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Pediatric and Adolescent Outpatient Clinic, Quisisana Hospital, Ferrara, Italy.
Acta Biomed. 2022 Aug 31;93(4):e2022291. doi: 10.23750/abm.v93i4.13330.
The conventional treatment of β-thalassemia (β-TM) patients is based on the correction of anemia through regular blood transfusions and iron chelation therapy. However, allogeneic hematopoietic stem cell transplantation (HSCT) remains the only currently available technique that has curative potential. Variable frequency and severity of long-term growth and endocrine changes after conventional treatment as well as after HSCT have been reported by different centers. The goal of this mini-review is to summarize and update knowledge about long-term growth and endocrine changes after HSCT in patients with β-TM in comparison to those occurring in β-TM patients on conventional treatment. Regular surveillance, early diagnosis, treatment, and follow-up in a multi-disciplinary specialized setting are suggested to optimize the patient's quality of life (www.actabiomedica.it).
β-地中海贫血(β-TM)患者的传统治疗方法基于通过定期输血和铁螯合疗法纠正贫血。然而,异基因造血干细胞移植(HSCT)仍然是唯一具有潜在治愈能力的现有技术。不同中心报道了常规治疗后以及 HSCT 后长期生长和内分泌变化的频率和严重程度不同。本综述的目的是总结和更新关于β-TM 患者 HSCT 后长期生长和内分泌变化的知识,并与接受常规治疗的β-TM 患者进行比较。建议在多学科专业环境中进行定期监测、早期诊断、治疗和随访,以优化患者的生活质量(www.actabiomedica.it)。