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Background: There is limited information on the natural course of chronic ITP in children. We aimed to evaluate the clinical and demographic characteristics of children with chronic ITP in the era before the availability of eltrombopag. Methods: A total of 86 children with chronic ITP between 1978–2014 were included. Demographic findings, laboratory results, clinical signs, bleeding scores, response time and time of complete remission were recorded. Results: The male/female ratio was 1.09, and median follow-up time was 3 years (range: 1.5–17 years). The median age at diagnosis of chronic ITP was 7 years (range: 2–17), and the median initial platelet count was 10 × 109/L (range: 1–66 × 109/L). Petechiae/ecchymoses were the most common clinical sign (86%) and followed by mucosal bleeding (39.5%). Severe bleeding was seen in 5% of the patients. None of them had intracranial hemorrhage. Twenty patients underwent splenectomy, and the rate of complete remission was 70%. Spontaneous complete remission was seen in 29% of the patients, and the median time to spontaneous complete remission was 3 years. Conclusions: Our study showed that almost one-third of patients with chronic ITP experienced spontaneous complete remission in an average of 3 years, and splenectomy provided satisfactory results in severe cases. This study demonstrates the natural history of chronic ITP in childhood before the era of eltrombopag. [ABSTRACT FROM AUTHOR]

Background: Primary immune thrombocytopenia (ITP) represents the commonest cause of isolated thrombocytopenia among children & adolescents; that's characterized by variable presentations and varied outcomes of disease differentiated into; acute, persistent, or chronic depending upon illness duration; with efforts made to increase knowledge about possible outcome predictors. Aim: we aimed to study disease characteristics & outcome predictors among 50 enrolled patients. Methods: This retrospective cohort study included collection of; demographic data, bleeding phenotype, Buchanan bleeding score onset of symptoms prior to diagnosis, proceeding viral infection or vaccination, initial platelet count & bone marrow finding, received treatment as well as; response to treatment among 50 patients already diagnosed as primary ITP child and adolescent which followed up since 2012 till 2020 in pediatric hematology clinic, Ain Shams University. Patients were further subdivided into acute, persistent & chronic ITP groups. Results: Among included cohort; 38 (76.0%) were males of median (IQR) age of 5.25 (2.5-8.5) years. Chronic ITP represented the most prevalent outcome in the cohort 58.0%(n=29), with acute ITP 32.0%(n=16) & persistent ITP 10.0% (n=5). Among the chronic ITP group in comparison to other groups; female was more than third the patients 34.5% (n=10), 72.4%(n=21) reported >2weeks disease related symptoms with no seasonal peaks of diagnosis, 10%(n=1) presented initially with menorrhagia, Yet no statistical differences between groups regarding associated autoimmune symptoms, bleeding score, nor initial platelet count (p= 0.91, p=0.078, p=0.955) respectively. All chronic ITP groups required steroids in initial treatment with significant difference in response to treatment & recurrence of the disease (p=0.00) as none showed complete remission and all patients experienced disease recurrence. Conclusion: Different presentation of primary ITP. e.g. gender, bleeding symptoms, treatment among children can potentially serve as clinical predictors of outcome. [ABSTRACT FROM AUTHOR]

Background: There is limited information on the natural course of chronic ITP in children. We aimed to evaluate the clinical and demographic characteristics of children with chronic ITP in the era before the availability of eltrombopag. Methods: A total of 86 children with chronic ITP between 1978–2014 were included. Demographic findings, laboratory results, clinical signs, bleeding scores, response time and time of complete remission were recorded. Results: The male/female ratio was 1.09, and median follow-up time was 3 years (range: 1.5–17 years). The median age at diagnosis of chronic ITP was 7 years (range: 2–17), and the median initial platelet count was 10 × 109/L (range: 1–66 × 109/L). Petechiae/ecchymoses were the most common clinical sign (86%) and followed by mucosal bleeding (39.5%). Severe bleeding was seen in 5% of the patients. None of them had intracranial hemorrhage. Twenty patients underwent splenectomy, and the rate of complete remission was 70%. Spontaneous complete remission was seen in 29% of the patients, and the median time to spontaneous complete remission was 3 years. Conclusions: Our study showed that almost one-third of patients with chronic ITP experienced spontaneous complete remission in an average of 3 years, and splenectomy provided satisfactory results in severe cases. This study demonstrates the natural history of chronic ITP in childhood before the era of eltrombopag.

Myosin heavy chain 9 (MYH9)-related platelet disorders (MYH9-RD) belong to the group of inherited thrombocytopenias characterized by giant platelets and Döhle bodies. The process leading to the diagnosis of MYH9-RD in a 13-year-old male patient, followed by the diagnosis of chronic immune thrombocytopenic purpura (ITP), is described. The patient had thrombocytopenia with increased mean platelet volume since he was a little boy. Low CD41, CD42 and CD61 levels were detected in blood tests sent to complete missing diagnostic tests. Platelet aggregation tests were also abnormal. The requested genetic test revealed a heterozygous mutation in the MYH9 gene. The patient's audiogram and kidney functions were normal. In conclusion, because MYH9-RD appears to be rare, it is of great importance to maintain a high index of suspicion when managing patients diagnosed with chronic ITP. Additional complaints and findings should be considered at every outpatient clinic examination to make a more accurate diagnosis and prevent unnecessary treatments. [ABSTRACT FROM AUTHOR]

Background: Primary immune thrombocytopenia (ITP) is a common autoimmune disorder. Secretion of TNF-α, TNF-β and IFN-γ plays a major role in the pathogenesis of ITP. Objective: This cross-sectional study aimed to detect TNF-α (−308 G/A) and TNF-β (+ 252 A/G) gene polymorphism in a cohort of Egyptian children with chronic ITP (cITP) to clarify their possible association with progression to chronic disease. Methods: The study included 80 Egyptian cITP patients and 100 unrelated age- and sex-matched controls. Genotyping was performed using polymerase chain reaction–restriction fragment length polymorphism (PCR–RFLP). Results: Patients with TNF-α homozygous (A/A) genotype had significantly higher mean age, longer disease duration and lower platelet counts (p values 0.005, 0.024 and 0.008, respectively). TNF-α wild (G/G) genotype was significantly more frequent among responders (p = 0.049). Complete response was more frequent among wild (A/A) TNF-β genotype patients (p = 0.011), and platelet count was significantly lower among homozygous (G/G) genotype (p = 0.018) patients. Combined polymorphisms were strongly associated with susceptibility to chronic ITP. Conclusion: Homozygosity in either gene might contribute to a worse course of disease, increased severity and poor response to therapy. Patients expressing combined polymorphisms are more prone to progression to chronic disease, severe thrombocytopenia and longer disease duration. [ABSTRACT FROM AUTHOR]

