Adult Idiopathic Thrombocytopenic Purpura Research Articles

Ways to Improve the Results of Treatment of Idiopathic Thrombocytopenic Purpura in Adults

Ways to Improve the Results of Treatment of Idiopathic Thrombocytopenic Purpura in Adults

Adult idiopathic thrombotic thrombocytopenic purpura therapeutic modalities and outcome: 15 years’ experience

Adult idiopathic thrombotic thrombocytopenic purpura therapeutic modalities and outcome: 15 years’ experience

Reference guide for the treatment of adult idiopathic thrombocytopenic purpura

The treatment strategy of adult idiopathic thrombocytopenic purpura (ITP) in Japan had consisted of corticosteroids as the first-line option, splenectomy as the second-line option, and others as the third-line option, respectively, for a long time. However, thrombopoietin receptor agonists (TPO-RAs) have been widely adopted recently for corticosteroid-resistant ITP, and indications for rituximab have been extended to adult ITP in Japan, suggesting that ITP treatment is dramatically changing. In the "Reference guide for adult ITP treatments in Japan" revised in 2019, TPO-RAs and rituximab are equally recommended as second-line treatments alongside splenectomy. It is suggested to select from among the second-line treatments with careful consideration of not only their advantages and disadvantages but also aspects of the condition and situation of each patient such as any comorbidities or lifestyle factors. Moreover, since multiple effective therapeutic options are now available as second-line options, it is preferable to consider an early transition to second-line treatments for corticosteroid-refractory/dependent ITP patients.

Reference guide for management of adult idiopathic thrombocytopenic purpura (ITP): 2019 version

Reference guide for management of adult idiopathic thrombocytopenic purpura (ITP): 2019 version

Effect of rhIL-11 on Th1/Th2 and T-bet/GATA-3 imbalance in idiopathic thrombocytopenic purpura

Objective To investigate the effect of recombinant human interleukin 11 (rhlL-11) in the treatment of idiopathic thrombocytopenic purpura (ITP) on the levels of Th1, Th2 and the expression of their transcription factors T-bet mRNA, GATA-3 mRNA. Methods Fifty-six cases adult ITP patients hospitalized in the department of hematology of the Second People’s Hospital of Datong from May 2015 to December 2016 were collected, including 21 males and 35 females, aged 29~73 years; 10 healthy people in the same period were enrolled as control group, 4 males and 6 females, aged 20~52 years.Th1 and Th2 cell ratio and Th1/Th2 ratio of ITP patients were detected by flow cytometry before and after treatment.The expression levels of transcription factor T-bet and GATA-3 were measured using real-time fluorescence quantitative reverse transcription polymerase chain reaction (RT-PCR) before and after treatment. Results The effective rate of rhlL-11 in ITP treatment was 76.8%(43/56). For the effective patients, the median PLT after treatment increased (25.0 (15.0, 36.0)×109/L vs.68.0 (49.0, 108.0)×109/L, Z=-5.712, P 0.05). Conclusion rhIL-11 can effectively correct the imbalance in Th1 and Th2 cells and the imbalance of T-bet and GATA-3 in ITP patients, but it has no obvious therapeutic effect on a small number of patients Key words: Idiopathic thrombocytopenic purpura; Recombinant human interleukin-1; T Helper cells 1; T helper cells 2; T-box expressed in T Cells; GATA binding protein 3

Rediscovering splenectomy in the application of the adult idiopathic thrombocytopenic purpura

Traditionally, splenectomy is the second-line treatment method of primary thrombocytopenic purpura and a choice after the ineffectual treatment of glucocorticoid, but doctors never evaluate the curative effect of splenectomy before the operation and assess the necessity of splenectomy. In light of recent progresses on this topic, the current review summaries and pinpoints traditional application of splenectomy, side effects, new drug , and predicted factors of evaluation of operation, aiming to make accurate splenectomy for idiopathic thrombocytopenic purpura. Key words: Purpura, thrombocytopenic; Splenectomy; Forecasting; The new medicine treatment

Analysis of clinical effects and mechanism of recombinant human interleukin-11 with glucocorticoids for treatment of idiopathic thrombocytopenic purpura.

