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Purpose
This review aims to summarize the evidence and pharmacological characteristics of treatment options for transthyretin amyloid cardiomyopathy (ATTR-CM). Additionally, this review highlights the role of clinical pharmacists in helping to secure newly introduced therapies.
Summary
ATTR-CM, a disease characterized by misfolded protein that is deposited in the myocardium and disrupts cardiac functioning, has historically been underdiagnosed due to the need for invasive biopsy and an illusion of rarity. Once diagnosed, limited treatment modalities for ATTR-CM have led providers to rely on nonpharmacological remedies or off-label use of medications with limited evidence of benefit. However, recent noninvasive diagnostic advancements and heightened disease state awareness have revealed increased prevalence of ATTR-CM. This has led to the introduction of several first-in-class pharmaceuticals with actions targeted at inhibiting the various phases of amyloidosis: TTR stabilizers include diflunisal and first-in-class, Food and Drug Administration (FDA)-approved tafamidis; TTR silencers include patisiran and inotersen; fibril disrupters include doxycycline with tauroursodeoxycholic acid; and alternative agents include green tea extract and curcumin.
Conclusion
ATTR-CM treatments have emerged and, despite current limited data, are continuing to evolve. Tafamidis, the only agent approved by FDA for ATTR-CM, shows promise to improve survival and quality of life in patients with ATTR-CM. Pharmacists can play a key role in assisting with agent selection for this disease state, as well as providing knowledge about current and future clinical trials evaluating the safety and efficacy of the available treatment modalities.
Three factor Xa inhibitors have been studied in the treatment of venous thromboembolism, both for acute therapy and as extended therapy to prevent recurrent events. Rivaroxaban, apixaban, and edoxaban have all proven to be effective in Phase III clinical trials for this indication when compared to current standard of therapy with similar or less bleeding. Nevertheless, the agents all offer different pharmacological profiles, which have an impact on patient selection and potential advantages in clinical practice.
Summary. Stroke prevention in atrial fibrillation is of paramount importance given its associated morbidity and mortality. The many challenges of warfarin limit its effective use in real‐world clinical practice. We are entering an exciting therapeutic era as new classes of anticoagulants, including direct thrombin inhibitors, factor Xa inhibitors and novel vitamin K antagonists, are being evaluated for possible use in this patient population. If proven to be as efficacious as warfarin and safer, expanded use of these novel agents to lower risk subgroups may be justified. It is imperative that providers be aware of the many advantages and potential challenges posed by use of these novel agents in routine clinical care. An understanding of individual pharmacokinetic profiles and potential drug‐drug and drug‐disease interactions will translate into improved effectiveness in real‐world practice.
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