Chronic immune thrombocytopenic purpura (ITP) is one of the most frequent immune hematological disorders in which development the leading part is played by the antibodies directed against a narrow spectrum of platelet antigens. Though death rate at chronic ITP is insignificant, no more than 1%; disease strongly reduces patients quality of a life, and frequently requires difficult and expensive treatment. Antiplatelets antibodies mediating ITP, are directed against glycoprotein GP IIb/IIIa and, less often, GP Ib/IX. At ITP can be registered both raised bone marrow platelet production and accelerated peripheral blood platelet clearance, and strongly decreased production and normal platelet life-span. Antibodies to platelet membrane glycoproteins directly disturb platelet production in bone marrow. Standard drug interventions at chronic ITP - glucocorticoids in various doses, high doses intravenous immunoglobulin, anti-D immunoglobulin, rituximab - rare lead to stable increase platelet number while in 2/3 patients splenectomy is effective. The new therapy approach in chronic ITP is stimulation of platelet production in bone marrow. Recombinant fusion protein Romiplostim stimulates trombopoietin receptor and it is effective at long-term therapy in 87% patients with chronic ITP irrespective of previous splenectomy. TPO antibodies development with romiplostim therapy was not revealed.

Background : : This study aimed to identify chronicity predictors of pediatric primary immune thrombocytopenia (ITP). Methods : : This study retrospectively reviewed the medical records of patients with primary pediatric ITP admitted to a tertiary medical center between 2010 and 2021. Forty-five patients with a platelet count

Immune thrombocytopenia in children is defined by a decrease in platelet counts < 100 x 109/L. The incidence of the disease varies from 1.6-6 per 100,000 children. Oral hemorrhagic manifestations (petechiae, epistaxis, gingivorrhagia, soft and hard palate hemorrhagic blisters) may be the first manifestations of the disease. Age over 1 year at diagnosis and the sudden onset of the symptom are the most important predictors of chronic disease. Oral hemorrhagic manifestations in ITP (immune thrombocytopenic purpura) in children are a warning sign in the evolution of the disease and a therapeutic challenge. Corticosteroid therapy and IV immunoglobulin, are the first therapeutic line to follow. A multidisciplinary approach is a necessity for the proper management of the child with ITP.

Background: Platelet glycoprotein VI (GPVI) receptor is essential for platelet adhesion and aggregation. Eltrombopag is as an effective treatment for chronic immune thrombocytopenia (ITP); yet, its effect on platelet function is not fully characterized. Aim: This prospective study investigated the effect of eltrombopag therapy on platelet function through assessment of GPVI receptor expression and soluble GPVI levels among pediatric patients with persistent or chronic ITP. Methods: Thirty-six children and adolescents with persistent or chronic ITP were divided equally into two groups either to receive eltrombopag therapy or the standard of care. All patients were followed-up for 12 months with assessment of bleeding score and complete blood count (CBC). Evaluation of GPVI expression using flow cytometry and measurement of its soluble form by ELISA was done at baseline and at 6 months. Results: ITP patients on eltrombopag had significantly lower bleeding score after 6 months of therapy while the quality of life has significantly improved. Platelet count was significantly increased throughout the study. GPVI expression by flow cytometry and soluble GPVI levels were significantly increased after eltrombopag therapy. After 12 months, ITP patients on eltrombopag were able to maintain a good quality of life and low bleeding score. Conclusion: Our data suggest that eltrombopag, through its effect on the GPVI receptor expression and its soluble form, might reduce bleeding manifestations and improve the quality of life of chronic and persistent ITP children independent of its effect on the platelet count. [ABSTRACT FROM AUTHOR]

Thrombopoietin receptor agonists are important therapeutic option in children with immune thrombocytopenia (ITP). We evaluated the response, efficacy, safety of a generic form of romiplostim manufactured in India for treating children with persistent/chronic ITP at our centre. Study of 45 children with persistent/chronic ITP was conducted of which 5 discontinued and 40 were included between 2019‐2020. Patients received romiplostim for 20 weeks, at a dose of 5 mcg/kg/week. Platelet count at week 1, 3, 20 and 26 was assessed. Predesigned algorithm was used for dose adjustment. After 20 weeks, patients who had platelet count of 50 × 109/L or above were tapered off medication and monitored till 26 weeks. Median platelet count at enrolment was 11 x 109/L (IQR 23 X 109/L). 13/40 children had received >/= three lines of prior ITP therapy. Platelet response (platelet count rise to more than 50×109/L without rescue medications) observed in 26 (65%) patients at week 20. Rescue medication was used in 12/40 children. Sustained platelet response after tapering and stopping romiplostim observed in 22/40 children. No adverse events were considered serious or led to discontinuation of treatment. Our data demonstrated generic romiplostim is well tolerated and efficacious in children with persistent/chronic ITP. [ABSTRACT FROM AUTHOR]

Background: Immune thrombocytopenia (ITP) is a benign childhood disease. The aim of this study was to assess Health Related Quality of Life (HRQoL) among chronic ITP children in Vietnam. Methods: A case - control study was conducted from January 2020 to April 2021. Case group consisted of 56 children with chronic ITP aged from 2 to 18 years and their parents selected in the Department of Nephrology-Hematology – Endocrinology, Haiphong Children's Hospital, Hai Phong city. Controls included 170 healthy children matched with the patients on sex, and age recruited from schools in the same city. The validated Vietnamese version of the Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scale was used to assess QoL of the participants. Results: The mean HRQoL physical, emotional, social, school functioning domains and total scores of the chronic ITP patients were significantly lower compared with that of the controls (p

High-dose dexamethasone (HD-DXM) is debated as a second-line therapy for chronic Immune thrombocytopenia (ITP) in children. The aim of this study is to evaluate the efficacy and safety of HD-DXM as an emergency therapy in uncontrolled bleeding in children with chronic ITP and to assess its immunological effect on dendritic cells (DCs) percentage and their co-stimulatory markers CD86 and CD83. Totally, 20 children previously diagnosed as chronic ITP were enrolled in this study and all admitted to hospital with uncontrolled bleeding. Patients received HD-DXM as a single daily dose for 4 days. Blood samples were withdrawn from patients just prior to HD-DXM therapy and on day 5 to evaluate the platelet count and for flowcytometric analysis of DCs. Daily assessment of bleeding severity was performed. The platelet counts significantly increased in patients after 5 days of initiation of therapy compared with platelet count before therapy (p-value = 0. 0002). Control of bleeding observed in (90%), complete response (CR) documented in (50%), response (R) documented in (40%), and no response (NR) documented in (10%) of patients. The time to respond was raging from 1 to 3 days and minor complication recorded in two patients. Both plasmacytoid DCs and myeloid DCs percentage and their expression of co-stimulatory markers, CD86 and CD83 decreased significantly after HD-DXM therapy. Conclusion: short course of HD-DXM as a rescue therapy seems to be an effective alternative emergency treatment for uncontrolled bleeding in chronic ITP children especially in nations with limited resources.

