Help restore stability with Nplate®11
Pivotal trials study design1,21,2

Nplate® was studied vs placebo in two parallel, double-blind, 24-week, phase 3 trials in patients with adult chronic immune thrombocytopenia (ITP). One trial enrolled splenectomized patients, another enrolled non-splenectomized patients.

  • 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.
  • Dose adjustments were based on platelet counts.
  • Patients could have doses of concurrent ITP medications adjusted only during the first 12 weeks.
Pivotal trials endpoint definitions1,21,2
Nplate (romiplostim) pivotal trials, primary and secondary endpoint definitions

Review the article for the Nplate® pivotal trials. Read more > (Please be advised that you are leaving an Amgen website and will be directed to a third party site. Amgen is not responsible for the content on the site you are about to enter.)

Some information included in this article may not be consistent with the approved product labeling. For specific information regarding the approved use of Nplate®, see the full Prescribing Information.

Results in non-splenectomized patientsNON-SPLENECTOMIZED
Results in splenectomized patientsSPLENECTOMIZED

Help restore stability: achieve and maintain target platelet counts11

Platelet response in
non-splenectomized patients1,21,2

Nplate (romiplostim) durable and overall platelet response in non-splenectomized patients

Review Nplate® pivotal trials safety information. Read more >

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.1,21,2

Help restore stability: achieve and maintain target platelet counts11

Platelet response in
non-splenectomized patients1,21,2

Review Nplate® pivotal trials safety information. Read more >

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.1,21,2

Deliver platelet stability and durability11

Sustained median platelet count ≥ 50 x 109/L1,21,2

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.22

61% of patients achieved a durable platelet response during the last 8 weeks of the study1,21,2

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.22

Concurrent or rescue ITP therapies11

Non-splenectomized patients who reduced or discontinued concurrent ITP therapies1,21,2

Concurrent ITP therapies Concurrent ITP therapies

n = number of patients receiving concurrent ITP medication baseline.22

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.22

Non-splenectomized patients receiving rescue therapy1,21,2


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.11

Non-splenectomized patients receiving rescue therapy1,21,2

Non-splenectomized patients with ITP who received rescue therapy

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.11

*Prior ITP treatments in both study groups included corticosteroids, immunoglobulins, rituximab,§ cytotoxic therapies, danazol, and azathioprine.1,21,2

Reduction was defined as > 25% dose reduction. For multiple baseline concurrent therapies, at least one therapy with > 25% dose reduction was observed without an increase in other ITP therapies.11

Discontinuation was defined as discontinuation of all therapies for patients receiving multiple concomitant baseline therapies.11

§Rituximab is not FDA approved for use in ITP.

Help restore stability: achieve and maintain target platelet counts11

Platelet response in splenectomized patients1,21,2

Nplate (romiplostim) durable and overall 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.1,21,2

Review Nplate® pivotal trials safety information. Read more >

Help restore stability: achieve and maintain target platelet counts11

Platelet response in splenectomized patients1,21,2

Nplate (romiplostim) sustained 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.1,21,2

Review Nplate® pivotal trials safety information. Read more >

Sustained median platelet count ≥ 50 x 109/L1,21,2

Nplate (romiplostim) sustained platelet response in splenectomized patients Nplate (romiplostim) sustained platelet response 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. Light-blue line indicates platelet count of 50 x 109/L.22

Comparable results were seen in non-splenectomized patients.22

Sustained median platelet count ≥ 50 x 109/L1,21,2

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.22

Comparable results were seen in non-splenectomized patients.22

Concurrent or rescue ITP therapies11

Splenectomized patients who reduced or discontinued concurrent ITP therapies1,21,2

Concurrent ITP therapies Concurrent ITP therapies

n = number of patients receiving concurrent ITP medication at baseline.22

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.22

Splenectomized patients receiving rescue therapy1,21,2

Splenectomized patients with ITP who received rescue therapy Splenectomized patients with ITP who received rescue therapy


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.11

*Prior ITP treatments in both study groups included corticosteroids, immunoglobulins, rituximab,§ cytotoxic therapies, danazol, and azathioprine.1,21,2

Reduction was defined as > 25% dose reduction. For multiple baseline concurrent therapies, at least one therapy with > 25% dose reduction was observed without an increase in other ITP therapies.11

Discontinuation was defined as discontinuation of all therapies for patients receiving multiple concomitant baseline therapies.11

§Rituximab is not FDA approved for use in ITP.

Data for bleeding events33

Bleeding events were captured as adverse events and were not a predefined endpoint in the pivotal trial.33

Incidence of grade 2 and above bleeding events across pivotal trials1,3,43,4

Bleeding events data

Definitions of grade 2 through 5 bleeding events33

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.1,21,2

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.33

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 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®.
  • 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.

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 trials, 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%).
  • 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.


Women who become pregnant during Nplate® treatment are encouraged to enroll in Amgen's Pregnancy Surveillance Program. Patients or their physicians should call 1‑800‑77‑AMGEN1‑800‑77‑AMGEN (1‑800‑772‑64361‑800‑772‑6436) to enroll.

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.

Please see full Prescribing Information and Medication Guide.

See More Close

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 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®.
  • 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.

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 trials, 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%).
  • 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.

Women who become pregnant during Nplate® treatment are encouraged to enroll in Amgen's Pregnancy Surveillance Program. Patients or their physicians should call 1‑800‑77‑AMGEN1‑800‑77‑AMGEN (1‑800‑772‑64361‑800‑772‑6436) to enroll.

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.

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 thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008;371:395-403. 3. 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;8:1372-1382. 4. Data on file, Amgen.

Back to top