T h e C u rr e n t o f P o t e n t i a l T h e r a p y f o r T h a l a ss e m i a Intan Nur’aini Nirmala Subarkah 1 , Syahrul Tuba 2 , Nafrizal Fakhruzain 3 Auditorium Theater RIDU, Indonesia Faculty of Military Pharmacy The Republic of Indonesia Defense University The 4 th International Conference ICOPMAP on Pharmaceutical Sciences and Military Pharmacy | September 6 – 7 2024, Sentul, Bogor
Thalassemia a blood disorder condition when the hemoglobin levels produced are abnormal therapeutic treatments that are still being explored for their efficacy in treating specific conditions. 1.3 million fatalities from thalassemia worldwide in 2021 Indonesia’s population with thalassemia 4.896 in 2012 to 10.973 in 2021 Source : Global burden, distribution and prevention of β- thalassemias and hemoglobin E disorders. Colah, R., et al . (2010). Potential Therapy Estimated number of β -thalassemia-affected newborns per year The 4 th International Conference ICOPMAP on Pharmaceutical Sciences and Military Pharmacy | September 6 – 7 2024, Sentul, Bogor Faculty of Military Pharmacy The Republic of Indonesia Defense University Background
Objective Thalassemia rare disease Bone Marrow Transplantation as Potential Therapy To analyze the current of scope of options available and in the future for treating thalassemia disorders based on the mechanism of action, benefits, and risks of new thalassemia therapies. The 4 th International Conference ICOPMAP on Pharmaceutical Sciences and Military Pharmacy | September 6 – 7 2024, Sentul, Bogor Faculty of Military Pharmacy The Republic of Indonesia Defense University
Materials and Methods The 4 th International Conference ICOPMAP on Pharmaceutical Sciences and Military Pharmacy | September 6 – 7 2024, Sentul, Bogor Faculty of Military Pharmacy The Republic of Indonesia Defense University Scientific journals from leading platforms like PubMed, CrossRef, and Google Scholar in English were collected to create a narrative review.
Results and Discussion Thalassemia Hereditary Disease (Genetic Disorder) Hematoglobin Level Analysis Thalassemia : 8- 10 g/dL Normal : 10- 15,5 g/dL (in age 6- 18 years) ALT Analysis Thalassemia : > 34 u/L Normal : 7-33 u/L Creatinine Analysis Thalassemia : 0,2 mg/ dL – 0,7 mg/dL Normal : [0,6 - 1,2 mg/dL (man); 0,5 - 1,1 mg/dL (woman)] Blood Transfusion POTENTIAL THERAPY Stem Cell Therapy with Bone Marrow Transplantation Using Parameter The 4 th International Conference ICOPMAP on Pharmaceutical Sciences and Military Pharmacy | September 6 – 7 2024, Sentul, Bogor Faculty of Military Pharmacy The Republic of Indonesia Defense University
Conclusion The 4 th International Conference ICOPMAP on Pharmaceutical Sciences and Military Pharmacy | September 6 – 7 2024, Sentul, Bogor Faculty of Military Pharmacy The Republic of Indonesia Defense University The risk of blood accumulation in blood transfusion therapy will cause more serious effects if not treated immediately. Bone marrow transplantation can be a potential therapy for thalassemia and replace blood transfusion therapy especially for β- thalassemia major patients.