6/28/23, 11:37 PM IRIS Pathway Feedback Search Clinical Topics Home Studies IRIS IRIS Disease Chronic myeloid leukemia Trial question What is the role of imatinib in patients with newly diagnosed chronic-phase CML? Study design Multi-center Open label RCT Population Characteristics of study participants 41.0% female N = 1106 59.0% male 1106 patients (455 female, 651 male) Inclusion criteria: patients with newly diagnosed chronic-phase CML Key exclusion criteria: uncontrolled serious medical conditions, prior chemotherapy or treatment with any investigational agent, hematopoietic-cell transplantation, major surgery within the preceding four weeks, or seropositive for the human immunodeficiency virus Interventions N=553 imatinib (400 mg PO daily) N=553 interferon alfa plus low-dose cytarabine (interferon alfa, target dose 5 million U per square meter of body-surface area per day and cytarabine subcutaneous 40 mg of maximal daily dose for 10 days every month) Primary outcome Complete cytogenetic response 76.2 % 76.2 https://web.pathway.md/studies/recGeHWBERvXXDDzR 1/2 6/28/23, 11:37 PM IRIS Pathway 57.2 % 38.1 % Significant increase 19.1 % NNT = 2 14.5 0.0 % Imatinib Interferon alfa plus low-dose cytarabine Significant increase in complete cytogenetic response (76.2% vs. 14.5%; RR 5.26, 95% CI 2.14 to 8.38) Secondary outcomes Significant increase in freedom from progression to accelerated-phase or blast-crisis CML (96.7% vs. 91.5%; RR 1.06, 95% CI 0.43 to 1.69) Significant increase in complete hematologic response (95.3% vs. 55.5%; RR 1.72, 95% CI 0.7 to 2.74) Safety outcomes Significant difference in discontinuation of treatment or crossover to the alternative treatment group (14.3% vs. 89.2%). Conclusion In patients with newly diagnosed chronic-phase CML, imatinib was superior to interferon alfa plus low-dose cytarabine with respect to complete cytogenetic response. Reference O'Brien SG, Guilhot F, Larson RA et al. Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med. 2003 Mar 13;348(11):994-1004. Open reference URL https://web.pathway.md/studies/recGeHWBERvXXDDzR 2/2