The Impact of COVID-19 Regulations on Adherence to Recombinant Human Growth Hormone Therapy: Evidence from Real-World Data

Worldwide regulations during COVID-19 positively and negatively impacted self-management in paediatric patients with chronic medical conditions. We investigated the impact of regulations on adherence to recombinant human growth hormone (r-hGH) therapy in paediatric patients with growth disorders, using real-world adherence data extracted March...

Curve matching to predict growth in patients receiving growth hormone therapy: An interpretable & explainable method

Curve matching may be used to predict growth outcomes using data of patients whose growth curves resemble those of a new patient with growth hormone deficiency (GHD) and those born small for gestational age (SGA). We aimed to investigate the validity of curve matching to predict growth in patients with GHD and those born SGA receiving...

Analysis of real-world data on growth hormone therapy adherence using a connected injection device

Background. Poor adherence to long-term recombinant human growth hormone (r-hGH) treatment can lead to suboptimal clinical outcomes; consequently, supporting and monitoring adherence is a crucial part of patient management. We assessed adherence to r-hGH treatment in children with growth disorders over 48 months using a connected monitoring...

Application of the Dutch, Finnish and British Screening Guidelines in a Cohort of Children with Growth Failure

Aims: To evaluate three guidelines for selecting short children for diagnostic workup in a general pediatric clinic. Methods: All patients (n = 131) aged 3.00-9.99 years who were referred for growth failure to a general pediatric clinic were evaluated for their medical history and growth and examined. All of them underwent the same standardized...

Association between head circumference and body size

Background/Aims: Studies on the association between head circumference (HC) and height or weight have shown variable results. Methods: Using data from the Dutch nationwide survey performed in 1997 (n = 14,500), we calculated correlations for different ages, and fitted a regression model for the estimation of HC. HC versus height charts were...

Jgz-richtlijn: 'Signalering van en verwijscriteria bij kleine lichaamslengte' [Guideline for preventive child health care: 'Detection and referral criteria in short stature']

The main goal of this guideline for preventive child health care (PCHC) is to improve early detection of disorders that induce short stature. Based on research, evidence-based referral criteria for children aged 0-10 years with a short stature were formulated. These criteria are important for all professionals working with children, such as PCHC...

Long-term health and quality-of-life consequences of mass screening for childhood celiac disease: A 10-year follow-up study

OBJECTIVE. Mass screening for celiac disease is controversial. The objective of this study was to determine whether detection of childhood celiac disease by mass screening improves long-term health status and health-related quality of life. METHODS.We conducted a prospective 10-year follow-up study of 32 children who were aged 2 to 4 years, had...

Developing evidence-based guidelines for referral for short stature

Objective: To establish evidence based guidelines for growth monitoring on a population basis. Study design: Several auxological referral criteria were formulated and applied to longitudinal growth data from four different patient groups, as well as three samples from the general population. Results: Almost 30% of pathology can be detected by...

Improved accuracy when screening for human growth disorders by likelihood ratios

The standard deviation score (SDS) is a powerful tool for screening for growth-related problems. However, referral rules of the type 'if SDS(Y)<d, then refer' (for some constant d) are not optimal for answering the question: 'Does this child with measurement Y belong to the reference or to the diseased population?'. If the growth standard for...

The diagnostic work up of growth failure in secondary health care ; An evaluation of consensus guidelines

Background: As abnormal growth might be the first manifestation of undetected diseases, it is important to have accurate referral criteria and a proper diagnostic work-up. In the present paper we evaluate the diagnostic work-up in secondary health care according to existing consensus guidelines and study the frequency of underlying medical...

Referral patterns of children with poor growth in primary health care

Background. To promote early diagnosis and treatment of short stature, consensus meetings were held in the mid nineteen nineties in the Netherlands and the UK. This resulted in guidelines for referral. In this study we evaluate the referral pattern of short stature in primary health care using these guidelines, comparing it with cut-off values...

Growth monitoring and diagnostic work-up of short stature: An International Inventorization

Background/Aims: Growth monitoring is almost universally performed, but few data are available on which referral criteria and diagnostic work-up are used worldwide for children with short stature. Methods: A short questionnaire, containing questions on auxological screening and on diagnostic criteria for short stature, was sent to all members of...

Nationwide age references for sitting height, leg length, and sitting height/height ratio, and their diagnostic value for disproportionate growth disorders

Aims: To obtain age references for sitting height (SH), leg length (LL), and SH/H ratio in the Netherlands; to evaluate how SH standard deviation score (SDS), LL SDS, SH/H SDS, and SH/LL SDS are related to height SDS; and to study the usefulness of height corrected SH/H cut-off lines to detect Marfan syndrome and hypochondroplasia. Methods:...

Many referrals under Dutch short stature guidelines

The number of referrals under strict adherence to the Dutch consensus guidelines for short stature was estimated using longitudinal data from 970 children (0-10 years). Results showed that over 38% of all children would have to be referred one or more times. There is therefore a clear need to revise the current guidelines.

Worm plot: A simple diagnostic device for modelling growth reference curves

The worm plot visualizes differences between two distributions, conditional on the values of a covariate. Though the worm plot is a general diagnostic tool for the analysis of residuals, this paper focuses on an application in constructing growth reference curves, where the covariate of interest is age. The LMS model of Cole and Green is used to...

Defective collagen crosslinking in bone, but not in ligament or cartilage, in bruck syndrome: Indications for a bone-specific telopeptide lysyl hydroxylase on chromosome 17

Bruck syndrome is characterized by the presence of osteoporosis, joint contractures, fragile bones, and short stature. We report that lysine residues within the telopeptides of collagen type I in bone are underhydroxylated, leading to aberrant crosslinking, but that the lysine residues in the triple helix are normally modified. In contrast to...