ORPHA:86849
Acute basophilic leukemia
Clinical definition (Orphanet)
A rare acute myeloid leukemia characterized by primary differentiation to basophils. Microscopically, peripheral blood and bone marrow blasts contain coarse cytoplasmic basophilic granules which are positive with metachromatic staining (toluidine blue). Electron microscopy confirms that granules show features characteristic of basophil precursors. Mature basophils are usually sparse. Patients may present with manifestations related to bone marrow failure, as well as , cutaneous involvement, lytic lesions, and hyperhistaminemia. The disease is associated with a poor prognosis.
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?
4,679
4,679 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.
4,679 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).
1,389 in the last 10 years · low confidence
Is a treatment being tested?
22
trials for this specific condition
22 interventional trials matched this specific condition name; none in our sample are currently recruiting.
Data as of 26 July 2026
22 interventional trials — more than 59.2% of diseases in the trials denominator have none at all (151 of 255; this disease is at the 91.4th percentile).
low confidence · 91.4th 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?
1,150
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.
- 01Zhang Y7 papers · 2026
Division of Rheumatology, Allergy, and Immunology, University of North Carolina at Chapel Hill, Chapel Hill, NC; Thurston Arthritis Research Center, University of North Carolina at Chapel Hill, Chapel Hill, NC; Division of Allergy and Immunology, Department of Pediatrics, Food Allergy Initiative, University of North Carolina at Chapel Hill, Chapel Hill, NC.
Papers in Europe PMC - 02Suzuki R6 papers · 2026
Graduate School of Pharmaceutical Sciences, Nagoya City University, Nagoya, Japan.
Papers in Europe PMC - 03Wang T6 papers · 2024
College of Tourism and Culinary Science, Yangzhou University, Yangzhou, Jiangsu 225127, China.
Papers in Europe PMC - 04Furuno T5 papers · 2026
School of Pharmacy, Aichi Gakuin University, 1-100 Kusumoto-cho, Chikusa-ku, Nagoya, 464-8650, Japan. furuno@dpc.agu.ac.jp.
Papers in Europe PMC - 05Jiang S5 papers · 2024
College of Tourism and Culinary Science, Yangzhou University, Yangzhou, Jiangsu 225127, China. Electronic address: 17600189209@163.com.
Papers in Europe PMC - 06Liu J5 papers · 2026
Northeast Asia Institute of Traditional Chinese Medicine, Changchun University of Chinese Medicine, Changchun 130117, China.
Papers in Europe PMC - 07Chen X4 papers · 2025
State Key Laboratory of Food Science and Resources, Nanchang University, Nanchang 330047, China.
Papers in Europe PMC - 08Han S4 papers · 2026
School of Pharmacy, Xi'an Jiaotong University, Xi'an, China.
Papers in Europe PMC - 09Huang Y4 papers · 2024
Department of Pediatrics, Peking University First Hospital, Beijing, China.
Papers in Europe PMC - 10Li X4 papers · 2026
State Key Laboratory of Food Science and Resources, Nanchang University, Nanchang 330047, China.
Papers in Europe PMC
Recruiting interventional trials
Trials testing a treatment from the matched ClinicalTrials.gov set
22 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
1 observational study matches 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 basophilic leukemia" OR "basophilic leukaemia" OR "basophilic leukemia" OR "leukaemia basophilic" OR "leukemia basophilic"
MeSH descriptor terms unioned into the query: Leukemia, Basophilic, Acute
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
"Acute basophilic leukemia" OR "basophilic leukaemia" OR "basophilic leukemia" OR "leukaemia basophilic" OR "leukemia basophilic" OR "Leukemia, Basophilic, Acute"
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: 22 interventional · 1 observational · 0 expanded access. Only interventional studies enter the trial headline.
Cross-references (from Mondo): MESH:D015471 UMLS:C0023437 NCIT:C3164
Query health: ok — strategies attempted: phrase, mesh; with hits: phrase, mesh
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 (4679) is implausibly high for prevalence class "<1 / 1 000 000" — treat as possible over-matching, not a measure of research intensity