-
-
关键词:
- 系统性轻链型淀粉样变性 /
- 诊断 /
- 治疗
Abstract: The amyloidosis are rare diseases that characterized by abnormal deposition of amyloid.Systemic light chain amyloidosis(AL)is the most common type of amyloidosis in clinical practice,which often affects the function of vital organs such as heart and kidney.The incidence of AL has been increased these years in China,but the diagnosis and treatment are relatively insufficient.Imaging methods,biological and genetic indicators are critical to the early diagnosis and the prognosis for AL.Currently,The treatment for light chain amyloidosis is mostly focused on the elimination of malignant monoclonal plasma cells,and some new mechanism drugs targeting amyloid have also shown good efficacy.This article reviews the recent progress of pathogenesis,diagnosis and treatment for light chain amyloidosis.-
Key words:
- systemic light chain amyloidosis /
- diagnosis /
- treatment
-
[1] Fotiou D,Dimopoulos MA,Kastritis E.Systemic AL Amyloidosis:Current Approaches to Diagnosis and Management[J].Hemasphere,2020,4(4):e454.
[2] Huang XH,Liu ZH.The Clinical Presentation and Management of Systemic Light-Chain Amyloidosis in China[J].Kidney Dis(Basel),2016,2(1):1-9.
[3] Quock TP,Yan T,Chang E,et al.Epidemiology of AL amyloidosis:a real-world study using US claims data[J].Blood Adv,2018,2(10):1046-1053.
[4] Liu Y,Wen L,Ma L,et al.MAGE genes:Prognostic indicators in AL amyloidosis patients[J].J Cell Mol Med,2019,23(8):5672-5678.
[5] Cuddy SAM,Bravo PE,Falk RH,et al.Improved Quantification of Cardiac Amyloid Burden in Systemic Light Chain Amyloidosis:Redefining Early Disease?[J].JACC Cardiovasc Imaging,2020,13(6):1325-1336.
[6] Muchtar E,Gertz MA,Lacy MQ,et al.Refininga myloid complete hematological response:Quantitative serum free light chains superior to ratio[J].Am J Hematol,2020,95(11):1280-1287.
[7] Wong SW,Hegenbart U,Palladini G,et al.Outcome of Patients With Newly Diagnosed Systemic Light-Chain Amyloidosis Associated With Deletion of 17p[J].Clin Lymphoma Myeloma Leuk,2018,18(11):e493-e499.
[8] Liu Y,Lai Y,Ma L,et al.Fluorescence in situ hybridisation combined with CD138 immunomagnetic sorting is effective to identify cytogenetic abnormalities which play significant prognostic roles in Chinese AL amyloidosis patients[J].Amyloid,2020,27(3):208-209.
[9] Kimura K,Tsukamoto S,Miyazaki K,et al.Identification of Clonal Immunoglobulin λ Light-Chain Gene Rearrangements in AL Amyloidosis Using Next Generation Sequencing[J].Blood,2019,134(Supplement_1):1748-1748.
[10] Yang T,Wan K,Song R,et al.Serum high-density lipoprotein cholesterol serves as a prognostic marker for light-chain cardiac amyloidosis[J].Int J Cardiol,2021,325:96-102.
[11] Sidana S,Sidiqi MH,Dispenzieri A,et al.Fifteen year overal lsurvival rates after autologous stem cell transplantation for AL amyloidosis[J].Am J Hematol,2019,94(9):1020-1026.
[12] Roussel M,Merlini G,Chevret S,et al.A prospective phase 2 trial of daratumumab in patients with previously treated systemic light-chain amyloidosis[J].Blood,2020,135(18):1531-1540.
[13] Sanchorawala V,Sarosiek S,Schulman A,et al.Safety,tolerability,and response rates of daratumumab in relapsed AL amyloidosis:results of aphase 2 study[J].Blood,2020,135(18):1541-1547.
[14] Efstathios Kastritis G,Minnema MC,Wechalekar AD,et al.Subcutaneous daratumumab cyclophosphamide,bortezomib,and dexamethasone(Cybord)in patients with newly diagnosed lightchain(AL)amyloidosis:primary results from the phase 3 ANDROMED Astudy[C].Proceedings of the 25thEHA Congress Frankfurt;2020.
