Materiały źródłowe

• #1 Treatment for Amyloidosis | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/amyloidosis/treatment.html

  Treatment for amyloidosis is complex and must be tailored to each individual, so its important to be treated at a specialized center with expertise in this disease. […] Fred Hutch is involved in research to better understand amyloidosis and to identify more effective treatments, such as amyloid-removing agents, like CAEL-101. In addition, studies are underway at our center to assess the benefit of adding the monoclonal antibody daratumumab (Darzalex) to standard treatment with the hope that this will speed and deepen the response to chemotherapy. […] We view treatment as a collaborative effort. Your Fred Hutch physician will explain all your options and recommend an individualized treatment plan based on your type of amyloidosis as well as your specific medical needs and personal preferences.

• #2 Amyloidosis – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/amyloidosis/diagnosis-treatment/drc-20353183

  There’s no cure for amyloidosis. But treatment can help manage signs and symptoms and limit further production of amyloid protein. If the amyloidosis has been triggered by another condition, such as rheumatoid arthritis or tuberculosis, treating the underlying condition can be helpful. […] Some cancer drugs are used in AL amyloidosis to stop the growth of abnormal cells that produce the protein that forms amyloid. […] If your heart is affected, you may need to take blood thinners to reduce the risk of clots. You may also need medications to control your heart rate. Drugs that increase urination can reduce the strain on your heart and kidneys. […] For certain types of amyloidosis, drugs such as patisiran (Onpattro) and inotersen (Tegsedi) can interfere with the commands sent by faulty genes that create amyloid. Other drugs, such as tafamidis (Vyndamax, Vyndaqel) and diflunisal, can stabilize bits of protein in the bloodstream and prevent them from getting transformed into amyloid deposits.

• #3 Amyloidosis: Symptoms, Types & Treatments

  https://my.clevelandclinic.org/health/diseases/23398-amyloidosis

  Amyloidosis is a rare disorder that happens when proteins in your body turn into misshapen clumps. The abnormal proteins gather on your organs. Some amyloidosis types affect specific organs like your heart, kidneys, liver and lungs. Other types spread throughout your body. Treatments include organ transplants, chemotherapy or targeted therapies. […] Healthcare providers can stop the production of abnormal proteins that cause amyloidosis. Your bodys own immune system can help remove these proteins in a few types of amyloidosis. This can improve your symptoms and the health of the involved organs. […] Healthcare providers manage amyloidosis by treating the condition thats causing it. Treatments can slow the progression of amyloidosis or prevent new fibrils from forming. They can also ease your symptoms with medications. In the case of Al amyloidosis, once treatment stops new amyloid deposits from forming, your immune system can remove those that are already there. Research is ongoing to find ways of enhancing this process of removing amyloids from organs.

• #4 Treatment – Multidisciplinary Amyloidosis Program | NewYork-Presbyterian

  https://www.nyp.org/multidisciplinary-amyloidosis-program/treatment

  Treatment for amyloidosis is aimed at managing symptoms, limiting the production of further amyloid proteins, and/or supporting affected organ dysfunction. […] Therefore, doctors at NYP place a high priority on intervening early before multiple organs are seriously affected. […] A tailored approach is taken with each patient and treatment is geared toward how many organs are affected, the physiologic age of the patient, and his/her activity level. […] If a patient is diagnosed with primary (AL) amyloidosis, usually doctors prescribe a chemotherapy-based regimen (similar to what is used to treat multiple myeloma) to destroy the abnormal plasma cells that produce precursors of the amyloid protein. […] A new class of drugs called proteasome inhibitors are usually used in different combinations with other drugs to treat AL amyloidosis.

• #5 Treatment of amyloidosis: present and future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10120969/

  Cardiac amyloidosis (CA) is an infiltrative heart disease resulting from the deposition of amyloid fibrils in the interstitial spaces of the myocardium. The treatment of both forms of CA is based on: (i) prevention and slowing of the generation and deposition of amyloid fibrils and (ii) supportive care of complications. The main success of recent years has been the development of effective therapies that have been possible thanks to the understanding of the pathophysiology of amyloidosis. For the AL form, new therapeutic combinations between a proteasome inhibitor and a monoclonal antibody have been developed. For ATTR forms, the main strategies are transthyretin (TTR) production silencers and TTR tetramer stabilizers. […] The treatment of AL, ATTR-wt, and ATTRv amyloidosis are very different. Therefore, it is crucial to identify the type of amyloidosis and to characterize the fibrils before starting treatment and to assure the patient of the correct care.

• #6 AL Amyloidosis (Primary Amyloidosis): Symptoms, Treatment & What it Is

  https://my.clevelandclinic.org/health/diseases/15718-amyloidosis-al-amyloid-light-chain

  AL amyloidosis (amyloid light chain or primary amyloidosis) is one type of the rare disorder amyloidosis. Healthcare providers may treat amyloidosis with chemotherapy and/or bone marrow or stem cell transplantation. […] Healthcare providers may treat AL amyloidosis with medication and other treatments to ease your symptoms and address any organ damage. They also use treatments to slow down or stop the process causing your body to produce too many amyloids. […] Providers may use chemotherapy, immunotherapy or steroids to treat AL amyloidosis. Most people take one or two chemotherapy drugs plus steroid medication. The drugs work together to destroy the plasma cells that make light chain proteins. […] It’s important to remember medication can slow or stop AL amyloidosis from getting worse, but treatment doesn’t remove the fibrils already in your organs.

• #7 Primary Systemic Amyloidosis Treatment & Management: Approach Considerations, Medical Care

  https://emedicine.medscape.com/article/1093258-treatment

  Therapy for systemic light-chain (AL) amyloidosis is directed to recovering the function of the affected organs by targeting the abnormal plasma cell clone and slowing deposition of amyloid fibrils. Patients with systemic AL amyloidosis should be considered for early treatment if they have symptomatic visceral organ involvement, significant soft tissue involvement, coagulopathy, or neuropathy. […] The current standard treatment regimen for AL amyloidosis is daratumumab plus hyaluronidase, in combination with, cyclophosphamide, bortezomib, and dexamethasone is the current standard regimen for first-line treatment. Autologous stem cell transplantation (ASCT) is performed in eligible patients, especially those with an incomplete response to drug therapy. […] The goal of treatment is to achieve a complete hematologic response, with difference between involved and uninvolved free light chains (FLC) 10mg/L or involved FLC 20mg/L. Patients who achieve less than a very good partial response by cycle 3, or less than a partial response by cycle 2, should be considered for treatment modification.

• #8 Victory as first treatment for AL amyloidosis approved after NICE U-turn – Myeloma UK

  https://www.myeloma.org.uk/news/victory-as-first-treatment-for-al-amyloidosis-approved-after-nice-u-turn/

  Patients with AL amyloidosis have claimed a hard-fought victory after the very first treatment for the incurable disease was approved on the NHS, following a U-turn by NICE. […] Up to 600 people each year will now benefit from the life-extending treatment, known as DaraCyBorD. […] The first treatment of its kind, DaraCyBorD has been shown to be effective in clinical trials with patients five times more likely to see their disease stopped in its tracks. […] 59% of patients who received DaraCyBorD also had no sign of AL amyloidosis 20 months after treatment and longer remission times. […] The treatment combines daratumumab (Darzalex®) with cyclophosphamide, bortezomib (Velcade®) and dexamethasone. […] This new drug combination is a game-changer and we know it can have a significant impact on people’s quality of life and remission times.

