Anti-CLL1-based CAR T-cells with 4-1-BB or CD28/CD27 stimulatory domains in treating childhood refractory/relapsed acute myeloid leukemia.

Background: Though the efficacy of anti C-type lectin-like molecule-1 (CLL1) CAR T-cells in refractory/relapsed acute myeloid leukemia (R/R-AML) have been occasionally reported, the influence of co-stimulatory domain CAR T-cells is not investigated so far.

Method: Seven R/R-AML children treated with anti-CLL1 CAR T-cells were enrolled onto this preliminary comparison study. Among these seven patients, four received CD28/CD27-based CAR T-cells therapy, and three received 4-1BB-based CAR T-cells therapy.

intronic mutation leads to diamond-blackfan anemia like disease.

is required for normal erythropoiesis. Exonic/intronic mutations causes Diamond-Blackfan Anemia (DBA)-like disease. Herein, we present a case of a 5-year-old boy with anemia of unknown etiology.

Observation of Alectinib- and Crizotinib- included chemotherapy in children with ALK-positive anaplastic large cell lymphoma: A single institutional experience.

Approximately one-third children with anaplastic large cell lymphoma (ALCL) relapse after completion of chemotherapy, particularly for those high-risk patients. The introduction of novel therapeutic modalities is much needed for these sub-group patients. Two groups (n = 3, n = 4) of ALCL patients were treated with crizotinib- and alectinib-included ALCL-99 therapy, respectively, achieving complete remission rates of 66.

Characteristics of anti-CLL1 based CAR-T therapy for children with relapsed or refractory acute myeloid leukemia: the multi-center efficacy and safety interim analysis.

C-type lectin-like molecule-1 (CLL1) is preferentially expressed on acute myeloid leukemia (AML) stem cells and AML blasts, which can be considered as AML-associated antigen. Anti-CLL1-based CAR-T cells exhibited effective tumor-killing capacity in vitro and in AML-bearing mouse model. In this report, eight children with relapsed or refractory AML (R/R-AML) were recruited for a phase 1/2 clinical trial of autologous anti-CLL1 CAR-T cell immunotherapy.

Effects of Mutations on Leukemogenesis and Targeting of Children With Acute Lymphoblastic Leukemia.

Through the advancements in recent decades, childhood acute lymphoblastic leukemia (ALL) is gradually becoming a highly curable disease. However, the truth is there remaining relapse in ∼15% of ALL cases with dismal outcomes. mutations, in particular mutations, were predominant mutations affecting relapse susceptibility.