Myelodysplastic syndrome

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ORPHA:52688OMIM:614286
Who is this for?
Show terms as
7FDA treatments8Active trials25Specialists8Treatment centers2Financial resources

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Myelodysplastic Syndrome is treated with 8 medications in our database, including Revlimid, AZACITIDINE, Decitabine, DECITABINE, Azacitidine, and 3 more. 8 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Bristol Myers Squibb, Pilnova Pharma Inc, BluePoint Laboratories, Eisai, Celgene and others. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Myelodysplastic Syndrome treatment below.

Also known as:

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

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

Treatments

7 available

Lenalidomide

LENALIDOMIDE· Teva Pharmaceuticals, Inc.■ Boxed Warning
Lenalidomide capsules are indicated for the treatment of adult patients with transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion

Lenalidomide capsules are indicated for the treatment of adult patients with transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities

Inqovi

CEDAZURIDINE AND DECITABINE· Taiho Pharmaceutical Co., Ltd.
INQOVI is indicated for treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-Briti

INQOVI is indicated for treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups

Grafapex

TREOSULFAN· Medexus Pharma, Inc.■ Boxed Warning
Use in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation in adult and pediatric patients 1 year of age and older with myelodysplastic syndrom

Use in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation in adult and pediatric patients 1 year of age and older with myelodysplastic syndrome (MDS)

Rytelo

IMETELSTAT SODIUM· Geron CorporationOrphan Drug
treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not respon

treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA)

Decitabine

DECITABINE· BluePoint LaboratoriesOrphan Drug
for treatment of patients with myelodysplastic syndromes (MDS) including previously treated and untreated, de novo and secondary MDS of all French-American-British subtypes (refractory anemia, refract

for treatment of patients with myelodysplastic syndromes (MDS) including previously treated and untreated, de novo and secondary MDS of all French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups

AZACITIDINE

AZACITIDINE· Pilnova Pharma Inc

* Patient Copay Amount: $0 per dose for select HCP-administered medications (e.g., OPDIVO, YERVOY, ABRAXANE); $0 per one-month supply for select oral medications (e.g., REVLIMID, SPRYCEL, POMALYST).

VIDAZA

azacitidine· Celgene

* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Eligible commercially insured patients; U.S. residents 18 years o

Clinical Trials

8 recruitingView all trials with filters →
Phase 31 trial
Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome
Phase 3
Active
PI: ABBVIE INC. (AbbVie) · Sites: Fullerton, California; Los Angeles, California +218 more · Age: 1899 yrs
Phase 25 trials
Eltrombopag as a Novel Therapeutic Approach for Low-risk MDS and CMML With TET2 Mutations
Phase 2
Actively Recruiting Prior treatment eligible
PI: Abhay Singh, MD, MPH (Case Comprehensive Cancer Center, Cleveland Clinic) · Sites: Cleveland, Ohio · Age: 1899 yrs
Roll-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.
Phase 2
Active
PI: Novartis Pharmaceuticals (Novartis Pharmaceuticals) · Sites: Portland, Oregon; Salt Lake City, Utah +18 more · Age: 1299 yrs
Intensity 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)
Phase 2
Actively Recruiting
PI: Matias Sanchez, MD (University of Illinois at Chicago) · Sites: Chicago, Illinois · Age: 1865 yrs
A Phase 2a Study of HT-6184 in Subjects With IPSS-R Very Low, Low or Intermediate Risk MDS and Anemia
Phase 2
Active
· Sites: Ahmedabad, Gujarat; Ahmedabad, Gujarat +8 more · Age: 1899 yrs
Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes
Phase 2
Active Prior treatment eligible
PI: Esther Natalie Oliva (QOL-ONE Associazione Culturale e di Ricerca) · Sites: Amiens; Avignon +18 more · Age: 1899 yrs
N/A1 trial
Involvement of CDA and/or dCK Metabolizing Enzymes in the Response to Azacytidine Treatment of Patients With Hematologic Malignancies
N/A
Actively Recruiting
· Sites: Marseille · Age: 1899 yrs