• #9 Primary Systemic Amyloidosis Treatment & Management: Approach Considerations, Medical Care

  https://emedicine.medscape.com/article/1093258-treatment

  All patients with AL amyloidosis should be considered for enrollment in a clinical trial. Several novel treatment approaches (eg, venetoclax, ixazomib) are being studied for this disorder. […] The treatment of primary systemic amyloidosis is directed toward the affected organ and the specific type of the disease. Chemotherapy regimens similar to those used for myeloma are employed. In addition, daratumumab, the first and only specific treatment for newly diagnosed AL amyloidosis, was granted accelerated approval in 2021 by the US Food and Drug Administration (FDA). […] This is currently the preferred regimen for first-line treatment. […] For second-line treatment, treatment selection is guided by the depth and duration of initial response, use of a class of agents not previously exposed, and limitations imposed by patients fitness/frailty and end-organ damage.

• #10 Victory as first treatment for AL amyloidosis approved after NICE U-turn – Myeloma UK

  https://www.myeloma.org.uk/news/victory-as-first-treatment-for-al-amyloidosis-approved-after-nice-u-turn/

  Patients with AL amyloidosis have claimed a hard-fought victory after the very first treatment for the incurable disease was approved on the NHS, following a U-turn by NICE. […] Up to 600 people each year will now benefit from the life-extending treatment, known as DaraCyBorD. […] The first treatment of its kind, DaraCyBorD has been shown to be effective in clinical trials with patients five times more likely to see their disease stopped in its tracks. […] 59% of patients who received DaraCyBorD also had no sign of AL amyloidosis 20 months after treatment and longer remission times. […] The treatment combines daratumumab (Darzalex®) with cyclophosphamide, bortezomib (Velcade®) and dexamethasone. […] This new drug combination is a game-changer and we know it can have a significant impact on people’s quality of life and remission times.

• #11 Future Developments in the Treatment of AL Amyloidosis

  https://www.mdpi.com/2673-6357/3/1/12

  The combination of Daratumumab, Cyclophosphamide, Bortezomib, Dexamethasone (Dara-VCd) is currently the novel and preferred standard of care for newly diagnosed patients with AL amyloidosis and the only Food and Drug Administration (FDA) and EMA-approved treatment for this disease. […] After a median follow-up of 20.3 months, the hematologic CR rate was 59% in the daratumumab group vs. 19% in the control group, and at least VGPR was seen in 79% vs. 50%, respectively. […] However, if daratumumab is not available or accessible, alternative options include VCd, Bortezomib-Melphalan-dexamethasone (BMdex), or bortezomib plus dexamethasone. […] A significant proportion of patients will not achieve a sufficiently deep hematologic response with first-line treatment, or the disease will relapse. […] The patient should be carefully evaluated and previous exposure and refractoriness to daratumumab and bortezomib are critical for the choice of salvage therapy; prior high dose therapy with ASCT or current eligibility for ASCT are also important considerations.

• #12 Future Developments in the Treatment of AL Amyloidosis

  https://www.mdpi.com/2673-6357/3/1/12

  The combination of Daratumumab, Cyclophosphamide, Bortezomib, Dexamethasone (Dara-VCd) is currently the novel and preferred standard of care for newly diagnosed patients with AL amyloidosis and the only Food and Drug Administration (FDA) and EMA-approved treatment for this disease. […] After a median follow-up of 20.3 months, the hematologic CR rate was 59% in the daratumumab group vs. 19% in the control group, and at least VGPR was seen in 79% vs. 50%, respectively. […] However, if daratumumab is not available or accessible, alternative options include VCd, Bortezomib-Melphalan-dexamethasone (BMdex), or bortezomib plus dexamethasone. […] A significant proportion of patients will not achieve a sufficiently deep hematologic response with first-line treatment, or the disease will relapse. […] The patient should be carefully evaluated and previous exposure and refractoriness to daratumumab and bortezomib are critical for the choice of salvage therapy; prior high dose therapy with ASCT or current eligibility for ASCT are also important considerations.

• #13 Future Developments in the Treatment of AL Amyloidosis

  https://www.mdpi.com/2673-6357/3/1/12

  The combination of Daratumumab, Cyclophosphamide, Bortezomib, Dexamethasone (Dara-VCd) is currently the novel and preferred standard of care for newly diagnosed patients with AL amyloidosis and the only Food and Drug Administration (FDA) and EMA-approved treatment for this disease. […] After a median follow-up of 20.3 months, the hematologic CR rate was 59% in the daratumumab group vs. 19% in the control group, and at least VGPR was seen in 79% vs. 50%, respectively. […] However, if daratumumab is not available or accessible, alternative options include VCd, Bortezomib-Melphalan-dexamethasone (BMdex), or bortezomib plus dexamethasone. […] A significant proportion of patients will not achieve a sufficiently deep hematologic response with first-line treatment, or the disease will relapse. […] The patient should be carefully evaluated and previous exposure and refractoriness to daratumumab and bortezomib are critical for the choice of salvage therapy; prior high dose therapy with ASCT or current eligibility for ASCT are also important considerations.

• #14 Future Developments in the Treatment of AL Amyloidosis

  https://www.mdpi.com/2673-6357/3/1/12

  The combination of Daratumumab, Cyclophosphamide, Bortezomib, Dexamethasone (Dara-VCd) is currently the novel and preferred standard of care for newly diagnosed patients with AL amyloidosis and the only Food and Drug Administration (FDA) and EMA-approved treatment for this disease. […] After a median follow-up of 20.3 months, the hematologic CR rate was 59% in the daratumumab group vs. 19% in the control group, and at least VGPR was seen in 79% vs. 50%, respectively. […] However, if daratumumab is not available or accessible, alternative options include VCd, Bortezomib-Melphalan-dexamethasone (BMdex), or bortezomib plus dexamethasone. […] A significant proportion of patients will not achieve a sufficiently deep hematologic response with first-line treatment, or the disease will relapse. […] The patient should be carefully evaluated and previous exposure and refractoriness to daratumumab and bortezomib are critical for the choice of salvage therapy; prior high dose therapy with ASCT or current eligibility for ASCT are also important considerations.

• #15 AL Amyloidosis | Cedars-Sinai

  https://www.cedars-sinai.org/health-library/diseases-and-conditions/a/al-amyloidosis.html

  AL Amyloidosis is caused by the accumulation of an immunoglobulin light chain protein. […] One must eliminate the proliferating cells to reduce the production of the harmful amyloid protein. […] There is no cure for patients with AL amyloidosis but more frequently patients can go into remission with drug therapy. […] Recently, drugs useful to treat multiple myeloma and lymphoma have improved. […] Melphalan A chemotherapy drug used in high doses as part of a stem cell transplant. […] Bortezomib A new agent that blocks the proteasome, an enzyme in all cells, found to be very helpful in patients with multiple myeloma and most recently in patients with AL Amyloidosis. […] Thalidomide A sleeping pill later found to cause birth defects was found to be extremely effective in treating patients with multiple myeloma.

• #16 AL Amyloidosis | Cedars-Sinai

  https://www.cedars-sinai.org/health-library/diseases-and-conditions/a/al-amyloidosis.html

  Lenalidomide A newer version of thalidomide with fewer side effects and greater efficacy in general. […] Rituximab Useful for patients with lymphoma, and Waldenstrom’s Macroglobulinemia and AL Amyloidosis. […] Cyclophosphamide Another chemotherapy drug which can target the cells causing AL Amyloidosis. […] Peripheral blood stem cell transplantation- Is a procedure that has the promise of leading to a long-term remission in patients with AL Amyloidosis often without the need for further medication. […] New versions of bortezomib and lenalidomide have been recently released or are in development.

