-
Cancer Cell International Jul 2022Intravenous daratumumab (DARA IV) has been increasingly used in the treatment of amyloid light-chain (AL) amyloidosis. However, the outcomes for patients administered... (Review)
Review
BACKGROUND
Intravenous daratumumab (DARA IV) has been increasingly used in the treatment of amyloid light-chain (AL) amyloidosis. However, the outcomes for patients administered with DARA IV have not been aggregated. The objective of this systematic review and meta-analysis was to investigate the efficacy and safety of DARA IV for AL amyloidosis.
METHODS
We searched Medline, EMBASE, Cochrane Library and Web of Science up to 17 June 2021. Response rates and survival rates, and the corresponding 95% confidence intervals (CIs) were pooled and calculated using a fixed-effects model.
RESULTS
Thirty studies (5 cohort studies and 25 single-arm studies) with 997 patients were included. In patients receiving DARA IV-based treatments, very good partial response or better response rate, complete response rate, very good partial response rate, partial response rate and overall response rate were 66% (95% CI, 62-69%), 30% (95% CI, 23-36%), 40% (95% CI, 33-46%), 17% (95% CI, 14-21%), and 77% (95% CI, 73-80%), respectively. Cardiac and renal responses were 41% (95% CI, 34-49%) and 43% (95% CI, 32-54%), respectively. 58% (95% CI, 49-66%) of patients achieved PFS one year or longer. 2.5% (range, 1-10.0%) of patients experienced grade 3 or 4 adverse events, of which the most common adverse event was lymphocytopenia (range, 13.6-25.0%).
CONCLUSION
This study supports the efficacy and safety of DARA IV for the treatment of patients with AL amyloidosis.
PubMed: 35788237
DOI: 10.1186/s12935-022-02635-6 -
Journal of Huazhong University of... Oct 2010Primary tracheobronchial amyloidosis (TBA) is a rare pulmonary disease. A systematic review was performed on 64 cases of primary TBA in China and progress in the... (Review)
Review
Primary tracheobronchial amyloidosis (TBA) is a rare pulmonary disease. A systematic review was performed on 64 cases of primary TBA in China and progress in the diagnosis and treatment of this disease is discussed. The Chinese biological and medical databases from 1970 to 2010 were searched and 75 cases of complete clinical and pathological data were identified. The clinical characteristics of the disease were summarized and longitudinal comparisons were made of diagnostic and treatment methods over time. The results showed that the morbidity associated with primary TBA has increased over recent years. The clinical manifestations were non-specific. Progressive dyspnea, cough and sputum were the most common symptoms. The percentage of patients undergoing computed tomography (CT) scan has increased over the years. The bronchoscopy and transbrochial lung biopsy (TBLB) were usually sufficient to establish the diagnosis. Treatment was reported for a total of 44 cases. Bronchoscopic Nd:YAG laser irradiation, argon plasma coagulation (APC) and drugs administration such as steroids and colchicines were reported to be effective in some patients. It is concluded that the demographic characteristics and clinical manifestations of primary TBA patients in China are largely consistent with findings reported in other countries. Dramatically more cases were reported in recent years, mainly due to the extensive application of bronchoscopy since 1990s. Chest CT scan provides important clues for the diagnosis of the disease. The definite diagnosis was confirmed by bronchoscopic findings and Congo red staining of biopsy specimen. Bronchoscopic Nd:YAG laser irradiation, argon plasma coagulation (APC) and drugs administration, such as steroids and colchicines were reported to be effective in some patients.
Topics: Adult; Aged; Aged, 80 and over; Amyloidosis; Biopsy; Bronchial Diseases; Bronchoscopy; Female; Humans; Laser Therapy; Male; Middle Aged; Tracheal Diseases; Young Adult
PubMed: 21063841
DOI: 10.1007/s11596-010-0549-7 -
Clinical Lymphoma, Myeloma & Leukemia May 2022With recent advances in novel chemotherapeutic agents and increasing use of autologous hematopoietic stem cell transplant, there has been a significant improvement in... (Review)
Review
With recent advances in novel chemotherapeutic agents and increasing use of autologous hematopoietic stem cell transplant, there has been a significant improvement in outcomes for patients with AL Amyloidosis. Daratumumab, with its excellent safety and efficacy profile, appears to be an ideal treatment option for patients with newly diagnosed as well as relapsed refractory AL amyloidosis. In this systematic review, we analyzed the published literature on the role of Daratumumab in pretreated relapsed and refractory AL-amyloidosis patients using PubMed, Embase, Cochrane, and clinicaltrials.gov databases. A total of 16 studies evaluated the role of Daratumumab as monotherapy (DMT) or in combination with other chemotherapeutic agents (DCT). DMT and DCT were associated with promising efficacy with hematologic and organ responses (cardiac/renal) seen in 50%-90% and 50%-80% of the patients, respectively. Daratumumab appeared to be well tolerated with no significant treatment-related adverse events as DMT or DCT.
