Successful Anti-CLL1 CAR T-Cell Therapy in Secondary Acute Myeloid Leukemia.

Secondary acute myeloid leukemia (sAML) is a high-risk AML evolving from heterogenous prior hematological disorders. Compared to de novo AML, sAML has even worse responses to current therapy and thus is associated with lower remission rates, inferior overall survival (OS) and higher relapse rates. Many efforts have been devoted to improving the overall but with limited success, and novel strategy is thus highly needed.

Assessment of iron overload in very young children with limited thalassemia care resources in South China.

Southern China has one of the world's largest population of patients needing transfusions. Transfusion and chelation are not uniformly available and no magnetic resonance imaging (MRI) assessment data exists to date. A total of 153 young β-thalassemia major (β-TM) patients were assessed using a validated 1.

[Influence of total nucleated cell dose on the efficacy of cord blood transplantation].

Objective: To study the influence of cord blood total nucleated cell (TNC) dose on the efficacy of cord blood transplantation in children.

Methods: Thirty-four children with hematological disease received cord blood transplantation. They were assigned to 3 groups according to the infused TNC dose: TNC>10 x 10(7)/kg (n=7), 10 x10(7)/kg>TNC> or =7 x 10(7)/kg (n=9) and TNC<7 x 10(7)/kg (n=18).

[Haploidentical hematopoietic stem cell transplantation for beta-thalassemia major in children].

Objective: Hematopoietic stem cell transplantation is currently a unique curative therapy for beta-thalassemia major. However, only 30% of patients have HLA-identical siblings to serve as donors. This study investigated the feasibility of hematopoietic stem cell transplantation from HLA mismatched related donors for beta-thalassemia major in children.

[Relationship between cellular immune function during conditioning and graft rejection in patients with beta-thalassemia major].

Objective: To analyze the relationship between cell-mediated immune function during conditioning and graft rejection in patients with beta-thalassemia major.

Methods: Allogeneic hematopoietic stem cell transplantation was performed in 25 children with beta-thalassemia major and 11 with acute leukemia group. The percentages of T lymphocytes and natural killer (NK) cells in peripheral blood of these patients were detected with dual color immunofluorescence on day -10 (before conditioning) and day -5 (after conditioning), and the relationship between the cellular immune function and graft rejection was analyzed.

[Relationship between growth disorders and iron overload in children with beta-thalassemia major].

Objective: To study the status of growth and development and the relationship between growth disorders and iron overload in children with beta-thalassemia major.

Methods: Fifty children with beta-thalassemia major and who received blood transfusion therapy regularly (age: 9 months-17 years) were enrolled. They were subjected to a thorough history taking, clinical examinations, and laboratory examinations, including complete blood count, alanine transferasa (ALT) and serum ferritin.

[Combined transplantation of umbilical cord blood and bone marrow from same sibling donor in children with beta-thalassemia major].

The objective of this study was to investigate the curative effect of combined sibling umbilical cord blood and bone marrow transplantation in treatment of beta-thalassemia major. Combined umbilical cord blood and bone marrow transplantation from an HLA-identical sibling were performed for 3 patients with beta-thalassemia major. The nucleated cells infused into 3 recipients were 19.

[Efficacy of low-dose heparin and prostaglandin E1 in the prevention of hepatic veno-occlusive disease after allogenic hematopoietic stem cell transplantation in children with beta-thalassemia major].

Objective: Hepatic veno-occlusive disease (HVOD) is one of the most serious complications after allogenic hematopoietic stem cell transplantation (allo-SCT). Endothelial injury, leading to deposition of coagulation factors in the terminal hepatic venules, is believed to the key event in the pathogenesis of HVOD. This study was designed to explore the efficacy of low-dose heparin and prostaglandin E1 (PGE1) in the prevention of HVOD after allo-SCT in children with beta-thalassemia major.

[Umbilical cord blood transplantation for patients with beta-thalassemia major].

Objective: The beta-thalassemia major is a common hereditary hematology disease in southern China. The combination of blood transfusion and iron chelation is now the reference treatment. The allogeneic hematopoietic stem cell transplantation is the only curative therapy for beta-thalassemia major.

[Two HLA-loci mismatched sibling cord blood transplantation in a severe beta-thalassemia patient].

Allogeneic hematopoietic stem cell transplantation is the only curative therapy for severe beta-thalassemia. This time, the experience of utilizing HLA 2-loci mismatched sibling cord blood transplantation (CBT) in a child with severe beta-thalassemia was firstly reported in our country. A 3-year-male patient had been diagnosed with severe beta-thalassemia at 6 months of age (HbF 86.