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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Boost their platelet counts with Nplate® to reduce the risk of bleeding1

Nplate® is approved 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.

EFFICACY

7 out of 10 pediatric patients reached target platelet counts with Nplate®1,*

Phase 3 pediatric pivotal trial (N = 62)1,2

  • Patients were refractory or relapsed after at least one prior ITP therapy with a platelet count ≤ 30 × 109/L
  • Patients were stratified by age and randomized (2:1) to receive a starting dose of 1 mcg/kg subcutaneously weekly either Nplate® or placebo
    • Age categories were ≥ 1 to < 6 years, 6 to < 12 years, and 12 to < 18 years
  • Over a 24-week treatment period, dose was titrated up to a maximum of 10 mcg/kg weekly of either Nplate® or placebo in an effort to maintain a target platelet count of ≥ 50-200 × 109/L

Pediatric pivotal trial endpoints1,2

Primary Endpoint:
Incidence of durable platelet response
  • Weekly platelet responses (platelet count > 50 x 109/L)
Secondary Endpoint:
Overall platelet response
  • Defined as a durable or a transient platelet response
  • A transient platelet response waas the acheivement of any weekly platelet counts ≥ 50 x 109/L for any 4 weeks during the treatment without a durable platelet response

Platelet responses were excluded for 4 weeks after receiving rescue medications.

*Based on overall platelet response.

Platelet response in the 24-week
phase 3 pediatric trial1
Achieving and maintaining a platelet count
≥ 50 x 109/L can reduce the risk for bleeding1
Nplate® was studied in a long-term extension study of up to ~7.5 years in pediatric ITP*

Long-term extension trial study design (N = 66)

  • Pediatric subjects who had previously completed an Nplate® ITP study were eligible to participate in the study
  • Primary endpoints were incidence and exposure-adjusted incidence of adverse events, including clinically significant changes in laboratory values and incidence of antibody formation
  • Secondary endpoint was platelet response to Nplate®
  • Nplate® was administered weekly by subcutaneous injection
  • Consider long-term study design limitations when interpreting results. This study includes patients from multiple prior studies, is not blinded, not controlled, and includes inherent self-selection bias
  • *Median study duration was 2.8 years (N = 65).3

*Median study duration was 2.8 years (N = 65).3

    Long-term extension study

    • ~ 94% of pediatric patients responded to treatment with Nplate®†
    • 30-month median duration of response
    • ~ 7.5 years maximum platelet response

    Defined as one or more platelet count ≥ 50 x 109/L in the absence of rescue medication.

    Response defined as a median monthly platelet count ≥ 50 x 109/L.



    SAFETY RESULTS: The most frequently occurring (> 45%) adverse events were headache (58.5%), contusion (50.8%), epistaxis, upper respiratory tract infection and vomiting (49.2% each), cough and oropharyngeal pain (46.2% each). The most frequently reported (> 5%) grade 3 or 4 adverse events were thrombocytopenia (9.2%) and headache (6.2%).

    SAFETY

    Established safety profile across clinical trials

    Pediatric safety profile was established in a 12- and 24-week trial1,*

    Adverse reactions with an incidence of ≥ 25% in the 2 placebo-controlled trials were contusion, upper respiratory tract infection, and oropharyngeal pain.

    Common adverse reactions from 2 placebo-controlled pediatric studies1,4,†

    ADVERSE REACTIONS BY BODY SYSTEM
    NPLATE® (%)
    (N = 59)
    PLACEBO (%)
    (N = 24)
    Infections and Infestations
    Upper Respiratory Tract Infection
    18 (31%)
    6 (25%)
    Ear Infection
    3 (5%)
    0 (0.0%)
    Gastroenteritis
    3 (5%)
    0 (0.0%)
    Sinusitis
    3 (5%)
    0 (0.0%)
    Respiratory, Thoracic, and Mediastinal Disorders
    Oropharyngeal Pain
    15 (25%)
    1 (4%)
    Gastrointestinal Disorders
    Diarrhea
    12 (20%)
    3 (13%)
    Abdominal Pain Upper
    8 (14%)
    1 (4%)
    Skin and Subcutaneous Tissue Disorders
    Rash
    9 (15%)
    2 (8%)
    Purpura
    4 (7%)
    0 (0.0%)
    Urticaria
    3 (5%)
    0 (0.0%)
    General Disorders and Administration Site Conditions
    Pyrexia
    14 (24%)
    2 (8%)
    Peripheral Swelling
    4 (7%)
    0 (0.0%)
    Injury, Poisoning, and Procedural Complications
    Contusion
    24 (41%)
    8 (33%)

    *The 12-week trial was the phase 1/2 trial. The 24-week trial was the phase 3 trial.

    ≥ 5% incidence and ≥ 5% more frequent in the Nplate® arm.

    Phase 1/2 study (N = 22)

    Nplate® was studied in patients diagnosed with ITP at least 6 months prior to enrollment with a platelet count ≤ 30 × 109/L.

    • Patients were stratified by age and randomized (3:1) to receive (1 mcg/kg subcutaneously weekly) Nplate® or placebo1
    • Over a 12-week treatment period dose was titrated up to a maximum of 10 mcg/kg weekly of either Nplate® or placebo in an effort to maintain a target platelet count of ≥ 50-250 × 109/L
    No monitoring for hepatotoxicity is required on Nplate.
    REDUCED COMPLEXITY

    Reduce dietary complexity for your pediatric ITP patients

    In-office weekly administration allows you to closely monitor your patients’ treatment1

    No dietary restrictions

    • No calcium or other dietary restrictions
    • Does not inhibit iron absorption
    Patients can freely consume food products containing calcium such as cheese while on Nplate®. Patients can freely consume food products containing calcium such as cheese while on Nplate®.

    Achieve and maintain platelet count goals with weekly dosing and personalized titration1

    • Initial dose: 1 mcg/kg based on actual body weight1
    • Adjust dose based on platelet response and reassess body weight every 12 weeks1

    Refer to the dosing for more information and Prescribing Information for dilution instructions and additional Important Safety Information.

    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.

    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®

    References: 1. Nplate® (romiplostim) prescribing information, Amgen. 2. Tarantino M, Bussel J, Blanchette V, et al. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet. 2016;388:45-54. 3. Data on file, Amgen; Pediatric long-term trial clinical study report; 2017. 4. Tarantino M, Bussel J, Blanchette V, et al. Final safety and efficacy data of long-term open-label dosing of subcutaneous (SC) romiplostim in children with immune thrombocytopenia (ITP) [abstract]. Presented at the 59th Annual Meeting of the American Society of Hematology; December 9, 2017: Atlanta, GA.