BURDEN OF THALASSEMIA IN SAUDI ARABIA: REAL-WORLD EVIDENCE FROM THE NATIONAL UNIFIED ELECTRONIC HEALTH RECORD SYSTEM
Author(s)
Hatoun Ezzat, Medical Doctor1, Rawan Almasuood, Pharmacy2, Wejdan Aburas, Pharmacy3, Hind Hajj, Pharmacy4, Nancy Awad, Pharmacy5.
1General Supervisor of Blood Disorders Administration, Ministry of Health, Riyadh, Saudi Arabia, 2Health Technology Assessment Manager, Therapeutic Affairs deputyship, Ministry of Health, Riyadh, Saudi Arabia, 3Director of the Drug Policy and Regulation, Therapeutic Affairs deputyship, Ministry of Health, Riyadh, Saudi Arabia, 4Sr. Health Policy and Access Strategy Manager, Masdar Al Hekmah, Riyadh, Saudi Arabia, 5Chief Executive Officer, Masdar Al Hekmah, Riyadh, Saudi Arabia.
1General Supervisor of Blood Disorders Administration, Ministry of Health, Riyadh, Saudi Arabia, 2Health Technology Assessment Manager, Therapeutic Affairs deputyship, Ministry of Health, Riyadh, Saudi Arabia, 3Director of the Drug Policy and Regulation, Therapeutic Affairs deputyship, Ministry of Health, Riyadh, Saudi Arabia, 4Sr. Health Policy and Access Strategy Manager, Masdar Al Hekmah, Riyadh, Saudi Arabia, 5Chief Executive Officer, Masdar Al Hekmah, Riyadh, Saudi Arabia.
OBJECTIVES: Thalassemia imposes a lifelong burden due to chronic anemia, transfusion dependence, iron overload, and multi-organ complications. In Saudi Arabia, the burden remains insufficiently characterized, highlighting the need for real-world evidence to support optimized healthcare planning. The objective is to evaluate the real-world burden of α- and β-thalassemia in Saudi Arabia using LEAN healthcare services data from the Ministry of Health perspective.
METHODS: De-identified linked electronic medical record, prescription, and laboratory data were extracted and analyzed using LEAN unified electronic health record system. Patients were identified using ICD-10-CM codes for α- and β-thalassemia. Adults with transfusion- or non-transfusion-dependent disease between 2016 and 2026 were included. The dataset captured demographics, comorbidities, healthcare utilization, and treatment patterns. Follow-up extended from index date to January 2026 or death (≥12 months minimum observation).
RESULTS: A total of 2,802 patients were included (mean age 35.9 years; 60.8% female), predominantly β-thalassemia (68.7%) and α-thalassemia (28.4%). β-thalassemia showed higher comorbidity burden than α-thalassemia, particularly diabetes, hypertension, and cardiovascular disease, while hospitalization rates were higher in α-thalassemia (0.19 vs 0.13 per patient-year). Laboratory testing was low, with hemoglobin testing in 4.5% and ferritin testing in 30.4%; among those tested, 20.2% had ferritin >1000 ng/mL and 50% had hemoglobin <10 g/dL, while 19.3% had ALT >40 U/L. Treatment was dominated by folic acid (28.7%) and ferrous sulfate (15.2%), with lower use of iron chelation therapy (3%). Routine monitoring tests remained low. Complications were driven by hemolysis, transfusion-related effects, iron overload-related endocrine disease, and arrhythmias, particularly in β-thalassemia. The annual cost of routine care was SAR 32.1 million, complications SAR 65.1 million, and comorbidities SAR 20.1 million.
CONCLUSIONS: Monitoring and treatment patterns among thalassemia patients remain suboptimal, underscoring gaps in care pathways and the need for improved surveillance and more effective therapies.
METHODS: De-identified linked electronic medical record, prescription, and laboratory data were extracted and analyzed using LEAN unified electronic health record system. Patients were identified using ICD-10-CM codes for α- and β-thalassemia. Adults with transfusion- or non-transfusion-dependent disease between 2016 and 2026 were included. The dataset captured demographics, comorbidities, healthcare utilization, and treatment patterns. Follow-up extended from index date to January 2026 or death (≥12 months minimum observation).
RESULTS: A total of 2,802 patients were included (mean age 35.9 years; 60.8% female), predominantly β-thalassemia (68.7%) and α-thalassemia (28.4%). β-thalassemia showed higher comorbidity burden than α-thalassemia, particularly diabetes, hypertension, and cardiovascular disease, while hospitalization rates were higher in α-thalassemia (0.19 vs 0.13 per patient-year). Laboratory testing was low, with hemoglobin testing in 4.5% and ferritin testing in 30.4%; among those tested, 20.2% had ferritin >1000 ng/mL and 50% had hemoglobin <10 g/dL, while 19.3% had ALT >40 U/L. Treatment was dominated by folic acid (28.7%) and ferrous sulfate (15.2%), with lower use of iron chelation therapy (3%). Routine monitoring tests remained low. Complications were driven by hemolysis, transfusion-related effects, iron overload-related endocrine disease, and arrhythmias, particularly in β-thalassemia. The annual cost of routine care was SAR 32.1 million, complications SAR 65.1 million, and comorbidities SAR 20.1 million.
CONCLUSIONS: Monitoring and treatment patterns among thalassemia patients remain suboptimal, underscoring gaps in care pathways and the need for improved surveillance and more effective therapies.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE433
Topic
Economic Evaluation, Health Technology Assessment, Real World Data & Information Systems
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)