Physical Activity Limitations In Children With Severe Haemophilia A. Does Emicizumab Make A Difference?

Objectives: To assess the effect of emicizumab on physical activity in children with severe haemophilia A. METHODS: The prospective cohortstudy was conducted from October 2021 to April 2022 at the Paediatric Department of Kafrelsheikh University Hospital, Egypt, in collaboration with the Haematology out-patient clinic of the Paediatric Department, Zagazig University, Egypt, and the Paediatric Department of Cairo University Hospital, Egypt, and comprised children aged 4-18 years with severe haemophilia A who received emicizumab prophylaxis. Paediatric Haemophilia Activities List was used to assess physical activity at baseline and aftersix months of regular emicizumab prophylaxis. Data was analysed using SPSS 26. RESULTS: There were 29 children, all (100%) boys, with mean age 8.7±3.51 years(range 4-15 years. Of them, 17(58.62%) patients were negative for inhibitors. Median Paediatric Haemophilia Activities Listsum score was 59.54 (interquartile range: 50.15-62.05) at baseline which moved up to 84 (interquartile range: 79.05-86.35) post-intervention (p<0.001). CONCLUSIONS: Emicizumab prophylaxis improved the level of physical activity in children with severe haemophilia A.

Assessment of plasmablast cells in immune thrombocytopenic purpura in children.

Primary immune thrombocytopenic purpura (ITP) is an autoimmune disorder with platelet destruction due to B- and T-cell dysregulation and antiplatelet autoantibodies production. Flow cytometry can be used to further characterize the B- and T-cell compartments involved in platelet destruction. This case-control study was to enumerate plasmablast cells in pediatric ITP patients and to correlate their levels with disease course. This study included 30 ITP patients and 10 controls. Identification and enumeration of Plasmablast were done by multicolor flow cytometry using specific antibody panels (CD19, CD27 & CD38) and sequential gating using FACSCanto flow cytometer and FlowJo software. We found that lymphocytes subpopulation in ITP patients and controls revealed increase in frequency of CD19 (B lymphocytes) in acute, persistent, and chronic ITP patients in comparison with controls (p < 0.001, 0.023, 0.001) respectively. Plasmablast cells could play a role in the pathogenesis of ITP and might guide therapy in ITP patients in the future.

Effect of iron deficiency anemia and its treatment on cell mediated immunity.

Iron deficiency anemia (IDA) is one of the most prevalent micronutrient deficiencies particularly in the developing countries. While there is evidence of an altered immune profile in iron deficiency, the exact immunoregulatory role of iron is not known. Knowledge particularly in children, who are vulnerable to iron deficiency and infection, is lacking. We aimed to study the effects of IDA and its treatment with oral iron supplementation on cell-mediated immunity. The levels of T-lymphocytes, their CD4(+), CD8(+) and CD1a(+) subsets, transferrin receptor (CD71) and serum ferritin were evaluated in 40 iron-deficient and 40 healthy children. The impact of oral iron supplementation for three months on the same parameters was also noted in children with IDA. The level of mature T-lymphocytes (CD4(+) and CD8(+)) was significantly lower (P<0.001) while that of the immature T-cells (CD1a(+)) was significantly higher (p<0.001) in IDA children compared to the control. The mature T-cell count was significantly improved after iron therapy. In spite of significant reduction in the immature T-cells (CD1a(+)) level after iron supplementation, it was significantly higher than the control. The present study demonstrated that T-lymphocytes maturation was defective in IDA and improved partially after 3 months of iron supplementation. Therefore, longer time of iron therapy may be required to induce complete maturation of T-lymphocytes.