Mean Height Velocity Research Articles

Adult and Near-Adult Height in Patients with Severe Insulin-Like Growth Factor-I Deficiency after Long-Term Therapy with Recombinant Human Insulin-Like Growth Factor-I

Background: Treatment with recombinant human insulin-like growth factor-I (IGF-I) stimulates linear growth in children with severe IGF-I deficiency (IGFD). Aims: To evaluate the efficacy and safety of treatment with IGF-I in patients with severe IGFD treated until adult or near-adult height. Methods: Twenty-one children with severe IGFD were treated until adult or near-adult height under a predominantly open-label design. All patients were naive to IGF-I. Recombinant human IGF-I was administered subcutaneously in doses between 60 and 120 µg/kg twice daily. Nine patients received additional therapy with gonadotropin- releasing hormone (GnRH) analog for a mean period of 2.9 ± 1.8 years. Results: Mean duration of treatment was 10.0 years. Mean height velocity increased from 3.1 cm/year prior to treatment to 7.4 cm/year during the first year of treatment. Height velocities during the subsequent years were lower, but remained above baseline for up to 12 years. Cumulative mean Δ height SD score at (near) adult height was +2. The observed mean gain in height was 13.4 cm more than had been expected without treatment. The adult height achieved by the patients also treated with GnRH analog was not different from those who received IGF-I therapy alone. There were no new safety signals identified in these patients, a subset of those previously reported. Conclusion: Long-term therapy with IGF-I improves adult height of patients with severe IGFD. Most patients did not bring their heights into the normal adult range.

SAT0438 Catch-Up Growth During Tocilizumab Therapy for Systemic Juvenile Idiopathic Arthritis: 2-Year Data from a Phase 3 Clinical Trial

BackgroundSystemic juvenile idiopathic arthritis (sJIA), characterised by chronic arthritis associated with prominent systemic features, has a significant impact on the growing skeleton, resulting in impaired linear growth and systemic osteoporosis....

Efficacy and safety of LB03002, a once-weekly sustained-release human GH for 12-month treatment in Korean children with GH deficiency

The purpose of this study was to investigate the efficacy and safety of LB03002, a sustained-release human GH (SR-hGH), compared with that of daily rhGH for 12 months in children with GH deficiency (GHD). A total of 73 children with GHD were screened and 63 eligible subjects were randomized in a 1:1 ratio of LB03002 (SR-hGH) to daily rhGH treatment group. LB03002 was administered once weekly at a dose of 0.5 mg/kg while daily rhGH was administered for 6 consecutive days with equally divided doses to make a total of 0.21 mg/kg per week. Treatments were given for 12 months by s.c. injections. Injection site reactions and adverse events were investigated throughout the study period. The mean (S.D.) height velocity (HV) SHOWED a clinically significant increase after the 6-month treatment: 3.00 (1.15) cm/year at screening to 9.78 (1.98) cm/year at 6 months in the LB03002 group; 2.39 (1.63) cm/year at screening to 10.56 (2.65) cm/year at 6 months in the daily rhGH group. The increased HV at 12 months was still maintained in both the groups: 9.06 (1.63) cm/year at 12 months in the LB03002 group; 9.72 (2.32) cm/year at 12 months in the daily rhGH group. Most of the adverse drug reactions were mild and tolerable. No subjects were withdrawn due to adverse events. Weekly injection of LB03002 at a dose of 0.5 mg/kg per week was confirmed to have comparable efficacy to daily injection of rhGH at a dose of 0.21 mg/kg per week. Both formulations were well tolerated.

Longitudinal Standards for Height and Height Velocity in Korean Children and Adolescents: the Kangwha Cohort Study

Longitudinal standards for height and height velocity are essential to monitor for appropriate linear growth. We aimed to construct standards in Korean children and adolescents through the population-based longitudinal Kangwha study. Our study was a part of a community-based prospective cohort study from 1986 to 1999 with 800 school children. Height and height velocity were recorded annually from age 6 until final height. Results were compared with cross-sectional data from the 2007 Korean National Growth Charts. Final height was 173.5 cm in boys and 160.5 cm in girls. Although final height was similar between longitudinal and cross-sectional standards, the mean height for age was higher in the longitudinal standard by 1-4 cm from age 6 until the completion of puberty. Using the longitudinal standard, age at peak height velocity (PHV) was 12 in boys and 10 in girls; height velocity at PHV was 8.62 cm/yr in boys and 7.07 cm/yr in girls. The mean height velocity was less than 1 cm/yr at age 17 in boys and 15 in girls. Thus, we have presented the first report of longitudinal standards for height and height velocity in Korean children and adolescents by analyzing longitudinal data from the Kangwha cohort.

