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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When a timely and efficacious response matters, Nplate® restores stability for 8 out of 10 patients1,2,*

Nplate (romiplostim) pivotal trials and efficacy data
STUDY DESIGN
Pivotal trials study design1,2

Nplate® was studied vs placebo in 2 parallel, double-blind, 24-week, phase 3 trials in patients with adult chronic immune thrombocytopenia (ITP). One trial enrolled splenectomized patients (n = 63), the other enrolled non-splenectomized patients (n = 62).

  • Patients must have completed ≥ 1 prior treatment and had a platelet count of ≤ 30 x 109/L prior to study entry
  • Patients were randomized (2:1) to 24 weeks of Nplate® (1 mcg/kg subcutaneous) or placebo
  • Nplate® dose adjustments were based on platelet counts, and dose adjustments of concomitant ITP therapies were allowed
Pivotal trials endpoint definitions1,2
Primary Endpoint:
Durable platelet response
  • Weekly platelet responses (platelet count ≥ 50 x 109/L) during at least 6 of the last 8 weeks of treatment in 24-week treatment period
  • No rescue therapy at any time during treatment
Secondary Endpoint:
Overall platelet response
  • Overall platelet response = durable plus transient platelet responses
  • Transient platelet response: ≥ 4 weekly responses without durable response from weeks 2 to 25, excluding platelet counts within 8 weeks of rescue therapy

Review the article for the Nplate® pivotal trials. Read more

*Based on Overall Platelet Response (88%) in non-splenectomized patients.1

Prior ITP treatments in both study groups included corticosteroids, immunoglobulins, rituximab, cytotoxic therapies, danazol, and azathioprine. (Rituximab is not FDA approved for use in ITP.)1,3

EFFICACY
Achieve and maintain target platelet counts

Results in non-splenectomized patients

The majority of non-splenectomized patients achieved a durable platelet response1,2
Overall and durable platelet response
in non-splenectomized patients

Patients in the control arm received placebo. Some patients in both arms were receiving concurrent ITP medications at baseline and were allowed to continue receiving them.1,2

Onset of response

In the pivotal trials (N = 125)1

  • 30% of patients treated with Nplate® responded after 1 dose4,*
    • 37% in non-splenectomized patients (15/41)
    • 24% in splenectomized patients (10/42)

*Defined as platelet count ≥ 50 x 109/L measured 1 week after the first dose. In patients who received placebo, 0% achieved this response.4

Patients maintained platelet counts ≥ 50 x 109/L throughout the 6-month pivotal trial5
Median weekly platelet counts in non-splenectomized patients2

Comparable results were seen in splenectomized patients.1,2 See those results below.

Median weekly platelet count includes all patients, even those receiving rescue therapy. Error bars indicate the range from the first to the third quartiles. Red line indicates platelet count of 50 x 109/L.2

Concurrent or rescue ITP therapies
Non-splenectomized patients who reduced or discontinued* concurrent ITP therapies1,2

n = number of patients receiving concurrent ITP medication baseline.2

Changes could be made to concurrent ITP therapies (corticosteroids, azathioprine, or danazol) during the first 12 weeks of study when platelet counts were > 100 x 109/L.2

Non-splenectomized patients
receiving rescue therapy1,2

Rescue therapies included corticosteroids, intravenous immunoglobulin (IVIG), platelet transfusions, and anti-D immunoglobulin and were permitted for bleeding, for wet purpura, or if the patient was at immediate risk for hemorrhage.1

*Discontinuation was defined as discontinuation of all therapies for patients receiving multiple concomitant baseline therapies.1

Results in splenectomized patients

The majority of splenectomized patients achieved target response1,2
Overall and durable platelet response in
splenectomized patients

Patients in the control arm received placebo. Some patients in both arms were receiving concurrent ITP medications at baseline and were allowed to continue receiving them.1

Patients maintained platelet counts ≥ 50 x 109/L throughout the 6-month pivotal trial2
Median weekly platelet counts in splenectomized patients

Median weekly platelet count includes all patients, even those receiving rescue therapy. Error bars indicate the range from the first to the third quartiles. Red line indicates platelet count of 50 x 109/L.2

Comparable results were seen in non-spelectomized patients.2 See those results above.

