ORPHA:98832
Acute myeloid leukemia with minimal differentiation
Also known as: AML M0 · Minimally differentiated acute myeloblastic leukemia
Publications
4,322
Trials
9
Interventional, condition-specific
Researchers
1,281
Distinct authors in sample
Gene link
—
Readiness
2/6
Stages with a signal
Clinical definition (Orphanet)
A rare subtype of acute myeloid leukemia characterized by clonal proliferation of poorly differentiated myeloid blasts in the bone marrow, blood or other tissues. It usually presents with anemia, thrombocytopenia and other nonspecific symptoms related to ineffective hematopoesis (fatigue, bleeding and bruising, recurrent infections, bone pain) and/or extramedullary site involvement (gingivitis, ). Low remission rates are reported.
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:0005223
- UMLS:C0522631
- NCIT:C8460
Additional Mondo synonyms (30)
AML with minimal differentiation · M0 acute granulocytic leukaemia · M0 acute granulocytic leukaemia with minimal differentiation · M0 acute granulocytic leukemia · M0 acute granulocytic leukemia with minimal differentiation · M0 acute myeloblastic leukaemia · M0 acute myeloblastic leukemia · M0 acute myelocytic leukaemia · M0 acute myelocytic leukemia · M0 acute myelogenous leukaemia · M0 acute myelogenous leukaemia with minimal differentiation · M0 acute myelogenous leukemia · M0 acute myelogenous leukemia with minimal differentiation · M0 myeloid leukaemia · M0 myeloid leukaemia with minimal differentiation · M0 myeloid leukemia · M0 myeloid leukemia with minimal differentiation · acute myeloblastic leukaemia with minimal differentiation · acute myeloblastic leukemia with minimal differentiation · acute myeloblastic leukemia, minimally differentiated · acute myelocytic leukaemia with minimal differentiation · acute myelocytic leukemia with minimal differentiation · acute myelogenous leukaemia with minimal differentiation · acute myelogenous leukemia with minimal differentiation · acute myeloid leukaemia with minimal differentiation (MO) · acute myeloid leukemia with minimal differentiation · acute myeloid leukemia with minimal differentiation (MO) · acute myeloid leukemia, minimally differentiated · minimally differentiated acute myeloblastic leukaemia · minimally differentiated acute myeloblastic leukemia
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.
- Gene identifiedNot found
No GenCC disease–gene assertion in this build
- LiteraturePresent
4,322 matched papers (1,292 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 trialPresent
9 matched on ClinicalTrials.gov
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?
4,322
4,322 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.
4,322 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).
1,292 in the last 10 years · low confidence
Phrase hits: 4,322 · MeSH hits: 0
Who's working on it?
1,281
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.
- 01Wang J6 papers · 2025
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Papers in Europe PMC - 02Chen X4 papers · 2022
The First Affiliated Hospital, Sun Yat-sen University, 58 Second Zhongshan Road, Guangzhou, 510080, China.
Papers in Europe PMC - 03Medeiros LJ4 papers · 2026
Department of Hematopathology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Papers in Europe PMC - 04Wang H4 papers · 2022
Department of Biochemistry, Zhongshan School of Medicine, Sun Yat-sen University, 74 Second Zhongshan Road, Guangzhou, 510080, China.
Papers in Europe PMC - 05Wang X4 papers · 2025
Department of Hematopathology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Papers in Europe PMC - 06Eguchi M3 papers · 2024
Department of Pediatrics, Ehime University Graduate School of Medicine.
Papers in Europe PMC - 07Kaya Z3 papers · 2026
Gazi University Faculty of Medicine, Department of Pediatric Hematology, Ankara, Türkiye
Papers in Europe PMC - 08Koçak Ü3 papers · 2026
Gazi University Faculty of Medicine, Department of Pediatric Hematology, Ankara, Türkiye
Papers in Europe PMC - 09Li H3 papers · 2025
MOE Key Laboratory of Protein Sciences, Beijing Frontier Research Center for Biological Structure, Department of Basic Medical Sciences, School of Medicine, Tsinghua University, Beijing 100084, China.
Papers in Europe PMC - 10Li P3 papers · 2025
State Key Laboratory of Membrane Biology, Beijing Frontier Research Center for Biological Structure, School of Life Sciences, Tsinghua University, Tsinghua-Peking Center for Life Sciences, Beijing 100084, China. Electronic address: pilongli@mail.tsinghua.edu.cn.
Papers in Europe PMC
Clinical research
Is a treatment being tested?
9
interventional trials for this specific condition
9 interventional trials matched this specific condition name; none in our sample are currently recruiting. 2,455 trials are registered for acute myeloid leukemia, the broader category — shown separately because they may or may not enrol this specific subtype.
Data as of 11 September 2026
9 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 92th percentile).
low confidence · 92th percentile (trials denominator)
Recruiting interventional trials
From the matched ClinicalTrials.gov set
9 interventional trials matched after quoted-phrase search and title/condition post-filter.
No currently recruiting studies in the matched set. Open the same search on ClinicalTrials.gov.
