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ORPHA:93585

Immune-mediated thrombotic thrombocytopenic purpura

low confidenceSubtype of disorder

Also known as: Acquired TTP · Acquired thrombotic thrombocytopenic purpura · Autoimmune thrombotic thrombocytopenic purpura · Thrombotic thrombocytopenic purpura due to anti-ADAMTS-13 antibodies · aTTP · iTTP

Publications

2,056

Trials

14

Interventional, condition-specific

Researchers

1,066

Distinct authors in sample

Gene link

—

Readiness

2/6

Stages with a signal

Clinical definition (Orphanet)

A rare, non- thrombotic thrombocytopenic purpura (TTP), characterized by profound peripheral thrombocytopenia, microangiopathic hemolytic anemia (MAHA) and single or multiple organ failure of variable severity.

How rare: How common this is has not been clearly measured.

Orphanet entry

Cross-references

Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.

Additional Mondo synonyms (4)

acquired ADAMTS13 deficiency · acquired TTP · acquired thrombotic thrombocytopenic purpura · autoimmune thrombotic thrombocytopenic purpura

Research stages

Trial readiness signals

Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”

2/6 stages with a signal

An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.

  1. Gene identifiedNot found

    No GenCC disease–gene assertion in this build

  2. LiteraturePresent

    2,056 matched papers (1,502 in last 10 years) Source

  3. Phenotype characterisedNot checked

    Not yet enriched from Monarch / HPO

  4. Animal modelNot checked

    Not yet enriched from Monarch / Alliance

  5. Orphan designationNot checked

    FDA/EMA orphan-drug designation not enriched yet

  6. Interventional trialPresent

    14 matched on ClinicalTrials.gov (4 recruiting in sample)

Biology

Genes and phenotypes

Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.

Do we know what causes it?

Not yet — the cause hasn't been pinned down in GenCC.

No strong gene–disease assertion joined for this Orphanet entity.

Phenotypes (Monarch / HPO)

Not enriched in this build — Monarch phenotype joins were not run for this record.

Animal models (Monarch / Alliance)

Not enriched in this build.

Literature

Is anyone studying this?

2,056

2,056 papers have been published on this condition. That is a real research literature — still far smaller than common diseases (breast cancer: over 700,000 papers). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=5449) is 41.

2,056 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 41 (publications denominator n=5449).

1,502 in the last 10 years · low confidence

Phrase hits: 2,055 · MeSH hits: 3

Open Europe PMC search

Who's working on it?

1,066

Distinct author names in 200 sampled papers — named people below.

Who's working on it?

People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.

  1. 01
    Coppo P32 papers · 2026

    Service d'hématologie, Hôpital Saint-Antoine, Assistance Publique - Hôpitaux de Paris, Paris, France.

    Papers in Europe PMC
  2. 02
    Veyradier A22 papers · 2026

    Service d'Hématologie biologique, Hôpital Lariboisière, Assistance Publique-Hôpitaux de Paris and EA3518, Institut de Recherche Saint Louis, Université de Paris, Paris, France.

    Papers in Europe PMC
  3. 03
    Joly BS15 papers · 2026

    Service d'Hématologie biologique, Hôpital Lariboisière, Assistance Publique-Hôpitaux de Paris and EA3518, Institut de Recherche Saint Louis, Université de Paris, Paris, France.

    Papers in Europe PMC
  4. 04
    Sakai K14 papers · 2026

    Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan.

    Papers in Europe PMC
  5. 05
    Matsumoto M13 papers · 2026

    Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan.

    Papers in Europe PMC
  6. 06
    Peyvandi F13 papers · 2026

    Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy; Università degli Studi di Milano, Department of Pathophysiology and Transplantation, and Fondazione Luigi Villa, Milan. flora.peyvandi@unimi.it.

    Papers in Europe PMC
  7. 07
    Vanhoorelbeke K13 papers · 2026

    Laboratory for Thrombosis Research, IRF Life Sciences, KU Leuven Campus Kulak Kortrijk, Kortrijk, Belgium.

    Papers in Europe PMC
  8. 08
    Poullin P10 papers · 2026

    Centre de Référence des Microangiopathies Thrombotiques (CNR-MAT) AP-HP Paris France.

    Papers in Europe PMC
  9. 09
    Artoni A9 papers · 2026

    Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan.

    Papers in Europe PMC
  10. 10
    Bouzid R9 papers · 2026

    INSERM UMRS-1138, Centre de Recherche des Cordeliers, CNRS, Sorbonne Université, Université Paris Cité, Paris, France.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

14

interventional trials for this specific condition

14 interventional trials matched this specific condition name; 4 currently recruiting in our sample. 21 trials are registered for thrombotic thrombocytopenic purpura, the broader category — shown separately because they may or may not enrol this specific subtype.

Data as of 27 July 2026

14 interventional trials — more than 73% of diseases in the trials denominator have none at all (5114 of 7003; this disease is at the 93.1th percentile).

low confidence · 93.1th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

14 interventional trials matched after quoted-phrase search and title/condition post-filter.

Broader category: thrombotic thrombocytopenic purpura

21

Interventional trials for the parent category, exclusive of NCT IDs already counted above. Eligibility for this subtype is not guaranteed.

Worth raising with a clinician. How we count trials.

Recruiting under the broader category

Observational and natural-history studies

5 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.

Recruiting or not-yet-recruiting

Open the complete matched search on ClinicalTrials.gov

Where to find support

We do not yet link condition-specific patient organisations. These umbrella groups support undiagnosed and ultra-rare families:

India — NPRD

Last verified 2026-07-26

This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.

Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.

How we counted this

Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.

"Immune-mediated thrombotic thrombocytopenic purpura" OR "Acquired TTP" OR "Acquired thrombotic thrombocytopenic purpura" OR "Autoimmune thrombotic thrombocytopenic purpura" OR "Thrombotic thrombocytopenic purpura due to anti-ADAMTS-13 antibodies" OR "acquired ADAMTS13 deficiency"

Run this search on Europe PMC

MeSH descriptor terms unioned into the query: Thrombotic thrombocytopenic purpura, acquired

ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):

"Immune-mediated thrombotic thrombocytopenic purpura" OR "Acquired TTP" OR "Acquired thrombotic thrombocytopenic purpura" OR "Autoimmune thrombotic thrombocytopenic purpura" OR "Thrombotic thrombocytopenic purpura due to anti-ADAMTS-13 antibodies" OR "acquired ADAMTS13 deficiency" OR "Thrombotic thrombocytopenic purpura, acquired"

Interventional trials matched via: both, phrase, mesh (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).

Study-type breakdown: 14 interventional · 5 observational · 0 expanded access. Only interventional studies enter the trial headline.

Parent-category trials query:

"thrombotic thrombocytopenic purpura"

Query health: ok — strategies attempted: phrase, mesh; with hits: phrase, mesh

Run this search on ClinicalTrials.gov

Synonyms dropped by stoplist: aTTP; iTTP

Confidence reasoning

  • Preferred label is multi-word and distinctive
  • 2 synonym(s) dropped by stoplist (may under-count)
  • No label/synonym collisions with other diseases in this corpus
  • Publication count (2056) is extremely high with unknown/missing prevalence — treat as possible over-matching, not proven research intensity

Ingested 2026-07-27T04:25:45.333Z