The aim of the present study was to evaluate the effectiveness and safety of recombinant human interleukin-11 (IL-11) with glucocorticoids for treatment of adult idiopathic thrombocytopenic purpura (ITP) and the regulatory effect on immune mechanisms. A total of 80 patients with initial diagnosis of ITP admitted to our hospital were selected. Patients were randomly divided into the control group and observation group, with 40 cases each. The control group received glucocorticoids treatment, and the observation group received IL-11 and glucocorticoids. The treatment effects were compared. The total effective rate and effective degree of the observation group was higher than in the control group and the difference was statistically significant (P<0.05); comparing the incidence of complications of the two groups, there was no statistical difference (P>0.05). In the observation group, onset time was reduced, platelet recovery level increased and platelet antibody positive rate decreased, and the differences were statistically significant (P<0.05). The total treatment course was shorter and recurrence rate was lower in the observation group compared with the control group, and the differences were statistically significant (P<0.05). The percentage of CD4+CD25+ regulatory T cells decreased in the two groups after treatment, and was more pronounced in the observation group. The difference was statistically significant (P<0.05). In conclusion, IL-11 with glucocorticoids for the treatment of adult ITP is safe and effective, and may be associated with decreased percentage of CD4+CD25+ regulatory T cells.

Identification of a potent small molecule capable of regulating polyploidization, megakaryocyte maturation, and platelet production.

BackgroundMegakaryocytic cell maturation involves polyploidization, and megakaryocyte (MK) ploidy correlates with their maturation and platelet production. Retardation of MK maturation is closely associated with poor MK engraftment after cord blood transplantation and neonatal thrombocytopenia. Despite the high prevalence of thrombocytopenia in a range of setting that affect infants to adults, there are still very limited modalities of treatment.MethodsHuman CD34+ cells were isolated from cord blood or bone marrow samples acquired from consenting patients. Cells were cultured and induced using 616452 and compared to current drugs on the market such as rominplostim or TPO. Ploidy analysis was completed using propidium iodide staining and flow cytometry analysis. Animal studies consisted of transplanting human CD34+ cells into NOD.Cg-PrkdcscidIl2rgtm1Wjl/SzJ mice followed by daily injections of 15 mg/kg of 616452.ResultsWithin one week of culture, the chemical was able to induce polyploidization, the process required for megakaryocyte maturation with the accumulation of DNA content, to 64 N or greater to achieve a relative adult size. We observed fold increases as high as 200-fold in cells of 16 N or greater compared to un-induced cells with a dose-dependent manner. In addition, MK differentiated in the presence of 616452 demonstrated a more robust capacity of MK differentiation than that of MKs cultured with rominplostim used for adult idiopathic thrombocytopenic purpura (ITP) patients. In mice transplanted with human cord blood, 616452 strikingly enhanced MK reconstitution in the marrow and human peripheral platelet production. The molecular therapeutic actions for this chemical may be through TPO-independent pathways.ConclusionOur studies may have an important impact on our fundamental understanding of fetal MK biology, the clinical management of thrombocytopenic neonates and leukemic differentiation therapy.Electronic supplementary materialThe online version of this article (doi:10.1186/s13045-016-0358-y) contains supplementary material, which is available to authorized users.

PSY34 - The Budget Impact Analysis of Thrombopoietin Receptor Agonists For The Treatment of Idiopathic Thrombocytopenic Purpura In Adults

PSY34 - The Budget Impact Analysis of Thrombopoietin Receptor Agonists For The Treatment of Idiopathic Thrombocytopenic Purpura In Adults

Cost-per-responder analysis comparing romiplostim to rituximab in the treatment of adult primary immune thrombocytopenia in Spain

Background and objectiveRomiplostim, a thrombopoietin-receptor agonist, is approved for second-line use in idiopathic thrombocytopenic purpura (ITP) patients where surgery is contraindicated. Anti-CD20 rituximab, an immunosuppressant, is currently used off-label. This analysis compared the cost per responder for romiplostim versus rituximab in Spain. Materials and methodA decision analytic model was constructed to estimate the 6-month cost per responding patient (achieving a platelet count ≥50×109/L) according to the most robust published data. A systematic literature review was performed to extract response rates from phase 3 randomised controlled trials. Romiplostim patients received weekly injections; rituximab patients received 4 weekly intravenous infusions. Medical resource costs were obtained from Spanish reimbursement lists. Treatment non-responders incurred bleeding-related event (BRE) management costs as reported in clinical trials. Medical resource utilisation and clinical practice were based on Spanish treatment guidelines and validated by local clinical experts. ResultsThe literature review identified phase 3 romiplostim trials with a response rate of 83%. Due to a lack of phase 3 controlled rituximab trials, a systematic review of studies was selected as the best source, reporting a response rate of 62.5%. The mean cost per patient for romiplostim was €16,289 and €13,459 for rituximab. Rituximab resulted in a 10% higher cost per responder (€21,535 versus €19,625 for romiplostim). Romiplostim use reduced drug administration, intravenous immunoglobulin, and bleeding-related costs compared to rituximab. ConclusionsDue to its high level of efficacy leading to lower BRE costs, romiplostim represents an efficient use of resources for adult ITP patients in the Spanish Healthcare System.

