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Myelodysplastic syndrome

Myelodysplastic syndrome (MDS) is a heterogeneous group of hematologic neoplasms defined as a clonal disorder of hematopoietic stem cells leading to dysplasia and ineffective hematopoiesis in the bone marrow

Prevalence

1-9 / 100 000

3,310 - 29,790

US Estimated

5,135 - 46,215

Europe Estimated

Age of Onset

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ICD-10

-

Inheritance

Autosomal dominant

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Autosomal recessive

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Mitochondrial/Multigenic

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X-linked dominant

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X-linked recessive

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5 Facts you should know

FACT

1

A heterogeneous group of closely related clonal hematopoietic disorders commonly found in the aging population.

FACT

2

Signs and symptoms may include dizziness, fatigue, weakness, shortness of breath, bruising and bleeding, frequent infections, and headaches.

FACT

3

Patients with MDS may present with clinical manifestations of anemia, thrombocytopenia, and/or neutropenia.

FACT

4

Risk factors include previous chemotherapy or radiation therapy, exposure to tobacco smoke, pesticides, and benzene, and exposure to heavy metals such as mercury or lead.

FACT

5

The typical age of onset is 70 years.

Myelodysplastic syndrome is also known as...

Myelodysplastic syndrome

MDS

What’s your Rare IQ?

This famous rock guitarist battled throat and lung cancer, but ultimately died of the complications of myelodysplastic syndrome.

Common signs & symptoms

Myelodysplasia

Somatic mutation

Current treatments

Decitabine(Brand name: Dacogen (injection))

Manufactured by Otsuka America Pharmaceutical, Inc.
FDA-approved indication: 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.

Lenalidomide(Brand name: Revlimid)

Manufactured by Celgene Corporation
FDA-approved indication: For use in combination with dexamethasone for the treatment of multiple myeloma. Also, for use for the treatment of multiple myeloma (MM), as maintenance following autologous hematopoietic stem cell transplantation (auto-HSCT).

Azacitidine(Brand name: Vidaza)

Manufactured by Celgene Corporation
FDA-approved indication: Treatment of patients with the following myelodysplastic syndrome subtypes: refractory anemia or refractory anemia with ringed sideroblasts (if accompanied by neutropenia or thrombocytopenia and requiring transfusions), refractory anemia.