Topics: Antibodies, Monoclonal; Antineoplastic Agents; Humans; Immunoglobulin Light-chain Amyloidosis; Neoplasm Recurrence, Local; Treatment Outcome
PubMed: 34879994
DOI: 10.1016/j.clml.2021.10.014 -
Lasers in Medical Science Mar 2022With the investigation of the efficacy of laser therapy in primary localized amyloidosis(PLCA) only recently starting to materialize, we aimed to review the currently... (Review)
Review
With the investigation of the efficacy of laser therapy in primary localized amyloidosis(PLCA) only recently starting to materialize, we aimed to review the currently available studies of laser therapy in the management of the disease. We searched PubMed, Scopus, Embase, Web of Science, Cochrane, and ProQuest online databases with a specified search strategy, assessed the quality of each study, and then extracted the eligible data. Five RCTs(randomized controlled trials), one non-randomized controlled trial, three case series, and nine case reports(18 in total) were included. Overall, carbon dioxide (CO2), neodymium-doped:yttrium aluminum garnet (Nd:YAG), pulsed dye (PDL), Er (Erbium):YAG, and yttrium/erbium fiber were the studied lasers. One hundred fifty-five cases in total underwent laser therapy, with CO2 being the most frequent laser. Almost all studies demonstrated significantly desirable outcomes, while only mild and transient side effects were noted. Even though the studies' results were significant, we noticed that implementing a consistent methodology and a standardized objective assessment method was missing. Therefore, we recommend that future studies be conducted with less heterogeneous data for a more definite conclusion.
Topics: Humans; Amyloidosis, Familial; Laser Therapy; Lasers, Solid-State; Skin Diseases, Genetic; Randomized Controlled Trials as Topic
PubMed: 34611800
DOI: 10.1007/s10103-021-03429-4 -
European Archives of... Jul 2023The aim of this review was to study the surgical management of laryngeal amyloidosis and estimate the rate of recurrence after surgery. (Review)
Review
PURPOSE
The aim of this review was to study the surgical management of laryngeal amyloidosis and estimate the rate of recurrence after surgery.
METHODS
A systematic review searching PubMed and EMBASE was performed. A qualitative synthesis of data regarding the surgical management of LA and a quantitative analysis of the recurrence rate after surgery was conducted.
RESULTS
This systematic review included 14 retrospective studies, one of whom is retrospective controlled. A total of 515 subjects were included, the mean age ranged from 43.3 to 58 years with a male-to-female ratio of 1:1.3. All cases had a localized laryngeal amyloidosis. The supraglottic region was the most affected laryngeal site and multiple sites were commonly involved. Surgical treatment consists of endoscopic excision using laser, cold or powered instruments. Open surgery is required for severe primary case or revision surgery. Surgical complications such as granulomatosis scar tissue formation, tracheostomy, laryngotracheal stenosis, pneumothorax and concomitant malignancy were developed in 17.5% of patients. The time onset to diagnosis varied from 1 months to 15 years and the duration of follow-up from 3 months to 25 years. The rate of recurrence was 28.4% (95% CI 24.5-32.6) and the timing of recurrences ranged from 3 months to 10 years.
CONCLUSION
The recurrence rate after primary surgery for laryngeal amyloidosis is high. A tailored surgical treatment based on the disease extension and a long-term follow up are recommended.
Topics: Humans; Male; Female; Adult; Middle Aged; Retrospective Studies; Laryngeal Diseases; Larynx; Laryngostenosis; Amyloidosis
PubMed: 36790723
DOI: 10.1007/s00405-023-07881-6 -
Future Cardiology Jun 2022Aortic stenosis with cardiac amyloidosis (CA-AS) is common in the elderly. We provide an overview and a meta-analysis of outcomes after aortic valve (AV) intervention.... (Meta-Analysis)
Meta-Analysis Review
Aortic stenosis with cardiac amyloidosis (CA-AS) is common in the elderly. We provide an overview and a meta-analysis of outcomes after aortic valve (AV) intervention. The primary end point was all-cause mortality. Weighted pooled analysis showed a non-significant higher risk of death in CA-AS patients following surgical or transcatheter AV replacement. After transcatheter AV replacement, the risk of death in CA-AS patients was comparable to that associated with aortic stenosis alone (risk ratio: 1.23; 95% CI: 0.77-1.96; p = 0.39; I = 0%). An AV intervention is possibly not futile in CA-AS and should not be denied to patients with this condition.
