Beta Thalassemia MajorNCT03947632
Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia
An observational study of Beta Thalassemia Major, run by Aga Khan University.
Trial summary
The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Beta Thalassemia Major
- What is being tested?
- serum ferritin and hemoglobin test
- How many people
- 367 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2013to 31 Dec 2014
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 17 years
- All sexes
- Child
Study population: 367 pediatric patients (age 5-17 year) with transfusion dependent β-thalassemia were recruited. All patients were diagnosed based on hemoglobin electrophoresis or high-performance liquid chromatography. Patient recruitment was conducted at a non- profit organization named Fatimid Foundation Karachi (FFK), which provides free of cost blood components to the patients with various blood disorders.
Who may be able to join
- Children with transfusion dependent beta thalassemia major
- Age 5 to 17 years
- Subjects with informed consent
Who cannot join
- Age more than 17 years
- Subjects who refused informed consent
About this study
Official title: Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia.
The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth
Read the detailed description
Material and methods
Setting The study was conducted at the Sections of Hematology and Clinical Chemistry in the Department of Pathology and Laboratory Medicine, Aga Khan University, Karachi, Pakistan during January 2013 to December 2014. Patient recruitment was conducted at a non-profit organization named Fatimid Foundation Karachi (FFK), which provides free of cost blood components to the patients with various blood disorders. There are more than 4000 registered patients of thalassemia and hemophilia at FFK.
Patient selection After informed consent from their parents, 367 pediatric patients (age 5-17 year) with transfusion-dependent β-thalassemia were recruited. All patients were diagnosed based on hemoglobin electrophoresis or high-performance liquid chromatography.
Data collection Detail history was collected by a trained research officer on a predetermined questionnaire. The following information was collected from medical records of the patients: demographics (age, gender, age at the time of diagnosis and at first transfusion), anthropometrics (height and weight), and clinical details (blood transfusion history, iron chelation, last pre-transfusion hemoglobin, and last serum ferritin levels).
Sample collection Five milliliter of blood samples were collected in two tubes (ethylenediaminetetraacetic acid and gel) at Fatimid foundation by a trained phlebotomist prior to blood transfusion to determine hemoglobin and serum ferritin.
Laboratory methodology Serum ferritin was measured by chemi-luminescent micro-particle immunoassay on Cobos® E-601 and hemoglobin was measured using Sysmex® XP-100.
Anthropometry World health organization (WHO) 2007 growth charts were used for boys and girls to assess their physical growth. Body mass index (BMI) was computed as weight in kg/height in square meters. z-Score of height (h-SDS), weight (w-SDS) and BMI. According to WHO reference 2007, a z-score of <-2 for height was considered as stunted growth and BMI of <-2 was considered as underweight or thin for age.
Statistical analysis Data were collected, managed, edited, entered, and analyzed by statistical package of social sciences (SPSS) version 22 and STATA 13 (Stata Corp, College Station, TX, U.S.A.). A descriptive analysis of all patients in the study was performed. In order to analyze the profile of the sample according to the variables studied, frequency tables for categorical variables like (gender, age group) were created. Male: female ratio was also computed. The mean ± standard deviation, minimum and maximum values were calculated for parametric and median and interquartile range (IQR; Q3-Q1) for non-parametric variables. Spearman rho correlation was used to test relationships between the non-parametric variables. The level of significance taken for all the statistical tests was a p-value of <0.05. The association of h-SDS with median serum ferritin levels was further confirmed by comparing these laboratory parameters in children with height z-scores <2 with those with the z-score ≥2 using the Mann-Whitney test.
- Conditions
- Beta Thalassemia Major
- Being tested
- serum ferritin and hemoglobin test
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
serum ferritin and hemoglobin testDiagnostic test
Five milliliter of blood samples were collected in two tubes (ethylenediaminetetraacetic acid and gel) at Fatimid foundation by a trained phlebotomist prior to blood transfusion to determine hemoglobin and serum ferritin.
What is being measured
The primary outcome is the main question the study is designed to answer.
Height in transfusion-dependent β-thalassemia pediatric patients in Pakistan were recorded to assess growth rate
Height in meters will be recorded from medical records of the patients
Measured: Jan 2013 to Dec 2014
Weight in transfusion-dependent β-thalassemia pediatric patients in Pakistan were recorded to assess growth rate
Wight in kilograms will be recorded from medical records of the patients
Measured: Jan 2013 to Dec 2014
Assessment of physical growth in transfusion-dependent β-thalassemia pediatric patients in Pakistan were recorded to assess growth rate
World health organization (WHO) 2007 growth charts were used for boys and girls to assess their physical growth. Body mass index (BMI) was computed as weight in kg/height in square meters (kg/m^2). z-Score of height (h-SDS), weight (w-SDS), and BMI (BMI-SDS) were also calculated.
Measured: Jan 2013 to Dec 2014
Serum ferritin levels and their association with z-Score of height
Correlation was assessed between height for age z-score and serum ferritin levels. Serum ferritin was measured by chemi-luminescent micro-particle immunoassay on Cobos® E-601. The association of z-Score of height with median serum ferritin levels was further confirmed by comparing these laboratory parameters in children with height z-scores <2 with those with the z-score ≥2 using the Mann-Whitney test.
Measured: Jan 2013 to Dec 2014
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2019
- Study start1 Jan 2013
- Primary completion31 Dec 2014
- Study completion31 Dec 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta Thalassemia MajorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 367 participants (actual)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Aga Khan University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Bushra Moiz, Aga Khan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03947632
- Sponsor's protocol ID
- 2305-Pat-ERC-12
- Status last verified
- May 2019
- First submitted
- 7 May 2019
- Last updated
- 13 May 2019
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia.
