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myelodysplastic syndrome (MDS) - Clinical Profile, Treatments & Trials | Kisho | Kisho

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

8 FDA-approved treatments are available for myelodysplastic syndrome, including IVOSIDENIB (TIBSOVO, approved 2018), IMETELSTAT SODIUM (RYTELO, approved 2024), and LUSPATERCEPT (REBLOZYL, approved 2019). An additional 45 compounds hold orphan drug designation.

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved	Market Status
GRAFAPEX	TREOSULFAN	—	2025	Available
RYTELO	IMETELSTAT SODIUM	—	2024	Available
INQOVI	CEDAZURIDINE AND DECITABINE	—	2020	Available
REBLOZYL	LUSPATERCEPT	—	2019	Available
TIBSOVO	IVOSIDENIB	—	2018	Available
DECITABINE	DECITABINE	—	2014	Available
DACOGEN	—	—	2006	Available
REVLIMID	LENALIDOMIDE	—	2005	Available

Orphan Drug Designations

The following drugs have received orphan drug designation from the FDA for myelodysplastic syndrome. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor	Designated	Exclusivity End	Designation Status
lisaftoclax	lisaftoclax	Ascentage Pharma Group, Inc.	2026	—	Designated
roxadustat	roxadustat	Kyntra Bio	2025	—	Designated
ofirnoflast	ofirnoflast

Venetoclax is referenced in active clinical trials for myelodysplastic syndrome (designated 2019).

Adverse Event Profiles

FDA adverse event reports (FAERS) include all outcomes reported during treatment and do not establish causation. Report counts reflect all approved indications for each drug, not only this disease.

1,333 adverse event reports have been filed with the FDA for DACOGEN (across all indications). Most commonly reported: haemoglobin decreased, pneumonia, and toxicity to various agents.

Gene therapy approaches for myelodysplastic syndrome have been reported in the published literature.

GeneReviews

Management Guidelines

Evaluations Following Initial Diagnosis To establish the spectrum of manifestations and medical needs in an individual diagnosed with Bohring-Opitz syndrome (BOS), the following evaluations are recommended if they have not already been completed. Table 3. Recommended Evaluations Following Initial Diagnosis of Bohring-Opitz Syndrome

System/Concern	Evaluation	Comment
Growth	Weight, length/height, head circumference measurements plotted on standard growth chart	Goal: normal weight-for-length or body mass index; Expected final adult height: 2nd centile
ENT/Mouth	Craniofacial evaluation if cleft lip/palate, micrognathia, or obstructive sleep apnea is present

Clinical Trials

522 trials found

Explore Trials

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

522 clinical trials registered, 179 recruiting. Interventions under study include drug therapy, other interventions, procedural interventions, and biologic therapy. Pipeline includes 25 PHASE3, 182 PHASE2, 201 PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT05554406](https://clinicaltrials.gov/study/NCT05554406)	Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)	PHASE2	National Cancer Institute (NCI)	RECRUITING
[NCT05010122](https://clinicaltrials.gov/study/NCT05010122)	ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome	PHASE1	M.D. Anderson Cancer Center	RECRUITING
[NCT04800458](https://clinicaltrials.gov/study/NCT04800458)	Contribution of Anti-platelet Antibodies Identified With MAIPA Assay in the Demonstration of the Auto-immune Character of a Thrombocytopenia at Diagnosis	NA	University Hospital, Bordeaux	RECRUITING
[NCT06569095](https://clinicaltrials.gov/study/NCT06569095)	Predictive Value of Myelodysplastic Syndrome Stem Cells Determined by Multiparameter Flow Cytometry	—	Peking University People's Hospital	RECRUITING
[NCT01890486](https://clinicaltrials.gov/study/NCT01890486)	The Prospective Collection, Storage and Reporting of Data on Patients Undergoing Hematopoietic Stem Cell Transplantation Utilizing a Standard Preparative Regimen	—	Wake Forest University Health Sciences	RECRUITING

500 publications have been identified in PubMed for myelodysplastic syndrome. Research spans Basic Science / Preclinical (22%), Case Report / Case Series (22%), and Review / Meta-Analysis (19%).

