Luspatercept enhances hemoglobin levels in a Chinese boy with congenital sideroblastic anemia: A case report.

Background: Congenital sideroblastic anemia (CSA) is a rare and heterogeneous group of genetic disorders. Conventional treatment include pyridoxine (vitamin B6) and allogeneic hematopoietic stem cell transplantation (allo-HSCT), and can alleviate anemia in the majority of cases. Nevertheless, some CSA cases remain unresponsive to pyridoxine or are unable to undergo allo-HSCT.

[Relationship between Changes of Lymphocyte Subsets and Early Hematologic Response in Immunosuppressive Therapy of Severe Aplastic Anemia Patients].

Objective: To explore the relationship between the change of lymphocyte subsets before and after immunosuppressive therapy (IST) with disease severity of severe aplastic anemia (SAA) and hematologic response to IST.

Methods: The clinical data of 94 patients with SAA/VSAA treated by r-ATG and CsA in our hospital from December 2009 to October 2011 was analyzed retrospectively. Among them, 26 patients who had sequential data of lymphocyte subsets and cytokines before and after treatment were enrolled.

Next generation sequencing reveals co-existence of hereditary spherocytosis and Dubin-Johnson syndrome in a Chinese gril: A case report.

Background: Hereditary spherocytosis (HS) is a hereditary disease of hemolytic anemia that occurs due to the erythrocyte membrane defects. Dubin-Johnson syndrome (DJS), which commonly results in jaundice, is a benign hereditary disorder of bilirubin clearance that occurs only rarely. The co-occurrence of HS and DJS is extremely rare.

[Predicting early response to immunosuppressive therapy in severe aplastic anemia by soluble transferrin receptor assay].

Objective: To evaluate the value of serum soluble transferrin receptor (sTfR) concentration in predicting early response to immunosuppressive therapy (IST) in severe aplastic anemia (SAA).

Methods: Clinical data and hematologic responses of 140 SAA patients treated with rabbit antithymocyte globulin (rATG) combination with cyclosporine in our hospital were retrospectively analyzed. Correlation of pre-IST baseline of sTfR and IST responses was statistically analyzed and receiver operating characteristic (ROC) curve was used to estimate the sensitivity and specificity of sTfR in prediction of early responses.

[Pure red cell aplasia with thymoma/T-cell large granular lymphocyte leukemia: two cases report and literatures review].

Objective: To investigate the clinical and laboratory features of 2 cases of pure red cell aplasia (PRCA) with thymoma/T-cell large granular lymphocyte leukemia (T-LGLL), and to improve the recognition of the disease and the role of lymphocyte in its mechanism.

Methods: Two cases of PRCA with thymoma/T-LGLL were reported and the related literatures were reviewed.

Results: Case 1 was a 63-years old male with hemoglobin level of 54 g/L at admission.

[High-dose cyclophosphamide for severe aplastic anemia associated with β-thalassemia: a case report and literatures review].

Objective: To investigate the clinical features and therapeutic method for severe aplastic anemia (SAA) associated with β-thalassemia, and to improve the recognition of the disease.

Methods: One patient hospitalized for pancytopenia was reported and the related literatures were reviewed.

Results: A 14-years old girl who presented with anemia from her childhood was hospitalized for acute onset of pancytopenia.

[Effects of concentration of cyclosporine A on the early response to immunosuppressive therapy in severe aplastic anemia].

Objective: To evaluate the effects of cyclosporine A (CsA) whole-blood concentration on the early response to immunosuppressive therapy (IST) in severe and very severe aplastic anemia (SAA/VSAA).

Methods: Ninety SAA/VSAA patients treated with rabbit antithymocyte globulin (ATG) plus CsA as first line therapy in our hospital were retrospectively analysed. CsA levels between the response group and non-response group, and response rates of patients with variant CsA levels were compared respectively.