Broader category: acute myeloid leukemia
2,455
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
- NCT00801489·RECRUITING·Fludarabine Phosphate, Cytarabine, Filgrastim-sndz, Gemtuzumab Ozogamicin, and Idarubicin Hydrochloride in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
Not reviewed·Conditions: Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11 · Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11 · Acute Myeloid Leukemia With t(8;21); (q22; q22.1); RUNX1-RUNX1T1 · de Novo Myelodysplastic Syndrome·Matched via name phrase
- NCT02727803·RECRUITING·Personalized NK Cell Therapy in CBT
Not reviewed·Conditions: Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive · Acute Biphenotypic Leukemia · Acute Lymphoblastic Leukemia · Acute Lymphoblastic Leukemia in Remission·Matched via name phrase
- NCT06345365·RECRUITING·MA+AZA Regimen for the Treatment of Newly Diagnosed Acute Myeloid Leukemia (AML)
Not reviewed·Conditions: Acute Myeloid Leukaemia·Matched via name phrase
- NCT07198867·RECRUITING·A Study of A-CAR028 Treatment in Subjects With Relapsed or Refractory Acute Myeloid Leukemia
Not reviewed·Conditions: Acute Myeloid Leukemia (AML) · CAR-T Cell Therapy·Matched via name phrase
- NCT03300492·RECRUITING·Expanded Natural Killer Cells Following Haploidentical HSCT for AML/MDS
Not reviewed·Conditions: Acute Myeloid Leukemia · Myelodysplastic Syndromes·Matched via name phrase
- NCT06401603·RECRUITING·A Phase I Study of Decitabine, Lisaftoclax, and Olverembatinib in Patients With Advanced Chronic Myeloid Leukemia and Philadelphia Chromosome-Positive Acute Myeloid Leukemia
Not reviewed·Conditions: Advanced Chronic Myeloid Leukemia · Philadelphia Chromosome-Positive Acute Myeloid Leukemia·Matched via name phrase
- NCT07356154·RECRUITING·A Study of Revumenib and Mezigdomide in People With Leukemia
Not reviewed·Conditions: Leukemia · Acute Leukemia · Relapse Leukemia · Refractory Leukemia·Matched via name phrase
- NCT07521124·NOT YET RECRUITING·ABC Maintenance Therapy for AML
Not reviewed·Conditions: Acute Myeloid Leukemia (AML) in Remission·Matched via name phrase
- NCT06013423·RECRUITING·Cord Blood Transplant, Cyclophosphamide, Fludarabine, and Total-Body Irradiation in Treating Patients With High-Risk Hematologic Diseases
Not reviewed·Conditions: Acute Leukemia of Ambiguous Lineage · Acute Lymphoblastic Leukemia · Acute Myeloid Leukemia · Blastic Plasmacytoid Dendritic Cell Neoplasm·Matched via name phrase
- NCT05949125·RECRUITING·Phase 1 Study of Allo-RevCAR01-T-CD123 in Patients With Selected CD123 Positive Hematologic Malignancies
Not reviewed·Conditions: Acute Myeloid Leukemia, in Relapse · Acute Myeloid Leukemia Refractory·Matched via name phrase
- NCT07025824·NOT YET RECRUITING·Evaluation of Treosulfan Versus Melphalan Conditioning Followed by PTCy in Patients With AML and MDS Undergoing Allogeneic Transplantation
Not reviewed·Conditions: AML - Acute Myeloid Leukemia · MDS (Myelodysplastic Syndrome)·Matched via name phrase
- NCT05991908·RECRUITING·Randomized Study of Conditioning of Fludarabine Combined With Single or Dual Alkylating Agents in Myeloid Malignancies
Not reviewed·Conditions: Acute Myeloid Leukemia · Myelodysplastic Syndromes·Matched via name phrase
- NCT04869683·RECRUITING·Biocollection in MyeloDysplastic Syndrome (P-MDS)
Not reviewed·Conditions: Myelodysplastic Syndromes · Myelodysplastic Anemia · Myelodysplastic Syndrome With Isolated Del(5Q) · Myelodysplastic Syndrome With Ring Sideroblasts·Matched via name phrase
- NCT07082452·NOT YET RECRUITING·A Multicenter Trial Evaluating Efficacy and Safety of A Reduced Venetoclax Exposure To Seven Days Versus Standard Continuous Venetoclax Exposure Combined With Azacitidine in Treatment Naïve Subjects With Acute Myeloid Leukemia Who Are Ineligible for Intensive Induction
Not reviewed·Conditions: Leukemia, B-Cell, Chronic · Leukemia·Matched via name phrase
- NCT06297941·RECRUITING·Study of REM-422 in Patients With AML or Higher Risk MDS
Not reviewed·Conditions: Myelodysplastic Syndromes · Higher Risk Myelodysplastic Syndromes · Acute Myeloid Leukemia · Acute Myeloid Leukemia Refractory·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 6 · after dedupe 6 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 6 · 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 (6)
- isrctn·ISRCTN18332222·No longer recruiting·A phase II multicentre study to assess the tolerability and efficacy of the addition of bevacizumab to standard induction therapy in Acute Myeloid Leukaemia and high risk myelodysplastic syndrome above 60 years
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN70542454·No longer recruiting·Reduced intensity chemotherapy given with and without imatinib mesylate in patients >/= 60 years considered unfit for standard chemotherapy with previously untreated acute myeloid leukemia (AML) and refractory anemia with excess of blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T): a randomized phase II study
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN77039377·No longer recruiting·Randomised induction and post induction therapy in older patients (greater than or equal to 61 years of age) with Acute Myelocytic Leukaemia (AML) and Refractory Anaemia with Excess of Blasts (RAEB, RAEB-t)
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN38648181·No longer recruiting·Randomised induction and post-induction therapy in adult patients (less than or equal to 60 years of age) with acute myelocytic leukaemia (AML) or refractory anaemia with excess of blasts (RAEB, RAEB-t) with IPSS score greater than or equal to 1.5
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN76815071·No longer recruiting·Risk adapted treatment of Acute Myelocytic Leukaemia (AML)
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN11571826·No longer recruiting·An open label randomised controlled study in elderly subjects with previously untreated acute myelogenous leukaemia, comparing treatment groups randomised to receive daunorubicin and cytarabine or daunorubicin, cytarabine and PSC-833
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 Acute myeloid leukemia with minimal differentiation — 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.
