{"disease":{"id":"myelodysplastic-syndromes","name":"Myelodysplastic Syndromes (MDS)","therapeutic_area":"Haematology","data":{"aiSummary":"The treatment landscape for MDS is evolving, with hypomethylating agents (HMAs) like azacitidine and decitabine forming the cornerstone of therapy for higher-risk patients. Lenalidomide is an option for low-risk patients with del(5q) cytogenetic abnormality. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative option. Research is ongoing with novel agents like oral decitabine/cedazuridine and other targeted therapies to improve outcomes and address unmet needs, especially in high-risk patients and those who fail HMAs.","drug_count":8,"description":"Myelodysplastic syndromes (MDS) are a group of clonal hematopoietic stem cell disorders characterized by ineffective hematopoiesis, cytopenias, and a risk of progression to acute myeloid leukemia (AML). The pathophysiology involves genetic mutations leading to abnormal blood cell development in the bone marrow. Treatment aims to improve blood counts, reduce transfusion dependence, and prevent transformation to AML.","subtype_count":12},"enrichment_level":2,"last_enriched_at":null,"visit_count":0,"created_at":"2026-03-25T12:16:34.948Z","updated_at":"2026-03-25T12:16:34.948Z","meddra_pt":null,"meddra_code":null,"icd10_codes":null,"synonyms":[],"prevalence_global":null,"prevalence_us":null,"marketed_drug_count":0,"pipeline_drug_count":0,"trial_count":0},"drugs":{"marketed":[{"drug_id":"cedazuridine","indication_name":"Myelodysplastic Syndromes (MDS)","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":null,"generic_name":"CEDAZURIDINE","company_name":"","drug_phase":"marketed","molecular_target":"DNA methyltransferase","drug_class":"Nucleoside Metabolic Inhibitor [EPC]","quality_score":45,"revenue":null,"mechanism":"Decitabine inhibits DNA methyltransferase, leading to DNA hypomethylation and cellular differentiation or apoptosis."},{"drug_id":"imetelstat","indication_name":"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)","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Rytelo","generic_name":"IMETELSTAT","company_name":"Geron","drug_phase":"marketed","molecular_target":"","drug_class":"","quality_score":50,"revenue":null,"mechanism":"Rytelo works by inhibiting the activity of telomerase, an enzyme that helps cancer cells grow and divide."},{"drug_id":"pf-07059013","indication_name":"Treatment of certain types of myelodysplastic syndromes","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Pf-07059013","generic_name":"pf-07059013","company_name":"Pfizer","drug_phase":"marketed","molecular_target":"Unknown","drug_class":"Unknown","quality_score":25,"revenue":null,"mechanism":""}],"pipeline":[],"offLabel":[],"totalMarketed":3,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT04889729","title":"GENOMED4ALL: Improving MDS Classification and Prognosis by AI","phase":"","overall_status":"UNKNOWN","enrollment_count":13284,"lead_sponsor_name":"Istituto Clinico Humanitas","has_results":false},{"nct_id":"NCT04676945","title":"Analysis of Risk in MDS Over Time - Comparison of Treated vs Untreated Patients","phase":"","overall_status":"COMPLETED","enrollment_count":9179,"lead_sponsor_name":"Michael Pfeilstöcker","has_results":false},{"nct_id":"NCT04174547","title":"An European 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Study","phase":"","overall_status":"COMPLETED","enrollment_count":2115,"lead_sponsor_name":"National Heart, Lung, and Blood Institute (NHLBI)","has_results":false},{"nct_id":"NCT01688011","title":"Connect® Myeloid Disease Registry","phase":"","overall_status":"TERMINATED","enrollment_count":2013,"lead_sponsor_name":"Celgene","has_results":false},{"nct_id":"NCT03904134","title":"Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation (BMT CTN 1702)","phase":"NA","overall_status":"COMPLETED","enrollment_count":1753,"lead_sponsor_name":"Center for International