TAK-771-4002: Evaluating the Safety of GAMMAGARD LIQUID for the Treatment of Patients With Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Non-randomized, active-comparator, new-user, retrospective study

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (J06BA02) immunoglobulins, normal human, for intravascular adm.

  immunoglobulins, normal human, for intravascular adm.
• Medical condition to be studied:
  Chronic inflammatory demyelinating polyradiculoneuropathy

Population studied

• Short description of the study population:
  The study population included adult patients aged ≥ 18 years with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) received treatment with intravenous immunoglobulin in the years 2008 through 2019 identified from IBM MarketScan Research Databases, and Optum Clinformatics Data Mart.; Inclusion criteria; To be eligible for inclusion in either study cohort:; • Have a minimum of 6 months of continuous enrollment in the study database with medical and pharmacy coverage before the index date (to accurately define patient characteristics). Gaps in continuous enrollment ≤ 31 days are permitted.; • Fulfill the CIDP diagnosis algorithm on or before the index date using all available baseline data for each patient.; To be eligible for the Ig-naive (new-to-class) cohort:; • Be free of any previous recorded use of any Ig product (i.e., study IVIG products, nonstudy IVIG products, or subcutaneous Ig products) at any point before IVIG initiation; To be eligible for the Ig-experienced (new-to-drug) cohort:; • Have any previous recorded use of an Ig product (i.e., study IVIG products, nonstudy IVIG products, or subcutaneous Ig products) at any point before the index date; Exclusion criteria:; Patients in both study cohorts will be excluded if they fulfill any of the following exclusion criteria:; • Having claims for ≥ 2 different IVIG products on the index date, as accurate categorization of the index IVIG product would not be possible; • Recorded diagnosis of any of the following conditions on or before the index date, to reduce the potential for misclassification of CIDP status among patients using IVIG; – PID, as PID is an approved indication for treatment with GGL; – Evidence of secondary immunodeficiency (SID), including patients with recorded diagnoses of hematological malignancy (e.g., diagnosis of multiple; myeloma or chronic lymphocytic leukemia) or treatment with rituximab, as short courses of IVIG may be used for SID treatment; – Idiopathic thrombocytopenic purpura (ITP)
• 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)
• Special population of interest:
  Other
• Special population of interest, other:
  Chronic inflammatory demyelinating polyradiculoneuropathy patients
• Estimated number of subjects:
  7000

Study design details

• Main study objective:
  The objective is to study to evaluate the rates of adverse events of special interest (AESIs) (thrombotic events, acute kidney injury AKI, and hemolytic events) among participants with CIDP initiating GGL compared with rates among participants with CIDP initiating comparator intravenous immunoglobulin (IVIG) products.
• Outcomes:
  1. Hazard ratio of Thrombotic Events (TEs) between GGL and comparator IVIG products 2. Hazard ratio of Acute kidney injury (AKI) between GGL and comparator IVIG products 3. Hazard ratio of Hemolytic Events (HEs) between GGL and comparator IVIG products, 1. Hazard ratio of Anaphylaxis between GGL and comparator IVIG products 2. Hazard ratio of Transfusion-related Acute Lung Injury (TRALI) between GGL and comparator IVIG products 3. Hazard ratio of transfusion-associated Circulatory Overload (TACO) between GGL and comparator IVIG products
• Data analysis plan:
  All analyses will be performed separately in the 2 data sources (MarketScan Research Databases, Optum Clinformatics Data Mart) and the data source-specific results will be reported separately. Pooling of the final results across data sources will be performed, if appropriate. Within each data source, the 2 cohorts (Ig naive and Ig experienced) will be analyzed and reported separately, except in secondary analyses combining the 2 cohorts.

Documents

Study results

TAK-771-4002-clinical-study-report-redact