Clinical characteristics and outcomes of treated patients
| Treated patient . | PIT dose . | Disease with relevant mutations . | Best response . | Outcome . | Grade 3-5 adverse events∗ . |
|---|---|---|---|---|---|
| 1 | 2 mg | AML (+9; ASXL1, TET2, ETV6; progression from MDS) | CR | Achieved CR after 1 cycle but was MRD-positive on flow cytometry. Passed away shortly thereafter due to infection. | Leukopenia (grade 3), neutropenia (4), thrombocytopenia (4), lung infection (5) |
| 2 | 2 mg | CLL (11q-, 13q-, unmutated IgVH) | CR | Treatment discontinued early due to neutropenia but was MRD-negative by clonoSEQ negative at the end of therapy. Had a history of cirrhosis and passed away during admission for acute encephalopathy; CLL was in remission when passed.† | Neutropenia (grade 4), pancreatitis (3) |
| 3 | 2 mg | SLL (11q-; IgVH unknown) | CR | Was in remission at the last follow-up.† | |
| 4 | 4 mg | AML (del 20q, +8; progression from MDS) | CR | Treatment discontinued due to recurrent pericardial effusion, unclear if related. Resumed AZA for treatment of MDS, but AML remained in remission at the last follow-up. | Febrile neutropenia (grade 3), vasovagal reaction (3) |
| 5 | 4 mg | CLL (IgVH-negative, FISH unable to be done) | CR | Clinically remains in remission. MRD positive by peripheral blood flow (0.01%).† | Leukopenia (grade 3), neutropenia (4), thrombocytopenia (4), anemia (3) |
| 6 | 4 mg | SLL (trisomy 12; IgVH status unknown) | CR | In CR based on CT scans. MRD-negative by peripheral blood flow. |
| Treated patient . | PIT dose . | Disease with relevant mutations . | Best response . | Outcome . | Grade 3-5 adverse events∗ . |
|---|---|---|---|---|---|
| 1 | 2 mg | AML (+9; ASXL1, TET2, ETV6; progression from MDS) | CR | Achieved CR after 1 cycle but was MRD-positive on flow cytometry. Passed away shortly thereafter due to infection. | Leukopenia (grade 3), neutropenia (4), thrombocytopenia (4), lung infection (5) |
| 2 | 2 mg | CLL (11q-, 13q-, unmutated IgVH) | CR | Treatment discontinued early due to neutropenia but was MRD-negative by clonoSEQ negative at the end of therapy. Had a history of cirrhosis and passed away during admission for acute encephalopathy; CLL was in remission when passed.† | Neutropenia (grade 4), pancreatitis (3) |
| 3 | 2 mg | SLL (11q-; IgVH unknown) | CR | Was in remission at the last follow-up.† | |
| 4 | 4 mg | AML (del 20q, +8; progression from MDS) | CR | Treatment discontinued due to recurrent pericardial effusion, unclear if related. Resumed AZA for treatment of MDS, but AML remained in remission at the last follow-up. | Febrile neutropenia (grade 3), vasovagal reaction (3) |
| 5 | 4 mg | CLL (IgVH-negative, FISH unable to be done) | CR | Clinically remains in remission. MRD positive by peripheral blood flow (0.01%).† | Leukopenia (grade 3), neutropenia (4), thrombocytopenia (4), anemia (3) |
| 6 | 4 mg | SLL (trisomy 12; IgVH status unknown) | CR | In CR based on CT scans. MRD-negative by peripheral blood flow. |
Patient 4 had febrile neutropenia that was likely related to PIT or disease, and the dose of PIT was reduced to 2 mg. The vasovagal reaction was not related to treatment. This patient also experienced grade 2 pleural effusion, possibly due to PIT or disease.
Patient 5 had thrombocytopenia before study enrollment. Leukopenia, neutropenia, and anemia were related to VEN and disease.
CR, complete response; CT, computed tomography; FISH, fluorescence in situ hybridization; IgVH, immunoglobulin heavy chain variable region; MDS, myelodysplastic syndrome.
Patient 1 had leukopenia, neutropenia, and thrombocytopenia that were related to a combination of the disease and VEN; grade 5 lung infection was believed to be a result of the disease. Patient 2 experienced neutropenia related to PIT and VEN, and pancreatitis was not related to treatment or disease. Dosing of VEN and PIT were both reduced.
Study was prematurely terminated for patients 2, 3, and 5. See the text for details.