Clinical research in newborn infants: difficulties and specificity

Ligi, Isabelle; Boubred, Farid; Grandvuillemin, Isabelle; Simeoni, Umberto

doi:10.1007/s00228-010-0921-3

Cited by 8 publications

(11 citation statements)

References 15 publications

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“…The actual efficacy of interventions in the pediatric population may be impacted by changes in pharmacokinetics and pharmacodynamics due to maturation and development [ 2 , 31 , 32 , 34 , 59 , 61 , 66 , 72 , 78 – 87 ], insufficient data of high-quality and/or low-quantity [ 72 , 73 , 75 , 76 , 79 ], and incorrect predictions through the extrapolation of adult data [ 30 , 32 , 64 , 65 , 88 – 93 ]. Specifically, the reporting of the rationale and evidence for the comparator treatments used in the trial allow the reader to make appropriate scientific interpretations of the results [ 2 , 30 , 86 , 87 , 94 – 101 ]. Finally, our research found abundant support for the reporting of the validity of the outcomes used in every included age group to allow the reader to assess any potential for invalid outcome selection and measurement.…”

Section: Resultsmentioning

confidence: 99%

“…This involves reducing the pain, discomfort, distress, and invasiveness of the procedures used [ 31 , 32 , 72 , 86 , 88 , 132 – 136 ]. Reporting blood-sampling procedures allows the reader to critically appraise whether the volume of blood drawn in pediatric studies was minimized and sampling was infrequent as possible [ 32 , 60 , 78 , 86 , 87 , 133 , 134 , 137 ]. Additionally, inclusion of whether a Data and Safety Monitoring Board was established enables the audience to evaluate the presence of quality and safety oversight before and during the trial [ 22 , 86 , 87 , 118 , 138 – 143 ].…”

Section: Resultsmentioning

confidence: 99%

“…Reporting blood-sampling procedures allows the reader to critically appraise whether the volume of blood drawn in pediatric studies was minimized and sampling was infrequent as possible [ 32 , 60 , 78 , 86 , 87 , 133 , 134 , 137 ]. Additionally, inclusion of whether a Data and Safety Monitoring Board was established enables the audience to evaluate the presence of quality and safety oversight before and during the trial [ 22 , 86 , 87 , 118 , 138 – 143 ]. Further, detailed reporting of the trial’s comparator treatments allows the ethical implications and risks associated with certain comparators to be assessed by the reader, like the risk of exposure to novel or off-label agents [ 134 ], the use of placebo or withholding of some standard care [ 61 , 87 , 101 , 144 , 145 ], and the inability of pediatric subjects to provide informed consent to use of some comparator groups [ 134 , 146 ] make reporting of the rationale and evidence for the comparators used in the trial a necessity.…”

Section: Resultsmentioning

confidence: 99%

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Recommendations and evidence for reporting items in pediatric clinical trial protocols and reports: two systematic reviews

Clyburne-Sherin

Thurairajah

Kapadia

et al. 2015

Trials

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Background: Complete and transparent reporting of clinical trial protocols and reports ensures that these documents are useful to all stakeholders, that bias is minimized, and that the research is not wasted. However, current studies repeatedly conclude that pediatric trial protocols and reports are not appropriately reported. Guidelines like SPIRIT (Standard Protocol Items: Recommendations for Interventional Trials) and CONSORT (Consolidated Standards of Reporting Trials) may improve reporting, but do not offer guidance on issues unique to pediatric trials. This paper reports two systematic reviews conducted to build the evidence base for the development of pediatric reporting guideline extensions: 1) SPIRIT-Children (SPIRIT-C) for pediatric trial protocols, and 2) CONSORT-Children (CONSORT-C) for pediatric trial reports. Method: MEDLINE, the Cochrane Methodology Register, and reference lists of included studies were searched. Publications of any type were eligible if they included explicit recommendations or empirical evidence for the reporting of potential items in a pediatric protocol (SPIRIT-C systematic review) or trial report (CONSORT-C systematic review). Study characteristics, recommendations and evidence for pediatric extension items were extracted. Recurrent themes in the recommendations and evidence were identified and synthesized. All steps were conducted by two reviewers. Results: For the SPIRIT-C and CONSORT-C systematic reviews 366 and 429 publications were included, respectively. Recommendations were identified for 48 of 50 original reporting items and sub-items from SPIRIT, 15 of 20 potential SPIRIT-C reporting items, all 37 original CONSORT items and sub-items, and 16 of 22 potential CONSORT-C reporting items. The following overarching themes of evidence to support or refute the utility of reporting items were identified:

