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Disease

Myelodysplastic syndrome

Late-stage therapeutic development
Also known as MDS, MDS, unclassifiable, MDS-U, Myelodysplastic Syndromes+14 more

MDS, MDS, unclassifiable, MDS-U, Myelodysplastic Syndromes, dysmyelopoietic syndrome, hematopoeitic - myelodysplastic syndrome (MDS), myelodysplasia, myelodysplastic neoplasm, myelodysplastic syndrome, somatic, myelodysplastic syndrome, unclassifiable, myelodysplastic syndrome/neoplasm, myelodysplastic syndromes, oligoblastic leukaemia, oligoblastic leukemia, preleukemia, smoldering leukemia, smouldering leukaemia, myelodysplastic syndrome, susceptibility to.

24
Clinical trials
12
Associated genes
12
Related proteins
Current focus
Cereblon biologyErythropoietin biologyTherapeutic development
Latest activity
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Recent clinical, regulatory, research and industry developments relating to this disease.

A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies

Clinical trial2026-08-20Status changed to Active, not recruiting · ClinicalTrials.gov

A Pilot Study of Liposomal Cytarabine and Daunorubicin (CPX-351) in Combination With Gemtuzumab Ozogamicin (GO) in Relapsed Refractory Patients With Acute Myeloid Leukemia (AML) and Post-Hypomethylating Agent (Post-HMA) Failure High-Risk Myelodysplastic Syndrome (HR-MDS)

Clinical trial2026-07-24Status changed to Completed · ClinicalTrials.gov

A Randomized Phase II Study Evaluating the Efficacy of Hyperbaric Oxygen in Improving Engraftment in Umbilical Cord Blood Stem Cell Transplantation

Clinical trial2026-07-20Status changed to Active, not recruiting · ClinicalTrials.gov

Haploidentical Allogeneic Peripheral Blood Transplantation: Clinical Trial and Laboratory Correlates Examining Checkpoint Immune Regulators' Expression

Clinical trial2026-07-17Status changed to Completed · ClinicalTrials.gov

Phase 1b Study of Pembrolizumab, Decitabine +/- Venetoclax Combination Therapy in Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

Clinical trial2026-07-14Status changed to Suspended · ClinicalTrials.gov

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 2 clinical trials expected to report results, the earliest in Q1 2028.
  • 1 industry development reported.
Upcoming Milestones
Clinical Milestones9View all 9
Results posted 1
Results expected 2
Recently completed 4
Other updates 2
+1 more in the activity timeline below
Industry & Market1View
Activity timeline10

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Imetelstatapproved

Approval — Treatment of transfusion-dependent anaemia in adults with low- to intermediate-1 risk mye… (2025)

Pegfilgrastimapproved

Approval — Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adul… (2025)

Azacitidineapproved

Approval — Azacitidine Kabi is indicated for the treatment of adult patients who are not eligible fo… (2024)

Efbemalenograstim alfaapproved

Approval — Ryzneuta is indicated for the reduction in the duration of neutropenia and the incidence… (2024)

Lenalidomideapproved

Approval — Multiple myelomaLenalidomide Krka as monotherapy is indicated for the maintenance treatme… (2021)

Luspaterceptapproved

Approval — Reblozyl is indicated in adults for the treatment of transfusion-dependent anaemia due to… (2020)

Clinical trials

13 sponsors · 0 new · 1 completed in the last 12 months (net -1)

The current development programme across all trial phases.

Clinical programme
24
All trials
6
Active
13
Late-stage
6
Completed
Late-stage studies

A Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled Study of Ivosidenib or Enasidenib in Combination With Induction Therapy and Consolidation Therapy Followed by Maintenance Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome With Excess Blasts-2, With an IDH1 or IDH2 Mutation, Respectively, Eligible for Intensive Chemotherapy.

