Median Height Z-score Research Articles

Comparison of clinical features of cystic fibrosis patients eligible but not on CFTR modulators to ineligible for CFTR modulators.

Cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs target the underlying defect and improve CFTR function. They are a part of standard care in many countries, but not all patients are eligible for these drugs due to age and genotype. Here, we aimed to determine the characteristics of non-eligible patients for CFTR modulators in the CF registry of Turkey (CFRT) to highlight their clinical needs. This retrospective cohort study included CF patient data from the CFRT in 2021. The decision of eligibility for the CFTR modulator was determined according to the 'Vertex treatment-Finder' on the Vertex® website. Demographic and clinical characteristics of patients were compared between eligible (group 1) and ineligible (group 2) groups for CFTR modulators. Among the study population (N = 1527), 873 (57.2%) were in group 1 and 654 (42.8%) were in group 2. There was no statistical difference between groups regarding sex, meconium ileus history, diagnoses via newborn screening, FEV1 z-score, CF-associated complications, organ transplant history, and death. Patients in group 2 had a higher incidence of pancreatic insufficiency (87.7% vs. 83.2%, p = .010), lower median height z-scores (-0.87 vs. -0.55, p < .001), lower median body mass index z-scores (-0.65 vs. -0.50, p < .001), longer days receiving antibiotics due to pulmonary exacerbation (0 [interquartile range, IQR: 0-2] vs. 0 [IQR: 0-7], p = 0.001), and more non-invasive ventilation support (2.6% vs. 0.9%, p = 0.008) than patients in group 1. The ineligible group had worse clinical outcomes than the eligible group. This highlights their need for life-changing drugs to improve clinical outcomes.

Impact of ketogenic diet therapy on growth in children with epilepsy

RationaleKetogenic diet therapy (KDT) is an effective treatment for drug-resistant epilepsy in children. There is conflicting evidence regarding the impact of KDT on growth. We aimed to determine whether linear growth and weight were affected in children who followed KDT in the UK, and to explore potential associations with clinical or demographic factors with impacted growth. MethodsA retrospective review of medical records of children with epilepsy following KDT at 3 UK centres was conducted. Height and weight measurements taken as part of routine clinical management were recorded at baseline, 1–8 years on diet, and 1-year post-diet. Measurements were converted into z-scores, and the differences from baseline analysed using Wilcoxon Signed Rank tests. Potential associations of impacted growth with feeding method, ambulatory status, diet type, age at diet onset and average daily protein intake were investigated using Mann-Whitney, Kruskal-Wallis tests or Spearman’s Rank correlation. Results265 individuals were included, of which 84 had post-diet data available. Median height z-score significantly decreased at 1- (n = 139, p = .018), 2- (n = 86, p < .0005) and 3 years (n = 27, p = .001) on diet. There was no significant change to height or weight z-score 1-year post-diet discontinuation. Median weight z-score significantly decreased from baseline at 4 years (n = 15, p = .020), and 6 years (n = 8, p = .025) on diet, but not at other time points. There was greater height z-score decrease in non-ambulatory children at 2 years (p = .009), in those following a classical diet compared with the modified ketogenic diet at 2 years (p = .006) and amongst younger children at 2 years (n = 86, p < .005) and 3 years (n = 27, p = .008) on diet. No significant differences were found in weight z-score change across any subgroup, following Bonferroni correction for multiple testing. ConclusionsMedian linear growth was significantly adversely affected for the first 3 years on KDT but catch-up growth post diet discontinuation was observed. Non-ambulatory children, younger children, and individuals following a classical diet may be more vulnerable to impacted growth when on KDT, although this was not consistent across all time points. The potential short-term impact on linear growth should be discussed with individuals considering KDT, and monitored closely.

