RARE DISEASERESEARCH ATLAS
Open navigation menu

ORPHA:853

Fetal and neonatal alloimmune thrombocytopenia

medium confidenceDisorder

Also known as: FNAIT · NAIT

Publications

557

80.1th percentile

Trials

4

Interventional, condition-specific

Researchers

912

Distinct authors in sample

Gene link

—

Readiness

4/6

Stages with a signal

Clinical definition (Orphanet)

A rare hematological disease characterized by maternal alloimmunisation against fetal platelet antigens that are inherited from the father and different from those present in the mother, and usually presents as a severe isolated thrombocytopenia in otherwise healthy newborns.

How rare: 1-5 / 10 000 — about one to five people per ten thousand (still uncommon, but less ultra-rare).

Orphanet entry

Cross-references

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

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.”

4/6 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

    557 matched papers (376 in last 10 years) Source

  3. Phenotype characterisedPresent

    17 HPO annotations (e.g. Neonatal alloimmune thrombocytopenia; Purpura; Abnormal bleeding) Source

  4. Animal modelNot found

    No Alliance genotype “model of” associations via Monarch for these Mondo IDs

  5. Orphan designationPartial

    2 EMA designations (none yet with FDA orphan-indication approval) — e.g. nipocalimab Source

  6. Interventional trialPresent

    4 matched on ClinicalTrials.gov (2 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)

17

Associated phenotypes · MONDO:0019415

  • Neonatal alloimmune thrombocytopenia
  • Purpura
  • Abnormal bleeding
  • Spontaneous hematomas
  • Abnormality of the nervous system

Showing 5 of 17 — open Monarch for the full list.

Animal models (Monarch / Alliance)

None returned for this Mondo ID. Empty here is not proof that no model organism work exists under another name or gene.

Monarch fetch 2026-07-29

Therapies

Designations, candidates, and chemicals

FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.

Orphan designation (FDA · EMA)

2

Designations · no FDA orphan-indication approval yet

  • EMA nipocalimabPrevention of fetal and neonatal alloimmune thrombocytopenia · PositiveEMA designation
  • EMA Human platelet antigen-1a immunoglobulinPrevention of fetal and neonatal alloimmune thrombocytopenia · 27/10/2011 · WithdrawnEMA designation

Sources: FDA OOPD · EMA orphan designations

Open Targets candidates

No drugs or clinical candidates returned for this Mondo ID on Open Targets.

CTD chemicals (MyDisease.info)

No CTD chemical associations returned for this Mondo ID.

Literature

Is anyone studying this?

557

557 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=3967) is 59.

557 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).

376 in the last 10 years · medium confidence · 80.1th percentile (publications denominator)

Phrase hits: 557 · MeSH hits: 0

Open Europe PMC search

Who's working on it?

912

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
    Oepkes D18 papers · 2025

    Department of Obstetrics, Leiden University Medical Center, Leiden, The Netherlands.

    Papers in Europe PMC
  2. 02
    de Haas M17 papers · 2026

    Department of Immunohematology Diagnostics, Sanquin, Amsterdam, the Netherlands; Department of Immunohematology and Blood Transfusion, Leiden University Medical Center, Leiden, the Netherlands; Center for Clinical Transfusion Research, Sanquin Research, Leiden, the Netherlands.

    Papers in Europe PMC
  3. 03
    Lopriore E16 papers · 2026

    Division of Neonatology, Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands.

    Papers in Europe PMC
  4. 04
    Winkelhorst D16 papers · 2026

    Department of Obstetrics, Leiden University Medical Center, Leiden, The Netherlands.

    Papers in Europe PMC
  5. 05
    Kjeldsen-Kragh J15 papers · 2025

    Toronto Platelet Immunobiology Group, Toronto, ON, Canada; Department of Clinical Immunology and Transfusion Medicine, University and Regional Laboratories Region Skåne, Lund, Sweden.

    Papers in Europe PMC
  6. 06
    Tiller H15 papers · 2026

    Immunology Research Group, Faculty of Health Sciences, UiT, The Arctic University of Norway.

    Papers in Europe PMC
  7. 07
    van der Schoot CE14 papers · 2026

    Department of Experimental Immunohematology, Sanquin, Amsterdam, The Netherlands.

    Papers in Europe PMC
  8. 08
    Bein G13 papers · 2026

    Institute for Clinical Immunology and Transfusion Medicine, Justus-Liebig-University, Giessen, Germany.

    Papers in Europe PMC
  9. 09
    de Vos TW12 papers · 2026

    Department of Pediatrics, Division of Neonatology, Leiden University Medical Centre, Leiden, the Netherlands.

    Papers in Europe PMC
  10. 10
    Porcelijn L12 papers · 2025

    Department of Immunohematology Diagnostics, Sanquin, Amsterdam, The Netherlands.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

4

interventional trials for this specific condition

4 interventional trials matched this specific condition name; 2 currently recruiting in our sample.

Data as of 11 September 2026

4 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 88.1th percentile).

medium confidence · 88.1th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

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

Observational and natural-history studies

3 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.

None of the matched observational studies is currently listed as recruiting.

Open the complete matched search on ClinicalTrials.gov

Other registries (secondary)

Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.

raw 6 · after dedupe 6 · already on CT.gov 1 · kept 0 · parent 0 · uncertain 5 · dropped 0 · fetched 2026-07-29

Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri

No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).

Uncertain / not reviewed (5)

Where to find support

Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.

Orphanet entry for Fetal and neonatal alloimmune thrombocytopenia — check Associations / patient organisations on that page.

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.

"Fetal and neonatal alloimmune thrombocytopenia" OR "FNAIT"

Run this search on Europe PMC

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

"Fetal and neonatal alloimmune thrombocytopenia" OR "FNAIT"

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

Study-type breakdown: 4 interventional · 3 observational · 0 expanded access. Only interventional studies enter the trial headline.

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

Run this search on ClinicalTrials.gov

Synonyms dropped by stoplist: NAIT

Confidence reasoning

  • Preferred label is multi-word and distinctive
  • 1 synonym(s) dropped by stoplist (may under-count)
  • No label/synonym collisions with other diseases in this corpus

Ingested 2026-07-26T15:39:27.158Z