The present case series described six chronic immune thrombocytopenia patients (cITP), with a median age of 7.7 (7.0 to 13.0) years and low platelet count at 15,500 (7,000 to 20,000)/uL. They were suffering from bleeding symptoms and side effects of treatment. After enrollment, they were treated with thrombopoietin receptor agonist (eltrombopag). Five patients responded positively, showing a median platelet count of 115,000 (39,000 to 433,000)/uL. The median dose of eltrombopag used was 1.3 (0.8 to 2.2) mg/kg/day. The quality of life (QoL) improved for all patients, with their median overall score using a Pediatric QoL questionnaire showing 25.0% improvement. Median scores also showed improvements in each sphere of life functioning as physical (30.8%), emotional (26.4%), social (16.4%), and school (21.4%). The present report demonstrated that a select group of cITP patients, with low platelet count and bleeding symptoms, benefitted from treatment with eltrombopag, as shown by increased platelet counts and improved QoL. [ABSTRACT FROM AUTHOR]

There are no definitive guidelines for management of chronic or refractory immune thrombocytopenia (ITP) in children. Dapsone is an inexpensive and efficacious, yet neglected, therapeutic option for treatment of chronic ITP. We evaluated the efficacy and safety of dapsone in the management of chronic ITP in children. Children with chronic ITP < 14 years with minimum grade 2 bleeds refractory to either splenectomy/rituximab/eltrombopag; who were offered dapsone therapy were retrospectively analyzed. Dapsone intolerance and G6PD deficiency were excluded. Dapsone was started at a dose of 1–2 mg/kg/day. Response to dapsone as per international working group definitions, time to response along with side-effects were noted. Forty-four children enrolled; 29 analyzed. Nineteen were refractory to rituximab, 8 to splenectomy and 6 to eltrombopag. Median age was 9.8 years (3–14) with 16/29 males. Median dapsone dose was 1.59 mg/kg/day (range 1–2.1). Overall response was seen in 21/29 (72%): Complete Response in 7/29 (24%), Partial Response in 14/29 (48%). All responses were sustained for minimum 3 months. Median duration to response was 2.9 months (2–6.6). Median follow up was 28 months (6–73) and relapse rate-21%. Major side effects noted: Methemoglobinemia-01, skin ulceration-02. In three cases dapsone could be tapered and stopped without relapse. Dapsone is an economical and efficacious agent with good safety profile in childhood chronic/refractory ITP. [ABSTRACT FROM AUTHOR]

Background: Chronic ITP is a very common disease in children. Most treatment options have focused on slowing platelet destruction like corticosteroids and IVIG. These treatments are not always effective, or have only a transient effect, and treatment related adverse events often restrict further use. Impaired platelet production has been evolved as a potential disease mechanism for ITP. As a result, growth factor and growth factor analogue stimulation of megakaryopoiesis has also been investigated. Objective: is to evaluate the efficacy and safety of eltrombopag for the treatment of children and adolescents with chronic ITP. Subjects and methods: It was a prospective cohort study which was conducted on forty patients (22 males and 18 females) with chronic ITP. They were treated with eltrombopag for a period ranged from 6 months to 2 years after failure of first line of treatment (steroids, IVIG or both). Patients started eltrombopag with mean initial platelet count 14.4 X 103/ uL and were evaluated after at least 6 months of therapy. The study was approved by the academic and medical ethics committee of Zagazig University and a written informed consent was obtained from all patients. Results: Thirty patients (75%) responded to eltrombopag with a mean platelet count of 100.9 X103/ uL while 10 patients (25%) did not respond to the drug. Mean platelet count was significantly higher after eltrombopag therapy (100.9 ± 65.97 versus 14.4 ± 8.87, P < 0.001). The longer the duration of treatment, the higher the mean platelet count after treatment (r = 0.4, P < 0.001). Only 43.3% of responders received combined treatment with IVIG and steroids versus 20% of non-responders (p = 0.006). Conclusion: Eltrombopag is an effective, well tolerated and safe drug to restore platelet counts at levels high enough to prevent bleeding events in children with chronic ITP. [ABSTRACT FROM AUTHOR]

High-dose dexamethasone (HD-DXM) is debated as a second-line therapy for chronic Immune thrombocytopenia (ITP) in children. The aim of this study is to evaluate the efficacy and safety of HD-DXM as an emergency therapy in uncontrolled bleeding in children with chronic ITP and to assess its immunological effect on dendritic cells (DCs) percentage and their co-stimulatory markers CD86 and CD83. Totally, 20 children previously diagnosed as chronic ITP were enrolled in this study and all admitted to hospital with uncontrolled bleeding. Patients received HD-DXM as a single daily dose for 4 days. Blood samples were withdrawn from patients just prior to HD-DXM therapy and on day 5 to evaluate the platelet count and for flowcytometric analysis of DCs. Daily assessment of bleeding severity was performed. The platelet counts significantly increased in patients after 5 days of initiation of therapy compared with platelet count before therapy (p-value = 0. 0002). Control of bleeding observed in (90%), complete response (CR) documented in (50%), response (R) documented in (40%), and no response (NR) documented in (10%) of patients. The time to respond was raging from 1 to 3 days and minor complication recorded in two patients. Both plasmacytoid DCs and myeloid DCs percentage and their expression of co-stimulatory markers, CD86 and CD83 decreased significantly after HD-DXM therapy. Conclusion: short course of HD-DXM as a rescue therapy seems to be an effective alternative emergency treatment for uncontrolled bleeding in chronic ITP children especially in nations with limited resources. [ABSTRACT FROM AUTHOR]

Inherited macrothrombocytopenia is increasingly being recognized as a relatively common condition. This descriptive review aims at focusing on the different areas of advancement that have taken place with this condition with particular reference to India. A pubmed search of articles between January 1990 and October 2017 with the key words-macrothrombocytopenia, asymptomatic macrothrombocytopenia, macrothrombocytopenia India, syndromic macrothrombocytopenia, molecular pathology, megakaryopoiesis and platelet formation were searched. The shortlisted articles were then read. Review articles provided additional references and the articles thus obtained were also read. Special interest and research conducted by the authors provided further sources of information. A total of 487 articles were found of which 68 articles were related to our subject of review. Review articles were read and additional articles from the reference quoted. Forty-four percent of nonsyndromic Inherited macrothrombocytopenia showed mutations of MYH9, GP1BB, GP1Ba, GPIX, ABCG5 and 8, ACTN, FLI, TUBB and RUNX1 frequently in heterozygous state. All types of inheritance pattern namely autosomal dominant, recessive and sex linked patterns have been described. Syndromic causes of this phenomenon are well known and have been described. Many asymptomatic patients do have mild or moderate bleeding history. Clinical algorithms to differentiate chronic ITP associated macrothrombocytopenia from inherited variety have been explored. Inherited macrothrombocytopenia is an emerging area of interest in platelet biology with its implication in diagnosis, prognosis, genetic counseling, management and in transfusion medicine. [ABSTRACT FROM AUTHOR]