- ClinicalTrials.gov ID
- NCT03947632
- Acronym / sponsor ID
- 2305-Pat-ERC-12
- Conditions
- Beta Thalassemia Major
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 367 participants (actual)
- Lead sponsor
- Aga Khan UniversityUniversity, hospital or other
- Study start
- 1 Jan 2013 (actual)
- Primary completion
- 31 Dec 2014 (actual)
- Study completion
- 31 Dec 2014 (actual)
- First posted
- 13 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth
Detailed description
Material and methods
Setting The study was conducted at the Sections of Hematology and Clinical Chemistry in the Department of Pathology and Laboratory Medicine, Aga Khan University, Karachi, Pakistan during January 2013 to December 2014. Patient recruitment was conducted at a non-profit organization named Fatimid Foundation Karachi (FFK), which provides free of cost blood components to the patients with various blood disorders. There are more than 4000 registered patients of thalassemia and hemophilia at FFK.
Patient selection After informed consent from their parents, 367 pediatric patients (age 5-17 year) with transfusion-dependent β-thalassemia were recruited. All patients were diagnosed based on hemoglobin electrophoresis or high-performance liquid chromatography.
Data collection Detail history was collected by a trained research officer on a predetermined questionnaire. The following information was collected from medical records of the patients: demographics (age, gender, age at the time of diagnosis and at first transfusion), anthropometrics (height and weight), and clinical details (blood transfusion history, iron chelation, last pre-transfusion hemoglobin, and last serum ferritin levels).
Sample collection Five milliliter of blood samples were collected in two tubes (ethylenediaminetetraacetic acid and gel) at Fatimid foundation by a trained phlebotomist prior to blood transfusion to determine hemoglobin and serum ferritin.
Laboratory methodology Serum ferritin was measured by chemi-luminescent micro-particle immunoassay on Cobos® E-601 and hemoglobin was measured using Sysmex® XP-100.
Anthropometry World health organization (WHO) 2007 growth charts were used for boys and girls to assess their physical growth. Body mass index (BMI) was computed as weight in kg/height in square meters. z-Score of height (h-SDS), weight (w-SDS) and BMI. According to WHO reference 2007, a z-score of <-2 for height was considered as stunted growth and BMI of <-2 was considered as underweight or thin for age.
Statistical analysis Data were collected, managed, edited, entered, and analyzed by statistical package of social sciences (SPSS) version 22 and STATA 13 (Stata Corp, College Station, TX, U.S.A.). A descriptive analysis of all patients in the study was performed. In order to analyze the profile of the sample according to the variables studied, frequency tables for categorical variables like (gender, age group) were created. Male: female ratio was also computed. The mean ± standard deviation, minimum and maximum values were calculated for parametric and median and interquartile range (IQR; Q3-Q1) for non-parametric variables. Spearman rho correlation was used to test relationships between the non-parametric variables. The level of significance taken for all the statistical tests was a p-value of <0.05. The association of h-SDS with median serum ferritin levels was further confirmed by comparing these laboratory parameters in children with height z-scores <2 with those with the z-score ≥2 using the Mann-Whitney test.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Target follow-up
- 2 Years
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Age groups
- Child
- Study population
- 367 pediatric patients (age 5-17 year) with transfusion dependent β-thalassemia were recruited. All patients were diagnosed based on hemoglobin electrophoresis or high-performance liquid chromatography. Patient recruitment was conducted at a non- profit organization named Fatimid Foundation Karachi (FFK), which provides free of cost blood components to the patients with various blood disorders.
Key inclusion criteria (3 listed)
- Children with transfusion dependent beta thalassemia major
- Age 5 to 17 years
- Subjects with informed consent
Key exclusion criteria (2 listed)
- Age more than 17 years
- Subjects who refused informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| serum ferritin and hemoglobin testFive milliliter of blood samples were collected in two tubes (ethylenediaminetetraacetic acid and gel) at Fatimid foundation by a trained phlebotomist prior to blood transfusion to determine hemoglobin and serum ferritin. | Diagnostic test | – |
Primary outcome
Height in transfusion-dependent β-thalassemia pediatric patients in Pakistan were recorded to assess growth rate
Time frame: Jan 2013 to Dec 2014
Height in meters will be recorded from medical records of the patients
Weight in transfusion-dependent β-thalassemia pediatric patients in Pakistan were recorded to assess growth rate
Time frame: Jan 2013 to Dec 2014
Wight in kilograms will be recorded from medical records of the patients
Assessment of physical growth in transfusion-dependent β-thalassemia pediatric patients in Pakistan were recorded to assess growth rate
Time frame: Jan 2013 to Dec 2014
World health organization (WHO) 2007 growth charts were used for boys and girls to assess their physical growth. Body mass index (BMI) was computed as weight in kg/height in square meters (kg/m^2). z-Score of height (h-SDS), weight (w-SDS), and BMI (BMI-SDS) were also calculated.
Serum ferritin levels and their association with z-Score of height
Time frame: Jan 2013 to Dec 2014
Correlation was assessed between height for age z-score and serum ferritin levels. Serum ferritin was measured by chemi-luminescent micro-particle immunoassay on Cobos® E-601. The association of z-Score of height with median serum ferritin levels was further confirmed by comparing these laboratory parameters in children with height z-scores <2 with those with the z-score ≥2 using the Mann-Whitney test.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2019.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Beta Thalassemia Major, run by Aga Khan University.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 May 2019. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Height in transfusion-dependent β-thalassemia pediatric patients in Pakistan were recorded to assess growth rate (time frame: Jan 2013 to Dec 2014). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2019.