Hereditary ATTR Amyloidosis (hATTR)
Amyloidoses are rare, life-threatening diseases that result from extracellular deposition of amyloid fibrils, which cause organ dysfunction and damage1.
Amyloidosis occurs in the presence of an abnormal protein, as in hereditary transthyretin amyloidosis (hATTR)2 or systemic immunoglobulin light chain (AL) amyloidosis3, or in abundance of a normal protein, such as reactive systemic amyloidosis1.
Variability in clinical presentation and multiorgan involvement pose a challenge for diagnosing amyloidosis1,4,5. Although there is no cure for this disease, recent randomised clinical trials have launched management of the amyloidoses into the realm of evidence-based medicine6-9.
For more on managing AL amyloidosis, click here.
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References
- Muchtar E, Dispenzieri A, Magen H, Grogan M, Mauermann M, McPhail ED, et al. Systemic amyloidosis from A (AA) to T (ATTR): a review. J Inter Med. 2021;289(3):268–292.
- Manganelli F, Fabrizi GM, Luigetti M, Mandich P, Mazzeo A, Pareyson D. Hereditary transthyretin amyloidosis overview. Neurol Sci. 2022;43(S2):595–604.
- Merlini G, Dispenzieri A, Sanchorawala V, Schönland SO, Palladini G, Hawkins PN, et al. Systemic immunoglobulin light chain amyloidosis. Nat Rev Dis Prim. 2018;4(1):38.
- D'Souza A, Osman K, Chase CC, Borham A, Bruno M. The hematologist's role in amyloidosis management: disease awareness, diagnostic workup, and practice patterns. Blood. 2020;136:28–29.
- Carroll A, Dyck PJ, De Carvalho M, Kennerson M, Reilly MM, Kiernan MC, et al. Novel approaches to diagnosis and management of hereditary transthyretin amyloidosis. J Neurol Neurosurg Psychiat. 2022;93(6):668–678.
- Kastritis E, Leleu X, Arnulf B, Zamagni E, Cibeira MT, Kwok F, et al. Bortezomib, melphalan, and dexamethasone for light-chain amyloidosis. J Clin Oncol. 2020;38(28):3252–3260.
- Dispenzieri A, Kastritis E, Wechalekar AD, Schönland SO, Kim K, Sanchorawala V, et al. A randomized phase 3 study of ixazomib-dexamethasone versus physician’s choice in relapsed or refractory AL amyloidosis. Leukemia. 2022;36(1):225–235.
- Kastritis E, Palladini G, Minnema MC, Wechalekar AD, Jaccard A, Lee HC, et al. Daratumumab-based treatment for immunoglobulin light-chain amyloidosis. New Eng J Med. 2021;385(1):46–58.
- Gertz MA, Mauermann ML, Grogan M, Coelho T. Advances in the treatment of hereditary transthyretin amyloidosis: A review. Brain Behav. 2019;9(9):e01371.