Variation Features of the Mindanao Eddy from Argo Data

Based on monthly mean Argo profiler float data from 2004 to 2008 and monthly mean sea surface height and current velocity data from the Simple Ocean Data Assimilation (SODA) reanalysis for the period 1958–2007, the three-dimensional structure of the Mindanao Eddy (ME), its seasonal and interannual variability, and its impact on the thermohaline structure are analyzed. In addition, the displacement of the ME centre and its relation to ocean circulations are also discussed. The results showed that the ME spreads vertically from about 600 db depth upward to about 40 db depth. In addition to its strong seasonal variation, the strength of the ME displays strong interannual variability resulting from the interannual variability in the North Equatorial Current (NEC), the North Equatorial Counter Current (NECC) and the Mindanao Current (MC). The meridional movement of the centre of the ME has strong seasonal and interannual variability, and the zonal movement has a strong seasonal variation. The meridional movement is associated with the meridional displacement of the interface between the NECC and the NEC, whereas the zonal movement is associated with the zonal displacement of the interface between the MC and the northward branch of the NECC. The ME variability can greatly affect the pattern of the thermohaline structure in the local upper ocean. When the eddy is strong, the cold and low salinity water within it moves vigorously upward from the deep layer; the thermocline shoals significantly; the subsurface high salinity water shows a large decrease; and the upper mixed layer becomes thinner, and vice versa. RÉSUMÉ [Traduit par la rédaction] En nous basant sur les données mensuelles moyennes des flotteurs profileurs Argo pour la période de 2004 à 2008 et sur les données mensuelles moyennes de hauteur de la surface de la mer et de vitesses vectorielles du courant fournies par les réanalyses SODA (Simple Ocean Data Assimilation) pour la période de 1958 à 2007, nous analysons la structure tridimensionnelle du remous de Mindanao, sa variabilité saisonnière et interannuelle et son impact sur la structure thermohaline. De plus, nous discutons du déplacement du centre du remous de Mindanao et de sa relation avec les circulations océaniques. Les résultats ont montré que le remous s'étend verticalement vers le haut à partir d'une profondeur de 600 db jusqu’à une profondeur d'environ 40 db. En plus de sa forte variabilité saisonnière, la force du remous de Mindanao affiche une forte variabilité interannuelle résultant de la variabilité interannuelle dans le courant nord-équatorial (NEC), le contre-courant nord-équatorial (NECC) et le courant de Mindanao (MC). Le mouvement méridien du centre du centre du remous de Mindanao affiche une forte variabilité saisonnière et interannuelle et le mouvement zonal possède une forte variabilité saisonnière. Le mouvement méridien est lié au déplacement méridien de l'interface entre le NECC et le NEC alors que le mouvement zonal est lié au déplacement zonal de l'interface entre le MC et le bras allant vers le nord du NECC. La variabilité du remous de Mindanao peut grandement modifier la configuration de la structure thermohaline dans les couches supérieures locales de l'océan. Quand le remous est fort, l'eau froide et de faible salinité qu'il renferme se déplace vigoureusement vers le haut à partir de la couche profonde; la thermocline s'élève de façon marquée; les eaux de subsurface de forte salinité affichent une diminution importante; et la couche de mélange supérieure devient plus mince, et inversement.

Treatment of growth failure with growth hormone in children with chronic kidney disease: an open-label long-term study

To assess long-term efficacy and safety of recombinant human growth hormone (GH) in children with chronic kidney disease (CKD). An open-label, international, multicenter study. Children with CKD and growth failure received GH (0.35 mg/kg/week). The primary efficacy endpoint was a significant change in height velocity (HV) and height standard deviation score (SDS) versus baseline after 12 months of treatment, extended to 24 months, then to 5 years. In total, 81 patients enrolled (CKD Stage 4 - 5 = 37, on dialysis = 27, post-transplant = 17). After 12 and 24 months of treatment, increases were seen in mean (SD) HV (4.6 (3.1) to 9.0 (3.6) cm/year and 4.5 (3.3) to 7.5 (2.9) cm/year, respectively; both p < 0.001), mean (SD) height SDS (-3.7 (1.7) to -3.0 (1.7) and -3.6 (1.5) to -2.5 (1.5), respectively; both p < 0.001) and mean (SD) HV SDS (-2.4 (2.5) to 3.8 (4.5) and -2.4 (2.2) to 1.1 (3.8), respectively; both p < 0.001). A normal height SDS was seen in 1% of children at baseline, 17% after 12 months and 43% after 24 months of treatment. Improvements were similar across CKD subgroups with the greatest improvements in CKD Stage 4 - 5. Among 31 patients who completed about 5 years of treatment, four reached final height. There was no undue bone age acceleration and no deterioration of kidney function. Ten adverse events were related to GH treatment. In this long-term study, GH treatment was associated with significant improvements in growth and height in children with CKD and growth failure, and was well tolerated.

