Importance of Growth Factors and Bone Maturation Ratio in the Response to Growth Hormone Therapy.

OBJECTIVE: The aim was to identify the influence of insulin-like growth factor I (IGF-1), IGF-binding protein-3 (IGFBP-3), and bone age (BA)/chronological age (CA) ratio on the response to GH therapy after 1 and 2 years of treatment and upon reaching final height. METHODS: Longitudinal, retrospective, observational study of 139 patients treated for idiopathic growth hormone deficiency. Variables examined during follow-up: (1) genetic background; (2) perinatal history; (3) anthropometry; (4) height velocity, BA, BA/CA and height prognosis; (5) analytical results (IGF-1, IGFBP-3). Final response variables: adult height (AH), AH with respect to target height, AH with respect to initial height prognosis, AH with respect to height at the start of treatment, and AH with respect to height at onset of puberty. RESULTS: Lower pretreatment IGF-1 levels and a greater increase in IGF-1 at the end of treatment imply a better response (r = -0.405, P = .007 and r = 0.274, P = .014, respectively), as does a greater increase in IGFBP-3 after 2 years of treatment and at the end of treatment (r = 0.207, P = .035 and r = 0.259, P = .020, respectively). A lower BA/CA ratio pretreatment and at the onset of puberty results in a better response (r = -0.502, P = .000 and r = -0.548, P = .000, respectively), as does a lower increase in BA and BA/CA ratio after the 1 and 2 years of treatment (r = -0.337, P = .000 and r = -0.332, P = .000, respectively). CONCLUSION: Low pretreatment IGF-1, a greater BA delay with respect to CA pretreatment and at the onset of puberty, a greater increase in IGFBP-3 after 2 years of treatment, and a lower increase in BA and BA/CA ratio after 1 and 2 years of treatment imply a better long-term response.

Translation and psychometric evaluation of the Spanish version of the problem areas in diabetes-pediatric version (PAID-Peds) survey.

BACKGROUND: Metabolic control and psychological management of paediatric type 1 diabetes mellitus (T1DM) can be challenging over time. Development of an instrument to assess the youth-reported burden could aid in preventing T1DM-associated diseases. METHODS: The aim of this study was to translate and validate the Spanish version of the Problem Area in Diabetes Survey-Pediatric version (PAID-Peds). A multicentre, cross-sectional translation and linguistic validation study was performed on a sample of 30 participants aged 8-17 years with a minimum 1-year history of T1DM diagnosed at the Miguel Servet University Hospital in Zaragoza (Aragon, Spain), Ramón y Cajal University Clinical Hospital in Madrid (Spain), and Sant Joan de Déu Hospital in Barcelona (Catalonia, Spain). The qualitative validation consisted of translation into Spanish and back-translation into English of the Paid-Peds survey and subsequent administration to the sample population. Data were gathered on parameters related to sociodemographic characteristics and metabolic control. Validity, feasibility, and test-retest reliability were evaluated. Internal consistency was determined using Cronbach's alpha coefficient, test-retest reliability by means of interclass correlation, and paired samples using the Wilcoxon W-test. The study was approved by the ethics and research committees at each participating centre. RESULTS: The study assessed 30 children (46.7% female) with an average age of 13.33 ± 2.98 years; mean age at onset was 5.70 ± 3.62 years, and the mean disease duration was 7.63 ± 4.36 years. The mean score on the PAID-Peds survey was 42.88 ± 17.85. Cronbach's alpha coefficient was 0.90. Test-retest reliability measured by interclass correlation coefficient was 0.8 (95% CI: 0.63-0.90). No significant differences in total scores were found between test and retest (Wilcoxon W-test: 289; p = 0.051). CONCLUSIONS: The Spanish version of the PAID-Peds survey is a feasible, valid, and reliable instrument to assess the youth-perceived burden of T1DM.

Intralymphatic GAD-Alum (Diamyd®) Improves Glycemic Control in Type 1 Diabetes With HLA DR3-DQ2.