• #17 Treatment for Amyloidosis | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/amyloidosis/treatment.html

  Throughout treatment, your team provides follow-up care on a schedule tailored to you. […] Treatment looks different for different people depending on your diagnosis. We tailor your treatment plan to you. […] Chemotherapy is a main treatment for primary amyloidosis (AL). Physicians may recommend a single medicine or a combination of medicines to destroy abnormal cells in your blood. […] Targeted therapies are newer treatments that work more selectively than standard chemotherapy. […] If you have the most common form of amyloidosis (light chain, or AL type), your physician may recommend having a peripheral blood stem cell (PBSC) transplant using your own cells (autologous transplant). […] Care for symptoms caused by organ damage and for side effects you might experience from treatment are important for every person with amyloidosis. […] Because of organ damage, some people with amyloidosis become candidates for an organ transplant, such as a liver, kidney or heart transplant.

• #18 Cardiac Amyloidosis Treatment

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8932359/

  The goal of treatment is to administer the strongest chemotherapy regimen that the patient can safely tolerate with the intent of improving organ function and survival. […] High-dose melphalan with ASCT provides the greatest opportunity for long-term remission. However, to qualify for ASCT as initial therapy, patients must be low risk, defined as age 70 years old with good functional status (Eastern Cooperative Oncology Group performance status [ECOG PS] 2) and without significant organ dysfunction, including New York Heart Association (NYHA) class III and biomarker levels below validated thresholds. […] The transthyretin (TTR) tetramer stabilizer, tafamidis, is the only FDA-approved treatment for transthyretin cardiac amyloidosis (ATTR-CA). However, there are promising novel therapies on the horizon that target pathogenic TTR fibril formation including TTR gene silencers, TTR tetramer stabilizers, oligomer aggregation inhibitors, anti-TTR fiber antibodies, and TTR amyloid degraders (ongoing clinical trials).

• #19 Amyloidosis | Conditions | UCSF Health

  https://www.ucsfhealth.org/conditions/amyloidosis

  For primary amyloid, treatments include the same agents used to treat multiple myeloma, such as chemotherapy, corticosteroid medicines (lenalidomide or thalidomide) and/or bortezomib (Velcade). These treatments slow organ deterioration and some have been shown to prolong life, but none provide a cure. […] Because primary amyloid is such a difficult disease to treat and survival is limited, researchers have begun to investigate the use of high-dose chemotherapy with autologous stem cell transplantation as a means of prolonging survival. The initial results with autologous stem cell transplantation are encouraging. […] High-dose melphalan chemotherapy is administered over one day. Then the patient’s own stem cells (bone marrow) are re-administered two to three days later. […] The hope is that this therapy will delay progression of the disease, and in some cases, improve symptoms through removal of the abnormal proteins from the organs. However, this therapy is not a cure, and amyloidosis will return in everyone.

• #20 Amyloidosis | Conditions | UCSF Health

  https://www.ucsfhealth.org/conditions/amyloidosis

  For primary amyloid, treatments include the same agents used to treat multiple myeloma, such as chemotherapy, corticosteroid medicines (lenalidomide or thalidomide) and/or bortezomib (Velcade). These treatments slow organ deterioration and some have been shown to prolong life, but none provide a cure. […] Because primary amyloid is such a difficult disease to treat and survival is limited, researchers have begun to investigate the use of high-dose chemotherapy with autologous stem cell transplantation as a means of prolonging survival. The initial results with autologous stem cell transplantation are encouraging. […] High-dose melphalan chemotherapy is administered over one day. Then the patient’s own stem cells (bone marrow) are re-administered two to three days later. […] The hope is that this therapy will delay progression of the disease, and in some cases, improve symptoms through removal of the abnormal proteins from the organs. However, this therapy is not a cure, and amyloidosis will return in everyone.

• #21 Current Updates on the Management of AL Amyloidosis | Elsayed | Journal of Hematology

  https://thejh.org/index.php/jh/article/view/866/579

  Current treatment strategies and updates to clinical guidelines and therapeutics for AL amyloidosis. […] Currently, there are no clear guidelines on systemic AL amyloidosis treatment; however, since both AL amyloidosis and multiple myeloma (MM) are monoclonal plasma cell dyscrasias, AL amyloidosis treatment strategies and medications are derived from the anti-plasma cell therapy that is used for MM. […] Therapy is aimed at achieving deep and rapid hematological response, which reverses amyloid-mediated organ dysfunction, and improves OS. […] High-dose melphalan followed by ASCT is considered the most commonly used first-line therapy for patients who fit the eligibility criteria for ASCT. […] However, bortezomib-based induction with two cycles prior to ASCT was found to improve hematologic remission and OS in a randomized controlled trial and several retrospective studies.

• #22 Treatment of amyloidosis: present and future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10120969/

  Treatment of cardiac AL amyloidosis should be guided by a multidisciplinary team involving oncohaematology and cardiology specialists and if possible should be followed by specialized amyloidosis centres. […] Current guidelines recommend the combination of cyclophosphamide, bortezomib, dexamethasone (CyBorD), and daratumumab as the first-line therapy in patients newly diagnosed with AL. […] The therapy is based on autologous stem cell transplantation and chemo/immunotherapy against plasma cell dyscrasia. […] Three monoclonal antibodies birtamimab, CAEL-101, and AT-03 are currently being studied which aim at the removal of amyloid fibrils from the affected organs, the results of these studies will be able to offer direct proof of the hypothesis that by removing the fibril deposits of light chains from the organs there is an improvement in their function.

• #23 Amyloidosis Treatment Options – NYC | Herbert Irving Comprehensive Cancer Center (HICCC) – New York

  https://www.cancer.columbia.edu/cancer-types-care/types/amyloidosis/treatments

  The drug has shown exceptional activity in AL amyloidosis. […] Currently most therapies in amyloidosis work by destroying the plasma cell and stopping the accumulation of the amyloid but unfortunately not reversing the buildup of an existing amyloid. […] Monoclonal antibodies that directly target the amyloid deposits such as this treatment are new and exciting treatments that have the potential to reverse the existing buildup of an amyloid in patients with amyloidosis. […] A new class of drugs called proteasome inhibitors may also be used in different combinations with chemotherapy drugs. […] Around 20% of patients with amyloidosis and an appropriate organ function are eligible for stem cell transplantation. […] If a patient with amyloidosis is in good condition (fewer than three organs affected, with adequate cardiac function and laboratory results), a bone marrow transplant specialist can perform the transplant.

• #24 Systemic AL amyloidosis: current approach and future direction | Oncotarget

  https://www.oncotarget.com/article/28415/text/

  Current first line induction therapy is daratumumab combined with bortezomib, cyclophosphamide and dexamethasone (Dara-CyBorD). […] Novel and evolving therapy include BCL-2 inhibitor venetoclax in AL patients with t(11;14). […] Finally, the clinical trials of new treatments against deposited amyloid fibrils include monoclonal antibodies such as CAEL 101 or Birtamimab which if found effective and approved will revolutionize the treatment against AL amyloidosis.

• #25 Systemic AL amyloidosis: current approach and future direction | Oncotarget

  https://www.oncotarget.com/article/28415/text/

  Current first line induction therapy is daratumumab combined with bortezomib, cyclophosphamide and dexamethasone (Dara-CyBorD). […] Novel and evolving therapy include BCL-2 inhibitor venetoclax in AL patients with t(11;14). […] Finally, the clinical trials of new treatments against deposited amyloid fibrils include monoclonal antibodies such as CAEL 101 or Birtamimab which if found effective and approved will revolutionize the treatment against AL amyloidosis.

