Management Of Primary Immune Thrombocytopenia Research Articles

Immune Thrombocytopenia in Very Elderly Patients: Particularities in Presentation and Management. Results from the Multicenter Prospective Carmen-France Registry

Immune Thrombocytopenia in Very Elderly Patients: Particularities in Presentation and Management. Results from the Multicenter Prospective Carmen-France Registry

Management of Adult Patients with Primary Immune Thrombocytopenia (ITP) in Clinical Practice: A Consensus Approach of the Spanish ITP Expert Group.

Background and Objective Diagnosis and management of primary immune thrombocytopenia (ITP) have changed dramatically in the last decade. The aim of the study was to obtain information about the opinion of the Spanish ITP Group (GEPTI) members regarding the best clinical practices for diagnosis and management of adult patients with ITP. Materials and Methods A two-round Delphi method was carried out by sending to 129 experts a 90-item questionnaire developed by 11 specialists, with a 4-point Likert scale (“never,” “sometimes,” “frequently,” and “always”) for the assessment of responses. Results Forty out of the 129 experts participated in the survey (participation rate 30.2%) and 39 completed the questionnaire (response rate 97.5%). Salient consensus points included the following: the need to indicate workup studies from a sustained platelet count < 100 x 109/L in the absence of a clear etiology; bone marrow aspiration in elderly patients with suspected ITP; beginning treatment in asymptomatic patients with a platelet count < 20 x 109/L; not exceeding 6-7 weeks of corticosteroid therapy; switching from corticosteroids to one thrombopoietin receptor agonist (TRA); switching to other TRA or other options as combinations of them with immunosuppressive drugs in case of failure; how to reduce tapering TRA; treating patients with symptomatic persistent ITP and platelet count > 20 x 109/L; and considering mucosal or severe bleeding as a basic criterion for hospital admission. Conclusions The present consensus document provides a reference framework for the management of patients with ITP in clinical practice.

Diagnosis and management of primary immune thrombocytopenia in adults.

Diagnosis and management of primary immune thrombocytopenia in adults.

Progress on management of primary immune thrombocytopenia in pregnancy

Primary immune thrombocytopenia (ITP) in pregnancy is a special type of ITP, its impact on the mother and fetus cannot be ignored. The correct diagnosis and effective treatment of ITP in pregnancy are the focus of the clinical practice and medical research. This article reviews the progress on the management of the primary immune thrombocytopenia in pregnancy. Key words: Pregnancy; ITP; Diagnosis; Treatment

Treatment characteristics, efficacy and safety of thrombopoietin analogues in routine management of primary immune thrombocytopenia.

: Thrombopoietin receptor agonist (TPO-RAs) have demonstrated good efficacy and tolerance in clinical trials in refractory chronic primary immune thrombocytopenia (ITP) or chronic ITP with contraindication for splenectomy. No head-to-head study is available, and differences in trials design do not allow comparisons. Information on the use of TPO-RAs in nonchronic ITP is scant. We described our experience with TPO-RAs in ITP (chronic, persistent and newly diagnosed ITP) in routine clinical practice. Retrospective series of 100 adult ITP patients was analysed; 41 treated with eltrombopag, 37 with romiplostim and 22 with both. Response-related and safety variables were evaluated. With a median follow-up of 86.5 weeks (interquartile range, 34.3-128 weeks), no differences were found in response rate, time to response, stability of response or response duration based on the type of TPO-RA used. Of all, 25% of patients with newly diagnosed or persistent ITP and 7.2% with chronic responded and maintained their response when TPO-RAs were stopped. Regarding safety, two developed bone marrow fibrosis grade 3, with loss of response to both drugs. Incidence of vascular events was 7%. Both TPO-RAs may be useful in all types of ITP, not only chronic but also persistent and newly diagnosed. Similar results were noted in efficacy and safety variables for both drugs.

