scholarly journals A large observational study of patients with primary immune thrombocytopenia receiving romiplostim in European clinical practice

2016 ◽  
Vol 98 (2) ◽  
pp. 112-120 ◽  
Author(s):  
Michael Steurer ◽  
Philippe Quittet ◽  
Helen A. Papadaki ◽  
Dominik Selleslag ◽  
Jean-François Viallard ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Observational Study ◽  
Immune Thrombocytopenia ◽  
Primary Immune Thrombocytopenia ◽  
Large Observational Study

Romiplostim for Primary Immune Thrombocytopenia (ITP) in Routine Clinical Practice: Results from a Multicentre Observational Study in Germany

2021 ◽  
Author(s):  
Marcel Reiser ◽  
Klaus M. Josten ◽  
Hermann Dietzfelbinger ◽  
Anouchka Seesaghur ◽  
Markus Schill ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Observational Study ◽  
Immune Thrombocytopenia ◽  
Routine Clinical Practice ◽  
Newly Diagnosed ◽  
Platelet Response ◽  
Chronic Itp ◽  
Primary Immune Thrombocytopenia ◽  
Post Hoc

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.

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 ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Immune Thrombocytopenia ◽  
Healthcare Burden ◽  
Primary Immune Thrombocytopenia

scholarly journals Sustained Remission in Patients with Primary Immune Thrombocytopenia after Romiplostim Tapering and Discontinuation: A Case Series in Real Life Management in Spain

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
Keyword(s):  
Clinical Practice ◽  
Predictive Factors ◽  
Immune Thrombocytopenia ◽  
Real Life ◽  
Case Series ◽  
Sustained Remission ◽  
Platelet Counts ◽  
Thrombopoietin Receptor ◽  
Primary Immune Thrombocytopenia ◽  
Effective Option

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.

Romiplostim for the Treatment of Adults with Primary Immune Thrombocytopenia (ITP) in Routine Clinical Practice in France – Interim Results From a Large Observational Study

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4643-4643
Author(s):  
Philippe Quittet ◽  
Mohamed Hamidou ◽  
Bernard Bonnotte ◽  
Olivier Fain ◽  
Kerry Dillingham ◽  
...  
Keyword(s):  
Platelet Count ◽  
Clinical Practice ◽  
Observational Study ◽  
Clinical Utility ◽  
Employment Equity ◽  
Large Observational Study ◽  
Equity Ownership ◽  
Grade 3 ◽  
Observation Period

Abstract Abstract 4643 Background: The thrombopoietin-receptor agonist (TPOra) romiplostim is recommended for second-line treatment of adult ITP (Provan, Blood 2010). Romiplostim registration studies were conducted in selected patient (pt) populations and may not reflect clinical practice. Moreover, regional variations may exist in the clinical utility of romiplostim. A recent French study enrolled adult ITP pts failing previous treatments and receiving romiplostim on a compassionate use basis before commercial availability (Khellaf, Blood 2011). We report interim data from French pts enrolled in a large observational study of romiplostim use in clinical practice, with broader inclusion criteria. Methods: This ongoing study enrolls ITP pts ≥18 years old, who have received romiplostim in clinical practice. Pts participating in another study, who initiated romiplostim prior to commercial launch or received other TPOra or related products are excluded. Data recorded as per clinical practice is collected for up to 2 years following romiplostim initiation. Study outcomes include pt characteristics (at romiplostim initiation), romiplostim dose, adverse drug reactions (ADRs) and bleeds, summarized for pts meeting the eligibility criteria (Full Analysis Set; FAS). Amgen (Europe) GmbH funded the study and medical writing assistance. Results: As of February 2012, 86 (95%) of 91 pts enrolled in France were included in the FAS. Of these, 59% (51/86) remained on study, 31% (27/86) had completed the 2-year observation period and 9% (8/86) had withdrawn (death, 6 [7%]; lost to follow-up, 2 [2%]). At romiplostim initiation, median (Q1, Q3) age, weight and platelet count were 65.0 (48.0, 77.0) years, 74.00 (62.20, 84.00) kg and 18.0 (7.0, 31.0) × 109/L; 38% (33/86) of pts had been diagnosed with ITP for < 1 year (median [Q1, Q3] time from diagnosis, 3.04 [0.51, 13.04] years), 74% (64/86) had received ≥3 prior ITP therapies, 71% (61/86) had received prior rituximab, 30% (26/86) were splenectomised and 52% (45/86) female. Romiplostim was initiated at 1 and ≥3 μg/kg/week in 63% (54/86) and 24% (21/86) of pts; 36% (31/86) of pts stopped romiplostim before the end of the 2-year observation period, with requirement for alternative therapy (11 [13%]), haemostatic platelet count/no further treatment necessary (6 [7%]; last platelet count before romiplostim stopped, 68–616 × 109/L; 1 pt splenectomised, 4 had ITP disease duration of <1 year, all had received 3–5 prior therapies, 1 later received corticosteroids) and ADR (4 [5%]) the most commonly specified reasons. Median (Q1, Q3) romiplostim exposure was 55.1 (29.9, 100.3) weeks (maximum 106 weeks); median follow-up after romiplostim initiation was 20.40 (14.50, 24.10) months. Median (Q1, Q3) average weekly dose over the observation period was 2.6 (1.4, 4.2) μg/kg/week, and 3.02 [2.00, 5.00] and 2.00 [1.14, 3.02] μg/kg/week after 1 and 2 years of treatment (months 10–12 [n=48] and 22–24 [n=23], respectively). Median platelet counts rose rapidly during the first 4 weeks of treatment and remained >50 × 109/L thereafter (Figure). Grade ≥3 bleeds were rare and occurred at a lower rate after romiplostim initiation (Table). The most commonly reported ADRs were thrombocytosis, asthenia, myalgia and headache (9.7–3.2 events per 100 pt-years). Three pts reported a total of 6 serious ADRs: pulmonary embolism, 2 events; deep vein thrombosis, drug ineffective, myelofibrosis (initial disease diagnosis inconsistent with ITP, myelofibrosis more likely due to MDS), and thrombocytosis (platelets 477 × 109/L), 1 event each. No fatal ADRs were reported. Summary/conclusions: This study provides further insight into the clinical utility of romiplostim in France. At an interim analysis, pts tended to have less chronic disease than those enrolled in a previous observational study (Khellaf, Blood 2011). With lower doses than reported by Khellaf, and no new safety signals, pts achieved sustained increases in platelet counts and experienced a lower rate of grade ≥3 bleeds following romiplostim initiation. Disclosures: Dillingham: Amgen: Employment, Equity Ownership. Kreuzbauer:Amgen: Employment, Equity Ownership.

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

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 ◽  
...  
Keyword(s):  
Platelet Count ◽  
Clinical Practice ◽  
Immune Thrombocytopenia ◽  
Adult Patients ◽  
Severe Bleeding ◽  
Clinical Practices ◽  
Questionnaire Response ◽  
Diagnosis And Management ◽  
Asymptomatic Patients ◽  
Primary Immune Thrombocytopenia

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.

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