• #26 Treatment and prognosis of immunoglobulin light chain (AL) amyloidosis – UpToDate

  https://www.uptodate.com/contents/treatment-and-prognosis-of-immunoglobulin-light-chain-al-amyloidosis

  Treatment and prognosis of immunoglobulin light chain (AL) amyloidosis […] The treatment and prognosis of AL amyloidosis will be reviewed in detail here. […] Goals of therapy […] Indications for systemic therapy […] Supportive therapy […] Induction therapy […] Planning an approach […] Dara-CyBorD (preferred) […] Mayo stage I to IIIA disease […] Mayo stage IIIB disease […] Other bortezomib-based regimens […] Bortezomib-free regimens […] Autologous hematopoietic cell transplant […] HCT efficacy and toxicity […] Timing of HCT […] Melphalan dosing […] Monitoring response […] Response criteria […] Choice of therapy […] Daratumumab-based regimens […] Proteasome inhibitor-based regimens […] Immunomodulatory derivatives […] Lenalidomide-based regimens […] Pomalidomide plus dexamethasone […] Bendamustine-based regimens […] Venetoclax for those with t(11;14) […] BCMA-directed therapies […] Impact of organ involvement […] Coexisting myeloma […] IgM-related amyloidosis […] false

• #27 Treatment of amyloidosis: present and future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10120969/

  Current guidelines for the treatment of ATTR amyloidosis include: (i) specific therapies that modify the synthesis, production, and aggregation of amyloid fibrils; (ii) symptomatic therapy for the treatment of neurological and cardiovascular complications; (iii) support; and (iv) genetic counselling for familial forms. […] There are currently two types of therapeutic strategies that have proven effective for the treatment of ATTR-induced CA: (i) the silencing of TTR synthesis and expression and (ii) the stabilization of TTR proteins with the aim of limiting their degradation. […] The liver produces 95% of the TTR measured in serum. Therefore, liver transplantation has historically (since 1990) been proposed as the first-line therapy for the elimination of the main source of amyloidogenic TTR in patients in the familial form (ATTRv), while it is not indicated in the ATTR-wt form.

• #28 Cardiac Amyloidosis Treatment

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8932359/

  The goal of treatment is to administer the strongest chemotherapy regimen that the patient can safely tolerate with the intent of improving organ function and survival. […] High-dose melphalan with ASCT provides the greatest opportunity for long-term remission. However, to qualify for ASCT as initial therapy, patients must be low risk, defined as age 70 years old with good functional status (Eastern Cooperative Oncology Group performance status [ECOG PS] 2) and without significant organ dysfunction, including New York Heart Association (NYHA) class III and biomarker levels below validated thresholds. […] The transthyretin (TTR) tetramer stabilizer, tafamidis, is the only FDA-approved treatment for transthyretin cardiac amyloidosis (ATTR-CA). However, there are promising novel therapies on the horizon that target pathogenic TTR fibril formation including TTR gene silencers, TTR tetramer stabilizers, oligomer aggregation inhibitors, anti-TTR fiber antibodies, and TTR amyloid degraders (ongoing clinical trials).

• #29 Amyloidosis – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/amyloidosis/diagnosis-treatment/drc-20353183

  There’s no cure for amyloidosis. But treatment can help manage signs and symptoms and limit further production of amyloid protein. If the amyloidosis has been triggered by another condition, such as rheumatoid arthritis or tuberculosis, treating the underlying condition can be helpful. […] Some cancer drugs are used in AL amyloidosis to stop the growth of abnormal cells that produce the protein that forms amyloid. […] If your heart is affected, you may need to take blood thinners to reduce the risk of clots. You may also need medications to control your heart rate. Drugs that increase urination can reduce the strain on your heart and kidneys. […] For certain types of amyloidosis, drugs such as patisiran (Onpattro) and inotersen (Tegsedi) can interfere with the commands sent by faulty genes that create amyloid. Other drugs, such as tafamidis (Vyndamax, Vyndaqel) and diflunisal, can stabilize bits of protein in the bloodstream and prevent them from getting transformed into amyloid deposits.

• #30 Treatment of amyloidosis: present and future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10120969/

  TTR expression can be pharmacologically reduced using agents that silence or block TTR protein synthesis. […] Treatment with inotersen significantly improves neurological symptoms, in rare cases causing severe thrombocytopenia and glomerulonephritis which are life-threatening adverse events. […] Patisiran uses lipid nanoparticles that facilitate its hepatic uptake. […] Treatment with patisiran is currently only approved to treat ATTR-PN. […] Another approach to the treatment of TTR amyloidosis is to stabilize the tetrameric TTR protein complex, thereby preventing its dissociation into amyloidogenic TTR monomers and oligomers. […] The drug was studied in the ATTR-ACT trial which randomized 441 subjects affected by cardiac TTR amyloidosis (wild-type and familial) to 80 or 20 mg of tafamidis or placebo for 30 months.

• #31 Transthyretin-Related Amyloidosis Treatment & Management: Approach Considerations, Medical Care, Surgical Care

  https://emedicine.medscape.com/article/335301-treatment

  Tafamidis and tafamidis meglumine were approved by the FDA in 2019 for the treatment of ATTR-CM. […] Tafamidis has been found to be an effective therapy, with an acceptable adverse effect profile, for patients with heart failure related to ATTR cardiomyopathy. […] Acoramidis received FDA approval in November 2024 for treatment of wild-type or hereditary (variant) ATTR-CM in adults. […] Vutrisiran gained FDA approval in 2022 for polyneuropathy of hATTR in adults. […] Tolcapone has Orphan Drug designation for treatment of ATTR. […] Diflunisal improved quality of life scores and reduced progression of neurologic impairment compared with placebo. […] Management of identified ATTR-CM should involve early consideration of tafamidis, tafamidis meglumine, or acoramidis, as earlier administration may slow the progressive disease process.

• #32 Major Treatments | Amyloidosis Center

  https://www.bu.edu/amyloid/what/treatments/

  Data from these trials were sufficient to permit FDA to approve both patisaran or OnpattroTM (Alnylam Pharmaceuticals) and inotersen or TegsediTM (Ionis/Akcea Therapeutics) as the first treatments for ATTR amyloidosis. […] While at our Center we utilize the oral drug diflunisal as a means to slow ATTR amyloidosis progression, this drug, while effective and inexpensive, is not tolerated by many patients. […] We await FDA action on the 3rd new drug reported in 2018, tafamidis (VyndaqelTM, Pfizer, Inc.) which will seek approval for ATTR amyloidosis with cardiomyopathy and provide needed treatment for these patients. […] We also use diflunisal, EGCG (green tea extract) and clinical trials for the treatment of this type of amyloidosis.

• #33 Treatment of amyloidosis: present and future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10120969/

  Acoramidis (AG10/ALXN2060, Eldos Therapeutics) is a promising new TTR stabilizer under investigation for the treatment of ATTR amyloidosis and appears to be highly effective for the variant form with polyneuropathy, slowing disease progression. […] The treatment of amyloidosis has undergone a major change in recent years. Even though we currently have only a few drugs to use for the treatment of CA, very promising new treatments will be available to the cardiologist in the near future.

• #34 Major Treatments | Amyloidosis Center

  https://www.bu.edu/amyloid/what/treatments/

  Data from these trials were sufficient to permit FDA to approve both patisaran or OnpattroTM (Alnylam Pharmaceuticals) and inotersen or TegsediTM (Ionis/Akcea Therapeutics) as the first treatments for ATTR amyloidosis. […] While at our Center we utilize the oral drug diflunisal as a means to slow ATTR amyloidosis progression, this drug, while effective and inexpensive, is not tolerated by many patients. […] We await FDA action on the 3rd new drug reported in 2018, tafamidis (VyndaqelTM, Pfizer, Inc.) which will seek approval for ATTR amyloidosis with cardiomyopathy and provide needed treatment for these patients. […] We also use diflunisal, EGCG (green tea extract) and clinical trials for the treatment of this type of amyloidosis.

