Contributions
Abstract: PB1755
Type: Publication Only
Session title: Quality of life, palliative care, ethics and health economics
Background
The National Hemoglobinopathy Registry of the Turkish Society of Hematology (TSH) includes all resident patients in Turkey.
Aims
It aims to assess the efficacy of the national hemoglobinopathy control program (HCP), the changing pattern of the epidemiology, and the standardization of high-quality health care overall in Turkey.
Methods
The registry has been approved by the ethics committee of the Ege University. Participating physicians recorded patients' data into the database after receiving informed consent from the patients/legal representatives. The data collected during the last 18 months were extracted onto an Excel spreadsheet for analysis in January 2021.
Results
The table shows the characteristics of the 3554 patients. Overall, 81.5% of patients had thalassemia, and 18.5% sickle-cell disorders (SCD). A comprehensive HCP was activated, in Turkey, in 2002. A total of 1262 affected births were registered in the database since 2003. While 40% of the parents of affected births had a pre-marital screening, only 50% of those couples stated having appropriate genetic counseling. Eighty percent of couples having genetic counseling did not get a prenatal diagnosis (PND). The rest (20%) gave birth to affected babies despite PND. Notably, 30% of patients had at least one affected sibling born before the patient's birth, and 24% of patients had at least one affected sibling after the patient's birth. Although there has been a 33% reduction in the number of affected subjects (~52/year) since 2015, compared to the prior 5-year cohort (~80/year), the rate is still high, and this declining pattern requires further confirmation. The registry also includes 428 affected children from refugee families, of whom 30% were born in Turkey during the last decade. Overall, 295 patients (13 refugees) with thalassemia and 9 (1 refugee) with SCD, aged 8.7±5.4 (median 7.0), had bone marrow transplantation (BMT). The majority (82%) of BMTs were performed after 2010. The 2-year overall survival was 98%, and event-free survival was found 89% in those (MSD 179, MFD 29, UD 29) followed at least 2-years after BMT. Cardiac failure and arrhythmias were the most common causes of death in thalassemia until the last decade. The rate of cardiac deaths in the 'at-risk' population significantly decreased, while the mean age of deaths was increased in the decades following (p=0.02), and infections, particularly in association with transplantation, have replaced cardiac deaths in the period between 2010-2019. Cardiac and infectious complications were also the leading causes of death in patients with SCD. Acute chest syndrome, renal failure, and thrombosis comprised other frequent causes of death in this group. Survival analysis of patients with thalassemia & SCD by 10-year intervals demonstrated that >90% of patients were alive in each 10-year cohort since 1960.
Conclusion
Turkey is a country with a high young patient burden of hemoglobinopathies. As shown by the present data, these patients will survive by using the advantages of full access to conventional treatment. Despite good outcomes, BMT as a curative approach has remained limited to a suitable donor. Therefore, a more effective HCP should be in place to reduce the economic burden on the healthcare system. The program should offer prenatal testing in couples of the immigrant population. The longitudinal hemoglobinopathy registry is needed for national surveillance to organize healthcare resources properly.
Keyword(s): Health care, Prevention, Sickle cell disease, Thalassemia
Abstract: PB1755
Type: Publication Only
Session title: Quality of life, palliative care, ethics and health economics
Background
The National Hemoglobinopathy Registry of the Turkish Society of Hematology (TSH) includes all resident patients in Turkey.
Aims
It aims to assess the efficacy of the national hemoglobinopathy control program (HCP), the changing pattern of the epidemiology, and the standardization of high-quality health care overall in Turkey.
Methods
The registry has been approved by the ethics committee of the Ege University. Participating physicians recorded patients' data into the database after receiving informed consent from the patients/legal representatives. The data collected during the last 18 months were extracted onto an Excel spreadsheet for analysis in January 2021.
Results
The table shows the characteristics of the 3554 patients. Overall, 81.5% of patients had thalassemia, and 18.5% sickle-cell disorders (SCD). A comprehensive HCP was activated, in Turkey, in 2002. A total of 1262 affected births were registered in the database since 2003. While 40% of the parents of affected births had a pre-marital screening, only 50% of those couples stated having appropriate genetic counseling. Eighty percent of couples having genetic counseling did not get a prenatal diagnosis (PND). The rest (20%) gave birth to affected babies despite PND. Notably, 30% of patients had at least one affected sibling born before the patient's birth, and 24% of patients had at least one affected sibling after the patient's birth. Although there has been a 33% reduction in the number of affected subjects (~52/year) since 2015, compared to the prior 5-year cohort (~80/year), the rate is still high, and this declining pattern requires further confirmation. The registry also includes 428 affected children from refugee families, of whom 30% were born in Turkey during the last decade. Overall, 295 patients (13 refugees) with thalassemia and 9 (1 refugee) with SCD, aged 8.7±5.4 (median 7.0), had bone marrow transplantation (BMT). The majority (82%) of BMTs were performed after 2010. The 2-year overall survival was 98%, and event-free survival was found 89% in those (MSD 179, MFD 29, UD 29) followed at least 2-years after BMT. Cardiac failure and arrhythmias were the most common causes of death in thalassemia until the last decade. The rate of cardiac deaths in the 'at-risk' population significantly decreased, while the mean age of deaths was increased in the decades following (p=0.02), and infections, particularly in association with transplantation, have replaced cardiac deaths in the period between 2010-2019. Cardiac and infectious complications were also the leading causes of death in patients with SCD. Acute chest syndrome, renal failure, and thrombosis comprised other frequent causes of death in this group. Survival analysis of patients with thalassemia & SCD by 10-year intervals demonstrated that >90% of patients were alive in each 10-year cohort since 1960.
Conclusion
Turkey is a country with a high young patient burden of hemoglobinopathies. As shown by the present data, these patients will survive by using the advantages of full access to conventional treatment. Despite good outcomes, BMT as a curative approach has remained limited to a suitable donor. Therefore, a more effective HCP should be in place to reduce the economic burden on the healthcare system. The program should offer prenatal testing in couples of the immigrant population. The longitudinal hemoglobinopathy registry is needed for national surveillance to organize healthcare resources properly.
Keyword(s): Health care, Prevention, Sickle cell disease, Thalassemia