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ORPHA:521

Chronic myeloid leukemia

medium confidenceDisorder

Also known as: CML · Chronic granulocytic leukemia · Chronic myelogenous leukemia

Publications

83,853

99.3th percentile

Trials

793

Interventional, condition-specific

Researchers

1,285

Distinct authors in sample

Gene link

—

Readiness

5/6

Stages with a signal

Clinical definition (Orphanet)

Chronic myeloid leukaemia (CML) is the most common myeloproliferative disorder accounting for 15-20% of all leukaemia cases.

How rare: 1-9 / 100 000 — about one to nine people per hundred thousand.

Orphanet entry

Cross-references

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

Additional Mondo synonyms (17)

BCR-ABL Positive chronic myelogenous leukaemia · BCR-ABL Positive chronic myelogenous leukemia · CML - chronic myelogenous leukaemia · CML - chronic myelogenous leukemia · chronic granulocytic leukemia · chronic myelocytic leukaemia · chronic myelocytic leukemia · chronic myelogenous leukaemia (CML) · chronic myelogenous leukemia · chronic myelogenous leukemia (CML) · chronic myelogenous leukemia, BCR-ABL1 Positive · chronic myelogenous leukemias · chronic myeloid leukemia · hematopoeitic - chronic myelocytic leukaemia (CML) · hematopoeitic - chronic myelocytic leukemia (CML) · leukimia, chronic myeloid · myeloid leukemia, chronic

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.”

5/6 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

    83,853 matched papers (39,907 in last 10 years) Source

  3. Phenotype characterisedPresent

    15 HPO annotations (e.g. Myeloproliferative disorder; Thrombocytopenia; Abnormal granulocyte morphology) Source

  4. Animal modelPresent

    48 genotype models (Mus musculus) Source

  5. Orphan designationPresent

    1 FDA designation (1 FDA orphan-indication approval) — e.g. Rebastinib Source

  6. Interventional trialPresent

    793 matched on ClinicalTrials.gov (77 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)

15

Associated phenotypes · MONDO:0011996

  • Myeloproliferative disorder
  • Thrombocytopenia
  • Abnormal granulocyte morphology
  • Abnormal basophil morphology
  • Fever

Showing 5 of 15 — open Monarch for the full list.

Animal models (Monarch / Alliance)

48

Model associations linked to this Mondo ID

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)

1

Designation · 1 with FDA orphan-indication approval

  • FDA RebastinibPhiladelphia Chromosome Positive Chronic Myeloid Leukemia · 2009-09-03 · Not FDA Approved for Orphan Indication

Sources: FDA OOPD · EMA orphan designations

Open Targets candidates

189

Drugs / clinical candidates · MONDO_0011996

CTD chemicals (MyDisease.info)

43 associated chemicals · 239 pathways. Therapeutic evidence is listed first when present — not a treatment recommendation.

  • Allopurinol · therapeutic
  • arsenic disulfide · therapeutic
  • Arsenic Trioxide · therapeutic
  • Arsenicals · therapeutic
  • Bortezomib · therapeutic
  • Busulfan · therapeutic
  • Cyclophosphamide · therapeutic
  • Cytarabine · therapeutic
  • danusertib · therapeutic
  • Dasatinib · therapeutic
  • Decitabine · therapeutic
  • Dexamethasone · therapeutic

Pathways: Glutathione metabolism; Arachidonic acid metabolism; Nitrogen metabolism; Metabolism of xenobiotics by cytochrome P450; Drug metabolism - cytochrome P450; Metabolic pathways; Endocrine resistance; Platinum drug resistance

MyDisease.info · MONDO:0011996

Literature

Is anyone studying this?

83,853

83,853 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=3967) is 59.

83,853 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).

39,907 in the last 10 years · medium confidence · 99.3th percentile (publications denominator)

Phrase hits: 83,853 · MeSH hits: 0

Open Europe PMC search

Who's working on it?

1,285

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 Y11 papers · 2026

    Department of Hematology, Henan Cancer Hospital, Affiliated Cancer Hospital of Zhengzhou University, Henan, China.

    Papers in Europe PMC
  2. 02
    Takahashi N8 papers · 2026

    Department of Hematology, Akita University Hospital, 1-1-1 Hondo, Akita City, Akita, 010-8543, Japan. naotot@doc.med.akita-u.ac.jp.