• #35 Treatment – Amyloidosis UK

  https://amyloidosisuk.org/treatment/

  Two recently developed genetic therapies (commonly referred to as gene-silencing therapies in the news), called patisiran and inotersen, aim to silence the TTR gene in the liver cells this gene acts as a blueprint to make TTR protein. […] Patisiran belongs to a class of medicines called small interfering RNA, whereas inotersen belongs to the antisense oligonucleotide drug class. In clinical trials in patients with hATTR amyloidosis both these drugs improved neuropathy symptoms, quality of life, daily activities and disability. […] Both patisiran and inotersen have been approved by the European Medicines Agency and the U.S. Food and Drug Administration (FDA) for treating neuropathy caused by hATTR amyloidosis and are licensed for use in the UK and Ireland for this purpose. […] Tafamidis, is a TTR stabiliser. It attaches to TTR, which prevents the protein from breaking up, thereby stopping the formation of amyloid and slowing down the progression of the nerve disease.

• #36 Transthyretin-Related Amyloidosis Treatment & Management: Approach Considerations, Medical Care, Surgical Care

  https://emedicine.medscape.com/article/335301-treatment

  Patisiran utilizes RNA interference, a cellular process in which small interfering RNAs (siRNAs) control gene expression by mediating the cleavage of specific messenger RNAs (mRNAs). […] Approval was based on the APOLLO clinical trial, in which patients taking patisiran (n=148) showed significantly improved scores on the Neuropathy Impairment Score+7 (NIS+7) and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (QOL-DN) at 18 months, compared with those taking placebo (n=77) (P 0.001). […] Inotersen was approved by the FDA in 2018. Like patisiran, it is indicated for polyneuropathy of hATTR in adults; unlike patisiran, inotersen is given as a once-weekly, subcutaneous injection that the patient or caregiver can administer. […] Eplontersen was approved by the FDA in December 2023. Like inotersen, it is a antisense oligonucleotide that causes degradation of mutant and wild-type transthyretin (TTR) mRNA through binding to the TTR mRNA, which results in a reduction of serum TTR protein levels and TTR protein deposits in tissues.

• #37 Transthyretin-Related Amyloidosis Treatment & Management: Approach Considerations, Medical Care, Surgical Care

  https://emedicine.medscape.com/article/335301-treatment

  Patisiran utilizes RNA interference, a cellular process in which small interfering RNAs (siRNAs) control gene expression by mediating the cleavage of specific messenger RNAs (mRNAs). […] Approval was based on the APOLLO clinical trial, in which patients taking patisiran (n=148) showed significantly improved scores on the Neuropathy Impairment Score+7 (NIS+7) and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (QOL-DN) at 18 months, compared with those taking placebo (n=77) (P 0.001). […] Inotersen was approved by the FDA in 2018. Like patisiran, it is indicated for polyneuropathy of hATTR in adults; unlike patisiran, inotersen is given as a once-weekly, subcutaneous injection that the patient or caregiver can administer. […] Eplontersen was approved by the FDA in December 2023. Like inotersen, it is a antisense oligonucleotide that causes degradation of mutant and wild-type transthyretin (TTR) mRNA through binding to the TTR mRNA, which results in a reduction of serum TTR protein levels and TTR protein deposits in tissues.

• #38 Amyloidosis – Wikipedia

  https://en.wikipedia.org/wiki/Amyloidosis

  Patisiran functions similarly to inotersen. Moderate-certainty evidence suggests that patisiran mitigates worsening of peripheral neuropathy and disability from disease progression. Additionally, low-certainty evidence suggests that patisiran mitigates decreases in quality-of-life and slightly reduces the rate of adverse events versus placebo. There is no evidence of an effect on mortality rate. […] The roles of inotersen and patisiran in cardiac ATTR amyloidosis are still being investigated. […] Vutrisiran was approved by the U.S. Food and Drug Administration (FDA) in June 2022, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

• #39 Study Reveals Exciting New Treatment for Cardiac Amyloidosis with Cardiomyopathy

  https://www.medstarhealth.org/blog/cardiac-amyloidosis-treatment-research

  MedStar Health was the first and among the most prominent U.S. enrollers of participants in this important global Phase III clinical trial of a new drug to treat transthyretin amyloidosis with cardiomyopathy. […] The groundbreaking HELIOS B clinical trial has demonstrated the effectiveness of a new treatment for one type of cardiac amyloidosis, a fatal condition that disproportionately impacts Black patients. […] The HELIOS B study has revealed an effective new treatment for this once-fatal condition. […] When we analyzed the results, vutrisiran had clear benefits. Compared to the placebo group, participants assigned to this medication were: More likely to survive to the end of the study, More likely to have a better quality of life, Less likely to need hospitalization for reasons related to their heart.

• #40 Treatment of amyloidosis: present and future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10120969/

  Current guidelines for the treatment of ATTR amyloidosis include: (i) specific therapies that modify the synthesis, production, and aggregation of amyloid fibrils; (ii) symptomatic therapy for the treatment of neurological and cardiovascular complications; (iii) support; and (iv) genetic counselling for familial forms. […] There are currently two types of therapeutic strategies that have proven effective for the treatment of ATTR-induced CA: (i) the silencing of TTR synthesis and expression and (ii) the stabilization of TTR proteins with the aim of limiting their degradation. […] The liver produces 95% of the TTR measured in serum. Therefore, liver transplantation has historically (since 1990) been proposed as the first-line therapy for the elimination of the main source of amyloidogenic TTR in patients in the familial form (ATTRv), while it is not indicated in the ATTR-wt form.

• #41 Major Treatments | Amyloidosis Center

  https://www.bu.edu/amyloid/what/treatments/

  Immunomodulary drugs (lenalidomide and pomalidomide) and Proteosome inhibitors (bortezomib, carfilzomib and ixazomib) for the treatment of AL amyloidosis are being developed and have been studied in clinical trials here at the center. […] At present, clinical trials are on-going with monoclonal antibodies called daratumumab and isatuximab. Both have been used with success in multiple myeloma. […] In AA (historically known as secondary) amyloidosis, the treatment depends on the underlying disease. […] For hereditary ATTR amyloidosis, the treatment has been liver transplantation. […] A new liver will make only normal transthyretin. […] In 2018, 3 large clinical trials of 3 new pharmaceuticals were published in the prestigious New England Journal of Medicine each showing benefit in ATTR amyloidosis.

• #42 Transthyretin-Related Amyloidosis Treatment & Management: Approach Considerations, Medical Care, Surgical Care

  https://emedicine.medscape.com/article/335301-treatment

  Patisiran, vutrisiran, inotersen, and eplontersen are approved by the US Food and Drug Administration (FDA) for treatment of polyneuropathy caused by hereditary ATTR (hATTR) in adults, and tafamidis and tafamidis meglumine are approved for ATTR cardiomyopathy (ATTR-CM), as is vutrisiran under an expansion of its indication by the FDA. […] Since the 1990s, liver transplantation has been the gold standard for therapy. In the past several years, however, with the introduction of disease-modifying pharmacologic therapy, the number of liver transplantation cases for hATTR has declined significantly. […] The FDA has approved vutrisiran (Amvuttra), patisiran (Onpattro), inotersen (Tegsedi), and eplontersen (Wainua) for the treatment of polyneuropathy caused by hATTR in adults. Tafamidis (Vyndamax), tafamidis meglumine (Vyndaqel), acoramidis (Attruby), and, under an expanded indication, vutrisiran are FDA approved for ATTR-CM.

• #43 Cardiac Amyloidosis Treatment

  https://pmc.ncbi.nlm.nih.gov/articles/PMC8932359/

  For end-stage CA, advanced interventions including heart or heart-kidney transplantation may need to be considered. For patients with hereditary ATTR-CA with neuropathy, heart-liver transplant has been historically performed to prevent progression of debilitating neuropathy but is now superseded by the availability of gene-silencing therapies such as patisiran or inotersen.

