INDICATIONS

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.

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PRESCRIBING INFORMATION INDICATIONS IMPORTANT SAFETY INFORMATION VISIT PATIENT SITE

Results From the Nplate® vs SOC Trial Were Consistent With Those in the Phase 3 Pivotal Trial in Non-Splenectomized Patients1

Two, Phase 3 Pivotal Trials: The pivotal trials consisted of two phase 3, parallel, double-blind studies where patients with chronic immune thrombocytopenia (ITP) who had completed at least one prior treatment were randomized to Nplate® or placebo. The primary endpoint was the achievement of a durable platelet response, defined as a weekly platelet count ≥ 50 x 109/L for at least 6 of the last 8 weeks of the 24-week treatment period in the absence of rescue therapies at any time.1,2

See the Nplate® pivotal trials information

Nplate® vs SOC

TREATMENT FAILURE

Reduced Treatment Failure vs SOC Therapies3

Incidence of treatment failure at 1 year3

Incidence of treatment failure at one year
  • Approximately 9 out of 10 non-splenectomized patients in the Nplate® arm did not experience treatment failure
  • Results were consistent with those in the pivotal phase 3 trial of non-splenectomized patients3
  • The percentage of patients on Nplate® achieving a platelet response (≥ 50 x 109/L) ranged from 71% (108/152) to 92% (127/138) at any scheduled visit (weeks 2–52)3
Review Nplate® vs SOC trial safety information

REDUCTION IN BLEEDS

Nplate® Significantly Reduced the Potential for Bleeding Events3

Rate of Grade 3 and above bleeding events3,4,*

Rate of grade 3 and above bleeding events

Rate = adverse event rate per 100 patient-weeks on study medication. Rate was calculated by dividing the total number of reported events by the total number of patient-weeks and then multiplying by 100.3

  • In the pivotal trial with non-splenectomized patients, 10% (4/42) of Nplate® patients vs 30% (6/20) of control patients experienced a Grade 2† or above bleeding event4
  • Bleeding events were captured as adverse events and were not a predefined endpoint in the pivotal trial

*Bleeding events Grade 2 through 5 were defined as follows: Grade 2 is moderate; Grade 3 is severe; Grade 4 is life-threatening; Grade 5 is fatal.3

†Grading was per the Common Terminology Criteria for Adverse Events, v3. http://ctep.cancer.gov.

RESPONSE ACHIEVED

Nplate® platelet response: Up to 92% of patients achieved platelet counts > 50 × 109/L at any scheduled visit throughout the 1-year trial3

sustained-response-image

Nplate® or SOC: Platelet response over 1 year3

Nplate®
  • At any scheduled visit between weeks 2 and 52, 71% (108/152) to 92% (127/138) of patients in the Nplate® group had a platelet response > 50 × 109/L
  • Median platelet count was 108–176 × 109/L with Nplate® and 35–52 × 109/L with SOC over the course of the 1-year trial3

STUDY DESIGN

Multicenter, Randomized, Controlled, Open-Label Evaluation Study for Non-Splenectomized Patients With ITP3

Treatment with Nplate® vs SOC was studied in a multicenter, randomized, controlled, 52-week, open-label evaluation of Nplate® and medical SOC therapy for non-splenectomized patients with ITP (N = 234)3

The design of this trial does not allow for comparison of Nplate® to the individual treatments received in the SOC arm

  • Co-primary endpoints were the incidence of treatment failure and splenectomy
  • Safety outcomes were measured as one of the secondary endpoints
  • Concomitant therapies were allowed for both treatment groups
  • To achieve a target platelet count of 50–200 x 109/L, Nplate® was administered once weekly at a starting dose* of 3 mcg/kg up to a maximum dose of 10 mcg/kg
  • Treatments for patients assigned to the SOC group were selected by the investigator according to standard institutional practices or therapeutic guidelines

Co-primary endpoints

1. INCIDENCE OF TREATMENT FAILURE

Study discontinuation before treatment failure was also considered treatment failure

Platelet count ≤ 20 x 109/L for 4 consecutive weeks at highest recommended dose of study treatment

