Prevention of Growth Failure in Turner Syndrome: Long-Term Results of Early Growth Hormone Treatment in the “Toddler Turner” Cohort

2021 ◽  
pp. 1-18
Author(s):  
Charmian A. Quigley ◽  
Patricia Y. Fechner ◽  
Mitchell E. Geffner ◽  
Erica A. Eugster ◽  
Judith L. Ross ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Pubertal Development ◽  
Standard Deviation Score ◽  
Growth Failure ◽  
Long Term Results ◽  
Extension Study ◽  
Normal Range ◽  
Gh Treatment ◽  
Catch Up

<b><i>Introduction:</i></b> In the randomized “Toddler Turner” study, girls who received growth hormone (GH) starting at ages 9 months to 4 years (early-treated [ET] group) had marked catch-up growth and were 1.6 ± 0.6 SD taller than untreated (early-untreated [EUT]) control girls after 2 years. However, whether the early catch-up growth would result in greater near-adult height (NAH) was unknown. Therefore, this extension study examined the long-term effects of toddler-age GH treatment on height, pubertal development, and safety parameters. <b><i>Methods:</i></b> Toddler Turner study participants were invited to enroll in a 10-year observational extension study for annual assessments of growth, pubertal status, and safety during long-term GH treatment to NAH for both ET and EUT groups. <b><i>Results:</i></b> The ET group was taller than the EUT group at all time points from preschool to maturity and was significantly taller at the onset of puberty (<i>p</i> = 0.016), however, the difference was not significant at NAH. For the full cohort (ET + EUT combined, <i>n</i> = 50) mean (± SD) NAH was 151.2 ± 7.1 cm at age 15.0 ± 1.3 years. NAH standard deviation score (SDS) was within the normal range (&#x3e;−2.0) for 76% of ET and 60% of EUT subjects (68% overall) and correlated strongly with height SDS at GH start (<i>r</i> = 0.78; <i>p</i> &#x3c; 0.01), which in turn had a modest inverse correlation with age at GH start (i.e., height SDS declined with increasing age in untreated girls [<i>r</i> = −0.30; <i>p</i> = 0.016]). No new safety concerns arose. <b><i>Conclusion:</i></b> Although the ET group was taller throughout, height SDS at NAH was not significantly different between groups due to catch-down growth of ET girls during lapses in GH treatment after the Toddler study and similar long-term GH exposure overall. Early initiation of GH by age 6 years, followed by uninterrupted treatment during childhood, can prevent ongoing growth failure and enable attainment of height within the normal range during childhood, adolescence, and adulthood.

Efficacy of long-term growth hormone therapy in short non-growth hormone-deficient children

2017 ◽  
Vol 30 (2) ◽  
Author(s):  
Lucia Schena ◽  
Cristina Meazza ◽  
Sara Pagani ◽  
Valeria Paganelli ◽  
Elena Bozzola ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Final Height ◽  
Standard Deviation Score ◽  
Bone Age ◽  
Gh Treatment ◽  
Short Children ◽  
Igf I ◽  
Height Gain ◽  
The Cost

AbstractBackground:In recent years, several studies have been published showing different responses to growth hormone (GH) treatment in idiopathic short stature children. The aim of the present study was to investigate whether non-growth-hormone-deficient (non-GHD) short children could benefit from long-term GH treatment as GHD patients.Methods:We enrolled 22 prepubertal children and 22 age- and sex-matched GHD patients, with comparable height, body mass index (BMI), bone age, and insulin-like growth factor 1 (IGF-I) circulating levels. The patients were treated with recombinant human GH (rhGH) and followed until they reach adult height.Results:During GH treatment, the two groups grew in parallel, reaching the same final height-standard deviation score (SDS) and the same height gain. On the contrary, we found significantly lower IGF-I serum concentrations in non-GHD patients than in GHD ones, at the end of therapy (p=0.0055).Conclusions:In our study, the response to GH treatment in short non-GHD patients proved to be similar to that in GHD ones. However, a careful selection of short non-GHD children to be treated with GH would better justify the cost of long-term GH therapy.

scholarly journals Long-Term Effectiveness and Safety of Childhood Growth Hormone Treatment in Noonan Syndrome

2020 ◽  
Vol 93 (6) ◽  
pp. 380-395
Author(s):  
Tilman R. Rohrer ◽  
Jennifer Abuzzahab ◽  
Philippe Backeljauw ◽  
Anna Camilla Birkegård ◽  
Joanne Blair ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Adverse Events ◽  
Noonan Syndrome ◽  
Standard Deviation Score ◽  
Hormone Treatment ◽  
Gh Treatment ◽  
Serious Adverse Events ◽  
Height Gain ◽  
The Mean