AIMS: Residual beta cell function in type 1 diabetes (T1D) is associated with lower risk of complications. Autoantigen therapy with GAD-alum (Diamyd) given in 3 intralymphatic injections with oral vitamin D has shown promising results in persons with T1D carrying the human leukocyte antigen (HLA) DR3-DQ2 haplotype in the phase 2b trial DIAGNODE-2. We aimed to explore the efficacy of intralymphatic GAD-alum on blood glucose recorded by continuous glucose monitoring (CGM). METHODS: DIAGNODE-2 (NCT03345004) was a multicenter, randomized, placebo-controlled, double-blind trial of 109 recent-onset T1D patients aged 12 to 24 years with GAD65 antibodies and fasting C-peptide > 0.12 nmol/L, which randomized patients to 3 intralymphatic injections of 4 µg GAD-alum and oral vitamin D, or placebo. We report results for exploratory endpoints assessed by 14-day CGM at months 0, 6, and 15. Treatment arms were compared by mixed-effects models for repeated measures adjusting for baseline values. RESULTS: We included 98 patients with CGM recordings of sufficient quality (DR3-DQ2-positive patients: 27 GAD-alum-treated and 15 placebo-treated). In DR3-DQ2-positive patients, percent of time in range (TIR, 3.9-10 mmol/L) declined less between baseline and month 15 in GAD-alum-treated compared with placebo-treated patients (-5.1% and -16.7%, respectively; P = 0.0075), with reduced time > 13.9 mmol/L (P = 0.0036), and significant benefits on the glucose management indicator (P = 0.0025). No differences were detected for hypoglycemia. GAD-alum compared to placebo lowered the increase in glycemic variability (standard deviation) observed in both groups (P = 0.0219). Change in C-peptide was correlated with the change in TIR. CONCLUSIONS: Intralymphatic GAD-alum improves glycemic control in recently diagnosed T1D patients carrying HLA DR3-DQ2.

Influence of the SARS-CoV-2 pandemic on paediatric patients with type 1 diabetes mellitus after one year of follow-up.

OBJECTIVES: Lockdown during the SARS-CoV-2 pandemic generated uncertainty regarding its effects on the control of type 1 diabetes (DM1). Our study aims to evaluate the influence of the pandemic on the control of paediatric patients with DM1. METHODS: Longitudinal, retrospective, observational study in patients with DM1 attended between 15/10/2019 and 15/03/2020. Data were collected at that visit and at the three subsequent visits. The second was remote in 50% of cases. The variables analysed were: type of insulin therapy, time in range (TIR), time in hypoglycaemia (THypo), time in hyperglycaemia (THyper), coefficient of variation (CV), glycosylated haemoglobin, insulin requirements and anthropometric data. RESULTS: 157 patients were recruited. At the post-lockdown visit, the TIR increased and the THyper decreased with respect to the first (p<0.00) and second (p<0.00) visits. Patients treated with subcutaneous infusion showed a higher TIR at the third visit (p=0.03) and lower insulin requirements at the fourth visit (p=0.03) compared to patients treated with multiple doses. Patients with a remote visit presented a higher TIR (p<0.00), a lower THyper (p=0.00) and lower insulin requirements (p=0.01) at the next visit. Patients aged less than 6 years presented a lower glycosylated haemoglobin (p=0.01) and insulin requirements at the third (p=0.03) and fourth (p=0.01) visits, and a lower increase in body mass index (p=0.03) over the year. CONCLUSIONS: Metabolic control improved at the post-lockdown visit. Patients treated with subcutaneous infusion, those who had a remote visit during strict lockdown and those aged less than 6 years had a better evolution.

Growth hormone deficit: Influence of puberty on the response to treatment.

INTRODUCTION: Short stature is the most frequent reason for consultation in Pediatric Endocrinology consultations and sometimes requires treatment with growth hormone. The aim of the study was to analyze the response to treatment based on its onset in pubertal or prepubertal stages and to analyze the possible benefit of an early onset. PATIENTS AND METHODS: Longitudinal, retrospective and observational study in 139 patients treated for idiopathic growth hormone deficiency up to adult height. MAIN VARIABLES STUDIED: (a) genetic background: maternal, paternal and genetic height; (b) perinatal history; (c) anthropometry during follow-up and at pubertal onset: weight, height, body mass index; (d) variables during follow-up and at pubertal onset: growth rate, bone age and growth prognosis. Final response variables: adult height, adult height with respect to target height, adult height with respect to initial growth prediction, adult height with respect to initial height at the start of treatment and adult height with respect to height at pubertal onset. RESULTS: Total pubertal gain was 0.84 ± 0.6 SD. 61.9% of the patients started treatment with rhGH in prepuberty. The initiation of treatment in the prepubertal stage and a higher total pubertal gain are correlated with a better final height (P = 0.001 and r = 0.507, P = 0.00, respectively). Furthermore, a longer duration of treatment in pre-puberty is correlated with a better final response (r = 0.328, P = 0.00). CONCLUSIONS: The start of treatment in the prepubertal stage and its longer duration during this period are determining factors to achieve a good long-term response. Total pubertal gain was greater in patients who started treatment in the pubertal stage.

