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An internal pilot study for a randomized trial aimed at evaluating the effectiveness of iron interventions in children with non-anemic iron deficiency: the OptEC trial.

ABSTRACT: The OptEC trial aims to evaluate the effectiveness of oral iron in young children with non-anemic iron deficiency (NAID). The initial sample size calculated for the OptEC trial ranged from 112-198 subjects. Given the uncertainty regarding the parameters used to calculate the sample, an internal pilot study was conducted. The objectives of this internal pilot study were to obtain reliable estimate of parameters (standard deviation and design factor) to recalculate the sample size and to assess the adherence rate and reasons for non-adherence in children enrolled in the pilot study.The first 30 subjects enrolled into the OptEC trial constituted the internal pilot study. The primary outcome of the OptEC trial is the Early Learning Composite (ELC). For estimation of the SD of the ELC, descriptive statistics of the 4 month follow-up ELC scores were assessed within each intervention group. The observed SD within each group was then pooled to obtain an estimated SD (S2) of the ELC. Correlation (?) between the ELC measured at baseline and follow-up was assessed. Recalculation of the sample size was performed using analysis of covariance (ANCOVA) method which uses the design factor (1- ?(2)). Adherence rate was calculated using a parent reported rate of missed doses of the study intervention.The new estimate of the SD of the ELC was found to be 17.40 (S2). The design factor was (1- ?2)?= 0.21. Using a significance level of 5%, power of 80%, S2 = 17.40 and effect estimate (?) ranging from 6-8 points, the new sample size based on ANCOVA method ranged from 32-56 subjects (16-28 per group). Adherence ranged between 14% and 100% with 44% of the children having an adherence rate ? 86%. Information generated from our internal pilot study was used to update the design of the full and definitive trial, including recalculation of sample size, determination of the adequacy of adherence, and application of strategies to improve adherence.ClinicalTrials.gov Identifier: NCT01481766 (date of registration: November 22, 2011).

SUBMITTER: Abdullah K

PROVIDER: S-EPMC4501099 | biostudies-literature | 2015 Jul

REPOSITORIES: biostudies-literature

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An internal pilot study for a randomized trial aimed at evaluating the effectiveness of iron interventions in children with non-anemic iron deficiency: the OptEC trial.

Abdullah Kawsari K Thorpe Kevin E KE Mamak Eva E Maguire Jonathon L JL Birken Catherine S CS Fehlings Darcy D Hanley Anthony J AJ Macarthur Colin C Zlotkin Stanley H SH Parkin Patricia C PC

Trials 20150714

<h4>Background</h4>The OptEC trial aims to evaluate the effectiveness of oral iron in young children with non-anemic iron deficiency (NAID). The initial sample size calculated for the OptEC trial ranged from 112-198 subjects. Given the uncertainty regarding the parameters used to calculate the sample, an internal pilot study was conducted. The objectives of this internal pilot study were to obtain reliable estimate of parameters (standard deviation and design factor) to recalculate the sample si ...[more]

PMID: 26170014

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Project description:PurposeErythropoiesis-stimulating agents (ESAs) are often used in treatment of patients with chemotherapy-induced anemia. Many studies have demonstrated an improved hemoglobin (Hb) response when ESA is combined with intravenous iron supplementation and a higher effectiveness of intravenous iron over traditional oral iron formulations. A new formulation of oral sucrosomial iron featuring an increased bioavailability compared to traditional oral formulations has recently become available and could provide a valid alternative to those by intravenous (IV) route. Our study evaluated the performance of sucrosomial iron versus intravenous iron in increasing hemoglobin in anemic cancer patients receiving chemotherapy and darbepoetin alfa, as well as safety, need of transfusion, and quality of life (QoL).Materials and methodsThe present study considered a cohort of 64 patients with chemotherapy-related anemia (Hb >8 g/dL <10 g/dL) and no absolute or functional iron deficiency, scheduled to receive chemotherapy and darbepoetin. All patients received darbepoetin alfa 500 mcg once every 3 weeks and were randomly assigned to receive 8 weeks of IV ferric gluconate 125 mg weekly or oral sucrosomial iron 30 mg daily. The primary endpoint was to demonstrate the performance of oral sucrosomial iron in improving Hb response, compared to intravenous iron. The Hb response was defined as the Hb increase ?2 g/dL from baseline or the attainment Hb ? 12 g/dL.ResultsThere was no difference in the Hb response rate between the two treatment arms. Seventy one percent of patients treated with IV iron achieved an erythropoietic response, compared to 70% of patients treated with oral iron. By conventional criteria, this difference is considered to be not statistically significant. There were also no differences in the proportion of patients requiring red blood cell transfusions and changes in QoL. Sucrosomial oral iron was better tolerated.ConclusionIn cancer patients with chemotherapy-related anemia receiving darbepoetin alfa, sucrosomial oral iron provides similar increase in Hb levels and Hb response, with higher tolerability without the risks or side effects of IV iron.