Topics: Aged; Amyloidosis; Aortic Valve; Aortic Valve Stenosis; Humans; Risk Factors; Transcatheter Aortic Valve Replacement; Treatment Outcome
PubMed: 35420047
DOI: 10.2217/fca-2021-0118 -
Pacing and Clinical Electrophysiology :... Dec 2021Cardiac amyloidosis (CA) is an increasingly recognized cause of heart failure, characterized by extracellular deposition of insoluble protein fibrils leading to...
Cardiac amyloidosis (CA) is an increasingly recognized cause of heart failure, characterized by extracellular deposition of insoluble protein fibrils leading to progressive myocardial dysfunction. The most common types of cardiac amyloidosis are immunoglobin light-chain (AL) and transthyretin (ATTR). Conduction abnormalities are commonly encountered among patients with cardiac amyloidosis and are an important cause of morbidity and mortality. Abnormalities range from infra-Hisian intraventricular conduction delay and bundle branch block to complete atrioventricular block. Pacemaker placement in CA patients follows established guidelines, similar to those for patients without CA, with generally good efficacy. The role and appropriate timing of pacemakers for primary prevention of brady-arrhythmias in CA remains uncertain. While biventricular (BiV) pacing has been shown to improve clinical outcomes in patients with systolic heart failure without CA, there are few data examining the utility of BiV pacing in patients with CA. With the advent of effective treatments for AL and ATTR, appropriate application of pacing is important to support patients with CA and conduction disease through therapeutic trials. This systematic review summarizes the current literature examining the utility of pacing in CA.
Topics: Amyloidosis; Cardiac Pacing, Artificial; Heart Block; Heart Conduction System; Humans
PubMed: 34632598
DOI: 10.1111/pace.14375 -
International Journal of Cardiology May 2024Cardiac amyloidosis is increasingly recognized as a significant contributor to cardiovascular morbidity and mortality. With the emergence of novel therapies, there is a... (Meta-Analysis)
Meta-Analysis
BACKGROUND
Cardiac amyloidosis is increasingly recognized as a significant contributor to cardiovascular morbidity and mortality. With the emergence of novel therapies, there is a growing interest in prognostication of patients with cardiac amyloidosis using cardiac magnetic resonance imaging (CMR). In this systematic review and meta-analysis, we aimed to examine the prognostic significance of myocardial native T1 and T2, and extracellular volume (ECV).
METHODS
Observational cohort studies or single arms of clinical trials were eligible. MEDLINE, EMBASE and CENTRAL were systematically searched from their respective dates of inception to January 2023. No exclusions were made based on date of publication, study outcomes, or study language. The study populations composed of adult patients (≥18 years old) with amyloid cardiomyopathy. All studies included the use of CMR with and without intravenous gadolinium contrast administration to assess myocardial native T1 mapping, T2 mapping, and ECV in association with the pre-specified primary outcome of all-cause mortality. Data were extracted from eligible primary studies by two independent reviewers and pooled via the inverse variance method using random effects models for meta-analysis.
RESULTS
A total of 3852 citations were reviewed. A final nine studies including a total of 955 patients (mean age 65 ± 10 years old, 32% female, mean left ventricular ejection fraction (LVEF) 59 ± 12% and 24% had NYHA class III or IV symptoms) with cardiac amyloidosis [light chain amyloidosis (AL) 50%, transthyretin amyloidosis (ATTR) 49%, other 1%] were eligible for inclusion and suitable for data extraction. All included studies were single centered (seven with 1.5 T MRI scanners, two with 3.0 T MRI scanners) and non-randomized in design, with follow-up spanning from 8 to 64 months (median follow-up = 25 months); 320 patients died during follow-up, rendering a weighted mortality rate of 33% across studies. Compared with patients with AL amyloid, patients with ATTR amyloid had significantly higher mean left ventricular mass index (LVMi) (102 ± 34 g/m vs 127 ± 37 g/m, p = 0.02). N-terminal pro-brain natriuretic peptide (NT-proBNP), troponin T levels, mean native T1 values, ECV and T2 values did not differ between patients with ATTR amyloid and AL amyloid (all p > 0.25). Overall, the hazard ratios for mortality were 1.33 (95% CI = [1.10, 1.60]; p = 0.003; I = 29%) for every 60 ms higher T1 time, 1.16 (95% CI = [1.09, 1.23], p < 0.0001; I = 76%) for every 3% higher ECV, and 5.23 (95% CI = [2.27, 12.02]; p < 0.0001; I = 0%) for myocardial-to-skeletal T2 ratio below the mean (vs above the mean).
