Myelodysplastic syndromes are a group of bone marrow disorders where blood cells don't develop properly, leading to low counts and a risk of progression to acute leukemia. Treatment ranges from supportive care and growth factors to drugs like azacitidine and lenalidomide, and stem cell transplant for younger or higher-risk patients. Many people live for years with stable disease.
What's actually going on in research
Trials are testing venetoclax combinations that borrow strategies from acute leukemia treatment, oral azacitidine to extend response after initial therapy, and drugs targeting specific mutations like TP53 and splicing factor genes. Researchers are also studying immune checkpoint inhibitors, CAR-T cells adapted for MDS, and ways to predict who will respond to existing drugs.
Venetoclax combinations
This pill blocks a protein that keeps cancer cells alive and is FDA-approved for some leukemias. Trials are pairing it with azacitidine or decitabine to see if the combination works better than either drug alone in higher-risk MDS.
Mutation-targeted drugs
Drugs aimed at TP53 mutations, common in hard-to-treat MDS, are in testing. Other trials target splicing factor mutations, which drive many cases of MDS and have no specific treatment yet.
Oral azacitidine maintenance
An oral form of azacitidine, taken daily at home, is FDA-approved to extend remission after initial treatment. Trials are exploring whether starting it earlier or combining it with other drugs improves outcomes.
What to know before you search
Eligibility often depends on MDS subtype, risk score, blood counts, genetic mutations, prior treatments, and whether you're a transplant candidate.
What types of trials are currently open
- Treatment trials — Testing new drug combinations or next-generation versions of azacitidine and lenalidomide to see if they improve blood counts or delay progression to leukemia.
- Transplant trials — Studies refining when to transplant, what conditioning regimen to use, and how to manage complications afterward.
- Maintenance trials — Testing whether drugs taken after initial treatment can keep MDS under control longer.
- Mutation-based trials — Enrolling people based on genetic features of their MDS cells to test drugs that target those specific mutations.
- Observational studies — Following people with MDS over time to learn how the disease changes, what predicts transformation to leukemia, and who benefits from which treatments.
Recently added Myelodysplastic Syndromes trials
Take two drugs to treat increased blast myelodysplastic syndrome
This study will evaluate the safety and preliminary effectiveness of sonrotoclax combined with azacitidine in adults aged 18-70 years with newly diagnosed myelodysplastic neoplasms with increased blasts-2 (MDS-IB2). Sonrotoclax blocks BCL-2, a protein that helps cancer cells survive. Combining sonrotoclax with azacitidine may improve disease control. This study aims to determine whether the combination can induce remission with an acceptable safety profile, facilitate allogeneic stem cell transplantation in suitable patients, and improve long-term clinical outcomes. This multicenter, open-label study has two sequential parts. Phase Ib will evaluate different sonrotoclax doses and identify the recommended dose for phase II. Phase II will further assess remission rates and safety at the selected dose. All participants will receive sonrotoclax plus azacitidine; there is no randomized control group. Participants will undergo regular blood tests, bone marrow examinations, and monitoring for side effects. Suitable participants may proceed to transplantation based on disease response and clinical condition. Those who are not proceeding to transplantation and continue to benefit may continue treatment according to the protocol. Follow-up will assess the duration of remission, disease progression, subsequent treatment, survival, and quality of life.
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
A non-profit, translational, prospective and retrospective study. Patients will be enrolled according to the procedures described in the protocol, and samples will be processed and analyzed as specified in the protocol. Since constitutional bone marrow failure syndromes (CMF) are rare genetic disorders characterized by a variable and incompletely defined clinical phenotype, a partially unknown genetic etiology, and pathogenetic mechanisms that are only partly understood, we hypothesize that a number of patients currently diagnosed with idiopathic acquired aplastic anemia (AAA) or idiopathic single-lineage/multilineage bone marrow failure (BMF) may have been misdiagnosed and may actually have an underlying CMF. Therefore, our primary objective is to identify CMF underlying BMF and vice versa, by defining well-characterized patient cohorts based on clinical and family history, clinical manifestations, and immunological findings; identifying disease-specific parameters and biomarkers; and providing disease-specific guidelines to facilitate early diagnosis and, consequently, enable timely and appropriate treatment. In this context, the use of appropriate artificial intelligence (AI) tools is likely to improve the diagnostic accuracy of this approach.
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