• #44 Amyloidosis: Causes, Types, Symptoms, Diagnosis, Treatment, and Prognosis

  https://www.webmd.com/cancer/lymphoma/amyloidosis-symptoms-causes-treatments

  There is no cure for amyloidosis. Your doctor will prescribe treatments to slow the development of the amyloid protein and manage your symptoms. If amyloidosis is related to another condition, then treatment will include targeting that underlying condition. […] Specific treatment depends on what type of amyloidosis you have and how many organs are affected. […] High-dose chemotherapy with stem cell transplant can help remove the substance that leads to amyloid formation in some people with primary AL amyloidosis. Chemotherapy medicines alone may be used to treat other patients with primary AL amyloidosis. […] Secondary (AA) amyloidosis is treated by controlling the underlying disorder and with powerful anti-inflammatory medicines called steroids, which fight inflammation. […] A liver transplant may treat the disease if you have certain types of hereditary amyloidosis.

• #45 Amyloidosis treatment – Leukaemia Foundation

  https://www.leukaemia.org.au/blood-cancer/types-of-blood-cancer/amyloidosis/treatment/

  There is a range of treatments available for amyloidosis with much research being carried out around the world to find new treatments. […] Once a firm diagnosis has been made and the subtype of amyloidosis is identified, your doctors will discuss their recommended treatment regime with you. Regardless of the type of amyloidosis you have the goals of treatment are: to stop or slow the production of the amyloid protein, to preserve and support affected organs and tissues, to improve your quality of life. […] AA amyloidosis is managed by controlling the underlying inflammatory disease and therefore reducing the production of the amyloid protein SAA. If the SAA level can be reduced to almost normal and remains there for a long time there is a chance that the existing amyloid will eventually regress, improving the organ function. New treatments that interfere with the process of amyloid deposition are also being studied in AA.

• #46 Amyloidosis: Causes, Types, Symptoms, Diagnosis, Treatment, and Prognosis

  https://www.webmd.com/cancer/lymphoma/amyloidosis-symptoms-causes-treatments

  There is no cure for amyloidosis. Your doctor will prescribe treatments to slow the development of the amyloid protein and manage your symptoms. If amyloidosis is related to another condition, then treatment will include targeting that underlying condition. […] Specific treatment depends on what type of amyloidosis you have and how many organs are affected. […] High-dose chemotherapy with stem cell transplant can help remove the substance that leads to amyloid formation in some people with primary AL amyloidosis. Chemotherapy medicines alone may be used to treat other patients with primary AL amyloidosis. […] Secondary (AA) amyloidosis is treated by controlling the underlying disorder and with powerful anti-inflammatory medicines called steroids, which fight inflammation. […] A liver transplant may treat the disease if you have certain types of hereditary amyloidosis.

• #47 Amyloidosis Treatment Options | CVS Specialty

  https://www.cvsspecialty.com/resource-center/understanding-amyloidosis/amyloidosis-treatment-options.html

  Treatments for amyloidosis can help: […] Several medications can help treat amyloidosis. Your treatment will depend on the type of amyloidosis you have. For example, vutrisiran (Amvuttra), inotersen (Tegsedi) and patisiran (Onpattro) are approved to help treat the nerve pain and other symptoms that can happen with hereditary transthyretin (ATTR) amyloidosis. […] Two medications are approved help treat the wild-type ATTR amyloidosis with amyloids in the heart. They are tafamidis (Vyndamax) and tafamidis meglumine (Vyndaqel). […] Biologics are often prescribed to treat the underlying diseases that may cause amyloid A (AA) amyloidosis. One example is tocilizumab (Actemra). […] Certain cancer medications may be used for people with amyloid light-chain (AL) amyloidosis. In some cases, high dose chemotherapy followed by a stem cell transplant (SCT) may be suggested. […] Kidney, liver, and heart transplantation may be an option in certain cases. Depending on the type of amyloidosis you have, theres a chance that the disease will come back after transplant. But, treating early with newer therapies may help slow down the progression.

• #48 Amyloidosis – Wikipedia

  https://en.wikipedia.org/wiki/Amyloidosis

  Treatment depends on the type of amyloidosis that is present. Treatment with high dose melphalan, a chemotherapy agent, followed by stem cell transplantation has shown promise in early studies and is recommended for stage I and II AL amyloidosis. However, only 20-25% of people are eligible for stem cell transplant. Chemotherapy treatment including cyclophosphamide-bortezomib-dexamethasone-daratumumab (Dara-Cybord) is currently the recommended treatment option for people with AL Amyloidosis not eligible for transplant. […] In AA, symptoms may improve if the underlying condition is treated. In people who have inflammation caused by AA amyloidosis, tumour necrosis factor (TNF)-alpha inhibitors such as infliximab and etanercept are used for an average duration of 20 months. If TNF-alpha inhibitors are not effective, Interleukin-1 inhibitors (e.g., anakinra, canakinumab, rilonacept) and interleukin-6 inhibitors (e.g., tocilizumab) may be considered.

• #49 Treatment for Amyloidosis | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/amyloidosis/treatment.html

  Throughout treatment, your team provides follow-up care on a schedule tailored to you. […] Treatment looks different for different people depending on your diagnosis. We tailor your treatment plan to you. […] Chemotherapy is a main treatment for primary amyloidosis (AL). Physicians may recommend a single medicine or a combination of medicines to destroy abnormal cells in your blood. […] Targeted therapies are newer treatments that work more selectively than standard chemotherapy. […] If you have the most common form of amyloidosis (light chain, or AL type), your physician may recommend having a peripheral blood stem cell (PBSC) transplant using your own cells (autologous transplant). […] Care for symptoms caused by organ damage and for side effects you might experience from treatment are important for every person with amyloidosis. […] Because of organ damage, some people with amyloidosis become candidates for an organ transplant, such as a liver, kidney or heart transplant.

• #50 Cardiac amyloidosis: epidemiology, diagnosis and therapy

  https://www.escardio.org/Journals/E-Journal-of-Cardiology-Practice/Volume-19/cardiac-amyloidosis-epidemiology-diagnosis-and-therapy

  The cornerstones of amyloidosis management are heart failure therapy and treatment of the underlying disease. […] Loop diuretics constitutes a mainstay of the management of heart failure in amyloidosis. There are no data to suggest that the three life-saving therapies in heart failure with reduced ejection fraction (angiotensin-converting enzyme inhibitors/angiotensin receptor blockers/angiotensin receptor neprilysin inhibitors, beta-blockers and mineralocorticoid receptor antagonists) improve survival, symptoms and quality of life in patients with cardiac amyloidosis. In addition, these medications are often poorly tolerated, resulting in hypotension. A possible explanation for these findings is the complex pathophysiology of alterations in the heart muscle by amyloid infiltration; the combination of small to normal ventricular size, significant diastolic dysfunction and impaired atrial contraction results in decreased stroke volume and cardiac output while the intracardiac pressures are frequently elevated. Heart transplantation in carefully selected patients can be an effective therapeutic option.

• #51 Cardiac amyloidosis: epidemiology, diagnosis and therapy

  https://www.escardio.org/Journals/E-Journal-of-Cardiology-Practice/Volume-19/cardiac-amyloidosis-epidemiology-diagnosis-and-therapy

  The cornerstones of amyloidosis management are heart failure therapy and treatment of the underlying disease. […] Loop diuretics constitutes a mainstay of the management of heart failure in amyloidosis. There are no data to suggest that the three life-saving therapies in heart failure with reduced ejection fraction (angiotensin-converting enzyme inhibitors/angiotensin receptor blockers/angiotensin receptor neprilysin inhibitors, beta-blockers and mineralocorticoid receptor antagonists) improve survival, symptoms and quality of life in patients with cardiac amyloidosis. In addition, these medications are often poorly tolerated, resulting in hypotension. A possible explanation for these findings is the complex pathophysiology of alterations in the heart muscle by amyloid infiltration; the combination of small to normal ventricular size, significant diastolic dysfunction and impaired atrial contraction results in decreased stroke volume and cardiac output while the intracardiac pressures are frequently elevated. Heart transplantation in carefully selected patients can be an effective therapeutic option.

