Growth of Children with Thalassaemia: Effect of Different Transfusion Regimens

Kattamis, C; Touliatos, N.; Haidas, S; Matsaniotis, N.

doi:10.1136/adc.45.242.502

Cited by 60 publications

(23 citation statements)

References 10 publications

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“…After the age of 4 years, significant growth retardation involves stature, sitting height, weight and skeletal maturation (1)(2)(3). Delayed or complete lack of pubescent changes are common in both girls and boys (4,5).…”

Section: Introductionmentioning

confidence: 99%

GH response to provocation and circulating IGF-I and IGF-binding protein-3 concentrations, the IGF-I generation test and clinical response to GH therapy in children with beta-thalassaemia

Soliman

Banna

Ansari

1998

European Journal of Endocrinology

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The causes of growth retardation of children with thalassaemia major are multifactorial. We studied the GH response to provocation by clonidine and glucagon, measured the circulating concentrations of insulin, IGF-I, IGF-binding protein-3 (IGFBP-3) and ferritin, and evaluated IGF-I generation after a single dose of GH (0.1 mg/kg per dose) in 15 prepubertal patients with thalassaemia, 15 age-matched children with constitutional short stature (CSS) (height standard deviation score less than ¹2, with normal GH response to provocation) and 11 children with isolated GH deficiency (GHD). Children with thalassaemia had significantly lower peak GH response to provocation by clonidine and glucagon (6:2 Ϯ 2:3 and 6:8 Ϯ 2:1 mg/l respectively) than the CSS group (18:6 Ϯ 2:7 and 16:7 Ϯ 3:7 mg/l respectively). They had significantly decreased circulating concentrations of IGF-I and IGFBP-3 (47:5 Ϯ 19 ng/ml and 1:2 Ϯ 0:27 mg/l respectively) compared with those with CSS (153 Ϯ 42 ng/ ml and 2:06 Ϯ 0:37 mg/l respectively), but the IGF-I and IGFBP-3 concentrations were not different from those with GHD (56 Ϯ 25 ng/ml and 1:1 Ϯ 0:32 mg/l respectively). These data demonstrate that the GH-IGF-I-IGFBP-3 axis in thalassaemic children is defective. Serum ferritin concentration correlated significantly with GH peak response to provocation (r ¼ ¹0:36, P < 0:05) and circulating IGF-I (r ¼ ¹0:47, P < 0:01) and IGFBP-3 (r ¼ ¹0:42, P < 0:01) concentrations. In the IGF-I generation test, after GH injection, the thalassaemic children had significantly lower IGF-I and IGFBP-3 levels 86:7 Ϯ 11:2 ng/ml and 2:05 Ϯ 0:51 mg/l respectively) than those in the CSS group (226 Ϯ 45:4 ng/ ml and 2:8 Ϯ 0:43 mg/l respectively). The IGF-I response was significantly higher in children with GHD (158 Ϯ 50 ng/ml) than in thalassaemic children. Six short (height standard deviation score less than ¹2) thalassaemic children who had defective GH response to provocation (<10 mg/l), all the children with GHD and eight short normal children (CSS) were treated for 1 year with human GH (18 units/m 2 per week divided into daily s.c. doses). After 1 year of GH therapy there was a marked acceleration of growth velocity in both thalassaemic children (from 3:8 Ϯ 0:6 cm/year to 7:2 Ϯ 0:8 cm/year) and controls. However, the linear acceleration of growth velocity on GH therapy was significantly slower in thalassaemic children (3:3 Ϯ 0:3 cm/year increment) compared with those with CSS (5:3 Ϯ 0:4 cm/year increment) and GHD (6:9 Ϯ 1:2 cm/year increment) (P < 0:05). Their circulating IGF-I concentration (105 Ϯ 36 ng/ml) was significantly lower than those for CSS (246 Ϯ 58 ng/ml) and GHD (189 Ϯ 52 ng/ml) after 1 year of GH therapy. These data prove that some children with b-thalassaemia major have a defective GH-IGF-I-IGFBP-3 axis and suggest the presence of partial resistance to GH.

