Beta-ThalassemiaNCT04947540
Iron Status in BTM With Blood Transfusion
An observational study of Beta-Thalassemia, run by Assiut University.
Trial summary
To assess the possible role of iron overload as a cause of liver dysfunction in thalassemic childrens receiving multiple blood transfusion and its correlation with serum aminotransferases. Read more about this study
- Who is it for?
- Ages 5 to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Beta-Thalassemia
- How many people
- 100 participants
- Where
- No sites listed yet
- Study dates
- Jul 2021to Mar 2023
- 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 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: all cases diagnosed as thalassemia major attending to thalassemic center at hematological department at Assuit university childern hospital.
Who may be able to join
- Thalassemic patients of both sex.
- Beta-thalassemia major patients diagnosed Clinical and laboratory .
- Age 5 : 18 years.
- Undergoing multiple blood transfusion.
Who cannot join
- -Age less than 5 years.
- Acute illness as fever and infections.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Hematological and Biochemical Markers of Iron Status in Thalassemic Children Receiving Multiple Blood Transfusion
To assess the possible role of iron overload as a cause of liver dysfunction in thalassemic childrens receiving multiple blood transfusion and its correlation with serum aminotransferases.
Read the detailed description
Thalassemia is derived from the Greek words, thals, which means sea, and emia, which means blood, signifying that it is more common in the Mediterranean region . Globally, among humans, thalassemia is the commonest single-gene disorder. It is defined as a group of inherited disorders characterized by decreased or absent beta globin chain synthesis, leading to a reduced level of hemoglobulin (Hb) in the red blood cells . Specifically in developing countries, thalassemia is a huge health dilemma.
-Beta Thalassemia is the most common chronic hemolytic anemia in Egypt (85.1%) with an estimated carrier rate of 9-10.2%.
Blood transfusion is the primary way of treating thalassemia; it allows the normal growth of the child as well as restrains abnormal erythropoiesis . Iron-chelating agents should be used properly;otherwise, multiple blood transfusions can lead to iron overload. Yet, with no blood transfusion, the increase rate of erythropoiesis intensifies dietary iron absorption from the gut, leading to a severe form of iron overload .
iron overload can result in serious damage to various organs, for example, by depositing in the liver, heart, and various other endocrine glands along with endocrine organ failure. .
During the last years, liver disease has emerged as a major cause of mortality in patients with B- thalassemia major (TM).
The liver is the only site for ferritin and transferrin synthesis, as well as the primary organ for iron storage.
The liver has the maximum capacity to store excess iron in the body, and various other organs, as well as the liver, are very susceptible to damage as a result of iron toxicity.
The correlation between serum ferritin and hepatic iron concentration has been reported in multiple blood-transfused thalassemia patients .
Thalassemia major patients who undergo routine transfusion have an increased risk of acquiring transfusion-transmitted infections (TTI), including hepatitis B and C. These diseases have serious implications and may affect the serum ferritin and aminotransferase levels of thalassemia major patients.
Although the risk of post-transfusion hepatitis C virus (HCV) infection dropped significantly after the national screening of blood in 1993, more than 20% of children who were multitransfused after that date were HCV-RNA positive .
In Egypt, prevalence rate of HCV antibodies seropositivity in thalassemic children at 2011was 51.7% while in the study of El-Faramawy (2012) , a prevalence of 48 % was reported.
Iron overload and hepatitis-C virus (HCV) infection, have been implicated in the evolution of liver disease, in patients with transfusion-dependent beta-thalassaemia major (BTM). The impact of these factors and liver with BTM, has not been extensively studied yet.
Hepatitis virus C infection is the main risk factor for liver injury in transfusion-dependent thalassemics . Dimitrios (2013) on the other hand suggested that in the late stages of liver disease in BTM patients, iron overload may be the critical determinant, since fibrosis is related to the minimal haemosiderosis, independently of HCV history. Injury to the liver, whether acute or chronic, eventually results in an increase in serum concentrations of Alanine transaminase (ALT) and Aspartate transaminase (AST)
- Conditions
- Beta-Thalassemia
- Medical subject headings
- beta-Thalassemia
What is being measured
The primary outcome is the main question the study is designed to answer.