<b><i>Introduction:</i></b> Few data exist on long-term growth hormone (GH) treatment in patients with Noonan syndrome (NS). <b><i>Objective:</i></b> To evaluate the effectiveness and safety of GH treatment in NS in clinical practice. <b><i>Methods:</i></b> Height gain, near-adult height (NAH), and safety were assessed in 2 complementary non-interventional studies: NordiNet® IOS and ANSWER. The safety analysis included 412 patients, and the effectiveness analysis included 84 GH-treated patients (male, <i>n</i> = 67) with ≥4 years’ height standard deviation score (HSDS) data. HSDS was determined using national reference (NR) and NS-specific (NSS) data. <b><i>Results:</i></b> The mean (SD) baseline age was 8.38 (3.57) years; HSDS, −2.76 (1.03); GH dose, 41.6 (11.1) µg/kg/day. The mean (SD) HSDS increase from baseline (ΔHSDS) was 0.49 (0.37) (first year), 0.79 (0.58) (second year), and 1.01 (0.60) (third year) (NR). The mean (SD) HSDS at year 3 was −1.66 (1.00) (NR; 1.06 [1.12] [NSS]). Twenty-four patients achieved NAH. The mean (SD) NAH SDS (NR) was −1.51 (0.60) (154.90 [3.21] cm) in females and −1.79 (1.09) (165.61 [7.19] cm) in males; 70.8% (17/24) had NAH SDS ≥ −2. Adverse drug reactions and GH-unrelated serious adverse events (<i>n</i> = 34) were reported in 22/412 (5.3%) patients. Four neoplasms and 3 cases of scoliosis were reported; no cardiovascular adverse events occurred. <b><i>Conclusions:</i></b> GH-treated children with NS achieved substantial height gain during the first 3 years of follow-up. Overall, 24 patients achieved NAH, with 70.8% having NAH SDS ≥ –2. There was no evidence to support a higher prevalence of neoplasm, or cardiac or other comorbidities.

scholarly journals Long-term results of GH therapy in GH-deficient children treated before 1 year of age

1999 ◽  
pp. 29-34 ◽  
Author(s):  
F Huet ◽  
JC Carel ◽  
JL Nivelon ◽  
JL Chaussain
Keyword(s):  
Clinical Presentation ◽  
Gh Deficiency ◽  
Growth Parameters ◽  
Long Term Results ◽  
Long Term Effects ◽  
Target Height ◽  
Gh Therapy ◽  
Gh Treatment ◽  
Catch Up

OBJECTIVES: To evaluate the long-term effects of GH therapy in early diagnosed GH-deficient patients treated before 1 year of age. Study design: We studied all 59 patients (33 males) recorded by Association France-Hypophyse and treated with GH (0.50+/-0.15 IU/kg (S.D.) per week) before 1 year of age. Clinical presentation and growth parameters under GH treatment were analyzed. RESULTS: Neonatal manifestations of hypopituitarism were frequent: hypoglycemia (n=50), jaundice (n=25) and micropenis (n=17/33). Although birth length was moderately reduced (-0.9+/-1.4), growth retardation at diagnosis (5.8+/-3.8 months) was severe (-3.5+/-1.9 standard deviation scores (SDS)). Fifty patients (85%) had thyrotropin and/or corticotropin deficiency. After a mean duration of GH therapy of 8.0+/-3.6 years, change in height SDS was +3.11+/-2.06 S.D., exceeding 4 SDS in 19 patients. Only 9 patients (15%) did not reach a height of -2 S.D. for chronological age and 20 patients (34%) exceeded their target height. Pretreatment height SDS was independently associated with total catch-up growth. CONCLUSION: Conventional doses of GH allow normalization of height in patients with early GH deficiency and treatment.

Long Term Experience with Growth Hormone Treatment in Children with Chronic Renal Failure

1999 ◽  
Vol 19 (2_suppl) ◽  
pp. 467-472 ◽  
Author(s):  
Franz Schaefer ◽  
Dieter Haffner ◽  
Elke Wühl ◽  
Otto Mehls
Keyword(s):  
Growth Hormone ◽  
Renal Failure ◽  
Chronic Renal Failure ◽  
Final Height ◽  
Recombinant Human Growth Hormone ◽  
Growth Failure ◽  
Skeletal Maturation ◽  
Height Velocity ◽  
Benign Intracranial Hypertension

After a decade of experience with recombinant human growth hormone (rhGH) in children with chronic renal failure (CRF), the long-term efficacy and safety of the drug is now established. In prepubertal children, partial catch-up growth is achieved during the first three treatment years, followed by sustained percentile-parallel growth. Discontinuation of rhGH treatment results in catch-down growth in 75% of patients. Treatment efficacy is inversely correlated with age and baseline height velocity, and positively influenced by genetic target height and residual renal function. Skeletal maturation is not accelerated, suggesting a true increase in final height potential. Side effects are limited to a stimulation of insulin secretion, which is not associated with changes in glucose tolerance, and occasional cases of benign intracranial hypertension. In summary, the advent of rhGH has opened a new era in the management of growth failure in CRF. Available evidence suggests that treatment should start in early childhood and early in the course of renal failure, and should be continued at least until renal transplantation. It remains to be seen whether the beneficial effect of rhGH on height observed during the prepubertal period will result in an eventual increase in adult height.