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Section: Introductionmentioning

confidence: 99%

GH response to provocation and circulating IGF-I and IGF-binding protein-3 concentrations, the IGF-I generation test and clinical response to GH therapy in children with beta-thalassaemia

Soliman

Banna

Ansari

1998

European Journal of Endocrinology

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“…According to sex, this result was different than the other studies that found most of growth impairments occurs in girls aged 10-15 years. 15 This study showed that bone age deficit that poor prognosis (>36 months) mostly occurs in boys. A study in Italy also found that bone lesion is more severe in boys compared with that in girls.…”

Section: Discussionmentioning

confidence: 99%

Association between age and serum ferritin level with bone age deficit in children with thalassemia major

Andayani¹,

Sekarwana²,

Fadil³

2008

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Background Multiple blood transfusions in thalassemia patientslead to iron overload in bone tissue. Iron overload can bedetermined by serum ferritin measurement. Several studies haveevaluated association between serum ferritin level and growth,but without bone age examination.Objective To determine the association between age and serumferritin level with bone age in children with thalassemia major.Methods This study was conducted at Hasan Sadikin HospitalBandung during March-May 2007. We performed physicalexamination, serum feritin measurement, and bone ageexamination. Data were analyzed with x 2 to determine associationbetween variables. The association between age and serum ferritinlevel with bone age deficit was analyzed with regression logisticmodel.Results Subjects consisted of 49 patients with thalassemia major.All subjects had bone age deficit. Most boys were in age group of>10 years and had bone age difference >36 months, while mostgirls were <10 years and had bone age difference <36 months.Subjects with bone age difference <36 months mostly had serumferritin level <5,000 ng/dL, while most subjects with bone agedifference >36 months had serum ferritin level =5,000 ng/dL.This was statistically significant (x 2 =4.573, P=0.032). There wasassociation between age and bone age deficit (OR=13.461, 95%CI 3.199;56.640), but not with serum ferritin level (OR= 2.199,95% CI 0.532;9.095).Conclusion In thalassemic children, bone age deficit is associatedwith age, but not with serum feritin level.

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“…Growth failure is present in up to 30% of TM patients. Characteristically, reduced growth is observed around the age of 10–11 years (2–5). The result is a final height which is 1–2 centiles below the genetically predicted height.…”

Section: First Decade Of Lifementioning

confidence: 99%

Clinical aspects of transfusional iron overload

Wonke¹,

Sanctis²

2000

Rev Clin Exp Hematol

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Most of our knowledge of transfusional iron overload has been obtained from patients with β‐thalassemia major (TM). Iron overload causes multiple endocrinopathies presenting in the first decade of life with growth disturbance, followed by thyroid dysfunction. By the second decade of life, carbohydrate metabolism is disturbed in up to 25% of patients and failure of sexual development, even in well‐chelated patients, appears as the commonest endocrine complication. In the third and fourth decades of life, osteopenia and osteoporosis are the causes of morbidity in over 50% of well‐chelated and transfused TM patients. Cardiac disease, secondary to iron damage, causes death in developed countries as a result of noncompliance to desferrioxamine mesylate (DFX) from the third decade of life. In underdeveloped countries, cardiac death starts from 12 years of age, due to the nonavailability of the iron chelating agent DFX. With the emergence of the advanced cardiac magnetic resonance imaging technique, early diagnosis of heart iron will allow the use of parenteral and oral chelators in an innovative way to improve survival and the quality of life for TM patients.

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Growth of Children with Thalassaemia: Effect of Different Transfusion Regimens

Cited by 60 publications

References 10 publications

GH response to provocation and circulating IGF-I and IGF-binding protein-3 concentrations, the IGF-I generation test and clinical response to GH therapy in children with beta-thalassaemia

GH response to provocation and circulating IGF-I and IGF-binding protein-3 concentrations, the IGF-I generation test and clinical response to GH therapy in children with beta-thalassaemia

Association between age and serum ferritin level with bone age deficit in children with thalassemia major

Clinical aspects of transfusional iron overload

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