Epidemiology and management of primary immune thrombocytopenia: A nationwide population-based study in Korea

IntroductionThe epidemiology of immune thrombocytopenia (ITP) is not well characterized in an Asian population. Materials and methodsFrom July 2010 to June 2014, ITP patients were identified using the Korean Health Insurance Review and Assessment Service database. ResultsThe overall incidence rate of ITP was 5.3 per 100,000 person-years (95% CI: 5.1–5.5). The overall incidence rate ratios of children under 15years old to adults and females to males were 3.8 (95% CI: 3.7–3.9) and 1.3 (95% CI: 1.2–1.4), respectively. Of the total 10,814 patients, 3388 patients (31%) needed treatment for ITP; of these, 54% continued treatment for more than three months. First-line therapy consisted of corticosteroids (CS) in 42%, immunoglobulin (IVIg) in 35%, CS with IVIg in 19%, and other immunosuppressive agents (ISA) in 4%. Among treated patients, 75% of adults and 33% of children continued treatment for more than three months. After three months, the most frequently used drug was CS alone in 63% of patients. Only 104 patients underwent splenectomy; of these, 51% received salvage treatment after a median of one month after surgery (range: 0–27). The proportion of patients who received platelet transfusions of 12units or more per month for at least two consecutive months was significantly higher among patients treated for more than three months compared with patients who completed treatment within three months. ConclusionsThis population-based study is the first to describe the incidence of ITP and its treatment reality for patients in Korea.

PSY13 - A Retrospective Study on The Management of Primary Immune Thrombocytopenia In Adult Patient In Hong Kong

PSY13 - A Retrospective Study on The Management of Primary Immune Thrombocytopenia In Adult Patient In Hong Kong

High-dose dexamethasone or oral prednisone for immune thrombocytopenia?

Immune thrombocytopenia is an acquired bleeding disorder characterised by antibody-mediated destruction of platelets and impaired thrombopoiesis. Patients who are severely affected show haemorrhagic signs due to thrombocytopenia. In most guidelines and recommendations, a short course of oral prednisone is considered first-line treatment (ie, 0·5–2·0 mg/kg per day for 2–3 weeks, then tapered over a few weeks). 1 Provan D Stasi R Newland AC et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010; 115: 168-186 Crossref PubMed Scopus (1449) Google Scholar , 2 Neunert C Lim W Crowther M Cohen A Solberg Jr, L Crowther MA American Society of HematologyThe American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood. 2011; 117: 4190-4207 Crossref PubMed Scopus (1342) Google Scholar All guidelines emphasise avoiding long-term corticosteroids because of side-effects. High-dose dexamethasone compared with prednisone for previously untreated primary immune thrombocytopenia: a systematic review and meta-analysisIn adults with previously untreated immune thrombocytopenia, high-dose dexamethasone did not improve durable platelet count responses compared with standard-dose prednisone. High-dose dexamethasone might be preferred over prednisone for patients with severe immune thrombocytopenia who require a rapid rise in platelet count. Full-Text PDF

Real-life management of primary immune thrombocytopenia (ITP) in adult patients and adherence to practice guidelines.

Very few data exist on the management of adult patients diagnosed with primary immune thrombocytopenia (ITP). The objectives of this study were to describe the diagnostic and treatment patterns for ITP and to compare the findings to recent ITP guidelines. We retrospectively analyzed the medical records of adult ITP patients diagnosed with primary ITP between January 2011 and June 2012 and examined whether management strategies were consistent or not with eight recent guideline-recommended practices. Overall, median age at the diagnosis of the disease (n = 101) was 58years and median platelet count 12 × 10(9)/L with 75.2% of patients having symptoms of ITP. The study perceived two major shortcomings in the diagnostic approach: (1) failure to perform peripheral blood film examination in 22.8% of patients, a test that is mandatory by all guidelines, and (2) ordinary bone marrow assessment in more than half of the patients at diagnosis (50.5%), a test not routinely recommended by guidelines. Low appropriateness in therapeutic management of patients included (1) unjustified use of intravenous immunoglobulin in the absence of bleeding in 54.8% of patients and (2) splenectomy not being deferred until 6-12months from diagnosis (median 161days). Data also reflect a trend towards the early use of thrombopoietin receptor agonists in the treatment of patients who are refractory to any first-line therapy. We have recognized important areas of inapropriateness in the diagnostic and therapeutic management of adult ITP patients. Compliance with established guidelines should be encouraged in order to improve patient outcomes.

Current Management of Primary Immune Thrombocytopenia.