Blood and Marrow Transplant Research","has_results":false},{"nct_id":"NCT01595295","title":"Registry on Hypomethylating Agents in Myeloid Neoplasms","phase":"","overall_status":"COMPLETED","enrollment_count":1500,"lead_sponsor_name":"Arbeitsgemeinschaft medikamentoese Tumortherapie","has_results":false},{"nct_id":"NCT07102849","title":"Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":1400,"lead_sponsor_name":"National Heart, Lung, and Blood Institute (NHLBI)","has_results":false},{"nct_id":"NCT07465029","title":"A Study of Incidence, Treatment Patterns, and Outcomes in Transfusion-dependent Lower-risk Myelodysplastic Syndromes in Spain","phase":"","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":1300,"lead_sponsor_name":"Bristol-Myers Squibb","has_results":false},{"nct_id":"NCT05350748","title":"Comprehensive Molecular and Clinical Evaluation of Pediatric and Adult MDS","phase":"","overall_status":"RECRUITING","enrollment_count":1100,"lead_sponsor_name":"National Cancer Institute (NCI)","has_results":false},{"nct_id":"NCT05588154","title":"Collection of Blood, Bone Marrow, Skin, Saliva, and Stool Samples From Healthy Volunteers Used for Comparative Analysis of Myeloid 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(CMML-2)","phase":"PHASE3","overall_status":"TERMINATED","enrollment_count":530,"lead_sponsor_name":"Novartis Pharmaceuticals","has_results":true},{"nct_id":"NCT07019155","title":"Study of Individuals and Families With Aberrations in DDX41 or Similar Cancer Predisposition Variants","phase":"","overall_status":"RECRUITING","enrollment_count":510,"lead_sponsor_name":"National Cancer Institute (NCI)","has_results":false},{"nct_id":"NCT07355478","title":"Asia Myelodysplastic Syndrome (MDS) Registry","phase":"","overall_status":"NOT_YET_RECRUITING","enrollment_count":500,"lead_sponsor_name":"Bristol-Myers Squibb","has_results":false},{"nct_id":"NCT00481273","title":"AVIDA The Vidaza® (Azacitidine) Patient Registry","phase":"","overall_status":"COMPLETED","enrollment_count":479,"lead_sponsor_name":"Celgene","has_results":false},{"nct_id":"NCT06971185","title":"A Study to Evaluate Treatment Patterns and Effectiveness of 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Miami","has_results":false},{"nct_id":"NCT02164071","title":"Validation of a Comprehensive Health Status Assessment Scale in Elderly Patients (≥ 65 Years) With Hematological Malignancies","phase":"","overall_status":"COMPLETED","enrollment_count":363,"lead_sponsor_name":"Celgene","has_results":false},{"nct_id":"NCT03682536","title":"A 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 Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve","phase":"PHASE3","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":363,"lead_sponsor_name":"Celgene","has_results":true},{"nct_id":"NCT00003816","title":"Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer","phase":"PHASE2, PHASE3","overall_status":"COMPLETED","enrollment_count":361,"lead_sponsor_name":"Roswell Park Cancer Institute","has_results":true},{"nct_id":"NCT05949684","title":"ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions","phase":"PHASE3","overall_status":"RECRUITING","enrollment_count":360,"lead_sponsor_name":"Bristol-Myers Squibb","has_results":false},{"nct_id":"NCT00071799","title":"A Survival Study in Patients With High Risk Myelodysplastic Syndromes Comparing Azacitidine Versus Conventional Care","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":358,"lead_sponsor_name":"Celgene","has_results":true},{"nct_id":"NCT06960993","title":"Mosaic Trial for Stem Cell Transplant Recipients","phase":"NA","overall_status":"RECRUITING","enrollment_count":356,"lead_sponsor_name":"Northwestern University","has_results":false},{"nct_id":"NCT02619565","title":"Prospective Study of Molecular Predictors of Survival in Myelodysplastic Syndromes","phase":"NA","overall_status":"COMPLETED","enrollment_count":349,"lead_sponsor_name":"Assistance