Pathogenesis of thrombocytopenia is heterogeneous and resistance to treatment is a great challenge. In this study, we reviewed the demographic features of thrombocytopenic Egyptian patients. We also analyzed the role of T cell in chronic immune cases. IL-12, IL-35, IL-17, and TGF-β were measured by ELISA. The median age at the time of diagnosis was 30 years and its range was 14-70. The median platelet count at the time of diagnosis was 15 × 109/L. Regarding treatment and follow-up, there was an indication for treatment in 96% of patients. Of the 150 ITP patients who were given first-line therapy (corticosteroid 1 mg/kg/day PO), there was a complete response (CR) in 40.3% while 59.7% patients were nonresponsive to therapy. Forty-five chronic cases fulfilled the criteria for cytokine assay. Comparison between the case group and the control group revealed statistically significant lower platelet count in cases, while the four measured cytokines were statistically significant higher in cases rather than the control. Correlation between the platelet count and the level of cytokines was statistically insignificant. The remission rate in ITP on steroid as first-line therapy is less than 50%. The higher expression of IL-12 and IL-35in chronic ITP is due to the persistently higher Th1 activity which explains the continuity of the disease, while the higher expression of Treg cytokines (IL-17 and TGF-β) may be explained by effect of immunosuppression or upregulation of the receptors on Treg cells. [ABSTRACT FROM AUTHOR]

Background: DNA methylation is an epigenetic process that refers to chromatin-based mechanisms in the regulation of gene expression without DNA alternation. It is mediated by DNA methyltransferases (DNMTs). The DNA methyltransferase 3B (DNMT3B) gene contains a C-to-T single nucleotide polymorphism (SNP; rs2424913) in the Promotor region, 149 base pairs from the transcription start site, which is reported to significantly increase the Promotor activity. Objective: To investigate the prevalance of rs2424913 single nucleotide polymorphism located in the DNMT3B gene Promotor. Methods: In the present study, we investigated the prevalence of rs2424913 single nucleotide polymorphism located in DNMT3B gene Promotor by restriction fragment length polymorphism (PCR-RFLP) in Egyptian pediatric chronic immune thrombocytopenia (ITP) patients and controls. Results: The homozygous genotype (TT) was significantly higher in our patient and conferred almost 3-fold increased risk of chronic ITP when compared to controls. Conclusion: The present study shows that DNMT3B rs2424913 promotor polymorphism represents a genetic risk factor that may play an important role in understanding the pathogenesis of chronic ITP. [ABSTRACT FROM AUTHOR]

Introduction: In patients with chronic idiopathic thrombocytopenic purpura (cITP), the platelet count tends to be quite variable and, in the majority of cases, specific therapy is not warranted on a regular basis. However, patients with low platelet count (<30 nL) or with bleeding complications would require therapy, such as prednisolone, intravenous immunoglobulin infusions, splenectomy and/or immunosuppression. Romiplostim, a thrombopoietin agonist, has also proven to be useful in improving platelet counts. cITP can be associated with bleeding complications perioperatively. As such, a higher platelet count is warranted (approximately 80 nL), particularly for invasive surgeries, such as orthopaedic surgery, cardio-thoracic surgery, head and neck surgery and abdominal surgery, where risk of bleeding is quite high already. Aim: The aim of this study is to evaluate the safety and efficacy of short-term use of romiplostim, perioperatively. Methods: Patients with chronic ITP requiring major surgical interventions were enrolled in the study. Patients with malignancies or myelodysplastic syndromes, major bleeding disorders, under 18 years of age or pregnancy were excluded. Conclusion: This study has shown that the use of romiplostim is safe and effective in improving platelet counts preoperatively and that this could achieve excellent haemostasis, with no associated bleeding complications or rebound thrombocytopenia. A larger study involving multiple centres is required to verify these findings. [ABSTRACT FROM AUTHOR]

Eltrombopag is a second-line therapy for refractory thrombocytopenia from immune thrombocytopenic purpura (ITP). The medication is generally well tolerated but can lead to adverse thromboembolic complications in rare instances. We present a case of lower extremity deep vein thrombosis with bilateral pulmonary embolism in an ITP patient receiving eltrombopag. The patient underwent catheter-directed thrombolysis for pulmonary embolism. Eltrombopag was stopped on discharge, considering the potential cause of venous thrombosis., Competing Interests: The authors have declared that no competing interests exist., (Copyright © 2023, Ghumman et al.)

Immune thrombocytopenic purpura (ITP) is the condition caused by increased platelet destruction and or decreased platelet production. Previous studies have demonstrated the association and efficacy of Helicobacter pylori ( H. pylori) eradication therapy in patients with chronic ITP. Data in Thai patients, however, are limited. A prospective cross-sectional analytic study was conducted in adult patients with chronic ITP to determine the prevalence and clinical predictive factors of H. pylori infection and evaluate the efficacy of H. pylori eradication therapy. H. pylori-infected patients received eradication therapy (omeprazole 40 mg/day, clarithromycin 1000 mg/day, amoxicillin 2000 mg/day) for 2 weeks. The platelet counts at baseline and monthly for 6 months after the end of treatment were evaluated. Of the 25 patients, 9 patients (36%) had H.pylori infection. H. pylori infection is higher among women than men. There were two clinical factors included 1) relapsed ITP 2) response after the first-line treatment statistically proven to be associated with H. pylori infection with an odds ratio and p value of 7.7, p = 0.035 and ND (not determined due to small sample size), p < 0.001. Nearly 80% of infected patients had the platelet count response after eradication therapy with the median time to response of 4 months. The prevalence of H. pylori infection is modest in Thai adult patients with chronic ITP. A history of relapsed ITP and high quality of response after first-line treatment indicated H. pylori infection. Therefore, the urea breath test should be recommended in patients who have a relapsed ITP condition with a history of good response after first-line therapy. [ABSTRACT FROM AUTHOR]