Primary immune thrombocytopenia is an autoimmune disorder of unknown cause affecting both children and adults. The low peripheral blood platelet count is caused by premature platelet destruction by self-reacting antibodies in addition to an impairment of platelet production. The disease is heterogeneous in its pathophysiology, clinical features and responses to treatment. To date, most of the treatments used have been immune-modulating drugs and these contribute to increased morbidity and mortality in patients. A new class of drugs, the thrombopoietin receptor agonists, has been developed for use in ITP. These have gone through randomised controlled trials in large numbers of patients with ITP. These drugs have high efficacy and are well tolerated. In addition, around 30% of patients receiving these drugs are able to stop them and maintain a safe or normal platelet count. Older treatments such as splenectomy are being used less than before, largely because of the introduction of the thrombopoietin receptor agonists. Currently there are trials underway evaluating novel therapies for ITP that will become available over the next few years once the trials are complete.

Diagnosis and management of primary immune thrombocytopenia in pregnancy

Diagnosis and management of primary immune thrombocytopenia in pregnancy

Management of Primary Immune Thrombocytopenia, 2012: A Survey of Oklahoma Hematologists-Oncologists

BackgroundManagement options for patients with primary immune thrombocytopenia (ITP) have increased, and treatment of patients with ITP has changed during the past 10 years. MethodsTo document current practice and to determine how current practice is related to recommendations of 2 recent practice guidelines for ITP, an international Consensus report and an American society of Hematology (ASH) guideline, the authors surveyed practicing hematologistsoncologists in Oklahoma. Surveys were specific for children or adults. Each survey had 3 questions describing patients with a new diagnosis and patients who had not achieved remission with initial treatment. Questions were adapted from the clinical scenarios of the ASH guideline. ResultsTwelve (92%) Oklahoma pediatric hematologistsoncologists responded; 82 (81%) Oklahoma adult hematologistsoncologists responded. For a child with a new diagnosis of ITP, a platelet count of 8000/μL and minor bleeding, 5 (42%) hematologistsoncologists selected observation without drug treatment (recommended by both guidelines). For an adult with a platelet count of 9000/μL who had failed to respond to initial treatment with corticosteroids and IVIg, 32 (39%) selected splenectomy (recommended by the ASH guideline); 30 (37%) selected rituximab and 13 (16%) selected thrombopoietin-receptor agonists (both recommended by the International Consensus report). Hematologists-oncologists who had more years in practice were more likely to select splenectomy (P = 0.047). ConclusionsIn a time of changing management for patients with ITP, these data document reported current management in Oklahoma and provide a basis for serial comparisons across time and for comparisons with other regions and comparison of management with patient outcomes.

Complications de la splénectomie au cours du purpura thrombopénique immunologique. Revue de la littérature et mesures de prévention

Management of primary immune thrombocytopenia (ITP) has changed, and clinical practice broadens the use of thrombopoietin receptor agonists and anti-CD20 antibody as options for second-line therapy, as alternative to splenectomy. Splenectomy remains a successful, definitive curative treatment. The purpose of this review about the complications of the splenectomy, in the context of ITP, is to increase the awareness of clinicians towards the preventive measures, which are often not correctly applied.

Recombinant Human Thrombopoietin and Rituximab vs Rituximab Monotherapy in Corticosteroid-Resistant Primary Immune Thrombocytopenia: a Multicenter Randomized Controlled Study

