Efficacy and Safety of HMAs Combined With Venetoclax Versus HMAs Alone in ND Int-and H-risk MDS or CMML

Efficacy and Safety of HMAs Combined With Venetoclax Versus HMAs Alone in Newly Diagnosed Patients With Intermediate-/High-Risk MDS and CMML : A Retro-prospective, Multicenter Observational Cohort Study

The aim of this study is to observe and analyze the clinical efficacy of Venetoclax+HMAs regimen in the treatment of newly diagnosed higher-risk MDS and CMML cases, and to compare the efficacy of venetoclax +HMAs regimen with that of HMAS regimen alone, in order to provide a normative scheme and basis for clinical use.

Study Overview

• Status: Active, not recruiting
• Conditions: MDS; CMML
• Intervention / Treatment: Drug: Venetoclax

• Study Type: Observational
• Enrollment (Estimated): 224

Contacts and Locations

Study Locations

• China
  • Guangdong
    • Guangzhou, Guangdong, China, 510000
      • Guangdong Provincial People's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Adults aged 18 years or older with newly diagnosed myelodysplastic agenda syndrome (MDS)and chronic myelomonocytic leukemia (CMML)according to the 2016WHO or 2022 WHO criteria

Description

Inclusion Criteria:

1. Age ≥18 years old, male or female;
2. MDS or CMML diagnosed by the 2016 or 222 WHO criteria, with a sustained survival of at least 12 weeks;
3. received more than two cycles of Venetoclax plus HMAs or HMAs alone without prior disease specific or allogeneic hematopoietic stem cell therapy (before initiating Venetoclax or HMAs, Medications such as hydroxyurea were allowed to reduce the white-cell count to 1.0×109 per liter or less.) ;
4. bone marrow blast cell count (BM blast > 5%) or IPSS-R score > 3 (intermediate risk, high risk, very high risk);
5. Eastern Cooperative Oncology Group (ECOG) performance status ≤2;
6. complete case information could be obtained.

Exclusion Criteria:

1. patients who did not meet the inclusion criteria;
2. inability to obtain complete case information or to follow protocol steps or follow up on time;
3. other conditions considered by the investigators to be unsuitable for inclusion.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Venetoclax+HMAs; treatment group, the intervention is the use of Venetoclax(400mg，QD, 1-14d)	Drug: Venetoclax; Previous phase Ib studies have fully demonstrated that venetoclax combined with HMAs is well tolerated in patients with MDS or CMML. Patients treated with VEN/AZA had a higher chance of SCT after remission and a higher survival benefit
Standard treatment(HMAs); The control group，Azacitidine（75mg/m2 ，QD, sc，d1-d7）or Decitabine（15mg/m2, q8h-q12h, d1-d3 or 20mg/m2,qd, d1-d5)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
overall response rate	CR+mCR+PR+HI	After the first cycles treatment（Day1, Month2)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
IWG2023 Composite response rate	CR+CR-L+CRh+CRequ	After the first cycles treatment（Day1, Month2 )
overall survival	Refers to the time from the start of treatment to the patient's death or last follow-up.	through study completion, an average of 1 year
Blood transfusion dependence	Change in proportion of transfusion dependence from the start of treatment	through study completion, an average of 1 year
adaverse event rate	Grade 3-4 adverse events	Up to 1 years after the last subject enrolled.

Collaborators and Investigators

• Sponsor: Guangdong Provincial People's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2020
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): March 30, 2027

Study Registration Dates

• First Submitted: November 16, 2025
• First Submitted That Met QC Criteria: January 4, 2026
• First Posted (Actual): January 6, 2026

Study Record Updates

• Last Update Posted (Actual): January 6, 2026
• Last Update Submitted That Met QC Criteria: January 4, 2026
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Keywords: MDS; Venetoclax; CMML; Hypomethylating agents
• Additional Relevant MeSH Terms: venetoclax
• Other Study ID Numbers: GDPH-2024-MDS-1.2

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No