Effect of Age at the Start of Iron Chelation Therapy on Gonadal Function in β-Thalassemia Major

Bronspiegel-Weintrob, N; Olivieri, NF; Tyler, Bev; Andrews, D. F.; Freedman, Mervin B.; Fj, Holland

doi:10.1056/nejm199009133231104

Cited by 153 publications

(72 citation statements)

References 31 publications

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“…13 In the present study, there was no overt hypothyroidism in our patients with BTM, the result which agreed with that of Bronspiegel-Weintrobe et al 1990, 14 but the study of Aydinok et al 2002 11 declared that 17-18% of their patients with betathalassemia have hypothyroidism. IGF1 was statistically significantly lower in our children patients with BTM than the control group p<0.03, the result which agreed with that of DeLuca et al 1995, 15 who attributed this low level to disturbed liver function secondary to iron overload and malnutrition.…”

Section: Discussionsupporting

confidence: 82%

Growth pattern in children with beta-thalassemia major and its relation with serum ferritin, IGF1 and IGFBP3

Nasr

Ebrahim²,

Salahedin³

2012

J Clin Exp Invest

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ÖZETAmaç: Beta-talasemi major'lu (BTM) çocuklarda büyüme geriliği aşırı demir yükü ve endokrinolojik anormallikleri de içeren birçok muhtemel etyoloji nedeniyledir. Biz BTM'li çocuklarda büyümeyi ve serum ferritin, tiroid hormonları, IGF1 ve IGFBP3 ile ilişkisini araştırmayı amaçladık. Gereç ve yöntem:Beta-talasemi majorlu 33 çocuk ve yaş, cinsiyet ve boy bakımından eşleştirilmiş 30 sağlıklı kontrol grubu çocuk için tam bir öykü ve fizik muayene yapılarak, antropometrik ölçümleri alındı. Her iki grupta açlık kan örnekleri alınarak tam kan sayımı, açlık kan şekeri, karaciğer fonksiyon testleri, serum ferritin, tiroid profile, IGF1 ve IGFP3 düzeyleri çalışıldı. Her iki grubun kemik yaşı radyolojik olarak belirlendi.Bulgular: Beta-talasemi major grubunda boy kısalığı %57.6 ve gecikmiş puberte % 45.5 oranında saptandı. Üst/Alt ekstremite oranı BTM grubunda kontrollere nazaran anlamlı düşük bulundu (p=0.035). Talasemi grubunda açık hipotiroidi görülmedi ancak BTM grubunda IGF1 ve IGFBP3 düzeyleri kontrollerden anlamlı düşük bulundu (sırasıyla, p=0.022 ve p=0.037). T4 ile üst/alt ekstremite oranı arasında anlamlı bağıntı mevcuttu. IGF1, boy, transfüzyon süresi ve şelasyon süresi ile anlamlı ilişkili bulundu. Serum ferritin ile diğer değişkenler arasında anlamlı bir korelasyon saptanmadı.Sonuç: Beta-talasemi major'lu çocuklarda düzenli transfüzyonlar ve şelasyon tedavisine ragmen özellikle büyük çocuklarda büyüme geriliği belirgindir ve bunun nedeni asıl olarak büyüme hormone-IGF1 ve IGBP3 eksenindedir.Anahtar kelimeler: Beta-talasemi major, çocuklar, büyü-me, tiroid fonksiyonları, IGF1, IGFBP3, ferritin ABSTRACT Objectives: Growth impairment in children with Betathalassemia major (BTM) has several possible etiologies including excess iron overload and endocrinologic abnormalities. We aimed to assess growth in children with BTM and its relation with serum ferritin, thyroid hormones, IGF1 and IGFBP3. Materials and methods:Thirty-three children with BTM and 30 healthy children (control group) matched in age, sex and height were subjected to full clinical history and examination, including anthropometric measurements. Fasting blood samples from both groups were taken for complete blood counts, fasting blood sugar, liver function tests, serum ferritin, thyroid profiles, IGF1 and IGFBP3. Bone ages for both groups were determined radiologically.Results: A total of 57.6% of BTM group had retarded linear growth and 45.5% of them were with delayed puberty. There was statistical significant decrease in upper/ lower segment (U/L) ratio in the BTM group compared to the control group (p=0.035). No apparent hypothyroidism was found in the thalassemia group, but significant decreases were found in both IGF1 and IGFBP3 levels of the thalassemia group compared with the control group (p=0.022 and p=0.037, respectively). There was a significant correlation between T4 and U/L ratio (p<0.05); IGF1 was significantly correlated with height, duration of transfusions and duration of chelation (p<0.05). No significant correlation was found betw...

