Table 1 Characteristics of studies and subjects included in the review
Study author (year) | Study design | Gender (M/F) | Case Experimental vs. control | Patients’ characteristics | Intervention methods |
|---|---|---|---|---|---|
Abou-Alfa 2020 | RCT phase 3 | 68/117 | 185 124vs61 | Patients from 49 hospitals in six countries aged at least 18 years with histologically confirmed, advanced, IDH1-mutant cholangiocarcinoma who had progressed on previous therapy, and had up to two previous treatment regimens for advanced disease, an Eastern Cooperative Oncology Group performance status score of 0 or 1, and a measurable lesion as defined by Response Evaluation Criteria in Solid Tumors version 1.1. | Oral ivosidenib 500 mg once daily vs. or matched placebo |
Botton 2023 | RCT phase 3 | 181/132 | 319 158vs161 | Patients aged ≥ 60 years with de novo or secondary AML (World Health Organization classification20), a confirmed IDH2 gene mutation, and an Eastern Cooperative Oncology Group performance status score ≤ 2. At screening, patients were to have received 2 or 3 prior AML-directed therapies; prior hypomethylating agent (HMA) therapy for higher-risk myelodysplastic syndromes (MDS) also constituted an eligible prior therapy if the patient experienced progression to AML during or within 60 days after receiving the HMA. | Enasidenib 100 mg per day vs. conventional care regimen (CCR) |
DiNardo 2021 | RCT phase 2 | / | 101 68vs33 | Eligible patients were aged 18 years or older and had newly diagnosed, mutant-IDH2 acute myeloid leukaemia, and an Eastern Cooperative Oncology Group performance status of 0–2 | Enasidenib plus azacitidine vs. azacitidine only |
Montesinos 2022 | RCT phase 3 | 80/66 | 146 72vs74 | Age of 18 years or older and a centrally confirmed diagnosis of previously untreated IDH1-mutated acute myeloid leukemia determined with the Food and Drug Administration–approved Abbott RealTime IDH1 in vitro polymerase-chain-reaction (PCR) assay. Additional eligibility criteria included no previous treatment with an IDH1 inhibitor or hypomethylating agent for myelodysplastic syndrome, an Eastern Cooperative Oncology Group (ECOG) performance-status score of 0 to 2 (on a 5-point scale in which higher scores indicate greater disability), and adequate hepatic and renal function. | Oral ivosidenib (500 mg once daily) and subcutaneous or intravenous azacitidine (75 mg per square meter of body-surface area for 7 days in 28-day cycles) or to receive matched placebo and azacitidine |