AZA+Lus VS AZA Monotherapy in HR-MDS
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Azacitidine (AZA), Luspatercept.
- Who it may be relevant to
- Registry conditions: Higher-risk Myelodysplastic Syndrome. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Azacitidine Plus Luspatercept Versus Azacitidine Monotherapy in Higher-risk Myelodysplastic Neoplasms: a Randomized Prospective Study
Overview
This study is a randomized, prospective, single-center, open-label cohort study involving untreated HR-MDS patients. The patients were divided randomized into AZA+Lus cohort and AZA monotherapy cohort.
Detailed description
The hypomethylating agents (HMA) azacitidine (AZA) and decitabine (DEC) have been shown to improve survival and delay disease progression in patients with high-risk MDS. They are recommended by the NCCN as first-line treatments for patients with high-risk MDS. Clinical trials have demonstrated an OR rate of approximately 40-50% with AZA in patients with high-risk MDS. Despite the efficacy of HMA therapy, the rate of transfusion independence remains low. Anemia remains the most prominent symptom in refractory patients, with very limited options for subsequent treatment. Prolonged anemia affects every organ function and seriously affects the prognosis of patients.
Luspatercept is currently approved for the treatment of patients with both erythropoiesis receptor agonist ( ESA) treatment failures in transfusion-dependent low-risk MDS-RS patients. In a randomized controlled phase III clinical trial, compared to a placebo group, luspatercept significantly improved transfusion dependence and improved hemoglobin and quality of life in refractory MDS-RS patients. A recent conference report suggested that there was no significant difference in efficacy between low-risk and high-risk patients treated with luspatercept and that the HI rate for high-risk patients treated with luspatercept monotherapy was approximately 50%.
Thus this study aimed to compare the efficacy of AZA+luspatercept and AZA monotherapy.
Interventions
- Drug Azacitidine (AZA)
Azacitidine 75mg/m/ day \*5 days, 28 days for 1 course - Drug Luspatercept
Luspatercept 1.0 mg/kg subcutaneously every 3 weeks, adjusted according to hemoglobin, up to 1.75mg/kg. If hemoglobin ≥120g/L, luspatercept can be discontinued.
Primary outcome measures
- overall response rate [Time frame: 3 months, 6 months]
Secondary outcome measures (6)
- complete response rate [Time frame: 3 months, 6 months]
- rate of transfusion independence [Time frame: 3 months, 6 months]
- adverse event rate [Time frame: through study completion, an average of 1 year]
- relapse rate [Time frame: through study completion, an average of 1 year]
- progress-free survival [Time frame: through study completion, an average of 1 year]
- overall survival [Time frame: through study completion, an average of 1 year]
Eligibility criteria
Inclusion criteria
- Age ≥18 years old
- Diagnosed as higher-risk MDS (IPSS intermediate-2/high-risk, or IPSS-R >3.5, or IPSS-M moderate high-, high-, very high-risk)
- Untreated patients
- Liver and kidney function less than 2 times of upper limit of normal
- ECOG≤2 and expected survival more than 6 months
- Informed consent signed
Exclusion criteria
- With active infection
- Other malignant tumors
- Obvious abnormal liver and kidney function, or abnormal function of other organs
- Combined with myelofibrosis
- Have undergone bone marrow transplantation
- Pregnant or lactating women, or men who have recent reproductive needs
- Allergic to azacytidine, Rotercept or excipients
- History of polysorbate 80 allergy
- Refuse to sign informed consent
- Researchers consider it inappropriate to participate in the experiment
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT06927232 · AZA-Lus-HRMDS