RepurpOS

Polycythaemia Vera / Myeloproliferative Neoplasms

Disease intelligence for Polycythemia Vera: 144 alterations and 277 ranked therapeutics from curated public databases.

US DALYs606.6KWHO GHE 2021 · all ages
Global DALYs11.04MWHO GHE 2021 · worldwide
US mortality26.9K deaths/yrWHO GHE 2021
Global mortality311.3K deaths/yrWHO GHE 2021 · worldwide
Spontaneous remissionPV/ET/MF: no spontaneous remission; progressive disorders with risk of transformation to MF (PV/ET) and blast phase…
Best-intervention remissionJAK2 inhibitor (ruxolitinib) for MF/PV: 35-42% spleen volume reduction, significant symptom improvement…
Gap sizeModerate — ruxolitinib is established but HSCT underused in eligible MF patients; ropeginterferon is superior in PV…
Primary barrieraccess/practice
Full remission profile →

Disease overview

Alterations
144
Molecular (A)
100
Clinical (B–E)
44
Direct drugs
53
Via biomarker
224
Natural agents
0
Natural products
0
Merged ranked
277
With trial/literature evidence
25

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, Seeds, GreenMedInfo · Pipeline phase 7 · Updated 2026-07-06

Remission & chronicity

Spontaneous remission
PV/ET/MF: no spontaneous remission; progressive disorders with risk of transformation to MF (PV/ET) and blast phase (all); median survival PV >20 years, MF 5-7 years
Best-intervention remission
JAK2 inhibitor (ruxolitinib) for MF/PV: 35-42% spleen volume reduction, significant symptom improvement; ropeginterferon alfa-2b: superior complete haematological response vs hydroxyurea (PROUD-PV); allogeneic HSCT (MF): curative in 30-60% of transplant-eligible patients
Drug-free remission
Relapse after remission
Chronicity
Gap size
Moderate — ruxolitinib is established but HSCT underused in eligible MF patients; ropeginterferon is superior in PV but access varies
Primary barrier
access/practice
Barrier detail: Hydroxyurea remains the most common cytoreductive agent for PV/ET despite ropeginterferon demonstrating superior molecular response; HSCT for MF is underutilised in patients who could benefit; JAK2 mutation molecular monitoring not performed systematically

Last SoC change: 2022 — ropeginterferon alfa-2b (Besremi) FDA approved for PV; 2019 fedratinib (JAK2/FLT3) for MF; pacritinib 2022 for thrombocytopenic MF; ruxolitinib 2011 for MF, 2014 for PV; hydroxyurea standard since 1960s

Remission data layers: disease_db_100 · source-locked manual data · confidence: high

Identifiers: MONDO: MONDO:0009891

Alterations

Showing 80 of 144 · Types A (molecular) through E (functional)