Déficit de hormona de crecimiento: influencia de la pubertad en la respuesta al tratamiento / Growth hormone deficit: Influence of puberty on the response to treatment

Introducción: La talla baja es motivo de consulta frecuente en Endocrinología Pediátrica, precisando en ocasiones tratamiento con hormona del crecimiento (GH). El objetivo del estudio fue analizar la respuesta al tratamiento en función de su inicio en la etapa puberal o prepuberal y analizar el posible beneficio de un comienzo precoz. Pacientes y métodos: Estudio longitudinal, retrospectivo y observacional en 139 pacientes tratados por déficit de GH idiopático (grave o parcial) hasta talla adulta. Principales variables estudiadas: a) antecedentes familiares: talla materna, paterna y genética; b) antecedentes perinatales; c) antropometría durante el seguimiento y al inicio puberal: peso, talla, índice de masa corporal, y d) variables durante el seguimiento y al inicio puberal: velocidad de crecimiento, edad ósea y pronóstico de crecimiento. Variables de respuesta final: talla adulta, talla adulta respecto a talla genética, talla adulta respecto al pronóstico de crecimiento inicial, talla adulta respecto a talla al inicio del tratamiento y talla adulta respecto a talla al inicio puberal. Resultados: La ganancia puberal total fue de 0,84±0,6 DE. Un 61,9% de los pacientes iniciaron tratamiento con GH en prepubertad. El inicio del tratamiento en la etapa prepuberal y una mayor ganancia puberal total se relacionaron con una mejor talla final (p=0,001, y r=0,507, p=0,00 respectivamente). Además, una mayor duración del tratamiento en la prepubertad se correlacionó con una mejor respuesta final (r=0,328, p=0,00). Conclusiones: El inicio del tratamiento en la prepubertad y una mayor duración durante este periodo son factores determinantes para alcanzar una mejor respuesta a largo plazo. La ganancia puberal total fue mayor en los pacientes que iniciaron el tratamiento en etapa puberal. (AU)

Introduction: Short stature is the most frequent reason for consultation in Pediatric Endocrinology consultations and sometimes requires treatment with growth hormone. The aim of the study was to analyze the response to treatment based on its onset in pubertal or prepubertal stages and to analyze the possible benefit of an early onset. Patients and methods: Longitudinal, retrospective and observational study in 139 patients treated for idiopathic growth hormone deficiency up to adult height. Main variables studied: (a) genetic background: maternal, paternal and genetic height; (b) perinatal history; (c) anthropometry during follow-up and at pubertal onset: weight, height, body mass index; (d) variables during follow-up and at pubertal onset: growth rate, bone age and growth prognosis. Final response variables: adult height, adult height with respect to target height, adult height with respect to initial growth prediction, adult height with respect to initial height at the start of treatment and adult height with respect to height at pubertal onset. Results: Total pubertal gain was 0.84±0.6 SD. 61.9% of the patients started treatment with rhGH in prepuberty. The initiation of treatment in the prepubertal stage and a higher total pubertal gain are correlated with a better final height (P=.001 and r=0.507, P=.00, respectively). Furthermore, a longer duration of treatment in pre-puberty is correlated with a better final response (r=0.328, P=.00). Conclusions: The start of treatment in the prepubertal stage and its longer duration during this period are determining factors to achieve a good long-term response. Total pubertal gain was greater in patients who started treatment in the pubertal stage. (AU)

Growth hormone deficit. Does the first year of treatment influence adult height?

INTRODUCTION: Short stature is the most frequent reason for Pediatric Endocrinology consultations and sometimes requires treatment with growth hormone. OBJECTIVE: The possible correlation of a good response to any early response factor with a better final response was studied, and also whether there was a difference in response to treatment according to the type of deficit. PATIENTS AND METHODS: This was a longitudinal, retrospective and observational study of 139 patients treated for idiopathic growth hormone deficiency up to adult height. There were good response criteria in the first year of treatment: a) an increase in growth rate ≥3 cm/year, b) a growth rate ≥1 standard deviation (SD), c) an increase in height ≥0.5 SD, d) an increase in height ≥0.3 SD. Study of the Index of Responsiveness to treatment in the first and second year. Final response variables: adult height with respect to target height, adult height with respect to initial growth prediction and adult height with respect to initial height at the start of treatment. The possible correlation of a good response to any of the early response factors with a better final response to treatment was studied, and also whether there was a difference in the response to treatment according to the type of deficit. RESULTS: The treatment produced a gain in adult height with respect to genetic height of 0.06 ±â€¯0.7 SD. Patients considered good responders in the first year of treatment presented a better final response (growth rate ≥3 cm: p = 0.000, growth rate ≥1 SD: p = 0.008, height gain ≥0.5 SD: p = 0.007, height gain ≥0.3 SD: p = 0.006), as well as patients with a severe deficit (p = 0.04). The index of responsiveness to treatment during the first year was associated with a better final response (r = 0.249, p = 0.003), with this correlation being maintained in the second year (r = 0.294, p = 0.01). CONCLUSIONS: Growth hormone treatment increased height in the genetic target. The percentage of good responders varied depending on the criteria used. The response in the first year of treatment and a severe deficit were determining factors for achieving a good long-term response.

