Tata Laksana Terkini Talasemia Beta: Terapi Target

Susi Susanah

Sari


Talasemia beta adalah bentuk hemoglobinopati yang merupakan penyakit monogenik diturunkan terbanyak di dunia ditandai defek yang menyebabkan produksi globin beta berkurang atau tidak ada.  Ketidakseimbangan rantai globin alfa/beta menyebabkan rangkaian proses eritropoesis inefektif dan peningkatan absorpsi besi yang pada akhirnya mengakibatkan anemia hemolitik kronis dan kelebihan besi.  Secara konvensional tata laksana utama talasemia beta berat adalah transfusi darah dan obat kelasi besi yang masih memiliki banyak keterbatasan dan tantangan meskipun telah berdampak pada peningkatan kesintasan dan kualitas hidup penyandang talasemia beta mayor. Pemahaman mendalam terhadap molekular dan patofisiologi talasemia-beta membuka jalan bagi strategi pendekatan terapi baru yang diklasifikasikan atas 3 kategori, yaitu koreksi ketidakseimbangan rantai globin melalui pengembangan transplantasi sumsum tulang dan terapi gen; mengintervensi eritropoesis inefektif sehingga transfusi darah dan kelasi besi berkurang; dan modulasi disregulasi besi untuk mengendalikan kadar besi. Dengan demikian, strategi pendekatan terapi baru menjanjikan penurunan kebutuhan transfusi darah dan kelasi besi yang lebih menyamankan pasien dan diharapkan juga menurunkan biaya tata laksana. 


Kata Kunci


talasemia beta; patofisiologi; eritropoesis inefektif; disregulasi besi; terapi target

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Referensi


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DOI: http://dx.doi.org/10.14238/sp24.4.2022.279-85

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