Immune thrombocytopenia (ITP) results in part from the presence of platelet antibodies, which can be demonstrated by the Monoclonal Antibody-Specific Immobilization of Platelet Antigens (MAIPA) assay. The aim of our study was to correlate the presence of antiplatelet autoantibodies and the natural history of ITP. We performed a retrospective, single-center study of 108 adults with newly diagnosed ITP who had indirect MAIPA assay performed at disease onset. Chronic ITP was defined by the presence of thrombocytopenia after 1 year. Bleeding diathesis was evaluated with a bleeding score. At baseline, patients with a positive indirect MAIPA have a greater bleeding score than patients with negative MAIPA assay [median (interquartile) = 8 (6-12) vs 2 (0-6), p = 0.002]. Patients with a positive indirect MAIPA also had a higher rate of chronic ITP (92.9 vs 68.7 %, p = 0.06). In multivariate analysis, a positive indirect MAIPA result and a platelet count at onset ≥10 × 10/L remained independently associated with chronic ITP [adjusted OR (aOR) = 8.01; 95 % confidence interval (CI), 0.98-66.6; p = 0.05 and aOR = 3.09; 95 % CI, 1.18-8.10; p = 0.02, respectively]. Furthermore, when we analyzed together the results of direct ( n = 41) and indirect MAIPA, the same results were observed. Thus, indirect MAIPA positivity at disease onset is associated with more severe hemorrhage and predicts a chronic course in adult ITP patients. MAIPA assay could be useful in the management of ITP patients when it is performed at diagnosis. [ABSTRACT FROM AUTHOR]

Background: The pathophysiology underlying primary adult immune thrombocytopenic purpura (ITP) has not yet been identified. However, many mechanisms affect the immune system, causing defective tolerance to self-platelets and megakaryocytes. Cluster of differentiation 40 (CD40) contributes to both humoral and cell-mediated immune responses., Methods: This case‒control study was conducted to detect rs4810485G>T and rs1883832C>T polymorphisms of CD40 in Egyptian patients with persistent/chronic ITP to clarify their possible association with chronic disease evolution. This study included 50 patients with persistent/chronic ITP and 50 healthy controls. Genotyping was performed using the polymerase chain reaction‒restriction fragment length polymorphism technique., Results: Genotyping of rs1883832 and rs4810485 revealed no statistically significant differences between the two groups. However, combined gene polymorphism genotyping showed a statistically significant difference between the two groups ( P <0.01)., Conclusion: Our results indicate a strong association between the combined polymorphism of both genes and susceptibility to developing ITP among adult Egyptian patients. Targeting this pathway using novel therapeutic approaches is promising.

Aim: To describe the clinical course, morbidity and platelet recovery in an unselected Nordic cohort of children with chronic Immune Thrombocytopenic Purpura (ITP). Methods: Prospective 5-year follow-up of 96 children with ITP lasting more than 6 months, with reporting of hospital admissions, severity of bleeding episodes and stabilization of platelet counts above 20, 50 and 150 × 109/L. Results: The estimated 5-year recovery rate was 52%; exclusion of 12 splenectomized children did not change the estimate. Events eliciting admission to hospital occurred in 39 (41%). Major haemorrhages occurred in eight children (8%), including a nonfatal intracranial haemorrhage in one child (1%). The overall admission rate was 0.4/year of thrombocytopenia, decreasing during follow-up as thrombocytopenia converted to milder degrees. Early recovery within 2 years of diagnosis occurred in 35%, was associated with low morbidity and was more likely in young children with abrupt onset of symptoms. Conclusion: In a Nordic cohort of children with chronic ITP, one half had recovered 5 years after diagnosis, more than half never required hospitalization and <10% experienced serious bleeding episodes, always with a platelet count <20 × 109/L. Aggressive management can be restricted to the minority of children with continuing severe thrombocytopenia and frequent, clinically significant bleeding events. [ABSTRACT FROM AUTHOR]

Purpose: To assess the validity and reliability of the fatigue subscale of the Functional Assessment of Chronic Illness Therapy (FACIT-F), a 6-item subset from the thrombocytopenia subscale of the Functional Assessment of Cancer Therapy (FACT-Th6) and the Short Form-36 Version 2 (SF-36v2) in 2 clinical trials of the thrombopoietin receptor agonist eltrombopag in chronic immune thrombocytopenia (ITP) patients. Methods: In the 6-month, RAndomized placebo-controlled ITP Study with Eltrombopag (RAISE; n = 197), the FACIT-F, FACT-Th6, and SF-36v2 were administered at baseline, day 43, weeks 14 and 26, or early withdrawal. In the ongoing open-label extension study, Eltrombopag EXTENDed Dosing Study (EXTEND; n = 154), measures were administered at baseline, at the beginning of each stage, and at permanent discontinuation of study medication. Results: FACIT-F, FACT-Th6, and SF-36v2 demonstrated acceptable internal consistency reliability (i.e., all Cronbach's alphas >0.70) and test-retest reliability (all intraclass correlation coefficients >0.70). Construct validity was supported by moderate (0.35 < r < 0.50) to strong ( r > 0.50) inter-measure correlations for baseline and change scores. A small to medium magnitude of effect was captured by the FACIT-F and FACT-Th6 among patients who experienced sustained platelet responses. Conclusions: Results provide support for the validity, reliability, and responsiveness of the FACIT-F, FACT-Th6, and SF-36v2 in chronic ITP patients. [ABSTRACT FROM AUTHOR]

Romiplostim stimulates thrombopoietin receptor to increase platelet production of megakaryocytes in idiopathic thrombocytopenic purpura (ITP). This study aimed to evaluate the safety and efficacy of romiplostim in children with chronic ITP. Eighteen patients with chronic ITP, either none responsive or failed to maintain response on two or more therapeutic modalities, were enrolled. Patients were randomized (2:1) to receive romiplostim or placebo for 12 weeks, initiated at 1 μg/kg/week, escalated to 5 μg/kg/week, and then tapered. Median patients' age was 8.5 years, and the median baseline platelet count (PC) was 10.5 × 10(9)/L. The median weekly dose of romiplostim was 2 μg/kg. Fifty percent of patients in both romiplostim and placebo arms had at least one adverse event (AE); none was serious. Ten patients on romiplostim (83.3%) maintained the efficacy endpoint (PC > 50,000). Romiplostim was well-tolerated and efficient in treating the children with chronic refractory ITP with no unexpected AEs. [ABSTRACT FROM AUTHOR]

Chronic ITP (immune thrombocytopenic purpura; now defined as duration of more than 12 months) is not always associated with significant bleeding problems so that most children and adults can be managed expectantly with no medication unless surgery, accidents or other pathology mandate it. A cut-off platelet count of 30 x 10(9)/l divides a group with no increased mortality from those whose risk is greater and in whom medication is usually appropriate. There is increasing recognition of long-term morbidity and mortality associated with immune suppression induced by medication and more recently new concerns have arisen about the long-term vascular complications of splenectomy. A more conservative approach to medication is warranted in many patients with chronic ITP. [ABSTRACT FROM AUTHOR]

