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Atlas / Disease / Acquired thrombotic thrombocytopenic purpura

Acquired thrombotic thrombocytopenic purpura

disease
On this page

Also known as acquired ADAMTS13 deficiencyacquired TTPautoimmune thrombotic thrombocytopenic purpuraidiopathic thrombotic thrombocytopenic purpuraMoschowitz syndromepurpura, thrombotic thrombocytopenicTTP

Summary

Acquired thrombotic thrombocytopenic purpura (MONDO:0019740) is a disease and 16 clinical trials. Top therapeutic interventions include caplacizumab, apadamtase alfa, and imlifidase. A subtype of acquired thrombocytopenia — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

  • Prevalence: Unknown (Worldwide) [Orphanet-validated]
  • Phenotypes (HPO): 41
  • Clinical trials: 16

Clinical features

Epidemiology

Prevalence records

1 prevalence record(s), Orphanet:

TypeClassValueGeographyValidation
Annual incidence1-9 / 1 000 000EuropeValidated

Signs & symptoms

Clinical features (HPO)

41 HPO clinical features (Orphanet curated; top 41 by frequency):

HPO IDTermFrequency
HP:0000979PurpuraVery frequent (80-99%)
HP:0001873ThrombocytopeniaVery frequent (80-99%)
HP:0001937Microangiopathic hemolytic anemiaVery frequent (80-99%)
HP:6000462Anti-ADAMTS13 antibody positivityVery frequent (80-99%)
HP:0000093ProteinuriaFrequent (30-79%)
HP:0000790HematuriaFrequent (30-79%)
HP:0001250SeizureFrequent (30-79%)
HP:0001259ComaFrequent (30-79%)
HP:0001289ConfusionFrequent (30-79%)
HP:0001297StrokeFrequent (30-79%)
HP:0001324Muscle weaknessFrequent (30-79%)
HP:0001923ReticulocytosisFrequent (30-79%)
HP:0001945FeverFrequent (30-79%)
HP:0001981SchistocytosisFrequent (30-79%)
HP:0002014DiarrheaFrequent (30-79%)
HP:0002018NauseaFrequent (30-79%)
HP:0002027Abdominal painFrequent (30-79%)
HP:0002094DyspneaFrequent (30-79%)
HP:0002315HeadacheFrequent (30-79%)
HP:0002829ArthralgiaFrequent (30-79%)
HP:0002904HyperbilirubinemiaFrequent (30-79%)
HP:0003326MyalgiaFrequent (30-79%)
HP:0003419Low back painFrequent (30-79%)
HP:0008282Unconjugated hyperbilirubinemiaFrequent (30-79%)
HP:0012378FatigueFrequent (30-79%)
HP:0020181Reduced haptoglobin levelFrequent (30-79%)
HP:0025435Increased circulating lactate dehydrogenase concentrationFrequent (30-79%)
HP:0033404Intestinal ischemiaFrequent (30-79%)
HP:0410173Increased circulating troponin I concentrationFrequent (30-79%)
HP:0000651DiplopiaOccasional (5-29%)
HP:0001635Congestive heart failureOccasional (5-29%)
HP:0001645Sudden cardiac deathOccasional (5-29%)
HP:0001658Myocardial infarctionOccasional (5-29%)
HP:0001919Acute kidney injuryOccasional (5-29%)
HP:0002381AphasiaOccasional (5-29%)
HP:0003115Abnormal EKGOccasional (5-29%)
HP:0003138Increased blood urea nitrogenOccasional (5-29%)
HP:0003259Elevated circulating creatinine concentrationOccasional (5-29%)
HP:0011675ArrhythmiaOccasional (5-29%)
HP:0030149Cardiogenic shockOccasional (5-29%)
HP:0100749Chest painOccasional (5-29%)

Identifiers

Disease identifiers

FieldValue
Canonical nameacquired thrombotic thrombocytopenic purpura
Mondo IDMONDO:0019740
MeSHC536901
Orphanet93585
NCITC131653
SNOMED CT438476003
UMLSC2584778
MedGen391723
GARD0004607
Is cancer (heuristic)no

Also known as: acquired ADAMTS13 deficiency · acquired thrombotic thrombocytopenic purpura · acquired TTP · autoimmune thrombotic thrombocytopenic purpura · idiopathic thrombotic thrombocytopenic purpura · Moschowitz syndrome · purpura, thrombotic thrombocytopenic · TTP

Disease family

This is a subtype of acquired thrombocytopenia. Genetic, therapeutic, and trial evidence is largely curated at the broader-term level — see the parent page for the associated-gene cohort and molecular evidence.