Hematological and biochemical markers of Iron status in thalassemic children receiving multiple blood transfusion
Assessment the possible role of iron overload as a cause of liver dysfunction in thalassemic childrens receiving multiple blood transfusion and its correlation with serum aminotransferases.
Measured: baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2021
- Study startJul 2021 (estimated)
- Primary completionJul 2022 (estimated)
- Study completionMar 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Beta-ThalassemiaStudies of the same condition, open and under way.
- More trials run by Assiut UniversityThe sponsor's other registered studies.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Rehab Mohamed Rashed Gad, Assiut University
- Investigators
- Mohamed M Ghazally, professor, Study director, Egypt
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04947540
- Sponsor's protocol ID
- IS in BTM patients
- Status last verified
- Jun 2021
- First submitted
- 24 Jun 2021
- Last updated
- 19 Jul 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Al-Moshary M, Imtiaz N, Al-Mussaed E, Khan A, Ahmad S, Albqami S. Clinical and Biochemical Assessment of Liver Function Test and Its Correlation with Serum Ferritin Levels in Transfusion-dependent Thalassemia Patients. Cureus. 2020 Apr 7;12(4):e7574. doi: 10.7759/cureus.7574.
PubMed 32391223Amjad F, Fatima T, Fayyaz T, Khan MA, Qadeer MI. Novel genetic therapeutic approaches for modulating the severity of beta-thalassemia (Review). Biomed Rep. 2020 Nov;13(5):48. doi: 10.3892/br.2020.1355. Epub 2020 Sep 2.
PubMed 32953110Salama KM, Ibrahim OM, Kaddah AM, Boseila S, Ismail LA, Hamid MM. Liver Enzymes in Children with beta-Thalassemia Major: Correlation with Iron Overload and Viral Hepatitis. Open Access Maced J Med Sci. 2015 Jun 15;3(2):287-92. doi: 10.3889/oamjms.2015.059. Epub 2015 May 28.
PubMed 27275237
Study at a glance
From the registry record, last updated by the sponsor on 19 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hematological and Biochemical Markers of Iron Status in Thalassemic Children Receiving Multiple Blood Transfusion
- ClinicalTrials.gov ID
- NCT04947540
- Acronym / sponsor ID
- IS in BTM patients
- Conditions
- Beta-Thalassemia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- Jul 2021 (estimated)
- Primary completion
- Jul 2022 (estimated)
- Study completion
- Mar 2023 (estimated)
- First posted
- 1 Jul 2021 (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.
To assess the possible role of iron overload as a cause of liver dysfunction in thalassemic childrens receiving multiple blood transfusion and its correlation with serum aminotransferases.
Detailed description
Thalassemia is derived from the Greek words, thals, which means sea, and emia, which means blood, signifying that it is more common in the Mediterranean region . Globally, among humans, thalassemia is the commonest single-gene disorder. It is defined as a group of inherited disorders characterized by decreased or absent beta globin chain synthesis, leading to a reduced level of hemoglobulin (Hb) in the red blood cells . Specifically in developing countries, thalassemia is a huge health dilemma.
-Beta Thalassemia is the most common chronic hemolytic anemia in Egypt (85.1%) with an estimated carrier rate of 9-10.2%.
Blood transfusion is the primary way of treating thalassemia; it allows the normal growth of the child as well as restrains abnormal erythropoiesis . Iron-chelating agents should be used properly;otherwise, multiple blood transfusions can lead to iron overload. Yet, with no blood transfusion, the increase rate of erythropoiesis intensifies dietary iron absorption from the gut, leading to a severe form of iron overload .
iron overload can result in serious damage to various organs, for example, by depositing in the liver, heart, and various other endocrine glands along with endocrine organ failure. .
During the last years, liver disease has emerged as a major cause of mortality in patients with B- thalassemia major (TM).