Impacto de faster aspart sobre el control glucémico en niños y adolescentes con diabetes tipo 1 en tratamiento con un sistema integrado / The impact of «faster aspart» on blood glucose control in children and adolescents with type 1 diabetes treated using a sensor-augmented insulin pump

Introducción y objetivos: El control glucémico postprandial es fundamental para conseguir los objetivos metabólicos en pacientes con diabetes mellitus tipo 1 (DM1). La nueva insulina faster aspart presenta un perfil farmacológico caracterizado por una absorción e inicio de acción más rápidos, mayor disponibilidad precoz y menor incremento de la glucosa postprandial. El objetivo principal del estudio fue analizar su eficacia en pacientes con DM1 tratados con un sistema integrado.Pacientes y métodos: Estudio analítico, longitudinal, prospectivo y multicéntrico, evaluando el empleo de faster aspart durante tres meses en pacientes en edad pediátrica con DM1 con sistema integrado MiniMed640G® tratados previamente con insulina aspart. Al inicio y final del estudio se analizaron para posterior comparación: glucosa media, porcentajes de tiempo en objetivo, tiempo en hipoglucemia e hiperglucemia, área bajo la curva (AUC) < 70 y > 180 mg/dL, glucosa media pre y postprandial en comidas principales, necesidades diarias de insulina, porcentaje basal/bolo y HbA1c. Se registraron complicaciones agudas y eventos adversos, y se evaluó grado de satisfacción mediante encuesta.Resultados: Se incluyeron 31 pacientes de 13,49 ± 2,42 años de edad con DM1 de 7,0 ± 3,67 años de evolución. Faster aspart se asoció con menor porcentaje de tiempo en hiperglucemia > 180 mg/dL (25,8 ± 11,3 vs. 22,4 ± 9,5; p = 0,011) y > 250 mg/dL (5,2 ± 4,9 vs. 4,0 ± 3,6; p = 0,04) y AUC > 180 mg/dL (10,8 ± 6,5 vs. 9,3 ± 6,1; p = 0,03), incrementándose el tiempo en objetivo (71,4 ± 10,0 vs. 74,3 ± 9,2; p = 0,03) sin aumentar hipoglucemias. Las necesidades de insulina, porcentajes basal/bolo y HbA1c no se modificaron significativamente. Faster aspart fue bien tolerada y valorada por los participantes.Conclusiones: Faster aspart consigue un mejor control glucémico, aumentando el tiempo de glucosa en objetivo en niños y adolescentes con DM1 en tratamiento con un sistema integrado. (AU)

Background and aims: Post-prandial glucose control is essential to achieve metabolic goals in patients with type 1 diabetes mellitus (T1DM). The new «faster aspart» insulin has a pharmacological profile noted for its faster absorption and onset of action, and increased early availability, resulting in improved blood glucose control after meals. The main objective of the study was to analyse the efficacy of «faster aspart» vs. «insulin aspart» in children and adolescents with DM1 on sensor-augmented pump treatment.Patients and methods: Multicentre, longitudinal and prospective analytical trial evaluating the use of faster aspart insulin for three months in children with T1DM with MiniMed640G® sensor-augmented pumps previously treated with aspart insulin. At the beginning and end of the study the following variables were analysed for subsequent comparison: mean sensor glucose, percentage of time in range, hypoglycaemia and hyperglycaemia, area under the curve (AUC) < 70 and > 180 mg/dL, mean sensor glucose pre and postprandial in main meals, daily insulin requirements, basal/bolus percentage, and HbA1c. Acute complications, adverse events and satisfaction survey were assessed.Results: The study included 31 patients with a mean of 13.49 ± 2.42 years of age and with T1DM of 7.0 ± 3.67 years of onset. The use of faster aspart was associated with lower time in hyperglycaemia > 180 mg/dL (25.8 ± 11.3 vs. 22.4 ± 9.5; p = 0.011) and > 250 mg/dL (5.2 ± 4.9 vs. 4.0 ± 3.6; p = 0.04), lower AUC > 180 mg/dL (10.8 ± 6.5 vs. 9.3 ± 6.1; p = 0.03), and increased time in range (71.4 ± 10.0 vs. 74.3 ± 9.2; p = 0.03). No significant changes in hypoglycaemia, HbA1c, insulin requirements, and basal/bolus percentages were detected. Faster aspart was safe and well-evaluated by patients and caregivers.Conclusions: Faster aspart achieves better glycaemic control by increasing glucose time in range in children and adolescents with T1DM on treatment with sensor-augmented pumps. (AU)

The impact of "faster aspart" on blood glucose control in children and adolescents with type 1 diabetes treated using a sensor-augmented insulin pump.