Publique - Hôpitaux de Paris","has_results":false},{"nct_id":"NCT00303719","title":"Allogeneic Bone Marrow Transplantation Using Less Intensive Therapy","phase":"PHASE2","overall_status":"TERMINATED","enrollment_count":342,"lead_sponsor_name":"Masonic Cancer Center, University of Minnesota","has_results":true},{"nct_id":"NCT04093570","title":"A Study for Participants Who Participated in Prior Clinical Studies of ASTX727 (Standard Dose)","phase":"PHASE2","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":332,"lead_sponsor_name":"Taiho Oncology, Inc.","has_results":false},{"nct_id":"NCT04986657","title":"Whole Genome Sequencing (ChromoSeq) as an Adjunct to Conventional Genomic Profiling in AML and MDS","phase":"NA","overall_status":"RECRUITING","enrollment_count":325,"lead_sponsor_name":"Washington University School of Medicine","has_results":false},{"nct_id":"NCT03310918","title":"A Collaborative Palliative and Leukemia Care Model for Patients With AML and MDS Receiving Non-Intensive Therapy","phase":"NA","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":320,"lead_sponsor_name":"Massachusetts General Hospital","has_results":false},{"nct_id":"NCT04256317","title":"A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)","phase":"PHASE2, PHASE3","overall_status":"RECRUITING","enrollment_count":316,"lead_sponsor_name":"Taiho Oncology, Inc.","has_results":false},{"nct_id":"NCT06001385","title":"HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis","phase":"PHASE2","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":313,"lead_sponsor_name":"Center for International Blood and Marrow Transplant Research","has_results":false},{"nct_id":"NCT04428489","title":"Follow-up Study of ICUS and CCUS Patients","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":300,"lead_sponsor_name":"The First Affiliated Hospital of Soochow University","has_results":false},{"nct_id":"NCT04370457","title":"MyPal ePRO-based Early Palliative Care System in Adult Patients With Hematologic Malignancies","phase":"NA","overall_status":"UNKNOWN","enrollment_count":300,"lead_sponsor_name":"Centre for Research and Technology Hellas","has_results":false},{"nct_id":"NCT04517175","title":"Could Ki-67 be Used as a Diagnostic or Prognostic Marker in Hemato-oncological Diagnostics?","phase":"","overall_status":"UNKNOWN","enrollment_count":300,"lead_sponsor_name":"Maastricht University","has_results":false},{"nct_id":"NCT06379945","title":"Unified platforM for a Better integRal Evaluation of MyeLodyspLastic Syndromes in SpAin-Strategy for Unraveling Personalized genoMic Medicine in Public heAlth 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Myelodysplastic Syndrome (MDS)","phase":"PHASE2, PHASE3","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":289,"lead_sponsor_name":"Geron Corporation","has_results":true},{"nct_id":"NCT04771130","title":"A Study of BGB-11417 in Participants With Myeloid Malignancies","phase":"PHASE1, PHASE2","overall_status":"RECRUITING","enrollment_count":260,"lead_sponsor_name":"BeiGene","has_results":false},{"nct_id":"NCT00544115","title":"Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders","phase":"PHASE2","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":260,"lead_sponsor_name":"City of Hope Medical Center","has_results":true}],"total":50},"guidelines":[{"drug_id":"lenalidomide","guideline_body":"FDA label","recommendation":"Lenalidomide is indicated for the treatment of adult patients with transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities.","line_of_therapy":"1L|2L|3L+","evidence_grade":"A","guideline_year":null},{"drug_id":"lenalidomide","guideline_body":"FDA label","recommendation":"Lenalidomide is indicated for the treatment of adult patients with mantle cell lymphoma whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib.","line_of_therapy":"3L+","evidence_grade":"A","guideline_year":null}],"source":"Drug Landscape verified database"}