With regards to research animal models related to immune thrombocytopenia (ITP), there is an extensive literature of over 300 publications published since 1959. It appears that many of these models either confirm what has been found in human ITP or, in some instances, are the first to describe a phenomenon related to ITP that is still of relevance today in human medicine. These models will undoubtedly play a significant role in the future research of human ITP particularly related to understanding of the pathogenesis of the disorder and the development of novel therapeutics. This review will highlight some of the major animal models utilized for ITP research and will present a somewhat historical aspect of the subject. [ABSTRACT FROM AUTHOR]

To evaluate the rituximab treatment in children with chronic immune thrombocytopenic purpura This study included ten children with chronic immune thrombocytopenic purpura, which were nonresponsive to Steroid (S), IVIG and anti-D treatments. Rituximab was given with a dosage of 375 mg/m2 weekly for 4–6 weeks. Initial platelet count was less than 30×109/L and responses were assessed in follow-up. The patients’ groups were categorized as complete remission (CR);a platelet count ≥150×109/L,partial remission (PR);a platelet count ranging from 50×109/L to 150×109/L, minimal remission (MR); a platelet count ranging from 30×109/L to 50×109/L and no response (NR); a platelet count less than 30×109/L. Of our patients, four female and six male, their ages ranged from 39 mth to 13 yr and the mean age was 83.4±44.58 mth. None of the patients was splenectomized. The follow-up period after rituximab treatment ranged between 12 to 42 mth and the mean follow-up period was 25.10±13.03 months. While on this treatment, we had a CR in two patients, a PR in one, a MR in three, but no response in four. The patients in CR/PR are still being followed as in remission and they have 40 mth of mean follow-up period. The three patients in MR had a decrease in values of platelets earliest in one mth and the latest in four mth. Adverse effects of rituximab, such as itching and scraps that were not clinically significant were observed in three patients during rituximab infusion. There were no increase in infections after rituximab in any patient. CR was found in 20% of our patients, PR in 10% and MR in 30% with rituximab. On this treatment, while some series had good outcomes with this treatment (72%–100%, remission ratios), but many series, such as ours, had a poor response rate contrast to many reported case series in the literature. This condition may be associated with the age of our most patients who were young at the time of commenced rituximab. However, we believe that more studies are required to elucidate the reasons for different results in different case series reported in literature. [ABSTRACT FROM AUTHOR]

Rituximab, a chimeric murine/human monoclonal anti-CD20 antibody, was licensed for the treatment of B-cell lymphoma and has also shown efficacy against autoimmune diseases such as immune thrombocytopenic purpura (ITP). It is relatively safe; however, about 1-20% of patients were reported to have developed rituximab-induced serum sickness, which is more common among patients with autoimmune conditions than among those with hematologic malignancies. Here we describe a pediatric patient with steroid-dependent chronic ITP who presented with arthralgia and fever ten days after the second infusion of rituximab (on day 10), and presented with malaise and maculopapular rash on day 21. Oral prednisolone was started and his symptoms resolved. He had an elevated level of human anti-chimeric antibody (HACA) on day 27; thereafter, the HACA level slowly decreased. To our knowledge, among pediatric patients who received rituximab for chronic ITP, this is the sixth documented case of serum sickness and the only one who manifested an elevated level of HACA. Rituximab is a beneficial treatment option against chronic ITP; however, the risk of serum sickness should be considered. Steroid, usually used for the treatment of serum sickness, may prevent the development of severe serum sickness when administered during and after rituximab treatment. [ABSTRACT FROM AUTHOR]

Immune thrombocytopenic purpura (ITP) is typically considered an autoimmune disorder related to the production of autoantibodies; however, recent evidence indicates that cell-mediated cytotoxicity may be important pathogenetically in some cases. We describe seven patients with chronic ITP and concurrent T-cell clonopathy of unknown significance (TCUS), who failed various treatment regimens for ITP, including steroids, gamma globulins, splenectomy and rituximab, but had anecdotal success with azathioprine. These observations support the notion that ITP has heterogeneous biologic mechanisms, and that patients with persistent chronic ITP should be evaluated for T-cell clonality and considered for treatment options that are directed against cytotoxic T-cells. [ABSTRACT FROM AUTHOR]

Abstract: Intracranial hemorrhage is a devastating complication of immune thrombocytopenic purpura (ITP). Subdural hematoma (SDH) is rare and the optimal management unsettled. We report a series of seven patients of ITP who developed isolated SDH and subsequently review the literature. Three patients had acute ITP (mean duration 2.3 months) while four had chronic ITP (mean duration 8.3 years). Mean platelet count at admission was 25×109/L (range 16×109/L to 30×109/L). Mean age at which SDH occurred was 38 years. Headache and bilateral papilloedema were the commonest clinical features at presentation. Diagnosis was made by CT/MR imaging and SDH was acute in two patients and chronic in five. Precipitating factors could not be identified in any patient. Neurological parameters were closely monitored including level of consciousness, pupillary size and development of new neurological deficits. Two patients with acute SDH developed worsening neurological parameters, underwent craniotomy and hematoma evacuation, and survived. Of the two patients who expired, one was brought in an advanced stage that precluded surgery and the other developed metabolic complications due to underlying lupus nephritis. Reports of isolated SDH in ITP are infrequent. Compared to those developing intracerebral hemorrhage, SDH occurs more in patients who are older, having chronic ITP and having a higher platelet count. Medical management with close monitoring of neurological parameters is a viable treatment option in patients of ITP developing SDH. [Copyright &y& Elsevier]

The aim of this study was to evaluate the long-term response to rituximab in patients with chronic and refractory immune thrombocytopenic purpura (ITP). Adults with ITP fail to respond to conventional therapies in almost 30% of cases, developing a refractory disease. Rituximab has been successfully used in these patients. We used rituximab at 375 mg/m2, IV, weekly for a total of four doses in 18 adult patients. Complete remission (CR) was considered if the platelet count was >100 × 109/l, partial remission (PR) if platelets were >50 × 109/l, minimal response (MR) if the platelet count was >30 × 109/l and <50 × 109/l, and no response if platelet count remained unchanged. Response was classified as sustained (SR) when it was stable for a minimum of 6 months. Median age was 43.5 years (range, 17 to 70). Median platelet count at baseline was 12.5 × 109/l (range, 3.0 to 26.3). CR was achieved in five patients (28%), PR in five (28%), MR in four (22%), and two patients were classified as therapeutic failures (11%). Two additional patients were lost to follow-up. The median time between rituximab therapy and response was 14 weeks (range, 4 to 32). SR was achieved in 12 patients (67%). There were no severe adverse events during rituximab therapy. During follow-up (median, 26 months; range, 12 to 59), no other immunosuppressive drugs were used. In conclusion, rituximab therapy is effective and safe in adult patients with chronic and refractory ITP. Overall response rate achieved is high, long term, and with no risk of adverse events. [ABSTRACT FROM AUTHOR]

