Doxycycline plus tauroursodeoxycholic acid for transthyretin amyloidosis: a phase II study

Obici, Laura; Cortese, Andrea; Lozza, Alessandro; Lucchetti, Jacopo; Gobbi, Marco; Palladini, Giovanni; Perlini, Stefano; Saraiva, Maria João; Merlini, Giampaolo

doi:10.3109/13506129.2012.678508

Cited by 198 publications

(128 citation statements)

References 5 publications

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“…Diflunisal, a non-steroid anti-inflammatory drug, has an identical effect and a combination of tauroursodeoxycholic acid (TUDCA) with doxycycline has an effect on the disruption of amyloid masses. Clinical studies are in progress [74][75][76][77][78] .…”

Section: Ttr Amyloidosismentioning

confidence: 99%

Amyloid cardiomyopathy

Karafiátová¹,

Pika²

2017

BIOMED PAP

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Section: Ttr Amyloidosismentioning

confidence: 99%

Amyloid cardiomyopathy

Karafiátová¹,

Pika²

2017

BIOMED PAP

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“…Flushing and dyspeptic symptoms due to doxycycline were the most frequent adverse events. Due to the preliminary results of that study, which included 20 patients (one patient had a domino transplantation), patients were stable in terms of neurologic and cardiac status (45).…”

Section: Doxycycline+tauroursodeoxycholic Acid (Tudca)mentioning

confidence: 99%

Transthyretin-Related Familial Amyloid Polyneuropathy: In the Light of New Developments

Çakar¹,

Tekçe²,

Deymeer³

et al. 2017

tnd

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“…An Italian phase 2 study of this drug combination has been started in patients with ATTR amyloidosis [49]. This might also be a useful approach in AL amyloidosis by reducing fibril formation as shown in a transgenic mouse model [50].…”

Section: Treatmentmentioning

confidence: 99%

The Prevalence and Management of Systemic Amyloidosis in Western Countries

Nienhuis

Bijzet

Hazenberg³

2016

Kidney Dis

106

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Background: Amyloidosis has been a mystery for centuries, but research of the last decennia has clarified many of the secrets of this group of diseases. A protein-based classification of amyloidosis helps to understand problems that were part of the obsolete clinical classification in primary, secondary, and familial amyloidosis. All types of amyloid are secondary to some underlying precursor-producing process: each type is caused by a misfolded soluble precursor protein that becomes deposited as insoluble amyloid fibrils. Summary: The incidence of amyloidosis is not well documented, but probably falls between 5 and 13 per million per year. Prevalence data are scarce, but one UK study indicates about 20 per million inhabitants. Amyloidosis can be localized (amyloid deposited in the organ or tissue of precursor production) or systemic (amyloid at one or more sites distant from the site of precursor production). The major systemic types of amyloidosis are AL (associated with a light chain-producing plasma cell dyscrasia), AA (associated with longstanding inflammation), wild-type ATTR (associated with normal transthyretin and old age), and hereditary ATTR (associated with a transthyretin mutation) amyloidosis. Imaging techniques, such as cardiac ultrasound, magnetic resonance imaging, bone scintigraphy, and serum amyloid P component scintigraphy, are useful both for diagnosing amyloidosis and for assessing disease severity. Serologic markers are useful for detecting organ disease and disease monitoring during follow-up. Current treatment modalities are directed against the ongoing supply of precursor proteins and thereby aim to stop further accumulation of amyloid. Novel treatment modalities, such as interference with amyloid formation and even removal of amyloid, are being studied. A well-thought and planned monitoring during follow-up helps to assess the effect of treatment and to early detect possible progression of amyloidosis. Key Messages: Clinical management comprises histologic proof of amyloid, evidence of systemic deposition, reliable typing, precursor assessment, severity of organ disease, risk assessment and prognosis, choice of treatment, and planned monitoring during follow-up. Facts from East and West: (1) AL amyloidosis is the most prevalent type of amyloidosis accounting for 65% of the amyloidosis-diagnosed patients in the UK and for 93% of the amyloidosis-diagnosed patients in China. The predisposition of men over women to develop AL amyloidosis might be higher in China than in Western countries (2:1 vs. 1.3:1). Both in the East and West, incidence increases with age. At the time of diagnosis, edema is twice as frequent and the proportion of renal involvement is higher in Chinese compared to Western patients. (2) Melphalan followed by autologous stem cell transplantation (ASCT) is the current standard therapy but is restricted to eligible patients. The efficacy and safety of bortezomib combined with dexamethasone were proven in Western patients and recently confirmed in a Chinese cohort. Recent stu...

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Doxycycline plus tauroursodeoxycholic acid for transthyretin amyloidosis: a phase II study

Cited by 198 publications

References 5 publications

Amyloid cardiomyopathy

Amyloid cardiomyopathy

Transthyretin-Related Familial Amyloid Polyneuropathy: In the Light of New Developments

The Prevalence and Management of Systemic Amyloidosis in Western Countries

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