NameTypeSubtypeDirection FrequencyEvidenceSourcesLinks
Abdominal pain Pathology Pathological finding Moderate HPO, Monarch
Abnormal thrombosis Pathology Pathological finding Moderate HPO, Monarch
Abnormality of vision Pathology Pathological finding Moderate HPO, Monarch
Acute leukemia Pathology Pathological finding Moderate HPO, Monarch
Angina pectoris Pathology Pathological finding Moderate HPO, Monarch
Arterial thrombosis Pathology Pathological finding Moderate HPO, Monarch
Arthralgia Pathology Pathological finding Moderate HPO, Monarch
Bruising susceptibility Pathology Pathological finding Moderate HPO, Monarch
Budd-Chiari syndrome Pathology Pathological finding Moderate HPO, Monarch
Cerebral hemorrhage Pathology Pathological finding Moderate HPO, Monarch
Cerebral ischemia Pathology Pathological finding Moderate HPO, Monarch
Early satiety Pathology Pathological finding Moderate HPO, Monarch
Epistaxis Pathology Pathological finding Moderate HPO, Monarch
Erythromelalgia Pathology Pathological finding Moderate HPO, Monarch
Fatigue Functional Functional test Moderate HPO, Monarch
Gastrointestinal hemorrhage Pathology Pathological finding Moderate HPO, Monarch
Gingival bleeding Pathology Pathological finding Moderate HPO, Monarch
Headache Pathology Pathological finding Moderate HPO, Monarch
Hepatomegaly Pathology Pathological finding Moderate HPO, Monarch
Hypertension Pathology Pathological finding Moderate HPO, Monarch
Increased circulating hemoglobin concentration Lab / Clinical Lab value Moderate HPO, Monarch
Increased hematocrit Pathology Pathological finding Moderate HPO, Monarch
Increased megakaryocyte count Pathology Pathological finding Moderate HPO, Monarch
Increased red blood cell mass Pathology Pathological finding Moderate HPO, Monarch
Increased total leukocyte count Pathology Pathological finding Moderate HPO, Monarch
Intermittent claudication Pathology Pathological finding Moderate HPO, Monarch
Myelodysplasia Pathology Pathological finding Moderate HPO, Monarch
Myelofibrosis Pathology Pathological finding Moderate HPO, Monarch
Paresthesia Pathology Pathological finding Moderate HPO, Monarch
Polycythemia Pathology Pathological finding Moderate HPO, Monarch
Portal hypertension Pathology Pathological finding Moderate HPO, Monarch
Portal vein thrombosis Pathology Pathological finding Moderate HPO, Monarch
Pruritus Pathology Pathological finding Moderate HPO, Monarch
Pulmonary embolism Pathology Pathological finding Moderate HPO, Monarch
Respiratory insufficiency Pathology Pathological finding Moderate HPO, Monarch
Splenomegaly Pathology Pathological finding Moderate HPO, Monarch
Stroke Pathology Pathological finding Moderate HPO, Monarch
Thrombocytopenia Pathology Pathological finding Moderate HPO, Monarch
Thrombocytosis Pathology Pathological finding Moderate HPO, Monarch
Thromboembolism Pathology Pathological finding Moderate HPO, Monarch
Tinnitus Pathology Pathological finding Moderate HPO, Monarch
Venous thrombosis Pathology Pathological finding Moderate HPO, Monarch
Vertigo Pathology Pathological finding Moderate HPO, Monarch
Weight loss Pathology Pathological finding Moderate HPO, Monarch
ABHD6
abhydrolase domain containing 6, acylglycerol lipase
Molecular Gene Emerging Open Targets
ABL1
ABL proto-oncogene 1, non-receptor tyrosine kinase
Molecular Gene Emerging Open Targets
ACVR1
activin A receptor type 1
Molecular Gene Emerging Open Targets
AFG1L
AFG1 like ATPase
Molecular Gene Emerging Open Targets
ALK
ALK receptor tyrosine kinase
Molecular Gene Emerging Open Targets
APC
APC regulator of Wnt signaling pathway
Molecular Gene Emerging Open Targets
ASAP1
ArfGAP with SH3 domain, ankyrin repeat and PH domain 1
Molecular Gene Emerging Open Targets
ASXL1
ASXL transcriptional regulator 1
Molecular Gene Emerging Open Targets
ATG10
autophagy related 10
Molecular Gene Emerging Open Targets
ATM
ATM serine/threonine kinase
Molecular Gene Emerging Open Targets
BCOR
BCL6 corepressor
Molecular Gene Emerging Open Targets
BCR
BCR activator of RhoGEF and GTPase
Molecular Gene Emerging Open Targets
BRAF
B-Raf proto-oncogene, serine/threonine kinase
Molecular Gene Emerging Open Targets
CALR
calreticulin
Molecular Gene Emerging Open Targets
CBL
Cbl proto-oncogene
Molecular Gene Emerging Open Targets
CELF2
CUGBP Elav-like family member 2
Molecular Gene Emerging Open Targets
CHEK2
checkpoint kinase 2
Molecular Gene Emerging Open Targets
CLMB
calcimembrin
Molecular Gene Emerging Open Targets
COL19A1
collagen type XIX alpha 1 chain
Molecular Gene Emerging Open Targets
DNMT3A
DNA methyltransferase 3 alpha
Molecular Gene Emerging Open Targets
DPP8
dipeptidyl peptidase 8
Molecular Gene Emerging Open Targets
DPT
dermatopontin
Molecular Gene Emerging Open Targets
EP300
EP300 lysine acetyltransferase
Molecular Gene Emerging Open Targets
EPAS1
endothelial PAS domain protein 1
Molecular Gene Emerging Open Targets
EZH2
enhancer of zeste 2 polycomb repressive complex 2 subunit
Molecular Gene Emerging Open Targets
FAT4
FAT atypical cadherin 4
Molecular Gene Emerging Open Targets
FGFR2
fibroblast growth factor receptor 2
Molecular Gene Emerging Open Targets
FLT3
fms related receptor tyrosine kinase 3
Molecular Gene Emerging Open Targets
GATA2
GATA binding protein 2
Molecular Gene Emerging Open Targets
GATA3
GATA binding protein 3
Molecular Gene Emerging Open Targets
GFI1B
growth factor independent 1B transcriptional repressor
Molecular Gene Emerging Open Targets
GNAS
GNAS complex locus
Molecular Gene Emerging Open Targets
GYPA
glycophorin A (MNS blood group)
Molecular Gene Emerging Open Targets
HDAC1
histone deacetylase 1
Molecular Gene Emerging Open Targets
HDAC10
histone deacetylase 10
Molecular Gene Emerging Open Targets
HDAC11
histone deacetylase 11
Molecular Gene Emerging Open Targets

