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The Utah Amyloidosis Program Contributes to the First FDA Approved Medications for Amyloidosis and Partners With the American Heart Association to Develop Scientific Statements

Jan 22, 2021

Amyloidosis was considered a fatal disease characterized by protein misfolding, fibril deposition, and multi-organ failure. In the last two years, novel therapies received FDA approval for the management of amyloidosis. The Utah Amyloidosis Program participated in the 1)ANDROMEDA clinical trial of Daratumumab in patients with light chain amyloidosis;¹ 2) the NeuroATTR extension study of Inotersen in patients with hereditary transthyretin amyloidosis,² and 3) the ATTR-ACT trial of Tafamidis in patients with wild-type transthyretin amyloidosis.³ These therapies are the first ones to improve the survival and quality of life, specifically for patients with amyloidosis, and provides new hope for a disease considered to be fatal.

The studies demonstrated that patients in the early stages have better outcomes, making an early diagnosis a priority for this population. With this importance, the American Heart Association (AHA) developed the first Scientific Statement to manage patients with transthyretin cardiac amyloidosis.ª The Utah Amyloidosis program participated in developing this statement, which summarizes the diagnostic algorithms, including non-invasive pathways with pyrophosphate scans, and describes the approach to different therapeutic options.

With all of these advancements in the field, the Utah Amyloidosis Program continues to grow with the addition of several new members in order to provide expedite, comprehensive care, with the recent additions of:

  • Amandeep Godara, MD – Hematology
  • Zachary Francom – Amyloidosis Program Coordinator
  • Chris Blackburn – Hematology/Cardiology Nurse
  • Samantha Birchfield – Hematology Nurse
  • Ivana Grbelja – Cardiovascular Genetic Counselor
  • Patricia Miguez – Cardiovascular Post Doctoral Amyloidosis Fellow

¹Palladini G, Kastritis E, Maurer MS, Zonder J, Minnema MC, Wechalekar AD, Jaccard A, Lee HC, Bumma N, Kaufman JL, Medvedova E, Kovacsovics T, Rosenzweig M, Sanchorawala V, Qin X, Vasey SY, Weiss BM, Vermeulen J, Merlini G, Comenzo RL. Daratumumab plus CyBorD for patients with newly diagnosed AL amyloidosis: safety run-in results of ANDROMEDA. Blood. 2020 Jul 2;136(1):71-80. doi: 10.1182/blood.2019004460.

²Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, Planté-Bordeneuve V, Barroso FA, Merlini G, Obici L, Scheinberg M, Brannagan TH 3rd, Litchy WJ, Whelan C, Drachman BM, Adams D, Heitner SB, Conceição I, Schmidt HH, Vita G, Campistol JM, Gamez J, Gorevic PD, Gane E, Shah AM, Solomon SD, Monia BP, Hughes SG, Kwoh TJ, McEvoy BW, Jung SW, Baker BF, Ackermann EJ, Gertz MA, Coelho T. Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018 Jul 5;379(1):22-31. doi: 10.1056/NEJMoa1716793.

³Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M, Kristen AV, Grogan M, Witteles R, Damy T, Drachman BM, Shah SJ, Hanna M, Judge DP, Barsdorf AI, Huber P, Patterson TA, Riley S, Schumacher J, Stewart M, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy. N Engl J Med. 2018 Sep 13;379(11):1007-1016. doi: 10.1056/NEJMoa1805689. Epub 2018 Aug 27.

ªMichelle M Kittleson, Mathew S Maurer, Amrut V Ambardekar, Renee P Bullock-Palmer, Patricia P Chang, Howard J Eisen, Ajith P Nair, Jose Nativi-Nicolau, Frederick L Ruberg, American Heart Association Heart Failure and Transplantation Committee of the Council on Clinical Cardiology. Cardiac Amyloidosis: Evolving Diagnosis and Management: A Scientific Statement From the American Heart Association.  Circulation . 2020 Jul 7;142(1):e7-e22. doi: 10.1161/CIR.0000000000000792.