ORPHA:98833
Acute myeloblastic leukemia without maturation
Also known as: AML M1 · Acute myeloblastic leukemia M1
Clinical definition (Orphanet)
A rare, acute myeloid leukemia characterized by no significant myeloid maturation and more than 90% blast cells in the non-erythroid population. Various degrees of anemia, thrombocytopenia, or pancytopenia are present. Frequent clinical manifestations include fatigue, fever, bleeding disorders, and organomegaly, especially .
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.
Is anyone studying this?
1,635
1,635 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=183) is 38.
1,635 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 38 (publications denominator n=183).
731 in the last 10 years · low confidence
Is a treatment being tested?
74
trials for this specific condition
74 interventional trials matched this specific condition name; none in our sample are currently recruiting.
Data as of 26 July 2026
74 interventional trials — more than 59.2% of diseases in the trials denominator have none at all (151 of 255; this disease is at the 95.5th percentile).
low confidence · 95.5th percentile (trials denominator)
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.
Who's working on it?
1,418
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.
- 01Bacher U7 papers · 2026
University Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, Bern, Switzerland.
Papers in Europe PMC - 02Pabst T7 papers · 2026
Department of Medical Oncology, Inselspital, Bern University Hospital, Bern, Switzerland.
Papers in Europe PMC - 03Seipel K6 papers · 2026
Department for Biomedical Research (DBMR), University of Bern, 2008 Bern, Switzerland.
Papers in Europe PMC - 04Li Y5 papers · 2026
Department of Pediatrics, the First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China.
Papers in Europe PMC - 05Liu Y4 papers · 2026
Department of Dermatology, Peking Union Medical College Hospital, Chinese Academy Medical Sciences, Beijing, China.
Papers in Europe PMC - 06Wang W4 papers · 2025
Institute of Hematology, School of Medicine, Jinan University, Guangzhou, China.
Papers in Europe PMC - 07Zhao Y4 papers · 2025
The Key Laboratory of Geriatrics, Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical Sciences, Dongcheng District, Beijing, China.
Papers in Europe PMC - 08Chen J3 papers · 2025
Department of Hematology, First Affiliated Hospital, Jinan University, Guangzhou, China.
Papers in Europe PMC - 09Huang Y3 papers · 2025
Department of Hematology, The Second Affiliated Hospital of Nanchang University, Nanchang, China.
Papers in Europe PMC - 10Kronig MN3 papers · 2026
Department of Medical Oncology, Inselspital, Bern University Hospital, Bern, Switzerland.
Papers in Europe PMC
Recruiting interventional trials
Trials testing a treatment from the matched ClinicalTrials.gov set
74 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.
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.
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 2026-07-26This 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 below but are not added to the query string.
"Acute myeloblastic leukemia without maturation" OR "AML M1" OR "Acute myeloblastic leukemia M1" OR "AML without maturation" OR "FAB M1" OR "M1 acute granulocytic leukaemia" OR "M1 acute granulocytic leukaemia without maturation" OR "M1 acute granulocytic leukemia" OR "M1 acute granulocytic leukemia without maturation" OR "M1 acute myeloblastic leukaemia" OR "M1 acute myeloblastic leukaemia without maturation" OR "M1 acute myeloblastic leukemia" OR "M1 acute myeloblastic leukemia without maturation" OR "M1 acute myelocytic leukaemia" OR "M1 acute myelocytic leukaemia without maturation" OR "M1 acute myelocytic leukemia" OR "M1 acute myelocytic leukemia without maturation" OR "M1 acute myelogenous leukaemia" OR "M1 acute myelogenous leukaemia without maturation" OR "M1 acute myelogenous leukemia" OR "M1 acute myelogenous leukemia without maturation" OR "M1 acute myeloid leukaemia" OR "M1 acute myeloid leukaemia without maturation" OR "M1 acute myeloid leukemia" OR "M1 acute myeloid leukemia without maturation" OR "acute M1 myeloid leukaemia" OR "acute M1 myeloid leukemia" OR "acute granulocytic leukaemia without maturation" OR "acute granulocytic leukemia without maturation" OR "acute myeloblastic leukaemia M1" OR "acute myelocytic leukaemia without maturation" OR "acute myelocytic leukemia without maturation" OR "acute myelogenous leukaemia without maturation" OR "acute myelogenous leukemia without maturation" OR "acute myeloid leukaemia without maturation" OR "acute myeloid leukaemia without maturation (FAB M1)" OR "acute myeloid leukemia without maturation" OR "acute myeloid leukemia without maturation (FAB M1)"
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
"Acute myeloblastic leukemia without maturation" OR "AML M1" OR "Acute myeloblastic leukemia M1" OR "AML without maturation" OR "FAB M1" OR "M1 acute granulocytic leukaemia" OR "M1 acute granulocytic leukaemia without maturation" OR "M1 acute granulocytic leukemia" OR "M1 acute granulocytic leukemia without maturation" OR "M1 acute myeloblastic leukaemia" OR "M1 acute myeloblastic leukaemia without maturation" OR "M1 acute myeloblastic leukemia" OR "M1 acute myeloblastic leukemia without maturation" OR "M1 acute myelocytic leukaemia" OR "M1 acute myelocytic leukaemia without maturation" OR "M1 acute myelocytic leukemia" OR "M1 acute myelocytic leukemia without maturation" OR "M1 acute myelogenous leukaemia" OR "M1 acute myelogenous leukaemia without maturation" OR "M1 acute myelogenous leukemia" OR "M1 acute myelogenous leukemia without maturation" OR "M1 acute myeloid leukaemia" OR "M1 acute myeloid leukaemia without maturation" OR "M1 acute myeloid leukemia" OR "M1 acute myeloid leukemia without maturation" OR "acute M1 myeloid leukaemia" OR "acute M1 myeloid leukemia" OR "acute granulocytic leukaemia without maturation" OR "acute granulocytic leukemia without maturation" OR "acute myeloblastic leukaemia M1" OR "acute myelocytic leukaemia without maturation" OR "acute myelocytic leukemia without maturation" OR "acute myelogenous leukaemia without maturation" OR "acute myelogenous leukemia without maturation" OR "acute myeloid leukaemia without maturation" OR "acute myeloid leukaemia without maturation (FAB M1)" OR "acute myeloid leukemia without maturation" OR "acute myeloid leukemia without maturation (FAB M1)"
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: 74 interventional · 3 observational · 0 expanded access. Only interventional studies enter the trial headline.
Cross-references (from Mondo): UMLS:C0026998 NCIT:C3249
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 (1635) is implausibly high for prevalence class "<1 / 1 000 000" — treat as possible over-matching, not a measure of research intensity