Research Type	Count	% of Total
Laboratory research	110	22%
Patient case studies	109	22%
Research summaries	95	19%
Disease patterns and progression	65	13%
Clinical study results	55	11%
Testing and diagnosis research	30

Recent Publications

Galanopoulos AG (2026). [PMID: 41733418](https://pubmed.ncbi.nlm.nih.gov/41733418/). *Eur J Haematol*. [Review / Meta-Analysis]
Zhao S (2026). [PMID: 41875887](https://pubmed.ncbi.nlm.nih.gov/41875887/). *Mol Cell*. [Basic Science / Preclinical]
Liu C (2026). [PMID: 41934278](https://pubmed.ncbi.nlm.nih.gov/41934278/). *Eur J Haematol*. [Basic Science / Preclinical]
Su Z (2026). [PMID: 42611798](https://pubmed.ncbi.nlm.nih.gov/42611798/). *Leuk Lymphoma*. [Epidemiology / Natural History]
Hu T (2026). [PMID: 42363987](https://pubmed.ncbi.nlm.nih.gov/42363987/). *Ann Hematol*. [Case Report / Case Series]
Miller CA (2026). [PMID: 42239263](https://pubmed.ncbi.nlm.nih.gov/42239263/). *bioRxiv*. [Basic Science / Preclinical]
Wei Q (2026). [PMID: 42320760](https://pubmed.ncbi.nlm.nih.gov/42320760/). *Mod Pathol*. [Basic Science / Preclinical]
Vilcassim S (2026). [PMID: 42240074](https://pubmed.ncbi.nlm.nih.gov/42240074/). *Am J Hematol*. [Basic Science / Preclinical]
Komrokji RS (2026). [PMID: 41549790](https://pubmed.ncbi.nlm.nih.gov/41549790/). *American journal of hematology*. [Clinical Trial Publication]
Oran B (2026). [PMID: 40702903](https://pubmed.ncbi.nlm.nih.gov/40702903/). *Haematologica*. [Clinical Trial Publication]

Kisho

myelodysplastic syndrome

Overview

Clinical Presentation

Clinical Guidance

Explore clinical features

Genetic Landscape

ASXL1

GNB1

SF3B1

TET2

Diagnosis

Diagnostic Criteria

Suggestive Findings

Differential Diagnosis

Treatment

Approved Therapies

Orphan Drug Designations

Adverse Event Profiles

Management Guidelines

Clinical Trials

Prognosis

Surveillance Guidelines

Research Landscape

Recent Publications

Patient Support

Patient Advocacy Groups

Medical References

External Resources

OMIM

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Latest News & Developments

Gelatinous transformation and concomitant fibrosis of bone marrow in a patient with relapsed myelodysplastic syndrome following unrelated bone marrow transplantation.

Metastatic Testicular Cancer Complicated by Myelodysplastic Syndrome.

Two cases of lysozyme-induced nephropathy with varying underlying diseases: myelodysplastic syndrome and chronic myelomonocytic leukemia.

myelodysplastic syndrome

Overview

Clinical Presentation

Clinical Guidance

Explore clinical features

Genetic Landscape

ASXL1

GNB1

SF3B1

TET2

Diagnosis

Diagnostic Criteria

Suggestive Findings

Differential Diagnosis

Treatment

Approved Therapies

Orphan Drug Designations

Adverse Event Profiles

Management Guidelines

Clinical Trials

Prognosis

Surveillance Guidelines

Research Landscape

Recent Publications

Patient Support

Patient Advocacy Groups

Medical References

External Resources

OMIM

Orphanet

GARD

Subtypes

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Latest News & Developments

Gelatinous transformation and concomitant fibrosis of bone marrow in a patient with relapsed myelodysplastic syndrome following unrelated bone marrow transplantation.

Metastatic Testicular Cancer Complicated by Myelodysplastic Syndrome.

Two cases of lysozyme-induced nephropathy with varying underlying diseases: myelodysplastic syndrome and chronic myelomonocytic leukemia.