Amyloidosis medical therapy: Difference between revisions
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==Medical Therapy== | ==Medical Therapy== | ||
=== Primary Amyloidosis (AL): === | ===Primary Amyloidosis (AL):=== | ||
Some patients with primary [[amyloidosis]] respond to [[chemotherapy]] focused on the abnormal [[plasma cell]]s. A [[stem cell transplant]] may be done, as in [[multiple myeloma]]. | Some patients with primary [[amyloidosis]] respond to [[chemotherapy]] focused on the abnormal [[plasma cell]]s. A [[stem cell transplant]] may be done, as in [[multiple myeloma]]. | ||
*The initial step in the treatment of this disorder is to correct the [[organ failure]], since the disease is discovered at an advanced stage when multiple [[organ systems]] may be affected. | *The initial step in the treatment of this disorder is to correct the [[organ failure]], since the disease is discovered at an advanced stage when multiple [[organ systems]] may be affected. | ||
**[[Nephrotic syndrome]] is treated using supportive therapy and [[diuretics]]. | **[[Nephrotic syndrome]] is treated using supportive therapy and [[diuretics]]. | ||
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Treatment options with limited success include [[melphalan]], [[prednisone]], and [[colchicine]]. | Treatment options with limited success include [[melphalan]], [[prednisone]], and [[colchicine]]. | ||
=== Secondary Amyloidosis (AA): === | ===Secondary Amyloidosis (AA):=== | ||
* Medical or surgical treatment of the underlying chronic infection or inflammatory disease is recommended among all patients who develop AA amyloidosis. | *Medical or surgical treatment of the underlying chronic infection or inflammatory disease is recommended among all patients who develop AA amyloidosis. | ||
* Aggressively treating the disease that is causing the excess [[amyloid]] protein can improve [[symptoms]] and slow down or halt the progression of the disease. | *Aggressively treating the disease that is causing the excess [[amyloid]] protein can improve [[symptoms]] and slow down or halt the progression of the disease. | ||
* Complications such as [[heart failure]], [[renal failure]], and other problems can sometimes be treated, when needed. | *Complications such as [[heart failure]], [[renal failure]], and other problems can sometimes be treated, when needed. | ||
*Although the choice of therapy depends on the underlying cause of chronic inflammation, the aim is always to suppress production of SAA to within the normal range. | *Although the choice of therapy depends on the underlying cause of chronic inflammation, the aim is always to suppress production of SAA to within the normal range. | ||
*Examples of treatments for the commonest disorders underlying AA amyloidosis:<ref name="pmid30274625">{{cite journal| author=Papa R, Lachmann HJ| title=Secondary, AA, Amyloidosis. | journal=Rheum Dis Clin North Am | year= 2018 | volume= 44 | issue= 4 | pages= 585-603 | pmid=30274625 | doi=10.1016/j.rdc.2018.06.004 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=30274625 }} </ref> | *Examples of treatments for the commonest disorders underlying AA amyloidosis:<ref name="pmid30274625">{{cite journal| author=Papa R, Lachmann HJ| title=Secondary, AA, Amyloidosis. | journal=Rheum Dis Clin North Am | year= 2018 | volume= 44 | issue= 4 | pages= 585-603 | pmid=30274625 | doi=10.1016/j.rdc.2018.06.004 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=30274625 }} </ref> | ||
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|- | |- | ||
| rowspan="3" style="padding: 5px 5px; background: #DCDCDC;" |Inflammatory arthritis | | rowspan="3" style="padding: 5px 5px; background: #DCDCDC;" |Inflammatory arthritis | ||
| style="padding: 5px 5px; background: #F5F5F5;" | Conventional disease-modifying agents | | style="padding: 5px 5px; background: #F5F5F5;" |Conventional disease-modifying agents | ||
| style="padding: 5px 5px; background: #F5F5F5;" | | | style="padding: 5px 5px; background: #F5F5F5;" | | ||
*Gold | *Gold | ||
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|- | |- | ||
| rowspan="3" style="padding: 5px 5px; background: #DCDCDC;" |Periodic fevers | | rowspan="3" style="padding: 5px 5px; background: #DCDCDC;" |Periodic fevers | ||
| style="padding: 5px 5px; background: #F5F5F5;" | On-demand agents | | style="padding: 5px 5px; background: #F5F5F5;" |On-demand agents | ||
| style="padding: 5px 5px; background: #F5F5F5;" | | | style="padding: 5px 5px; background: #F5F5F5;" | | ||
*Nonsteroidal anti-inflammatory drugs | *Nonsteroidal anti-inflammatory drugs | ||
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|- | |- | ||
| rowspan="5" style="padding: 5px 5px; background: #DCDCDC;" |Inflammatory bowel disease | | rowspan="5" style="padding: 5px 5px; background: #DCDCDC;" |Inflammatory bowel disease | ||