Project description:Early iron deficiency is associated with impaired (cognitive) development, the severity of which depends on the timing and duration of the under-supply of iron. To design effective treatment and prevention strategies for iron deficiency in humans, suited animal models are needed. In an earlier study (Antonides et al., 2015b) we separated 10 pairs of piglets from their mothers within a few days after birth and reared one sibling with artificial iron-deficient (ID) and the other with balanced control milk until weaning. ID piglets grew slower and showed poorer reference memory (RM) performance than their controls in a spatial holeboard task, even weeks after iron repletion. One putative intervening factor in that study was pre-weaning maternal deprivation. In an attempt to refine the piglet iron-deficiency model, we assessed whether piglets reared by sows, but withheld iron supplementation, can serve as animal model of iron deficiency. As sow milk is inherently ID, piglets normally receive a prophylactic iron injection. Ten pairs of piglets were housed with foster sows until weaning (4 weeks). One sibling per pair was randomly assigned to the control group (receiving iron dextran injections: 40 mg iron per kilogram body mass on days 3 and 10), the other to the ID group. From weaning, all pigs were fed a balanced commercial diet. Blood samples were taken in week 1, 3.5, 6, and 12. Pre-weaning blood iron values of ID piglets were lower than those of controls, but recovered to normal values after weaning. Hemoglobin of ID piglets did not reach anemic values. Hematocrit and hemoglobin of ID animals did not decrease, and serum iron even increased pre-weaning, suggesting that the piglets had access to an external source of iron, e.g., spilled feed or feces of the foster sows. Growth, and spatial memory assessed in the holeboard from 10 to 16 weeks of age, was unaffected in ID pigs. We conclude that sow-raised piglets are not a suitable model for iron-deficiency induced cognitive deficits in humans. Based on our previous and the present study, we conclude that growth and memory are only impaired in piglets that suffered from pre-weaning anemia.

Project description:Implementation research is concerned with bridging the gap between evidence and practice through the study of methods to promote the uptake of research into routine practice. Good quality evidence has been summarised into guideline recommendations to show that peri-operative fasting times could be considerably shorter than patients currently experience. The objective of this trial was to evaluate the effectiveness of three strategies for the implementation of recommendations about peri-operative fasting.A pragmatic cluster randomised trial underpinned by the PARIHS framework was conducted during 2006 to 2009 with a national sample of UK hospitals using time series with mixed methods process evaluation and cost analysis. Hospitals were randomised to one of three interventions: standard dissemination (SD) of a guideline package, SD plus a web-based resource championed by an opinion leader, and SD plus plan-do-study-act (PDSA). The primary outcome was duration of fluid fast prior to induction of anaesthesia. Secondary outcomes included duration of food fast, patients' experiences, and stakeholders' experiences of implementation, including influences. ANOVA was used to test differences over time and interventions.Nineteen acute NHS hospitals participated. Across timepoints, 3,505 duration of fasting observations were recorded. No significant effect of the interventions was observed for either fluid or food fasting times. The effect size was 0.33 for the web-based intervention compared to SD alone for the change in fluid fasting and was 0.12 for PDSA compared to SD alone. The process evaluation showed different types of impact, including changes to practices, policies, and attitudes. A rich picture of the implementation challenges emerged, including inter-professional tensions and a lack of clarity for decision-making authority and responsibility.This was a large, complex study and one of the first national randomised controlled trials conducted within acute care in implementation research. The evidence base for fasting practice was accepted by those participating in this study and the messages from it simple; however, implementation and practical challenges influenced the interventions' impact. A set of conditions for implementation emerges from the findings of this study, which are presented as theoretically transferable propositions that have international relevance.ISRCTN18046709--Peri-operative Implementation Study Evaluation (POISE).

