Help restore stability: raise and sustain platelet counts

Pivotal trials study design1,2

In the two parallel, double-blind, phase 3 trials, patients with chronic immune thrombocytopenia (ITP) who had completed at least one prior treatment* and had a platelet count of ≤ 30 x 109/L prior to study entry 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 definitions1,2

Primary endpoint

Secondary endpoint

platelet response
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
  • 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.2 Read more >

Help restore stability:
achieve and maintain target platelet counts

Nplate®: Significant platelet response across two 6-month pivotal trials1,2

non-splenectomized patients
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

Help restore stability:
raise and sustain platelet counts

Nplate®: Sustained median platelet count 50 x 109/L throughout 6-month pivotal trials1,2

non-splenectomized patients
non-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

  • Comparable results were seen in splenectomized patients.2

Review the most common adverse events from the Nplate® pivotal trials.1 Learn more >

Nplate® or SOC trial and results3

Trial design

The incidence of treatment failure and splenectomy (co-primary endpoints), as well as patient safety, were evaluated in a multicenter, randomized, controlled, 52-week, open-label evaluation of Nplate® and medical SOC therapy for non-splenectomized patients with ITP. Concomitant therapies were allowed for both treatment groups in this study.

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

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.

Therapies used in SOC arm (percentage of patients)3

SOC Therapies
SOC Therapies

Co-primary endpoints:

1. Incidence of treatment failure3

Study discontinuation before treatment failure was also considered treatment failure.

Treatment Failure
Treatment Failure

2. Incidence of splenectomy3

Study discontinuation before splenectomy was also considered splenectomy.

Trial results

Nearly 90% of non-splenectomized patients in the Nplate® arm avoided treatment failure.3

Incidence of treatment failure at 1 year3

Incidence of Treatment
Incidence of Treatment

For Nplate®, the percentage of patients 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 the most common Adverse Events from the Nplate® pivotal trials.3 Read more >

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

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.1

Rituximab is not FDA approved for use in ITP.

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

The study also counted splenectomy as a treatment.2

Important Safety Information

Risk of Progression of Myelodysplastic Syndromes to Acute Myelogenous Leukemia

  • In Nplate® 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®

Laboratory Monitoring

Adverse Reactions


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.