| style="padding: 5px 5px; background: #F5F5F5;" | Conventional disease-modifying agents | | style="padding: 5px 5px; background: #F5F5F5;" |Conventional disease-modifying agents | ||
| style="padding: 5px 5px; background: #F5F5F5;" | | | style="padding: 5px 5px; background: #F5F5F5;" | | ||
*Sulfasalazine | *Sulfasalazine | ||
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ileo-cecal resection and primary reconstruction | ileo-cecal resection and primary reconstruction | ||
|- | |- | ||
| rowspan="2" style="padding: 5px 5px; background: #DCDCDC;" | Immunodeficiency | | rowspan="2" style="padding: 5px 5px; background: #DCDCDC;" |Immunodeficiency | ||
| style="padding: 5px 5px; background: #F5F5F5;" | Immunoglobulins | | style="padding: 5px 5px; background: #F5F5F5;" |Immunoglobulins | ||
| style="padding: 5px 5px; background: #F5F5F5;" | | | style="padding: 5px 5px; background: #F5F5F5;" | | ||
* | * | ||
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*Miconazole | *Miconazole | ||
|- | |- | ||
| rowspan="2" style="padding: 5px 5px; background: #DCDCDC;" | Chronic infections | | rowspan="2" style="padding: 5px 5px; background: #DCDCDC;" |Chronic infections | ||
| style="padding: 5px 5px; background: #F5F5F5;" | Antibiotics and surgery | | style="padding: 5px 5px; background: #F5F5F5;" |Antibiotics and surgery | ||
| style="padding: 5px 5px; background: #F5F5F5;" | | | style="padding: 5px 5px; background: #F5F5F5;" | | ||
* | * | ||
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* | * | ||
|- | |- | ||
| rowspan="2" style="padding: 5px 5px; background: #DCDCDC;" | Immunodeficiency | | rowspan="2" style="padding: 5px 5px; background: #DCDCDC;" |Immunodeficiency | ||
| style="padding: 5px 5px; background: #F5F5F5;" | Immunoglobulins | | style="padding: 5px 5px; background: #F5F5F5;" |Immunoglobulins | ||
| style="padding: 5px 5px; background: #F5F5F5;" | | | style="padding: 5px 5px; background: #F5F5F5;" | | ||
* | * | ||
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*Miconazole | *Miconazole | ||
|- | |- | ||
| rowspan="2" style="padding: 5px 5px; background: #DCDCDC;" | Neoplasia | | rowspan="2" style="padding: 5px 5px; background: #DCDCDC;" |Neoplasia | ||
| style="padding: 5px 5px; background: #F5F5F5;" | Chemotherapy and surgery | | style="padding: 5px 5px; background: #F5F5F5;" |Chemotherapy and surgery | ||
| style="padding: 5px 5px; background: #F5F5F5;" | | | style="padding: 5px 5px; background: #F5F5F5;" | | ||
Varies according to type of cancer | Varies according to type of cancer | ||
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Biologic agents (in Castleman disease) | Biologic agents (in Castleman disease) | ||
| style="padding: 5px 5px; background: #F5F5F5;" | | | style="padding: 5px 5px; background: #F5F5F5;" | | ||
* Tocilizumab | *Tocilizumab | ||
|- | |- | ||
|} | |} | ||
* Long-term inflammatory control can be accompanied by gradual regression of amyloid deposits and improvement in renal function. | *Long-term inflammatory control can be accompanied by gradual regression of amyloid deposits and improvement in renal function. | ||
*Currently a second clinical trial is in progress in order to evaluate a targeted inhibitor molecule, Kiacta, in the management of secondary amyloidosis. | *Currently a second clinical trial is in progress in order to evaluate a targeted inhibitor molecule, Kiacta, in the management of secondary amyloidosis. | ||
*Novel therapies aimed at promoting clearance of existing amyloid deposits soon may be an effective treatment approach. | *Novel therapies aimed at promoting clearance of existing amyloid deposits soon may be an effective treatment approach. | ||
=== | ===Senile Systemic or Wild-type Amyloidosis (ATTRwt)=== | ||
*In patients with transthyretin amyloid cardiomyopathy, tafamidis was associated with reductions in all-cause mortality and cardiovascular-related hospitalizations and reduced the decline in functional capacity and quality of life as compared with placebo.<ref name="pmid30145929">{{cite journal| author=Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M et al.| title=Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy. | journal=N Engl J Med | year= 2018 | volume= 379 | issue= 11 | pages= 1007-1016 | pmid=30145929 | doi=10.1056/NEJMoa1805689 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=30145929 }}</ref> | |||
=== Familial Amyloidosis === | |||
== References == | ==References== | ||
{{reflist|2}} | {{reflist|2}} | ||
Revision as of 21:29, 29 October 2019
Amyloidosis Microchapters |
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Shyam Patel [2]; Associate Editor(s)-in-Chief:
Overview
There are few available treatments for primary amyloidosis. Since the disease is typically discovered at an advanced stage, the initial treatment is aimed at preventing further organ damage and correcting the effects of organ failure.
