ORPHA:398109
Neonatal autoimmune hemolytic anemia
Also known as: Neonatal AHA · Neonatal AIHA
Publications
8
24.4th percentile
Trials
0
Interventional, condition-specific
Researchers
50
Distinct authors in sample
Gene link
—
Readiness
2/6
Stages with a signal
Clinical definition (Orphanet)
A rare, secondary, autoimmune disease characterized by onset of hemolytic anemia in the period associated with a positive direct antiglobulin test. may be associated.
How rare: <1 / 1 000 000 — fewer than one in a million. In a city the size of Kolkata, that might mean on the order of fifteen people.
Cross-references
Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.
- MONDO:0018358
- UMLS:C4751517
Additional Mondo synonyms (2)
neonatal AHA · neonatal AIHA
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
No specific-condition interventional trial, but broader-category trials exist — discuss eligibility with a clinician.
- Gene identifiedNot found
No GenCC disease–gene assertion in this build
- LiteraturePresent
8 matched papers (7 in last 10 years) Source
- Phenotype characterisedNot found
No HPO disease–phenotype associations via Monarch for these Mondo IDs
- Animal modelNot found
No Alliance genotype “model of” associations via Monarch for these Mondo IDs
- Orphan designationNot found
No FDA or EMA orphan-drug designation matched this disease via UMLS or preferred name Source
- Interventional trialPartial
None under the specific name; 75 for broader category autoimmune hemolytic anemia
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)
None returned for this Mondo ID. That often means “not linked under this ID,” not “no clinical features.”
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)
No designation matched this disease via UMLS or preferred name on the FDA OOPD mirror or EMA orphan register. Absence here is not proof that none exists under another wording.
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?
8
8 papers have ever been indexed under this name. For scale, breast cancer has over 700,000. Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.
8 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).
7 in the last 10 years · high confidence · 24.4th percentile (publications denominator)
Phrase hits: 8 · MeSH hits: 0
Who's working on it?
50
Distinct author names in 8 sampled papers — named people below.
Who's working on it?
No interventional trial matched this name; these authors publish on it in the sampled literature — a practical starting point for contact.
- 01Alsaleem M1 paper · 2020
Pediatrics Department, Neonatology, Children's Mercy Hospital, Kansas City, MO 64108, USA.
Papers in Europe PMC - 02Aydin F1 paper · 2021
Department of Pediatrics, Division of Pediatric Hematology and Oncology & Pediatric HSCT Unit.
Papers in Europe PMC - 03Barcellini W1 paper · 2023
Hematology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Papers in Europe PMC - 04Black LV1 paper · 2009
Division of Pediatric Hemato-Oncology, University of Alabama at Birmingham, Birmingham, AL 35233, USA.
Papers in Europe PMC - 05Bortolotti M1 paper · 2023
Hematology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Papers in Europe PMC - 06
- 07Cansever M1 paper · 2021
Pediatrics, Division of Pediatric Immunology, Faculty of Medicine.
Papers in Europe PMC - 08Chen F1 paper · 2023
Clinical Hematology, Barts Health National Health Service Trust, Queen Mary University, London, United Kingdom.
Papers in Europe PMC - 09Couto M1 paper · 2026
Haematology, Mater Misericordiae University Hospital, Dublin, IRL.
Papers in Europe PMC - 10
Clinical research
Is a treatment being tested?
0
interventional trials for this specific condition
No interventional trial testing a treatment matched this specific condition name on ClinicalTrials.gov (observational studies and pan-disease registries are listed separately when present). 75 trials are registered for autoimmune hemolytic anemia, the broader category — shown separately because they may or may not enrol this specific subtype.
Data as of 11 September 2026 · last trial check 11 September 2026
No matched interventional trials. This is true for 77.2% of diseases in the trials denominator (5501 of 7126). Here are the researchers publishing on it.
high confidence · 38.6th percentile (trials denominator)
Recruiting interventional trials
From the matched ClinicalTrials.gov set
No interventional trial testing a treatment was found for this specific condition name on ClinicalTrials.gov.
75 interventional trials matched autoimmune hemolytic anemia, the broader category — listed below. Those studies are not counted in the condition-specific total.
Broader category: autoimmune hemolytic anemia
75
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
- NCT07748533·RECRUITING·HY001N for Patients With Autoimmune Hemolytic Anemia After Failure ≥3 Lines of Therapy.
