Myelodysplastic/myeloproliferative neoplasm

Summary

Myelodysplastic/myeloproliferative neoplasm (MONDO:0006311) is a cancer and 193 clinical trials. Top therapeutic interventions include azacitidine, busulfan, and alemtuzumab. A subtype of myeloproliferative neoplasm — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

• Classification: Cancer
• Clinical trials: 193

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	myelodysplastic/myeloproliferative neoplasm
Mondo ID	MONDO:0006311
EFO	EFO:1000388
DOID	DOID:4972
NCIT	C27262
SNOMED CT	445738007
UMLS	C1301355
MedGen	226981
GARD	0024368
Is cancer (heuristic)	yes

Also known as: MDS-MPD · MDS/MPD · MDS/MPN · MPD-MDS · MPD/MDS · myelodysplastic myeloproliferative disease · myelodysplastic/myeloproliferative disease · myelodysplastic/myeloproliferative diseases · myelodysplastic/myeloproliferative disorder · myelodysplastic/myeloproliferative disorders · myelodysplastic/myeloproliferative neoplasm · myelodysplastic/myeloproliferative neoplasms · myeloproliferative/myelodysplastic disorders · myeloproliferative/myelodysplastic syndromes

Disease family

This is a subtype of myeloproliferative neoplasm. Genetic, therapeutic, and trial evidence is largely curated at the broader-term level — see the parent page for the associated-gene cohort and molecular evidence.

Classification path: disease › human disease › disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › hematopoietic and lymphoid system neoplasm › hematopoietic and lymphoid cell neoplasm › myeloid neoplasm › myeloproliferative neoplasm › myelodysplastic/myeloproliferative neoplasm

Related subtypes (12): myeloid leukemia, essential thrombocythemia, therapy-related myeloid neoplasm, transient myeloproliferative syndrome, primary myelofibrosis, myeloproliferative disease, autosomal recessive, thrombocytopenia 6, chronic eosinophilic leukemia, chronic neutrophilic leukemia, myeloproliferative neoplasm, unclassifiable, erythroid neoplasm, myelofibrosis

Subtypes (2): atypical chronic myeloid leukemia, BCR-ABL1 negative, chronic myelomonocytic leukemia

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

No tiered GWAS variants or ClinVar records for this disease.

Genes & proteins

No associated-gene cohort resolved for this disease. Atlas builds the molecular and therapeutic sections — associated genes, protein families, druggability, pathways, interactions, and drug associations — by aggregating over a disease’s associated genes (resolved via GWAS / GenCC / ClinVar / CIViC), and none resolved here. This is expected for antibody-mediated, autoimmune, or otherwise non-gene-defined conditions; the curated evidence for this disease is its clinical features, GWAS susceptibility, and clinical trials (above).

Function

No pathway enrichment — requires an associated-gene cohort.

Therapeutics

Drugs indicated or in trials for this disease

No drug has an approved disease-direct ChEMBL indication for this disease.

11 drugs in clinical trials for this disease (phase 2–3, investigational): efficacy not established — a trial record, not an indication.

Drug	Highest phase
Bexarotene	Phase 2
Busulfan	Phase 2
Cyclosporine	Phase 2
Filgrastim	Phase 2
Fludarabine Phosphate	Phase 2
Human Immunoglobulin G	Phase 2
Melphalan	Phase 2
Methotrexate	Phase 2
Methylprednisolone	Phase 2
Sargramostim	Phase 2
Tacrolimus Anhydrous	Phase 2