VP.14 Growth patterns in treated SMA children in the UK

Spinal muscular atrophy (SMA) is a neuromuscular disorder characterized by degeneration of alpha motor neurons in the spinal cord. SMA type 1 (SMA1) is the most severe form, with patients never achieving sitting, whilst patients with SMA type 2 (SMA2) are sitters who are not able to walk independently. Disease modifying treatments such as nusinersen, onasemnogene abeparvovec, and risdiplam have led to increases in motor function and life expectancy. However, not much is known about the effect of these treatments on growth. The aim of this retrospective study was to analyse the patterns in weight, height, and BMI of treated SMA1 and SMA2 patients compared with the UK90 reference population. Cross-sectional data on growth was obtained from the SMA Research and Clinical Hub UK (SMA REACH UK) national database. Results are based on last available measurements of SMA1 (n=57) and SMA2 (n=41) nusinersen treated children. Median age was 3.02 (IQR: 1.44-4.78), and 6.93 (IQR: 4.73-8.81) years for SMA1 and SMA2, respectively. The results showed that compared to the paediatric normative data, SMA1 and SMA2 had a lower weight with the median weight z-score of -0.96 (IQR: -1.97; -0.16) and -1.20 (IQR: -2.02; 0.16), respectively. SMA2 children presented with a lower median height z-score (-1.37 (IQR: -2.19; -0.22)), compared to those with SMA1 (-0.91 (IQR: -2.28; 0.83)). Consequently, median BMI z-score was -0.90 (IQR: -2.82; 0.80) for SMA 1, and -0.63 (IQR: -1.46; 1.45) for SMA2 patients. Approximately a quarter of the children in this group are considered underweight (Weight SD score <=-2) for both SMA1 (25%) and SMA2 (27%). Children with SMA2 are more likely to be overweight (BMI SD score >=2) than patients with SMA1, with rates of 15% and 4% respectively. This analysis sheds light on how growth is affected by novel disease modifying treatments in SMA.

Early corticosteroid withdrawal is associated with improved adult height in pediatric kidney transplant recipients.

Catch-up growth after pediatric kidney transplantation (kTx) is usually insufficient to reach normal adult height. We aimed to analyze the effect of pre-transplant recombinant human growth hormone (rhGH) and corticosteroid withdrawal on linear growth in the first year after kidney transplantation and identify factors associated with final height (FH). Patients who underwent kTx between 1996 and 2018 at below 18years old in five Belgian and Dutch centers were included. We analyzed the differences between height Z-scores at kTx and 1year post-transplant (Δ height Z-score) in children with and without corticosteroids at 1year (CS + /CS -) and with and without rhGH treatment before kTx (rhGH + /rhGH -). Univariable and multivariable linear regression analysis was applied to identify factors associated with height Z-score at 1year post-kTx, Δ height Z-score, and FH Z-score. A total of 177 patients were included, with median age 9.3years at kTx. Median height Z-scores pre-kTx and 1year later in the CS - /rhGH - , CS + /rhGH - , CS - /rhGH + , and CS + /rhGH + groups were - 1.42/ - 0.80, - 0.90/ - 0.62, - 1.35/ - 1.20, and - 1.30/ - 1.60 (p = 0.001). CS use 1year post-kTx was the only factor associated with Δ height (p = 0.003) on multivariable analysis. CS use at 1year was the only variable associated with FH (p = 0.014) in children with pre-transplant height Z-score below - 1 (n = 52). Increase in height Z-score in the first year post-kTx was highest in the CS - /rhGH - group and lowest in the CS + /rhGH + group. The use of corticosteroids at 1year post-kTx is associated with catch-up growth and in children with pre-transplant height Z-score below - 1 also with final height. A higher resolution version of the Graphical abstract is available as Supplementary information.

Nutrient intake in school-aged children with food allergies: a case-control study

Most studies assessed nutrient intake of young children with food allergy (FA) compared to healthy children. We aimed to compare macro- and micronutrient intake of school-aged children with FA to non-allergic children. This case-control study included 93 Italian children (52 with FA and 41 controls, median age 7.5 and 8.3 years, respectively). Macro- and micronutrient intake was assessed by a three-day food dietary record. Anthropometric measurements were also collected. The median height z-score was significantly lower in the FA group, despite a similar daily energy and protein intake. Calcium, iron and vitamin D intake was suboptimal in both groups, while protein intake was higher than recommended in both groups. Unexpectedly, children with FA consume more protein than controls, while having lower micronutrient intake, especially calcium. Our data suggest the importance of nutritional counseling for children with FA to ensure a balanced nutrient intake while on elimination diet.