Safety and effectiveness of growth hormone therapy in infants with Prader-Willi syndrome younger than 2 years: a prospective study

2019 ◽  
Vol 32 (8) ◽  
pp. 879-884 ◽  
Author(s):  
Raquel Corripio ◽  
Carla Tubau ◽  
Laura Calvo ◽  
Carme Brun ◽  
Núria Capdevila ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Prospective Study ◽  
Mixed Model ◽  
Standard Deviation Score ◽  
Uniparental Disomy ◽  
Chromosome 15 ◽  
Prader Willi Syndrome ◽  
Gh Treatment ◽  
Maternal Uniparental Disomy ◽  
A Prospective Study

Abstract Background There is little evidence of the effects of early treatment with growth hormone (GH) in infants with Prader-Willi syndrome (PWS). A prospective study was conducted to assess the safety of GH therapy in infants younger than 2 years of age with PWS. Methods A total of 14 patients with PWS started treatment with GH under the age of 2 years and were followed over a 2-year period. A deletion of chromosome 15 was present in nine infants (64.3%) and maternal uniparental disomy 15 in five infants (35.7%). The median age at start of GH treatment was 9.6 months (interquartile range [IQR] 9.0–18.3 months). Changes in height standard deviation score (SDS), body mass index (BMI) SDS and subcapsular and tricipital skinfolds in the follow-up period were evaluated with a mixed-model regression analysis using the Package R. Results There were no fatal adverse events. A significant decrease (p < 0.001) in tricipital and subcapsular skinfold thickness, with an upward trend of height SDS and a downward trend of BMI SDS, was observed. Infants who started GH before 15 months of age started walking at a median of 18.0 [17.0–19.5] months vs. 36.6 [36.3–37.8] months for those who began treatment with GH after 15 months of age (p = 0.024). Conclusions GH treatment in infants with PWS less than 2 years of age is safe and improved body composition. Infants who received GH before the age of 15 months started to walk earlier.

scholarly journals Still too little, too late? Ten years of growth hormone therapy baseline data from the NordiNet® International Outcome Study

2018 ◽  
Vol 31 (5) ◽  
pp. 521-532 ◽  
Author(s):  
Michel Polak ◽  
Daniel Konrad ◽  
Birgitte Tønnes Pedersen ◽  
Gediminas Puras ◽  
Marta Šnajderová
Keyword(s):  
Growth Hormone ◽  
Czech Republic ◽  
Standard Deviation Score ◽  
Outcome Study ◽  
Gh Treatment ◽  
The Czech Republic ◽  
Serbia And Montenegro ◽  
The Mean ◽  
Late Start ◽  
The Uk

AbstractBackground:We investigated time trends in age, gender, growth hormone (GH) dose and height standard deviation score (SDS) in children with GH deficiency (GHD), born small for gestational age (SGA) or with Turner syndrome (TS) starting GH treatment.Methods:Data were obtained from children enrolled in the NordiNet®International Outcome Study (IOS) between 2006 and 2015 in the Czech Republic, France, Germany, Serbia and Montenegro (all indications), and Switzerland and the UK (GHD only). Trends were analyzed by linear regression. Patients were divided by age into early-, medium- or late-start groups in three different time periods.Results:Approximately one-third of children starting treatment for GHD were girls, with no apparent increase in proportion over time. The mean baseline age for starting treatment decreased significantly (p<0.001) for both GHD and SGA in the Czech Republic and Germany. In the other countries studied, over 40% of children started treatment for GHD and SGA late (girls >10, boys >11 years) between 2013 and 2015. The mean baseline GH doses were largely within recommended ranges for GHD and SGA, but below the lowest recommended starting dose for TS in almost every year since 2011 except in France.Conclusions:Approximately one-third of children starting treatment for GHD were girls. Between 2013 and 2015, more than 40% of children started treatment for GHD and SGA late except in Germany and the Czech Republic. TS patients received below-recommended doses. These results highlight the need for earlier identification of short stature in children, particularly girls, and for dose optimization in TS.

Long-term results of gamma knife surgery for growth hormone—producing pituitary adenoma: is the disease difficult to cure?