BACKGROUND AND AIMS: Post-prandial glucose control is essential to achieve metabolic goals in patients with type 1 diabetes mellitus (T1DM). The new "faster aspart" insulin has a pharmacological profile noted for its faster absorption and onset of action, and increased early availability, resulting in improved blood glucose control after meals. The main objective of the study was to analyse the efficacy of "faster aspart" vs. "insulin aspart" in children and adolescents with DM1 on sensor-augmented pump treatment. PATIENTS AND METHODS: Multicentre, longitudinal and prospective analytical trial evaluating the use of faster aspart insulin for three months in children with T1DM with MiniMed640G® sensor-augmented pumps previously treated with aspart insulin. At the beginning and end of the study the following variables were analysed for subsequent comparison: mean sensor glucose, percentage of time in range, hypoglycaemia and hyperglycaemia, area under the curve (AUC) <70 and >180 mg/dL, mean sensor glucose pre- and postprandial in main meals, daily insulin requirements, basal/bolus percentage, and HbA1c. Acute complications, adverse events and satisfaction survey were assessed. RESULTS: The study included 32 patients with a mean of 13.49 ± 2.42 years of age and with T1DM of 7.0 ± 3.67 years of onset. The use of faster aspart was associated with lower time in hyperglycaemia >180 mg/dL (25.8 ± 11.3 vs. 22.4 ± 9.5; p = .011) and >250 mg/dL (5.2±4.9 vs. 4.0 ± 3.6; p = .04), lower AUC >180 mg/dL (10.8 ± 6.5 vs. 9.3 ± 6.1; p = .03), and increased time in range (71.4 ± 10.0 vs. 74.3 ± 9.2; p = .03). No significant changes in hypoglycaemia, HbA1c, insulin requirements, and basal/bolus percentages were detected. Faster aspart was safe and well-evaluated by patients and caregivers. CONCLUSIONS: Faster aspart achieves better glycaemic control by increasing glucose time in range in children and adolescents with T1DM on treatment with sensor-augmented pumps.

Impact on the well-being perceived by caregivers of children and adolescents with type 1 diabetes following the use of interstitial glucose measurement systems.

INTRODUCTION: Type 1 diabetes mellitus (DM-1) is one of the most common chronic childhood diseases, and it is essential to optimize glycemic control in order to avoid complications. For years, interstitial glucose measurement systems (MGI systems) have been among the new technologies at the forefront of self-care. OBJECTIVES: To determine the impact on the well-being of the caregivers of patients with DM-1 under 18 years of age, controlled at a Pediatric Diabetes Unit of a third level hospital, of the use of MGI systems. MATERIAL AND METHODS: This was an observational, descriptive and analytical cohort study based on a questionnaire completed by the patients' caregivers, as well as from the patient's clinical history. RESULTS: There were 120 participants (55.5% males), with a mean age 13.20 +/- 3.71 years and mean glycosylated haemoglobin (HbA1c) 7.36% +/- 0.90. 52.5% of the sample used MGI systems. The caregivers of patients using MGI systems showed significantly higher scores (p < 0.05) regarding well-being, compared to the caregivers of patients not using this technology. In the former, a significant improvement (p < 0.05) in these variables with respect to the values prior to the beginning of their use was observed. CONCLUSIONS: The use of MGI systems for diabetes self-management in our study led to a greater sense of well-being on the part of caregivers compared with before their introduction, as well as in comparison with those who continued to perform measurements using daily capillary glycemias.

Raquitismo carencial en un paciente de raza negra / Vitamin D deficiency as cause of rickets in a patient of African origin

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[Growth hormone deficit: Influence of puberty on the response to treatment]. / Déficit de hormona de crecimiento: influencia de la pubertad en la respuesta al tratamiento.

INTRODUCTION: Short stature is the most frequent reason for consultation in Pediatric Endocrinology consultations and sometimes requires treatment with growth hormone. The aim of the study was to analyze the response to treatment based on its onset in pubertal or prepubertal stages and to analyze the possible benefit of an early onset. PATIENTS AND METHODS: Longitudinal, retrospective and observational study in 139 patients treated for idiopathic growth hormone deficiency up to adult height. MAIN VARIABLES STUDIED: (a) genetic background: maternal, paternal and genetic height; (b) perinatal history; (c) anthropometry during follow-up and at pubertal onset: weight, height, body mass index; (d) variables during follow-up and at pubertal onset: growth rate, bone age and growth prognosis. Final response variables: adult height, adult height with respect to target height, adult height with respect to initial growth prediction, adult height with respect to initial height at the start of treatment and adult height with respect to height at pubertal onset. RESULTS: Total pubertal gain was 0.84±0.6 SD. 61.9% of the patients started treatment with rhGH in prepuberty. The initiation of treatment in the prepubertal stage and a higher total pubertal gain are correlated with a better final height (P=.001 and r=0.507, P=.00, respectively). Furthermore, a longer duration of treatment in pre-puberty is correlated with a better final response (r=0.328, P=.00). CONCLUSIONS: The start of treatment in the prepubertal stage and its longer duration during this period are determining factors to achieve a good long-term response. Total pubertal gain was greater in patients who started treatment in the pubertal stage.

