Mediterranean Journal of Hematology and Infectious Diseases最新文献

Background: Iron deficiency anemia (IDA) is a major public health problem among children worldwide. Iron deficiency without anemia (IDWA) is at least twice as common as IDA. Some studies propose that oral iron fortification can modify the infant's gut microbiome, leading to intestinal inflammation.

Objectives: To determine whether oral iron therapy can lead to intestinal inflammation in children with IDA or IDWA.

Patients and methods: Fifty-six patients aged 6 months to 16 years (median age 7.6 years) with IDA or IDWA were randomly assigned to receive either iron (III)-hydroxide polymaltose complex (IPC) 5 mg/kg once daily (maximum dose 100 mg) or sucrosomial iron (SI)1.4 mg/kg once daily (maximum dose 29.4 mg). Safety and efficacy were studied after 30 and 90 days of treatment. In addition, fecal calprotectin as a marker of intestinal inflammation was measured simultaneously and compared to results obtained before therapy.

Results: A significant increase in serum ferritin was noted in both groups as the median ferritin level at baseline was 6.7 μg/L in the IPC group and 6.6 μg/L in the SI group, increasing to 15.9 μg/L and 12.1 μg/L respectively, after 90 days of treatment. However, there was no significant change in fecal calprotectin in either group. In addition, no differences in the trend over time were observed between the two groups regarding fecal calprotectin, serum ferritin, and hemoglobin.

Conclusions: IPC and SI were equally effective in treating IDA and IDWA. At the recommended doses, oral iron therapy does not seem to induce intestinal inflammation.

Multiple myeloma (MM), characterized by abnormal proliferation of clonal plasma cells, is an incurable hematological malignancy. Various immunotherapy strategies have emerged as an efficacious approach for the treatment of MM, including monoclonal antibodies, antibody-drug conjugates, bispecific antibodies, and chimeric antigen receptor T (CAR-T) cells. Anti-B-cell maturation antigen (BCMA) CAR-T cells have revolutionized the treatment of MM patients with relapsed/refractory disease and their clinical use was approved for the treatment of these patients. Despite this progress, the efficacy of CAR-T cells in MM is limited by the responsiveness of only a part of the treated patients, the relapse of other patients, the cost of the treatment and the diminished response in patients with prior exposure to anti-BCMA targeting agents. Ongoing clinical trials are evaluating the use of CAR-T cells at an earlier stage of MM disease and the use of CAR-T cells targeting other membrane antigens expressed on malignant plasma cells.

Pediatric Hodgkin lymphoma (HL) is a curable disease for more than 90% of children and adolescents in high-income countries. However, similar results cannot be achieved, particularly for advanced disease, in low- and middle-income countries (LMICs), where challenging socio-economic realities and the consequent scarcity of local resources heavily impact the treatment and patients' outcome. Information regarding the management and outcome of pediatric HL in LMICs is still limited. In this narrative review, we summarize the results reported in the literature so far and discuss the critical key points that have emerged from this overview.

Background: Antiphospholipid Syndrome (APS) is one of the most common acquired causes of hypercoagulability. The 2023 American College of Rheumatology (ACR) / European Alliance of Associations for Rheumatology (EULAR) APS Classification Criteria were specified as new APS classification criteria with high specificity for use in observational studies and research. The primary objective of this study was to evaluate the performance of the 2023 ACR/EULAR APS classification criteria in a real-world rheumatology department.

Methods: This is a retrospective, single-center study evaluating the sensitivity and specificity of the 2006 revised Sapporo and 2023 ACR/EULAR APS classification criteria in patients diagnosed with APS through clinical evaluation. A total of 184 patients, 103 of whom were diagnosed with APS, were included in the study.

Results: The 2023 ACR/EULAR APS classification criteria demonstrate higher specificity 98.8% (95% CI 93.3-99.8) and positive predictive value (PPV) 98.7% (95% CI 93.2-99.8). The revised Sapporo criteria exhibit higher sensitivity 90.3% (95% CI 83-96.6), negative predictive value (NPV) 88.1% (95% CI 79.4-93.4), and accuracy 90.8% (95% CI 85.7-94.1). When the diagnosis of APS was accepted according to the revised Sapporo criteria, the sensitivity of the 2023 ACR/EULAR APS classification criteria was 77% (95% CI 67.8-84.2), specificity 97.6% (95% CI 91.7-99.3), PPV 97.5% (95% CI 69.3-84.9) and NPV 78.1% (95% CI 69.3-84.9).

Conclusion: The 2023 ACR/EULAR APS classification criteria have low sensitivity and high specificity compared to the revised Sapporo APS classification criteria. The increase in specificity is due to risk assessment in thromboses and strict obstetric and laboratory criteria.

In the last ten years, a consistent number of clinical studies have evaluated different gene approaches for the treatment of patients with sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Initial studies of gene therapy for hemoglobinopathies involved the use of lentiviral vectors to add functional copies of the gene encoding β-globin in defective CD34 cells; more recently, gene editing techniques have been used involving either CRISPR-Cas9, transcription activation-like effector protein nuclease, zinc finger nuclease, and base editing to either induce fetal hemoglobin production at therapeutic levels or to genetically repair the underlying molecular defect causing the disease. Here, we review recent gene editing studies that have started the development of a new era in the treatment of hemoglobinopathies and, in general, monoallelic hereditary diseases.