Sequential design with boundaries approach in pediatric intervention research reduces sample size

Lee, Johanna H. van der; Wesseling, J; Tanck, Michael W.T.; Offringa, Martin

doi:10.1016/j.jclinepi.2009.07.005

Cited by 16 publications

(15 citation statements)

References 49 publications

(83 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Simulations have demonstrated that the expected sample size for sequential trials is smaller than the corresponding fixed sample size [7,44]. Challenges include analyzing multiple treatments, calculating study power [35], averting the risk of type I error [7], timing the first interim analysis, and planning the optimal number of interim analyses.…”

Section: Sequential Designsmentioning

confidence: 99%

A framework for applying unfamiliar trial designs in studies of rare diseases

Gupta

Faughnan

Tomlinson

et al. 2011

Journal of Clinical Epidemiology

View full text Add to dashboard Cite

Section: Sequential Designsmentioning

confidence: 99%

A framework for applying unfamiliar trial designs in studies of rare diseases

Gupta

Faughnan

Tomlinson

et al. 2011

Journal of Clinical Epidemiology

View full text Add to dashboard Cite

“…This design may be suitable in trials where outcome results are available quickly in relation to the patient recruitment rate. Analyses from a systematic review of paediatric sequential trials 176 (24 were published between 1963 and 2005) indicated a median reduction in sample size of 52 subjects (range −22 to 229 subjects) or 35% (range −42% to 90%) for sequential trials when comparing with a classical fixed sample size approach. Only nine trials reported sufficient information about assumptions to allow calculation of a corresponding fixed sample size.…”

Section: Recruitmentmentioning

confidence: 99%

“…The unknown sample size at the start of sequential trials may be problematic for funders, although some trials pre specify a maximum number of participants. 176 Another strategy to address likely sample size problems is responsive-adaptive randomisation: a 'play the winner' approach that maximises allocation to the most effective treatment. Outcomes for previous participants affect subsequent treatment allocation probabilities.…”

Section: Recruitmentmentioning

confidence: 99%

Treatment of extravasation injuries in infants and young children: a scoping review and survey

Corbett

Marshall

Harden

et al. 2018

Health Technol Assess

View full text Add to dashboard Cite

BackgroundExtravasation injuries are caused by unintended leakages of fluids or medicines from intravenous lines, but there is no consensus on the best treatment approaches.ObjectivesTo identify which treatments may be best for treating extravasation injuries in infants and young children.DesignScoping review and survey of practice.PopulationChildren aged < 18 years with extravasation injuries and NHS staff who treat children with extravasation injuries.InterventionsAny treatment for extravasation injury.Main outcome measuresWound healing time, infection, pain, scarring, functional impairment, requirement for surgery.Data sourcesTwelve database searches were carried out in February 2017 without date restrictions, including MEDLINE, CINAHL (Cumulative Index to Nursing and Allied Health Literature) Plus and EMBASE (Excerpta Medica dataBASE).MethodsScoping review – studies were screened in duplicate. Data were extracted by one researcher and checked by another. Studies were grouped by design, and then by intervention, with details summarised narratively and in tables. The survey questionnaire was distributed to NHS staff at neonatal units, paediatric intensive care units and principal oncology/haematology units. Summary results were presented narratively and in tables and figures.ResultsThe evidence identified in the scoping review mostly comprised small, retrospective, uncontrolled group studies or case reports. The studies covered a wide range of interventions including conservative management approaches, saline flush-out techniques (with or without prior hyaluronidase), hyaluronidase (without flush-out), artificial skin treatments, debridement and plastic surgery. Few studies graded injury severity and the results sections and outcomes reported in most studies were limited. There was heterogeneity across study populations in age, types of infusate, injury severity, location of injury and the time gaps between injury identification and subsequent treatment. Some of the better evidence related to studies of flush-out techniques. The NHS survey yielded 63 responses from hospital units across the UK. Results indicated that, although most units had a written protocol or guideline for treating extravasation injuries, only one-third of documents included a staging system for grading injury severity. In neonatal units, parenteral nutrition caused most extravasation injuries. In principal oncology/haematology units, most injuries were due to vesicant chemotherapies. The most frequently used interventions were elevation of the affected area and analgesics. Warm or cold compresses were rarely used. Saline flush-out treatments, either with or without hyaluronidase, were regularly used in about half of all neonatal units. Most responders thought a randomised controlled trial might be a viable future research design, though opinions varied greatly by setting.LimitationsPaucity of good-quality studies.ConclusionsThere is uncertainty about which treatments are most promising, particularly with respect to treating earlier-stage injuries. Saline flush-out techniques and conservative management approaches are commonly used and may be suitable for evaluation in trials.Future workConventional randomised trials may be difficult to perform, although a randomised registry trial may be an appropriate alternative.FundingThe National Institute for Health Research Health Technology Assessment programme.

show abstract

“…In such cases, it is desirable to adjust the sample size according to the effect size for the ongoing trial thus reducing the risk of trial failure. Early stopping is pursued when the efficacy or futility of the experimental drug becomes obvious during the trial [16]. Finally, through responseadaptive randomization, allocation probability may change during the trial based on the responses of the previous patients and more patients are expected to be randomised to the group that is proving superiority.…”

Section: Adaptive Designmentioning

confidence: 99%