Growth hormone deficit. Does the first year of treatment influence adult height? / Déficit de hormona de crecimiento, ¿influye el primer año de tratamiento en la talla final?

INTRODUCTION: Short stature is the most frequent reason for Pediatric Endocrinology consultations and sometimes requires treatment with growth hormone. OBJECTIVE: The possible correlation of a good response to any early response factor with a better final response was studied, and also whether there was a difference in response to treatment according to the type of deficit. PATIENTS AND METHODS: This was a longitudinal, retrospective and observational study of 139 patients treated for idiopathic growth hormone deficiency up to adult height. There were good response criteria in the first year of treatment: a) an increase in growth rate≥3cm / year, b) a growth rate≥1 standard deviation (SD), c) an increase in height≥0.5 SD, d) an increase in height≥0.3 SD. Study of the Index of Responsiveness to treatment in the first and second year. Final response variables: adult height with respect to target height, adult height with respect to initial growth prediction and adult height with respect to initial height at the start of treatment. The possible correlation of a good response to any of the early response factors with a better final response to treatment was studied, and also whether there was a difference in the response to treatment according to the type of deficit. RESULTS: The treatment produced a gain in adult height with respect to genetic height of 0.06±0.7 SD. Patients considered good responders in the first year of treatment presented a better final response (growth rate≥3cm: p=0.000, growth rate≥1 SD: p=0.008, height gain≥0.5 SD: P=0.007, height gain≥0.3 SD: P=0.006), as well as patients with a severe deficit (P=0.04). The index of responsiveness to treatment during the first year was associated with a better final response (r=0.249, P=0.003), with this correlation being maintained in the second year (r=0.294, P=0.01). CONCLUSIONS: Growth hormone treatment increased height in the genetic target. The percentage of good responders varied depending on the criteria used. The response in the first year of treatment and a severe deficit were determining factors for achieving a good long-term response.

Impact on the well-being perceived by caregivers of children and adolescents with type 1 diabetes following the use of interstitial glucose measurement systems. / Impacto en el bienestar percibido por cuidadores de niños y adolescentes con diabetes tipo 1 mediante la utilización de sistemas de medición de glucosa intersticial.

INTRODUCTION: Type 1 diabetes mellitus (DM-1) is one of the most common chronic childhood diseases, and it is essential to optimize glycemic control in order to avoid complications. For years, interstitial glucose measurement systems (MGI systems) have been among the new technologies at the forefront of self-care. OBJECTIVES: To determine the impact on the well-being of the caregivers of patients with type 1 diabetes mellitus under 18 years of age, controlled at a Pediatric Diabetes Unit of a third level hospital, of the use of MGI systems. MATERIAL AND METHODS: This was an observational, descriptive and analytical cohort study based on a questionnaire completed by the patients' caregivers, as well as from the patient's clinical history. RESULTS: There were 120 participants (55.5% males), with a mean age 13.20+/-3.71 years and mean glycosylated haemoglobin (HbA1c) 7.36%+/-0.90. 52.5% of the sample used MGI systems. The caregivers of patients using MGI systems showed significantly higher scores (p<.05) regarding well-being, compared to the caregivers of patients not using this technology. In the former, a significant improvement (p<.05) in these variables with respect to the values prior to the beginning of their use was observed. CONCLUSIONS: The use of MGI systems for diabetes self-management in our study led to a greater sense of well-being on the part of caregivers compared with before their introduction, as well as in comparison with those who continued to perform measurements using daily capillary glycemias.

Osteogénesis imperfecta: análisis de 40 pacientes / Osteogenesis imperfecta: Review of 40 patients

INTRODUCCIÓN: La osteogénesis imperfecta (OI) es una enfermedad genética heterogénea manifestada como fragilidad ósea y fracturas. PACIENTES Y MÉTODOS: Estudio descriptivo retrospectivo analizando características clínicas, genéticas y tratamiento de pacientes diagnosticados de OI (1989-2017) en el Hospital Universitario Miguel Servet, Zaragoza (Endocrinología Pediátrica y Reumatología). RESULTADOS: Incluidos 40 pacientes; 32,5% varones, 67,5% mujeres; 29 niños, 11 adultos. Media de fracturas al diagnóstico en OI leve 4,6±6,4 (edad media al diagnóstico 7,8±12,8años), en OI moderada 1,7±2,4 (edad media al diagnóstico 0,04±0,3años), en OI grave 3,7±2,1 y en OI muy grave 12,5±7,8, ambos grupos diagnosticados al nacimiento. Estudio genético en 32 pacientes, 25 con variante patogénica/probablemente patogénica, siendo COL1A1 el gen más frecuentemente afectado. En 7 pacientes no fue encontrada la variante responsable, 5 con confirmación diagnóstica (estudio bioquímico colágenoI). Tratamiento con bifosfonatos 19 pacientes; 7 asociando hormona de crecimiento. Los tratados con bifosfonatos han presentado mejoría clínica (reducción de dolor óseo y/o irritabilidad) y reducción del número de fracturas. CONCLUSIONES: El gen COL1A1 es el más frecuentemente afectado en nuestros pacientes. El tratamiento debe ser multidisciplinar y el uso de bifosfonatos proporciona mejoría