Racial/Ethnic Disparities in the Investigation and Treatment of Pediatric Growth Hormone Deficiency

Introduction: In the United States, non-Hispanic white (NHW) children are disproportionately over-represented relative to children of racial and ethnic minorities in pediatric growth hormone (GH) treatment registries. This study sought to determine if this racial inequity is due to differences in GH stimulation testing and/or GH prescribing patterns in children referred for endocrine evaluation of short stature. Methods: Retrospective chart review was performed including children aged 2-16 years, with height z-score ≤ -1.5, and of NHW, non-Hispanic black (NHB) or Hispanic race/ethnicity, referred for endocrine growth evaluation between January 1, 2012 and December 31, 2019. Age, sex, anthropometry, GH stimulation test results and GH treatment data were extracted. Comparisons between NHB, NHW and Hispanic children were performed using analysis of variance, chi-squared tests, Mann Whitney U and logistic regression tests. Results: This study included 7,425 patients (5,905 NHW, 800 NHB, and 720 Hispanic). GH stimulation testing was performed in 992, and 576 were prescribed GH. NHW children were 1.4 (95% CI 1.04 - 1.8) times more likely than NHB children and 1.7 (95% CI 1.2 - 2.2) times more likely than Hispanic children to undergo GH stimulation testing. NHB children treated with GH had: 1) lower median peak GH concentration when compared with NHW (p=0.02) and Hispanic (p=0.08) children (NHB 4.7 [1.2, 8.3] ng/ml, NHW 7.2 [4.9, 9.7] ng/ml, Hispanic 7.1 [4.3, 11.9] ng/ml); 2) lower median height z-scores than NHW (p=0.01) but not Hispanic children (p=0.5); and 3) a greater height deficit from mid-parental height when compared with NHW (p=0.01) and Hispanic (p=0.002) children Discussion: Racial and ethnic disparities are present in the evaluation and treatment of children with disordered growth. This likely results from both over-investigation of NHW children as well as under-investigation and under-treatment of children from minority communities. The evaluation and treatment of children with short stature should be determined by clinical concern alone, but this is unfortunately not current practice.

Racial and Ethnic Disparities in the Investigation and Treatment of Growth Hormone Deficiency

To determine if the racial/ethnic inequity in growth hormone (GH) use is due to differences in GH stimulation testing and/or prescribing patterns in children referred for endocrine evaluation of short stature. Retrospective chart review was performed including children aged 2-16years, height z-score of ≤-1.5, and of non-Hispanic White (NHW), non-Hispanic Black (NHB), or Hispanic race/ethnicity, referred for endocrine growth evaluation between January 2012 and December2019. This study included 7425 children (5905 NHW, 800 NHB, and 720 Hispanic). GH stimulation testing was performed in 992, and 576 were prescribed GH. NHW children were 1.4 (95% CI, 1.04-1.8) times more likely than NHB children and 1.7 (95% CI, 1.2-2.2) times more likely than Hispanic children to undergo GH stimulation testing. GH-treated NHB children had (1) a lower median peak GH concentration when compared with NHW (P=.02) and Hispanic (P=.08) children (NHB 4.7ng/mL [95% CI, 1.2-8.3ng/mL] ng/mL, NHW 7.2ng/mL [95% CI, 4.9-9.7ng/mL], Hispanic 7.1ng/mL [95% CI, 4.3-11.9ng/mL]); (2) lower median height z-scores than NHW (P=.01) but not Hispanic children (P=.5); and (3) a greater height deficit from midparental height when compared with NHW (P=.01) and Hispanic (P = .002) children. Racial and ethnic disparities exist in the evaluation and treatment of children with disordered growth. This likely results from both overinvestigation of NHW children as well as underinvestigation and undertreatment of children from minority communities. The evaluation and treatment of children with short stature should be determined by clinical concern alone, but this is not current practice.

Gene polymorphisms in leptin and its receptor and the response to growth hormone treatment in patients with idiopathic growth hormone deficiency.

This study aimed to investigate the relationships between genetic polymorphisms of leptin/receptor genes and clinical/biochemical characteristics in children with growth hormone deficiency (GHD). Ninety-three GHD children and 69 age-matched normal controls were enrolled. Anthropometric measurements, bone age, and laboratory test results were obtained. Polymorphisms in the LEP gene promoter locus (LEP-2548, rs7799039) and LEPR genes (K109R, rs1137100 and Q223R, rs1137101) were analyzed using PCR-RFLP. The serum leptin levels were measured using an ELISA kit. The median height and BMI z-scores of all GHD subjects were -2.20 and -0.26, respectively, and those of normal controls were -0.30 and -0.13, respectively. The serum leptin levels were similar between GHD subjects and normal controls (p = 0.537), but those were different between the complete GHD (6.97 ng/mL) and partial GHD (4.22 ng/mL) groups (p = 0.047). There were no differences in the genotypic distributions of LEP-2548, LEPR K109R, and Q223R between GHD subjects and normal controls. However, GHD subjects with the G allele at LEP-2548 showed higher IGF-1 (p = 0.047) and IGFBP-3 SDSs (p = 0.027) than GHD subjects with the A allele. GHD subjects with the G allele at LEPR Q223R showed lower stimulated GH levels (p = 0.023) and greater height gain after 1 year of GH treatment (p = 0.034) than GHD subjects with the A allele. In conclusion, leptin/leptin receptor genes are suggested to have the role of growth-related factors, which can affect various growth responses in children who share the same disease entity.