• #52 Treating Cardiac Amyloidosis – UChicago Medicine

  https://www.uchicagomedicine.org/conditions-services/heart-vascular/cardiac-amyloidosis/treatment-services

  Treatment options for cardiac amyloidosis depends on the type and severity. Our multidisciplinary team of experts will work with you on a strategy to manage your health and preserve your quality of life. […] Since AL amyloidosis starts in in the bone marrow, chemotherapy and stem cell transplantation are first-line treatments. […] Chemotherapy destroys the cells in the blood that produce the abnormal protein. For AL amyloidosis, we offer both oral and intravenous chemotherapy. […] Some patients with AL amyloidosis may be candidates for a stem cell transplant. Receiving donor marrow from someone who is not affected by amyloidosis can prevent production of amyloid proteins and offer long-term management for the condition. […] For patients diagnosed with TTR amyloidosis, newer drug therapies are being developed to help manage the condition and prevent the cell mutation that causes the disorder. However, transplantation currently offers the best chance to reverse a patient’s failing health so he can live a full, active life. […] Since amyloidosis is not reversible, the ideal treatment for patients who have advanced cardiac amyloidosis is heart transplantation.

• #53 Primary Systemic Amyloidosis Treatment & Management: Approach Considerations, Medical Care

  https://emedicine.medscape.com/article/1093258-treatment

  Congestive heart failure may respond to diuretics, but larger doses are often required as the disease progresses. […] Patients with nephrotic syndrome require supportive therapy and diuretics. Kidney failure can be successfully treated with dialysis. […] Gastrointestinal involvement and neuropathy are treated symptomatically.

• #54 Primary Systemic Amyloidosis Treatment & Management: Approach Considerations, Medical Care

  https://emedicine.medscape.com/article/1093258-treatment

  Congestive heart failure may respond to diuretics, but larger doses are often required as the disease progresses. […] Patients with nephrotic syndrome require supportive therapy and diuretics. Kidney failure can be successfully treated with dialysis. […] Gastrointestinal involvement and neuropathy are treated symptomatically.

• #55 Treatment side effects – Myeloma Patients Europe

  https://www.mpeurope.org/myeloma-and-al-amyloidosis/about-al-amyloidosis/treatment-side-effects/

  As it is mentioned in the treatment section, since AL amyloidosis is a disease which manifests quite differently from patient to patient, treatment usually is highly individualised as well. Your doctor might take different approaches involving different types of drugs or procedures. […] If constipation becomes a big problem, your doctor can prescribe several types of laxatives, which either reduce the removal of water from the faeces in the intestine, making them softer, or increase their bulk, or stimulate the movement of the bowel. […] If you are experiencing diarrhoea, you should report it to your doctor or healthcare team so the most appropriate treatment can be prescribed. […] Treatment depends on the symptoms experienced but avoiding meat (which can be hard to swallow) may help, and therapy can help reduce the chance of choking.

• #56 Treatment side effects – Myeloma Patients Europe

  https://www.mpeurope.org/myeloma-and-al-amyloidosis/about-al-amyloidosis/treatment-side-effects/

  As it is mentioned in the treatment section, since AL amyloidosis is a disease which manifests quite differently from patient to patient, treatment usually is highly individualised as well. Your doctor might take different approaches involving different types of drugs or procedures. […] If constipation becomes a big problem, your doctor can prescribe several types of laxatives, which either reduce the removal of water from the faeces in the intestine, making them softer, or increase their bulk, or stimulate the movement of the bowel. […] If you are experiencing diarrhoea, you should report it to your doctor or healthcare team so the most appropriate treatment can be prescribed. […] Treatment depends on the symptoms experienced but avoiding meat (which can be hard to swallow) may help, and therapy can help reduce the chance of choking.

• #57 9 Natural Therapies for Amyloidosis

  https://www.healthline.com/health/amyloidosis/natural-therapies-for-amyloidosis

  Amyloidosis occurs due to a buildup of amyloid proteins in the body. Along with treatment, complementary therapy can help with symptoms. Some examples include movements, such as yoga, sleep therapy, and a balanced diet. […] To prevent the progression of amyloidosis and the damage it can cause, your doctor can recommend a treatment plan. The type of treatment will depend on the kind of amyloidosis you have. It will likely include medication, such as chemotherapy or corticosteroids. It could also involve a procedure, such as a transplant or surgery. Still, treatment for amyloidosis doesn’t have to stop with conventional medication. […] Natural and complementary therapies can ease symptoms and improve well-being. Here are nine ways to get you started. […] If you’re having trouble with insomnia and daytime fatigue, talk with your doctor about sleep therapy.

• #58 9 Natural Therapies for Amyloidosis

  https://www.healthline.com/health/amyloidosis/natural-therapies-for-amyloidosis

  Reducing the amount of salt in your diet can help if fluid buildup in the body causes swelling. […] Your doctor may recommend following a low salt diet to help with swelling. This can also protect your heart and kidneys from further damage. […] If eating traditional meals isn’t appealing to you, you may want to talk with your doctor about meal replacement shakes or smoothies. This can help you get the nutrients your body needs to maintain energy and brain and muscle function. […] While there’s no clear amyloidosis diet, dietary modifications have helped some people feel better. […] Diuretics (also called water pills) are available by prescription and can help alleviate amyloidosis-related fluid retention. […] Research has shown acupuncture can be beneficial for amyloidosis symptoms. […] Even if you take medications or undergo other treatments for amyloidosis, complementary therapies can help. When used in conjunction with conventional medication, these therapies can make it easier to help manage your symptoms and medication side effects.

• #59 Treatment side effects – Myeloma Patients Europe

  https://www.mpeurope.org/myeloma-and-al-amyloidosis/about-al-amyloidosis/treatment-side-effects/

  To reduce the risk of fluid overload, your doctor can recommend that you limit your fluid intake (likely to be around or less than 1.5 litres) and keep your salt intake to a minimum. […] If, despite an appropriate diet and fluid intake limitations, you experience fluid overload, it can be treated with diuretics. […] Renal failure is usually treated by dialysis. […] If you are working, investigate whether you could work from home, or reduce your hours or responsibilities. […] Your doctor may be able to refer you to a fertility specialist to discuss what can be done. […] In case these are chemotherapy-related, they can be treated with anti-emetics. […] If you develop neutropaenia, you will be given detailed advice by your doctor or dietician about which foods to avoid and which are good alternatives.

• #60 9 Natural Therapies for Amyloidosis

  https://www.healthline.com/health/amyloidosis/natural-therapies-for-amyloidosis

  Amyloidosis occurs due to a buildup of amyloid proteins in the body. Along with treatment, complementary therapy can help with symptoms. Some examples include movements, such as yoga, sleep therapy, and a balanced diet. […] To prevent the progression of amyloidosis and the damage it can cause, your doctor can recommend a treatment plan. The type of treatment will depend on the kind of amyloidosis you have. It will likely include medication, such as chemotherapy or corticosteroids. It could also involve a procedure, such as a transplant or surgery. Still, treatment for amyloidosis doesn’t have to stop with conventional medication. […] Natural and complementary therapies can ease symptoms and improve well-being. Here are nine ways to get you started. […] If you’re having trouble with insomnia and daytime fatigue, talk with your doctor about sleep therapy.