Major bleeding event

Change in treatment due to side effects or bleeding symptoms

2. INCIDENCE OF SPLENECTOMY

Study discontinuation before splenectomy was also considered splenectomy

Therapies used in SOC arm3

Therapies used in standard of care arm
  • Patients could receive one or more types of SOC therapies, including watchful waiting3
  • Patients already receiving ITP therapies at baseline (21/157 in Nplate® group; 5/77 in SOC group) could continue receiving these throughout the trial3
  • Other ITP treatments used in the Nplate® arm at any time during the 1-year trial included corticosteroids (37% of patients), immunoglobulins (7%), rituximab (1%), azathioprine (1%), danazol (2%), other medications‡ (6%), and platelet transfusions (6%)3
See the Nplate® vs SOC safety data

*The recommended starting dose for Nplate® is 1 mcg/kg. Please see Nplate® Prescribing Information for complete dosing instructions, including guidelines for dose adjustments. In the pivotal trials, the median dose of Nplate® was 2 mcg/kg (25th–75th percentile: 1–3 mcg/kg) in the study of non-splenectomized patients and 3 mcg/kg (25th–75th percentile: 2–7 mcg/kg) in the study of splenectomized patients.5

†Rituximab is not FDA-approved for use in ITP. 5

‡Including vincristine, cyclosporine, tranexamic acid, ascorbic acid, calcium, ethamsylate, pantoprazole, and Expasyl®. Expasyl is a registered trademark and entire property of Pierre Rolland.

SOC, standard-of-care.

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Important Safety Information

Risk of Progression of Myelodysplastic Syndromes to Acute Myelogenous Leukemia

  • In Nplate® (romiplostim) 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 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®.
  • 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.

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 (1‑800‑772‑64361‑800‑772‑6436). 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.
Adverse Reactions
Adult ITP
  • In the placebo-controlled trials of adult ITP patients, headache was the most commonly reported adverse drug reaction, occurring in 35% of patients receiving Nplate® and 32% of patients receiving placebo. Adverse drug reactions in adults with a ≥ 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%).
  • The safety profile of Nplate® was similar across patients, regardless of ITP duration. The following adverse reactions (at least 5% incidence and at least 5% more frequent with Nplate® compared with placebo or standard of care) occurred in Nplate® patients with ITP duration up to 12 months: bronchitis, sinusitis, vomiting, arthralgia, myalgia, headache, dizziness, diarrhea, upper respiratory tract infection, cough, nausea and oropharyngeal pain. The adverse reaction of thrombocytosis occurred with an incidence of 2% in adults with ITP duration up to 12 months.
Pediatric ITP
  • The most common adverse reactions experienced by ≥ 5% of patients receiving Nplate® with ≥ 5% higher incidence in the Nplate® arm across the two placebo-controlled trials were contusion (41%), upper respiratory tract infection (31%), oropharyngeal pain (25%), pyrexia (24%), diarrhea (20%), rash (15%), and upper abdominal pain (14%).
  • In pediatric patients of age ≥ 1 year receiving Nplate® for ITP, adverse reactions with an incidence of ≥ 25% in the two randomized trials were: contusion (41%), upper respiratory tract infection (31%), and oropharyngeal pain (25%).
  • In a long-term, single arm, open label pediatric safety study, headache occurred in 78/203 patients (38%); the incidence rates of other adverse reactions were similar to those reported in the placebo-controlled studies.

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.

INDICATIONS

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.

Important Safety Information

Risk of
Progression of Myelodysplastic Syndromes to Acute Myelogenous Leukemia

  • In Nplate® (romiplostim) 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 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®.
  • 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.

Loss of Response to Nplate®

References: 1. Nplate® (romiplostim) prescribing information, Amgen. 2. Kuter DJ, Bussel JB, Lyons RM, et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenia purpura: a double-blind randomised controlled trial. Lancet. 2008;371(9610):395-403. 3. Kuter DJ, Rummel M, Boccia R, et al. Romiplostim or standard of care in patients with immune thrombocytopenia. N Engl J Med. 2010;363:1889-1899. 4. Gernsheimer T. Chronic idiopathic thrombocytopenic purpura: mechanisms of pathogenesis. Oncologist. 2009;14(1):12-21. 5. RITUXAN® (rituximab) full Prescribing Information, Genentech, Inc.