BackgroundThough great progresses had been achieved in the management of primary immune thrombocytopenia (ITP), still 25%-30% of the patients show no response to the conventional therapy. Both thrombopoietin (TPO) and rituximab (RTX) are recommended as the second-line treatments. TPO has shown potent activity in ITP, but the therapeutic efficacy is dependent on continual administration. Rituximab is an anti-CD20 chimeric monoclonal antibody which could induce prolonged remission in ITP. However, longer time to response was also reported. The combination of rhTPO and rituximab could complement each other in both mechanism of action and time window, exert powerful effect, which may be a choice for the glucocorticosteroids-resistant/relapsed patients. MethodPatients with platelet counts ≤ 30 x109/L, or ≤ 50 x109/L with bleeding symptoms from 12 centers were enrolled in the study. Subjects enrolled in the study were assigned randomly as 1: 2 to RTX group and rhTPO plus RTX group. Eligibility criteria were set according to the recently published guideline (Rodeghiero F, et al, Blood 2009). Approval for the study was obtained from the Ethics Committee of the School of Medicine and Qilu Hospital. Informed consent in accordance with the Declaration of Helsinki was given to each patient.Rituximab (MabThera; Roche) was given with a fixed dose of 100mg weekly for four weeks. And in the combination group, rhTPO (Tebiao, 3SBio, China) was added in the regimen with a dose of 300U/kg/day in the first 14 days, and a modified frequency of rhTPO was given according to the platelet counts after two weeks, but no longer than four weeks.Primary outcomes include response (R), complete response (CR), no response (NR) and relapse. All the criteria were consent with the definition and outcome criteria. (Rodeghiero F, et al. Blood, 2009.) Secondary outcomes are listed below. Time to response (TTR) was considered as the duration from baseline to response. Time to relapse was also measured with Kaplan-Meier analysis. Besides, safety was reflected as adverse events graded according to the Common Toxicity Criteria. This clinical trial was registered at http://clinicaltrials.gov as NCT 01525836. ResultA total of 114 patients were recruited: monotherapy group (n = 35) and combination group (n = 79). All patients failed to response to glucocorticosteroids or relapsed after corticosteroids treatment. Response rate was reached in 71.4% (25/35) of patients in monotherapy group vs. 78.5% (62/79) in the RTX and rhTPO combination group, while complete responses were achieved as 10/35 (28.6%) in RTX monotherapy group and 34/79 (43.0%) in rhTPO plus RTX group respectively. Median time to response was 28 days (range 4-90 days) in RTX group, while in RhTPO plus RTX group, TTR was 7 days (range 4-28 days) (P = 0.002). There was significantly longer time to relapse in the combination group (shown in Fig. 1).Table 1Characteristics and result of RTX group and rhTPO plus RTXparameterRTX (n = 35)rhTPO + RTX(n = 79)P value fordifferencebetweengroupsAge, median (range)46(14-68)42(13-82).664Female / Male (n)23/1251/28.905Baseline platelet count, median(range)13(3-36)8(0-32).221OR, %71.4%(25/35)78.5%(62/79).414CR, %0.2860.43.143NR, %28.6%21.5%.414TTR, day(s)28(4-90)7(4-28).002* [Display omitted] Adverse effects were observed in RTX group in 5 patients, most of whom were affected with inflammation. One patient was reported as viral myocarditis, and there were also complaints about chill, rash and hyperpyrexia. And incidence of adverse events was slightly increased in rhTPO plus RTX group. There were 14 adverse effects reported in the combination group, such as fatigue, pulmonary inflammatory, but all were below Grade 2. ConclusionOur findings suggest that combination of RTX and RTX in ITP yields shorter time to response compared with monotherapy of RTX, and moreover, combination extended time to relapse. Thus, combination therapy may represent an effective treatment option for glucocorticosteroids-resistant or relapsed patients.The results were compared with Gehan-Breslow-Wilcoxon Test. (P = 0.0140) Disclosures:No relevant conflicts of interest to declare.

Pathophysiology and management of primary immune thrombocytopenia

Primary immune thrombocytopenia, or idiopathic thrombocytopenic purpura (ITP), is an autoimmune disorder characterized by isolated thrombocytopenia due to accelerated platelet destruction and impaired platelet production. Autoantibodies against platelet surface glycoproteins, such as GPIIb/IIIa and GPIb/IX complexes, play major roles in both platelet destruction and impaired platelet production, although autoantibody-independent mechanisms, such as T cell-mediated cytotoxicity, may also be involved in its pathogenesis. Recent advances in the localization of autoantigenic epitopes and the characterization of T cell functional abnormalities in ITP patients have improved our understanding of the pathophysiology of this disease. Although corticosteroids and splenectomy remain central to the treatment of ITP, a new class of drugs, i.e., thrombopoietin receptor agonists (TPO-RAs) and rituximab, have substantially broadened the therapeutic options for refractory ITP patients. Moreover, the success of TPO-RAs in ITP patients shows that reduced platelet production caused by impaired megakaryocytopoiesis plays a greater role in ITP than previously recognized.