Phase 3 · Active, not recruiting · Stichting Hemato-Oncologie voor Volwassenen Nederland

A Phase 3, Open-label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) vs Epoetin Alfa for the Treatment of Anemia Due to Revised International Prognostic Scoring System (IPSS-R) Very Low, Low, or Intermediate-Risk Myelodysplastic Syndrome (MDS) in Erythropoiesis-Stimulating Agent (ESA)-Naive Participants Who Are Non-Transfusion Dependent (NTD): The "ELEMENT-MDS" Trial

Phase 3 · Active, not recruiting · Bristol-Myers Squibb

A Phase 3, Multicenter, Open-label, Randomized, Study of Gilteritinib Versus Midostaurin in Combination With Induction and Consolidation Therapy Followed by One-year Maintenance in Patients With Newly Diagnosed Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes With Excess Blasts-2 (MDS-EB2) With FLT3 Mutations Eligible for Intensive Chemotherapy (HOVON 156 AML / AMLSG 28-18)

Phase 3 · Active, not recruiting · Stichting Hemato-Oncologie voor Volwassenen Nederland

A Phase 2/3, Multicenter, Randomized, Dose Optimization (Part I), Double-blind (Part II) Study to Compare the Efficacy and Safety of Oral Azacitidine (Oral-Aza, ONUREG®) Plus Best Supportive Care (BSC) Versus Placebo Plus BSC in Participants With IPSS-R Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)

Phase 2/3 · Active, not recruiting · Bristol-Myers Squibb

A Phase 3b, Open-label Study Evaluating the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) Initiated at Maximum Approved Dose in LR-MDS With IPSS-R Very Low-, Low-, or Intermediate-risk Who Require RBC Transfusions (MAXILUS)

Phase 3 · Active, not recruiting · Bristol-Myers Squibb

A Phase III Randomized Study of Farnesyl Transferase Inhibitor R115777 in Acute Myeloid Leukemia (AML) Patients in Second or Subsequent Remission or in Remission After Primary Induction Failure or Patients Over Age 60 in First Remission

Phase 3 · Completed · National Cancer Institute (NCI)
Recently completed

Efficacy and Safety of Ustekinumab for Intestinal Behçet's Syndrome Complicated by Myelodysplastic Syndrome

Phase 2 · Completed · Liu Tian

A Phase III Randomized Study of Farnesyl Transferase Inhibitor R115777 in Acute Myeloid Leukemia (AML) Patients in Second or Subsequent Remission or in Remission After Primary Induction Failure or Patients Over Age 60 in First Remission

Phase 3 · Completed · National Cancer Institute (NCI)

A Multi-Center Study of Conditioning With Treosulfan, Fludarabine and Escalating Doses of TBI for Allogeneic Hematopoietic Cell Transplantation in Patients With Acute Myeloid Leukemia (AML) Myelodysplastic Syndrome (MDS), and Acute Lymphoblastic Leukemia (ALL)

Phase 2 · Completed · Fred Hutchinson Cancer Center

A Programme of Treatment Development for Older Patients With Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome

Phase 2/3 · Completed · The University of New South Wales

A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 in Patients Diagnosed With Low and INT-1 Risk Myelodysplastic Syndrome (MDS) and Transfusion-dependent Iron Overload

Phase 4 · Completed · Novartis Pharmaceuticals

A PHASE III STUDY IN CHILDREN WITH UNTREATED ACUTE MYELOGENOUS LEUKEMIA (AML) OR MYELODYSPLASTIC SYNDROME (MDS)

Phase 3 · Completed · National Cancer Institute (NCI)