INTRODUCTION: Osteogenesis imperfecta (OI) is a heterogeneous genetic disease manifesting as bone fragility and fractures. PATIENTS AND METHODS: Retrospective descriptive study analysing clinical and genetic features, and treatment of patients with OI. RESULTS: Forty patients were included; 32.5% males, 67.5% females; 29 children, 11 adults. Number of fractures at diagnosis with mild OI was 4.6±6.4 (average age at diagnosis 7.8±12.8years), with moderate OI 1.7±2.4 (age at diagnosis .04±.3years), in severe OI 3.7±2.1 and in extremely severe forms 12.5±7.8, both groups diagnosed at birth. Genetic study in 32 patients, 25 with a positive genetic study (pathogenic/probably pathogenic variant). COL1A1 gene was the most frequently affected. In 7 patients, no pathogenic or probably pathogenic variant was found (5 diagnosed by biochemical study of typeI collagen). Nineteen patients were treated with bisphosphonates; 7 combined with growth hormone. The patients treated with bisphosphonates showed clinical improvement (reduction of bone pain and/or irritability) and reduction of fractures. CONCLUSIONS: The COL1A1 gene is the most frequently affected. OI patients should receive multidisciplinary management and bisphosphonates can improve their quality of life

Crecimiento hasta edad adulta en una población nacida pequeña para la edad gestacional tratada con hormona de crecimiento / Adult height in short children born small for gestational age treated with growth hormone

ANTECEDENTES Y OBJETIVO: El tratamiento con hormona de crecimiento recombinante humana (rhGH) en niños nacidos pequeños para la edad gestacional (PEG) se ha mostrado eficaz, aunque con variedad en la respuesta. Se evalúa la talla adulta y los factores que determinan la respuesta a largo plazo. PACIENTES Y MÉTODOS: Estudio retrospectivo de 80 pacientes PEG tratados con rhGH por baja talla y seguidos hasta la talla adulta (23 varones). RESULTADOS: El grupo que inició el tratamiento antes de la pubertad alcanzó mayor talla adulta (−1,4±0,6 vs. −1,9±0,6 púberes). Existió mayor ganancia de talla en los tratados durante ≥ 2 años en prepubertad (1,32±0,5 SDE). Los factores asociados con una mayor ganancia de talla fueron: a) la menor talla, peso e IMC al inicio; b) la menor edad cronológica, ósea y el menor nivel de IGF-I iniciales; c) la mayor distancia con la talla genética; d) la mayor velocidad de crecimiento el primer y el segundo año y la mayor ganancia de talla previa y durante la pubertad. El porcentaje de buena respuesta en el primer año varió entre el 46,6 y el 81,6% en función del criterio empleado. El incremento de la velocidad de crecimiento≥3cm/año es el que mejor se relaciona con buena respuesta a largo plazo. CONCLUSIONES: El tratamiento con rhGH en niños PEG produce un incremento variable de talla adulta, que les permite alcanzar su rango genético. Los mejores resultados se producen en el grupo con mayor número de años en tratamiento en la prepubertad y no dependen de la respuesta hipofisaria de GH

BACKGROUND AND OBJECTIVE: Recombinant human growth hormone (rhGH) treatment in small for gestational age (SGA) children has been effective, although there is significant variability in the response. Adult height and the factors that determine the long-term response are evaluated. Patients and DESIGN: A retrospective study of 80 patients born SGA with short stature treated with rhGH and followed until adult height (23 males). RESULTS: The group starting treatment pre-puberty reached a higher Adult height (−1.4±0.6 vs. −1.9±.6 in pubertal children), the highest final height gain was achieved in those treated for at least 2years prepuberty (1.32±.5 SDS). Factors associated with greater adult height gain were: a) less height, weight and BMI at start of treatment, b) lower chronological and bone age with lower IGF-I before treatment, c) greater distance to target height, d) higher growth velocity the first and second year of treatment, and higher height gain before and during puberty. The percentage of patients with good response in the first year ranged from 46.6% to 81.6% depending on the criteria. Growth velocity increase ≥3cm/ first year correlated best with long-term response. CONCLUSION: rhGH treatment in children born SGA produced a varying increase in adult height that allowed them to reach their adult height. The best results occurred in the prepubertal group and did not depend on pituitary GH response

Osteogenesis imperfecta: Review of 40 patients. / Osteogénesis imperfecta: análisis de 40 pacientes.