Pubertal development and adult height in patients with congenital hypothyroidism detected by neonatal screening in southern Brazil.

Objectives Adequate treatment of congenital hypothyroidism (CH) is required for normal growth and sexual development. To evaluate pubertal development in patients with permanent CH detected by a statewide Neonatal Screening Program of Paraná and, secondly, to evaluate adult height (AH) in a subgroup of patients. Methods Clinical, laboratory, and auxological data obtained from medical records of 174 patients (123 girls). Results Median chronological age (CA) at treatment initiation was 24days, and mean initial levothyroxine dose was 11.7±1.9μg/kg/day; mean CA at puberty onset was 11.5±1.3 years (boys) and 9.7±1.2 years (girls); mean CA in girls who underwent menarche (n=81) was 12.1±1.1 years. Thyroid-stimulating hormone (TSH) values above the normal range were observed in 36.4% of the boys and 32.7% of the girls on puberty onset, and in 44.6% around menarche. Among 15 boys and 66 girls who had reached the AH, the median height z-score value was significantly greater than the target height (TH) z-score value in boys (p=0.01) and in girls (p<0.001). Boys with normal TSH values at puberty onset had greater mean AH z-score compared with boys with TSH values above the normal range (p=0.04). Conclusions In this group, pubertal development in girls with CH was not different from that reported in healthy girls in the general Brazilian population. Boys with higher TSH at puberty onset may have an increased risk of not reaching their potential height compared with those with normal TSH during this period. In a subgroup who attained AH, the median AH z-score was greater than the median TH z-score.

Rituximab for Children With Difficult-to-Treat Nephrotic Syndrome: Its Effects on Disease Progression and Growth.

Background: Since the early 2000s rituximab (RTX) has been thought of as an alternative treatment for steroid-sensitive nephrotic syndrome (SSNS) and steroid-resistant nephrotic syndrome (SRNS).Objective: This study aimed to determine the effects of RTX treatment on disease outcome and growth in pediatric SSNS and SRNS patients.Materials and Methods: The medical records of pediatric SSNS and SRNS patients that began RTX treatment at the mean age of 10.8 ± 5.1 years between 2009 and 2017 were retrospectively reviewed. Additionally, the effect of RTX on growth was evaluated based on patient height, weight, and BMI z scores.Results: The study included 41 children, of which 21 had SSNS and 20 had SRNS. Mean age at diagnosis of NS was 5.8 ± 4.7 years. Mean duration of post-RTX treatment follow-up was 2.3 ± 1.6 years. Among the SSNS patients, 6 and 11 patients were steroid free and calcineurin inhibitor free at the last follow-up visit, respectively. The 1-year cumulative steroid and calcineurin inhibitor doses both decreased after RTX treatment, as compared to before RTX (P = 0.001 and P = 0.015, respectively). The median height z-score at the time of RTX initiation was −1.2 and the median height z-score at the last follow-up visit was −0.6 (P = 0.044). The median BMI z-score decreased from 1.6 (IQR; 0.9–3.0) at the time RTX was initiated to 1.1 IQR; [(−0.7)−2.5] at the last follow-up visit (P = 0.007). At the last follow-up visit 4 SRNS patients had complete remission and 4 had partial remission. The 1-year cumulative steroid dosage in the SRNS patients decreased significantly after RTX, as compared to before RTX (P = 0.001). The median height z-score at the time of RTX initiation was −0.8 and the median height z-score at the last follow-up visit was −0.7 (P = 0.81). The median BMI z-score decreased from 0.3 at the time RTX was initiated to −0.1 at the last follow-up visit (P = 0.11).Conclusion: RTX has a more positive effect on disease outcome and growth in SSNS patients than in those with SRNS.

