Disease

Thrombocytopenia 4

Also known as CYCS thrombocytopenia, thrombocytopenia caused by mutation in CYCS, thrombocytopenia type 4, THC4+1 more

CYCS thrombocytopenia, thrombocytopenia caused by mutation in CYCS, thrombocytopenia type 4, THC4, thrombocytopenia, autosomal dominant, 4.

Associated genes

Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

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Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies

Eltrombopag olamineapproved

Approval — Eltrombopag Viatris is indicated for the treatment of adult patients with primary immune… (2024)

Eltrombopagapproved

Approval — Revolade is indicated for the treatment of adult patients with primary immune thrombocyto… (2010)

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals

2024emaApproval — Eltrombopag olamine· Eltrombopag Viatris is indicated for the treatment of adult patients with primary immune thrombocytopenia (ITP) who are refractory to other treatments (e.g. corticosteroids, immunoglobulins).Eltrombopag Viatris is indicated for the treatment of paediatric patients aged 1 year and above with primary immune thrombocytopenia (ITP) lasting 6 months or longer from diagnosis and who are refractory to other treatments (e.g. corticosteroids, immunoglobulins) (see sections 4.2 and 5.1).Eltrombopag Viatris is indicated in adult patients with chronic hepatitis C virus (HCV) infection for the treatment of thrombocytopenia, where the degree of thrombocytopenia is the main factor preventing the initiation or limiting the ability to maintain optimal interferon-based therapy (see sections 4.2 and 5.1). source ↗

2010emaApproval — Eltrombopag· Revolade is indicated for the treatment of adult patients with primary immune thrombocytopenia (ITP) who are refractory to other treatments (e.g. corticosteroids, immunoglobulins) (see sections 4.2 and 5.1). Revolade is indicated for the treatment of paediatric patients aged 1 year and above with primary immune thrombocytopenia (ITP) lasting 6 months or longer from diagnosis and who are refractory to other treatments (e.g. corticosteroids, immunoglobulins) (see sections 4.2 and 5.1). Revolade is indicated in adult patients with chronic hepatitis C virus (HCV) infection for the treatment of thrombocytopenia, where the degree of thrombocytopenia is the main factor preventing the initiation or limiting the ability to maintain optimal interferon-based therapy (see sections 4.4 and 5.1). Revolade is indicated in adult patients with acquired severe aplastic anaemia (SAA) who were either refractory to prior immunosuppressive therapy or heavily pretreated and are unsuitable for haematopoietic stem cell transplantation (see section 5.1). source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

CYCS0.75 ARHGEF30.46 TPM40.46 GCSAML0.43 THPO0.41 BAK10.37 SIRPA0.34 HBS1L0.31 AK30.28 GTF3C50.28 S1PR30.26 RCL10.26

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Sphingosine 1-phosphate receptor 3via S1PR30.26

Reference

Authoritative identity, definition & identifiers.

Synonyms

CYCS thrombocytopenia, thrombocytopenia caused by mutation in CYCS, thrombocytopenia type 4, THC4, thrombocytopenia, autosomal dominant, 4

References & data sources

Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
Related entities are derived from literature co-mention (studied together) — associative, not causal.