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

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Recommendations and evidence for reporting items in pediatric clinical trial protocols and reports: two systematic reviews

Clyburne-Sherin

Thurairajah

Kapadia

et al. 2015

Trials

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“…Furthermore, some methodological problems and ethical considerations may limit the quality of paediatric clinical research. [188][189][190] The authors of this review hope that this gap between adult and paediatric clinical studies of BNP will be reduced in the near future. Certainly, international guidelines and/or expert consensus opinion on the use of BNP in paediatric patients with CHD are long overdue.…”

Section: Bnp In the Follow-up Of Corrected/palliated Chdmentioning

confidence: 99%

BNP in children with congenital cardiac disease: is there now sufficient evidence for its routine use?

et al. 2015

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Interest in brain natriuretic peptide (BNP) and N-terminal pro-brain natriuretic peptide (NT-proBNP) in the management of children with CHD has increased. There are, however, no current guidelines for their routine use. The aim of this review article is to provide an update on the data regarding the use of BNP/NT-proBNP in the evaluation and surgical treatment of children with CHD. BNP/NT-proBNP levels in children with CHD vary substantially according to age, laboratory assay methods, and the specific haemodynamics associated with the individual congenital heart lesion. The accuracy of BNP/NT-proBNP as supplemental markers in the integrated screening, diagnosis, management, and follow-up of CHD has been established. In particular, the use of BNP/NT-proBNP as a prognostic indicator in paediatric cardiac surgery has been widely demonstrated, as well as its role in the subsequent follow-up of surgical patients. Most of the data, however, are derived from single-centre retrospective studies using multivariable analysis; prospective, randomised clinical trials designed to evaluate the clinical utility and cost-effectiveness of routine BNP/NT-proBNP use in CHD are lacking. The results of well-designed, prospective clinical trials should assist in formulating guidelines and expert consensus recommendations for its use in patients with CHD. Finally, the use of new point-of-care testing methods that use less invasive sampling techniques -capillary blood specimens -may contribute to a more widespread use of the BNP assay, especially in neonates and infants, as well as contribute to the development of screening programmes for CHD using this biomarker.

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“…Because of differences in body composition in different age groups, pharmacokinetic studies have sometimes resulted in incomplete and incorrect conclusions [96]. Trials that include different paediatric ages may need appropriate dose adjustments by weight or body surface area [96,106]. Researchers are increasingly recognizing the importance of qualitative outcome measures that are relevant to the child and family, including the impact of the illness and treatment on the quality of life [16].…”

Section: Outcome Measuresmentioning

confidence: 99%

Clinical trials in children

Joseph

Craig

Caldwell

2015

Brit J Clinical Pharma

307

274

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Safety and efficacy data on many medicines used in children are surprisingly scarce. As a result children are sometimes given ineffective medicines or medicines with unknown harmful side effects. Better and more relevant clinical trials in children are needed to increase our knowledge of the effects of medicines and to prevent the delayed or non-use of beneficial therapies. Clinical trials provide reliable evidence of treatment effects by rigorous controlled testing of interventions on human subjects. Paediatric trials are more challenging to conduct than trials in adults because of the paucity of funding, uniqueness of children and particular ethical concerns. Although current regulations and initiatives are improving the scope, quantity and quality of trials in children, there are still deficiencies that need to be addressed to accelerate radically equitable access to evidence-based therapies in children.

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Clinical research in newborn infants: difficulties and specificity

Cited by 8 publications

References 15 publications

Recommendations and evidence for reporting items in pediatric clinical trial protocols and reports: two systematic reviews

Recommendations and evidence for reporting items in pediatric clinical trial protocols and reports: two systematic reviews

BNP in children with congenital cardiac disease: is there now sufficient evidence for its routine use?

Clinical trials in children

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