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Title
Abstract
Objectives
Search Methods
Main Results
Background
Study Characteristics
Key Results
Conclusions
№ of Participants (Studies)
Why It is Important to Do This Review
Types of Participants
Search Methods for Identification of Studies
Data Collection and Analysis
Data Extraction and Management
Cochrane Database of Systematic Reviews
Unit of Analysis Issues
Dealing with Missing Data
Data Synthesis
Sensitivity Analysis
Summary of Findings Table
Results of the Search
Trial Design
Participants
Incomplete Outcome Data
Participant or Parental Satisfaction
Summary of Main Results
Potential Biases in the Review Process
Implications for Practice
Implications for Research
Were Incomplete Outcome Data Adequately Addressed?
References
All Topics
Medicine and Health Sciences
Pediatrics

Growth hormone therapy for people with thalassaemia

Profile image of Nai Ming LaiNai Ming Lai

2020, The Cochrane library

https://doi.org/10.1002/14651858.CD012284.PUB3
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Abstract

Guidelines for the management of transfusion dependent thalassaemia (TDT). 3rd edition. Thalassaemia International Federation, 2014.

Related papers

Growth deficiency in polytransfused β‐thalassaemia patients is not growth hormone dependent
Francesco Gallo

Clinical Endocrinology, 1997

OBJECTIVE Growth deficiency is commonly seen in polytransfused b-thalassaemia patients, especially in adolescence. It is not completely dependent on the lack of their pubertal growth spurt. GH impairment at different levels (hypothalamic or pituitary) and/or a reduced IGF-1 synthesis have been suggested the main causes of stunted growth in these patients. We evaluated the relationship between GH reserve and growth in short b-thalassaemia patients. PATIENTS Twenty-nine short patients (height <¹1. 8 SDS for chronological age) were divided into two groups (low and normal responders) on the basis of their GH peak during insulin and clonidine tests (Յ and > 20 mU/l, respectively). All but one low responders underwent the GHRH test to exclude the impairment of somatotroph function and in eight of them an IGF-1 generation test was also performed. The two groups were compared with each other with respect to growth (height deficiency, height velocity, bone age and bone delay), haematological characteristics (serum ferritin levels, age at the start both of low (subcutaneous) s.c. infusion of desferrioxamine and of transfusional therapy) and serum IGF-1 and IGF-1 binding protein 3 levels. RESULTS Thirteen patients (45%) (11 males, two females) were low responders, all but two having serum IGF-1 < 5th centile (< 0. 1 centile in 42%); the GHRH test excluded the impairment of somatotroph function in 8/12. Height deficiency, serum ferritin levels, and age at the start of s.c. chelating therapy

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Study of Growth Hormone Levels in Thalessemia Major Patients in Children
rasika hattewar

Journal of Medical Science And clinical Research, 2020

Introduction: Thalassemia is an inherited disorder of hemoglobin synthesis. Regular blood transfusions and chelation therapy have notice ably prolonged survival in thalassemic patients. [1] Despite a significant increase in the life span of these patients, they suffer from multiple abnormalities probably due to iron over load, including endocrinal abnormalities such as hypogonadism, diabetes mellitus, hypothyroidism and hyperparathyroidism. [2] The pattern of levels of growth hormone levels among the patients with thalassemia major undergoing repeated blood transfusions remain unexplored. Very few studies have been undertaken among Indian population. Methodology: It is a prospective study and was conducted at St George hospital, Mumbai, on diagnosed patients of thalassemia major admitted to the paediatric wards. Duration of the study was 3 months and 20 patients were included. Inclusion Criteria 1. All admitted patients of thalassemia major aged 18 years and below. Exclusion Criteria 1. Chronic haemolytic anemia apart from thalassemia major 2. Those that was very sick 3. Those suffering from malnutrition 4. Those who were on supplementation of growth hormone. Patients who were willing to participate and sign the inform consent were enrolled in the study. Growth hormone levels were done using CLIA. Results: The mean age of the studied thalassemia patients was 9.65 ± 4.23 years with no gender preponderance. Mean (SD) Growth hormone levels was 0.769 ng/dl ± 0.540 ng/dl and was not found to be statistically significant (p>0.001). Conclusion: It is evident from the present study that the levels of Growth Hormone are normal among βthalassemia major patients on repeated blood transfusion. But GH supplementation can help these patients. Frequent monitoring and supplementation in deficient states is recommended.

