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Journal of Pediatric Endocrinology &... Jun 2010Genotropin (somatropin) is licensed for the treatment of children with growth hormone deficiency, Prader-Willi syndrome, Turner syndrome, chronic renal insufficiency and... (Review)
Review
Genotropin (somatropin) is licensed for the treatment of children with growth hormone deficiency, Prader-Willi syndrome, Turner syndrome, chronic renal insufficiency and in children born small for gestational age. This systematic review (SR) evaluated the clinical efficacy and effectiveness of Genotropin in these conditions to inform a NICE Technology Appraisal of growth hormone for the treatment of growth failure in children. Search terms were used to search seven databases, including Medline and Embase, for English language studies. Randomised controlled trials (RCTs) or observational studies investigating Genotropin in children were included. Out of 30 RCTs identified, one reported final height data. Eleven observational studies reported final height and seven were based on the Pfizer International Growth Survey (KIGS). This SR highlights the lack of long-term RCTs reporting final height data and other important qualitative outcomes, such as quality of life. Observational data, such as those from KIGS, remain vital for informing therapy.
Topics: Adolescent; Body Height; Child; Clinical Trials, Phase III as Topic; Databases, Bibliographic; Female; Growth Disorders; Human Growth Hormone; Humans; Male; Randomized Controlled Trials as Topic; Recombinant Proteins
PubMed: 20662327
DOI: 10.1515/jpem.2010.092 -
Research in Developmental Disabilities 2010Skin-picking is a type of self-injurious behavior involving the pulling, scratching, lancing, digging, or gouging of one's own body. It is associated with social... (Review)
Review
Skin-picking is a type of self-injurious behavior involving the pulling, scratching, lancing, digging, or gouging of one's own body. It is associated with social impairment, and increased medical and mental health concerns. While there are several reports showing that skin-picking is common in individuals with developmental disabilities, knowledge about effective treatment approaches is sparse. We therefore reviewed studies involving the treatment of chronic skin-picking in individuals with developmental disabilities. Systematic searches of electronic databases, journals, and reference lists identified 16 studies meeting the inclusion criteria. These studies were evaluated in terms of: (a) participants, (b) functional assessment procedures and results, (c) intervention procedures, (d) results of the intervention, and (e) certainty of evidence. Across the 16 studies, intervention was provided to a total of 19 participants aged 6-42 years. Functional assessment procedures included direct observations, analog functional analyses, and functional assessment interviews. The most commonly identified function was automatic reinforcement. Treatment approaches included combinations of differential reinforcement, providing preferred items and activities stimuli (e.g., toys), wearing protective clothing (e.g., helmets or gloves), response interruption and redirection, punishment, and extinction. Improvements in behavior were reported in all of the reviewed studies. Suggestions for future intervention research are offered.
Topics: Autistic Disorder; Behavior Therapy; Child; Chronic Disease; Developmental Disabilities; Humans; Prader-Willi Syndrome; Self Mutilation; Skin
PubMed: 19963341
DOI: 10.1016/j.ridd.2009.10.017 -
Psychiatric Genetics Dec 2005Autism spectrum disorders (ASDs) have been linked with maternally derived duplications/triplications of chromosome 15q11-13 and therefore might occur more frequently in... (Review)
Review
Autism spectrum disorders (ASDs) have been linked with maternally derived duplications/triplications of chromosome 15q11-13 and therefore might occur more frequently in people with Prader-Willi syndrome (PWS) when due to uniparental disomy (UPD), than in other forms of chromosomal abnormality involving this region [i.e. deletion (DEL) forms of PWS and DEL+UPD forms of Angelman's syndrome -(AS)]. Twelve studies regarding ASD in PWS and AS were reviewed. It was noteworthy that among the genetically confirmed UPD and DEL cases of PWS and AS, the rate of ASD was 25.3% (38/150; range 0-36.5%) in PWS and 1.9% in AS (2/104; range 0-100%) (Fisher's exact P<0.0001). Among the subset of cases with confirmed UPD or DEL, the rate of ASD in the UPD cases of PWS was significantly higher (20/53) than in the remaining combined samples (i.e. DEL PWS+UPD AS+DEL AS cases; 20/201) (Fisher's exact P<0.0001). ASD in UPD PWS cases (20/53) compared with DEL PWS cases (18/97) was also statistically significant (Fisher's exact P=0.0176). Thus, the limited available evidence supported the prediction that overexpression of maternally imprinted genes in 15q11-13 confers a risk for ASD. Further research will be required to confirm these findings.
Topics: Angelman Syndrome; Autistic Disorder; Chromosome Aberrations; Databases, Factual; Humans; Prader-Willi Syndrome
PubMed: 16314754
DOI: 10.1097/00041444-200512000-00006 -
Health Technology Assessment... 2002
Review
Topics: Child; Cost-Benefit Analysis; Human Growth Hormone; Humans; Kidney Failure, Chronic; Practice Guidelines as Topic; Prader-Willi Syndrome; Quality of Life; Randomized Controlled Trials as Topic; State Medicine; Treatment Outcome; Turner Syndrome; United Kingdom
PubMed: 12433316
DOI: 10.3310/hta6180