Cost-effectiveness of adding rituximab to splenectomy and romiplostim for treating steroid-resistant idiopathic thrombocytopenic purpura in adults.

BackgroundIdiopathic thrombocytopenic purpura (ITP) is an autoimmune disease in which the platelet count falls to <100 × 109/L. Corticosteroids are recommended as the first-line treatment, splenectomy is recommended as the second-line treatment, and thrombopoietin receptor agonists (TPO-RAs) and rituximab are recommended as the third-line treatments for ITP in Japanese ITP treatment guidelines. However, in Japan, rituximab is not eligible for reimbursement for the treatment of ITP. The cost-effectiveness of ITP treatment has not been investigated in Japan. Therefore, in this study, the cost-effectiveness of adding rituximab treatment to the existing treatments indicated for ITP in Japan, namely splenectomy and the TPO-RA romiplostim, was investigated based on the scenario that rituximab is eligible for reimbursement in Japan as a treatment for ITP.MethodsThe efficacy endpoint was set as the number of years with a platelet count ≥30 × 109/L. The analysis was conducted from the healthcare payer’s perspective. If the first treatment is ineffective or relapse occurs, then the patient is given the following treatment. The analyzed treatment order consisted of three patterns: splenectomy-romiplostim (sequence 1), splenectomy-romiplostim-rituximab (sequence 2), and splenectomy-rituximab-romiplostim (sequence 3). A Markov model was built for ITP, and the analysis period was set as 2 years. The discount rate was an annual rate of 2%.Sensitivity analyses of the efficacy of splenectomy, romiplostim, and rituximab; treatment cost; and romiplostim dose were performed.ResultsThe expected costs per patient over a 2-year period for sequences 1, 2, and 3 were USD 40,980, USD 39,822, and USD 33,551, respectively. The expected years with a platelet count ≥30 × 109/L for the three sequences were 1.75, 1.79, and 1.78 years, respectively. The sensitivity analyses illustrated that the results of the base case analysis were robust.ConclusionsAdding rituximab to standard treatment for ITP (sequences 2–3) is less costly and marginally more effective than standard therapy in adults. According to the study results, if rituximab is reimbursed for the treatment of ITP in Japan, medical expenses are expected to decline.

Late results after splenectomy in adult idiopathic thrombocytopenic purpura.

Background:We performed a retrospective study on patients with idiopathic thrombocytopenic purpura (ITP) to evaluate the response to splenectomy in relation to preoperative platelet count.Materials and Methods:Two groups of patients operated on with laparoscopic or open splenectomy for ITP, with a platelet count ≤30,000/μL (study group: 22 patients) and >30,000/μL (control group: 18 patients), respectively, were compared. The two groups were homogeneous in relation to age, sex, length of preoperative steroid therapy, and time interval between diagnosis and surgery (Student t test with P > .1). The results of surgery were evaluated at one year after splenectomy. Positive response to surgery, according to the American Society of Hematologic Guidelines, was considered in patients with a postoperative platelet count ≥100,000/μL or in patients with a postoperative platelet count ≥30,000/μL and a twofold increase in platelet count from baseline, in the absence of bleeding.The postoperative platelet count increase rate was statistically related to preoperative platelet count in both the study and control groups. Statistical analysis was performed using the Student's t test for independent sample and the Pearson correlation in a 2-tailed test.Results:No relationship between preoperative platelet count and postoperative platelet percent increase was observed in the control group (r = –0.41; P = .089), whereas a significant negative correlation (r = –0.68; P = .0004) was found in the study group.Conclusions:A higher increase of postoperative percent platelet count may be predicted in patients with a low preoperative platelet count.

Roles of peripheral B1 cells in the individualized treatment of adult idiopathic thrombocytopenic purpura.