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, merged ranking, and clinical trial / literature evidence (top agents)

DrugTypePhaseEvidence ScoreTrials & literatureSourcesLinks
ABEMACICLIB Repurposing Small molecule Approved Strong 95 DGIdb
ABIRATERONE ACETATE Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
ACETAZOLAMIDE Repurposing Small molecule Approved Strong 95 ChEMBL
ACYCLOVIR Repurposing Small molecule Approved Strong 95 DGIdb
ADENOSINE TRIPHOSPHATE Repurposing Small molecule Approved Strong 95 DGIdb
AFATINIB Repurposing Small molecule Approved Strong 95 DGIdb
ALECTINIB Repurposing Small molecule Approved Strong 95 DGIdb
ALECTINIB HYDROCHLORIDE Repurposing Small molecule Approved Strong 95 DGIdb
ALPELISIB Repurposing Small molecule Approved Strong 95 DGIdb
AMPHETAMINE Repurposing Small molecule Approved Strong 95 ChEMBL
ASCIMINIB Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
ASCIMINIB HYDROCHLORIDE Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
ATEZOLIZUMAB Repurposing Small molecule Approved Strong 95 DGIdb
AVAPRITINIB Repurposing Small molecule Approved Strong 95 DGIdb
AVELUMAB Repurposing Small molecule Approved Strong 95 DGIdb
AXITINIB Repurposing Small molecule Approved Strong 95 DGIdb
AZACITIDINE Repurposing Small molecule Approved Strong 95 DGIdb
BENDAMUSTINE Repurposing Small molecule Approved Strong 95 DGIdb
BEVACIZUMAB Repurposing Small molecule Approved Strong 95 DGIdb
BICALUTAMIDE Repurposing Small molecule Approved Strong 95 ChEMBL
BINIMETINIB Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
BLINATUMOMAB Repurposing Small molecule Approved Strong 95 DGIdb
BOSUTINIB Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
BOSUTINIB MONOHYDRATE Repurposing Small molecule Approved Strong 95 DGIdb
BRENTUXIMAB VEDOTIN Repurposing Small molecule Approved Strong 95 DGIdb
BRIGATINIB Repurposing Small molecule Approved Strong 95 DGIdb
BUSULFAN Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
CABOZANTINIB S-MALATE Repurposing Small molecule Approved Strong 95 DGIdb
CAMPTOTHECIN Repurposing Small molecule Approved Strong 95 DGIdb
CAPECITABINE Repurposing Small molecule Approved Strong 95 DGIdb
CAPMATINIB Repurposing Small molecule Approved Strong 95 DGIdb
CARBACHOL Repurposing Small molecule Approved Strong 95 ChEMBL
CARBAMAZEPINE Repurposing Small molecule Approved Strong 95 DGIdb
CELECOXIB Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
CEMIPLIMAB Repurposing Small molecule Approved Strong 95 DGIdb
CERITINIB Repurposing Small molecule Approved Strong 95 DGIdb
CETUXIMAB Repurposing Small molecule Approved Strong 95 DGIdb
CHLOROQUINE Repurposing Small molecule Approved Strong 95 DGIdb
CIPROFLOXACIN Repurposing Small molecule Approved Strong 95 ChEMBL
CISPLATIN Repurposing Small molecule Approved Strong 95 DGIdb
CLOMIPRAMINE Repurposing Small molecule Approved Strong 95 ChEMBL
COBIMETINIB Repurposing Small molecule Approved Strong 95 DGIdb
CRIZOTINIB Repurposing Small molecule Approved Strong 95 DGIdb
CYCLOPHOSPHAMIDE ANHYDROUS Repurposing Small molecule Approved Strong 95 DGIdb
CYTARABINE Repurposing Small molecule Approved Strong 95 DGIdb