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2025emaApproval — Pegfilgrastim· Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2025emaApproval — Pegfilgrastim· Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2025emaApproval — Imetelstat· Treatment of transfusion-dependent anaemia in adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have failed to respond or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA). source ↗
2024emaApproval — Efbemalenograstim alfa· Ryzneuta is indicated for the reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2024emaApproval — Azacitidine· Azacitidine Kabi is indicated for the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation(HSCT) with: Intermediate-2 and high-risk myelodysplastic syndromes (MDS) according to the international prognostic scoring system (IPSS), Chronic myelomonocytic leukaemia (CMML) with 10-29% marrow blasts without myeloproliferative disorder, Acute myeloid leukaemia (AML) with 20-30% blasts and multi-lineage dysplasia, according to World Health Organization (WHO) classification, AML with > 30% marrow blasts according to the WHO classification. source ↗
2021emaApproval — Lenalidomide· Multiple myelomaLenalidomide Krka as monotherapy is indicated for the maintenance treatment of adult patients with newly diagnosed multiple myeloma who have undergone autologous stem cell transplantation. Lenalidomide Krka as combination therapy with dexamethasone, or bortezomib and dexamethasone, or melphalan and prednisone (see section 4.2) is indicated for the treatment of adult patients with previously untreated multiple myeloma who are not eligible for transplant. Lenalidomide Krka in combination with dexamethasone is indicated for the treatment of multiple myeloma in adult patients who have received at least one prior therapy. Myelodysplastic syndromesLenalidomide Krka as monotherapy is indicated for the treatment of adult patients with transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.Mantle cell lymphomaLenalidomide Krka as monotherapy is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (see sections 4.4 and 5.1). Follicular lymphomaLenalidomide Krka in combination with rituximab (anti-CD20 antibody) is indicated for the treatment of adult patients with previously treated follicular lymphoma (Grade 1 – 3a). Multiple myelomaLenalidomide Krka as monotherapy is indicated for the maintenance treatment of adult patients with newly diagnosed multiple myeloma who have undergone autologous stem cell transplantation. Lenalidomide Krka as combination therapy with dexamethasone, or bortezomib and dexamethasone, or melphalan and prednisone (see section 4.2) is indicated for the treatment of adult patients with previously untreated multiple myeloma who are not eligible for transplant. Lenalidomide Krka in combination with dexamethasone is indicated for the treatment of multiple myeloma in adult patients who have received at least one prior therapy. Myelodysplastic syndromesLenalidomide Krka as monotherapy is indicated for the treatment of adult patients with transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate. Mantle cell lymphomaLenalidomide Krka as monotherapy is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (see sections 4.4 and 5.1). Follicular lymphomaLenalidomide Krka in combination with rituximab (anti-CD20 antibody) is indicated for the treatment of adult patients with previously treated follicular lymphoma (Grade 1 – 3a). source ↗
2020emaApproval — Pegfilgrastim· Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2020emaApproval — Luspatercept· Reblozyl is indicated in adults for the treatment of transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS).Reblozyl is indicated in adults for the treatment of anaemia associated with transfusion dependent and non transfusion dependent beta thalassaemia.  Reblozyl is indicated in adults for the treatment of transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS).Reblozyl is indicated in adults for the treatment of anaemia associated with transfusion dependent and non transfusion dependent beta thalassaemia.  source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

TET20.80GATA20.77ASXL10.76DNMT3A0.74SRSF20.68DDX410.68SETBP10.66CSF3R0.64SF3B10.64DNMT10.62TGFB10.58CRBN0.57

Disease biology

12 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

DNA (cytosine-5)-methyltransferase 3Avia DNMT3A0.74Granulocyte colony-stimulating factor receptorvia CSF3R0.64DNA (cytosine-5)-methyltransferase 1via DNMT10.62Transforming growth factor beta-1 proproteinvia TGFB10.58Protein cereblonvia CRBN0.57Erythropoietin receptorvia EPOR0.57DNA damage-binding protein 1via DDB10.56E3 ubiquitin-protein ligase RBX1via RBX10.56Transforming growth factor beta-2 proproteinvia TGFB20.56DNA polymerase delta subunit 3via POLD30.52Breakpoint cluster region proteinvia BCR0.52Cytidine deaminasevia CDA0.51

Reference

Authoritative identity, definition & identifiers.

Synonyms

MDS, MDS, unclassifiable, MDS-U, Myelodysplastic Syndromes, dysmyelopoietic syndrome, hematopoeitic - myelodysplastic syndrome (MDS), myelodysplasia, myelodysplastic neoplasm, myelodysplastic syndrome, somatic, myelodysplastic syndrome, unclassifiable, myelodysplastic syndrome/neoplasm, myelodysplastic syndromes, oligoblastic leukaemia, oligoblastic leukemia, preleukemia, smoldering leukemia, smouldering leukaemia, myelodysplastic syndrome, susceptibility to

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.