First-year response to rhGH therapy in children with CKD: a National Cooperative Growth Study Report

A clear definition of the appropriate growth response during recombinant human growth hormone (rhGH) treatment has never been established in the pediatric chronic kidney disease (CKD) population. We present here data from Genentech's National Cooperative Growth Study (NCGS) on the first-year growth response in prepubertal children with CKD. Using NCGS data, we constructed response curves for the first year of rhGH therapy in 270 (186 males, 84 females) naïve-to-treatment, prepubertal children with CKD prior to transplant or dialysis. Data from both genders were combined because gender was not significantly related to height velocity (p = 0.51). Response to rhGH was expressed as height velocity (HV) in cm/year. Mean, mean + or - 1SD, and mean - 2SD for HV during the first year of rhGH treatment as well as pretreatment HV were plotted versus age. Age-specific HV plots for rhGH-treated children with CKD are presented. At all ages, the first-year mean HV was greater than the mean pretreatment HV. The mean - 2SD for HV in children on rhGH treatment was similar to the mean pretreatment HV. These growth plots will be useful to clinicians for assessing a patient's first-year growth response. We propose that a HV below the mean - 1SD is an inadequate response. These curves may help identify patients with a suboptimal growth response due to confounding medical factors and/or non-compliance.

Recombinant human growth hormone treatment in short children with renal disease: Our first experience

Growth retardation is a hallmark of chronic illnesses such as chronic kidney disease in children, and it is associated with increased morbidity and mortality. The growth hormone (GH) resistance observed in uraemia can be overcome by supraphysiological doses of exogenous GH. We would like to present our first results of recombinant human growth hormone (rhGH) treatment, mainly in children on haemodialysis. Sixteen children, aged 4.5-17.1 years (mean age 11.25 +/- 3.57) with height below -2.0 standard deviation score (SDS) for age or height velocity below -2.0 SDS for age, were selected to receive rhGH therapy at our Nephrology and Haemodialysis Department. Most of them were on haemodialysis (14 children) with mean spent time 2.88 +/- 2.68 years (0-9 years) before the initiation of rhGH therapy. One half of patients were prepubertal (8 children) and the second half were in early puberty (testicular volume between 4 and 8 ml for boys and breast development B2 or B3 in girls). All patients received 28-30 IU/m2 rhGH per week by daily subcutaneous injection. The year before rhGH therapy served as a control period. During the first year of treatment, mean height velocity in haemodialysis patients increased from 2.25 cm/year to 6.59 cm/year (p < 0.0001) and in the second year it was 5.25 cm/ year (p = 0.004). The mean height SDS in haemodialysis children did not improve significantly during the first year of rhGH treatment (from -3.01 SDS to -2.77 SDS, p = 0.063). Neither weight nor the body mass index varied compared with the pretreatment period. Two patients developed worsened secondary hyperparathyroidism and were excluded from the study, but the relationship with rhGH remains uncertain. Mean height velocity significantly improved during rhGH therapy in haemodialysis patients. No significant side-effects were observed in children during three-year treatment with GH.

Growth in children with cystic fibrosis‐related diabetes

Cystic fibrosis-related diabetes (CFRD) is associated with a shortened life expectancy and greater deterioration in lung function than in CF patients with normal glucose metabolism. There are few published data on how CFRD affects growth in childhood. We carried out a retrospective case controlled study of growth and lung function in 34 children with CFRD attending three specialist centers in London. We found that for the 2 years leading to CFRD diagnosis (at a mean age of 13.1 years), the mean height velocity was significantly less compared to controls: 4.9 (standard deviation-SD 1.6) cm/year vs. 6.0 (SD 1.9) cm/year (P = 0.005). For the 2 years following diagnosis, height velocity remained significantly lower (3.4 (SD 2.2) cm/year vs. 4.4 (SD 2.2) cm/year, P = 0.02). Mean FEV(1) was reduced prior to diagnosis and at diagnosis, but was similar to controls 2 years after diagnosis. This study highlights the compromise in height velocity and lung function that occurs prior to diagnosis of CFRD in children with CF, and a reduction in height velocity should be considered an indicator of impaired glucose metabolism. It would be useful to know whether early treatment with insulin can help promote catch up growth.

