Myelodysplastic syndromes (MDS) are a group of bone marrow disorders that can cause low blood cell counts and may progress to acute leukemia. Treatment options for patients with intermediate-to-high-risk MDS are limited, especially for older patients or those who are not suitable for intensive chemotherapy or hypomethylating agents.
Chidamide is an oral histone deacetylase inhibitor that has shown antitumor activity in several hematologic malignancies. This study aims to evaluate the effectiveness and safety of chidamide used alone in patients with intermediate-to-high-risk MDS.
This is a prospective, single-arm, open-label phase II study conducted at a single center. Eligible participants will receive oral chidamide twice weekly and will be followed for treatment response and side effects. The results of this study may help determine whether chidamide could be a potential treatment option for patients with intermediate-to-high-risk MDS who have limited therapeutic choices.
Aged 60 years or older at the time of informed consent.
Diagnosis of myelodysplastic syndromes (MDS) according to the World Health Organization (WHO) classification.
International Prognostic Scoring System (IPSS) risk category of Intermediate-2 or High, with bone marrow blasts <15%.
Revised International Prognostic Scoring System (IPSS-R) risk category of Intermediate, High, or Very High, with bone marrow blasts <15%.
Bone marrow blasts >=15% at screening.
Prior treatment with chidamide.
Concurrent diagnosis of acute myeloid leukemia (AML) or other active hematologic malignancy.
Receipt of intensive chemotherapy, hypomethylating agents, or other investigational agents within 4 weeks before enrollment.
Single group
Chidamide
DrugChidamide will be administered orally at an initial dose of 20 mg twice weekly. Each dose consists of four 5-mg tablets taken approximately 30 minutes after meals. Treatment may continue for up to 3 months, with a planned follow-up period of 6 months. Dose modifications are permitted based on hematologic and non-hematologic toxicities. In the event of grade 3 or higher hematologic toxicity or clinically significant non-hematologic toxicity, chidamide will be temporarily withheld and may be resumed at a reduced dose after recovery, according to the investigator's judgment. Treatment will be discontinued if unacceptable toxicity persists despite dose interruption or dose reduction.
Overall Response Rate Assessed According to Study Protocol-Defined Response Criteria
Overall response rate is defined as the proportion of participants who achieve a protocol-defined treatment response after chidamide treatment. Response assessment will be based on hematologic parameters and bone marrow evaluation, as specified in the study protocol.
Incidence of Treatment-Emergent Adverse Events
Treatment-emergent adverse events will be assessed based on clinical evaluation, laboratory testing, and investigator assessment during the study period.
Proportion of Participants With Dose Interruption, Dose Reduction, or Treatment Discontinuation Due to Adverse Events
This outcome will assess the proportion of participants who require dose interruption, dose reduction, or permanent discontinuation of chidamide due to adverse events.
Proportion of Participants Achieving Hematologic Improvement
Hematologic improvement will be assessed based on changes in peripheral blood counts and transfusion requirements according to the study protocol.
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