When a timely and efficacious response matters, Nplate^® restores stability for 8 out of 10 patients^1,2,*

Nplate (romiplostim) pivotal trials and efficacy data

STUDY DESIGN

Pivotal trials study design^1,2

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 definitions^1,2

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 response^1,2

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 dose^4,*
- 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
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 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
Watch an expert discuss personalized Nplate^® dosing for ITP control.¹ Click here

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 therapies^1,2

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 therapy^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

*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 response^1,2

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 response^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

Onset of response

In the pivotal trials (N = 125)¹

30% of patients treated with Nplate^® responded after 1 dose^2,‡
- 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 trial^1,2

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
study^1,2

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 therapies^1,2

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 therapy^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

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

EXPERT VIDEOS

These physicians choose Nplate^® for chronic ITP in adults

Watch Drs Steven Fein, Michael Tarantino, and Terry Gernsheimer discuss why Nplate^® is right for their chronic ITP patients

SAFETY

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

NEXT Discover more about Nplate^® long-term data

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.

INDICATIONS