High Prevalence of Pituitary Magnetic Resonance Abnormalities and Gene Mutations in a Cohort of Brazilian Children with Growth Hormone Deficiency and Response to Treatment

Data were retrospectively collected from 69 Brazilian patients (45 boys) with growth hormone deficiency (GHD) who received exogenous growth hormone (GH) for a median duration of 4 years (range 1-13 years). Forty-two patients had multiple pituitary hormone deficiencies and 27 had isolated GHD. Peak GH was < 7 ng/ml (IRMA) or < 3.2 ng/ml (IFMA) after two stimulation tests. Therapy was started at median age of 10.0 years (range 2.2-21.6 years), bone age of 5.8 years (0.5-13.5 years) and height standard deviation score -4.4 (range -9.3 to -1.6). MRI revealed pituitary abnormalities in 87% of patients. Homozygous mutations in PROP-1, GHRH-R, GH-1 or HESX-1 genes were found in 12 patients. Mean height velocities were 3.3 pretreatment and 10.3, 7.8, 7.4 and 6.4 cm/yr, respectively, during 1-4 years of treatment with GH. In conclusion, the high prevalence (96%) of genetic and/or pituitary abnormalities probably reflects the stringent diagnostic criteria used, and GH replacement resulted in significant catch-up growth.

Efficacy and Safety of Growth Hormone Treatment in Children Born Small for Gestational Age in Japan

Growth-promoting effects and safety of growth hormone (GH) treatment in prepubertal short-statured children born small for gestational age (SGA) were evaluated in a multicenter, open-label, randomized parallel-group comparison study. Patients were randomized to two dose groups; 34 and 33 patients received GH at 0.033 and 0.067 mg/kg/day for one year, respectively. The increase of the mean height velocity standard deviation score (SDS) was significantly (p <0.0001) higher in the 0.067-mg group (from -1.4 to 4.7) than that in the 0.033-mg group (-1.9 to 2.6). A significant (p <0.0001) increase in the mean height SDS was established in the 0.067-mg group; increases of -3.1 to -2.5 vs -3.1 to -2.2 in the 0.033- and 0.067-mg groups, respectively. The trial was non-eventful. Oral glucose tolerance tests indicated a mostly normal pattern of plasma glucose before and after 12-month GH treatment. The growth-promoting effect was significantly higher with GH treatment at 0.067 mg/kg/day.

Effect of a gluten‐free diet on growth and small‐bowel histology in children with celiac disease in India

Follow-up studies on growth and histological recovery of children with celiac disease (CD) while on a gluten-free diet (GFD) are lacking from Asia. We therefore assessed the effects of this diet. Forty-two children with CD were enrolled. Weight and height were expressed as weight for height (WfH) and height standard deviation scores (HSDS), respectively. Twenty-five children had repeated duodenal biopsies after 1-2 years and 14 had a third biopsy after 3-7 years of GFD. Compliance was checked by regular interview and IgA antiendomysial antibody estimation (EMA). At diagnosis (n = 25), mean HSDS was -3.3 +/- 1.6 with 76% having a HSDS of <-2; 60% were undernourished (WfH mean 81.6 +/- 5.7). Over a mean follow up of 3.7 years, HSDS improved to -1.3 +/- 1.7 and 84% cases achieved normal nutrition. Mean height velocity was 13.9 cm during first year and 5.6 cm in subsequent years. Small-bowel biopsies at diagnosis showed subtotal villous atrophy (Marsh IIIb) in 18 (72%) and partial villous atrophy (Marsh IIIa) in seven (28%) patients. Repeat biopsy at 1-2 years showed shift from subtotal to partial villous atrophy in 94% (n = 17/18) and normalization in one patient. In patients with Marsh IIIa improvement of partial villous atrophy was observed in all. Immunoglobulin A endomysial antibody was negative in 81%. Repeat biopsies at 5 years of GFD showed improvement to Marsh I-II, but none normalized. The majority of children with CD show normalization of nutrition and growth after GFD. Small-bowel histology improves markedly but does not normalize even after 5 years of GFD.