The liver is the only site for ferritin and transferrin synthesis, as well as the primary organ for iron storage.
The liver has the maximum capacity to store excess iron in the body, and various other organs, as well as the liver, are very susceptible to damage as a result of iron toxicity.
The correlation between serum ferritin and hepatic iron concentration has been reported in multiple blood-transfused thalassemia patients .
Thalassemia major patients who undergo routine transfusion have an increased risk of acquiring transfusion-transmitted infections (TTI), including hepatitis B and C. These diseases have serious implications and may affect the serum ferritin and aminotransferase levels of thalassemia major patients.
Although the risk of post-transfusion hepatitis C virus (HCV) infection dropped significantly after the national screening of blood in 1993, more than 20% of children who were multitransfused after that date were HCV-RNA positive .
In Egypt, prevalence rate of HCV antibodies seropositivity in thalassemic children at 2011was 51.7% while in the study of El-Faramawy (2012) , a prevalence of 48 % was reported.
Iron overload and hepatitis-C virus (HCV) infection, have been implicated in the evolution of liver disease, in patients with transfusion-dependent beta-thalassaemia major (BTM). The impact of these factors and liver with BTM, has not been extensively studied yet.
Hepatitis virus C infection is the main risk factor for liver injury in transfusion-dependent thalassemics . Dimitrios (2013) on the other hand suggested that in the late stages of liver disease in BTM patients, iron overload may be the critical determinant, since fibrosis is related to the minimal haemosiderosis, independently of HCV history. Injury to the liver, whether acute or chronic, eventually results in an increase in serum concentrations of Alanine transaminase (ALT) and Aspartate transaminase (AST)
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 5 to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- all cases diagnosed as thalassemia major attending to thalassemic center at hematological department at Assuit university childern hospital.
Key inclusion criteria (4 listed)
- Thalassemic patients of both sex.
- Beta-thalassemia major patients diagnosed Clinical and laboratory .
- Age 5 : 18 years.
- Undergoing multiple blood transfusion.
Key exclusion criteria (2 listed)
- -Age less than 5 years.
- Acute illness as fever and infections.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Hematological and biochemical markers of Iron status in thalassemic children receiving multiple blood transfusion
Time frame: baseline
Assessment the possible role of iron overload as a cause of liver dysfunction in thalassemic childrens receiving multiple blood transfusion and its correlation with serum aminotransferases.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jul 2021.
- Background: PubMed 32391223Al-Moshary M, Imtiaz N, Al-Mussaed E, Khan A, Ahmad S, Albqami S. Clinical and Biochemical Assessment of Liver Function Test and Its Correlation with Serum Ferritin Levels in Transfusion-dependent Thalassemia Patients. Cureus. 2020 Apr 7;12(4):e7574. doi: 10.7759/cureus.7574.
- Background: PubMed 32953110Amjad F, Fatima T, Fayyaz T, Khan MA, Qadeer MI. Novel genetic therapeutic approaches for modulating the severity of beta-thalassemia (Review). Biomed Rep. 2020 Nov;13(5):48. doi: 10.3892/br.2020.1355. Epub 2020 Sep 2.
- Background: PubMed 27275237Salama KM, Ibrahim OM, Kaddah AM, Boseila S, Ismail LA, Hamid MM. Liver Enzymes in Children with beta-Thalassemia Major: Correlation with Iron Overload and Viral Hepatitis. Open Access Maced J Med Sci. 2015 Jun 15;3(2):287-92. doi: 10.3889/oamjms.2015.059. Epub 2015 May 28.
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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.
Mohamed M Ghazally, professor study director
Egypt
Questions about this trial
What is this clinical trial studying?
An observational study of Beta-Thalassemia, run by Assiut 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 4 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: status not confirmed, as of the sponsor's last update on 19 Jul 2021. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Rehab M Rashed, pediatrician (01064959241 ext. Egypt, rhabrashed950@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Hematological and biochemical markers of Iron status in thalassemic children receiving multiple blood transfusion (time frame: baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04947540, which also lists 3 linked publications.
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 19 Jul 2021.