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.

... Read More 
Health-Related Quality of Life data

Patients treated with Nplate® experienced changes in Health-Related Quality of Life (HRQoL) domains compared to placebo1


Patient questionnaire assessed issues specific to patients with immune thrombocytopenia (ITP)1

The ITP-patient assessment questionnaire (ITP-PAQ) is the first disease-specific HRQoL questionnaire developed for use in adults with chronic ITP. The domains listed below investigate the impact of ITP on a patient’s functioning and well-being.1

Review the ITP-PAQ and its questions. View here 

In a post hoc analysis, patients treated with Nplate® experienced changes in HRQoL domains1

ITP Patient Assessment Questionnaire (ITP-PAQ) mean change scores from
baseline for non-splenectomized patients (n = 61)1,*,†





ITP Patient Assessment Questionnaire (ITP-PAQ) mean change scores from
baseline for splenectomized patients (n = 58)1,*,†

These data come from an analysis of a secondary descriptive endpoint from the pivotal trials that examined the change from baseline of scores from a validated Patient Reported Outcome (PRO) instrument over a 24-week period.1

  • The PRO instrument—ITP patient assessment questionnaire (ITP-PAQ)—is a validated, specialized HRQoL questionnaire to investigate the impact of ITP on a patient’s functioning and well-being in the 10 domains noted in the graphs above1
  • Patients self-administered the ITP-PAQ at baseline (before Nplate® or placebo) and at weeks 4, 12, and 24. Patients were blinded to their current platelet count results before completing the ITP-PAQ1
  • Important consideration: This analysis was not powered to show a treatment effect and contains data from a secondary descriptive endpoint1

In pooled data of the post-hoc analysis, patients treated with Nplate® experienced changes in HRQoL domains1

Mean ITP-PAQ score change from baseline to week 24 comparing
Nplate® (n = 84) vs placebo (n = 41) in all patients1,*,†

In pooled data of splenectomized and non-splenectomized patients compared with placebo, patients treated with Nplate® experienced changes in HRQoL domains.1

  • Important consideration: This analysis was not powered to show a treatment effect and contains data from a secondary descriptive endpoint1

*Placebo comparison may limit real-world comparison.

Length of disease may influence the change in quality of life.

Overall quality of life was one of the domains and not a composite endpoint of the other domains.1,2

Immune thrombocytopenia patient assessment questionnaire (ITP-PAQ)

In the past 4 weeks, ...

  • How much have your ITP symptoms or the effects of its treatments interfered with your ability to exercise?
  • To what extent has having ITP limited the types of physical or sporting activities you participate in?
*5-point scale from extremely to not at all.

In the past 4 weeks, ...

  • How often did you feel physically unattractive due to bruising, scarring, wounds, or the effects of ITP medications?*
  • Overall, to what extent have ITP and its treatments affected your physical health?
  • Overall, how bothered have you been by the effect of ITP and its treatments on your physical health?
*5-point scale from all the time to never. 7-point scale from extremely to not at all.

In the past 4 weeks, how often did ITP or its treatments...

  • Cause you to have difficulty falling asleep at bedtime?
  • Cause you to awaken during the night?
  • Cause you to feel sleepy during the daytime?
  • Cause you to feel physically fatigued?
*5-point scale from all the time to never

In the past 4 weeks, ...

  • How fearful have you been of having a bleeding episode (nose bleeds, gum bleeds, etc)?
  • How fearful have you been of death or dying?
  • How fearful have you been of being too far away from your doctor in case you needed medical help?
  • How fearful have you been about getting an infection?
  • How fearful have you been of needing to have emergency surgery (due to concerns about bleeding during surgery)?
*5-point scale from extremely fearful to not at all fearful.
  • How much has having ITP made it less likely that you would get pregnant?
  • How much has having ITP made it less likely that you would give birth?
  • How much has having ITP made it less likely that you would adopt?
*5-point scale from extremely to not at all. Questions completed by women only.

Thinking about your last period, ...

  • How bothered were you by heavier bleeding than before having ITP?
  • How bothered were you by bleeding for more days than before having ITP?
  • How bothered were you by more pain than before having ITP?
*5-point scale from extremely to not at all. Questions completed by women only.

In the past 4 weeks, ...

  • Overall, to what extent have ITP and its treatments affected your quality of life?*
  • Overall, how bothered have you been by the effect of ITP and its treatments on your quality of life?*
  • I have made significant changes to my lifestyle because I have ITP.
  • My ITP prevents me from doing things in life that I want to do.
  • My ITP prevents my spouse, partner, or family members from doing things in life that they want to do.
*7-point scale from extremely to not at all. 4-point scale from strongly agree to strongly disagree.

In the past 4 weeks, ...

  • How often did you feel like you had no control over your health because of your ITP or its treatments?*
  • How often did you feel like you were unable to manage stress because of your ITP or its treatments?*
  • How often did you have feelings of sadness or depression because of your ITP or its treatments?*
  • Overall, how much has ITP or its treatments affected you psychologically (mental state, emotions)?
  • Overall, how bothered have you been by the effect that ITP or its treatments has had on you psychologically (mental state, emotions)?
*5-point scale from all the time to never. 5-point scale from extremely to not at all.

In the past 4 weeks, ...

  • How often has having ITP limited your ability to participate in social activities?*
  • How often have you avoided social activities to limit your exposure to infection?*
  • How bothered were you by what people might think about your bruising or scarring?
  • To what extent have you been unable to lead a normal social life because of your ITP?
*5-point scale from all the time to never. 5-point scale from extremely to not at all.

In the past 4 weeks, how often did you...