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Selected highlights of the VIII International Symposium of Clinicians for Endocrinopathies in Thalassemia and Adolescent Medicine (ICET-A) on Growth, Puberty and Endocrine Complications in Thalassaemia. Auditorium of the Sultan Qaboos University (SQU) Muscat (Sultanate of Oman), 20th of December ...
Saif Al Yaarubi, Ashraf Soliman

Pediatric endocrinology reviews : PER, 2015

The VIII ICET-A International Symposium was held in Muscat (Sultanate of Oman) on the 20th of December, 2014. The symposium included four sessions on a wide range of topics covering growth disorders and endocrine complications in thalassaemia. Despite the fact that endocrine complications are very common in multi-transfused thalassaemia patients a recent survey conducted by the International Network of Clinicians for Endocrinopathies in Thalassemia and Adolescent Medicine (ICET-A) in 2014 in Acitrezza (Catania, Italy) showed that the major difficulties reported by hematologists or pediatricians experienced in thalassaemias or thalassaemia syndromes in following endocrine complications included: Lack of familiarity with medical treatment of endocrine complications, interpretation of endocrine tests, lack of collaboration and on-time consultation between thalassaemic centres supervised by haematologists and endocrinologists. Endocrine monitoring of growth, pubertal development, reprod...

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Linear Growth Deficiency in B-Thalassemia Patients: Is It Growth Hormone Dependent?
Dr. Mahdi Shahriari

Iranian Journal of Medical Sciences, 2002

Background: b-Thalassemia major is a serious medical problem. Growth retardation is commonly seen in poly-transfused b- thalassemia patients. The exact mechanism of short stature in children with thalassemia major is not well understood, however, it is believed to be multi-factorial. Objective: To study the growth state and its relationship to growth hormone (GH) deficiency in b-thalassemia patients. Methods: The extent of growth and growth hormone deficiency were evaluated in 150 patients aged 10-22 years (84 males, 66 females) residing in Shiraz. The age, sex, height, weight and serum ferritin levels were recorded using a questionnaire. Growth hormone secretion was determined with L-Dopa provocative test in 138 b- thalassemia patients. Results: Short stature was present in 64% of patients (63.6% of girls, 69% of boys). Growth hormone deficiency was present in 57.5% of 138 patients, (53.5% of boys, 46.5% of girls). Short stature was found in 83.5% of GH deficient and 74.6% of GH su...

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Highlights of the IX International Symposium of Clinicians for Endocrinopathies in Thalassemia and Adolescent Medicine (ICET-A) on Growth, and Endocrine Complications in Thalassaemia
Ashraf Soliman

The IX Symposium of the International Network of Clinicians for Endocrinopathies in Thalassemia and Adolescent Medicine (ICET-A) was held in the glamorous city of Abu Dhabi on the 6th of February, 2015 in the Course of IV Pan Arab Hematology Conference and XIII Saudi Society of Hematology Congress. Both meetings were merged with the highlights of the European Hematology Association (EHA).The symposium included four sessions on a wide range of topics covering growth disorders and endocrine complications in thalassaemia. The goals of the meeting were to provide an update on current research, to inspire younger investigators in this field, to promote interaction between different countries, and to introduce the ICET-A group, its aims and directions for its progress.

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Endocrine investigation and follow up in thalassaemia. Time for specific guidelines
Nicos Skordis

