Efficacy and safety of anti-CD19 CAR T-cell therapy in 110 patients with B-cell acute lymphoblastic leukemia with high-risk features.

Anti-CD19 chimeric antigen receptor (CAR) T-cell therapy is effective in patients with advanced B-cell acute lymphoblastic leukemia (B-ALL). However, efficacy data is sparse in subgroups of patients with high-risk features such as BCR-ABL+, TP53 mutation, extramedullary disease (including central nervous system leukemia) or posttransplant relapse. It is also uncertain whether there is an added benefit of transplantation after anti-CD19 CAR T-cell therapy. We conducted a phase 1/2 study of 115 enrolled patients with CD19+ B-ALL. A total of 110 patients were successfully infused with anti-CD19 CAR T cells. In all, 93% of patients achieved a morphologic complete remission, and 87% became negative for minimal residual disease. Efficacy was seen across all subgroups. One-year leukemia-free survival (LFS) was 58%, and 1-year overall survival (OS) was 64% for the 110 patients. Seventy-five nonrandomly selected patients (73.5%) subsequently received an allogeneic hematopoietic stem cell transplant (allo-HSCT). LFS (76.9% vs 11.6%; P < .0001; 95% confidence interval [CI], 11.6-108.4) and OS (79.1% vs 32.0%; P < .0001; 95% CI, 0.02-0.22) were significantly better among patients who subsequently received allo-HSCT compared with those receiving CAR T-cell therapy alone. This was confirmed in multivariable analyses (hazard ratio, 16.546; 95% CI, 5.499-49.786). Another variate that correlated with worse outcomes was TP53 mutation (hazard ratio, 0.235; 95% CI, 0.089-0.619). There were no differences in complete remission rate, OS, or LFS between groups of patients age 2 to 14 years or age older than 14 years. Most patients had only mild cytokine release syndrome and neurotoxicity. Our data indicate that anti-CD19 CAR T-cell therapy is safe and effective in all B-ALL subgroups that have high-risk features. The benefit of a subsequent allo-HSCT requires confirmation because of nonrandom allocation. This trial was registered at www.clinicaltrials.gov as #NCT03173417.

5 years of clinical DC-CIK/NK cells immunotherapy for acute myeloid leukemia - a summary.

Aim: To assess the efficacy of dendritic cells-cytokine induced killer (DC-CIK) and natural killer (NK) cell-based immunotherapy in treating the low- and intermediate-risk acute myeloid leukemia. Patients & methods: DC-CIK or NK cells were infused once every 3 months for 2-4 cycles to 85 patients. Results & conclusion: The 5-year overall survival (OS) and relapse-free survival (RFS) rates were 90.5 and 65.2%, respectively. The OS of the very favorable, the favorable and the intermediate-risk groups was 94.4, 86.3 and 93.3% (p = 0.88), and the RFS 83.3, 81.8 and 62.2% (p = 0.14), respectively. The OS and RFS of the 60 patients treated with DC-CIK alternating with NK cells were better than the 25 patients treated with DC-CIK or NK alone (96.5 vs 71.2%; p = 0.003. 79.5 vs 28.9%; p < 0.001).

Application of minimally invasive video-assisted technique in papillary thyroid microcarcinoma.

BACKGROUND: Minimally invasive video-assisted technique (MIVAT) was initially described by Miccoli and colleagues in Italy. The MIVAT without gas infusion was introduced in the clinical practice to treat small benign thyroid nodules and had advantages in terms of cosmetic results compared with the conventional approach. OBJECT: To compare the outcome in papillary thyroid microcarcinoma (PTMC) patients treated with MIVAT and conventional procedure, and evaluate the feasibility of MIVAT applied in PTMC patients. MATERIALS AND METHODS: Data of 35 PTMC patients with MIVAT and 33 others with conventional procedure were analyzed and compared, including postoperative outcomes, operative time, incidence of complications, the completeness of operation, and the prognosis of tumor after a follow-up of 5 years. RESULTS: With regard to the postoperative outcomes, a significantly statistical difference was found between MIVAT group and the conventional procedure group. The mean operative time in the MIVAT group was longer than that in the conventional group. However, it was similar to the convention group when only the mean operative time of the last 5 patients in the MIVAT group was estimated. The rate of temporary hypoparathyroidism was significantly lower in MIVAT group compared with the convention group, and the incidences of other complications had no significant difference. With regard to the completeness of operation and the prognosis of tumor, no differences were found between the 2 groups. CONCLUSIONS: MIVAT can be safely applied in PTMC patients with positive impact on patients outcome by comparable results to a conventional procedure after a median follow-up of 5 years. Thus it is a better alternative therapeutic treatment for patients with PTMC.