  • Have either bruising or petechiae (broken blood vessels)?
  • Have wounds or scars from blood tests, injections, or IV needles?
  • Have blood blisters in your mouth?
  • Have bleeding episodes (nose bleeds, gum bleeds, etc)?
  • Have muscle aches?
  • Have cramps in your legs?
*5-point scale from all the time to never

See Fertility and Menstrual symptoms.

Since you were diagnosed, ...

  • To what degree has ITP negatively interfered with your choice of careers?
  • How much has ITP negatively interfered with your ability to get a promotion at your job?
  • How much has ITP negatively interfered with your relationships with coworkers?
  • How fearful have you been of losing your job because of your ITP?
*5-point scale from extremely to not at all, with a not applicable option.

Immune thrombocytopenia patient assessment questionnaire (ITP-PAQ)

  • Activity

    In the past 4 weeks, ...

    • How much have your ITP symptoms or the effects of its treatments interfered with your ability to exercise?
    • To what extent has having ITP limited the types of physical or sporting activities you participate in?
    *5-point scale from extremely to not at all.
  • Bother3

    In the past 4 weeks, ...

    • How often did you feel physically unattractive due to bruising, scarring, wounds, or the effects of ITP medications?*
    • Overall, to what extent have ITP and its treatments affected your physical health?
    • Overall, how bothered have you been by the effect of ITP and its treatments on your physical health?
    *5-point scale from all the time to never. 7-point scale from extremely to not at all.
  • Fatigue/sleep3,*

    In the past 4 weeks, how often did ITP or its treatments...

    • Cause you to have difficulty falling asleep at bedtime?
    • Cause you to awaken during the night?
    • Cause you to feel sleepy during the daytime?
    • Cause you to feel physically fatigued?
    *5-point scale from all the time to never.
  • Fear3,*

    In the past 4 weeks, ...

    • How fearful have you been of having a bleeding episode (nose bleeds, gum bleeds, etc)?
    • How fearful have you been of death or dying?
    • How fearful have you been of being too far away from your doctor in case you needed medical help?
    • How fearful have you been about getting an infection?
    • How fearful have you been of needing to have emergency surgery (due to concerns about bleeding during surgery)?
    *5-point scale from extremely fearful to not at all fearful.
  • Fertility (subscale under Women’s reproductive health)3,*,†

    • How much has having ITP made it less likely that you would get pregnant?
    • How much has having ITP made it less likely that you would give birth?
    • How much has having ITP made it less likely that you would adopt?
    *5-point scale from extremely to not at all. Questions completed by women only.
  • Menstrual symptoms (subscale under women’s reproductive health )3,*,†

    Thinking about your last period, ...

    • How bothered were you by heavier bleeding than before having ITP?
    • How bothered were you by bleeding for more days than before having ITP?
    • How bothered were you by more pain than before having ITP?
    *5-point scale from extremely to not at all. Questions completed by women only.
  • Overall quality of life (QoL) 3,*

    In the past 4 weeks, ...

    • Overall, to what extent have ITP and its treatments affected your quality of life?*
    • Overall, how bothered have you been by the effect of ITP and its treatments on your quality of life?*
    • I have made significant changes to my lifestyle because I have ITP.
    • My ITP prevents me from doing things in life that I want to do.
    • My ITP prevents my spouse, partner, or family members from doing things in life that they want to do.
    *7-point scale from extremely to not at all. 4-point scale from strongly agree to strongly disagree.
  • Psychological3

    In the past 4 weeks, ...

    • How often did you feel like you had no control over your health because of your ITP or its treatments?*
    • How often did you feel like you were unable to manage stress because of your ITP or its treatments?*
    • How often did you have feelings of sadness or depression because of your ITP or its treatments?*
    • Overall, how much has ITP or its treatments affected you psychologically (mental state, emotions)?
    • Overall, how bothered have you been by the effect that ITP or its treatments has had on you psychologically (mental state, emotions)?
    *5-point scale from all the time to never. 5-point scale from extremely to not at all.
  • Social activity3

    In the past 4 weeks, ...

    • How often has having ITP limited your ability to participate in social activities?*
    • How often have you avoided social activities to limit your exposure to infection?*
    • How bothered were you by what people might think about your bruising or scarring?
    • To what extent have you been unable to lead a normal social life because of your ITP?
    *5-point scale from all the time to never. 5-point scale from extremely to not at all.
  • Symptoms3,*

    In the past 4 weeks, how often did you...

    • Have either bruising or petechiae (broken blood vessels)?
    • Have wounds or scars from blood tests, injections, or IV needles?
    • Have blood blisters in your mouth?
    • Have bleeding episodes (nose bleeds, gum bleeds, etc)?
    • Have muscle aches?
    • Have cramps in your legs?
    *5-point scale from all the time to never.
  • Women's reproductive health3

    See Fertility and Menstrual symptoms.

  • Work3,*

    Since you were diagnosed, ...

    • To what degree has ITP negatively interfered with your choice of careers?
    • How much has ITP negatively interfered with your ability to get a promotion at your job?
    • How much has ITP negatively interfered with your relationships with coworkers?
    • How fearful have you been of losing your job because of your ITP?
    *5-point scale from extremely to not at all, with a not applicable option.

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.

References: 1. George JN, Mathias SD, Go RS, et al. Improved quality of life for romiplostimtreated patients with chronic immune thrombocytopenic purpura: results from two randomized, placebo-controlled trials. Br J Haematol. 2009;144:409-415. 2. Mathias SD, Bussel JB, George JN, McMillan R, Okano GJ, Nichol JL. A disease-specific measure of health-related quality of life for use in adults with immune thrombocytopenia purpura: its development and validation. Health Qual Life Outcomes. 2007;5:11.

Back To Top

You are now leaving NplateHCP.com.

Please be aware that sponsors of this site are not responsible for content on the site you are about to enter.