Clinical characteristics of proximal tubular acidosis induced by deferasirox in children with β-thalassemia major
- VernacularTitle:重型β地中海贫血患儿使用地拉罗司致近端肾小管酸中毒的临床特征
- Author:
Juanjuan CHEN
1
;
Chaoyang LYU
;
Manqing DENG
Author Information
- Publication Type:Research Article
- Keywords: deferasirox; β-thalassemia major; proximal tubular acidosis; β2-microglobulin; renal tubular function; iron deposition
- From: Journal of Clinical Medicine in Practice 2024;28(24):124-128
- CountryChina
- Language:Chinese
- Abstract: Objective To analyze the clinical characteristics of proximal tubular acidosis induced by deferasirox in children with β-thalassemia major. Methods This study retrospectively analyzed the clinical manifestations, laboratory test results, radiological features, treatment methods and outcomes of proximal tubular acidosis in 5 children with β-thalassemia major. Results Five patients aged 5 to 16 years, included 2 males and 3 females. Two patients were diagnosed with Fanconi syndrome. Four patients received deferasirox doses exceeding the conventional dose, including one with a rapid decline in ferritin levels. Gastrointestinal symptoms were the initial manifestations in 3 patients, while only mild anorexia was observed in 2 patients. There were significant correlations between the severity of clinical manifestations, the degree of acidosis and its duration. All patients had abnormally elevated urine β2-microglobulin levels, and 4 patients had hepatic iron deposition. After reducing or discontinuing deferasirox and providing symptomatic treatment, the renal tubular function of all 5 patients gradually recovered. Conclusion Proximal tubular acidosis induced by deferasirox in patients with β-thalassemia major can manifest as partial or complete proximal tubular acidosis, which may be associated with high-dose deferasirox administration and rapid decreases in body iron levels. Regular monitoring of relevant indicators is recommended for children receiving long-term deferasirox treatment. Once proximal tubular acidosis is detected, drug dosage adjustment or discontinuation should be promptly implemented, and renal tubular function can recover in most patients.
