1-Year Trial Evaluating Nplate® and Medical Standard of Care in Non-splenectomized Patients
Nplate®: Sustained response maintained treatment goals
The data from this Nplate® or SOC trial were recently published in N Engl J Med.
Nearly 90% of Nplate® patients avoided treatment failure in the 1-year trial in non-splenectomized patients[5]
- Most Nplate® patients in the phase 3b trial achieved raised and sustained platelet counts without major bleeding or intolerable side effects during the 1-year treatment period.[5]
- Results were consistent with those in the pivotal phase 3 trial in non-splenectomized patients.[3][5][6]
This analysis includes only patients who experienced a treatment failure and not patients who discontinued from the study.
Sustained response, with up to 92% of Nplate® patients achieving platelet counts > 50 x 109/L throughout the
1-year trial[5]
*Per the protocol, the starting dose of Nplate® was 3 mcg/kg. The Nplate® dosing adjustment algorithm was as follows: For
platelet counts < 30 x 109/L, the dose was increased by 1 mcg/kg every week; for platelet counts 30 to 450 x 109/L, the dose may have been
adjusted (increased or decreased) in increments of 1 mcg/kg at the investigator’s discretion no more frequently than
every 2 weeks; for platelet counts > 450 x 109/L, the dose was held, and the dose was reduced by 1 mcg/kg at the next scheduled
dosing day when the platelet count had fallen to < 200 x 109/L. The recommended range for maintaining platelet counts
was 50 x 109/L to 200 x 109/L. The dose was held when the current dose was 1 mcg/kg and a dose reduction
was required. Once the platelet count was ≤ 50 x 109/L, dosing was resumed at 1 mcg/kg.[3]
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- 71% (108/152) to 92% (127/138) of Nplate® patients and 26% (16/62) to 51% (26/51) of SOC patients achieved weekly platelet counts > 50 x 109/L from week 2 through the end of the 1-year treatment period.[5]
- Median platelet count was 108–176 x 109/L with Nplate® and 35–52 x 109/L with SOC over the course of the 1-year trial.[5]
- Increased platelet counts were maintained with a relatively low and stable dose of Nplate®.[5]
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Significantly reduced severe, life-threatening bleeding events
Very low rate of ≥ grade 3 bleeding events throughout the 1-year study[5]
*Grading was per the Common Terminology Criteria for Adverse Events, v3. http://ctep.cancer.gov.
- Nplate® significantly reduced the rate of overall bleeding events: 3.56 events per 100 patient-weeks (260 events in 7294 patient-weeks) with Nplate® and 5.02 events per 100 patient-weeks (153 events in 3050 patient-weeks) with SOC
(P = 0.001).[5] - Bleeding events ≥ grade 3 were reported in 3% (5/154) of patients in the Nplate® group and 7% (5/75) of patients in the SOC group.[5]
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INDICATION
Nplate® is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in patients with chronic immune thrombocytopenia (ITP) 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 chronic 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.
Important Safety Information
Risk of Progression of Myelodysplastic Syndromes to Acute Myelogenous Leukemia
- In Nplate® clinical trials of patients with myelodysplastic syndromes (MDS) and severe thrombocytopenia, progression from MDS to acute myelogenous leukemia (AML) has been observed.
- Nplate® is not indicated for the treatment of thrombocytopenia due to MDS or any cause of thrombocytopenia other than chronic ITP.
Thrombotic/Thromboembolic Complications
- Thrombotic/thromboembolic complications may result from increases in platelet counts with Nplate® use. Portal vein thrombosis has been reported in patients with chronic liver disease receiving Nplate®. Nplate® should be used with caution in patients with ITP and chronic liver disease.
- To minimize the risk for thrombotic/thromboembolic complications, do not use Nplate® in an attempt to normalize platelet counts. Follow the dose adjustment guidelines to achieve and maintain a platelet count of ≥ 50 x 109/L.
Bone Marrow Reticulin Formation and Risk for Bone Marrow Fibrosis
- 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 study, one patient with ITP and hemolytic anemia developed marrow fibrosis with collagen during Nplate® therapy. Clinical studies are in progress to assess the risk of bone marrow fibrosis and clinical consequences with cytopenias.
- If new or worsening morphological abnormalities or cytopenia(s) occur, consider a bone marrow biopsy to include staining for fibrosis.
Worsened Thrombocytopenia after Cessation of Nplate®
- In clinical studies of patients with chronic ITP who had Nplate® discontinued, four of 57 patients developed thrombocytopenia of greater severity than was present prior to Nplate® therapy. This worsened thrombocytopenia resolved within 14 days.
- Following discontinuation of Nplate®, obtain weekly CBCs, including platelet counts, for at least 2 weeks and consider alternative treatments for worsening thrombocytopenia, according to current treatment guidelines.
Lack or Loss of Response to Nplate®
- Hyporesponsiveness or failure to maintain a platelet response with Nplate® should prompt a search for causative factors, including neutralizing antibodies to Nplate®.
- To detect antibody formation, submit blood samples to Amgen (1-800-772-6436). Amgen will assay these samples for antibodies to Nplate® and thrombopoietin (TPO).
- Discontinue Nplate® if the platelet count does not increase to a level sufficient to avoid clinically important bleeding after 4 weeks at the highest weekly dose of 10 mcg/kg.
Laboratory Monitoring
- Obtain CBCs, including platelet counts, weekly during the dose adjustment phase of Nplate® therapy and then monthly following establishment of a stable Nplate® dose.
- Obtain CBCs, including platelet counts, weekly for at least two weeks following discontinuation of Nplate®.
Adverse Reactions
- In the placebo-controlled studies, headache was the most commonly reported adverse drug reaction, occurring in 35% of patients receiving Nplate® and 32% of patients receiving placebo. Headaches were usually of mild or moderate severity.
- Most common adverse reactions (≥ 5% higher patient incidence in Nplate® versus placebo) were 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%), and Paresthesia (6%, 0%).
Please see Prescribing Information and Medication Guide
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Clinical studies have evaluated the safety and efficacy of Nplate® treatment in adult patients with chronic ITP. Nplate® clinical trials have been conducted in both splenectomized and non-splenectomized patients, with some patients receiving Nplate® treatment for > 5 years.
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