ORPHA:86843
Acute panmyelosis with myelofibrosis
Also known as: Acute myelodysplasia with myelofibrosis · Acute myelofibrosis
Publications
1,065
Trials
0
Interventional, condition-specific
Researchers
1,456
Distinct authors in sample
Gene link
—
Readiness
2/6
Stages with a signal
Clinical definition (Orphanet)
A rare unclassified acute myeloid leukemia characterized by an acute panmyeloid proliferation with blasts constituting more than 20% of cells in the bone marrow or peripheral blood, accompanied by fibrosis of the bone marrow. Patients typically present with acute onset of severe constitutional symptoms, bone pain, and pancytopenia. is minimal or absent. The disease is rapidly with poor therapy response.
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:0019455
- UMLS:C0334674
- NCIT:C4344
Additional Mondo synonyms (7)
APMF · acute (malignant) myelofibrosis · acute (malignant) myelosclerosis · acute myelodysplasia with myelofibrosis · acute myelofibrosis · acute myelosclerosis · acute panmyelosis
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
Research-stage checklist from open sources (GenCC, literature, Monarch when enriched, ClinicalTrials.gov). Not a prognosis or care recommendation.
- Gene identifiedNot found
No GenCC disease–gene assertion in this build
- LiteraturePresent
1,065 matched papers (396 in last 10 years) Source
- Phenotype characterisedPresent
15 HPO annotations (e.g. Increased micromegakaryocyte count; Myelofibrosis; Abnormality of bone marrow stromal cells) Source
- 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 trialNot found
No matched interventional trial under our ClinicalTrials.gov rules
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)
15
Associated phenotypes · MONDO:0019455
- Increased micromegakaryocyte count
- Myelofibrosis
- Abnormality of bone marrow stromal cells
- Acute myelomonocytic leukemia
- Megakaryocyte nucleus hypolobulation
Showing 5 of 15 — open Monarch for the full list.
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?
1,065
1,065 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.
1,065 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).
396 in the last 10 years · low confidence
Phrase hits: 1,065 · MeSH hits: 0
Who's working on it?
1,456
Distinct author names in 200 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.
- 01Chen Y4 papers · 2026
Avian Immunosuppressive Diseases Division, State Key Laboratory of Animal Disease Control and Prevention, Harbin Veterinary Research Institute, The Chinese Academy of Agricultural Sciences, Harbin 150069, China.
Papers in Europe PMC - 02Kim HJ4 papers · 2019
Department of Hematology, Catholic Blood and Marrow Transplantation Center, Leukemia Research Institute, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
Papers in Europe PMC - 03Li J4 papers · 2025
Department of Urology, Changhai Hospital, Naval Medical University (Second Military Medical University), Shanghai, China.
Papers in Europe PMC - 04Cho BS3 papers · 2019
Department of Hematology, Catholic Blood and Marrow Transplantation Center, Leukemia Research Institute, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
Papers in Europe PMC - 05Kobayashi T3 papers · 2025
Division of Hematology and Oncology, Kyoto Prefectural University of Medicine, Kyoto, Japan.
Papers in Europe PMC - 06Kojima K3 papers · 2025
Division of Hematology, Respiratory Medicine and Oncology, Department of Internal Medicine, Faculty of Medicine Saga University, 5-1-1 Nabeshima, Saga, 849-8501, Japan.
Papers in Europe PMC - 07
- 08Lee JW3 papers · 2019
Department of Hematology, Catholic Blood and Marrow Transplantation Center, Leukemia Research Institute, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
Papers in Europe PMC - 09Min WS3 papers · 2017
Department of Hematology, Catholic Blood and Marrow Transplantation Center, Leukemia Research Institute, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
Papers in Europe PMC - 10Orazi A3 papers · 2009
Department of Pathology and Laboratory Medicine, Indiana University School of Medicine, Indianapolis, Indiana 46202-5200, USA. aorazi@iupui.edu
Papers in Europe PMC
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).
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.
low 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.
See who's working on it — people publishing on this disease are often the practical next contact when no trial is listed.
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 11 · after dedupe 11 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 11 · 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 (11)
- ctis·2023-509635-89-00·Expired·A Phase 1b/2 Study of BMS-986158 Monotherapy and in Combination with Either Ruxolitinib or Fedratinib in Participants with DIPSS-Intermediate or High Risk Myelofibrosis
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN16783472·No longer recruiting·Assessing the safety and tolerability of oral ruxolitinib in combination with 5-azacitidine in patients with advanced phase myeloproliferative neoplasms (MPN), including myelodysplastic syndromes (MDS) or acute myeloid leukaemia (AML) arising from MPN.
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·ISRCTN88102629·No longer recruiting·A study on the safety and effectiveness of fedratinib with ropeginterferon alfa-2b in patients with myelofibrosis
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN50290131·No longer recruiting·Comparing interventions for the prevention of graft-versus-host disease after unrelated donor stem cell transplantation
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN12885480·Recruiting·Ruxolitinib versus hydroxycarbamide or interferon as first-line therapy in high-risk polcythemia vera
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN65011803·No longer recruiting·Effects of tamoxifen in patients with myeloproliferatIve disorders
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)
- isrctn·ISRCTN72251782·No longer recruiting·Randomised study to compare aspirin versus hydroxyurea/aspirin in 'intermediate risk' primary thrombocythaemia (PT) and hydroxyurea/aspirin versus anagrelide/aspirin in 'high risk' primary thrombocythaemia
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 panmyelosis with myelofibrosis — 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 panmyelosis with myelofibrosis" OR "Acute myelodysplasia with myelofibrosis" OR "Acute myelofibrosis" OR "acute (malignant) myelofibrosis" OR "acute (malignant) myelosclerosis" OR "acute myelosclerosis" OR "acute panmyelosis"
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
"Acute panmyelosis with myelofibrosis" OR "Acute myelodysplasia with myelofibrosis" OR "Acute myelofibrosis" OR "acute (malignant) myelofibrosis" OR "acute (malignant) myelosclerosis" OR "acute myelosclerosis" OR "acute panmyelosis"
Study-type breakdown: 0 interventional · 0 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
Synonyms dropped by stoplist: APMF
Confidence reasoning
- Preferred label is multi-word and distinctive
- 1 synonym(s) dropped by stoplist (may under-count)
- No label/synonym collisions with other diseases in this corpus
- Publication count (1065) is implausibly high for prevalence class "<1 / 1 000 000" — treat as possible over-matching, not a measure of research intensity
Ingested 2026-07-27T03:10:44.280Z