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ORPHA:86830

Chronic myeloproliferative disease, unclassifiable

high confidenceDisorder

Also known as: CMPD-U · Undifferentiated myeloproliferative disease

Publications

630

82.5th percentile

Trials

46

Interventional, condition-specific

Researchers

1,614

Distinct authors in sample

Gene link

—

Readiness

3/6

Stages with a signal

Clinical definition (Orphanet)

A rare hematological neoplasm characterized by clonal proliferation of myeloid precursors in the bone marrow, blood and other tissues (spleen, liver), with clinical, morphological and molecular features of myeloproliferative neoplasms (MPN), failing to meet criteria of a specific MPN. The presentation is nonspecific and variable and often includes leukocytosis, thrombocytosis and anemia. , as well as fatigue, malaise or weight loss may appear in advanced stages.

How rare: 1-9 / 1 000 000 — roughly one to nine people per million. In a city the size of Kolkata, perhaps a few dozen.

Orphanet entry

Cross-references

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

Additional Mondo synonyms (9)

CMPD, U · MPN, U · MPN-U · chronic myeloproliferative disease, unclassifiable · chronic myeloproliferative disorder, unclassifiable · myeloproliferative neoplasm, unclassifiable · unclassifiable chronic myeloproliferative disease · unclassifiable chronic myeloproliferative disorder · undifferentiated myeloproliferative disease

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.”

3/6 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

    630 matched papers (461 in last 10 years) Source

  3. Phenotype characterisedPresent

    3 HPO annotations (e.g. Increased total eosinophil count; Malignant eosinophil proliferation; Myeloproliferative disorder) Source

  4. Animal modelNot found

    No Alliance genotype “model of” associations via Monarch for these Mondo IDs

  5. Orphan designationNot found

    No FDA or EMA orphan-drug designation matched this disease via UMLS or preferred name Source

  6. Interventional trialPresent

    46 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)

3

Associated phenotypes · MONDO:0019452

  • Increased total eosinophil count
  • Malignant eosinophil proliferation
  • Myeloproliferative disorder

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

1

Drugs / clinical candidates · MONDO_0019452

CTD chemicals (MyDisease.info)

No CTD chemical associations returned for this Mondo ID.

Literature

Is anyone studying this?

630

630 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.

630 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).

461 in the last 10 years · high confidence · 82.5th percentile (publications denominator)

Phrase hits: 630 · MeSH hits: 0

Open Europe PMC search

Who's working on it?

1,614

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
    Gangat N9 papers · 2025

    Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, Minnesota, USA.

    Papers in Europe PMC
  2. 02
    Andersson TM6 papers · 2025

    Department of Medical Epidemiology and Biostatistics, Karolinska Institutet, Stockholm, Sweden.

    Papers in Europe PMC
  3. 03
    Bak M6 papers · 2026

    Department of Haematology, Zealand University Hospital, University of Copenhagen, 4000 Roskilde, Denmark.

    Papers in Europe PMC
  4. 04
    George TI6 papers · 2026

    Department of Pathology, School of Medicine, University of Utah, Salt Lake, UT, USA.

    Papers in Europe PMC
  5. 05
    Hultcrantz M6 papers · 2025

    Department of Medicine, Solna, Karolinska Institutet, Stockholm, Sweden.

    Papers in Europe PMC
  6. 06
    Orazi A6 papers · 2025

    Department of Pathology, Texas Tech University Health Sciences Center, El Paso, TX, United States. Electronic address: Attilio.Orazi@ttuhsc.edu.

    Papers in Europe PMC
  7. 07
    Tefferi A6 papers · 2025

    Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, Minnesota, USA. tefferi.ayalew@mayo.edu.

    Papers in Europe PMC
  8. 08
    Xu Z6 papers · 2025

    State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. xuzefeng@ihcams.ac.cn.

    Papers in Europe PMC
  9. 09
    Björkholm M5 papers · 2024

    Department of Medicine, Solna, Karolinska Institutet, Stockholm, Sweden.

    Papers in Europe PMC
  10. 10
    Cattaneo D5 papers · 2025

    Hematology Division, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

46

interventional trials for this specific condition

46 interventional trials matched this specific condition name; none in our sample are currently recruiting.

Data as of 11 September 2026

46 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 97th percentile).

high confidence · 97th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

46 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

5 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

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 4 · after dedupe 4 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 4 · 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 (4)

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 Chronic myeloproliferative disease, unclassifiable — check Associations / patient organisations on that page.

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.

"Chronic myeloproliferative disease, unclassifiable" OR "CMPD-U" OR "Undifferentiated myeloproliferative disease" OR "CMPD, U" OR "MPN, U" OR "MPN-U" OR "chronic myeloproliferative disorder, unclassifiable" OR "myeloproliferative neoplasm, unclassifiable" OR "unclassifiable chronic myeloproliferative disease" OR "unclassifiable chronic myeloproliferative disorder"

Run this search on Europe PMC

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

"Chronic myeloproliferative disease, unclassifiable" OR "CMPD-U" OR "Undifferentiated myeloproliferative disease" OR "CMPD, U" OR "MPN, U" OR "MPN-U" OR "chronic myeloproliferative disorder, unclassifiable" OR "myeloproliferative neoplasm, unclassifiable" OR "unclassifiable chronic myeloproliferative disease" OR "unclassifiable chronic myeloproliferative disorder"

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: 46 interventional · 5 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

Ingested 2026-07-27T03:08:34.103Z