Top ▲

Cold agglutinin disease

GtoPdb Disease Summaries

This section gives an overview of the disease, and where available shows the following:

  • Synonyms: Shows known synonyms for the disease.
  • Description: Gives a basic description/definition of the disease.
  • Database Link: External links to the same disease at the Disease Ontology, OMIM or Orphanet sites.
  • Immunopharmacology comments: General comments about the target's role in immunopharmacology, provided by GtoImmuPdb curators.
  • Associated with: Counts are displayed for the total targets the disease is associated with in GtoPdb. The counts of targets and ligands of immunological relevance associated to the disease are also shown.

More information can be found in the help pages.

✖
Disease ID:1253
Name:Cold agglutinin disease
Associated with:0 target
1 immuno-relevant ligand
Synonyms
autoimmune hemolytic anemia | chronic cold agglutinin disease | cold agglutinin syndrome
Description
Cold agglutinin disease is an autoimmune hemolytic anemia caused by autoantibodies that destroy erythrocytes via activation of the C1-complex of the classical complement pathway. The disease causes chronic anemia, severe fatigue and potentially fatal thrombotic events.
Database Links
Orphanet: ORPHA56425

Targets

GtoPdb Disease Summaries - Targets

Click on the target name to link to its detailed view page

Where available, information is display on the role of the target in the disease; drugs which target the disease and their therapeutic use and side-effects.

If there is mutation data curated in GtoPdb this is indicated, with a link back to the appropriate section on the target detailed view page

Immuno ligand interactions - If available, a table of immuno-relevant ligands is shown. These ligands have been curated as having an association to the disease and possess interaction data with the target in GtoPdb. The approval status of the ligand is shown, along with curator comments and an indication of whether the target is considered the primary target of the ligand.

More information can be found in the help pages.

✖
Key to terms and symbols

No target related data available for Cold agglutinin disease

Ligands

GtoPdb Disease Summaries - Ligands

Click ligand name to view ligand summary page

  • Approved: If the ligand is an approved drug this is indicated, along with approval bodies.
  • Immuno: Immuno icon indicates the ligand is immuno-relevant

Click the arrow in the final column to expand comments

  • Immuno Disease Comments: Curatorial comments specifically added as part of GtoImmuPdb. They give more information on the association between the ligand and disease in the context of immunopharmacology.
  • Clinical Use: General clinical comments relating to the ligand and may not necessarily be specific to the disease in question. With hyperlink to more details on the ligand summary pages.
  • Bioactivty Comments: Curatorial comments specifically about the compounds biological activity - with hyperlink to more details on the ligand summary pages.

More information can be found in the help pages.

✖
Key to terms and symbols Click ligand name to view ligand summary Click column headers to sort
Ligand References Clinical and Disease comments
sutimlimab
Immuno Disease Comments: TNT009 is a first-in-class C1-complex-targeting mAb, that is hoped to provide an effective treatment for CAD and other complement-mediated disorders. It is a Phase 3 clinical candidate for CAD (see NCT03347396) and Phase 1 for other disorders (see NCT02502903 and NCT03275454). TNT009 is an FDA and EMA orphan drug for the treatment of hemolysis in patients with primary CAD.
Clinical Use: Click here to link to ClinicalTrials.gov's list of current TNT009 (and BIVV009) trials. In May 2020 the FDA granted priority review of sutimlimab for the treatment of complement C1-activated hemolysis in adult patients with cold agglutinin disease (CAD). In February 2022 the FDA authorised sutimlimab as the first and only approved treatment for the prevention of hemolysis in CAD patients. | View clinical data
Bioactivity Comments: Preclinical details of TNT009 in vitro activity are published in [3], and results from the first-in-human Phase 1 trial are reported in [2]. In kidney transplant patients, TNT009 (BIVV009) effectively blocks alloantibody-triggered complement pathway activation, but this fails to translate to therapeutic benefit in the short term [1]. Further studies are needed to fully evaluate the therapeutic value of complement pathway blockade in transplant patients. | View biological activity

References

Show »

1. Eskandary F, Jilma B, Mühlbacher J, Wahrmann M, Regele H, Kozakowski N, Firbas C, Panicker S, Parry GC, Gilbert JC et al.. (2018) Anti-C1s monoclonal antibody BIVV009 in late antibody-mediated kidney allograft rejection-results from a first-in-patient phase 1 trial. Am J Transplant, 18 (4): 916-926. [PMID:28980446]

2. Mühlbacher J, Jilma B, Wahrmann M, Bartko J, Eskandary F, Schörgenhofer C, Schwameis M, Parry GC, Gilbert JC, Panicker S et al.. (2017) Blockade of HLA Antibody-Triggered Classical Complement Activation in Sera From Subjects Dosed With the Anti-C1s Monoclonal Antibody TNT009-Results from a Randomized First-in-Human Phase 1 Trial. Transplantation, 101 (10): 2410-2418. [PMID:28926521]

3. Wahrmann M, Mühlbacher J, Marinova L, Regele H, Huttary N, Eskandary F, Cohen G, Fischer GF, Parry GC, Gilbert JC et al.. (2017) Effect of the Anti-C1s Humanized Antibody TNT009 and Its Parental Mouse Variant TNT003 on HLA Antibody-Induced Complement Activation-A Preclinical In Vitro Study. Am J Transplant, 17 (9): 2300-2311. [PMID:28251805]