Summary. In chronic immune thrombocytopenic purpura (ITP), autoantibodies bind to platelet surface proteins, particularly αIIb , resulting in platelet destruction by the reticulo-endothelial system. In order to better localize the autoepitopes on αIIb , we studied the binding of antibodies to Chinese hamster ovary (CHO) cells expressing either αIIb β3 or αIIb -αv β3 chimaeras in which a segment of αIIb (either amino acids L1-Q459, L1-F223 or F223-Q459) was substituted for that portion of αv . We evaluated platelet-associated autoantibodies from 14 ITP patients with αIIb -dependent antibodies. Ten of 14 bound to αIIb (L1-Q459) -αv β3 , showing that autoepitopes were often localized to this region of αIIb . In addition, each of the autoantibodies binding to αIIb (L1-Q459) -αv β3 , also bound to CHO cells expressing either αIIb(L1-F223) -αv β3 or αIIb(F223-Q459) -αv β3 ). In two of the three eluates tested, > 95% of the autoantibody binding to αIIb could be adsorbed using CHO cells expressing any of the three chimaeras, showing that the epitope(s) have contact points on either side of amino acid F223; in the third eluate, only a portion (∼40%) could be adsorbed by the chimaeric cell lines showing that, in this patient, an additional antibody was also present, directed to a site distal to amino acid Q459. The remaining four eluates bound to CHO cells expressing αIIb β3 but to none of the chimaeras, suggesting that these epitopes are also distal to amino acid Q459. We conclude that the binding of many anti-αIIb... [ABSTRACT FROM AUTHOR]

Summary. Chronic idiopathic thrombocytopenic purpura (ITP) is an autoimmune disorder in which activated T-helper (Th) cells and different Th-cell cytokines might play an important role. We have recently reported that chronic ITP patients in remission had elevated plasma levels of the Th3 cytokine transforming growth factor-β1 (TGF-β1), possibly as a part of a bystander immune suppression. In the present study we found that, in ITP patients with active disease [platelet count (plc) < 50 × 109 /l], mitogen-stimulated peripheral blood mononuclear cells (PBMC) had a significantly reduced production of TGF-β1 (444 ± 178 pg/ml; n = 6) compared with patients with plc 50–150 × 109 /l (1293 ± 374 pg/ml; n = 9; P < 0·05), patients with plc > 150 × 109 /l (1894 ± 244 pg/ml; n = 12; P < 0·005) and healthy controls (1698 ± 241 pg/ml; n = 10; P < 0·01). Nineteen per cent of ITP patients expressed a platelet-induced PBMC proliferation. Surprisingly, 22% of the ITP patients had a PBMC proliferation below the normal range, i.e. a suppressed proliferation in the presence of platelets; five of these six patients had active disease. In summary, this study demonstrated that chronic ITP patients with active disease had reduced PBMC production of the Th3 cytokine TGF-β1. This result gives further support to the theory that chronic ITP in active phase is associated with a downregulated Th3-response. [ABSTRACT FROM AUTHOR]

The objectives of this study were to investigate the effectiveness of oral megadose methylprednisolone (OMMP) therapy in children with chronic immune thrombocytopenic purpura (ITP). Twenty‐two patients were given oral methylprednisolone daily for 7 d (30 mg/kg for 3 d and then 20 mg/kg for 4 d). OMMP therapy was repeated once per month if the platelet count was less than 20,000/mm3 at the 30th day of therapy, for up to six courses. The number of platelets of all patients increased gradually during the OMMP therapy, with a peak number at the 7th day, then decreased until the 14th day, and remained relatively stable until 12 months. During the study no patient had a platelet count less than 20,000/mm3 at the 3rd day and 50,000/mm3 at the 7th day. Although the number of platelets was gradually decreased between the 7th and 14th days, it remained above 100,000/mm3 for at least 12 months in the nine patients, and above 20,000/mm3 in the four patients. None of these 13 patients required hospitalization or therapy during the follow‐up period. All of the patients tolerated the medication well. None of them reported side‐effects that were severe enough to discontinue therapy. We conclude that OMMP therapy is a safe, easy and effective therapy in children with refractory chronic ITP, and it may provide long‐term remission in about two thirds of the patients. [ABSTRACT FROM AUTHOR]

Immune thrombocytopenia (ITP) is a rare autoimmune disease that presents along a spectrum of disease severity, ranging from asymptomatic thrombocytopenia to potentially life-threatening bleeding complications. Recent case reports and case series suggest that a COVID-19 infection can trigger secondary ITP and may be associated with higher rates of bleeding and lower nadir platelet counts compared to patients with ITP of other etiologies. Multiple ITP relapses have also been described in some COVID-19 patients. We report the case of a 30-year-old otherwise healthy woman who presented to the hospital with fatigue, easy bruising, and a platelet count of 11 x 10 3 /µL. She responded well to our initial treatment with prednisone and intravenous immunoglobulin (IVIG) but experienced a persistent disease course with nine ITP relapses (defined as platelet count <30 x 10 3 /µL) over the next 10.5 months, requiring six additional hospital admissions for acute management as well as long-term maintenance medication adjustments. It is important for clinicians to recognize ITP as a potential complication of a COVID-19 infection and to initiate early therapy to prevent serious bleeding in these patients. Further studies will be needed to understand the natural history, optimal treatment, and prognosis for patients with relapsing COVID-19-associated ITP., Competing Interests: The authors have declared that no competing interests exist., (Copyright © 2022, Boehm et al.)