• #61 9 Natural Therapies for Amyloidosis

  https://www.healthline.com/health/amyloidosis/natural-therapies-for-amyloidosis

  Reducing the amount of salt in your diet can help if fluid buildup in the body causes swelling. […] Your doctor may recommend following a low salt diet to help with swelling. This can also protect your heart and kidneys from further damage. […] If eating traditional meals isn’t appealing to you, you may want to talk with your doctor about meal replacement shakes or smoothies. This can help you get the nutrients your body needs to maintain energy and brain and muscle function. […] While there’s no clear amyloidosis diet, dietary modifications have helped some people feel better. […] Diuretics (also called water pills) are available by prescription and can help alleviate amyloidosis-related fluid retention. […] Research has shown acupuncture can be beneficial for amyloidosis symptoms. […] Even if you take medications or undergo other treatments for amyloidosis, complementary therapies can help. When used in conjunction with conventional medication, these therapies can make it easier to help manage your symptoms and medication side effects.

• #62 Treatment Centers & Clinical Trials – Amyloidosis Research Consortium

  https://arci.org/patients-and-caregivers/treatment-centers-clinical-trials/

  Finding the best amyloidosis care is not easy. ARC created My Amyloidosis Pathfinder (MAP) to help you discover, learn about, and contact treatment centers and clinical trials that are right for you. By answering our short questionnaire, MAP is able to match you to centers and trials specific to your condition. […] Finding the right treatment center is a crucial part of your care. MAP only shows you treatment centers with the capability to provide excellent treatment for your type of amyloidosis. We provide extensive information on each center, allowing you to make an informed decision. […] Clinical trials allow researchers to test the safety and effectiveness of new, promising drugs. Before any drug can be approved, it must be rigorously tested in clinical trials. Without the participation of patients, new treatments and cures will never happen. In addition, participating in a clinical trial may be a great way for patients to access new treatments before they become available. Especially in a rare disease such as amyloidosis, clinical trials can be a vital resource for your care.

• #63 Treatment Centers & Clinical Trials – Amyloidosis Research Consortium

  https://arci.org/patients-and-caregivers/treatment-centers-clinical-trials/

  Finding the best amyloidosis care is not easy. ARC created My Amyloidosis Pathfinder (MAP) to help you discover, learn about, and contact treatment centers and clinical trials that are right for you. By answering our short questionnaire, MAP is able to match you to centers and trials specific to your condition. […] Finding the right treatment center is a crucial part of your care. MAP only shows you treatment centers with the capability to provide excellent treatment for your type of amyloidosis. We provide extensive information on each center, allowing you to make an informed decision. […] Clinical trials allow researchers to test the safety and effectiveness of new, promising drugs. Before any drug can be approved, it must be rigorously tested in clinical trials. Without the participation of patients, new treatments and cures will never happen. In addition, participating in a clinical trial may be a great way for patients to access new treatments before they become available. Especially in a rare disease such as amyloidosis, clinical trials can be a vital resource for your care.

• #64 Northwestern Medicine Amyloidosis Therapies

  https://northwesternmedicine.nm.org/amyloidosis/

  Treatment options may include: Medications to address symptoms associated with nerve damage, heart failure and/or kidney failure. Well-tolerated chemotherapy drugs to suppress cells in the bone marrow. Organ transplantation that may include heart, liver, or heart and liver. […] Researchers at Northwestern Medicine conduct clinical trials that provide access to innovative therapies for the treatment of heart and vascular disease. Participating in a clinical trial is an opportunity to evaluate the effectiveness and safety of medications or study devices. […] ATTR CM: Enrolling patients with hereditary or wild-type transthyretin-mediated amyloid cardiomyopathy. Eligible participants will be randomized to receive an investigational medicine, AKCEA-TTR-LRx, or placebo. […] MAGNITUDE: This phase 3, randomized, placebo-controlled study aims to evaluate the efficacy and safety of NTLA-2001 in participants with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM). NTLA-2001, an investigational CRISPR/Cas9 based genome editing therapy, is intended to edit TTR in hepatocytes so the gene stops making the TTR protein.

• #65 Treatment for Amyloidosis | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/amyloidosis/treatment.html

  Treatment for amyloidosis is complex and must be tailored to each individual, so its important to be treated at a specialized center with expertise in this disease. […] Fred Hutch is involved in research to better understand amyloidosis and to identify more effective treatments, such as amyloid-removing agents, like CAEL-101. In addition, studies are underway at our center to assess the benefit of adding the monoclonal antibody daratumumab (Darzalex) to standard treatment with the hope that this will speed and deepen the response to chemotherapy. […] We view treatment as a collaborative effort. Your Fred Hutch physician will explain all your options and recommend an individualized treatment plan based on your type of amyloidosis as well as your specific medical needs and personal preferences.

• #66 Amyloidosis Treatment Options – NYC | Herbert Irving Comprehensive Cancer Center (HICCC) – New York

  https://www.cancer.columbia.edu/cancer-types-care/types/amyloidosis/treatments

  Our comprehensive multidisciplinary approach to amyloidosis treatment involves hematologists, nephrologists, neurologists, cardiologists, pathologists, and research experts. […] The many treatments for amyloidosis include: […] Primary amyloidosis (AL) is usually treated with a chemotherapy-based regimen (similar to that used for multiple myeloma) to destroy the abnormal plasma cells that give rise to the amyloid proteins or plaque. […] Chemotherapy is often given through an IV in our infusion center, but it can sometimes be given with pills. […] Targeted therapies work by targeting specific genes, proteins, or other aspects that contribute to the development of amyloidosis. […] One of the newest and most exciting new developments in therapy of AL amyloidosis is the development of CD38 antibody (Daratumumab), which works by binding to the malignant plasma cells and exposing them to the immune system of the patient.

• #67 Our Approach | AL Amyloidosis | Dana-Farber Cancer Institute

  https://www.dana-farber.org/cancer-care/treatment/hematologic-oncology/programs/amyloidosis/approach

  Treatment for AL amyloidosis involves chemotherapy to eradicate the plasma cells responsible for the production of amyloidogenic free light chain, thus stopping the production of the abnormal protein. For some patients, stem cell transplantation may be an option for long-term disease management. Many patients also require treatment for cardiac complications that arise from the disease. This may include an implantable cardioverter defibrillator or pacemaker for the treatment of atrial and ventricular arrhythmias that sometimes develop as the disease progresses. […] Each patient with AL amyloidosis is unique and requires thoughtful treatment planning by a multi-specialty team considering every aspect of the patient’s specific situation.

• #68 Treatment of amyloidosis: present and future

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10120969/

  Acoramidis (AG10/ALXN2060, Eldos Therapeutics) is a promising new TTR stabilizer under investigation for the treatment of ATTR amyloidosis and appears to be highly effective for the variant form with polyneuropathy, slowing disease progression. […] The treatment of amyloidosis has undergone a major change in recent years. Even though we currently have only a few drugs to use for the treatment of CA, very promising new treatments will be available to the cardiologist in the near future.

• #69 Cardiac Amyloidosis: Symptoms & Treatment | Mass General Brigham

  https://www.massgeneralbrigham.org/en/patient-care/services-and-specialties/heart/conditions/cardiac-amyloidosis

  Cardiac amyloidosis treatment varies based on the type of amyloid protein involved. […] Effective medications can slow or stop the deposition of amyloid in the heart, slowing the progression of the disease. However, existing amyloid deposits are not significantly affected by these drugs. […] Treatment typically involves chemotherapy or similar drugs that stop the production of new amyloid. Once abnormal protein production is stopped, patients may experience an improvement in their symptoms, even though existing amyloid deposits remains in the organs. […] Progress in the diagnosis and treatment of cardiac amyloidosis has been significant over the past decade. Early diagnosis is has enabled successful treatment, allowing patients to lead active lives. Ongoing clinical trials for novel therapies also offer hope for removing amyloid from the heart and other affected organs. The prognosis can be very good when patients are diagnosed early and receive treatment from a specialist in cardiac amyloidosis who can accurately identify the type of amyloid and develop a personalized treatment plan.

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