Thalassemia Reports, 2012

Iron overload due to multiple transfusions affects the endocrine glands especially the anterior pituitary, the pancreas, the thyroid and the parathyroids. This leads to a variety of endocrinopathies and growth failure. Delayed puberty, hypogonadism, growth hormone deficiency in adults, hypothyroidism, hypoparathyroidism and diabetes are common and around 20% of patients have more than one endocrinopathy. In this paper suggestions for guidelines concerning diagnosis, investigation and treatment are proposed for the following clinical entities encountered in thalassaemia patients: i) Growth failure: after the age of 9-10 rears there is a slowing of growth velocity, the pathogenesis of which is multifactorial and anaemia, folate deficiency and hypersplenism are implicated. Desferrioxamine toxicity has been reported as cause of the abnormal upper to lower segment ratio. Growth hormone is given in selected cases. ii) Delayed puberty and hypogonadism: are the most obvious clinical consequences of iron overload in both sexes. Primary and secondary amenorrhoea are very common in women. Sex steroid replacement therapy is the optimal therapeutic regime which has a great impact on the quality of life of adult thalassaemia patients. iii) Fertility: Women with TM, who are regularly transfused and are well chelated can now become pregnant either spontaneously or by inducing ovulation. Pregnancy must carefully monitored. iv) Growth hormone deficiency in adult thalassaemics: This occurs in a high prevalence and since GH in adults is involved in numerous biological functions, especially of the heart, proper assessment of this hormone is needed and consideration of the need for replacement. v) Hypothyroidism and hypoparathyroidism: these deficiencies are also discussed.

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Prevalence of growth and endocrine disorders in Malaysian children with transfusion-dependent thalassaemia
Muhammad Yazid Jalaludin

Singapore Medical Journal, 2018

Endocrine dysfunction due to iron overload secondary to frequent blood transfusions is a common complication in children with transfusion-dependent thalassaemia (TDT). We ascertained the prevalence of endocrine dysfunction in children with TDT seen in a hospital setting in Malaysia. We reviewed all patients with TDT who had ≥ 8 blood transfusions per year. Patients who had a history of stem cell transplantation, concurrent autoimmune diseases or were newly diagnosed to have TDT were excluded. Standard diagnostic criteria were used in the diagnosis of various endocrine dysfunctions. Of the 82 patients with TDT, 65% had at least one endocrine dysfunction. Short stature was the commonest (40.2%), followed by pubertal disorders (14.6%), hypoparathyroidism (12.3%), vitamin D deficiency (10.1%), hypocortisolism (7.3%), diabetes mellitus (5.2%) and overt hypothyroidism (4.9%). Subclinical hypothyroidism and prediabetes mellitus were seen in 13.4% and 8.6% of the patients, respectively. For children aged < 10 years, the prevalence of both thyroid dysfunction and hypoparathyroidism was 9.1%. CONCLUSION Two-thirds of children with TDT experienced at least one endocrine dysfunction. Thyroid dysfunction and hypoparathyroidism may be missed if endocrine screening is only performed in children with TDT > 10 years of age. Close monitoring for endocrine dysfunction and hormonal therapy is essential to prevent long-term adverse outcomes.

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Growth Hormone and Some Other Parameters Estimation in Thalassemia major Patients
Nawal Mahdi

Kirkuk University Journal-Scientific Studies, 2016

group. This study revealed that there was highly significant decrease in all parameters in thalassemic patients (except serum ferritin was highly significant increase in patients) as compare with controls (P< 0.001). Positive correlation found between GH and both Hb and PCV, while negative correlation was found between GH and ferritin level.

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Growth and endocrine disorders in thalassemia: The international network on endocrine complications in thalassemia (I-CET) position statement and guidelines
Nicos Skordis

Indian Journal of Endocrinology and Metabolism, 2013

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Highlights of the VIII International Symposium of Clinicians for Endocrinopathies in Thalassemia and Adolescent Medicine (ICET-A ) on Growth, Puberty and Endocrine Complications in Thalassaemia. Auditorium of the Sultan Qaboos University (SQU) Muscat (Sultanate of Oman), 20th of December 2014
Ashraf Soliman

T he VIII ICET-A International Symposium was held in Muscat (Sultanate of Oman) on the 20th of December, 2014. The symposium included four sessions on a wide range of topics covering growth disorders and endocrine complications in thalassaemia. Despite the fact that endocrine complications are very common in multi-transfused thalassaemia patients a recent survey conducted by the International Network of Clinicians for Endocrinopathies in Thalassemia and Adolescent Medicine (ICET-A) in 2014 in Acitrezza (Catania, Italy) showed that the major difficulties reported by hematologists or pediatricians experienced in thalassaemias or thalassaemia syndromes in following endocrine complications included: Lack of familiarity with medical treatment of endocrine complications, interpretation of endocrine tests, lack of collaboration and on-time consultation between thalassaemic centres supervised by haematologists and endocrinologists. Endocrine monitoring of growth, pubertal development, reproductive ability and endocrine function in general are essential to achieve a good quality of life as well as controlling the pain which results from the defects of bone structure, all of which increase with the age of patients. Such comprehensive care is best provided by coordinated, multidisciplinary teams working in expert centres. The multidisciplinary team must include an endocrinologist, preferably someone experienced in the management of hormonal deficiencies