Medical Therapy
Primary Amyloidosis (AL):
Some patients with primary amyloidosis respond to chemotherapy focused on the abnormal plasma cells. A stem cell transplant may be done, as in multiple myeloma.
- The initial step in the treatment of this disorder is to correct the organ failure, since the disease is discovered at an advanced stage when multiple organ systems may be affected.
- Nephrotic syndrome is treated using supportive therapy and diuretics.
- Renal failure is treated with dialysis.
- Heart failure is treated using diuretics.
- Gastrointestinal and nerve involvement are treated symptomatically.
The most commonly used regimen for AL amyloidosis is CyBorD, which consists of cyclophosphamide, bortezomib, and dexamethasone.[1]
Treatment options with limited success include melphalan, prednisone, and colchicine.
Secondary Amyloidosis (AA):
- Medical or surgical treatment of the underlying chronic infection or inflammatory disease is recommended among all patients who develop AA amyloidosis.
- Aggressively treating the disease that is causing the excess amyloid protein can improve symptoms and slow down or halt the progression of the disease.
- Complications such as heart failure, renal failure, and other problems can sometimes be treated, when needed.
- Although the choice of therapy depends on the underlying cause of chronic inflammation, the aim is always to suppress production of SAA to within the normal range.
- Examples of treatments for the commonest disorders underlying AA amyloidosis:[4]
Underlying Condition | Treatment Options | Examples |
---|---|---|
Inflammatory arthritis | Conventional disease-modifying agents |
|
Other immunosuppressant agents |
| |
Biologic agents |
| |
Periodic fevers | On-demand agents |
|
Colchicine (for familial mediterranean fever) |
Colchicine | |
Biologic agents |
| |
Inflammatory bowel disease | Conventional disease-modifying agents |
|
Biologic agents |
| |
Antibiotics |
| |
Biologic agents |
| |
Surgery |
ileo-cecal resection and primary reconstruction | |
Immunodeficiency | Immunoglobulins |
|
Antibiotics |
| |
Chronic infections | Antibiotics and surgery |
|
Physiotherapy (in case of bronchiectasis) |
| |
Immunodeficiency | Immunoglobulins |
|
Antibiotics |
| |
Neoplasia | Chemotherapy and surgery |
Varies according to type of cancer |
Biologic agents (in Castleman disease) |
|
- Long-term inflammatory control can be accompanied by gradual regression of amyloid deposits and improvement in renal function.
- Currently a second clinical trial is in progress in order to evaluate a targeted inhibitor molecule, Kiacta, in the management of secondary amyloidosis.
- Novel therapies aimed at promoting clearance of existing amyloid deposits soon may be an effective treatment approach.
Senile Systemic or Wild-type Amyloidosis (ATTRwt)
- In patients with transthyretin amyloid cardiomyopathy, tafamidis was associated with reductions in all-cause mortality and cardiovascular-related hospitalizations and reduced the decline in functional capacity and quality of life as compared with placebo.[5]
Familial Amyloidosis
References
- ↑ Milani P, Merlini G, Palladini G (2018). "Novel Therapies in Light Chain Amyloidosis". Kidney Int Rep. 3 (3): 530–541. doi:10.1016/j.ekir.2017.11.017. PMC 5976806. PMID 29854961.
- ↑ Adams D, Suhr OB, Dyck PJ, Litchy WJ, Leahy RG, Chen J; et al. (2017). "Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy". BMC Neurol. 17 (1): 181. doi:10.1186/s12883-017-0948-5. PMC 5594468. PMID 28893208.
- ↑ van de Donk NW, Janmaat ML, Mutis T, Lammerts van Bueren JJ, Ahmadi T, Sasser AK; et al. (2016). "Monoclonal antibodies targeting CD38 in hematological malignancies and beyond". Immunol Rev. 270 (1): 95–112. doi:10.1111/imr.12389. PMC 4755228. PMID 26864107.
- ↑ Papa R, Lachmann HJ (2018). "Secondary, AA, Amyloidosis". Rheum Dis Clin North Am. 44 (4): 585–603. doi:10.1016/j.rdc.2018.06.004. PMID 30274625.
- ↑ Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M; et al. (2018). "Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy". N Engl J Med. 379 (11): 1007–1016. doi:10.1056/NEJMoa1805689. PMID 30145929.