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Current Pediatric Reviews
ISSN: 1573-3963
Current Pediatric Reviews
Volume 2, Number 2, May 2006
Contents
Cystic Fibrosis Gene Therapy: Key Questions and Prospects
Pp. 99-105
Isabelle Fajac, Stéphanie Grosse, Annie-Claude
Roche and Michel Monsigny
[Abstract]
Not Just any Fat for Cystic Fibrosis? Docosahexaenoic
Acid in Cystic Fibrosis Pp. 107-113
Stephanie van Biervliet, Jean-Pierre van Biervliet, Eddy
Robberecht and Armand Christophe
[Abstract]
Specific Nutritional Needs for Children: Are Children
Small Adults? Pp. 115-122
Yvan Vandenplas, Bruno Hauser and Jean De Schepper
[Abstract]
Severe Meningococcal Infections in Children and Adolescents
Pp. 123-130
Alan Graham and Brahm Goldstein
[Abstract]
Cellular Mechanisms in Perinatal Hypoxic-Ischemic
Brain Injury Pp. 131-141
Enrique Hilario, Antonia Alvarez, Francisco J. Alvarez,
Elena Gastiasoro and Adolf Valls-i-Soler
[Abstract]
Evaluating a Child with Partial Developmental
Delay (ParDD), Global Developmental Delay (GDD)/Mental Retardation
(MR): Clinical Expertise Based or Evidence-Based? Pp.
143-153
Virginia C.N. Wong and Brian H.Y. Chung
[Abstract]
Update on the Treatment of Childhood Asthma
Pp. 155-164
H. William Kelly
[Abstract]
Childhood Systemic Lupus Erythematosus: New and Old
Treatments Pp. 165-171
Brigitte Bader-Meunier and Elie Haddad
[Abstract]
Primary Vesicoureteric Reflux and Reflux Nephropathy
– New Insights Pp. 173-176
Laura Santoro, Alessandra Ferrarini, Laura Crosazzo
and Mario G. Bianchetti
[Abstract]
Recent Advances in Minimal Access Surgery for Infants
and Children Pp. 177-186
Ken K.Y. Wong and Paul K.H. Tam
[Abstract]
Abstracts
[Back to top]
Cystic Fibrosis Gene Therapy: Key Questions and Prospects
Isabelle Fajac, Stéphanie Grosse, Annie-Claude
Roche and Michel Monsigny
Cystic fibrosis is a monogenic disorder with significant
morbidity and mortality, despite advances in conventional
treatment. It is a good candidate for gene therapy and this
field has progressed rapidly since the cystic fibrosis transmembrane
conductance regulator gene was cloned. We will review the
specific questions to address for successful cystic fibrosis
gene therapy, such as the extra- and intracellular barriers
to airway gene transfer, the target cells and the endpoints
to assess efficacy. We will discuss recent advances in viral
and nonviral gene transfer agents, delivery techniques and
novel strategies to enhance airway gene transfer and expression.
[Back to top]
Not Just any Fat for Cystic Fibrosis? Docosahexaenoic
Acid in Cystic Fibrosis
Stephanie van Biervliet, Jean-Pierre van Biervliet, Eddy
Robberecht and Armand Christophe
Once the genetic defect of cystic fibrosis (CF), the most
common autosomal recessive disease, was discovered, there
was hope for quick positive results with gene-therapy. These
did not came true, however. Recent CF mice studies by Freedman
et al. demonstrated a disease reversal by high doses
of docosahexaenoic acid (DHA). Although it is known for long
that the same essential fatty acid imbalance with low levels
of DHA is present in CF patients and that DHA has anti-inflammatory
actions, caution is urged to prevent false expectations when
we extrapolate mice model results. This review explores the
present scientific data on DHA supplementation in CF.
[Back to top]
Specific Nutritional Needs for Children: Are Children
Small Adults?
Yvan Vandenplas, Bruno Hauser and Jean De Schepper
Nutrition is age specific, in all aspects. The intake of
young children depends on their caretakers. It is obvious
that growth and development cause major changes in children.
However, these changes not only consider size but also body
composition and maturation of many (digestive) functions.
