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Association details:
| Biomarker: | SF3B1 mutation |
|---|---|
| Cancer: | Myelodysplastic Syndrome |
| Drug: | Reblozyl (luspatercept-aamt) (TGFβ inhibitor) |
| Direction: | Sensitive |
Evidence:
Source:
Excerpt:
Myelodysplastic syndrome:...In patients...with an SF3B1 mutation, if no response is observed after 2 months of ESA treatment with or without G-CSF, treatment with luspatercept is recommended
Source:
Title:
A Study to Evaluate the Efficacy, Pharmacokinetics and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) in Chinese and Japanese Subjects With Ring Sideroblasts Who Require Red Blood Cell Transfusions
Excerpt:
...- Ring sideroblast ≥ 15% of erythroid precursors in bone marrow or ≥ 5% (but <15%) if SF3B1 mutation is present....
Source:
Title:
A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India
Excerpt:
...If the SF3B1 mutation is present, RS ≥ 5% will be included....
Trial ID:
More C2 evidence
Source:
Title:
Luspatercept for Anemia in Lower Risk MDS or Non-proliferative MDS/MPN Neoplasms
Excerpt:
...Cohort 1: detectable splicing mutation other than SF3B1 2....
Trial ID:
Source:
Title:
Assessment of Effectiveness and Safety of Luspatercept in Patients Suffering From Lower-risk Myelodysplastic Syndrome.
Excerpt:
...- Ring sideroblasts (RS) ≥ 15% of erythroid precursors in bone marrow or ≥ 5% if SF3B1 mutation is present...
Trial ID:
Source:
Title:
A Study of Luspatercept (ACE-536) to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes
Excerpt:
...Documented diagnosis of MDS according to World Health Organization (WHO)/French American British (FAB) classification that meets IPSS R classification of very low, low, or intermediate risk disease, and: Ring sideroblast ≥ 15% of erythroid precursors in bone marrow or ≥ 5% (but < 15%) if SF3B1 mutation is present....
Trial ID:
Source:
Title:
Determination of the effectiveness and safeness of the drug luspatercept in patients who suffering from a low risk type of cancer when blood-forming cells in the bone marrow become abnormal and having characterized by decreased red blood cells below normal.
Excerpt:
...Subject has documented diagnosis of MDS according to WHO classification that meets IPSS-R classification[4] of very low-, low-, or intermediate-risk disease, and the following:• Ring sideroblasts (RS) ≥ 15% of erythroid precursors in bone marrow or ≥ 5% if SF3B1 mutation is present• Less than 5% blasts in bone marrow• Peripheral blood white blood cell (WBC) count < 13,000/μL4. ...
Trial ID:
Source:
Title:
Efficacy and Safety of Luspatercept: A Study by Fondazione Italiana Sindromi Mielodisplastiche
Excerpt:
...- Ring Sideroblasts (RS) ≥15% of erythroid precursors in bone marrow in the absence of SF3B1 mutation, or ≥5% in the presence of SF3B1 mutation;...
Trial ID:
Less C2 evidence
Source:
Title:
1871 Improved Overall Survival Among Luspatercept Responders and Predictors of Response
Published date:
11/02/2023
Excerpt:
Among pts with SF3B1 MT, we observed a trend for higher response in pts with the hotspot K700E, 62.7% (37/59) vs 47.4% (27/57) in non-K700E, p=.097. In pts with SF3B1 MT, divided by median VAF, we observed a trend for better HI with VAF was >38, 63.5% (33/52) vs 45.3% (24/53), p=.062...Our RWD in the largest luspa treated cohort demonstrates OS benefit to luspa response...Low baseline RBC-TB dependency, lower risk IPSS-M, SF3B1 MT, SF3B1-K700E mutation and SF3B1α co-mutations correlated with higher response rates.
Source:
Title:
1757 Luspatercept for Treatment of Lower Risk Myelodysplastic Syndromes: Real World Data Replicates Medalist Study Results and Confirms Activity Among Hypomethylating Agents and Lenalidomide Treated Patients
Published date:
11/03/2022
Excerpt:
We present to our knowledge first real-world data (RWD) in a large cohort of pts treated at our center with luspatercept for LR-MDS....Between February 2020 and December 2021, 114 pts were treated with luspatercept….The response rates were higher among SF3B1 MT pts compared to wild type, 48% (34/80) compared to 16% (5/32), p=.002….In multivariable analysis both SF3B1 mutation status and baseline RBC TB independently correlated with luspatercept response but not HMA failure....Low baseline RBC-TB dependency and SF3B1 MT correlated with higher response rates.
DOI:
https://doi.org/10.1182/blood-2022-169690
