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ORPHA:98835

Acute undifferentiated leukemia

low confidenceDisorder

Also known as: Acute myeloid leukemia, minimal differentiation, FAB M0

Publications

36,319

Trials

41

Interventional, condition-specific

Researchers

1,536

Distinct authors in sample

Gene link

—

Readiness

2/6

Stages with a signal

Clinical definition (Orphanet)

A rare acute leukemia of ambiguous lineage characterized by clonal proliferation of primitive hematopoietic cells, primarily in the bone marrow and blood, lacking lineage-specific markers and detectable genotypic alterations. The patients present with leukocytosis, anemia, variable platelet count and a variety of nonspecific symptoms related to ineffective hematopoesis (fatigue, bleeding and bruising, recurrent infections, bone pain) and/or extramedullary site involvement (lymphadenopathy, , ).

Orphanet entry

Cross-references

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

Additional Mondo synonyms (10)

acute myeloid leukemia, minimal differentiation, FAB M0 · acute undifferentiated leukemia · leukaemia stem cell · leukemia stem cell · stem cell acute leukaemia · stem cell acute leukemia · stem cell leukaemia · stem cell leukemia · undifferentiated acute leukaemia · undifferentiated acute 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.

  1. Gene identifiedNot found

    No GenCC disease–gene assertion in this build

  2. LiteraturePresent

    36,319 matched papers (18,303 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

    41 matched on ClinicalTrials.gov (7 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?

36,319

36,319 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=5449) is 41.

36,319 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).

18,303 in the last 10 years · low confidence

Phrase hits: 36,319 · MeSH hits: 0

Open Europe PMC search

Who's working on it?

1,536

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
    Zhang Y6 papers · 2026

    The First Hospital of Jilin University, Changchun, Jilin, China.

    Papers in Europe PMC
  2. 02
    Chen W4 papers · 2026

    Department of Pathology, The University of Texas Southwestern Medical Center, Dallas, Texas.

    Papers in Europe PMC
  3. 03
    chen x4 papers · 2026

    Department of Medicinal Chemistry, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Shanghai, China.

    Papers in Europe PMC
  4. 04
    Ciceri F4 papers · 2026

    Acute Leukemia Working Party of EBMT, PARIS, France.

    Papers in Europe PMC
  5. 05
    Li J4 papers · 2026

    Zhongshan Institute for Drug Discovery, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Zhongshan, China. jli@simm.ac.cn.

    Papers in Europe PMC
  6. 06
    Li X4 papers · 2026

    Zhongshan Institute for Drug Discovery, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Zhongshan, China.

    Papers in Europe PMC
  7. 07
    Li Y4 papers · 2026

    Department of Nursing, West China Second University Hospital, Sichuan University, China/Key Laboratory of Birth Defects and Related Diseases of Women and Children (Sichuan University), Ministry of Education, Chengdu, Sichuan, China.

    Papers in Europe PMC
  8. 08
    Liu Y4 papers · 2026

    Department of Hematology, The 960th Hospital of The Chinese People's Liberation Army Joint Logistics Support Force, Jinan, China.

    Papers in Europe PMC
  9. 09
    Medeiros LJ4 papers · 2026

    Department of Hematopathology, The University of Texas MD Anderson Cancer Center, Houston, Texas.

    Papers in Europe PMC
  10. 10
    Wang J4 papers · 2026

    Department of Hematology and Hematology Research Laboratory, West China Hospital of Sichuan University, Chengdu, Sichuan, China.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

41

interventional trials for this specific condition

41 interventional trials matched this specific condition name; 7 currently recruiting in our sample.

Data as of 27 July 2026

41 interventional trials — more than 73% of diseases in the trials denominator have none at all (5114 of 7003; this disease is at the 96.6th percentile).

low confidence · 96.6th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

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

Observational and natural-history studies

4 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.

"Acute undifferentiated leukemia" OR "Acute myeloid leukemia, minimal differentiation, FAB M0" OR "leukaemia stem cell" OR "leukemia stem cell" OR "stem cell acute leukaemia" OR "stem cell acute leukemia" OR "stem cell leukaemia" OR "stem cell leukemia" OR "undifferentiated acute leukaemia" OR "undifferentiated acute leukemia"

Run this search on Europe PMC

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

"Acute undifferentiated leukemia" OR "Acute myeloid leukemia, minimal differentiation, FAB M0" OR "leukaemia stem cell" OR "leukemia stem cell" OR "stem cell acute leukaemia" OR "stem cell acute leukemia" OR "stem cell leukaemia" OR "stem cell leukemia" OR "undifferentiated acute leukaemia" OR "undifferentiated acute leukemia"

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: 41 interventional · 4 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

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 (36319) is extremely high with unknown/missing prevalence — treat as possible over-matching, not proven research intensity

Ingested 2026-07-27T05:31:49.320Z