DABRAFENIB Repurposing Small molecule Approved Strong 95 DGIdb
DABRAFENIB MESYLATE Repurposing Small molecule Approved Strong 95 DGIdb
DASATINIB ANHYDROUS Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
DECITABINE Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
DENOSUMAB Repurposing Small molecule Approved Strong 95 DGIdb
DrugTypePhaseEvidence ScoreSourcesLinks
ANAGRELIDE Small molecule Phase III Moderate 80 Open Targets
GIVINOSTAT Small molecule Phase III Moderate 80 Open Targets
HYDROXYUREA Small molecule Phase III Moderate 80 Open Targets
INTERFERON Biologic Phase III Moderate 80 Open Targets
INTERFERON ALFA Biologic Phase III Moderate 80 Open Targets
INTERFERON ALFA-2A Biologic Phase III Moderate 80 Open Targets
INTERFERON ALFA-2B Biologic Phase III Moderate 80 Open Targets
NOVAFERON Small molecule Phase III Moderate 80 Open Targets
PEGINTERFERON ALFA-2B Biologic Phase III Moderate 80 Open Targets
PIPOBROMAN Small molecule Phase III Moderate 80 Open Targets
RUSFERTIDE Biologic Phase III Moderate 80 Open Targets
SAPABLURSEN Small molecule Phase III Moderate 80 Open Targets
ASPIRIN Small molecule Phase II Emerging 60 Open Targets
BOMEDEMSTAT Small molecule Phase II Emerging 60 Open Targets
CLOPIDOGREL Small molecule Phase II Emerging 60 Open Targets
CYCLOPHOSPHAMIDE Small molecule Phase II Emerging 60 Open Targets
CYCLOSPORINE Biologic Phase II Emerging 60 Open Targets
DASATINIB Small molecule Phase II Emerging 60 Open Targets
DASATINIB ANHYDROUS Small molecule Phase II Emerging 60 Open Targets
DOXORUBICIN Small molecule Phase II Emerging 60 Open Targets
ERLOTINIB Small molecule Phase II Emerging 60 Open Targets
ETOPOSIDE Small molecule Phase II Emerging 60 Open Targets
FLUDARABINE Small molecule Phase II Emerging 60 Open Targets
GANDOTINIB Small molecule Phase II Emerging 60 Open Targets
IDASANUTLIN Small molecule Phase II Emerging 60 Open Targets
IMATINIB Small molecule Phase II Emerging 60 Open Targets
IMATINIB MESYLATE Small molecule Phase II Emerging 60 Open Targets
IMETELSTAT Small molecule Phase II Emerging 60 Open Targets
LESTAURTINIB Small molecule Phase II Emerging 60 Open Targets
LUMINESPIB Small molecule Phase II Emerging 60 Open Targets
METHOTREXATE Small molecule Phase II Emerging 60 Open Targets
MIRABEGRON Small molecule Phase II Emerging 60 Open Targets
MOMELOTINIB Small molecule Phase II Emerging 60 Open Targets
NAVTEMADLIN Small molecule Phase II Emerging 60 Open Targets
PREDNISONE Small molecule Phase II Emerging 60 Open Targets
RITUXIMAB Biologic Phase II Emerging 60 Open Targets
VINCRISTINE Small molecule Phase II Emerging 60 Open Targets
ZINPENTRAXIN ALFA Biologic Phase II Emerging 60 Open Targets
ACETYLCYSTEINE Small molecule Phase I Emerging 35 Open Targets
POMALIDOMIDE Small molecule Phase I Emerging 35 Open Targets
DrugTypePhaseEvidence ScoreTrials & literatureSourcesLinks
ABEMACICLIB Repurposing via BRAF Small molecule Approved Strong 95 DGIdb
ABIRATERONE ACETATE Repurposing via APC Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
ACETAZOLAMIDE Repurposing via ABHD6 Small molecule Approved Strong 95 ChEMBL