Efficacy and safety of a new ready-to-use recombinant human growth hormone solution

We report 24-month interim results of two multicenter phase III studies in previously untreated children with growth failure secondary to GH deficiency (GHD) that were paramount to the development of a new recombinant human GH (rh- GH, somatropin), approved as the first 'biosimilar' in Europe. Study 1 consisted of 3 parts performed in 89 children. The objective was to compare efficacy and safety of the lyophilized formulation of the new somatropin [Somatropin Powder (Sandoz)] with a licensed reference rhGH preparation and the liquid formulation of the new somatropin [Somatropin Solution (Sandoz)] and to assess long-term efficacy and safety of this ready-to-use Somatropin Solution. Study 2 was performed in 51 children and designed to demonstrate efficacy and safety of Somatropin Powder and to confirm its low immunogenic potential; rhGH was given sc at a daily dose of 0.03 mg/kg. Primary [body height, height SD score (HSDS), height velocity, and height velocity (HV) SD score (HVSDS)] and secondary [IGF-I and IGF binding protein 3 (IGFBP-3)] efficacy endpoints and safety parameters were assessed regularly. In study 1, all treatments showed comparable increases in growth. The baseline-adjusted difference between Somatropin Powder and the reference rhGH product in mean HV was -0.20 cm/yr (95% confidence interval (CI) [-1.34;0.94]) and in mean HVSDS was 0.76 (95% CI [-0.57;2.10]) after 9 months. These very small differences demonstrate comparable therapeutic efficacy between the two treatments. The results of study 2 were consistent with those seen in study 1. Equivalent therapeutic efficacy and clinical comparability in terms of safety and immunogenicity between Somatropin Powder and the reference rhGH product and between Somatropin Powder and Somatropin Solution was demonstrated. The safety and immunogenicity profiles were similar and as expected from experience with rhGH preparations.

Early Recombinant Human Growth Hormone Treatment in Glucocorticoid-Treated Children with Juvenile Idiopathic Arthritis: A 3-Year Randomized Study

Long-term glucocorticoid therapy adversely affects growth and body composition in children with juvenile idiopathic arthritis (JIA). In previous studies, recombinant human GH (rhGH) halted the progression of these complications without inducing catch-up growth. The objective of the study was to evaluate the impact on growth and body composition of rhGH started early after glucocorticoid initiation and to record adverse effects in children with JIA. This is a 3-yr randomized controlled study. This study was conducted in a teaching hospital. Thirty children, 12-15 months into glucocorticoid therapy for severe JIA, were enrolled. Patients received rhGH (0.46 mg/kg.wk) in daily sc injections (n = 15) or no rhGH therapy (n = 15) for 3 yr. Difference in height sd score (SDS) change between the two groups was assessed. Height velocity, body composition, and oral glucose tolerance were evaluated yearly. Mean height SDS increase was larger with rhGH (+0.37 +/- 1.5 SDS) than without (-0.96 +/- 1.2 SDS) (P = 0.04). Mean height velocity returned to normal within the first year of rhGH treatment and remained normal thereafter. Mean lean mass increase was greater with rhGH treatment (+7.3 +/- 2.9 kg vs. +4.4 +/- 2.8 kg; P = 0.03). Fat mass and bone mineralization were not significantly different in the two groups. Fasting serum insulin increased significantly in rhGH-treated patients (5.2 +/- 16 mIU/liter) compared with untreated controls (-2.3 +/- 5 mIU/liter) (P = 0.04); fasting glycemia was unchanged. rhGH started early in the course of JIA preserved normal growth velocity and height. Although rhGH was well tolerated, carbohydrate metabolism should be monitored closely.

Catch-up growth in autosomal dominant isolated growth hormone deficiency (IGHD type II)

Objective Data on the GH-induced catch-up growth of severely GH-deficient children affected by monogenetic defects are missing. Patients Catch-up growth of 21 prepubertal children (6 females, 15 males) affected with IGHD type II was analyzed in a retrospective chart review. At start of therapy, mean age was 6.2 years (range, 1.6–15.0), mean height SDS was −4.7 (−7.6 to −2.2), mean IGF-I SDS was −6.2 (−10.1 to −2.2). GH was substituted using a mean dose of 30.5 μg/kg*d. Results Catch-up growth was characterized by a mean height gain of +0.92, +0.82, and +0.61 SDS after 1, 2, and 3 years of GH therapy, respectively. Mean height velocities were 10.7, 9.2 and 7.7 cm/year during the first three years. Mean duration of complete catch-up growth was 6 years (3–9). Mean height SDS reached was −0.97 (−2.3 to +1.1), which was within the range of the estimated target height of −0.60 SDS (−1.20 to −0.15). The younger and shorter the children were at start of therapy the better they grew during the first year independent of the dose. Mean bone age was delayed at start by 2.1 years and progressed by 2.5 years during the first two years of therapy. Incomplete catch-up growth was caused by late initiation or irregular administration of GH in four cases. Conclusions Our data suggest that GH-treated children with severe IGHD show a sustained catch-up growth over 6 years (mean) and reach their target height range. This response to GH is considered to be characteristic for young children with severe growth retardation due to IGHD.

