59 research outputs found
Growth hormone treatment modalities in girls with Turner syndrome
The November 1938 issue of Endocrinology published a paper by the
American physician Henry Turner which described seven females exhibiting
certain physical features including sh0l1 stature, sexual infantilism, webbing of
the neck, low posterior hairline, and increased carrying angle of the elbow.
This syndrome now bears his name, although the Munich paediatrician Otto
Ullrich had already described patients with similar physical characteristics in
1930. For this reason, German studies refer to this syndrome as Ullrich-Turner
syndrome
Bone mineral density assessed by phalangeal radiographic absorptiometry before and during long-term growth hormone treatment in girls with Turner's syndrome participating in a randomized dose-response study
To assess bone mineral density (BMD) in girls with Turner's syndrome
before and during long-term treatment with GH, longitudinal measurements
using phalangeal radiographic absorptiometry were performed in 68 girls
with Turner's syndrome. These previously untreated girls, age 2-11 y,
participating in a randomized, dose-response trial, were randomly assigned
to one of three GH dosage groups: group A, 4 IU/m(2)/d ( approximately
0.045 mg/kg/d); group B, first year 4 IU/m(2)/d, thereafter 6 IU/m(2)/d (
approximately 0.0675 mg/kg/d); or group C, first year 4 IU/m(2)/d, second
year 6 IU/m(2)/d, thereafter 8 IU/m(2)/d ( approximately 0.090 mg/kg/d).
In the first 4 y of GH treatment, no estrogens for pubertal induction were
prescribed to the girls. Thereafter, girls started with 17beta-estradiol
(5 microg/kg body weight/d, orally) when they had reached the age of 12 y.
BMD results were adjusted for bone age and sex, and expressed as SD scores
using reference values of healthy Dutch girls. At baseline, almost every
individual BMD value of bone consisting predominantly of cortical bone, as
well as that of bone consisting predominantly of trabecular bone, was
within the normal range of healthy girls and the SD scores were not
significantly different from zero [mean (SE) 0.38 (0.22) and -0.04
(0.13)]. During 7 y of GH treatment, BMD SD scores showed a significant
increase to values significantly higher than zero [mean (SE) 0.87 (0.15)
and 0.95 (0.14)]. The increment in BMD SD score of bone consisting
predominantly of cortical bone was significantly higher in group C
compared with that of the other two GH dosage groups. The pretreatment
bone age was significantly negatively related to the increment in BMD SD
score. We found no significant influence of spontaneous puberty or the use
of low-dose estrogens in the last 3 y of the study period on the increment
in BMD SD score during 7 y of GH treatment. In conclusion, most untreated
young girls with Turner's syndrome have a normal volumetric BMD. During 7
y of GH treatment with 4, 6, or 8 IU/m(2)/d, the BMD SD score increased
significantly
Towards a nanospecific approach for risk assessment.
In the current paper, a new strategy for risk assessment of nanomaterials is described, which builds upon
previous project outcomes and is developed within the FP7 NANoREG project. NANoREG has the aim to
develop, for the long term, new testing strategies adapted to a high number of nanomaterials where
many factors can affect their environmental and health impact. In the proposed risk assessment strategy,
approaches for (Quantitative) Structure Activity Relationships ((Q)SARs), grouping and read-across are
integrated and expanded to guide the user how to prioritise those nanomaterial applications that may
lead to high risks for human health. Furthermore, those aspects of exposure, kinetics and hazard
assessment that are most likely to be influenced by the nanospecific properties of the material under
assessment are identified. These aspects are summarised in six elements, which play a key role in the
strategy: exposure potential, dissolution, nanomaterial transformation, accumulation, genotoxicity and
immunotoxicity.
With the current approach it is possible to identify those situations where the use of nanospecific
grouping, read-across and (Q)SAR tools is likely to become feasible in the future, and to point towards the
generation of the type of data that is needed for scientific justification, which may lead to regulatory
acceptance of nanospecific applications of these tools.The research leading to these results has been partially funded
by the European Union Seventh Framework Programme (FP7/
2007e2013) under the project NANoREG (A common European
approach to the regulatory testing of nanomaterials), grant agreement
310584.info:eu-repo/semantics/publishedVersio
A Dutch paediatric palliative care guideline: a systematic review and evidence-based recommendations for symptom treatment
Background: Children with life-threatening and life-limiting conditions can experience high levels of suffering due to multiple distressing symptoms that result in poor quality of life and increase risk of long-term distress in their family members. High quality symptom treatment is needed for all these children and their families, even more so at the end-of-life. In this paper, we provide evidence-based recommendations for symptom treatment in paediatric palliative patients to optimize care. Methods: A multidisciplinary panel of 56 experts in paediatric palliative care and nine (bereaved) parents was established to develop recommendations on symptom treatment in paediatric palliative care including anxiety and depression, delirium, dyspnoea, haematological symptoms, coughing, skin complaints, nausea and vomiting, neurological symptoms, pain, death rattle, fatigue, paediatric palliative sedation and forgoing hydration and nutrition. Recommendations were based on evidence from a systematic literature search, additional literature sources (such as guidelines), clinical expertise, and patient and family values. We used the GRADE methodology for appraisal of evidence. Parents were included in the guideline panel to ensure the representation of patient and family values. Results: We included a total of 18 studies that reported on the effects of specific (non) pharmacological interventions to treat symptoms in paediatric palliative care. A few of these interventions showed significant improvement in symptom relief. This evidence could only (partly) answer eight out of 27 clinical questions. We included 29 guidelines and two textbooks as additional literature to deal with lack of evidence. In total, we formulated 221 recommendations on symptom treatment in paediatric palliative care based on evidence, additional literature, clinical expertise, and patient and family values. Conclusion: Even though available evidence on symptom-related paediatric palliative care interventions has increased, there still is a paucity of evidence in paediatric palliative care. We urge for international multidisciplinary multi-institutional collaboration to perform high-quality research and contribute to the optimization of symptom relief in palliative care for all children worldwide
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