ACYCLOVIR Repurposing via ABL1 Small molecule Approved Strong 95 DGIdb
ADENOSINE TRIPHOSPHATE Repurposing via ABL1 Small molecule Approved Strong 95 DGIdb
AFATINIB Repurposing via BRAF Small molecule Approved Strong 95 DGIdb
ALECTINIB Repurposing via BRAF Small molecule Approved Strong 95 DGIdb
ALECTINIB HYDROCHLORIDE Repurposing via ALK Small molecule Approved Strong 95 DGIdb
ALPELISIB Repurposing via BRAF Small molecule Approved Strong 95 DGIdb
AMPHETAMINE Repurposing via ABHD6 Small molecule Approved Strong 95 ChEMBL
ASCIMINIB Repurposing via BCR Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
ASCIMINIB HYDROCHLORIDE Repurposing via BCR Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
ATEZOLIZUMAB Repurposing via BRAF Small molecule Approved Strong 95 DGIdb
AVAPRITINIB Repurposing via CBL Small molecule Approved Strong 95 DGIdb
AVELUMAB Repurposing via ATM Small molecule Approved Strong 95 DGIdb
AXITINIB Repurposing via BRAF Small molecule Approved Strong 95 DGIdb
AZACITIDINE Repurposing via ABL1 Small molecule Approved Strong 95 DGIdb
BENDAMUSTINE Repurposing via ATM Small molecule Approved Strong 95 DGIdb
BEVACIZUMAB Repurposing via BRAF Small molecule Approved Strong 95 DGIdb
BICALUTAMIDE Repurposing via ABHD6 Small molecule Approved Strong 95 ChEMBL
BINIMETINIB Repurposing via APC Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
BLINATUMOMAB Repurposing via BCR Small molecule Approved Strong 95 DGIdb
BOSUTINIB Repurposing via BCR Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
BOSUTINIB MONOHYDRATE Repurposing via BCR Small molecule Approved Strong 95 DGIdb
BRENTUXIMAB VEDOTIN Repurposing via ALK Small molecule Approved Strong 95 DGIdb
BRIGATINIB Repurposing via ALK Small molecule Approved Strong 95 DGIdb
BUSULFAN Repurposing via BCR Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
CABOZANTINIB S-MALATE Repurposing via ABL1 Small molecule Approved Strong 95 DGIdb
CAMPTOTHECIN Repurposing via ATM Small molecule Approved Strong 95 DGIdb
CAPECITABINE Repurposing via BRAF Small molecule Approved Strong 95 DGIdb
CAPMATINIB Repurposing via BRAF Small molecule Approved Strong 95 DGIdb
CARBACHOL Repurposing via ABHD6 Small molecule Approved Strong 95 ChEMBL
CARBAMAZEPINE Repurposing via ALK Small molecule Approved Strong 95 DGIdb
CELECOXIB Repurposing via APC Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
CEMIPLIMAB Repurposing via ALK Small molecule Approved Strong 95 DGIdb
CERITINIB Repurposing via ALK Small molecule Approved Strong 95 DGIdb
CETUXIMAB Repurposing via BRAF Small molecule Approved Strong 95 DGIdb
CHLOROQUINE Repurposing via BRAF Small molecule Approved Strong 95 DGIdb
CIPROFLOXACIN Repurposing via ABHD6 Small molecule Approved Strong 95 ChEMBL
CISPLATIN Repurposing via ALK Small molecule Approved Strong 95 DGIdb

Alterations and therapeutics are aggregated from public databases (Open Targets, HPO, ChEMBL, DGIdb, and others). Natural agents are candidates from OSMF narrative reviews on opensourcemed.info/chronic-disease.html — not approved treatments. This page is for research orientation only — not medical advice.

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