Nplate (romiplostim) pivotal trials and efficacy data When a timely and efficacious response
matters, Nplate® restores stability
for 8 out of 10 patients1,2,*1,2,*
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 (n = 63), another 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 definitions1,21,2
Nplate (romiplostim) pivotal trials, primary and secondary endpoint definitions

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

Results in non-splenectomized patients NON-SPLENECTOMIZED
Results in splenectomized patients SPLENECTOMIZED

Achieve and maintain target platelet counts in non-splenectomized patients11

The majority of non-splenectomized patients achieved target response1,21,2

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

Onset of response

In the pivotal trials (N = 125)11

  • 30% of patients treated with Nplate® responded after 1 dose44,‡
    • 37% in non-splenectomized patients (study n = 62)
    • 24% in splenectomized patients (study n = 63)

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

Achieve and maintain target platelet counts in non-splenectomized patients1 1

The majority of non-splenectomized patients achieved target response1 ,2 1,2

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 1,2

Onset of response

In the pivotal trials (N = 125)11

  • 30% of patients treated with Nplate® responded after 1 dose4 4,‡
    • 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 1

Patients maintained platelet counts ≥ 50 x 109/L throughout the 6-month pivotal trial1,21,2

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.2 2

Nplate® is the only weekly treatment that delivers the lasting stability chronic ITP demands1,21,2

In the final analysis of the long-term extension study

  • The majority of patients achieved a platelet response for 27 weeks or greater. The maximum number of weeks with a platelet response was 1844 4
Nplate (romiplostim) pivotal trials response and lasting stability

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.

Watch Dr. Michael Tarantino from the Bleeding and Clotting Disorders Institute, IL discuss relapse in adult chronic ITP and first-line treatment failure
First-line Treatment Failure
Watch Dr. Terry Gernsheimer from the Seattle Cancer Care Alliance, WA, discuss relapse in adult chronic ITP following splenectomy
Relapse Following Splenectomy

Concurrent or rescue ITP therapies1 1

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.2 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 2

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.1 1

Non-splenectomized patients receiving rescue therapy1,2 1,2

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 1

*Based on Overall Platelet Response (88%) in non-splenectomized patients.1,21,2

Prior ITP treatments in both study groups included corticosteroids, immunoglobulins, rituximab, cytotoxic therapies, danazol, and azathioprine.1,21,2 (Rituximab is not FDA approved for use in ITP.)

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 4

§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.1 1

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

Achieve and maintain target platelet counts in splenectomized patients1 1

The majority of splenectomized patients achieved target response1,21,2

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

Onset of response

In the pivotal trials (N = 125)11

  • 30% of patients treated with Nplate® responded after 1 dose44,‡
    • 37% in non-splenectomized patients (study n = 62)
    • 24% in splenectomized patients (study n = 63)

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.1,2 1,2

Achieve and maintain target platelet counts in splenectomized patients11

The majority of splenectomized patients achieved target response1,21,2

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

Onset of response

In the pivotal trials (N = 125)11

  • 30% of patients treated with Nplate® responded after 1 dose4 4,‡
    • 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 Response11

Patients maintained platelet counts ≥ 50 x 109/L throughout the 6-month pivotal trial1,21,2

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.2 2

Comparable results were seen in non-splenectomized patients.2 2

Achieve a Durable Target Response1 1

Patients maintained platelet counts ≥ 50 x 109/L throughout the 6-month pivotal trial1,21,2

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.2 2

Nplate® is the only weekly treatment that delivers the lasting stability chronic ITP demands1,21,2


In the final analysis of the long-term extension study

  • The majority of patients achieved a platelet response for 27 weeks or greater. The maximum number of weeks with a platelet response was 1844 4

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.

Watch Dr. Terry Gernsheimer from the Seattle Cancer Care Alliance, WA, discuss relapse in adult chronic ITP following splenectomy
Relapse Following Splenectomy
Watch Dr. Michael Tarantino from the Bleeding and Clotting Disorders Institute, IL discuss relapse in adult chronic ITP and first-line treatment failure
First-line Treatment Failure

Concurrent or rescue ITP therapies1 1

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.2 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 2

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

*Based on Overall Platelet Response (88%) in non-splenectomized patients.1,21,2

Prior ITP treatments in both study groups included corticosteroids, immunoglobulins, rituximab, cytotoxic therapies, danazol, and azathioprine.1,21,2 (Rituximab is not FDA approved for use in ITP.)

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 4

§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.1 1

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

Data for bleeding events5 5

Bleeding events were captured as adverse events and were not a predefined endpoint in the pivotal trial.5 5

Incidence of grade 2 and above bleeding events across pivotal trials1,4,51,4,5

Definitions of grade 2 through 5 bleeding events5 5

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.5 5

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.

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. Headaches were usually of mild or moderate severity.
  • Most common adverse reactions in adults (≥ 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%).

Pediatric ITP

  • In pediatric patients of age ≥ 1 year receiving romiplostim 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%).
  • Most common adverse reactions (≥ 5% incidence and ≥ 5% more frequent in the romiplostim 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%).

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.

INDICATIONS

Nplate® is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in adult patients with chronic 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 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.

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. Headaches were usually of mild or moderate severity.
  • Most common adverse reactions in adults (≥ 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%).

Pediatric ITP

  • In pediatric patients of age ≥ 1 year receiving romiplostim 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%).
  • Most common adverse reactions (≥ 5% incidence and ≥ 5% more frequent in the romiplostim 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%).

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.

INDICATIONS

Nplate® is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in adult patients with chronic 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 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. Kuter DJ, Bussel JB, Newland A, et al. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. Br J Haematol. 2013;161:411-423. 4. Data on file, Amgen. 5. 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.

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