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fetal and neonatal alloimmune thrombocytopenia (NAIT) - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

fetal and neonatal alloimmune thrombocytopenia

MONDO:0019415

Also known as:NAIT

Overview

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.

TreatmentsNo approved treatments · 3 in pipeline

Patient Orgs1 organizations · Naitbabies

Diagnosis

Biomarker and diagnostic research for fetal and neonatal alloimmune thrombocytopenia has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for fetal and neonatal alloimmune thrombocytopenia. An additional 3 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for fetal and neonatal alloimmune thrombocytopenia, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for fetal and neonatal alloimmune thrombocytopenia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Prognosis

Estimated prevalence: 1-5 in 10,000 (Uncommon).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

No clinical trials have been registered for fetal and neonatal alloimmune thrombocytopenia.

40 publications have been identified in PubMed for fetal and neonatal alloimmune thrombocytopenia. Research spans Review / Meta-Analysis (25%), Clinical Trial Publication (15%), and Epidemiology / Natural History (15%).

Research Type	Count	% of Total
Research summaries	10	25%

Data assembled from 4 of 12 sources · Last updated Sep 20, 2026, 8:29 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Patient Support

Patient Advocacy Groups

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NaitbabiesPrimary Focus

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Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.

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Medical References

External Resources

Orphanet

European rare disease database

Orphanet:853

GARD

Genetic and Rare Diseases Info Center

GARD:2295

Questions for Your Care Team

→With 3 compounds in the orphan drug pipeline, which are furthest along in development and which clinical trials should I be aware of?
→Are there specialists or centers of excellence for this condition in my area?
→How can I connect with Naitbabies and other patient organizations for support?

← Back to All Diseases

Kisho

fetal and neonatal alloimmune thrombocytopenia

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Patient Support

Patient Advocacy Groups

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Patient perspectives on hemolytic disease of the fetus and newborn and fetal and neonatal alloimmune thrombocytopenia: common experiences and insights.

fetal and neonatal alloimmune thrombocytopenia

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Patient Support

Patient Advocacy Groups

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Patient perspectives on hemolytic disease of the fetus and newborn and fetal and neonatal alloimmune thrombocytopenia: common experiences and insights.