Specialists

25 foundView all specialists →
MM
Minocher Battiwalla, MD
NASHVILLE, TN
Specialist
PI on 2 active trials
CM
Corey Cutler, MD, MPH
PARK CITY, UT
Specialist
PI on 3 active trials
EO
Esther Natalie Oliva
Amiens, MA
Specialist

Rare Disease Specialist

2 Myelodysplastic syndrome publications
AM
Abhay Singh, MD, MPH
Cleveland, Ohio
Specialist

Rare Disease Specialist

PI on 1 active trial
TM
Thomas G. Martin, MD
Specialist
PI on 4 active trials
MS
Mikkael A Sekeres
MIAMI, FL
Specialist
PI on 1 active trial3 Myelodysplastic syndrome publications
GM
Guillermo M Bravo, MD
CHANDLER, AZ
Specialist
PI on 1 active trial1 Myelodysplastic syndrome publication
AK
Anne Kuan
SAN ANTONIO, TX
Specialist
PI on 1 active trial
CP
Claudio G. Brunstein, MD, PhD
MINNEAPOLIS, MN
Specialist
PI on 6 active trials
NS
Nobuhito Sanada
Specialist
PI on 2 active trials
MM
Margaret L. MacMillan, MD
Specialist
PI on 4 active trials
DM
David A Sallman, MD
TAMPA, FL
Specialist
PI on 1 active trial
MD
Michael Dickinson, Dr
Specialist
PI on 2 active trials
GD
Guillermo Garcia-Manero, M D
SIDNEY, NE
Specialist
PI on 1 active trial
KG
Katsuhisa Goto
Specialist
PI on 1 active trial
MM
Marc Gautier, MD
LEBANON, NH
Specialist
PI on 1 active trial
JD
Jürgen Wehmeyer, Dr.
Specialist
PI on 1 active trial
JD
Jeffrey Bubis, DO
FLEMING ISLAND, FL
Specialist
PI on 1 active trial
AA
Adam Al-Hakim
Specialist
2 Myelodysplastic syndrome publications
SS
Sinisa Savic
Specialist
2 Myelodysplastic syndrome publications
AZ
Amer M Zeidan
NEW HAVEN, CT
Specialist
2 Myelodysplastic syndrome publications
SB
Stefan Balabanov
Specialist
2 Myelodysplastic syndrome publications
DR
Dr Kavita Raj
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

2 grants

Myelodysplastic syndrome medication copay assistance

PAN Foundation

Apply ↗

BMS Access Support - ONUREG (azacitidine) Patient Assistance Program

Bristol-Myers Squibb

Apply ↗

Community

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Latest news about Myelodysplastic syndrome

5 articles
NewsANNALS 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
NewsANNALS 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-MD
NewsJOURNAL 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 myelo
NewsCURRENT 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 path
NewsHAEMATOLOGICAApr 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 refrac
See all news about Myelodysplastic syndrome

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Myelodysplastic syndrome

What is Myelodysplastic syndrome?

Myelodysplastic Syndrome is treated with 8 medications in our database, including Revlimid, AZACITIDINE, Decitabine, DECITABINE, Azacitidine, and 3 more. 8 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Bristol Myers Squibb, Pilnova Pharma Inc, BluePoint Laboratories, Eisai, Celgene and others. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Myelodysplastic Syndrome treatment below.

Are there clinical trials for Myelodysplastic syndrome?

Yes — 8 recruiting clinical trials are currently listed for Myelodysplastic syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Myelodysplastic syndrome?

25 specialists and care centers treating Myelodysplastic syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Myelodysplastic syndrome?

5 FDA-approved treatments are currently tracked on UniteRare for Myelodysplastic syndrome. See the treatments and support programs sections for copay assistance, eligibility, and contact details.