Nplate® is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in adult patients with immune thrombocytopenia (ITP) who have had an insufficient corticosteroids, immunoglobulins, or splenectomy.Nplate® is indicated for the treatment of thrombocytopenia in pediatric patients 1 year of age and older with ITP for at least 6 months who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
Nplate® is not indicated for the treatment of thrombocytopenia due to myelodysplastic syndrome (MDS) or any cause of thrombocytopenia other than ITP. Nplate® should be used only in patients with ITP whose degree of thrombocytopenia
and clinical condition increase the risk for bleeding. Nplate® should not be used in an attempt to normalize platelet counts.
Nplate® is the once-weekly, in-office treatment that offers:
No new safety signals observed up to 5 years of continuous treatment1
No need to remember to take a pill every day2-5
No liver monitoring, and no known drug interactions or dietary restrictions2,6
Pivotal Trials (Pooled)
Adverse Reactions in Placebo-Controlled Phase 3 Trials2
Adverse drug reactions with ≥ 5% higher incidence with Nplate® vs placebo control
ADVERSE REACTIONS | NPLATE® (n = 84) | CONTROL (n = 41) |
---|---|---|
Arthralgia | 26% | 20% |
Dizziness | 17% | 0% |
Insomnia | 16% | 7% |
Myalgia | 14% | 2% |
Pain in extremity | 13% | 5% |
Abdominal pain | 11% | 0% |
Shoulder pain | 8% | 0% |
Dyspepsia | 7% | 0% |
Paresthesia | 6% | 0% |
Headache was the most commonly reported adverse drug reaction, occurring in 35% of patients receiving Nplate® and 32% of patients receiving placebo2
Incidence of Bleeding Events*
Bleeding events were captured as adverse events and were not a predefined endpoint in the pivotal trial.2
Incidence of Grade 2 and above bleeding events across pivotal trials2
*Bleeding events Grade 2 through 5 were defined as follows: Grade 2 is moderate, causing enough discomfort to interfere with usual activity; Grade 3 is severe, incapacitating, making it impossible to work or engage in usual activities; Grade 4 is life-threatening, patient is at risk of death at the time of the event; Grade 5 is fatal.7
Long-Term Extension Data
Adverse Events (AEs) Did Not Increase in Frequency or Type With Longer Nplate® Drug Exposure1
Long-term safety profile in open-label extension study
AEs WITH NPLATE® | % of patients (n) |
---|---|
Serious treatment-related AEs | 8% (24/291) |
Thrombotic events | 6.5% (19/291) |
Bone marrow reticulin increase | 1.4% (4/291) |
Neutralizing antibodies | < 1% (4/291) |
Deaths | 5% (16/291) |
Most common AEs
The most common AEs included headache (38% of patients), nasopharyngitis (34%), fatigue (32%), contusion (31%), upper respiratory tract infection (26%), diarrhea (25%), and epistaxis (25%).1
5-year follow-up study established long-term safety, demonstrating no increase in AEs or new AE types with longer Nplate® exposure1
CHANGES IN BM MORPHOLOGY
Nplate® Showed Minimal Changes in Reticulin Fiber Formation and Collagen Fibrosis8
– Of the 9 patients who had increases of ≥ 2 on the modified Bauermeister scale, 1 had neutropenia detected by laboratory test, and 2 had adverse events of anemia
Percentage of patients treated with romiplostim that had changes in collagen fibrosis or bone marrow reticulin formation8
Progression of reticulin fiber formation ≥ 2 grades or an increase to Grade 4 (presence of collagen)
Presence of collagen (Grade 4 findings)
Of the 9 patients who had increases of ≥ 2 on the modified Bauermeister scale, one had neutropenia detected by laboratory test, and two had adverse events of anemia.8
STUDY DESIGN & BASELINE CHARACTERISTICS
A Phase 4, Open-label, Multicenter Trial Evaluating Changes in BM Morphology2
A phase 4, open-label, multicenter trial prospectively evaluated bone marrow for reticulin formation and collagen fibrosis in adult patients with immune thrombocytopenia (ITP) receiving Nplate® (romiplostim) or a non-US-approved romiplostim product. A modified Bauermeister grading scale was used for both assessments.2
Modified Bauermeister scale: quantification of bone marrow reticulin and collagen8
GRADE 0 | No reticulin |
GRADE 1 | Occasional fine individual fibers and foci of a fine fiber network |
GRADE 2 | Fine fiber network throughout most of the section; no coarse fibers |
GRADE 3 | Diffuse fiber network with scattered thick coarse fibers but no mature collagen (negative trichrome staining) |
GRADE 4 | Diffuse, coarse fiber network with areas of collagenization (positive trichrome staining) |
Patients were administered Nplate® by subcutaneous injection once weekly for up to 3 years. Based on cohort assignment at the time of study enrollment, patients were evaluated for bone marrow reticulin and collagen at year 1 (cohort 1), year 2 (cohort 2), or year 3 (cohort 3) in comparison to the baseline bone marrow at the start of the study. A total of 169 patients were enrolled in the 3 cohorts.2
Baseline characteristics8
Study limitations8
Nplate® administration may increase the risk for development or progression of reticulin fiber formation within the bone marrow. This formation may improve upon discontinuation of Nplate®. In a clinical trial, one patient with ITP and hemolytic anemia developed marrow fibrosis with collagen during Nplate® therapy.
Nplate® is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in adult patients with immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. Nplate® is indicated for the treatment of thrombocytopenia in pediatric patients 1 year of age and older with ITP for at least 6 months who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
Nplate® is not indicated for the treatment of thrombocytopenia due to myelodysplastic syndrome (MDS) or any cause of thrombocytopenia other than ITP. Nplate® should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding. Nplate® should not be used in an attempt to normalize platelet counts.
Please see full Prescribing Information and Medication Guide.
References: 1. Kuter DJ, Bussel JB, Newland A. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. Br J Haematol. 2013;161(3):411-423. 2. Nplate® (romiplostim) prescribing information, Amgen. 3. Promacta® (eltrombopag) full Prescribing Information, Novartis. 4. Doptelet® (avatrombopag) full Prescribing Information, Sobi. 5. Tavalisse® (fostamatinib disodium hexahydrate) full Prescribing Information, Rigel. 6. Data on file, Amgen; Clinical Study Report 20080435; 2014. 7. Gernsheimer T. Chronic idiopathic thrombocytopenic purpura: mechanisms of pathogenesis. Oncologist. 2009;14(1):12-21. 8. Janssens A, Rodeghiero F, Anderson D, et al. Changes in bone marrow morphology in adults receiving romiplostim for the treatment of thrombocytopenia associated with primary immune thrombocytopenia. Ann Hematol. 2015;95(7):1077-1087.