The effect of deferasirox on endocrine complications in children with thalassemia

Bilgin, Burçak; Yozgat, Ayça Koca; Işık, Pamir; Çulha, Vildan; Kaçar, Dilek; Kara, Abdurrahman; Özbek, Namık; Yaralı, Neşe

doi:10.1080/08880018.2020.1734124

Cited by 12 publications

(10 citation statements)

References 22 publications

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“…In the present study, we found that oral deferasirox is not superior to deferoxamine when used as monotherapy. These data not agree with other reports [27,28] showing that long-term oral deferasirox therapy may decrease the prevalence of endocrinopathies in TM. The main explanation for this may be the short duration of therapy in our study and the different age groups.…”

Section: Discussioncontrasting

confidence: 81%

Detection of endocrine disorders in young children with multi-transfused thalassemia major

2021

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Background Beta thalassemia major (TM) is the most common inherited genetic disorder worldwide. Patients are at risk of iron overload, which leads to various forms of tissue damage, including endocrinopathies. The aim of this study was to evaluate the prevalence and risk factors of endocrine disorders in young patients with multi-transfused TM receiving iron chelation therapy. Methods The inclusion criteria included all known cases of TM according to hemoglobin electrophoresis data, aged 12 years or younger, during the study period. The patient’s age, gender, parent’s consanguinity, clinical examination, and types of iron chelating agents used were recorded. Serum ferritin level, complete blood count (CBC), blood glucose homeostasis, thyroid, and parathyroid functions were determined. Results One hundred twenty patients met the inclusion criteria; 70% of them had malnutrition. The presence of endocrine disorders was observed in 28/120 (23.33%) patients. The most common endocrine disorders were thyroid disorders, either subclinical or clinical hypothyroidism in 11/120 (9.17%) patients, followed by abnormalities in glucose homeostasis 9/120 (7.5%). The prevalence of impaired glucose tolerance, impaired fasting glucose, and diabetes mellitus in the present study was 5 (4.17%), 4 (3.33%), and 0 (00%), respectively, while the least frequent endocrine disorder seen in our patients was hypoparathyroidism in 8/120 (6.66%). We noted that high serum ferritin levels and poor patient compliance to therapy were significantly associated with increased endocrine disorders (OR 0.98, 95% CI 0.96–0.99, P = 0.003 and OR 0.38, 95% CI 0.16:0.93, P = 0.03, respectively). Combined chelating iron agents significantly decreased the prevalence of endocrine disorders when compared with monotherapy (OR 0.40, 95% CI 0.16:0.97, P = 0.04). Conclusion Endocrine disorders could occur in TM patients early before or equal to 12 years of life in about one-fourth of the patients. A high serum ferritin level and poor patient compliance to therapy were significantly associated with increased endocrine disorders. Combined iron-chelating agents were associated with a decreased prevalence of endocrine disorders when compared with monotherapy.

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Section: Discussioncontrasting

confidence: 81%

Detection of endocrine disorders in young children with multi-transfused thalassemia major

2021

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“…The prevalence of the other endocrine complications in our pediatric population was hypogonadism, hypothyroidism and GH deficit—5.9%, and hypoparathyroidism—4.4%. This prevalence was consistent with some studies [ 66 , 67 ], while other studies reported a significantly higher incidence of endocrinopathies [ 14 , 16 ]. These discrepancies could be attributed to differences in age distribution and therapeutic management, and to genetic, geographical, cultural, and economic factors.…”

Section: Discussionsupporting

confidence: 93%

Link between Genotype and Multi-Organ Iron and Complications in Children with Transfusion-Dependent Thalassemia

Meloni

Pistoia

Ricchi

et al. 2022

JPM

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We evaluated the impact of the genotype on hepatic, pancreatic and myocardial iron content, and on hepatic, cardiac and endocrine complications in children with transfusion-dependent β-thalassemia (β-TDT). We considered 68 β-TDT patients (11.98 ± 3.67 years, 51.5% females) consecutively enrolled in the Extension-Myocardial Iron Overload in Thalassemia network. Iron overload was quantified by T2* technique and biventricular function by cine images. Replacement myocardial fibrosis was evaluated by late gadolinium enhancement technique. Three groups of patients were identified: homozygous β+ (N = 19), compound heterozygous β0β+ (N = 24), and homozygous β0 (N = 25). The homozygous β0 group showed significantly lower global heart and pancreas T2* values than the homozygous β+ group. Compared to patients with homozygous β+ genotype, β0β+ as well as β0β0 patients were more likely to have pancreatic iron overload (odds ratio = 6.53 and 10.08, respectively). No difference was detected in biventricular function parameters and frequency of replacement fibrosis. No patient had cirrhosis/fibrosis, diabetes or heart failure, and the frequency of endocrinopathies was comparable among the groups. In pediatric β-TDT patients, there is an association between genotype and cardiac and pancreatic iron overload. The knowledge of patients’ genotype can be valuable in predicting some patients’ phenotypic features and in helping the clinical management of β-TDT patients.

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“…From the clinical practice point of view, the unfavorable effect of ferritin on pubertal development can be minimized by the use of appropriate chelation therapy in proper time [21,22]. A very recent study showed the good effect of appropriate chelation therapy on endocrinologic complications in children with thalassemia [23].…”

Section: Discussionmentioning

confidence: 99%

Pancreatic MR imaging and endocrine complications in patients with beta-thalassemia: a single-center experience

et al. 2021

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Iron deposition in various organs can cause endocrine complications in patients with transfusiondependent beta-thalassemia. The aim was to investigate the relationship between endocrine complications and pancreatic iron overload using magnetic resonance imaging (MRI). Forty patients with transfusion-dependent thalassemia (TDT) were enrolled in the study. The magnetic resonance imagings of the patients were performed using a 1.5 Tesla Philips MRI scanner. Two out of three patients had at least one clinical endocrine complication. The rate of iron deposition was 62.5% in liver, and 45% in pancreas tissue, and was 12.5% in heart tissue. Pancreatic T2* and hepatic T2* values were signi cantly positively correlated (p = 0.006). Pancreatic T2* and ferritin were signi cantly negatively correlated (p = 0.03). Cardiac T2* values were negatively correlated with fasting blood glucose (p = 0.03). Patients with short stature had signi cantly higher cardiac iron burden (22.3 vs. 36.6 T2*ms; p00.01) and patients with hypothyroidism had higher liver iron concentrations (9.9 vs. 6.4 LIC mg/g; p = 0.05). The ferritin level of 841 ng/mL, and liver iron concentration (LIC) value of 8.7 mg/g were detected as the threshold level for severe pancreatic iron burden (AUC 70%, p:0.04, AUC 80%, p = 0.002, respectively). Moreover, males were found to have decreased pancreas T2* values compared with the values in females (T2* 19.3 vs. 29.9, p = 0.05). Patients with higher ferritin levels over than 840 ng/mL should be closely monitored for pancreatic iron deposition, and patients with endocrine complications should be assessed in terms of cardiac iron burden.

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The effect of deferasirox on endocrine complications in children with thalassemia

Cited by 12 publications

References 22 publications

Detection of endocrine disorders in young children with multi-transfused thalassemia major

Detection of endocrine disorders in young children with multi-transfused thalassemia major

Link between Genotype and Multi-Organ Iron and Complications in Children with Transfusion-Dependent Thalassemia

Pancreatic MR imaging and endocrine complications in patients with beta-thalassemia: a single-center experience

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