Conditions: Autoimmune Hemolytic Anemia (AIHA)·Matched via name phrase
- NCT07361094·RECRUITING·Autologous CD19/BCMA Dual-Target CAR-T for Relapsed/Refractory Autoimmune Diseases
Conditions: Relapsed/Refractory Systemic Lupus Erythematosus · Relapsed/Refractory Systemic Sclerosis · Relapsed/Refractory Idiopathic Inflammatory Myopathies · Relapsed/Refractory sjögren's Syndrome·Matched via name phrase
- NCT07441525·RECRUITING·UCAR-T Targeting CD19/BCMA in Subjects With Autoantibody-Mediated Autoimmune Benign Hematological Diseases
Conditions: Autoimmune Hemolytic Anemia · Primary Immune Thrombocytopenic Purpura · Evans Syndrome·Matched via name phrase
- NCT06847607·NOT YET RECRUITING·A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)
Conditions: AIHA - Warm Autoimmune Hemolytic Anemia · AIHA - Cold Autoimmune Hemolytic Anemia·Matched via name phrase
- NCT07453368·RECRUITING·Orelabrutinib in the Treatment of Relapsed/Refractory AIHA
Conditions: Autoimmune Hemolytic Anemia (AIHA)·Matched via name phrase
- NCT07287930·NOT YET RECRUITING·A Study of YTS109 Cell in Subjects With Relapsed/Refractory Autoimmune Hemolytic Anemia
Conditions: Autoimmune Hemolytic Anemia·Matched via name phrase
- NCT05925023·RECRUITING·Sirolimus in the Treatment of Refractory/Relapsed wAIHA
Conditions: Warm Autoimmune Hemolytic Anemia·Matched via name phrase
- NCT07743242·RECRUITING·Autoimmune Hemolytic Anemia and Its Clinical Implication on Antiphospholipid Syndrome
Conditions: Anti Phospholipid Syndrome·Matched via name phrase
- NCT05089227·RECRUITING·Efficacy of Prolonged Anticoagulation for Primary Prevention of Venous Thromboembolic Disease in Autoimmune Hemolytic Anemia: a Prospective, Phase II, Randomized, Multicenter Study
Conditions: Prolonged Anticoagulation · Venous Thromboembolic Disease · Autoimmune Hemolytic Anemia·Matched via name phrase
- NCT04138927·ENROLLING BY INVITATION·A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
Conditions: Warm Antibody Autoimmune Hemolytic Anemia·Matched via name phrase
- NCT07738822·NOT YET RECRUITING·A Phase 1b/2a Study of Velinotamig in Adults With Relapsed/Refractory ITP and wAIHA
Conditions: Immune Thrombocytopenia (ITP) · Warm Autoimmune Hemolytic Anemia (WAIHA)·Matched via name phrase
- NCT07205315·RECRUITING·A Clinical Study Evaluating the Safety and Efficacy of GT801 Injection in Adult Patients With Relapsed/Refractory CD19-positive B-cell Hematologic Malignancies and Autoimmune Hemolytic Anemia
Conditions: B-cell Acute Lymphoblastic Leukemia (B-ALL) · Chronic Lymphocytic Leukemia (CLL) · B-cell Non-Hodgkin's Lymphoma (B-NHL) · Autoimmune Hemolytic Anemia (AIHA)·Matched via name phrase
- NCT07175493·RECRUITING·A Study of CM336 in Patients With Relapsed or Refractory Autoimmune Cytopenia
Conditions: Autoimmune Cytopenia · Immune Thrombocytopenia (ITP) · Autoimmune Hemolytic Anemia · Evans Syndrome·Matched via name phrase
- NCT07324889·NOT YET RECRUITING·An Open-label, Single-arm, Prospective, Multicenter, Phase I/II Clinical Study on the Safety and Efficacy of CD19/BCMA CAR-T Cell Therapy for Relapsed/Refractory Warm Antibody Autoimmune Hemolytic Anemia
Conditions: Relapsed/Refractory Warm Antibody Autoimmune Hemolytic Anemia·Matched via name phrase
- NCT07689604·NOT YET RECRUITING·META 10-19 in Patients With Relapsed/Refractory Autoimmune Hemolytic Anemia
Conditions: Autoimmune Hemolytic Anemia (AIHA)·Matched via name phrase
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 1 · after dedupe 1 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 1 · dropped 0 · fetched 2026-07-30
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 (1)
- isrctn·ISRCTN12057804·Recruiting·A study of nipocalimab in pregnancies for severe hemolytic disease of the fetus and newborn (HDFN)
skipped — LLM skipped (--skip-llm)
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 Neonatal autoimmune hemolytic anemia — check Associations / patient organisations on that page.
India — NPRD
Last verified 2026-07-26This 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.
"Neonatal autoimmune hemolytic anemia" OR "Neonatal AHA" OR "Neonatal AIHA"
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
"Neonatal autoimmune hemolytic anemia" OR "Neonatal AHA" OR "Neonatal AIHA"
Study-type breakdown: 0 interventional · 0 observational · 0 expanded access. Only interventional studies enter the trial headline.
Parent-category trials query:
"autoimmune hemolytic anemia"
Query health: ok — strategies attempted: phrase; with hits: phrase
Run this search on ClinicalTrials.gov
Confidence reasoning
- Preferred label is multi-word and distinctive
- No synonyms dropped by stoplist
- No label/synonym collisions with other diseases in this corpus
Ingested 2026-07-27T15:19:56.004Z
