scholarly journals Primary immune thrombocytopenia in US clinical practice: incidence and healthcare burden in first 12 months following diagnosis

2019 ◽  
Vol 23 (2) ◽  
pp. 184-192 ◽  
Author(s):  
Derek Weycker ◽  
Ahuva Hanau ◽  
Mark Hatfield ◽  
Hongsheng Wu ◽  
Anjali Sharma ◽  
...  
2016 ◽  
Vol 98 (2) ◽  
pp. 112-120 ◽  
Author(s):  
Michael Steurer ◽  
Philippe Quittet ◽  
Helen A. Papadaki ◽  
Dominik Selleslag ◽  
Jean-François Viallard ◽  
...  

2017 ◽  
Vol 2017 ◽  
pp. 1-8 ◽  
Author(s):  
María-Eva Mingot-Castellano ◽  
Carlos Grande-García ◽  
David Valcárcel-Ferreiras ◽  
Clara Conill-Cortés ◽  
Loreto de Olivar-Oliver

Romiplostim, a thrombopoietin-receptor agonist (TPO-ra), is a highly effective option in primary immune thrombocytopenia (ITP), with 80–90% of patients achieving platelet responses after few weeks of treatment. The evidence showing remissions, that is, sustained platelet counts after romiplostim discontinuation, in patients with ITP refractory to immunosuppressive therapy is steadily increasing. However, there is a lack of guidelines or recommendations addressing how and when to taper romiplostim in clinical practice in patients maintaining elevated and stable platelet counts. Furthermore, given the high heterogeneity of ITP patients, no associated predictive factors have been currently identified. Here, we present 4 representative clinical cases of the daily clinical practice in Spain comprising newly diagnosed, persistent, and both splenectomized and nonsplenectomized chronic ITP patients treated with romiplostim, achieving and maintaining clinical remission (platelet count ≥ 50×109/L for 24 consecutive weeks in the absence of any treatment for ITP) after treatment tapering and discontinuation, without observed safety concerns. Prospective studies identifying clinical and biological predictive factors of sustained response are warranted.


2021 ◽  
Author(s):  
Marcel Reiser ◽  
Klaus M. Josten ◽  
Hermann Dietzfelbinger ◽  
Anouchka Seesaghur ◽  
Markus Schill ◽  
...  

Introduction: The effectiveness and safety of romiplostim were evaluated by immune thrombocytopenia (ITP) phase (newly diagnosed/persistent/chronic) at romiplostim initiation. Methods: Post hoc analysis of a prospective, German, multicentre, observational study in adults with ITP who received ≥1 dose of romiplostim. Follow-up data were collected for ≤2 years. Outcomes included overall platelet response (≥1 platelet count ≥50 × 109/L at 2–24 weeks after romiplostim initiation) or durable platelet response (≥75% of measurements ≥50 x 109/L at 14–24 weeks), and adverse drug reactions (ADRs), evaluated by ITP phase. Results: Data from 96 patients were analysed (newly diagnosed, n=18; persistent, n=25; chronic, n=53). During the 2–24-week follow-up, overall platelet response was achieved in 100% (95% confidence interval [CI]: 81.5–100), 100% (86.3–100), and 96.2% (87.0–99.5) of patients with newly diagnosed, persistent, or chronic ITP, respectively; platelet responses were durable in 88.2% (63.6–98.5), 65.0% (40.8–84.6), and 69.4% (54.6–81.7) of patients. During the 2-year follow-up, ADRs occurred in 24.0–35.8% of patients across phases. Two patients with chronic ITP experienced bone marrow ADRs; no thrombotic ADRs occurred. Conclusion: Romiplostim was effective and well tolerated in patients with newly diagnosed, persistent, or chronic ITP in routine clinical practice.


2019 ◽  
Vol 2019 ◽  
pp. 1-11
Author(s):  
M. Eva Mingot-Castellano ◽  
M. Teresa Álvarez Román ◽  
Luis Fernando Fernández Fuertes ◽  
Tomás José González-López ◽  
José María Guinea de Castro ◽  
...  

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.


Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 1094-1094
Author(s):  
Kaelyn H. Lu ◽  
James N. George ◽  
Sara K. Vesely ◽  
Deirdra R. Terrell

Abstract Abstract 1094 Introduction With 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. Methods Separate 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. Results All 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). Discussion Although 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. Conclusion These 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.


2021 ◽  
Vol 69 (3) ◽  
Author(s):  
Edgar Vladimir Cabrera-Bernal ◽  
Ana Marcela Torres-Amaya ◽  
María Teresa Vallejo-Ortega ◽  
Adriana LInares-Ballesteros ◽  
Isabel Cristina Sarmiento-Urbina ◽  
...  

Introduction: Primary immune thrombocytopenia (ITP) is the most frequent cause of thrombocytopenia in pediatric population, with a reported incidence of 1.1-12.5 cases per 100 000 children. However, currently there are different definitions of ITP, as well as diagnostic and therapeutic approaches. Objective: To develop an evidence-based clinical practice guideline (CPG) to unify ITP definitions, and this way, reduce the variability of its diagnosis, and to provide indications for the treatment of acute, persistent, and chronic ITP in patients under 18 years of age Materials and methods: The CPG was developed by a multidisciplinary group that followed the standard methods of the Grading of Recommendations, Assessment, Development and Evaluations (GRADE) guidelines for the development of CPGs, formulated PICO clinical questions, and conducted systematic reviews. GRADE evidence profiles were created and recommendations, with their respective level of evidence and strength, were made after a panel of experts assessed the benefit-risk balance, the quality of evidence, the patients’ values and preferences and the context in which they should be implemented. Results: A total of 23 recommendations for the treatment of acute, persistent and chronic ITP by pediatricians, hematologist and health professionals working in emergency services were made. Overall, the evidence of the GPC is of low quality and the recommendations were formulated in order to improve the treatment success rate of ITP and the prognosis of children with this condition. Conclusions: Despite ITP is the main cause of thrombocytopenia in pediatric population, so far there is not enough high quality evidence supporting the recommendations presented here for its proper classification and treatment in children. Thus, further studies providing high quality evidence on this issue are required.


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