Single Center Retrospective Analysis of Outcomes Following Hematopoietic Cell Transplantation (HCT) for Primary Immune Deficiency (PID)

Background Successful hematopoietic stem cell transplantation (HCT) is curative in primary immune deficiency (PID), but data on post HCT late effects are scarce. We describe outcomes and late effects in PID HCT survivors at 1 year post HCT. Methods Retrospective data analysis was conducted on consecutive PID HCT recipients from Sept 2007-2017 on IRB approved protocol 0707. Outcomes queried included engraftment, graft versus host disease (GVHD), and late effects in survivors. Data review at 2, 5, and 10 years post HCT including analysis of cognitive outcomes and immune reconstitution are ongoing; specifically impact of disease type, age at HCT, and graft source. Results Thirty-nine PID HCT patients received reduced intensity conditioning (RIC) HCT with 1-year overall survival (OS) of 82%. Median age at HCT was 0.5 years (range 0.08 – 17.4 years) and 72% (28/39) were males. Stem cell source was peripheral blood in 72% (19 unrelated, 9 related), umbilical cord blood in 25.5% (10 unrelated), and bone marrow in 2.5% (1 unrelated). Donors were 16 HLA matched and 23 mismatched. RIC regimen was Busulfan AUC 5000 mcmol/L*min x 2 days, Fludarabine 60 mg/m2 × 6 days, rabbit ATG 2mg/kg x 4 days ± Thiotepa 5mg/kg x 1 day. Incidence of > Grade 2 acute GVHD and limited chronic GVHD was 5% (2/39) and 5% (2/39), respectively. Seven patients died due to infection (4), veno-occlusive disease (1), Grade 4 acute GVHD (1), and intracranial hemorrhage (1). Graft failure (GF) rate was 5% (1 primary GF and 1 secondary GF with EBV infection). Donor chimerism (DC) including T cell, was >95% in 60% and those with mixed chimerism had full T cell DC at 1 year post HCT. In 32 survivors, no cardiac, liver, renal, pulmonary abnormalities, or secondary malignancies were noted. Growth was significantly impacted with median height z-scores of -3, with 69% being Conclusion Successful RIC HCT is curative with excellent 1 year OS of 82% with minimal organ toxicities in PID patients; however, significant growth impairment was observed in the majority with some endocrine abnormalities at 1 year post HCT. While further ongoing analysis is imperative, this emphasizes the need for standardized prospective medical surveillance in order to earlier identify post HCT problems systematically and intervene appropriately to improve outcomes in post HCT PID patients.

Comparison of Growth Pattern of School Children in Sylhet, Bangladesh Using the 2000 CDC Standards and 2007 WHO Standards

Background: Growth assessment is an essential component of pediatric health surveillance because almost any problem within the physiologic, interpersonal and social domains can adversely affect growth. The most powerful tool in the growth assessment is the growth chart. Growth parameters of children are usually interpreted in relation to international standards like the National Center for Growth Statistics (NCHS) and Centers for Disease Control and Prevention (CDC) Growth Charts of 1977 and 2000, respectively. In 2007, the World Health Organisation (WHO) published a new set of growth charts believed to be applicable to children reared healthily in all parts of the globe. The aim of current study was determine the growth pattern of school children in Sylhet using the CDC standards and WHO standards.&#x0D; Methods: A Comparative cross-sectional study was done in the department of paediatrics, Sylhet MAG Osmani Medical College Hospital from July, 2012 to Dec, 2012. Multi-stage random sampling technique was adopted for the survey. This study was carried out in selected primary schools in Sylhet Metropoliton City area. The participants were healthy school children, aged 6 to 11 years who was purposively selected. A total of 504 children were included into this study. The primary end point was to determine weight, height and body mass index (BMI) and the secondary end point was to compare the values using 2000 CDC standards and 2007 WHO standards.&#x0D; Results: The mean weights, height and BMI of the studied child were lower from CDC and WHO standards but closer with the WHO standards and wider from CDC. The weight Z-scores have got closer on the two reference charts for both male and female but differ at age 6 and 7 years. The median height Z-scores were almost closer to the median for male and female at all different ages on both CDC and WHO charts. The median BMI Z-scores for both sexes were below the reference values on both CDC and WHO charts. The prevalence of underweight and stunting were higher in both sexes when determined using the CDC standards compared to WHO standards.&#x0D; Conclusion: The WHO references would under diagnose under-nutrition and over-diagnose overweight/obesity in the population studied. However, this study results are closer to the WHO reference and wider from the CDC standards.&#x0D; Birdem Med J 2019; 9(1): 35-43

Late effects in high-risk neuroblastoma survivors treated with high-dose chemotherapy and stem cell rescue.