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Growth hormone therapy for people with thalassaemia (Review)
Amutha Ramadas

Cochrane Library Cochrane Database of Systematic Reviews , 2020

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Growth hormone therapy for people with thalassaemia (Protocol)
Amutha Ramadas

This is the protocol for a review and there is no abstract. The objectives are as follows: To assess the risks and benefits of GH therapy in people with thalassaemia.

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Cochrane Database of Systematic Reviews Growth hormone therapy for people with thalassaemia (Review) Growth hormone therapy for people with thalassaemia (Review
Chin Fang Ngim, Amutha Ramadas

ackground Thalassaemia is a recessively-inherited blood disorder that leads to anaemia of varying severity. In those affected by the more severe forms, regular blood transfusions are required which may lead to iron overload. Accumulated iron from blood transfusions may be deposited in vital organs including the heart, liver and endocrine organs such as the pituitary glands which can affect growth hormone production. Growth hormone deficiency is one of the factors that can lead to short stature, a common complication in people with thalassaemia. Growth hormone replacement therapy has been used in children with thalassaemia who have short stature and growth hormone deficiency. Objectives To assess the benefits and safety of growth hormone therapy in people with thalassaemia. Search methods We searched the Cochrane Haemoglobinopathies Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books. We also searched the reference lists of relevant articles, reviews and clinical trial registries. Our database and trial registry searches are current to 10 August 2017 and 08 August 2017, respectively. Selection criteria Randomised and quasi-randomised controlled trials comparing the use of growth hormone therapy to placebo or standard care in people with thalassaemia of any type or severity. Data collection and analysis Two authors independently selected trials for inclusion. Data extraction and assessment of risk of bias were also conducted independently by two authors. The quality of the evidence was assessed using GRADE criteria. Main results One parallel trial conducted in Turkey was included. The trial recruited 20 children with homozygous beta thalassaemia who had short stature; 10 children received growth hormone therapy administered subcutaneously on a daily basis at a dose of 0.7 IU/kg per week and 10 children received standard care. The overall risk of bias in this trial was low except for the selection criteria and attrition bias which were unclear. The quality of the evidence for all major outcomes was moderate, the main concern was imprecision of the estimates due to the small sample size leading to wide confidence intervals. Final height (cm) (the review's pre-specified primary outcome) and change in height were not assessed in the included trial. The trial reported no clear difference between groups in height standard deviation (SD) score after one year, mean difference (MD) -0.09 (95% confidence interval (CI) -0.33 to 0.15 (moderate quality evidence). However, modest improvements appeared to be observed in the following key outcomes in children receiving growth hormone therapy compared to control (moderate quality evidence): change between baseline and final visit in height SD score, MD 0.26 (95% CI 0.13 to 0.39); height velocity, MD 2.28 cm/year (95% CI 1.76 to 2.80); height velocity SD score, MD 3.31 (95% CI 2.43 to 4.19); and change in height velocity SD score between baseline and final visit, MD 3.41 (95% CI 2.45 to 4.37). No adverse effects of treatment were reported in either group; however, while there was no clear difference between groups in the oral glucose tolerance test at one year, fasting blood glucose was significantly higher in the growth hormone therapy group compared to control, although both results were still within the normal range, MD 6.67 mg/dL (95% CI 2.66 to 10.68). There were no data beyond the one-year trial period. Authors' conclusions A small single trial contributed evidence of moderate quality that the use of growth hormone for a year may improve height velocity of children with thalassaemia although height SD score in the treatment group was similar to the control group. There are no randomised controlled trials in adults or trials that address the use of growth hormone therapy over a longer period and assess its effect on final height and quality of life. The optimal dosage of growth hormone and the ideal time to start this therapy remain uncertain. Large well-designed randomised controlled trials over a longer period with sufficient duration of follow up are needed.

