Table 2.
Efficacy and toxicity of BET inhibitors in patients with hematological cancers.
| BETi | Drug Combination | Target Disease and Criteria of Inclusion | Clinical Trial (Number of Patients, Age) | Response | Adverse Effects |
|---|---|---|---|---|---|
| ABBV-075 | Venetoclax | AML, MM |
NCT02391480 (n = 128, ≥18 years old) |
No data available | No data available |
| FT-1101 | Azacitidine |
R/R AML (FLT3-ITD or FLT3-TKD mutated) MDS (eligible to receive azacitidine), R/R NHL (primary mediastinal, DLBCL and B-cell lymphoma) |
NCT02543879 (n = 94, ≥18 years old) |
No data available | No data available |
| OTX015/MK-8628 | - |
AML Ph + ALL MM (exposed to at least one alkylating agent/corticosteroid/IMiD and bortezomib) DLBCL (≥1 nonirradiated tumor mass ≥750 mm3). (failure of all standard therapies and life expectancy ≥3 months) |
NCT01713582 (n = 141, ≥18 years old) |
DOR in 6,6% of DLBCL patients and clinical activity without ORC in 13,3% patients | Serious adverse effects: Thrombocytopenia, febrile neutropenia, leukocytosis, sinus bradycardia, gastrointestinal events (grade 1–2), fatigue, asthenia (grade 1–2) and infections |
| Azacitidine | AML | NCT02303782 † | No data available | No data available | |
| CPI0610 | - | MM (progressed after at least one line of standard therapy) |
NCT02157636 (n = 30, ≥18 years old) |
No data available | No data available |
| - | Lymphoma (progressed after/prior treatment and without available/effective standard therapy) | NCT01949883 (n = 64, ≥18 years old) |
No data available | No data available | |
| PLX51107 | - | R/R AML or NHL (life expectancy ≥3 months) | NCT02683395 †† (n = 50, ≥18 years old) |
No data available | No data available |
| BAY1238097 | - | MM (refractory to standard treatment or with no standard therapy available, life expectancy ≥3 months) | NCT02369029 †† (n = 8, ≥18 years old) |
No data available | No data available |
| GS-5829 | Exemestane, Fulvestrant | DLBCL, PTCL (refractory to or intolerant of standard therapy or no standard therapy available) | NCT02392611 (n = 33, ≥18 years old) |
No data available | No data available |
| INCB054329 | - | DLBCL, AML, MM, ALL, BL (progressed following at least 1 line of therapy without further approved therapy available) | NCT02431260 †† (n = 69, ≥18 years old) |
PK variations among individuals |
Serious adverse effects Anemia, neutropenia, thrombocytopenia, gastrointestinal disorders, fatigue, hyperbilirubinemia, pneumonia, multi-organ failure, hepatic vein thrombosis, infections increased alanine and aspartate aminotransferases, hyperbilinubinemia, respiratory, thoracic, and mediastinal disorders |
| RO6870810 | - | R/R AML and MDS (allogeneic stem cell transplant not treated with immunosuppressors for ≥2 weeks, life expectancy ≥2 months) |
NCT02308761 (n = 26, ≥18 years old) |
No data available | No data available |
Abbreviations: Acute lymphoblastic leukemia (ALL), Acute myeloid leukemia (AML), Burkitt lymphoma (BL), Diffuse large B-cell lymphoma (DLBCL), Multiple myeloma (MM), Myelodysplastic syndrome (MDS), Non-Hodgkin lymphoma (NHL), Relapsed/Refractory (R/R), Durable Objective Response (DOR), Objective Response Criteria (ORC). † Withdrawn †† Terminated. Philadelphia chromosome positive (Ph+), Immunomodulatory drug (IMiD).