Further Development and Assessment of Tools to Measure Risk Factors for and Treatment of Thiamine Deficiency Disorders

NCT05390086 | Completed

• 1 other identifier: 1867468
• Study Type: observational
• Target: 42
• Locations: 1 country
• Sites: 2

Brief Summary

Thiamine deficiency, including the most severe form infantile beriberi, is a public health concern across South and Southeast Asia, where monotonous diets rely on thiamine-poor white rice. Food insecurity, food preparation and cooking practices, anti-thiamine compound consumption and culturally determined postpartum food restrictions precipitate thiamine deficiency in these high-risk regions. The risk of thiamine deficiency is highest in the first year of life, especially among exclusively or predominantly breastfed infants of mothers who are thiamine deficient themselves, as thiamine content of breastmilk is related to maternal thiamine status. However, diagnosis of infantile thiamine deficiency is challenging due to the highly variable, non-specific clinical manifestations, referred to as thiamine deficiency disorders (TDD), that often overlap with other conditions, resulting in misdiagnosis and missed treatment opportunities, which can be fatal or have irreversible consequences. Considering that breastfed infants are at highest risk, a large proportion of infant deaths could be avoided if: 1) infants with TDD were immediately treated with thiamine when medically indicated and, importantly; 2) thiamine deficiency was prevented by improving thiamine status among breastfeeding women. The latter is important given emerging evidence of long-term neurocognitive deficits of severe and even subclinical thiamine deficiency. In light of these diagnostic uncertainties, it was recognised that a case definition for thiamine responsive disorders (TRD) would help to better identify infants with TDD who would benefit from timely thiamine treatment. This study will test the usefulness of a recently developed case definition for TRD and practical tool in different contexts in Lao PDR where TDD have been reported. Secondly, data on diet, maternal and household risk factors for TDD in different contexts will be used to propose a community risk factor screening tool to better identify populations at risk of thiamine deficiency and help advocate for and guide planning of preventive programmes.

Conditions

Thiamine Deficiency

Outcome Measures

Primary Outcomes (1)

• Thiamine responsive disorder (TRD)

  Presenting signs and symptoms compatible with the thiamine responsive disorders (TRD) case definition and physician diagnosis of beriberi

  From hospital admission to hospital discharge

Secondary Outcomes (1)

• Risk factors for TDD/TRD

  Baseline

Interventions

Standard of care OTHER

The study is being implemented among children who are seeking care at collaborating hospitals for signs and symptoms suggestive of TDD. All children will be treated by the hospital physicians following the usual hospital procedures. The study will not interfere in any with the administration of required treatments and interventions.

Eligibility Criteria

• Age: 21 Days - 18 Months
• Sex: all
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Infants and young children in the target age range (21 days - \<18 months) who are admitted to the collaborating hospitals with at least one of the signs/symptoms suggestive of TDD will be eligible for participation. Mothers of all participating children will be invited to participate in the study.

You may qualify if:

• Infants and young children aged 21 days - \<18 months presenting to the collaborating hospitals and presenting with at least one of the following signs and symptoms consistent with TDD:
• Enlarged liver (liver edge \>2 cm below the right costal margin)
• Edema
• Tachypnea (respiratory rate \>60/min for 3-8 weeks; \>50/min for 2-11 months; \>40/min for 12-18 months)
• Tachycardia (heart rate \>160/min for \<12 months; \>120/min for 12-18 months)
• Oxygen saturation \<92%
• Difficulty breathing (chest indrawing or nasal flaring)
• Refusal to breastfeed or of infant formula or food for \>24 hours
• Repetitive or recurrent vomiting with no obvious other cause (\>3 times in past 24 hours)
• Persistent crying not relieved by soothing or feeding with no obvious other cause
• Hoarse voice/cry or loss of voice
• Nystagmus or other unusual eye movements
• Muscle twitching
• Loss of consciousness
• Convulsion

You may not qualify if:

• Infants and young children who do not meet any of the TDD-like signs and symptoms will not be eligible for study participation.

Sponsors & Collaborators

• University of California, Davis: lead
• Lao Tropical and Public Health Institute: collaborator

Study Sites (2)

Lao Friends Hospital for Children

Luang Prabang, Laos

Location

Children's Hospital

Vientiane, Laos

Location

MeSH Terms

Conditions

Thiamine Deficiency

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

Vitamin B Deficiency; Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 16, 2022
• First Posted: May 25, 2022
• Study Start: June 15, 2022
• Primary Completion: August 26, 2022
• Study Completion: August 26, 2022
• Last Updated: April 23, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Within 2 years of completion of the data collection

Locations

LA (2)