Droxidopa for the treatment of neurogenic orthostatic hypotension in neurodegenerative diseases

Pérez-Lloret, Santiago; Quarracino, Cecilia; Otero‐Losada, Matilde; Rascol, Olivier

doi:10.1080/14656566.2019.1574746

Cited by 11 publications

(9 citation statements)

References 78 publications

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“… 70 , 71 Since orthostatic hypotension can be challenging to treat, droxidopa may be used as an adjunct to midodrine and compression stockings and was recently approved specifically for management of symptomatic neurogenic orthostatic hypotension. 72 Pyridostigmine, the acetylcholinesterase inhibitor, can also be used but has not been specifically studied in CA. 73 Fludrocortisone should be avoided due to risk of volume retention.…”

Section: General Cardiac Managementmentioning

confidence: 99%

Cardiac Amyloidosis Treatment

Stern

Patel

2022

Methodist DeBakey Cardiovascular Journal

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Cardiac amyloidosis (CA) is a restrictive cardiomyopathy with a traditionally poor prognosis. Until recently, CA treatment options were limited and consisted predominantly of managing symptoms and disease-related complications. However, the last decade has seen significant advances in disease-modifying therapies, increased awareness of CA, and improved diagnostic methods resulting in earlier diagnoses. In this review, we provide an overview of current and experimental treatments for the predominant types of CA: transthyretin cardiac amyloidosis (ATTR-CA) and immunoglobulin light chain (AL)-mediated CA (AL-CA). The mainstay of AL-CA treatment is proteasome inhibitor-based chemotherapy with daratumumab and, when feasible, autologous stem cell transplantation. For ATTR-CA, the stabilizer tafamidis is the only US Food and Drug Administration (FDA)-approved treatment. However, promising novel therapies on the horizon target various points in the ATTR-CA amyloidogenic cascade. These include transthyretin gene ( TTR) silencing agents to prevent TTR formation, TTR tetramer stabilization and inhibition of oligomer aggregation to prevent fibril formation, anti-TTR fiber antibodies, and amyloid degradation. For end-stage CA, advanced interventions may need to be considered, including heart, heart-kidney, and, for hereditary ATTR-CA, heart-liver transplantation. Despite the evolution of treatment options, CA management remains complex due to patient frailty and therapeutic side effects or intolerance with advanced cardiac disease. This is particularly relevant for those with AL-CA, when active teamwork between the hematologist-oncologist and the cardiologist is critical for treatment success. Often, referral to an expert center is necessary for timely diagnosis, initiation of treatment, and participation in clinical trials.

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Section: General Cardiac Managementmentioning

confidence: 99%

Cardiac Amyloidosis Treatment

Stern

Patel

2022

Methodist DeBakey Cardiovascular Journal

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“…The anticholinergic pyridostigmine can also improve neurogenic orthostatic hypotension, 59 and some centres are also using droxidopa as a support for orthosthatic blood pressure in both neurodegenerative diseases and AL. 60,61 Fludrocortisone can be helpful in some patients who do not show a good response to midodrine but may cause or exacerbate fluid retention. disease-specific therapies due to cardiovascular compromise may be considered for orthotopic heart transplantation (OHT) in experienced centres with established collaborations between cardiovascular and haematology teams.…”

Section: Supportive Therapiesmentioning

confidence: 99%

Progress and challenges in the treatment of cardiac amyloidosis: a review of the literature

et al. 2021

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Cardiac amyloidosis is a restrictive cardiomyopathy determined by the accumulation of amyloid, which is represented by misfolded protein fragments in the cardiac extracellular space. The main classification of systemic amyloidosis is determined by the amyloid precursor proteins causing a very heterogeneous disease spectrum, but the main types of amyloidosis involving the heart are light chain (AL) and transthyretin amyloidosis (ATTR). AL, in which the amyloid precursor is represented by misfolded immunoglobulin light chains, can involve almost any system carrying the worst prognosis among amyloidosis patients. This has however dramatically improved in the last few years with the increased usage of the novel therapies such as proteasome inhibitors and haematopoietic cell transplantation, in the case of timely diagnosis and initiation of treatment. The treatment for AL is directed by the haematologist working closely with the cardiologist when there is a significant cardiac involvement. Transthyretin (TTR) is a protein that is produced by the liver and is involved in the transportation of thyroid hormones, especially thyroxine and retinol binding protein. ATTR results from the accumulation of transthyretin amyloid in the extracellular space of different organs and systems, especially the heart and the nervous system. Specific therapies for ATTR act at various levels of TTR, from synthesis to deposition: TTR tetramer stabilization, oligomer aggregation inhibition, genetic therapy, amyloid fibre degradation, antiserum amyloid P antibodies, and antiserum TTR antibodies. Treatment of systemic amyloidosis has dramatically evolved over the last few years in both AL and ATTR, improving disease prognosis. Moreover, recent studies revealed that timely treatment can lead to an improvement in clinical status and in a regression of amyloid myocardial infiltration showed by imaging, especially by cardiac magnetic resonance, in both AL and ATTR. However, treating cardiac amyloidosis is a complex task due to the frequent association between systemic congestion and low blood pressure, thrombo-embolic and haemorrhagic risk balance, patient frailty, and generally poor prognosis. The aim of this review is to describe the current state of knowledge regarding cardiac amyloidosis therapy in this constantly evolving field, classified as treatment of the cardiac complications of amyloidosis (heart failure, rhythm and conduction disturbances, and thrombo-embolic risk) and the disease-modifying therapy.

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“…Finalmente, la droxidopa es un profármaco que se convierte en noradrenalina en tejidos periféricos y en el sistema nervioso central. Aunque no está comercializada ni aprobada en España, sí ha demostrado eficacia en la EP y la AMS, aunque los efectos a medio y largo plazo son más discutibles 51 . Muchas personas con AMS (40%) pueden presentar hipertensión supina por la noche, por lo que en ocasiones son necesarios antihipertensivos de corta duración (captopril 25 mg o parches de nitroglicerina).…”

Section: Disautonomíaunclassified

“…El estreñimiento es frecuente y se ha de tratar con recomendaciones dietético-higiénicas: buena hidratación, dieta rica en fibra, aceite de oliva, probióticos y ejercicio. Si el problema persiste se pueden añadir laxantes formadores de volumen, laxantes osmóticos y enemas en casos resistentes 51 .…”

Section: Disautonomíaunclassified

Atrofia multisistémica

Soriano¹

2021

KRANION

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Droxidopa for the treatment of neurogenic orthostatic hypotension in neurodegenerative diseases

Cited by 11 publications

References 78 publications

Cardiac Amyloidosis Treatment

Cardiac Amyloidosis Treatment

Progress and challenges in the treatment of cardiac amyloidosis: a review of the literature

Atrofia multisistémica

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