AZA+Lus VS AZA Monotherapy in HR-MDS

Chinese Academy of Medical Sciences & Peking Union Medical College

Status and phase

Not yet enrolling

Phase 3

Conditions

Higher-risk Myelodysplastic Syndrome

Treatments

Drug: Azacitidine (AZA)

Drug: Luspatercept

Study type

Interventional

Funder types

Other

Identifiers

NCT06927232

AZA-Lus-HRMDS

Details and patient eligibility

About

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.

Full 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.

Enrollment

86 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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