Current management of primary immune thrombocytopenia in Japan

Current management of primary immune thrombocytopenia in Japan

Management of Primary Immune Thrombocytopenia, 2012: A Survey of Oklahoma Hematologists-Oncologists

Abstract 1094 IntroductionWith the availability of multiple new agents, management of patients with primary immune thrombocytopenia (ITP) has changed substantially since the initial practice guideline for ITP was developed by the American Society of Hematology (ASH). To document current practice and to determine how current practice is related to recommendations of 2 recent guidelines for ITP, an International Consensus report (ICR), 2010, and an updated ASH guideline, 2011, we surveyed practicing hematologists-oncologists in Oklahoma. MethodsSeparate surveys were developed for children and adults. Each survey had 3 clinical scenarios; each scenario had 5–7 management choices. Hematologists-oncologists were instructed to select 1 management choice. Questions were adapted from the clinical scenarios of the ASH guideline. Additionally, data were collected for [1] number of years in practice; [2] site of practice, (either University of Oklahoma Medical Center or community); and [3] the estimated number of patients with ITP seen each year. Oklahoma hematologists-oncologists were identified by searching the Oklahoma Board of Medical Licensure and the Oklahoma Osteopathic Association websites for all active physicians certified in adult/pediatric hematology/oncology as of 12/31/2011. Exclusion criteria were: physicians whose careers were devoted to full-time research/administration. Data was collected between 1/31/2012–3/15/2012. Only the stronger grades of recommendations by the ICR and ASH guidelines were used for comparison to the hematologists-oncologists’ responses. Comparison of treatment responses to the number of years in practice was selected a priori, because it was hypothesized that hematologists-oncologists with fewer years in practice may more readily accept newer treatments while hematologists-oncologists with more years in practice may prefer more traditional management. Chi-square or Fisher’s exact test were used to determine if treatment responses differed by number of years in practice. SAS version 9.2 was used; alpha was set at 0.05. ResultsAll 13 (100%) Oklahoma pediatric hematologists-oncologists responded. For a child with a new diagnosis of ITP, a platelet count of 8,000/μL, and minor bleeding, 5 (38%) pediatric hematologists-oncologists selected observation without drug treatment (recommended by both guidelines). Because of the small number of pediatric hematologists-oncologists, no comparison of their responses to years in practice was performed. Eighty-three (82%) adult hematologists-oncologists responded. For an adult with a platelet count of 9,000/μL who had failed to respond to initial treatment with corticosteroids and IVIg, 32 (39%) selected splenectomy (recommended by ASH guideline); 31 (37%) selected rituximab and 13 (16%) selected thrombopoietin (TPO)-receptor agonists (both recommended by ICR). There was not a statistically significant difference between the number of years in practice for those who selected splenectomy versus all other treatment options (p = 0.07). DiscussionAlthough both recent guidelines recommended that children with no/mild bleeding may be managed with observation regardless of the platelet count, only 5 (38%) pediatric hematologists-oncologists selected observation in this scenario. The practice guidelines have different recommendations for second-line treatment in adults: the ICR recommends TPO-receptor agonists and rituximab but not splenectomy; the ASH guideline recommends splenectomy but not TPO-receptor agonists or rituximab. For this clinical scenario, 39% of hematologists-oncologists selected splenectomy, 37% selected rituximab and 16% selected TPO-agents. These different choices and recommendations reflect the changing clinical practice. Although not statistically significant, the hematologists-oncologists who had been in practice for >20 years appeared to be more likely to select splenectomy (p=0.07), consistent with greater acceptance of newer treatments by younger hematologists-oncologists. Although these data may not be generalizable, the high survey response rate provides confidence that these data accurately reflect the judgment, if not the actual practice, of Oklahoma hematologists-oncologists. ConclusionThese observations provide an estimate of actual current clinical practice for management of patients with ITP. Disclosures:Off Label Use: Rituximab for primary immune thrombocytopenia (ITP). George:Alexion, Inc.: Consultancy; Baxter, Inc.: Consultancy; Amgen, Inc.: Consultancy, PI for clinical trial involving romiplostim, PI for clinical trial involving romiplostim Other, Research Funding. Terrell:Amgen, Inc.: Consultancy; Baxter, Inc.: Consultancy.