Nordic Country Patient Registry for Romiplostim (NCPRR): Population-Based Prospective Annual Assessment of Safety of Romiplostim Treatment in Adult Patients with Chronic Idiopathic (Immune) Thrombocytopenic Purpura (ITP) Based on National Health Registry Systems in Denmark, Sweden and Norway (20070797)

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

20070797_Executive Summary_04JAN2022