We aimed to explore the changes of peripheral B1 cells before and after treatment of adult idiopathic thrombocytopenic purpura (ITP) and to investigate the association of these changes with the disease condition and prognosis. Ninety-seven ITP patients were divided into the effective or ineffective groups, based on their response to hormone therapy. Forty healthy volunteers were enrolled into the control group (HC). The percentages of CD19+ cells, B1 cells, and platelet-associated immunoglobulin (PAIg) in peripheral blood from healthy volunteers and ITP patients before and after treatment were evaluated, and blood platelet (PLT) counts were determined. The percentages of CD19+ cells [(21 ± 10.0) vs (11.2 ± 7.1)%], B1 cells [(8.85 ± 5.23) vs (2.2 ± 1.3)%], and PAIg [(28 ± 19) vs (11.7 ± 8)%] in whole blood from ITP patients before treatment were significantly higher than those in whole blood from healthy controls (P < 0.05). Before treatment, the percentage of B1 cells and PAIg in ITP patients was negatively correlated with the PLT level (r = -0.89, P < 0.05 and r = -0.814, P < 0.05, respectively). Further, the B1 cell percentage was positively associated with the PAIg percentage in ITP patients before treatment. In the effective group, the B1 cell percentage was reduced sharply at 1 month after treatment [(2.45 ± 1.75) vs (8.74 ± 5.04)%, P < 0.05)], so as at 3 and 6 months. However, in the ineffective group, there was no difference in the B1 cell percentage before and after treatment [(7.9 ± 5.6) vs (8.76 ± 5.26)%]. This obvious association of changes in peripheral B1 cells with disease condition and prognosis in ITP patients may be of certain clinical significance for guiding the individualized treatment of ITP.

Coste por paciente con respuesta a romiplostim y rituximab en el tratamiento de la trombocitopenia inmune primaria en España

Background and objectiveRomiplostim, a thrombopoietin-receptor agonist, is approved for second-line use in idiopathic thrombocytopenic purpura (ITP) patients where surgery is contraindicated. Anti-CD20 rituximab, an immunosuppressant, is currently used off-label. This analysis compared the cost per responder for romiplostim versus rituximab in Spain. Materials and methodA decision analytic model was constructed to estimate the 6-month cost per responding patient (achieving a platelet count≥50×109/l) according to the most robust published data. A systematic literature review was performed to extract response rates from phase 3 randomized controlled trials. Romiplostim patients received weekly injections; rituximab patients received 4 weekly intravenous infusions. Medical resource costs were obtained from Spanish reimbursement lists. Treatment non-responders incurred bleeding-related event (BRE) management costs as reported in clinical trials. Medical resource utilization and clinical practice were based on Spanish treatment guidelines and validated by local clinical experts. ResultsThe literature review identified phase 3 romiplostim trials with a response rate of 83%. Due to a lack of phase 3 controlled rituximab trials, a systematic review of studies was selected as the best source, reporting a response rate of 62.5%. The mean cost per patient for romiplostim was €16,289 and €13,459 for rituximab. Rituximab resulted in a 10% higher cost per responder (€21,535 versus €19,625 for romiplostim). Romiplostim use reduced drug administration, intravenous immunoglobulin, and bleeding-related costs compared to rituximab. ConclusionsDue to its high level of efficacy leading to lower BRE costs, romiplostim represents an efficient use of resources for adult ITP patients in the Spanish Healthcare System.

Association between Helicobacter pylori Infection and Idiopathic Thrombocytopenic Purpura in Adults

A Gram-negative spiral bacterium, Helicobacter pylori, has been known to be a causative agent of chronic gastritis and peptic ulcer disease and plays an important role in increasing the risk for the development of gastric cancer. In addition, the infection has also been implicated in the development of extragastric diseases, such as hematological, systemic, cardiovascular and neurological diseases. Accumulating evidence indicates that successful eradication of H. pylori results in an increase of the platelet count in approximately half of H. pylori-positive ITP patients. However, the efficacy of the eradication was quite different in ITP patients among different countries worldwide. In this review, we summarize the recent evidences with regard to the association between H. pylori infection and ITP. We also provide information about the possible pathogenetic mechanisms and the factors for predicting platelet recovery.

