Management of transfusion-dependent β-thalassemia (TDT): Expert insights and practical overview from the Middle East

El-Beshlawy, Amal; Dewedar, Hany; Hindawi, Salwa; Alkindi, Salam; Tantawy, Azza A.; Yassin, Mohamed A.; Taher, Ali T.

doi:10.1016/j.blre.2023.101138

Blood Reviews

2023

DOI: 10.1016/j.blre.2023.101138

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Management of transfusion-dependent β-thalassemia (TDT): Expert insights and practical overview from the Middle East

Amal El-Beshlawy,

Hany Dewedar,

Salwa Hindawi

et al.

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2024

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Cited by 4 publications

References 112 publications

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Iron chelation therapy for children with transfusion‐dependent β‐thalassemia: How young is too young?

Forni,

Kattamis,

Kuo

et al. 2024

Pediatric Blood & Cancer

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In this review, we provide a summary of evidence on iron overload in young children with transfusion‐dependent β‐thalassemia (TDT) and explore the ideal timing for intervention. Key data from clinical trials and observational studies of the three available iron chelators deferoxamine, deferiprone, and deferasirox are also evaluated for inclusion of subsets of young children, especially those less than 6 years of age. Evidence on the efficacy and safety of iron chelation therapy for children ≥2 years of age with transfusional iron overload is widely available. New data exploring the risks and benefits of early‐start iron chelation in younger patients with minimal iron overload are also emerging.

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Iron chelation therapy for children with transfusion‐dependent β‐thalassemia: How young is too young?

Forni,

Kattamis,

Kuo

et al. 2024

Pediatric Blood & Cancer

Self Cite

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Constructing a novel clinical indicator model to predict the occurrence of thalassemia in pregnancy through machine learning algorithm

Long,

Bai

2024

Front. Hematol.

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Thalassemia is one of the inherited hemoglobin disorders worldwide, resulting in ineffective erythropoiesis, chronic hemolytic anemia, compensatory hemopoietic expansion, hypercoagulability, etc., and when a mother carries the thalassemia gene, the child is more likely to have severe thalassemia. Furthermore, the economic and time costs of genetic testing for thalassemia prevent many thalassemia patients from being diagnosed in time. To solve this problem, we performed least absolute shrinkage and selection operator (LASSO) regression to analyze the correlation between thalassemia and blood routine indicators containing mean corpuscular volume (MCV), mean corpuscular hemoglobin (MCH), mean corpuscular hemoglobin concentration (MCHC), and red blood cell (RBC). We then built a nomogram to predict the occurrence of thalassemia, and receiver operating characteristic (ROC) curve was used to verify the prediction efficiency of this model. In total, we obtained 7,621 cases, including 847 thalassemia patients and 6,774 non-thalassemia. Among the 847 thalassemia patients, with a positivity rate of 67.2%, 569 cases were positive for α-thalassemia, and with a rate of 31.5%, 267 cases were positive for β-thalassemia. The remaining 11 cases were positive for both α- and β-thalassemia. Based on machine learning algorithm, we screened four optimal indicators, namely, MCV, MCH, RBC, and MCHC. The AUC value of MCV, MCH, RBC, and MCHC were 0.907, 0.906, 0.796, and 0.795, respectively. Moreover, the AUC value of the prediction model was 0.911. In summary, a novel and effective machine learning model was built to predict thalassemia, which functioned accurately, and may provide new insights for the early screening of thalassemia in the future.

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Renal Findings in Patients with Thalassemia at Abdominal Ultrasound: Should We Still Talk about “Incidentalomas”? Results of a Long-Term Follow-Up

Fatigati,

Meloni,

Costantini

et al. 2024

Diagnostics

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We retrospectively collected all ultrasound imaging data of our thalassemia patients over a period of 10 years with the aim of assessing the prevalence and the risk factors of renal stones and cysts. Moreover, we assessed the incidence of renal-cell carcinoma (RCC) among thalassemia patients (133 with thalassemia major (TM) and 157 with thalassemia intermedia (TI)) and its association with demographic and clinical findings. Renal stones were detected in 15.2% of patients. In the multivariable Cox regression analysis, the independent predictors were blood consumption, splenectomy, and proteinuria. Renal cysts were detected in 18.4% of patients. In the multivariable analysis, age emerged as the only independent predictor. After the first detection, 35% of the patients showed changes in the number, size, or grading of renal cysts. During the study period, the crude incidence rate of RCC was 75.9 cases per 100,000 person-years. The most frequent histological subtype (80%) included clear-cell RCC. In total, 80% of patients with RCC had TM and all were positive for hepatitis C virus antibodies. Thalassemia patients are significantly affected by asymptomatic renal diseases such as stones, cysts, and cancer, suggesting the need for regular screening by imaging.

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Management of transfusion-dependent β-thalassemia (TDT): Expert insights and practical overview from the Middle East

Cited by 4 publications

References 112 publications

Iron chelation therapy for children with transfusion‐dependent β‐thalassemia: How young is too young?

Iron chelation therapy for children with transfusion‐dependent β‐thalassemia: How young is too young?

Constructing a novel clinical indicator model to predict the occurrence of thalassemia in pregnancy through machine learning algorithm

Renal Findings in Patients with Thalassemia at Abdominal Ultrasound: Should We Still Talk about “Incidentalomas”? Results of a Long-Term Follow-Up

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