Project description:Iron-deficiency anemia is the most frequent nutritional deficiency, with women of reproductive age being particularly at risk of its development. The aim of the systematic review was to assess the effectiveness of dietary interventions to treat iron-deficiency anemia in women based on the randomized controlled trials. The systematic review was conducted according to the PRISMA guidelines and registered in the PROSPERO database (CRD42021261235). The searching procedure was based on PubMed and Web of Science databases, while it covered records published until June 2021. It included all randomized controlled trials assessing effectiveness of various dietary interventions on treatment of iron-deficiency anemia in women of childbearing age. The total number of 7825 records were screened, while 14 of them were finally included in the systematic review. The studies were screened, included, and reported, and the risk of bias was assessed using the revised Cochrane risk-of-bias tool for randomized trials by two independent researchers. The included studies compared the effectiveness of various dietary interventions with supplementation, placebo, control, or any other dietary intervention, while the assessed dietary interventions were based either on increasing iron supply and/or on increasing its absorption (by increasing vitamin C or vitamin D or decreasing phytate intake). The duration of applied intervention was diversified from 3 months or less, through 4 or 5 months, to half of a year or more. Among the assessed biochemical measures, the following were analyzed in majority of studies: hemoglobin, ferritin, transferrin receptor, hematocrit, and transferrin. The majority of included studies supported the influence of dietary interventions on the treatment of iron-deficiency anemia, as the applied dietary intervention was not effective in only three studies. The majority of included studies were assessed as characterized by medium risk of bias, while the overall risk was high for only four studies, which resulted from the randomization process, deviations from the intended interventions, and selection of the reported result. The majority of included studies were conducted for increasing iron supply and/or increasing vitamin C supply; however, only for the interventions including increasing iron supply and simultaneously increasing its absorption by vitamin C supply were all results confirmed effective. Vitamin D also seems to be an effective dietary treatment, but further studies are necessary to confirm the observations. Considering this fact, dietary interventions recommended for anemic female patients should include increased intake of iron and vitamin C.

Project description:BackgroundThree decades of research suggests that prevention of iron deficiency anemia (IDA) in the primary care setting may be an unrealized and unique opportunity to prevent poor developmental outcomes in children. A longitudinal study of infants with IDA showed that the developmental disadvantage persists long term despite iron therapy. Early stages of iron deficiency, termed non-anemic iron deficiency (NAID), provide an opportunity for early detection and treatment before progression to IDA. There is little research regarding NAID, which may be associated with delayed development in young children. The aim of this study is to compare the effectiveness of four months of oral iron treatment plus dietary advice, with placebo plus dietary advice, in improving developmental outcomes in children with NAID and to conduct an internal pilot study.Methods/designFrom a screening cohort, those identified with NAID (hemoglobin ≥110 g/L and serum ferritin <14 μg/L) are invited to participate in a pragmatic, multi-site, placebo controlled, blinded, parallel group, superiority randomized trial. Participating physicians are part of a primary healthcare research network called TARGet Kids! Children between 12 and 40 months of age and identified with NAID are randomized to receive four months of oral iron treatment at 6 mg/kg/day plus dietary advice, or placebo plus dietary advice (75 per group). The primary outcome, child developmental score, is assessed using the Mullen Scales of Early Learning at baseline and at four months after randomization. Secondary outcomes include an age appropriate behavior measure (Children's Behavior Questionnaire) and two laboratory measures (hemoglobin and serum ferritin levels). Change in developmental and laboratory measures from baseline to the end of the four-month follow-up period will be analyzed using linear regression (analysis of covariance method).DiscussionThis trial will provide evidence regarding the association between child development and NAID, and the effectiveness of oral iron to improve developmental outcomes in children with NAID. The sample size of the trial will be recalculated using estimates taken from an internal pilot study.Trial registrationThis trial was registered with Clinicaltrials.gov (identifier: NCT01481766 ) on 22 November 2011.

Dataset Information

An internal pilot study for a randomized trial aimed at evaluating the effectiveness of iron interventions in children with non-anemic iron deficiency: the OptEC trial.

Publications

An internal pilot study for a randomized trial aimed at evaluating the effectiveness of iron interventions in children with non-anemic iron deficiency: the OptEC trial.

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