Study type

Study topic

Human medicinal product

Study type

Non-interventional study

Scope of the study

Assessment of risk minimisation measure implementation or effectiveness
Disease epidemiology
Safety study (incl. comparative)

Data collection methods

Secondary use of data
Non-interventional study

Non-interventional study design

Cohort
Other

Non-interventional study design, other

Post-marketing safety surveillance
Study drug and medical condition

Name of medicine

NPLATE

Study drug International non-proprietary name (INN) or common name

ROMIPLOSTIM

Anatomical Therapeutic Chemical (ATC) code

(B02BX04) romiplostim
romiplostim
Population studied

Short description of the study population

The study population includes chronic ITP patients, defined as patients who have been diagnosed with ITP for more than 6 months, and who continually receive ITP treatment (eg, ITP medication) and/or have had a splenectomy procedure. All qualified adult chronic ITP patients will be the source population, regardless of romiplostim therapy status.
Inclusion Criteria
Subjects in the NCPRR will be those whose health information is recorded by the NHRS of Denmark, Norway, or Sweden.
The eligible adult cITP romiplostim-exposed patients for the NCPRR must meet all of the following criteria:
• Patients must be 18 years or older at the time of chronic ITP diagnosis,
• Patients will have romiplostim therapy between 01 January 2009 and 31 December 2018, and
• Patients will have at least 6 months of medical information prior to initial date of receiving romiplostim treatment (Index Date), data from which will establish a
baseline of study subjects.
The eligible adult cITP romiplostim-unexposed patients for the NCPRR must meet all following criteria:
• Patients must be 18 years or older at the time of chronic ITP diagnosis,
• Patients will not have received romiplostim therapy prior to or during the study period between 01 January 2009 and 31 December 2019, and
• Patients will have at least 6 months of medical information in the study period. The data from this period will establish baseline of subjects in the comparison group.
Exclusion Criteria
Patients who are enrolled in a clinical trial for other TPO receptor agonists (eg, clinical trials for eltrombopag) will be excluded from the study.
Patients are excluded from the cohorts of chronic ITP patients if either of the following is present:
1. Any diagnosis of another condition associated with thrombocytopenia other than chronic ITP listed in Appendix 2 at any time prior to or within 6 months after the qualifying diagnosis for chronic ITP. Examples of thrombocytopenia associated with other diseases and conditions include system

Age groups

Adults (18 to < 46 years)
Adults (46 to < 65 years)
Adults (65 to < 75 years)
Adults (75 to < 85 years)
Adults (85 years and over)

Estimated number of subjects

623
Study design details

Main study objective

The overarching aim of the study is to characterize the long-term safety profile of romiplostim therapy in adult chronic ITP patients.

Outcomes

To estimate the incidence rate of increased bone marrow (BM) reticulin and/or BM fibrosis with associated clinical signs (any of the following: splenomegaly, hepatomegaly, leukocytosis, and/or, cytopenia), confirmed by BM biopsy findings of adults with chronic ITP receiving romiplostim, Describe worsened thrombocytopeniaStudy incidence rate of thrombocytosisDescribe incidence rate of thrombotic eventsAssess incidence rate of hematological malignancies/pre-malignant statesDescribe bleeding and/or use of rescue medicationDescribe incidence rate of concurrent leukocytosis/anemiaDescribe safety in patients with renal impairmentMeasure impact of EU dosing

Data analysis plan

The study is descriptive in nature. Number and frequency distributions of patients who experience events will be reported. Rates of occurrence of events over available follow-up time will be reported.
Documents
Study results
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