"Acute myeloid leukemia with minimal differentiation" OR "AML M0" OR "Minimally differentiated acute myeloblastic leukemia" OR "AML with minimal differentiation" OR "M0 acute granulocytic leukaemia" OR "M0 acute granulocytic leukaemia with minimal differentiation" OR "M0 acute granulocytic leukemia" OR "M0 acute granulocytic leukemia with minimal differentiation" OR "M0 acute myeloblastic leukaemia" OR "M0 acute myeloblastic leukemia" OR "M0 acute myelocytic leukaemia" OR "M0 acute myelocytic leukemia" OR "M0 acute myelogenous leukaemia" OR "M0 acute myelogenous leukaemia with minimal differentiation" OR "M0 acute myelogenous leukemia" OR "M0 acute myelogenous leukemia with minimal differentiation" OR "M0 myeloid leukaemia" OR "M0 myeloid leukaemia with minimal differentiation" OR "M0 myeloid leukemia" OR "M0 myeloid leukemia with minimal differentiation" OR "acute myeloblastic leukaemia with minimal differentiation" OR "acute myeloblastic leukemia with minimal differentiation" OR "acute myeloblastic leukemia, minimally differentiated" OR "acute myelocytic leukaemia with minimal differentiation" OR "acute myelocytic leukemia with minimal differentiation" OR "acute myelogenous leukaemia with minimal differentiation" OR "acute myelogenous leukemia with minimal differentiation" OR "acute myeloid leukaemia with minimal differentiation (MO)" OR "acute myeloid leukemia with minimal differentiation (MO)" OR "acute myeloid leukemia, minimally differentiated" OR "minimally differentiated acute myeloblastic leukaemia"
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
"Acute myeloid leukemia with minimal differentiation" OR "AML M0" OR "Minimally differentiated acute myeloblastic leukemia" OR "AML with minimal differentiation" OR "M0 acute granulocytic leukaemia" OR "M0 acute granulocytic leukaemia with minimal differentiation" OR "M0 acute granulocytic leukemia" OR "M0 acute granulocytic leukemia with minimal differentiation" OR "M0 acute myeloblastic leukaemia" OR "M0 acute myeloblastic leukemia" OR "M0 acute myelocytic leukaemia" OR "M0 acute myelocytic leukemia" OR "M0 acute myelogenous leukaemia" OR "M0 acute myelogenous leukaemia with minimal differentiation" OR "M0 acute myelogenous leukemia" OR "M0 acute myelogenous leukemia with minimal differentiation" OR "M0 myeloid leukaemia" OR "M0 myeloid leukaemia with minimal differentiation" OR "M0 myeloid leukemia" OR "M0 myeloid leukemia with minimal differentiation" OR "acute myeloblastic leukaemia with minimal differentiation" OR "acute myeloblastic leukemia with minimal differentiation" OR "acute myeloblastic leukemia, minimally differentiated" OR "acute myelocytic leukaemia with minimal differentiation" OR "acute myelocytic leukemia with minimal differentiation" OR "acute myelogenous leukaemia with minimal differentiation" OR "acute myelogenous leukemia with minimal differentiation" OR "acute myeloid leukaemia with minimal differentiation (MO)" OR "acute myeloid leukemia with minimal differentiation (MO)" OR "acute myeloid leukemia, minimally differentiated" OR "minimally differentiated acute myeloblastic leukaemia"
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: 9 interventional · 0 observational · 0 expanded access. Only interventional studies enter the trial headline.
Parent-category trials query:
"acute myeloid leukemia"
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
- Publication count (4322) is implausibly high for prevalence class "<1 / 1 000 000" — treat as possible over-matching, not a measure of research intensity
Ingested 2026-07-27T05:30:50.037Z