Nplate® is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in adult patients with immune... Read More
In pediatric ITP: For one less thing that may keep your patients from doing what they love
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.
7 out of 10 pediatric patients reached target platelet counts with Nplate®1,*
Phase 3 pediatric pivotal trial (N = 62)1,2
Nplate® was studied vs placebo in a double-blind, 24-week, multicenter, randomized, phase 3 study in pediatric patients 1 year to 17 years with immune thrombocytopenia (ITP).
- 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 < 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
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 × 109/L can reduce the risk for bleeding.1
Nplate® was studied in a long-term extension study of up to ~7.5 years in pediatric ITP3,*
Long-term extension trial study design (N = 66)4:
- 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 study3,4
- ~ 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%).
Hear from an expert pediatric hematologist
Watch why Dr. Michael Tarantino chooses Nplate® for second-line treatment in pediatric ITP patients.
Established safety profile across clinical trials1
Pediatric safety profile was established in a 12- and 24-week trial1,*
Adverse reactions with an incidence of ≥ 25% in the two placebo-controlled trials were contusion, upper respiratory tract infection, and oropharyngeal pain.1
Common adverse reactions from two placebo-controlled pediatric studies1,†
(N = 59)
(N = 24)
*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 last 6 months prior to enrollment with a platelet count ≤ 30 × 109/L.1
- Patients were stratified by age and randomized (3:1) to receive (1 mcg/kg subcutaneously weekly) Nplate® or placebo.1
- 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.1
No routine monitoring of hepatotoxicity required
Reduce 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
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
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 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 (1‑800‑772‑6436). 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.
- 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.
- 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%).
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.
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.
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.