Energy and nutrient requirements do not only depend on intake,
but also on expenditure. The latter depends on many variables
such as physical activity.
Physiological needs for calories differ according to age.
Also the needs for macro- and micronutrients are age-dependent.
As a consequence, it is impossible to deduce recommendations
for children from adult data.
Because of changing physical activity, and because of errors
in earlier techniques to measure intake and expenditure, recommendations
for energy intake may have to be updated.
[Back to top]
Severe Meningococcal Infections in Children and Adolescents
Alan Graham and Brahm Goldstein
Infection with Neisseria meningitidis remains one
of the most devastating illnesses in pediatrics. Affected
patients can progress from a mild viral-like illness to death
within a matter of hours. Additionally, the definitive clinical
diagnosis cannot be reached until the characteristic petechial
rash appears, making differentiating meningococcal infection
from a benign viral illness impossible. This review will discuss
the epidemiology, bacteriology, pathophysiology, treatment,
and potential new therapies for severe meningococcal infections
in children and adolescents.
[Back to top]
Cellular Mechanisms in Perinatal Hypoxic-Ischemic
Brain Injury
Enrique Hilario, Antonia Alvarez, Francisco J. Alvarez,
Elena Gastiasoro and Adolf Valls-i-Soler
Hypoxia-ischemia is responsible for a high morbidity and
mortality rate, both in infant and adult patients. In humans,
the brain is immature from fetal to postnatal life, demonstrating
more specific vulnerability than the adult one, induced by
exposure at perinatal asphyxia. The extension and distribution
of brain lesions depend to a large extent on the intensity
and exposure to injury, the maturational stage, the affected
brain region and the survival time of the patient. Different
cellular events are developed in response to hypoxic-ischemic
injury. These events have been related to the excitotoxicity
(activation of glutamate receptors), energy failure (decrease
in ATP levels), inflammatory cascade (delivery of inflammatory
mediators), and gene and transcriptional activation.
[Back to top]
Evaluating a Child with Partial Developmental Delay
(ParDD), Global Developmental Delay (GDD)/Mental Retardation
(MR): Clinical Expertise Based or Evidence-Based?
Virginia C.N. Wong and Brian H.Y. Chung
Developmental delay (DD) is a commonly encountered clinical
problem. Nearly most children with neurodevelopmental disorders
present with developmental delay. The ultimate diagnosis may
take months or even years to be confirmed. The final verdict
may be purely developmental with a benign outcome, or there
might be slow deterioration ending up in a more sinister cause
like neurodegenerative diseases. At times, the developmental
course might plateau off with the final developmental outcome
underachieved. With standardized assessment at a later age,
these children could then be labeled as suffering from “mental
retardation’ (MR) or “intellectual disability”
(ID). Thus, the crucial issue during the process of making
the final diagnosis for any child presenting with developmental
delay is when can one decide that an etiological diagnosis
can confidently be made during the phase of development. When
can one be really sure that there is no underlying organic
cause to account for the developmental delay. Often than not,
the clinician is faced with the dilemma of how exhaustive
one should persue in investigating a case of DD, and when
to stop investigating further. It is very important for us
to develop a logical and practical approach in managing these
DD children at the “first” presentation and to
establish one’s clinical judgment to find the “Best
timing” for referral or diagnostic workup. The first
step to the approach is the awareness and the differentiation
of Partial Developmental Delay (ParDD) from Global Developmental
Delay (GDD). We aim to provide the readers an understanding
of a practical and evidence based approach for the diagnostic
evaluation of a child with DD/MR. We hope this approach can
help to improve the diagnostic yield of DD/MR.
