Pure red cell aplasia in children: a clinical analysis of 16 cases / 中国当代儿科杂志

OBJECTIVE@#To study the clinical features, treatment, and prognosis of pure red cell aplasia (PRCA) in children.@*METHODS@#A retrospective analysis was performed for the clinical data of 16 children with PRCA. The outcome and prognosis of patients treated with prednisone combined with Huaiqihuang granules versus prednisone alone were evaluated.@*RESULTS@#All the 16 children complained of symptoms of anemia including pale or sallow complexion. Of 12 children undergoing pathogen test, 7 (58%) were found to have pathogen infection, among which human cytomegalovirus was the most common. Lymphocyte subsets were measured for 7 children, among whom 5 (71%) had lymphocyte immune disorder. Six children were found to have abnormalities in immunoglobulin and complement. The 8 children treated with prednisone combined with Huaiqihuang granules had a median follow-up time of 21.5 months, among whom 1 was almost cured, 1 was relieved, and 6 were obviously improved; the median onset time of treatment was 1 month, and 2 children had disease recurrence in the course of drug reduction or withdrawal. The 8 children in the prednisone alone treatment group had a median follow-up time of 34 months, among whom 4 were almost cured, and 4 were obviously improved; the median onset time of treatment was 2.5 months, and 4 children had recurrence during drug reduction or withdrawal.@*CONCLUSIONS@#Children with PRCA usually complain of anemia-related symptoms. Laboratory tests show pathogen infection in some children with PRCA, and most of children have immune disorders. Glucocorticoids have a good therapeutic effect, but some children relapse in the course of drug reduction or withdrawal. Combined treatment with prednisone and Huaiqihuang granules may have a faster onset of action and less possibility of recurrence.

Clinical and biological characteristics of childhood acute myeloid leukemia with EVI1 gene positive expression / 中国当代儿科杂志

<p><b>OBJECTIVE</b>To study the expression of ecotropic viral integration site (EVI1) gene in childhood acute myeloid leukemia (AML) and the clinical features of EVI1-positive children with AML.</p><p><b>METHODS</b>The clinical data of EVI1-positive children with AML were collected and analyzed. RT-PCR and real-time quantitative PCR were used for qualitative and quantitative analysis of expression of EVI1. Flow cytometry (FCM) was used for determining the immunophenotypes of bone marrow cells. Multiparameter FCM was used for monitoring minimal residual disease. The karyotypes were determined.</p><p><b>RESULTS</b>Of 241 children with AML, 33 (13.7%) were positive for EVI1 expression. There were no significant differences in age at first visit as well as the white blood cell count, hemoglobin level, and platelet count in peripheral blood between EVI1-positive and EVI1-negative children with AML (P>0.05), but EVI1-positive children had a significantly increased proportion of females compared with EVI1-negative children (P<0.05). The change in EVI1 expression was not synchronous with clinical remission and the change of MRD: some children had clinical remission or negative conversion of MRD before negative conversion of EVI1, while some had negative conversion of EVI1 before clinical remission or while MRD showed positive. EVI1 gene was usually co-expressed with other fusion genes. CD33 (100%), CD38 (88%), and HLADR (76%) were highly expressed in EVI1-positive children with AML. Abnormal chromosome structure or number was found in 15 patients. Compared with EVI1-negative children, EVI1-positive children had significantly lower complete remission rates after the first course of treatment (P<0.05).</p><p><b>CONCLUSIONS</b>EVI1-positive children with AML have a poor short-term prognosis. In the development of AML, the activation of EVI1 gene is not isolated, but the result of interactions with other genes or chromosome abnormalities, and the mechanism of activation and its function need further study.</p>

Clinical analysis of pediatric acute myeloid leukemia with EVI1 gene positive / 中华实用儿科临床杂志

Objective To analyze the clinical features and prognosis of pediatric acute myeloid leukemia (AML) with EVI1 gene positive.Methods The nested RT-PCR was performed to detect the EVI1 expression in pediatric AML patients from Jan.2009 to Dec.2011.The patients with EVI1 were investigated on clinical features,curative effects and prognosis.The differences between EVI1 (+) and EVI1 (-) patients were also analyzed.Results The frequency of EVI1 (+) expression was 15.65％ (13/83 cases)in pediatric AML,with the highest incidence in high-risk patients.EVI1 (+) was obviously associated with some unfavorable molecular genetic changes such as complex karyotype,MLL rearrangement,and monosomy 7.There were significant differences for EVI1 (+) group and EVI1 (-) group in the complete remission rate (45.5％ vs 79.3％,x2 =5.497,P ＜ 0.05) and complete remission(CR) rate after the first chemotherapy (18.2％ vs 63.8％,x2 =7.828,P ＜0.01).Although significant difference in death rate was not observed,EVI1(+) group had significantly higher early-death rate (45.5％ vs 8.6％,P ＜0.01).The EVI1(+) group also had lower 4 years event-free survival (EFS) [(21.2 ± 13.8) ％ vs (50.2 ± 9.1) ％,x2 =4.493,P ＜ 0.05] and lower 4 years overall survival(OS) [(32.4 ± 7.1) ％ vs (60.3 ± 10.9) ％,x2 =4.602,P ＜ 0.05] compared with EVI1 (-) group.But binary Logistic analysis did not identify EVI1 (+) as an independent unfavorable prognostic factor.Conclusions The pediatric AML with positive EVI1 expression had lower CR rate,higher early death rate and lower EFS.Positive EVI1 expression is related with an adverse outcome,but is not an independent poor prognostic factor.

