Description of the methodology used in an ongoing pediatric care interventional study of children born with cleft lip and palate in South America [NCT00097149]

Abstract

Background: The contribution of birth defects, including cleft lip and palate, to neonatal and infant mortality and morbidity is substantial. As other mortality and morbidity causes including infections, hygiene, prematurity, and nutrition are eradicated in less developed countries, the burden of birth defects will increase proportionally.

Methods/design: We are using cleft lip and palate as a sentinel birth defect to evaluate its burden on neonatal and infant health and to assess the effectiveness of systematic pediatric care during the first month and first two years of life in decreasing this burden. The neonatal intervention, consisting of weekly pediatric evaluation and referral to appropriate care, is delivered to about 696 infants born with cleft lip and/or palate in 47 hospitals in South America. Neonatal mortality in this group will be compared to that in a retrospective control group of about 464 infants born with cleft lip and/or palate in the same hospitals. The subgroup of infants with isolated clefts of both the lip and palate (about 264) is also randomized into two groups, intervened and non-intervened, and further followed up over 2 years. Intervened cases are evaluated by pediatricians every three months and referred for appropriate care. The intervened and non-intervened cases will be compared over study outcomes to evaluate the intervention effectiveness. Non-intervened cases are matched and compared to healthy controls to assess the burden of cleft lip and palate. Outcomes include child's neurological and physical development and family social and economic conditions.

Discussion: Large-scale clinical trials to improve infant health in developing countries are commonly suggested, making it important to share the methods used in ongoing studies with other investigators implementing similar research. We describe here the content of our ongoing pediatric care study in South America. We hope that this may help researchers targeting this area to plan their studies more effectively and encourage the development of similar research efforts to target other birth defects or infant outcomes such as prematurity and low birth weight.

Trial registration: ClinicalTrials.gov NCT00097149.

Figures

Figure 1

Study Design. Figure 1 presents a flowchart of the study design of the neonatal and the two-year subprojects. All infants born with a typical form of cleft lip and palate in the study hospitals are enrolled in the neonatal subproject. Only isolated cases with both cleft of lip and cleft palate who are singleton births, normal birth weight, and without complications other than cleft lip and palate that require systematic care are enrolled at birth in the two-year subproject and randomized into intervened and non-intervened groups. A group of healthy controls is matched to the non-intervened group by gender, date and hospital of birth at a ratio of 2 to 1. A = Evaluating the impact of the intervention on neonatal mortality. B1 = Evaluating the impacts of cleft lip and palate on child's development and family socioeconomic outcomes (Two-year subproject). B2 = Evaluating the impact of the two-year intervention on child development and family socioeconomic outcomes.