The correlation of the IGF-I, IGFBP-3, and ALS generation test to height velocity after 6 months of recombinant growth hormone therapy in girls with Turner syndrome

Background/aims Girls with Turner syndrome (TS) have short stature and benefit from growth hormone therapy (hGH). Some TS present a significant change in height velocity in response to hGH while others have only a mild increment. Our objective was to correlate the response to hGH (height velocity after 6 months of therapy) to biochemical data prior to and after the beginning of hGH to try to define a tool to predict the response to hGH. Methods Thirteen TS participated in the study (ages 3.5–14 years). Levels of IGF-I and IGFBP-3 were measured before and 5, 30 and 90 days after starting hGH (0.05 mg/kg/day), ALS levels were measured only prior and after 5 days. Results The mean height velocity (±SD) increased from 4.27 (±1.18) cm/year to 8.46 (±2.17) cm/year ( p = 0.0001). There was no correlation between the height velocity encountered and the expected height velocity using published mathematical models. Basal ALS values correlated to height velocity SDS and IGF-I and IGFBP-3, after 90 days, correlated to height velocity. Most of the data was too scattered to be used individually for each patient. Conclusions Even though we observed a relationship between biochemical markers and height velocity in TS treated with hGH, the response to hGH therapy in this condition is highly variable.

Growth Hormone Effects on Adult Height in Idiopathic Short Stature

Growth Hormone Effects on Adult Height in Idiopathic Short Stature

Effect of renal transplantation in childhood on longitudinal growth and adult height

Severe growth failure is frequently observed in children suffering from end-stage renal disease (ESRD). We analyzed the effect of renal transplantation (RTx) on longitudinal growth and final height in 37 children (19 girls) with ESRD with a mean follow-up of 8.5 years. The mean age at RTx was 11.3 years. In children transplanted before start of puberty, mean height velocity increased significantly from 4.9 to 8.0 cm/year (P < 0.01), resulting in an increase in standardized height of 0.6 SD within two years post RTx. Although peak height velocity during puberty was significantly increased compared with healthy children, total pubertal height gain was reduced by 20% because of its shortened duration. Mean standardized height significantly increased from the time of RTx until final height by 1.3 SD and 0.7 SD in children transplanted before and after start of puberty, respectively. Mean adult height (boys 170 cm; girls 151 cm) was normal (> -2 SD) in 68% of patients. Change in standardized height from RTx until adult height was associated with initial degree of stunting and glomerular filtration rate (GFR; cumulative r2 0.49). Total pubertal height gain was associated with the age at start of puberty, GFR, and age at RTx (cumulative r2 0.57). RTx in children with ESRD induces moderate catch-up growth during the prepubertal growth period. However, final height is reduced in about one third of patients due to the reduced pubertal height gain and preexisting height deficit at the time of RTx.

Growth and Metabolic Control during Puberty in Girls with X-Linked Hypophosphataemic Rickets

Objective: X-linked hypophosphataemic rickets (XLH) results in defective bone mineralization and impaired growth. Treatment with oral phosphate (Pi) and calcitriol improves but does not normalize growth. This study assessed whether pubertal growth and metabolic control contribute to the height deficit. Methods: Study included patients with XLH who were treated with Pi-calcitriol from diagnosis to adult height; their hospital records, biochemistry and radiographs were reviewed. Results: Six females with XLH were included. Their mean peak height velocity and total height gain during puberty were nearly normal despite deteriorating metabolic control. Conclusions: In treated girls with XLH, the pubertal growth is nearly normal despite suboptimal metabolic control. The major height loss occurs prior to puberty and is not recovered during the pubertal growth spurt.

Persistent short stature, other potential outcomes, and the effect of growth hormone treatment in children who are born small for gestational age.

Persistent short stature, other potential outcomes, and the effect of growth hormone treatment in children who are born small for gestational age.