Mediterranean Journal of Hematology and Infectious Diseases最新文献

Background: Febrile neutropenia (FN) remains a frequent and potentially life-threatening complication in pediatric oncology, where prompt recognition of bacteremia is critical for risk-adapted therapy and antimicrobial stewardship. Traditional biomarkers such as C-reactive protein (CRP) and procalcitonin (PCT) are widely used, yet their early predictive value is inconsistent across studies. Cellular activation markers measured by flow cytometry, particularly CD48, have been scarcely investigated in this setting. This study aimed to evaluate conventional, metabolic, and immune biomarkers for predicting bacteremia in children with FN and to assess the incremental diagnostic value of CD48.

Methods: This prospective single-center cohort enrolled 38 pediatric oncology patients presenting with 46 FN episodes over 9 months. Clinical data, blood cultures, and serial measurements of CRP, PCT, lactate, interleukin-6, interleukin-8, MCP-1, sTREM-1, CD48, and CD64 were obtained at 0, 24, 48, and 72 hours. Bacteremia was defined by positive culture for a recognized pathogen. Receiver operating characteristic (ROC) analyses were performed to determine the area under the curve (AUC), sensitivity, and specificity. A multivariable logistic regression model evaluated the combined performance of biomarkers.

Results: Bacteremia occurred in 12 (26.1%) FN episodes. Sepsis, tachycardia, and elevated lactate were more common among bacteremic patients. CRP showed limited early discrimination (AUC 0.62 on day 2) but improved by day 4 (AUC 0.74). PCT was consistently higher in bacteremia (AUC 0.89 at day 4), and lactate demonstrated strong early predictive value (AUC 0.81). CD48 was significantly elevated from 0-24 h (AUC 0.78), outperforming CD64 (AUC 0.60) and preceding the rise in CRP. In combined modeling, PCT + CD48 + lactate achieved the highest discrimination (AUC 0.92; sensitivity 92%, specificity 85%). Post-hoc power analysis showed 82% power to detect AUC differences ≥0.15.

Conclusion: Integration of CD4 with PCT and Lactate markedly improved diagnostic accuracy in this cohort; however, given the limited number of bacteremic episodes, these findings should be considered exploratory and require external validation in larger, multicenter studies before clinical implementation.

Background: Thalassemia is a common hereditary hemoglobin disorder in Vietnam. Elucidating the epidemiological and genetic patterns in children is essential for developing screening and prevention strategies.

Methods: A retrospective analysis of 1,240 children under 10 years of age with Thalassemia treated at the National Institute of Hematology and Blood Transfusion in Vietnam, between 2014 and 2023.

Results: The median age at treatment initiation was 1 year (range 0-9 years), with 94.5% of patients aged 0-5 years. Children born after 2020 were diagnosed and treated earlier than those born before 2020 (0 year (range 0-2 years) vs 1 year (range 0-9 years); p < 0.0001), concurrent with the implementation of the national prenatal screening program. β-thalassemia and β-thalassemia/HbE accounted for nearly 90% of cases, with subtype distribution varying by ethnicity and region. β-thalassemia/HbE predominated in the Northwest and North Central regions, particularly among the Thai and Muong populations. In contrast, β-thalassemia was more prevalent in the Northeast, notably among the Tay and Nung populations. Eight α-globin and thirteen β-globin mutated types were detected. The common β-globin variants (CD17, CD41/42, CD71/72, -28, and IVSI-1) and HbE (CD26) mirror patterns reported in neighboring Laos and Guangxi Province, China. For α-globin genotypes, --^SEA (49.83%), Hb CS (31.53%), and -α^3.7 (8.47%) were most frequent.

Conclusion: Geography, ethnicity, and genetic background strongly shape Thalassemia epidemiology in Northern Vietnam. Targeted genetic counseling, early carrier screening, and region-focused community programs are urgently needed to reduce disease burden in high-risk populations.

Sickle cell disease (SCD) and transfusion-dependent β-thalassemia are no longer pediatric death sentences. With newborn screening, transfusions, and chelation therapy, patients now survive into their 4th-6th decade. Yet as they age, they face mounting complications -pain that never truly resolves, organs failing one by one, and profound isolation. Ironically, palliative care remains scarce despite the clinical complexity. This narrative review examines end-of-life care in these hemoglobinopathies, focusing on pain management, ethical tensions, and the psychosocial needs that intensify as death approaches. We reviewed literature from 2020 to 2025, international guidelines, and European frameworks. The evidence is clear: terminal SCD involves unpredictable crises and intractable pain; β-thalassemia brings slow cardiac decline and iron-laden organ failure. Both demand early palliative integration, yet both are drastically undertreated. Cultural beliefs heavily shape how families accept or reject end-of-life discussions. Disparities in opioid access, lack of disease-specific referral criteria, and absence of flexible hospice models create barriers that disproportionately harm marginalized patients. We conclude that hemoglobinopathy patients deserve the same anticipatory, culturally informed, multidisciplinary palliative care that we increasingly offer to cancer patients. Health systems must establish referral pathways specific to these diseases, permit palliative transfusions in hospice when appropriate, ensure equitable opioid access, and embed psychosocial support in hemoglobinopathy centers.