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Section: Discussionsupporting

confidence: 82%

Growth pattern in children with beta-thalassemia major and its relation with serum ferritin, IGF1 and IGFBP3

Nasr

Ebrahim²,

Salahedin³

2012

J Clin Exp Invest

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“…The prevalence of hypogonadism and/or delayed puberty was reported at 40%-80% [3,4] by previous studies. This prevalence depended on serum ferritin levels, type of thalassemia, the onset of blood transfusion and chelating therapy [4,7,13,14]. We had no patients with abnormal testicular function or delayed puberty in this study.…”

Section: Discussionmentioning

confidence: 99%

“…This can be attributed to adequate treatment with blood transfusion at an appropriate time (3.8 ± 2.3 years old) and effective iron chelation shown by a mean serum ferritin level during the past year of 1,575 ± 642 ng/mL. Bronspiegel-Weintrob et al [13] reported that patients with abnormal puberty had higher serum ferritin levels before chelation and had received suboptimal chelation therapy (serum ferritin levels 4,734 ± 3,081 and 3,572 ± 1,870 ng/mL, respectively). Important factors for developing hypogonadism and/ or delayed puberty are a high serum ferritin level [10][11][12].…”

Section: Discussionmentioning

confidence: 99%

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Testicular function in patients with regular blood transfusion for thalassemia major

et al. 2015

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Background: Regular blood transfusion and iron chelation therapy have improved the quality of life of patients with thalassemia and increased their longevity, but transfusion also increases the frequency of endocrine complications, possibly because of iron deposition in the pituitary gland or the gonads, or both. Objective: To evaluate testicular function in patients with thalassemia major by basal hormonal study, and identify risk factors for dysfunction. Methods: We performed a cross-sectional study of 28 patients with thalassemia major aged 11.7 ± 1.8 (8−14.9) years (15 in prepuberty, 13 in puberty with no delayed puberty) who had regular blood transfusions. A normal control group comprised 64 boys who were matched for age and Tanner genital stage. Results: The mean level of serum ferritin in the previous year was 1,575 ± 642 ng/mL, and the onset of blood transfusion was at 3.8 ± 2.3 years and iron chelation therapy was 6.6 ± 2.8 years. The trend for anti-Müllerian hormone levels in patients and controls was similar with age, and although higher in the patients, particularly at Tanner stage II, was not significantly different. Testosterone levels were lower in the patients compared with controls; particularly at Tanner stages IV-V (290.88 vs. 537.4 ng/dL, P < 0.05). Serum follicle-stimulating hormone and luteinizing hormone levels were not significantly different between the groups at any Tanner stage. Conclusion: Patients who received regular blood transfusions had normal Sertoli cell function. Leydig cell dysfunction may occur, even though the patients had a normal pubertal onset.

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“…The most common complication of iron loading in thalassemia, hypogonadism, is also prevented by effective use of deferoxamine (Bronspiegel-Weintrob et al 1990), although secondary amenorrhea in women, and secondary hypogonadism in men, may develop after age 21, even in those who have attained normal puberty (Gamberini et al 2008). …”

Section: Clinical Benefitsmentioning

confidence: 99%

Management of the Thalassemias

Olivieri

Brittenham

2013

Cold Spring Harbor Perspectives in Medicine

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During the last 30 years, in addition to the considerable progress made in control and prevention of thalassemias 3 , there have also been major advances in their symptomatic management, at least in wealthier countries where appropriate facilities are available. Remarkable improvements in survival in the severe forms of thalassemia have followed the more judicious use of blood transfusion and, in particular, the ability to manage the iron accumulation resulting from transfusion with its severe and ultimately lethal effects on endocrine and cardiac function.

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Effect of Age at the Start of Iron Chelation Therapy on Gonadal Function in β-Thalassemia Major

Abstract: Beginning chelation treatment with deferoxamine before the age of puberty can help children with transfusion-dependent thalassemia major to attain normal sexual maturation.

Cited by 153 publications

References 31 publications

Growth pattern in children with beta-thalassemia major and its relation with serum ferritin, IGF1 and IGFBP3

Growth pattern in children with beta-thalassemia major and its relation with serum ferritin, IGF1 and IGFBP3

Testicular function in patients with regular blood transfusion for thalassemia major

Management of the Thalassemias

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