Recently completed CMML-specific clinical trials
| Therapeutic agent . | Mechanism . | Phase . | Patient population . | Subjects enrolled (n) . | Outcome . | References . |
|---|---|---|---|---|---|---|
| Decitabine (vs Hydroxyurea) | HMA | 3 | Untreated, advanced proliferative CMML | 170 (decitabine, 84) | EFS HR, 0.83, (95% CI, 0.59-1.16), OS HR 1.08 (95% CI, 0.76-1.54) | DACOTA study13 |
| Ruxolitinib | JAK 1/2 inhibitor | 1/2 | CMML (prior therapy allowed, not required) | 50 | ORR, 38%∗ | 16 |
| Lenzilumab | Recombinant anti-GM-CSF monoclonal antibody | 1 | CMML; R/R, intolerant, or ineligible for prior treatment | 15 | ORR, 33%∗ | 17 |
| Tipifarnib | Farnesyltransferase inhibitor | 2 | CMML and MDS/MPN | 44 (CMML, 37) | ORR, 21.9%∗ in patients with CMML | 18 |
| Tagraxofusp | CD123-directed cytotoxin (IL-3 fused to diphtheria toxin) | 1/2 | CMML; R/R and 1L | 36 | 11% bone marrow morphologic CR; 42% spleen response† | 19 |
| Therapeutic agent . | Mechanism . | Phase . | Patient population . | Subjects enrolled (n) . | Outcome . | References . |
|---|---|---|---|---|---|---|
| Decitabine (vs Hydroxyurea) | HMA | 3 | Untreated, advanced proliferative CMML | 170 (decitabine, 84) | EFS HR, 0.83, (95% CI, 0.59-1.16), OS HR 1.08 (95% CI, 0.76-1.54) | DACOTA study13 |
| Ruxolitinib | JAK 1/2 inhibitor | 1/2 | CMML (prior therapy allowed, not required) | 50 | ORR, 38%∗ | 16 |
| Lenzilumab | Recombinant anti-GM-CSF monoclonal antibody | 1 | CMML; R/R, intolerant, or ineligible for prior treatment | 15 | ORR, 33%∗ | 17 |
| Tipifarnib | Farnesyltransferase inhibitor | 2 | CMML and MDS/MPN | 44 (CMML, 37) | ORR, 21.9%∗ in patients with CMML | 18 |
| Tagraxofusp | CD123-directed cytotoxin (IL-3 fused to diphtheria toxin) | 1/2 | CMML; R/R and 1L | 36 | 11% bone marrow morphologic CR; 42% spleen response† | 19 |
Summary of key features from recently completed trials conducted in the patient population with CMML.
CI, confidence interval; CR, complete remission; EFS, event-free survival; GM-CSF, granulocyte-macrophage colony-stimulating factor; HR, hazard ratio; IL-3, interleukin-3; ORR, overall response rate; R/R, relapsed/refractory; 1L, frontline.
Used MDS/MPN specific response criteria; overall response rate defined by complete remission + complete cytogenetic remission + partial remission + marrow response + clinical benefit.20
In evaluable patients with baseline splenomegaly; spleen response is defined by MDS/MPN response criteria.