Current treatment strategies have improved the outcome of high-risk neuroblastoma (HRNB) at the cost of increasing acute and late effects of treatment. Although high-dose chemotherapy with stem cell rescue (HDC-SCR) has replaced total body irradiation (TBI) based HRNB therapy, late effects of therapy remain a significant concern. To describe late effects prevalence, severity, and risks after HDC-SCR. Retrospective chart review of relapse-free HRNB survivors ≥1 year after single HDC-SCR between 2000 and 2015 at Fred Hutchinson Cancer Research Center. Sixty-one survivors (30 males) were eligible. Median age (years) at SCR was 3.5 years (range 0.7-27 years) and median posttransplant follow-up was 5.4 years (1.2-16.3 years) . Fifty-three (86.9%) survivors developed late effects that increased over time (P<0.001) and varied in severity from grade 1 (35) to grade 5 (1). These were unrelated to gender or age. High-frequency hearing loss seen in 82% of survivors was the most common abnormality present and 43% of those required hearing aids. Seventeen (27.9%) survivors developed dental late effects and these were most common in children<2 years of age at transplant (P=0.008). Other toxicities included endocrine (18%), orthopedic (14.8 %), renal (3.9%), melanotic nevi (8.2%), neuropsychological impairments (8.2%), subsequent malignancies (4.9%), pulmonary (4.9%), cardiac (4.9%), and focal nodular liver hyperplasia (3.3%). At 9 years posttransplant, the median height and weight Z-scores were significantly lower than Z-scores at the time of HDC-SCR (-0.01/-1.08, P<0.001; -0.14/-0.78, P=0.005). Avoidance of TBI does not mitigate the need to provide diligent, ongoing surveillance for late effects.

Diet and Anthropometrics of Children With Inflammatory Bowel Disease: A Comparison With the General Population

There is a lack of knowledge regarding the diet of children with inflammatory bowel disease (IBD). Therefore, we investigated dietary intake in pediatric IBD compared to the general population and assessed anthropometrics and food avoidance. In this cross-sectional cohort study, patients younger than 18 years with IBD were included (2014-2017). Dietary intake (total energy, macro nutrients, food types) was assessed using a Food Frequency Questionnaire (FFQ) and compared with a matched reference population (n = 306). There were 102 patients included (55% male, median age 15.0 years). Median height Z-score was -0.39 (interquartile range [IQR]: -1.17 to 0.26). Growth failure (height Z-scores<-1.64) was present in 11% of patients. Median BMI Z-score was 0.36 (IQR: -0.70 to 0.96). Acute malnutrition (weight-for-height Z-score<-2) was present in 3% of patients. Energy intake was lower in pediatric IBD compared to the reference population (mean kilojoule/day: 8286 vs 9794, P < 0.001). Protein intake did not differ, while fat intake was higher in pediatric IBD (mean gram/day: 101 vs 91, P < 0.001), with higher intake of vegetable oils/fats. Carbohydrate intake was lower in pediatric IBD (mean gram/day: 267 vs 305, P < 0.001), with lower intake of food types high in sugar. Food avoidance was reported in 53% of patients, with frequent avoidance of spicy (46%), high-fat food (30%), and dairy (30%). The diet of children with IBD differs from the general pediatric population, with lower energy intake and high rates of food avoidance. Evaluation of the dietary intake alongside anthropometrics may be important to prevent nutrition deficiencies and promote health.

Growth patterns in early juvenile idiopathic arthritis: Results from the Childhood Arthritis Prospective Study (CAPS).

