Abstract: Objective　To investigate clinical and biological characteristics and prognosis of pediatric inv(16)/CBFβMYH11-positive acute myeloid leukemia. Methods　From December 2008 to September 2017, 30 patients diagnosed as inv(16)/ CBFβ-MYH11-positive acute myeloid leukemia were enrolled in this study and their clinical and biological features were retrospectively analyzed. Following-up of 30 patients were ended in November 2017, and the survival status was calculated by K-M curve and prognostic factors were analyzed by COX model. Results　At preliminary diagnosis of the total 30 patients, 19 patients had hepatomegaly (63.3%), 18 cases had splenomegaly (60.0%), and 19 cases had lymphoadenopathy (63.3%). Patients at diagnosis with high leukocyte counts （≥50×109/L） accounted for 63.3%. In all patients, the 5-year anticipated EFS and OS were （84.2±7.6）% and（89.8±5.6）%, respectively. Cox multivariate regression analysis showed that gender, age at onset, leukocyte count, lactate dehydrogenase level, extramedullary leukemia, c-kit gene mutation, chromosome inv (16) alone, initial CBFβ-MYH11 quantification and the response to the first course of treatment did not statistically affect EFS and OS (P>0.05).  The level of CBFβ-MYH11 gene at diagnosis was used as the baseline to determine whether the level of gene after treatment had a more than 2 logarithmic (2-log) reduction. When patients with a more than 2-log reduction of CBFβ-MYH11 transcript levels (≥2-log) compared with other patients after a course of induction therapy, the differences of 5-year EFS and OS were not statistically significant (P>0.05). When periodically monitoring the CBFβ-MYH11 gene, three cases with progressively increasing CBFβ-MYH11 fusion gene during the consolidation therapy were identified, and no relapse was found after adjustment of treatment intensity. All of them survived so far. Conclusions　In patients with inv(16)/CBFβ-MYH11-positive AML, hepatosplenomegaly, lymphadenopathy and high initial leukocyte counts are most common features, and the therapeutic effect is good. It is important to regularly monitor CBFβ-MYH11 quantitation in the consolidation therapy phase.