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Recombinant human growth hormone treatment in children with thalassemia major
Sabri Çetinkaya

Pediatrics International, 1999

AbstractBackground: To evaluate the growth hormone reserve and the growth hormone response to recombinant human growth hormone (GH) in prepubertal thalassemic children with growth retardation.Methods: Twenty thalassemic patients with short stature and delayed bone age were studied. Patients were randomized into GH-treated (n=10) and non-GH treated (control; n=10) groups. The GH-treated group received recombinant human (rh)-GH (Genotropin) at the dose of 0.7 IU/kg per week for 12 months.Results: There was a significant discordance between GH response to pharmacologic stimuli and physiological secretion of GH/GHRH testing. Following the administration of rhGH, growth velocity increased from 2.47~0.48 cm/year to 6.27~0.76 cm/year (P=0.005), whereas there was not a similar change in the non-GH-treated group. The height velocities of the two groups during the 1 year follow-up period were significantly different (6.27~0.76 vs 3.99~0.34 cm/year; P=0.025). There were significant differences between the height velocity improvements and height velocity standard deviation scores of the two groups as well.Conclusion: The present study has demonstrated that rhGH is a safe and efficacious mode of treatment in thalassemic children.

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Growth hormone secretion in adult patients with thalassaemia
Maria Galati

Clinical Endocrinology, 2005

Growth retardation and short stature are frequent clinical features of patients with beta-thalassaemia major. Dysfunction of the GH-IGF-1 axis has been described in many thalassaemic children and adolescents with short stature and reduced growth velocity. Several studies have demonstrated that recombinant GH treatment improves growth velocity in these patients, although response to the treatment is variable and not predictable. A reassessment of the GH-IGF-1 axis must be performed in young adults with childhood-onset GH deficiency (GHD), after attainment of final height, to select those who are candidates for replacement therapy as adults. To our knowledge there are no data available on retesting the GH-IGF-1 axis in adult thalassaemic patients with childhood-onset GHD. The aim of our study was to investigate GH secretion in adult thalassaemic patients with childhood-onset GHD. Design We reassessed GH secretion in a group of adult thalassaemic patients in whom partial GHD had been diagnosed during childhood.

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Related topics

  • Pediatrics
  • Medicine
  • Growth hormone deficiency
  • Short stature
  • Medical and Health Sciences

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    Gian Forni

    Journal of Clinical Medicine, 2022

    Background: The treatment of endocrinopathies in haemoglobinopathies is a continually expanding research area; therefore, recommendations supporting the appropriateness of treatments are a pressing need for the medical community. Methods: The Management Committee of SITE selected and gathered a multidisciplinary and multi-professional team, including experts in haemoglobinopathies and experts in endocrinopathies, who have been flanked by experts with methodological and organizational expertise, in order to formulate recommendations based on the available scientific evidence integrated by personal clinical experience. The project followed the systematic approach for the production of clinical practice guidelines according to the methodology suggested by the National Center for Clinical Excellence, Quality and Safety of Care (CNEC). Results: Out of 14 topics, 100 clinical questions were addressed, and 206 recommendations were elaborated on. The strength of recommendations, panel agree...

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    New Entity—Thalassemic Endocrine Disease: Major Beta-Thalassemia and Endocrine Involvement
    Cristina Vasiliu

    Diagnostics

    Beta-thalassemia (BTH), a recessively inherited haemoglobin (Hb) disorder, causes iron overload (IO), extra-medullary haematopoiesis and bone marrow expansion with major clinical impact. The main objective of this review is to address endocrine components (including aspects of reproductive health as fertility potential and pregnancy outcome) in major beta-thalassemia patients, a complex panel known as thalassemic endocrine disease (TED). We included English, full-text articles based on PubMed research (January 2017–June 2022). TED includes hypogonadism (hypoGn), anomalies of GH/IGF1 axes with growth retardation, hypothyroidism (hypoT), hypoparathyroidism (hypoPT), glucose profile anomalies, adrenal insufficiency, reduced bone mineral density (BMD), and deterioration of microarchitecture with increased fracture risk (FR). The prevalence of each ED varies with population, criteria of definition, etc. At least one out of every three to four children below the age of 12 y have one ED. E...

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