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

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 12 ◽  
pp. 204062072110483
Author(s):  
Monica Carpenedo ◽  
Erminia Baldacci ◽  
Claudia Baratè ◽  
Alessandra Borchiellini ◽  
Francesco Buccisano ◽  
...  

Introduction: In patients with primary immune thrombocytopenia (ITP), a short course of steroids is routinely given as first-line therapy. However, the response is often transient and additional therapy is usually needed. Thrombopoietin receptor agonists (TPO-RAs) are frequently used as second-line therapy, although there is little clinical guidance on the timing of their administration and on tapering/discontinuation of the drug. To provide clinical recommendations, we used the Delphi technique to obtain consensus for statements regarding administration and on tapering/discontinuation of second-line TPO-RAs among a group of Italian clinicians with expertise in management of ITP. Methods: The Delphi process was used to obtain agreement on five statements regarding initiation and on tapering/discontinuation of second-line TPO-RAs. Agreement was considered when 75% of participants approved the statement. Eleven experts participated in the voting. Results: Full consensus was reached for three of the five statements. The experts held that an early switch from corticosteroids to a TPO-RA has the dual advantage of sparing patients from corticosteroid abuse and improve long-term clinical outcomes. All felt that dose reduction of TPO-RAs can be considered in patients with a stable response and platelet count >100 × 109/L that is maintained for at least 6 months in the absence of concomitant treatments, although there was less agreement in patients with a platelet count >50 × 109/L. Near consensus was reached regarding the statement that early treatment with a TPO-RA is associated with an increase in clinically significant partial or complete response. The experts also agreed that optimization of tapering and discontinuation of TPO-RA therapy in selected patients can improve the quality of life. Conclusion: The present consensus can help to provide guidance on use of TPO-RAs in daily practice in patients with ITP. Plain language summary Second-line administration of thrombopoietin receptor agonists in immune thrombocytopenia There is little guidance on the timing of administration and tapering/discontinuation of thrombopoietin receptor agonists (TPO-RAs) in patients with primary immune thrombocytopenia (ITP). The Delphi technique was used to obtain consensus for five statements. The present consensus among Italian clinicians aims to provide guidance on second-line use of TPO-RAs for patients with ITP in daily practice.


Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 3388-3388 ◽  
Author(s):  
Tatyana Semenovna Konstaninova ◽  
Ivanova Valentina Leonidovna ◽  
Andrzej Hellmann ◽  
Slawomira Kyrcz-Krzemien ◽  
Sascha Tillmanns ◽  
...  

Abstract Abstract 3388 Background: Primary Immune Thrombocytopenia (ITP) is an autoimmune disorder characterized by a low platelet count below 100 × 109/L and an exclusion of secondary causes such as bacterial infections or hematological malignancies. Fc-gamma-receptors (FcγRs) are involved in the clearance of immune complexes, the antibody-dependent cellular cytotoxicity, the secretion of mediators and indirectly the regulation of B cell proliferation and differentiation. The investigational product SM101 represents the non-glycosylated C-terminally engineered human soluble FcγRIIB. SM101 has been investigated successfully in autoimmune models, toxicology studies and safety clinical trials in healthy subjects and is currently being investigated in phase II clinical studies in patients with ITP and Systemic Lupus Erythematosus (SLE). Recently, the dose-escalation part of the phase Ib/IIa clinical trial in ITP was completed. Objectives: The primary objective was to evaluate the safety and tolerability of SM101 at increasing dose levels in patients with chronic ITP. Secondary objectives included efficacy in terms of platelet count and pharmacokinetic (PK) evaluation of SM101. Methods: The Ib part of the clinical trial was designed as a randomized, double-blind, placebo-controlled, multi-center dose escalation study in 36 ITP patients. Each dosing cohort consisted of 4 verum and 2 placebo subjects. Major inclusion criteria included a diagnosis of chronic ITP for at least 6 months and a platelet count of less than 30 × 109/L at baseline. Concomitant corticosteroids were allowed at constant doses during the study. Eligible patients were dosed on day 1 with a 5 week safety period, followed up by the intended treatment cycle of 4 weekly infusions between day 35 and 56 and a final follow-up of 3 months. An independent Data Safety Monitoring Board was in place to review safety data after each dose cohort. Results: In total, 36 ITP patients in 6 dose groups received 5 infusions of 0.3 to 12.0 mg/kg/week SM101or placebo. 56% of the patients were females, 39% of the patients were splenectomized and 39% received concomitant corticosteroids prior to randomization. In the placebo group, 42% received rescue medication compared to 0 to 25% in the higher dose groups with SM101. No dose-limiting toxicity (DLT) occurred and no serious adverse event (SAE) was observed for patients treated with SM101. SM101 plasma levels increased proportionally with dose and the mean t1/2 across all dose groups was 42.7 h with a range of 14.0 to 99.9 h. In terms of immunogenicity, no anti-drug-antibodies against SM101 were observed. For patients without ITP rescue therapy, the highest dose group with 12 mg/kg/week SM101 showed a sustained median platelet response over 50 × 109/L in comparison to placebo after one treatment cycle with 4 infusions (Figure 1). This response persisted during the 3 months follow-up period with a median platelet count of approximately 70 × 109/L at the end of the clinical trial. The platelet response in the 12 mg/kg/week group started approximately 20 days after the beginning of the treatment cycle. Conclusions: - To the author's knowledge, this is the first time that a soluble Fcγ receptor showed efficacy in a clinical trial - In the 12 mg/kg/week group, SM101 showed a clear platelet response in patients with ITP - The duration of the platelet response persisted for at least 3 months after the last administration of SM101 - Multiple doses of SM101 up to 12 mg/kg/week were well tolerated with no DLT or SAE - Further clinical trials with SM101 in ITP to support the current findings are ongoing. Disclosures: Tillmanns: SuppreMol GmbH: Employment. Sondermann:SuppreMol GmbH: Employment. Buckel:SuppreMol GmbH: Employment.


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.


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 ◽  
...  

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