The majority of non-splenectomized patients achieved target response1,2

Patients in the control arm received placebo. Some patients in both arms were receiving concurrent ITP medications at baseline and were allowed to continue receiving them.1,2

Onset of response

In the pivotal trials (N = 125)1

  • 30% of patients treated with Nplate® responded after 1 dose2,‡
    • 37% in non-splenectomized patients (study n = 62)
    • 24% in splenectomized patients (study n = 63)

Review Nplate® pivotal trials safety information. Read more

Achieve a Durable Target Response1

Patients maintained platelet counts ≥ 50 x 109/L throughout the 6-month pivotal trial1,2

Nplate (romiplostim) sustained median platelet response in non-splenectomized patients Nplate (romiplostim) sustained median platelet response in non-splenectomized patients

Comparable results were seen in splenectomized patients.2

61% of patients achieved a durable platelet response during the last 8 weeks of the
study1,2

Median weekly platelet count includes all patients, even those receiving rescue therapy. Error bars indicate the range from the first to the third quartiles. Light-blue line indicates platelet count of 50 x 109/L.2

Concurrent or rescue ITP therapies
Splenectomized patients who reduced or
discontinued* concurrent ITP therapies1,2

n = number of patients receiving concurrent ITP medication baseline.2

Changes could be made to concurrent ITP therapies (corticosteroids, azathioprine, or danazol) during the first 12 weeks of study when platelet counts were > 100 x 109/L.2

Splenectomized patients receiving rescue therapy1,2

Rescue therapies included corticosteroids, intravenous immunoglobulin (IVIG), platelet transfusions, and anti-D immunoglobulin and were permitted for bleeding, for wet purpura, or if the patient was at immediate risk for hemorrhage.1

*Discontinuation was defined as discontinuation of all therapies for patients receiving multiple concomitant baseline therapies.1

These physicians choose Nplate® for chronic ITP in adults. Learn why.

Watch Dr. Michael Tarantino from the Bleeding and Clotting Disorders Institute, IL, and Dr. Terry Gernsheimer from the Seattle Cancer Care Alliance, WA, as they discuss why they turn to Nplate® after relapse in adult chronic ITP.

First-line Treatment Failure
Relapse Following Splenectomy
SAFETY
In Nplate (romiplostim) clinical trials, Adverse & Side Effects were mild to moderate Adverse reactions in placebo-controlled phase 3 trials1

 

Pivotal trials: adverse drug reactions with ≥ 5% higher incidence
with Nplate® vs placebo control in phase 3 trials1
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 placebo1
In the placebo-controlled studies, the majority of adverse drug reactions were mild to moderate in severity1

Adverse reactions in placebo-controlled phase 3 trials1

Pivotal trials: adverse drug reactions with ≥ 5% higher incidence with Nplate® vs placebo control in phase 3 trials1

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 placebo.1

Data for bleeding events

Bleeding events were captured as adverse events and were not a predefined endpoint in the pivotal trial.1

Incidence of grade 2 and above bleeding
events across pivotal trials1

Definitions of grade 2 through 5 bleeding events6

Grade 2 Moderate; causing enough discomfort to interfere with usual activity
Grade 3 Severe; incapacitating, making it impossible to work or engage in usual activities
Grade 4 Life-threatening; patient is at risk of death at the time of the event
Grade 5 Fatal

Patients in the control arm received placebo. Some patients in both arms were receiving concurrent ITP medications at baseline and were allowed to continue receiving them.1

Post hoc analysis showed that no bleeding events of grade 2 or higher severity occurred at platelet counts > 50 x 109/L in these pivotal trials.6

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.

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;371:395-403. 3. RITUXAN® full Prescribing Information, Genentech, Inc. 4. Data on file, Amgen; Onset of Response; 2018. 5. Data on file, Amgen; Duration of Response; 2017. 6. Gernsheimer TB, George JN, Aledort LM, et al. Evaluation of bleeding and thrombotic events during long-term use of romiplostim in patients with chronic immune thrombocytopenia (ITP). J Thromb Haemost. 2010;8(6):1372-1382.

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