Table 1 Characteristics of the prospective clinical studies (including randomized and non-randomized studies) and patients included in the meta-analysis
Author/year | NCT number | Study design | Nation | Stage | IDH mutations type | Median age (year) | Sample size | Interventions | Median follow-up (months) | Outcomes | Scores* |
|---|---|---|---|---|---|---|---|---|---|---|---|
Venugopalet et al. [14] | NCT03683433 | Prospective phase 2 study | United States | Newly diagnosed IDH2-mutated AML | IDH2-mutated | NA | 7 | Enasidenib + Azacitidine + venetoclax or FLT3i | 13.1 | A | 15 |
Venugopal [14] et al | NCT03683433 | Prospective phase 2 study | United States | IDH2-mutated R/R AML | IDH2-mutated | NA | 19 | Enasidenib + Azacitidine + venetoclax or FLT3i | NA | A | 15 |
Montesinos et al. [21] | NCT03173248 | Double-blind, randomized, placebo-controlled, phase 3 trial | United States, Australia, and Brazil et. al | Newly diagnosed IDH1-mutated AML | R132C, R132H, R132G, R132L, R132S | 76.0 | 72 | Ivosidenib + Azacitidine | 12.4 | A,B,C,D,E,G,H | 7 |
DiNardo et al. [22] | NCT02677922 | Prospective phase Ib study | United States, Canada, and France et. al | Newly diagnosed IDH1-mutated AML | R132C, R132H, R132L, | 76.0 | 23 | Ivosidenib + Azacitidine | 16.1 | A,B,H | 15 |
DiNardo et al. [23] | NCT02677922 | Open-label, randomized, phase 1b/2 trial | United States, Canada, and France et. al | Newly diagnosed IDH2-mutated AML | R140(75%), R172 (24%), data missing (1%) | 75 | 68 | Enasidenib + Azacitidine | 14·9 | A,B,C,E,G,H | 3 |
Stein et al. [24] | NCT02632708 | Prospective phase 1 study | United States, Germany, and Netherlands | Newly diagnosed IDH-mutated AML | IDH1-mutated and IDH2-mutated | NA | 151 | Enasidenib or Ivosidenib + intensive chemotherapy | NA | A,B,C | 16 |
Botton et al. [25] | NCT02577406 | Open-label, randomized, phase 3 trial | United States, Brazil, and Belgium et. al | IDH2-mutated R/R AML | IDH2-R140 (72.8%), IDH2-R172 (27.2%) | 72 | 158 | Enasidenib | NA | A,B,C,D,E,F,G,H | 3 |
Roboz et al. [26] | NCT02074839 | Prospective phase 1 study | United States and France | Newly diagnosed IDH1-mutant AML | IDH1-R132 (100%) | 76.5 | 34 | Ivosidenib | 23.5 | A,B,C,G,H | 14 |
DiNardo et al. [27] | NCT02074839 | Prospective phase 1 study | United States and France | IDH1-mutated R/R AML | R132C(60.8%), R132H (21.6%), R132G/L/S(15.2%), wild-type (0.8%), other (1.6%) | 76.5 | 258 | Ivosidenib | 14.8 | A,B,C,D,H | 16 |
Stein et al. [28] | NCT01915498 | Prospective phase 1/2 study | United States and France | IDH2-mutated R/R AML | R140(75%), R172 (25%) | 68 | 280 | Enasidenib | 7.8 | A,B,C,D,F,G | 16 |
Pollyea et al. [29] | NCT01915498 | Prospective phase 1/2 study | United States and France | Newly diagnosed IDH2-mutated AML | R140(67%), R172 (31%), other/missing(3%) | 77 | 39 | Enasidenib | 8.4 | A,B,C,D,E,F,G,H | 15 |