[Back to top]
Update on the Treatment of Childhood Asthma
H. William Kelly
The prevalence of asthma in children continues to increase
in many parts of the world, with the highest increases in
industrialized urban societies. Although the exact cause of
the increase remains undefined the “hygiene hypothesis”
where lower exposures to infectious agents that stimulate
the production of Th1 lymphocytes thus allowing proliferation
of Th2 lymphocytes that mediate allergic diseases has gained
popularity. Asthma is an inflammatory disorder of the airways
mediated by eosinophils and Th2 cytokines. Thus far, efforts
at primary prevention have fallen short and therapy is targeted
at preventing symptoms, morbidity and mortality and improving
the patient’s quality of life. International guidelines
recommend a stepwise approach to therapy with the inhaled
corticosteroids as the most effective therapy for children
with all levels of persistent asthma. Patients with intermittent
disease (symptoms less than 2 times weekly) can be effectively
treated with as needed short-acting β2
agonists. Leukotriene receptor antagonists and cromolyn/nedocromil
are alternatives to the inhaled corticosteroids though not
as effective. Children inadequately controlled on low daily
doses of inhaled corticosteroids can increase the dosage or
attempt adjunctive therapy with long-acting inhaled β2
agonists or leukotriene receptor antagonists. In infants and
young children less than 5 years of age adjunctive therapy
has not been studied. Both inhaled corticosteroids and leukotriene
receptor antagonists reduce the risk of exacerbations in wheezing
infants. Comparative studies have not been done. Attention
has to be paid to delivery systems for aerosolized drugs in
children and patient acceptance and compliance requires monitoring.
Aerosolized drugs can be effectively delivered by MDI plus
valved holding chamber spacer devices with a mask in or nebuliser
and mask in young infants.
[Back to top]
Childhood Systemic Lupus Erythematosus: New and Old
Treatments
Brigitte Bader-Meunier and Elie Haddad
Systemic lupus erythematosus (SLE) is more severe in children
than in adults, and often needs aggressive treatment. Classical
(old ) therapies include mostly non-steroid anti-inflammatory
drugs, hydroxychloroquine, corticosterois and cyclophosphamide.
However, these two latter drugs can induce severe side effects.
Recently, new drugs, especially Mycophenolate Mofetyl and
anti-CD20 antibody, have been proved to be efficient and well
tolerated in adult-onset SLE and may become mainstay therapeutic
option for severe chidhood-onset lupus manifestations. Management
of complications such as growth failure, osteoporosis, premature
atherosclerosis and anti-phospholipid syndrome has also progressed
within the last years. Beside these promising perspectives
of treatment, psychological support, long-term follow-up and
program of transition from the pediatric to the adult care
unit remain essential.
[Back to top]
Primary Vesicoureteric Reflux and Reflux Nephropathy
– New Insights
Laura Santoro, Alessandra Ferrarini, Laura Crosazzo
and Mario G. Bianchetti
Primary vesicoureteric reflux was felt to result from a
congenitally short mucosal tunnel length with oblique intravescical
insertion but more recent data suggest that primary vesicoureteric
reflux is often associated with functional urodynamic abnormalities.
There is now sound evidence that primary vesicoureteric reflux
has a familial basis; most findings suggest a dominant inheritance
with incomplete penetrance. Primary vesicoureteric reflux
is often associated with kidney damage, mostly referred to
as reflux nephropathy. It has been traditionally assumed that
in primary vesicoureteric reflux kidney damage results from
reflux of infected urine into the renal tissue but recent
observations indicate that kidney damage sometimes occurs
prenatally. In the overwhelming majority of patients with
prenatally acquired renal damage the damage is linked with
high-grade vesicoureteric reflux. In conclusion there are
two categories of reflux disorder: a reflux associated with
an acquired renal scarring secondary to infections and a prenatal
high-grade vesicoureteric reflux linked with a congenital
nephropathy.
[Back to top]
Recent Advances in Minimal Access Surgery for Infants
and Children
Ken K.Y. Wong and Paul K.H. Tam
Paediatric surgeons were among the pioneers of laparoscopic
surgery in the early 1970s. However, the vast potential of
minimal access surgery in infants and children has only been
more fully realised in the past decade. This has been brought
about by increasing experience in paediatric laparoscopic
procedures, and advances in video technology and miniaturised
instruments. Very few complex paediatric surgical conditions
are now beyond the scope of minimal access surgery. Benefits
of minimal access surgery may include reduced postoperative
pain, shortened length of stay, and faster resumption of normal
activities. The question now is no longer whether laparoscopic
surgery should be done in children, but what conditions should
be treated laparoscopically. In this review, we will discuss
the scope of laparoscopic surgery in children and focus on
recent advance and controversial issues.
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