Clinical trials & evidence

Clinical trials

Clinical trials: 193.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	76
PHASE1	40
Not specified	39
PHASE1/PHASE2	21
PHASE3	15
PHASE2/PHASE3	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05153226	PHASE3	ACTIVE_NOT_RECRUITING	GvHD Prophylaxis in Unrelated Donor HCT: Randomized Trial Comparing PTCY Versus ATG
NCT00003593	PHASE3	COMPLETED	Chemotherapy in Treating Children With Down Syndrome and Myeloproliferative Disorder, Acute Myelogenous Leukemia, or Myelodysplastic Syndrome
NCT00003816	PHASE2/PHASE3	COMPLETED	Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer
NCT00005823	PHASE3	COMPLETED	Intensive Compared With Nonintensive Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome
NCT00005863	PHASE3	COMPLETED	Combination Chemotherapy With or Without Filgrastim and/or Tretinoin in Treating Patients With Acute Myeloid Leukemia
NCT00043134	PHASE3	UNKNOWN	Low-Dose Decitabine Compared With Standard Supportive Care in Treating Older Patients With Myelodysplastic Syndrome
NCT00047268	PHASE3	UNKNOWN	Donor Stem Cell Transplant With or Without Chemotherapy in Treating Children With Primary Myelodysplastic Syndrome
NCT00075478	PHASE3	COMPLETED	Total-Body Irradiation With or Without Fludarabine Phosphate Followed By Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer
NCT00265837	PHASE3	COMPLETED	Laboratory-Treated Donor Bone Marrow in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Hematologic Cancer
NCT00324324	PHASE3	TERMINATED	Moxifloxacin in Preventing Bacterial Infections in Patients Who Have Undergone Donor Stem Cell Transplant
NCT00516503	PHASE3	COMPLETED	Baclofen-Amitriptyline Hydrochloride-Ketamine Gel in Treating Peripheral Neuropathy Caused by Chemotherapy in Patients With Cancer
NCT00666211	PHASE3	COMPLETED	Opioid Titration Order Sheet or Standard Care in Treating Patients With Cancer Pain
NCT00719563	PHASE3	COMPLETED	American Ginseng in Treating Patients With Fatigue Caused by Cancer
NCT00750009	PHASE3	COMPLETED	Personalized Information or Basic Information in Helping Patients Make Decisions About Participating in a Clinical Trial
NCT00755040	PHASE3	COMPLETED	Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder
NCT00782145	PHASE3	COMPLETED	A Web-Based Stem Cell Transplant Support System or Standard Care in Young Patients Undergoing Stem Cell Transplant and Their Families
NCT00544115	PHASE2	ACTIVE_NOT_RECRUITING	Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders
NCT02727803	PHASE2	RECRUITING	Personalized NK Cell Therapy in CBT
NCT02861417	PHASE2	ACTIVE_NOT_RECRUITING	Busulfan, Fludarabine Phosphate, and Post-Transplant Cyclophosphamide in Treating Patients With Blood Cancer Undergoing Donor Stem Cell Transplant
NCT03289910	PHASE2	ACTIVE_NOT_RECRUITING	Topotecan Hydrochloride and Carboplatin With or Without Veliparib in Treating Advanced Myeloproliferative Disorders and Acute Myeloid Leukemia or Chronic Myelomonocytic Leukemia
NCT04061421	PHASE1/PHASE2	RECRUITING	Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes Overlap Syndromes (ABNL-MARRO)
NCT04140487	PHASE1/PHASE2	RECRUITING	Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, or High-Risk Myelodysplastic Syndrome/Myeloproliferative Neoplasm
NCT04282187	PHASE2	RECRUITING	Decitabine With Ruxolitinib, Fedratinib or Pacritinib for the Treatment of Accelerated/Blast Phase Myeloproliferative Neoplasms