INTRODUCTION: Osteogenesis imperfecta (OI) is a heterogeneous genetic disease manifesting as bone fragility and fractures. PATIENTS AND METHODS: Retrospective descriptive study analysing clinical and genetic features, and treatment of patients with OI. RESULTS: Forty patients were included; 32.5% males, 67.5% females; 29 children, 11 adults. Number of fractures at diagnosis with mild OI was 4.6±6.4 (average age at diagnosis 7.8±12.8years), with moderate OI 1.7±2.4 (age at diagnosis .04±.3years), in severe OI 3.7±2.1 and in extremely severe forms 12.5±7.8, both groups diagnosed at birth. Genetic study in 32 patients, 25 with a positive genetic study (pathogenic/probably pathogenic variant). COL1A1 gene was the most frequently affected. In 7 patients, no pathogenic or probably pathogenic variant was found (5 diagnosed by biochemical study of typeI collagen). Nineteen patients were treated with bisphosphonates; 7 combined with growth hormone. The patients treated with bisphosphonates showed clinical improvement (reduction of bone pain and/or irritability) and reduction of fractures. CONCLUSIONS: The COL1A1 gene is the most frequently affected. OI patients should receive multidisciplinary management and bisphosphonates can improve their quality of life.

[The impact of «faster aspart¼ on blood glucose control in children and adolescents with type 1 diabetes treated using a sensor-augmented insulin pump]. / Impacto de faster aspart sobre el control glucémico en niños y adolescentes con diabetes tipo 1 en tratamiento con un sistema integrado.

BACKGROUND AND AIMS: Post-prandial glucose control is essential to achieve metabolic goals in patients with type 1 diabetes mellitus (T1DM). The new «faster aspart¼ insulin has a pharmacological profile noted for its faster absorption and onset of action, and increased early availability, resulting in improved blood glucose control after meals. The main objective of the study was to analyse the efficacy of «faster aspart¼ vs. «insulin aspart¼ in children and adolescents with DM1 on sensor-augmented pump treatment. PATIENTS AND METHODS: Multicentre, longitudinal and prospective analytical trial evaluating the use of faster aspart insulin for three months in children with T1DM with MiniMed640G® sensor-augmented pumps previously treated with aspart insulin. At the beginning and end of the study the following variables were analysed for subsequent comparison: mean sensor glucose, percentage of time in range, hypoglycaemia and hyperglycaemia, area under the curve (AUC) < 70 and > 180 mg/dL, mean sensor glucose pre and postprandial in main meals, daily insulin requirements, basal/bolus percentage, and HbA1c. Acute complications, adverse events and satisfaction survey were assessed. RESULTS: The study included 31 patients with a mean of 13.49 ± 2.42 years of age and with T1DM of 7.0 ± 3.67 years of onset. The use of faster aspart was associated with lower time in hyperglycaemia > 180 mg/dL (25.8 ± 11.3 vs. 22.4 ± 9.5; p = 0.011) and > 250 mg/dL (5.2 ± 4.9 vs. 4.0 ± 3.6; p = 0.04), lower AUC > 180 mg/dL (10.8 ± 6.5 vs. 9.3 ± 6.1; p = 0.03), and increased time in range (71.4 ± 10.0 vs. 74.3 ± 9.2; p = 0.03). No significant changes in hypoglycaemia, HbA1c, insulin requirements, and basal/bolus percentages were detected. Faster aspart was safe and well-evaluated by patients and caregivers. CONCLUSIONS: Faster aspart achieves better glycaemic control by increasing glucose time in range in children and adolescents with T1DM on treatment with sensor-augmented pumps.

Adult height in short children born small for gestational age treated with growth hormone. / Crecimiento hasta edad adulta en una población nacida pequeña para la edad gestacional tratada con hormona de crecimiento.

BACKGROUND AND OBJECTIVE: Recombinant human growth hormone (rhGH) treatment in small for gestational age (SGA) children has been effective, although there is significant variability in the response. Adult height and the factors that determine the long-term response are evaluated. PATIENTS AND DESIGN: A retrospective study of 80 patients born SGA with short stature treated with rhGH and followed until adult height (23 males). RESULTS: The group starting treatment pre-puberty reached a higher Adult height (-1.4±0.6 vs. -1.9±.6 in pubertal children), the highest final height gain was achieved in those treated for at least 2years prepuberty (1.32±.5 SDS). Factors associated with greater adult height gain were: a) less height, weight and BMI at start of treatment, b) lower chronological and bone age with lower IGF-I before treatment, c) greater distance to target height, d) higher growth velocity the first and second year of treatment, and higher height gain before and during puberty. The percentage of patients with good response in the first year ranged from 46.6% to 81.6% depending on the criteria. Growth velocity increase ≥3cm/ first year correlated best with long-term response. CONCLUSION: rhGH treatment in children born SGA produced a varying increase in adult height that allowed them to reach their adult height. The best results occurred in the prepubertal group and did not depend on pituitary GH response.