ObjectivesTo investigate early vertical growth patterns and factors associated with poor growth in a modern inception cohort of UK children with juvenile idiopathic arthritis (JIA) using data from the Childhood Arthritis Prospective Study (CAPS).MethodsA study period of 3 years was chosen. Children included in this analysis had a physician diagnosis of JIA and had height measurements available at both baseline and at 3-years of follow-up. Height is presented as z-scores calculated using World Health Organisation growth standards for age and gender. Growth over the 3-year period was assessed using change in z-score and height velocity. Univariable and multivariable linear regressions were used to identify factors associated with height z-score at baseline and change of height z-score at 3 years.Results568 patients were included; 65% female, median baseline age 7.4 years [interquartile range (IQR) 3.6, 11.2], median symptom duration at presentation 5.5 months [IQR 3.1, 11.6]. Height z-score decreased significantly from baseline to 3 years (p ≤ 0.0001); baseline median height z-score was −0.02 (IQR −0.71, 0.61), decreasing to −0.47 (IQR −1.12, 0.24) at 3 years. Growth restriction, defined as change of height z-score ≤−0.5, was observed in 39% of patients. At 3 years, higher baseline height z-score was the strongest predictor for a negative change in height z-score [−0.3 per unit of baseline height z-score (95% CI: −0.36, −0.24), p < 0.0001].ConclusionsAlthough overall height at 3 years after initial presentation to rheumatology is within the population norm, as a cohort, children with JIA experience a reduction of growth in height over the first 3 years of disease. Late presentation to paediatric rheumatology services is associated with lower height at presentation. However, patients with the lowest height z scores at presentation were also the most likely to see an improvement at 3 years. The impact of JIA on growth patterns is important to children and families and this study provides useful new data to support informed clinical care.

The Relationship between Dietary Intake, Growth, and Body Composition in Inborn Errors of Intermediary Protein Metabolism

To examine relationships between dietary intake, growth and body composition patterns in patients with inborn errors of intermediary protein metabolism and to determine a safe protein:energy ratio (P:E ratio) associated with optimal growth outcomes. Retrospective longitudinal data of growth and dietary intake in patients (n = 75) with isovaleric acidemia (IVA; n = 7), methylmalonic acidemia/propionic acidemia (MMA/PA; n = 14), urea cycle defects (UCD; n = 44), classical maple syrup urine disease (MSUD; n = 10) were collected. Prospective longitudinal data of growth, dietary intake, and body composition from 21 patients: IVA (n = 5), MMA/PA (n = 6), UCD (n = 7), and MSUD (n = 3) were collected at clinic visits. Fifty-two of 75 (66%), 49 of 74 (68%), and 44 of 65 (68%) patients had a z-score of 0 (±1) for lifetime weight, height, and body mass index, respectively. Patients with MMA/PA had the lowest median height and weight z-scores, and MSUD patients had highest median body mass index z-score at all ages. In IVA, MMA/PA, and UCD, total natural protein intake met or exceeded the Food and Agriculture Organization of the United Nations (FAO)/World Health Organization (WHO)/United Nations University (UNU) recommended safe levels. Median percentage fat mass was 17.6% in IVA, 20.7% in MMA/PA, 19.4% in UCD, and 17.8% in MSUD. There was a significant negative correlation between percentage fat mass and total protein intake in IVA, MMA/PA, and UCD (r = -0.737; P = .010). The correlation between the P:E ratio and growth variables in IVA, MMA/PA, and UCD suggest a safe P:E ratio (>1.5 to < 2.9) g protein:100 kcal/day. Growth outcomes in inborn errors of intermediary protein metabolism are not always ideal. Most patients with IVA, MMA/PA, and UCD consume sufficient natural protein to meet FAO/WHO/UNU recommendations. A P:E ratio range of (>1.5 to < 2.9)g protein/100 kcal/day correlates with optimal growth outcomes.

Comparison of height and weight after 12 vs. 18 Gy cranial radiation therapy in pediatric acute lymphoblastic leukemia (ALL) patients

PurposeTo compare the effect of 12 versus 18 Gy cranial radiation therapy (RT) on height and weight indices among pediatric patients with acute lymphoblastic leukemia (ALL). Methods and materialsRecords of children with ALL who were 2 to 14 years old at the time of RT and were treated at a single institution between 2000 and 2011 were reviewed. Patients' height, weight, and body mass index were converted into z-scores using the Centers for Disease Control growth charts to normalize the values to number of standard deviations from the mean. These values were measured at the pre-RT clinic visit and subsequent yearly intervals. The z-scores of the growth indices were fitted into a generalizing estimating equations model and analyzed by various clinical factors. ResultsA total of 48 patients met the study criteria, including 32 boys and 16 girls. The median age at the time of RT was 7 years (range, 2-14 years). Patients were separated into 2 dose groups: 12 Gy (n = 30) and 18 Gy (n = 18). Median follow-up was 4.9 years (range, 3.0-11.8 years) and 6.0 years (range, 3.1-10.5 years) and the median pre-RT height z-scores were −0.55 (range, −2.2 to 1.4) and −0.85 (range, −3.1 to 0.8) for the 2 groups, respectively (P = .65). Patients who received 18 Gy had a significant difference in change in height compared with those who received 12 Gy, who were able to maintain normal growth during the first 3 years of follow-up. This did not appear to be sex-specific, and there was no difference in change in weight or body mass index. ConclusionsCompared with 18 Gy, patients with ALL who received 12 Gy of cranial RT had less height impairment in the first 3 years post-RT, but further prospective studies are needed.