Top Clinical Trials

TitleDescriptionPhasesStatusInterventionsMore Information
Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic SyndromeMyelodysplastic Syndrome (MDS) is a group of disorders that gradually affect the ability of a person's bone marrow (semi-liquid tissue present in many bones like backbones) to produce normal blood cells. Some people with MDS have a risk of the disease progressing to acute myeloid leukemia (AML), and a risk of death from the disease itself. Symptoms of MDS include fatigue, shortness of breath, unusual paleness due to anemia (low red blood cell count), easy or unusual bruising, and red spots just beneath the skin caused by bleeding. The purpose of this study is to see how safe and effective venetoclax and azacitidine (AZA) combination are when compared to AZA and a placebo (contains no medicine), in participants with newly diagnosed higher-risk MDS.Phase 3RecruitingVenetoclax| AzacitidineClick here for more information
Efficacy and Safety Study of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in ESA Naïve Subjects Who Require Red Blood Cell TransfusionsThe study will be conducted in compliance with the International Council for Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements.
This is an interventional active-controlled, open-label, randomized Phase 3 study to compare the efficacy and safety of luspatercept (ACE-536) versus epoetin alfa for the treatment of anemia due to IPSS-R very low, low or intermediate risk MDS in ESA naïve subjects who require RBC transfusions.
The study is divided into the Screening Period, a Treatment Period and a Post-Treatment Follow-up Period.
Phase 3RecruitingLuspatercept| Epoetin alfaClick here for more information
SY-1425 Plus Azacitidine in Participants With Newly Diagnosed RARA-positive Higher-Risk Myelodysplastic SyndromeThis study compares the efficacy of SY-1425 in combination with azacitidine to azacitidine in combination with placebo in participants who are Retinoic Acid Receptor Alpha (RARA) positive, and newly diagnosed with higher-risk myelodysplastic syndrome (MDS), and who have not received treatment for this diagnosis. The primary goal of the study is to compare the complete remission rate between the two treatment arms.Phase 3RecruitingSY-1425 + AzacitidineClick here for more information
Magrolimab + Azacitidine Versus Azacitidine + Placebo in Untreated Participants With Myelodysplastic Syndrome (MDS)The primary objective of this study is to evaluate the efficacy of magrolimab in combination with azacitidine compared to that of azacitidine plus placebo in previously untreated participants with intermediate/high/very high risk myelodysplastic syndrome (MDS) by Revised International Prognostic Scoring System (IPSS-R) as measured by complete remission (CR) and overall survival (OS).Phase 3RecruitingMagrolimab| AzacitidineClick here for more information
A Study of ASTX030 (Cedazuridine in Combination With Azacitidine) in MDS, CMML, or AMLStudy ASTX030-01 is designed to move efficiently from Phase 1 to Phase 3. Phase 1 consists of an open-label Dose Escalation Stage (Stage A) using multiple cohorts at escalating dose levels of oral cedazuridine and azacitidine (only one study drug will be escalated at a time) followed by a Dose Expansion Stage (Stage B) of ASTX030. Phase 2 is a randomized open-label crossover study to compare oral ASTX030 to subcutaneous (SC) azacitidine. Phase 3 is a randomized open-label crossover study comparing the final oral ASTX030 tablet to SC azacitidine. The duration of the study is expected to be approximately 36 months.Phase 2|Phase 3RecruitingAzacitidine|ASTX030 (cedazuridine + azacitidine) CedazuridineClick here for more information
Study of ASTX727 vs IV Decitabine in MDS, CMML, and AMLMulticenter, randomized, open-label, crossover PK study of ASTX727 versus IV decitabine. Adult subjects who are candidates to receive IV decitabine will be randomized 1:1 to receive the ASTX727 tablet Daily×5 in Cycle 1 followed by IV decitabine 20 mg/m^2 Daily×5 in Cycle 2, or the converse order. After completion of PK studies during the first 2 treatment cycles, subjects will continue to receive treatment with ASTX727 from Cycle 3 onward (in 28-day cycles) until disease progression, unacceptable toxicity, or the subject discontinues treatment or withdraws from the study.Phase 3RecruitingASTX727/ DacogenClick here for more information
Efficacy and Safety of FG-4592 for Treatment of Anemia in Patients With Lower Risk MDS With Low Red Blood Cell Transfusion BurdenThe purpose of this study is to determine whether FG-4592 is safe and effective in the treatment of anemia in patients with Lower Risk Myelodysplastic Syndrome and Low Red Blood Cell Transfusion Burden.Phase 3RecruitingFG-4592Click here for more information
A Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical TrialsA Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept. The study design is divided into the Transition Phase, Treatment Phase and Follow-up Phase. Subjects will enter transition phase and depending on their background will enter either the treatment phase or the Long-term Post-treatment Follow-up (LTPTFU) phase.Phase 3RecruitingLuspaterceptClick here for more information

Top Treatments in Research

AgentClass/Mechanism of ActionDevelopment StatusCompanyClinical StudiesMore Information
VenetoclaxVenetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It works by blocking the action of a certain protein in the body that helps cancer cells survive. This helps to kill cancer cells.Phase 3AbbVie|Genentech, Inc.Click here for more informationClick here for more information
SY-1425SY-1425, a clinical stage RARα agonist with improved pharmacokinetics, potency, and selectivity over pan-retinoic acid agonists, induces differentiation in non-APL AML cell lines and primary patient samples with a RARA super-enhancer associated biomarker (RARA-high)Phase 3Syros PharmaceuticalsClick here for more informationClick here for more information
MagrolimabMagrolimab is a first-in-class investigational monoclonal antibody against CD47 and macrophage checkpoint inhibitor that is designed to interfere with recognition of CD47 by the SIRPα receptor on macrophages, thus blocking the "don't eat me" signal used by cancer cells to avoid being ingested by macrophages. Magrolimab is being developed in several hematologic and solid tumor malignancies, including MDS.Phase 3Gilead SciencesClick here for more informationClick here for more information
ASTX030 (cedazuridine + azacitidine)ASTX030 is an investigational oral formulation of azacitidine + cedazuridine designed to enhance systemic bioavailability of azacitidine delivered orally to the level achieved with parenteral administration of azacitidine alone. Cedazuridine is a cytidine deaminase inhibitor and inhibits azacitidine inactivation in the gut and liver.Phase 2|Phase 3Astex Pharmaceuticals, Inc.Click here for more informationClick here for more information
ASTX727Oral decitabine and cedazuridine (ASTX727) is a unique fixed-dose combination of the hypomethylating agent decitabine, the active ingredient in Dacogen®, and the novel cytidine deaminase inhibitor, E7727 (cedazuridine). Oral decitabine and cedazuridine was designed to deliver decitabine by oral administration. By inhibiting cytidine deaminase, cedazuridine inhibits the major mechanism by which decitabine is degraded in the gut and liver, and the combination therefore permits the efficient delivery of decitabine orally.Phase 3Astex Pharmaceuticals, Inc.Click here for more informationClick here for more information