Myelodysplastic syndrome

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At a Glance

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Regulatory Watchboard

10 events
Apr 2026RYTELO: New indication approved
FDAcompleted
Jun 2025Eltrombopag as a Novel Therapeutic Approach for Low-risk MDS and CMML With TET2 Mutations

Abhay Singh, MD MPH — PHASE2

TrialRECRUITING
Mar 2025A Phase 1B/2A Trial of NADUNOLIMAB in Combination With Azacitidine (With/Without Venetoclax) in Patients With Myelodysplastic Syndrome (MDS) and Acute Myelogenous Leukemia (AML)

M.D. Anderson Cancer Center — PHASE1, PHASE2

TrialRECRUITING
Feb 2025Intensity Modulated Total Marrow Irradiation in Fully Human Leukocyte Antigen (HLA)-Matched and Partially-HLA Mismatched Allogeneic Transplantation Patients With High-Risk Acute Myeloid Leukemia (AML), Chronic Myeloid Leukemia (CML), and Myelodysplastic Syndrome (MDS)

University of Illinois at Chicago — PHASE2

TrialRECRUITING
Jun 2024RYTELO: FDA approved
FDAcompleted
Dec 2023A Phase 2a Study of HT-6184 in Subjects With IPSS-R Very Low, Low or Intermediate Risk MDS and Anemia

Halia Therapeutics, Inc. — PHASE2

TrialACTIVE NOT RECRUITING
Feb 2023Roll-over Study for Patients Who Have Completed a Prior Novartis-sponsored Sabatolimab (MBG453) Study and Are Judged by the Investigator to Benefit From Continued Treatment With Sabatolimab.

Novartis Pharmaceuticals — PHASE2

TrialACTIVE NOT RECRUITING
Jun 2021Involvement of CDA and/or dCK Metabolizing Enzymes in the Response to Azacytidine Treatment of Patients With Hematologic Malignancies

Assistance Publique Hopitaux De Marseille — NA

TrialRECRUITING
Sep 2020Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome

AbbVie — PHASE3

TrialACTIVE NOT RECRUITING
Jun 2011Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes

Associazione Qol-one — PHASE2

TrialACTIVE NOT RECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

2 programs

FINANCIAL LANDSCAPE SUMMARY

2

Total programs

1

Open now

2

Travel grants

Travel Grants2

BMS Access Support - ONUREG (azacitidine) Patient Assistance Program

Bristol-Myers Squibb

Contact for detailsApply ↗

Myelodysplastic syndrome medication copay assistance

PAN Foundation

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Approved Treatments

5 FDA-approved

Rytelo

(IMETELSTAT SODIUM)Orphan drug

Geron Corporation

12.1. Mechanism of Action Imetelstat is an oligonucleotide human telomerase inhibitor that binds to the template region of the RNA component of human ...

Approved Jun 2024FDA label ↗

Decitabine

(DECITABINE)Orphan drug

BluePoint Laboratories

Nucleoside Metabolic Inhibitor [EPC]

12.1 Mechanism of Action Decitabine is believed to exert its antineoplastic effects after phosphorylation and direct incorporation into DNA and inhibi...

Approved May 2006FDA label ↗

Lenalidomide

(LENALIDOMIDE)Orphan drugstandard

Teva Pharmaceuticals, Inc.

Thalidomide Analog [EPC]

12.1 Mechanism of Action Lenalidomide is an analogue of thalidomide with immunomodulatory, antiangiogenic, and antineoplastic properties. Cellular act...

FDA label ↗

Inqovi

(CEDAZURIDINE AND DECITABINE)Orphan drugstandard

Taiho Pharmaceutical Co., Ltd.

Nucleoside Metabolic Inhibitor [EPC]

12.1 Mechanism of Action Decitabine is a nucleoside metabolic inhibitor that is believed to exert its effects after phosphorylation and direct incorpo...

FDA label ↗

Grafapex

(TREOSULFAN)Orphan drugstandard

Medexus Pharma, Inc.

FDA label ↗

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Clinical Trial Landscape

8 active trials
1Phase 3
5Phase 2
1N/A
1PHASE1, PHASE2
8Total recruiting
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Recent News & Research

6 articles
Annals of hematologyApr 21, 2026

Health-related quality of life of patients with acute myeloid leukemia and myelodysplastic syndromes/neoplasms treated with decitabine: a systematic literature review.

Decitabine, including its new oral formulation (decitabine-cedazuridine, DEC-C), is commonly used in AML and MDS, particularly in older or unfit patients. While its clinical efficacy and tolerability ...

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Annals of hematologyApr 20, 2026

Very-low-dose decitabine and rhTPO for thrombocytopenia in lower-risk myelodysplastic syndrome.

Myelodysplastic syndrome (MDS) represents a heterogeneous group of myeloid neoplasms, with approximately two-thirds identified as lower-risk MDS (LR-MDS). LR-MDS with thrombocytopenia is associated wi...

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Journal of immunology (Baltimore, Md. : 1950)Apr 19, 2026

HIF-1α impairs NK cell differentiation-maturation and cytotoxicity in myelodysplastic syndrome via JAK1/STAT5/SOCS2 pathway.

Myelodysplastic syndrome (MDS) is a heterogeneous group of myeloid neoplasms characterized by treatment difficulties and a propensity to progress to acute myeloid leukemia. Impaired natural killer (NK...

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Current hematologic malignancy reportsApr 17, 2026

Clonal Signatures of Telomere Biology Disorders.

In inherited bone marrow failure syndromes (IBMFS), clonal hematopoiesis (CH) has been increasingly recognized as a molecular fingerprint of the underlying pathophysiology. A specific clonal profile h...

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HaematologicaApr 1, 2026

TGFβ-activated kinase-1 knockdown in hematopoietic stem-progenitor cells causes PANoptosis and myelodysplastic syndrome-like disease in mice.

Mutant SF3B1 (SF3B1mut) in hematopoietic stem/progenitor cells (HSPC) primarily affects erythropoiesis, resulting in myelodysplastic syndromes (MDS) with refractory macrocytic anemia and ring siderobl...

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British journal of haematologyMar 17, 2026

Safety and effectiveness of the combination of 5-azacitidine and ruxolitinib in VEXAS syndrome: A single-centre experience.

Safety and effectiveness of the combination of 5-azacitidine and ruxolitinib in VEXAS syndrome: A single-centre experience.

Read ↗

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Specialist Network

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