Growth in Children with Autosomal Recessive Polycystic Kidney Disease in the CKiD Cohort Study

Previous studies have suggested that some children with autosomal recessive polycystic kidney disease (ARPKD) have growth impairment out of proportion to their degree of chronic kidney disease (CKD). The objective of this study was to systematically compare growth parameters in children with ARPKD to those with other congenital causes of CKD in the chronic kidney disease in Children (CKiD) prospective cohort study. Height SD scores (z-scores), proportion of children with severe short stature (z-score < -1.88), rates of growth hormone use, and annual change in height z-score were analyzed in children with ARPKD (n = 22) compared with two matched control groups: children with aplastic/hypoplastic/dysplastic kidneys (n = 44) and obstructive uropathy (OU) (n = 44). Differences in baseline characteristics were tested by Wilcoxon rank-sum test or Fisher's exact test. Matched differences in annual change in height z-score were tested by Wilcoxon signed-rank test. Median height z-score in children with ARPKD was -1.1 [interquartile range -1.5, -0.2]; 14% of the ARPKD group had height z-score < -1.88, and 18% were using growth hormone. There were no significant differences in median height z-score, proportion with height z-score < -1.88, growth hormone use, or annual change in height z-score between the ARPKD and control groups. Children with ARPKD and mild-to-moderate CKD in the CKiD cohort have a high prevalence of growth abnormalities, but these are similar to children with other congenital causes of CKD. This study does not support a disease-specific effect of ARPKD on growth, at least in the subset of children with mild-to-moderate CKD.

Hepatopathy of Mauriac syndrome: a retrospective review from a tertiary liver centre

BackgroundMauriac syndrome is characterised by growth failure, cushingoid appearance and hepatomegaly which occurs in patients with insulin dependent diabetes and was first described shortly after the introduction of insulin as...

Imatinib Has a Negative Impact On Growth In Children With Previously Untreated Chronic Myeloid Leukaemia (CML) In Early Chronic Phase (CP): Results Of The French National Study

AimsThe French pediatric CML working group conducted a retrospective study analysing the growth of patients under 18 years with CML treated with imatinib front-line. Patients and MethodsData from 78 patients receiving imatinib (260 mg/m2) for newly diagnosed CML in chronic phase between July 2001 and August 2012 were collected. Height was expressed as standard deviation score (SDS). Height SDS was calculated using French growth standard, as patient height minus mean height for age and sex, divided by standard deviation of height for age and sex. Pre-pubertal age was defined as being younger than 9 years for girls or 11 years for boys. ResultsA gradual decrease in the median of the height SDS score is observed over time since the start of imatinib. Sixty three patients were assessable for paired analysis at onset of imatinib and 12 months later. The median height-SDS in this group of patients was significantly lower (p < 10-4) 12 months after the start of imatinib. Variance analysis was performed in 36 children at onset of imatinib, 12 months and 24 months later. The median height-Z score in this group of patients was significantly lower 12 months and 24 months after the start of imatinib (p < 10-4) compared to initial diagnosis. The 63 patients assessable for paired data analysis at onset of imatinib and 12 months later were classified by sex and pubertal status. The median height Z score in boys and girls was significantly (p < 10-4) lower 12 months after the start of imatinib. When the patients were analysed according to the pubertal status at onset of imatinib, the median height SDS score was significantly (p < 10-4) lower 12 months after the start of imatinib in the prepurbertal group as well as in the postpubertal group. A similar finding was observed in the subgroups of boys and girls starting imatinib at a prebubertal or postpubertal age. No significant difference was observed when the median M12 height SDS score in patients who started imatinib at a prepubertal stage or a pospubertal stage were compared. Moreover, no significant difference was observed when the median M12 height-Z score in boys and girls were compared. ConclusionThis retrospective analysis revealed a statistically significant deceleration in growth during the first 2 years of imatinib treatment in children and adolescents with CML and an absence of influence of sex and pubertal status on the depth of growth deceleration. Disclosures:No relevant conflicts of interest to declare.