Classification path: disease › human disease › disease by body system or component › hematologic disorder › blood platelet disease › thrombocytopenia › acquired thrombocytopenia › acquired thrombotic thrombocytopenic purpura

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

No tiered GWAS variants or ClinVar records for this disease.

Genes & proteins

No associated-gene cohort resolved for this disease. Atlas builds the molecular and therapeutic sections — associated genes, protein families, druggability, pathways, interactions, and drug associations — by aggregating over a disease’s associated genes (resolved via GWAS / GenCC / ClinVar / CIViC), and none resolved here. This is expected for antibody-mediated, autoimmune, or otherwise non-gene-defined conditions; the curated evidence for this disease is its clinical features, GWAS susceptibility, and clinical trials (above).

Function

No pathway enrichment — requires an associated-gene cohort.

Therapeutics

No druggable-target or therapeutic data for this disease’s cohort.

Clinical trials & evidence

Clinical trials

Clinical trials: 16.

Phase distribution (across all retrieved trials)

PhaseTrials
PHASE26
Not specified4
PHASE33
PHASE42
EARLY_PHASE11

Top trials by phase / activity

NCTPhaseStatusTitle
NCT01754545PHASE4WITHDRAWNProphylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura
NCT04767828PHASE4UNKNOWNA Single Arm Study of Brain Metastasis in Patients With HER2-positive Breast Cancer
NCT01433003PHASE3WITHDRAWNThe Plasma Large-Volume Exchange RCT
NCT02553317PHASE3COMPLETEDPhase III Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura
NCT02878603PHASE3COMPLETEDFollow-up Study for Patients Who Completed Study ALX0681-C301 (Post-HERCULES)
NCT06794008PHASE2RECRUITINGBCMA-CD19 CAR-T Therapy for Refractory Autoimmune Diseases
NCT00632242PHASE2COMPLETEDARC1779 Injection in Patients With Von Willebrand Factor-Related Platelet Function Disorders
NCT01151423PHASE2COMPLETEDStudy to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
NCT02854059PHASE2TERMINATEDIdeS in Asymptomatic Antibody-Mediated Thrombotic Thrombocytopenic Purpura (TTP) Patients
NCT03922308PHASE2COMPLETEDStudy of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
NCT04021173PHASE2UNKNOWNA Clinical Study of Anfibatide in Acquired Thrombotic Thrombocytopenic Purpura (TTP)
NCT01808521EARLY_PHASE1COMPLETEDA Pilot Study of N-acetylcysteine in Thrombotic Thrombocytopenia Purpura
NCT04985318Not specifiedACTIVE_NOT_RECRUITINGRetrospective Analysis of the Efficiency of Caplacizumab in the Treatment of aTTP
NCT05004493Not specifiedRECRUITINGBiorepository and Registry for Plasma Exchange Patients
NCT05046717Not specifiedACTIVE_NOT_RECRUITINGImprovement of Immunologic and Molecular Techniques for the Diagnosis and Follow-up of Patients With Thrombotic Thrombocytopenic Purpura
NCT05876221Not specifiedCOMPLETEDPlatelet Response to Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura

Drugs tested across these trials (top 30)

MoleculeMax phaseTrials referencing
CAPLACIZUMAB44
APADAMTASE ALFA41
IMLIFIDASE41
ANFIBATIDE21
EGAPTIVON PEGOL21
CHEMBL543550001

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.4
BioBTree
v2.0.2

Sources 20

This page pulls from 20 datasets indexed by BioBTree:

chembl_moleculecivic_evidenceclinical_trialsclinvarefogardgenccgwasgwas_studyhgncmedgenmeshmimmondomondochildmondoparentncitorphanetsctidumls