NCT04771130	PHASE1/PHASE2	RECRUITING	A Study of BGB-11417 in Participants With Myeloid Malignancies
NCT05038592	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Phase I/II Study of Tagraxofusp in Combination With Decitabine for Patients With Myelomonocytic/Myeloproliferative Neoplasm and High Risk Myelodysplastic Syndromes
NCT05600894	PHASE2	ACTIVE_NOT_RECRUITING	Venetoclax in Combination With ASTX727 for the Treatment of Chronic Myelomonocytic Leukemia and Other Myelodysplastic Syndrome/Myeloproliferative Neoplasm
NCT05817955	PHASE2	RECRUITING	Clinical Study of Azacitidine Combined With Ruxolitinib in the Treatment of Higher-risk MDS/MPN
NCT06523556	PHASE1/PHASE2	RECRUITING	Axatilimab With or Without Azacitidine for the Treatment of Patients With Advanced Phase Myeloproliferative Neoplasms, Myeloproliferative Neoplasm/Myelodysplastic Syndrome Overlap or High Risk Chronic Myelomonocytic Leukemia
NCT06599762	PHASE2	RECRUITING	Evaluation of Tranexamic Acid in Myelodysplastic Syndromes and Acute Myeloid Leukemia
NCT07228624	PHASE2	RECRUITING	Ruxolitinib Before, During and After Hematopoietic Cell Transplant in Older Patients With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes
NCT07468916	PHASE2	NOT_YET_RECRUITING	Ropeginterferon Alfa-2b for the Treatment of Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes and Chronic Myelomonocytic Leukemia
NCT07612280	PHASE1/PHASE2	RECRUITING	Phase 1/ Phase 2 Study to Assess Safety and Efficacy of Orally Administered JBI-802 in Subjects With Myeloproliferative Neoplasms (MPN) and Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN) With Thrombocytosis
NCT00002718	PHASE2	COMPLETED	T-cell Depleted Bone Marrow and G-CSF Stimulated Peripheral Stem Cell Transplantation From Related Donors in Treating Patients With Leukemia, Lymphoblastic Lymphoma, Myelodysplastic Syndrome, or Aplastic Anemia
NCT00002792	PHASE2	COMPLETED	Combination Chemotherapy Plus Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Myeloproliferative Disorders
NCT00002809	PHASE2	COMPLETED	Bone Marrow Transplant Plus Cyclophosphamide and Total-Body Irradiation in Treating Patients With Hematologic Cancer
NCT00003116	PHASE2	COMPLETED	High-Dose Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Patients With Hematologic Cancer
NCT00003335	PHASE2	COMPLETED	Umbilical Cord Blood Transplantation in Treating Patients With High-Risk Hematologic Cancer
NCT00003336	PHASE2	COMPLETED	Pilot Study Of Unrelated UCB Transplant for Non-Malignant Hematologic Conditions
NCT00003913	PHASE2	COMPLETED	Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Nonmalignant Hematologic Disease
NCT00003961	PHASE2	COMPLETED	Sargramostim After Bone Marrow Transplantation in Treating Patients With Myelodysplastic Syndrome

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
AZACITIDINE	4	7
BUSULFAN	4	6
ALEMTUZUMAB	4	4
ARSENIC TRIOXIDE	4	4
CYTARABINE	4	4
PHENYLBUTANOIC ACID	4	4
THIOTEPA	4	4
AMITRIPTYLINE	4	3
DAUNORUBICIN HYDROCHLORIDE	4	3
ETOPOSIDE	4	3
FLUDARABINE PHOSPHATE	4	3
MERCAPTOPURINE ANHYDROUS	4	3
CEDAZURIDINE	4	2
EDETATE CALCIUM DISODIUM MONOHYDRATE	4	2
GANCICLOVIR	4	2
HYDROXYUREA	4	2
MOXIFLOXACIN HYDROCHLORIDE	4	2
PENTOSTATIN	4	2
ROMIDEPSIN	4	2
RUXOLITINIB PHOSPHATE	4	2
SARGRAMOSTIM	4	2
THIOGUANINE	4	2
TOPOTECAN HYDROCHLORIDE	4	2
TRETINOIN	4	2
ALCOHOL	4	1
AMIFOSTINE	4	1
ASPARAGINASE	4	1
ATORVASTATIN CALCIUM	4	1
AXATILIMAB	4	1
BACLOFEN	4	1