Comparisons of clinical features of chronic aplastic anemia and myelodysplastic syndrome in children / 中国当代儿科杂志

<p><b>OBJECTIVE</b>This study compared the differences in clinical features between chronic aplastic anemia (CAA) and myelodysplastic syndrome (MDS) in children in order to provide a basis for the differential diagnosis of the two diseases.</p><p><b>METHODS</b>A retrospective study of 23 cases of CAA and 9 cases of MDS from September 2007 to September 2010 was performed. The clinical data including routine blood test results, reticulocyte counts, serum lactate dehydrogenase level, serum ferritin level, cytological examination of bone marrow, bone marrow CD34+ cell counts, bone marrow chromosome and FISH test results were compared between the CAA and MDS groups.</p><p><b>RESULTS</b>Neutrophils, reticulocytes, and serum ferritin and lactate dehydrogenase levels increased in the MDS group compared with those in the CAA group. There were significant differences in bone marrow blast cell counts and dyshematopoiesis phenomena of three lines blood cells between the CAA and MDS groups. The bone marrow CD34+ cell counts and the rate of chromosomal abnormalities detected in bone marrow cytogenetic analysis in the MDS group were significantly higher than those in the CAA group.</p><p><b>CONCLUSIONS</b>There are differences in the results of laboratory examinations and morphological and cytogenetic examinations of bone marrow between the children with CAA and MDS. The differences are useful to the differential diagnosis of the two diseases.</p>

Therapeutic effects of a combination of high-dose immunoglobulin and cyclosporine A in children with aplastic anemia / 中国当代儿科杂志

<p><b>OBJECTIVE</b>To evaluate the therapeutic effects of a combined immunotherapy, high-dose immunoglobulin (HDIG) plus cyclosporine A (CsA) plus prednisone (P), in children with aplastic anemia (AA) and to explore the association of peripheral blood lymphocyte subsets, peripheral blood cells and marrow CD34+ cells with therapeutic effects in AA.</p><p><b>METHODS</b>The clinical data of 46 children with AA and who received the combined immunotherapy of HDIG + CsA + P were retrospectively studied.</p><p><b>RESULTS</b>Of the 46 children with AA, 31 (67.4%) were responded to the combined immunotherapy. The binary logistic regression analysis showed low absolute neutrophil count (B=4.703, p<0.05), low percentage of peripheral blood CD4+ cells (B=0.142, p<0.05) and low ratio of peripheral blood CD4+/CD8+ (B=2.945, p<0.05)were associated with poor therapeutic effects. The ratio of CD34+/karyocytes of bone marrow in children with AA was lower than that in normal individuals, but it was not significantly related to the therapeutic effect.</p><p><b>CONCLUSIONS</b>The combined immunotherapy (HDIG+CsA+P) was effective in children with AA. The absolute neutrophilcount, the percentage of peripheral blood CD4+ and the ratio of peripheral blood CD4+/CD8+ were important prognostic factors in AA.</p>

Effects of ouabain at low concentrations on growth of leukemia cells / 中国实验血液学杂志

This study was aimed to investigate the effects of ouabain at low concentrations on growth regulation in various leukemia cell lines and to determine the therapeutic potential of ouabain in leukemia. By using MTT, flow cytometry (FCM), the changes in cell growth and cell cycle of leukemia cell lines were observed after treating with ouabain at low concentrations (<or=10 nmol/L). The expression of sodium pump alpha1 subunit was evaluated by Western blot. The results showed that in megakaryocytic leukemia M07e and Meg-01 cell lines, the low concentrations of ouabain (<or=10 nmol/L) inhibited the cell growth, blocked the cell cycle at G1 phase, and decreased the protein expression of sodium pump alpha1 subunit. The same concentrations of ouabain induced the proliferation of lymphocytic leukemia B95 and Jhhan cell lines, increased the percentage of cells at S phase and G2/M phase and up-regulated the expression of sodium pump alpha1 subunit. The basic expression of the sodium pump alpha1 subunit in M07e and Meg-01 was lower than that in B95 and Jhhan. It is concluded that the low concentrations of ouabain inhibit the cell growth of the megakaryocytic leukemia cell lines M07e and Meg-0, which may be related with the low expression of sodium pump alpha1 subunit of that cell lines and the negative regulation of the protein induced by ouabain.