrombopoietin (TPO) receptor agonists. This study aims to discuss the treatment methods and results from 100 patients with ITP at the Muğla Training and Research Hospital through a pharmacological approach. Materials and Methods: Demographic characteristics, clinical findings, bone marrow aspiration and biopsy results, and treatments and treatment responses at the time of diagnosis of the 100 patients with ITP who were treated and followed up in the period 2015-2023 were evaluated retrospectively. Results: In the third month after treatment, the overall response percentage was 100% in patients who received steroids only and 88% in patients who received IVIg treatment alone or in combination with steroids (p > 0.05). The most preferred second-line treatments were splenectomy (41%), eltrombopag (26%), and rituximab (10%). Bone marrow biopsy was performed in 54% of patients, where 35.1% showed increased megakaryocytes, 44.4% adequate megakaryocytes, and 14.8% decreased megakaryocytes. It is noted that eltrombopag and rituximab, in particular, yield higher complete remission rates than immunosuppressive drugs. Conclusions: Considering the side effects of immunosuppressive medications, IVIg, splenectomy, and steroid therapy, the use of new agents such as eltrombopag, which are easily tolerated and have a lower risk of side effects, is expected to increase.

Real-world clinical outcomes with fostamatinib for the treatment of refractory chronic immune thrombocytopenia: a single-center experience.

Fostamatinib is a spleen tyrosine kinase inhibitor indicated for the treatment of chronic immune thrombocytopenia (ITP) unresponsive to a previous treatment. Real-world studies evaluating the utilization and effectiveness of fostamatinib outside the context of a clinical trial are lacking. The objective of this analysis was to evaluate the effectiveness of fostamatinib for the treatment of ITP in a real-world cohort. We conducted a single-center, retrospective, observational study to evaluate the effectiveness of fostamatinib for the treatment of ITP. The primary endpoint was durable response as defined by the American Society of Hematology ITP response criteria. Secondary endpoints included overall response rate, time to response, and safety. Subgroup analysis was performed to assess frequency of durable response in key subgroups of patients based on prior therapies. Thirty-one patients treated with fostamatinib for ITP were included in our analysis. Patients had received a median of four prior lines of therapy. Ten patients (32%) achieved a durable response. Most durable responders maintained their response at 24 months ( n = 7; 70%). The median time to response was 9 days. Four patients (13%) discontinued fostamatinib due to an adverse event. Subgroups who had higher rates of durable responses included those who had received two to three prior lines of therapy (40%), splenectomized patients (50%), and those who had not received prior rituximab (55%). Fostamatinib therapy in a real-world population of patients with heavily pretreated ITP led to a durable response in a third of patients, which was maintained for most responders.

Первичная иммунная тромбоцитопения у детей: анализ эффективности лечения в соответствии с Национальными клиническими рекомендациями

AIM. To assess the treatment efficacy in children with primary immune thrombocytopenia (ITP). MATERIALS &amp; METHODS. The analysis included 13 ITP patients (6 girls and 7 boys) with the mean age of 9.5 years (range 4–17 years) admitted to the Pediatric Hospital of the Tver region in 2023. Primary ITP was preceded by infection in 9 (69 %) children and by measles vaccination in 1 (8 %) child. The mean time after infection onset was 11 days (range 5–15 days). As assessed by the pediatric bleeding score, hemorrhage severity was of grade 1 in 4 (31 %), grade 2 in 3 (23 %), and grade 3 in 6 (46 %) patients. Hematuria was identified in 3 (23 %) patients, and menorrhagia was detected in 1 (8 %) patient. The mean platelet count by the time of admission to the hospital was 9 × 109/L (range 1–86 × 109/L). RESULTS. The incidence of newly diagnosed primary ITP appeared to be 5.7 cases per 100,000 children in the Tver region. Treatment started with 20 mg/m2 IV dexamethasone on Day 1–3 in 7 (54 %) children, 1000 mg/kg IVIG on Day 1 in 2 (15 %) children, and 2 mg/kg prednisolone per os during 21 days in 1 (8 %) child. Dynamic follow-up was conducted on 3 (23 %) patients. In 2 (17 %) patients, steroid therapy had to be discontinued and IVIG was started due to increased hemorrhage and/or developing corticosteroid complications. Partial and complete responses were achieved in 8 (62 %) and 4 (31 %) patients, respectively. In 1 (8 %) case, the response was not assessed. The overall response rate to the first-line therapy was 92 %. The mean platelet count at hospital discharge was 107 × 109/L (range 20–307 × 109/L), and the mean number of hospital days was 10.2 (range 2–23). The platelet count decrease to grade 1/2 was observed in 3 (23 %) children during the first 6 weeks followed by spontaneous complete recovery during the subsequent 1–2 weeks. CONCLUSION. The treatment of pediatric primary ITP in full compliance with the National Clinical Guidelines showed very high efficacy in terms of achieving primary response and preventing relapses. The relatively high overall IVIG use rate was accounted for by a slow response to corticosteroids, increased hemorrhage, risk factors, and parental concern.

Real-world experience of thrombopoietin receptor agonists in pediatric immune thrombocytopenia: a report from a Chinese tertiary children's hospital.

Primary immune thrombocytopenia (ITP) is the most common bleeding disorder in children. There are approximately 20% pediatric ITP patients respond poor to corticosteroids as first-line treatment. Recently thrombopoietin receptor agonists (TPO-RAs) have been used to treat refractory ITP and have achieved certain therapeutic effects. To investigate the efficacy and safety of TPO-RAs in the treatment of pediatric ITP, we conducted this real-world study. Fifty-three pediatric patients with ITP who did not respond well to corticosteroids were treated with TPO-RAs. Clinical data, including therapeutic response rate, changes in platelet (PLT) count, and adverse events (AEs) were collected. Of the 51 evaluable patients, 37 (72.5%) responded to TPO-RAs. Patients aged >4 years had a higher response rate than those aged ≤4 years (81.1% vs. 50.0%, P=0.04). There was no effect of sex, duration of disease, prior therapy, Mycoplasma pneumoniae (MP) immunoglobulin M (IgM) positivity, antinuclear antibody (ANA) positivity, CD4/CD8 ratio or baseline PLT count on the response rate (P>0.05). Other than 10 patients with PLT counts that exceeded the upper limit of normal, AEs were sporadic, including increased aminotransferase levels, cough, headache, and vomiting. TPO-RAs exhibited good clinical efficacy in pediatric ITP patients who failed to respond to first-line treatment, especially patients aged >4 years, and the side effects were minor.

Treatment of Immune Thrombocytopenia: Contextualization from a Historical Perspective.

Immune thrombocytopenia (ITP) is an autoimmune disease characterized by an isolated decrease in platelet count and an increased risk of bleeding. The pathogenesis is complex, affecting multiple components of the immune system and causing both peripheral destruction of platelets and inadequate production in the bone marrow. In this article, we review the treatment of ITP from a historical perspective, discussing first line and second line treatments, and management of refractory disease.