2005 ◽  
Vol 102 (Special_Supplement) ◽  
pp. 119-123 ◽  
Author(s):  
Tatsuya Kobayashi ◽  
Yoshimasa Mori ◽  
Yukio Uchiyama ◽  
Yoshihisa Kida ◽  
Shigeru Fujitani
Keyword(s):  
Growth Hormone ◽  
Gamma Knife ◽  
Pituitary Adenomas ◽  
Gamma Knife Surgery ◽  
Long Term Results ◽  
Content Type ◽  
The Mean ◽  
Dose Radiation ◽  
Fine Print

Object. The authors conducted a study to determine the long-term results of gamma knife surgery for residual or recurrent growth hormine (GH)—producing pituitary adenomas and to compare the results with those after treatment of other pituitary adenomas. Methods. The series consisted of 67 patients. The mean tumor diameter was 19.2 mm and volume was 5.4 cm3. The mean maximum dose was 35.3 Gy and the mean margin dose was 18.9 Gy. The mean follow-up duration was 63.3 months (range 13–142 months). The tumor resolution rate was 2%, the response rate 68.3%, and the control rate 100%. Growth hormone normalization (GH < 1.0 ng/ml) was found in 4.8%, nearly normal (< 2.0 ng/ml) in 11.9%, significantly decreased (< 5.0 ng/ml) in 23.8%, decreased in 21.4%, unchanged in 21.4%, and increased in 16.7%. Serum insulin-like growth factor (IGF)—1 was significantly decreased (IGF-1 < 400 ng/ml) in 40.7%, decreased in 29.6%, unchanged in 18.5%, and increased in 11.1%, which was almost parallel to the GH changes. Conclusions. Gamma knife surgery was effective and safe for the control of tumors; however, normalization of GH and IGF-1 secretion was difficult to achieve in cases with large tumors and low-dose radiation. Gamma knife radiosurgery is thus indicated for small tumors after surgery or medication therapy when a relatively high-dose radiation is required.

scholarly journals Long-Term Effect of Growth Hormone (GH) Treatment on Body Composition in Children with GH Deficiency

1999 ◽  
Vol 46 (Suppl) ◽  
pp. S35-S38 ◽  
Author(s):  
RYUICHI KUROMARU ◽  
HITOSHI KOHNO ◽  
NAMI UEYAMA ◽  
HASSAN MOHAMED SALAMA HASSAN ◽  
SUNAO HONDA ◽  
...  
Keyword(s):  
Growth Hormone ◽  
Body Composition ◽  
Gh Deficiency ◽  
Term Effect ◽  
Gh Treatment ◽  
Long Term Effect

Growth Hormone Therapy in Children with Kabuki Syndrome: 1-year Treatment Results

2017 ◽  
Vol 88 (3-4) ◽  
pp. 258-264 ◽  
Author(s):  
Dina A. Schott ◽  
Willem J.M. Gerver ◽  
Constance T.R.M. Stumpel
Keyword(s):  
Growth Hormone ◽  
Gh Deficiency ◽  
Standard Deviation Score ◽  
Height Velocity ◽  
Kabuki Syndrome ◽  
Body Proportions ◽  
Leg Length ◽  
Gh Treatment ◽  
Igf I ◽  
Rhgh Treatment

Background/Aims: Kabuki syndrome (KS) is a rare genetic malformation syndrome, resulting in characteristic features such as short stature. We investigate whether growth hormone (GH) treatment increases linear height and influences body proportions in KS children. Methods: In this prospective study, 18 genetically confirmed prepubertal KS children (9 females and 9 males) aged from 3.8 to 10.1 years (mean 6.8 ± 2.1 years) were treated with recombinant human GH (rhGH) for 1 year. Calculations for height, height velocity, BMI, sitting height, and subischial leg length were made. Bone age, insulin-like growth factor (IGF-I), and IGF binding protein 3 (IGFBP-3) were also measured. Results: This study showed an increase in height standard deviation score (SDS) for the whole group from –2.40 to –1.69 (p < 0.05) after 1 year of rhGH treatment. The change in height SDS within 1 year was >0.7 SDS for 10 subjects and >0.5 SDS for 3 subjects. The mean IGF-I SDS at the start of the study was –0.70 (±1.07), which increased after 12 months to 1.41 (±0.91) (p < 0.05). KS children who received rhGH at a younger age displayed significantly greater increases in height than those who started when they were older. The same was true for both gene mutation KMT2D versus KDM6A and for GH deficiency versus non-GH deficiency KS children (p < 0.05). Throughout the course of rhGH treatment, the subjects’ body proportions remained normal. Conclusions: All participants experienced catch-up growth during the year of rhGH treatment, but without an influence on body proportions.

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