CONCLUSION
Higher native T1 time and ECV, and lower myocardial to skeletal T2 ratio, on CMR are associated with worse mortality in patients with cardiac amyloidosis. Therefore, tissue mapping using CMR may offer a useful non-invasive technique to monitor disease progression and determine prognosis in patients with cardiac amyloidosis.
Topics: Adult; Humans; Female; Middle Aged; Aged; Adolescent; Male; Cardiomyopathies; Stroke Volume; Ventricular Function, Left; Magnetic Resonance Imaging; Myocardium; Amyloid Neuropathies, Familial; Disease Progression; Magnetic Resonance Imaging, Cine; Predictive Value of Tests; Contrast Media; Observational Studies as Topic
PubMed: 38382853
DOI: 10.1016/j.ijcard.2024.131892 -
Orphanet Journal of Rare Diseases Jul 2022Amyloid light-chain (AL) amyloidosis is an ultra-rare disease associated with significant morbidity and mortality. Few studies have examined the global epidemiology of...
BACKGROUND
Amyloid light-chain (AL) amyloidosis is an ultra-rare disease associated with significant morbidity and mortality. Few studies have examined the global epidemiology of this condition.
METHODS
This study estimated the diagnosed incidence and 1-year, 5-year, 10-year, and 20-year period prevalence of AL amyloidosis in 2018 for countries in and near Europe, and in the United States (US), Canada, Brazil, Japan, South Korea, Taiwan, and Russia. A systematic literature review (SLR) was conducted to identify country-specific, age- and gender-specific diagnosed incidence of AL amyloidosis and observed survival data-point inputs for an incidence-to-prevalence model. Extrapolations were used to estimate incidence and prevalence for countries without registry or published epidemiological data.
RESULTS
Of 171 publications identified in the SLR, 10 records met the criteria for data extraction, and two records were included in the final incidence-to-prevalence model. In 2018, an estimated 74,000 AL amyloidosis cases worldwide were diagnosed during the preceding 20 years. The estimated incidence and 20-year prevalence rates were 10 and 51 cases per million population, respectively.
CONCLUSIONS
Orphan medicinal product designation criteria of the European Medicines Agency or Electronic Code of Federal Regulations indicate that a disease must not affect > 5 in 10,000 people across the European Union or affect < 200,000 people in the US. This study provides up-to-date epidemiological patterns of AL amyloidosis, which is vital for understanding the burden of the disease, increasing awareness, and to further research and treatment options.
Topics: Europe; Humans; Immunoglobulin Light-chain Amyloidosis; Incidence; Prevalence; Registries; United States
PubMed: 35854312
DOI: 10.1186/s13023-022-02414-6 -
International Journal of Surgery... 2008Amyloidosis refers to a variety of conditions in which amyloid proteins are abnormally deposited in organs and/or tissues. Amyloidosis is a rare occurrence in thyroid... (Review)
Review
BACKGROUND AND AIM
Amyloidosis refers to a variety of conditions in which amyloid proteins are abnormally deposited in organs and/or tissues. Amyloidosis is a rare occurrence in thyroid gland.
METHODS
A systematic review of the published data on amyloid goiter was carried out by searching Medline and other online databases (such as Scopus and Endnote) for the period from 1951 to March 2008. A total of 127 publications (case series, single case reports and reviews) was found, of which 31 were case series published from February 1995 to March 2008. Six articles have been considered for our review because they regard amyloid goiter as a manifestation of both primary and secondary amyloidosis (a total of 30 cases have been analyzed). Exclusion criterion was the presence of primary thyroid cancer.
RESULTS
The preoperative diagnosis of amyloid goiter should be considered in patients with known systemic amyloidosis or with a long-standing predisposing disease who present a rapidly growing thyroid volume in association with a euthyroid state. Fine-needle aspiration biopsy can be performed to exclude primary malignant lesions of thyroid gland and immunohistochemical studies can identify and characterize the amyloid deposits.
CONCLUSION
Amyloid goiter has to be suspected in all patients with a progressive, rapidly growing, bilateral thyroid enlargement and a concomitant history of chronic inflammatory processes. Moreover, this should be suspected in patients who are known to have disease predisposing to amyloid deposition.
Topics: Amyloidosis; Biopsy, Fine-Needle; Diagnosis, Differential; Goiter; Humans; Thyroid Gland; Tomography, X-Ray Computed; Ultrasonography
PubMed: 19168408
DOI: 10.1016/j.ijsu.2008.12.025