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

    Dept of Translational and Precision Medicine, Sapienza University-Az, Policlinico Umberto, Rome, Italy. Electronic address: breccia@bce.uniroma1.it.

    Papers in Europe PMC
  4. 04
    Liu Z6 papers · 2026

    Department of Hematology, First Affiliated Hospital of Guangxi Medical University, Guangxi, China.

    Papers in Europe PMC
  5. 05
    Iqbal Z5 papers · 2026

    College of Applied Medical Sciences (CoAMS) , King Saud Bin Abdulaziz University of Health Sciences, Riyadh, Saudi Arabia.

    Papers in Europe PMC
  6. 06
    Abdulgayoom M4 papers · 2026

    Hematology Department, National Center for Cancer Care and Research (NCCCR), Doha 3050, Qatar.

    Papers in Europe PMC
  7. 07
    Chen C4 papers · 2026

    Department of Hematology, Qilu Hospital of Shandong University, Jinan, China.

    Papers in Europe PMC
  8. 08
    Cortes JE4 papers · 2026

    Georgia Cancer Center at Augusta University, Augusta, GA. jorge.cortes@augusta.edu.

    Papers in Europe PMC
  9. 09
    Jiang Q4 papers · 2026

    National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China; Department of Hematology, peking University People's Hospital, Qingdao, China. Electronic address: jiangqian@medmail.com.cn.

    Papers in Europe PMC
  10. 10
    Khan S4 papers · 2026

    Khyber Medical College Peshawar KPK Pakistan.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

793

interventional trials for this specific condition

793 interventional trials matched this specific condition name; 77 currently recruiting in our sample. 2,246 trials are registered for myeloid leukemia, the broader category — shown separately because they may or may not enrol this specific subtype.

Data as of 11 September 2026

793 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 99.8th percentile).

medium confidence · 99.8th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

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

Broader category: myeloid leukemia

2,246

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

Observational and natural-history studies

152 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 53 · after dedupe 52 · already on CT.gov 1 · kept 0 · parent 0 · uncertain 51 · dropped 0 · fetched 2026-07-29

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 (51)

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 myeloid leukemia — 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 myeloid leukemia" OR "Chronic granulocytic leukemia" OR "Chronic myelogenous leukemia" OR "BCR-ABL Positive chronic myelogenous leukaemia" OR "BCR-ABL Positive chronic myelogenous leukemia" OR "CML - chronic myelogenous leukaemia" OR "CML - chronic myelogenous leukemia" OR "chronic myelocytic leukaemia" OR "chronic myelocytic leukemia" OR "chronic myelogenous leukaemia (CML)" OR "chronic myelogenous leukemia (CML)" OR "chronic myelogenous leukemia, BCR-ABL1 Positive" OR "chronic myelogenous leukemias" OR "hematopoeitic - chronic myelocytic leukaemia (CML)" OR "hematopoeitic - chronic myelocytic leukemia (CML)" OR "leukimia, chronic myeloid" OR "myeloid leukemia, chronic"

Run this search on Europe PMC

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

"Chronic myeloid leukemia" OR "Chronic granulocytic leukemia" OR "Chronic myelogenous leukemia" OR "BCR-ABL Positive chronic myelogenous leukaemia" OR "BCR-ABL Positive chronic myelogenous leukemia" OR "CML - chronic myelogenous leukaemia" OR "CML - chronic myelogenous leukemia" OR "chronic myelocytic leukaemia" OR "chronic myelocytic leukemia" OR "chronic myelogenous leukaemia (CML)" OR "chronic myelogenous leukemia (CML)" OR "chronic myelogenous leukemia, BCR-ABL1 Positive" OR "chronic myelogenous leukemias" OR "hematopoeitic - chronic myelocytic leukaemia (CML)" OR "hematopoeitic - chronic myelocytic leukemia (CML)" OR "leukimia, chronic myeloid" OR "myeloid leukemia, chronic"

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: 793 interventional · 152 observational · 11 expanded access. Only interventional studies enter the trial headline.

Parent-category trials query:

"myeloid leukemia"

Query health: ok — strategies attempted: phrase; with hits: phrase

Run this search on ClinicalTrials.gov

Synonyms dropped by stoplist: CML

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

Ingested 2026-07-26T14:10:33.328Z