Background: Occult hemorrhage can occur in any internal organ in ITP patients. Four sites of occult hemorrhage deserve special attention including microscopic hematuria, fecal occult blood, retinal hemorrhage, and silent intracranial hemorrhage. Aim: The aim of this study was to investigate for the frequency of subclinical bleeding in Egyptian children with ITP and its relation to different clinical and laboratory parameters of the disease. Methods: This cross-sectional study included 40 ITP patients recruited from the Pediatric Hematology& Oncology unit, Children Hospital, Ain Shams University. Occult blood in stools and urine analysis, fundus examination, and non-contrast brain MRI, for brain microbleed, were done. Results: The total number of patients with occult bleeds was eleven. Two patients had occult blood in stool, five had microscopic hematuria, one had retinal bleeds and three patients had brain microbleeds. Nine patients with occult bleeding were chronic, one persistent and one acute ITP patients. Their mean age was 10.23 6 4.18 and their mean initial bleeding score was 2.55 6 0.82.There were no significant differences between patients with occult bleeding and those without as regards the platelet counts & hemoglobin level, as well as the mean platelet counts & mean hemoglobin level over the disease duration. Conclusion: Our results suggest that subclinical bleeding is a potential risk in children with ITP, more commonly chronic ITP patients. We could not demonstrate a significant relation of occult bleeding to the laboratory findings; nevertheless, the significance of the routine assessment of occult bleeding in ITP and the identification of high-risk patients require additional studies. [ABSTRACT FROM AUTHOR]

Idiopathic thrombocytopenic purpura is not an uncommon bleeding disorder with a prevalence of 40-80 per million children per year. Over the last six decades, the subject of ITP has attracted the attention of pediatricians and hematologists. It is one of the subjects which has many controversies because of its unpredictable course heralded by remission relapses, and chronicity with mortality in less than 1% of cases. In the present review only the controversies in the management of acute and chronic ITP have been reviewed as it interests most pediatricians. Management of intracranial hemorrhage (ICH), severe gastrointestinal hemorrhage and menorrhagia continues to still remain a challenge in spite of newer therapies. [ABSTRACT FROM AUTHOR]

Three patients with a history of chronic idiopathic thrombocytopenic purpura stretching back over 20 years are reported. Despite splenectomy and immunosuppressive therapy satisfactory control of their disease has not been achieved. They had remained refractory to all therapeutic manoeuvres with corticosteroids and immunosuppressives for years with thrombocyte counts between 5,000 and 25,000/μl and the concommitant risk of bleeding. This report describes the treatment of bleeding complications in these patients with high dose intravenous immunoglobulin; the peripheral blood thrombocyte count increased in all three patients from subnormal towards normal, but 2 to 4 weeks later returned to its initial low value. During the therapeutically induced raised thrombocyte count a normal bleeding time and only a moderate inhibition of thrombocyte adhesion and aggregation was observed resulting in reasonable haemostasis. High dose intravenous immunoglobulin is therefore a practical method for the control of bleeding complications in patients with refractory chronic idiopathic thrombocytopenic purpura. A clear explanation for its mode of action has not been found - the lymphocyte subpopulations remained unchanged and immunoglobulin production in vitro during the course of treatment was only minimally decreased. [ABSTRACT FROM AUTHOR]

Background: Immune Thrombocytopenia (ITP) is characterized by low platelet counts. Splenectomy has been in practice for the treatment of ITP since the early 20th century. We aimed to analyze the data of ITP patients from our hospital who underwent splenectomy and further present the long-term outcome and safety profile in these patients., Method: This study was a single-center, registry based study conducted at a tertiary care hospital in Northern India. Patients aged 18 years or more, who underwent splenectomy after at least one line of therapy, were included in the study. The primary outcome was the overall response rate (ORR) at one month after splenectomy. Secondary outcomes were sustained response, relapse-free survival, factors affecting the ORR, and adverse events after splenectomy., Results: Forty-five patients of ITP were included in the study. Thirty-six patients underwent splenectomy in the first half (2001-2010), of the study period. The median age of the patients was 38 (19-56) years. The median duration from diagnosis to splenectomy was 1.76 (0.47-2.58) years. The median number of therapy received before splenectomy was 3 (1-6). The overall response rate (ORR) post-splenectomy at day 30 was 89.2% with 61.8% complete response (CR). The ORR was 88.5% at 1-year, with 48.8% CR. The relapse-free survival (RFS) at 5-years was 57.38% (95% Confidence Interval 40.59-71.02%), There was no effect of duration of disease, age, gender, and prior therapy received, on the ORR at one-month. At one year, the platelet response was significantly better in patients who had a CR at one-month than patients who had a partial response at one month. The relapse-free survival was better in patients who achieved CR after 1-month of splenectomy. During the median follow-up of 5.02 (1 month-20 years) years, there were five cases of overwhelming post-splenectomy infection (OPSI). There was no recorded incidence of perioperative mortality, deep vein thrombosis, or mesenteric thrombosis., Discussion: Despite the variation in outcome from different studies, splenectomy gives the best possible long-term treatment-free remission amongst all the available second-line agents. It is also, one of the most financially affordable therapies. Despite advantages, the number of ITP patients undergoing splenectomy has been on the decline and largely attributable to the newer and more effective second-line therapies. There is no pre-surgery variable predicting the ORR after splenectomy., Conclusion: Splenectomy in ITP offers a long-term sustained response at an economical cost., Competing Interests: None., (AJBR Copyright © 2021.)

Background: Immune thrombocytopenia (ITP) is a benign hematological disorder characterized by low platelet counts in peripheral blood and spectrum of various bleeding manifestations. Azathioprine is one of the effective, readily available, and affordable immunosupressants available for ITP management in developing countries. We aimed to study the efficacy and long-term safety profile of our patients with ITP who were treated with azathioprine., Method: This was a retrospective, single-center study conducted at a tertiary care hospital in Northern India. The patients who had received at least one line of therapy before receiving azathioprine were included in this study. All patients received oral azathioprine at a dose of 1 mg/kg/day (50 mg or 100 mg tablet formulations were used), which was increased up to 2 mg/kg/day depending upon the response and adverse effects., Result: Sixty-three patients were analyzed. Their median age was 28 years (range 15-68); 29/63 patients (46.03%) were females. The median duration from diagnosis to azathioprine initiation was 539 days (323 days-980.5 days). The patients included in the study had received a median of 3 (range 1-6) prior lines of therapies; 38/63 patients (60.32%) had received ≥3 prior therapies. Six patients (9.5%) had relapsed after splenectomy, and 16 patients (25.4%) had relapsed after receiving rituximab. The mean baseline platelet count was 10000/μL. The median time to response was 95 days (90 days-not reached) and the cumulative overall response rate (complete and partial response) at day 90 was 38.1%. Only one patient achieved complete response with azathioprine in our study. The cumulative rate of relapse at five years was 21.2%. Twenty-six patients stopped azathioprine after achieving some response (CR/PR) with Azathioprine for a median duration of 1067.5 days (range: 236 days-2465 days). They were followed up for a median of 870 days (range: 392 days-1928 days), and twelve of them relapsed. Twenty-six patients (26/63, 41.27%) reported one or more adverse events while on azathioprine. Leucopenia was the most frequent adverse event, followed by anemia and hepatobiliary laboratory abnormalities. Serious adverse events (grade ≥3 CTCAEv4) were noted in three patients (4.7%). One patient succumbed to severe sepsis multiorgan dysfunction while being on treatment., Conclusion: We conclude that azathioprine has a good response rate in chronic ITP patients. It is well-tolerated with minimal and manageable side effects., Competing Interests: None., (AJBR Copyright © 2021.)