Quick Recertification Series

Quick Recertification Series

Long-term Relapse-free Rurvival Rate and Predictive Factors of Idiopathic Thrombocytopenic Purpura in Adults Undergoing Splenectomy

Our aim is to identify the long-term relapse-free rate and predictive factors of response to splenectomy in adults with idiopathic thrombocytopenic purpura (ITP). Between 1999 and 2005, 54 patients of ITP, who underwent splenectomy in Chang Gung Memorial Hospital, Linkou, Taiwan were retrospectively reviewed. Various pre, intra and postoperative factors were analyzed to determine the predictive factors of response and long-term disease-free rate after splenectomy in adult patients. The relapse free survival rates in complete response patients of splenectomy at 1-, 3- and 5-yr are 91.9%, 88.4%, and 88.4%, respectively. Younger age, response to steroids, pre- and postsplenectomy high platelet counts were found to be have significant p value of < 0.05 as predictive factors in univariate analysis. In multivariate analysis, only pre-op and post-op platelet counts were significant. Significant long-term relapse-free survival rate is achieved by splenectomy in adults with ITP. In univariate analysis, age, response to steroids, pre- and postsplenectomy platelet counts were the significant predictive factors of response. But in multivariate analysis, only pre-op and post-op platelet counts were significant.

Long-Term Results After Splenectomy in Adult Idiopathic Thrombocytopenic Purpura: Comparison Between Open and Laparoscopic Procedures

Only a few studies have addressed long-term results comparing laparoscopic and open splenectomy in idiopathic thrombocytopenic purpura (ITP). We analyzed the 1-year results comparing age, sex, length of preoperative steroid therapy, diagnosis-to-splenectomy interval, and preoperative platelet count in relation to postoperative response after open and laparoscopic splenectomy. Data collected from two groups, treated by laparoscopic and open splenectomy, respectively, of 20 patients each were retrospectively reviewed. Positive response to splenectomies, evaluated according to the International Working Group guidelines reported by the American Society of Hematology, was statistically related through Student's t test and the Pearson correlation test to the above-mentioned factors. Positive response to splenectomy was observed in 80% and 85% of patients, respectively, in the laparoscopic and open groups (P > .10). No statistical differences were observed comparing each of the studied factors between laparoscopic and open splenectomy responder patients (P > .10). When percentage increase of postoperative platelet count was related to diagnosis-to-splenectomy interval, a positive correlation was found in the laparoscopic group (r = 0.544, P < .05). In addition, a significant negative correlation in both groups was observed comparing preoperative platelet count and percentage postoperative platelet increase, with a greater increase of postoperative platelet count in patients with a lower preoperative platelet count (laparoscopic group, r = -0.663; open group, r = -0.656; P < .01). In this series long-term results after laparoscopic splenectomy in ITP patients were as effective as after the open approach. Higher postoperative platelet percentage increase was achieved in both groups in patients with a lower preoperative platelet count. Finally, laparoscopic splenectomy in this study seems to be superior to the open approach in patients with a longer diagnosis-to-splenectomy interval.

Management of Idiopathic Thrombocytopenic Purpura in Adult: A Review Article

Idiopathic thrombocytopenic purpiira (ITP), also known as primary immune or autoimmune thmmbocytopenic purpura, is a common cause of thrombocytopenia and bleeding complications in children and adults. It mav be confused with other causes of thrombocvtopenia and is treated with agents that varv in efjicacv, toxicity, and cost. Clinical presentation very in children and adult. In children, ITP is usually an acute, self-limited disorder that resolves spontaneously: in adults, it is typically a chronic disorder with a more insidious onset. In about one third of adults with ITP, the condition is persistent and relatively resistant to most treatments .Available evidence suggests that only about 5% of adults with chronic ITP have spontaneous remission. The principal therapeutic options for ITP include glucocorticoids, intravenous immunoglobulin and splenectomy. Other treatments have been used for refractory cases; these include intravenous anti-Rh (D), azathioprine, cyclophosphamide, danazol, vinca alkaloids, ascorbic acid, colchicine, interferon-alpha, combination chemotherapy, protein A, immunoadsorption, cyclosporine, epsilon-aminocaproic acid, plasma exchange, and accessory splenectomy. Journal of Shaheed Suhrawardy Medical College Vol 2No.1 June 2010 page 24-28 DOI: http://dx.doi.org/10.3329/jssmc.v2i1.12348

Helicobacter Pylori-Associated Idiopathic Thrombocytopenic Purpura: A Narrative Review

The Gram-negative bacterium Helicobacter pylori has a well-demonstrated role in several gastroduodenal diseases, including peptic ulcer disease, chronic active gastritis, mucosa-associated lymphoid tissue lymphoma, and gastric adenocarcinoma. In addition, more recently, several studies have focused on the possible causal role of H. pylori in various extragastric disorders, such as cardiovascular, respiratory, neurological, skin, and autoimmune conditions. The current status of the research on the pathogenesis, clinical and therapeutic aspects of H. pylori-associated idiopathic thrombocytopenic purpura in adults and children will be addressed in this narrative review.