[15] Suzuki K,Wechalekar AD,Kim K,et al.Subcutaneous Daratumumab(DARASC)+Bortezomib,Cyclophosphamide,and Dexamethasone(VCd)in Asian Patients with Newly Diagnosed Light Chain(AL)Amyloidosis:Subgroup Analysis from the Phase 3 And romeda Study[J].Blood,2020,136(Supplement1):11-11.
[16] Parker TL,Rosenthal A,Sanchorawala V,et al.A Phase II Study of Isatuximab(SAR650984)(NSC-795145) for Patients with Previously Treated AL Amyloidosis(SWOGS1702;NCT#03499808)[J].Blood,2020,136(Supplement1):20-21.
[17] Oliva L,Orfanelli U,Resnati M,et al.The amyloido genic light chain is a stress or that sensitizes plasma cells to proteasome inhibitor toxicity[J].Blood,2017,129(15):2132-2142.
[18] Minnema MC,Nasserinejad K,Hazenberg B,et al.Bortezomib-based induction followed by stem cell transplantation in light chain amyloidosis:results of the multicenter HOVON104 trial[J].Haematologica,2019,104(11):2274-2282.
[19] Kastritis E,Leleu X,Arnulf B,et al.Bortezomib,Melphalan,and Dexamethasone for Light-Chain Amyloidosis[J].J Clin Oncol,2020,38(28):3252-3260.
[20] Cohen AD,Landau H,Scott EC,et al.Safety and Efficacy of Carfilzomib(CFZ)in Previously-Treated Systemic Light-Chain(AL)Amyloidosis[J].Blood,2016,128(22):645-645.
[21] Sanchorawala V,Palladini G,Kukreti V,et al.Aphase 1/2 study of the oral proteasome inhibito rixazomib in relapsed or refractory AL amyloidosis[J].Blood,2017,130(5):597-605.
[22] Dispenzieri A,Kastritis E,Wechalekar AD,et al.Primary Results from the Phase 3 Tourmaline-AL1 Trial of Ixazomib-Dexamethasone Versus Physician's Choice of Therapy in Patients(Pts)with Relapsed/Refractory Primary Systemic AL Amyloidosis(RRAL)[J].Blood,2019,134(Supplement_1):139-139.
[23] Cohen OC,Sharpley F,Gillmore JD,et al.Use of ixazomib,lenalidomide and dexamethasone in patients with relapsed amyloid light-chain amyloidosis[J].Br J Haematol,2020,189(4):643-649.
[24] Muchtar E,Gertz MA,Laplant B,et al.Phase 2 Trial of Ixazomib,Cyclophosphamide and Dexamethasone for Treatment of Previously Untreated Light Chain Amyloidosis[J].Blood,2020,136(Supplement1):52-53.
[25] Sharpley FA,Manwani R,Mahmood S,et al.Real world outcomes of pomalidomide for treatment of relapsed light chain amyloidosis[J].Br J Haematol,2018,183(4):557-563.
[26] Palladini G,Milani P,Foli A,et al.A phase 2 trial of pomalidomide and dexamethason eres cue treatment in patients with AL amyloidosis[J].Blood,2017,129(15):2120-2123.
[27] Milani P,Sharpley F,Schönland SO,et al.Pomalidomide and dexamethasone grant rapid haematologic responses in patients with relapsed and refractory AL amyloidosis:a European retrospective series of 153 patients[J].Amyloid,2020,27(4):231-236.
[28] Sidiqi MH,AlSaleh AS,Leung N,et al.Venetoclax for the treatment of translocation(11;14) AL amyloidosis[J].Blood Cancer J,2020,10(5):55.
[29] Gran C,BorgBruchfeld J,Ellin F,et al.Rapid Complete Response to Single-Agent Bcl-2 Inhibitor Venetoclax in a Heart-Transplanted Patient with Triple Refractory Immunoglobulin Light-Chain Amyloidosis[J].Acta Haematol,2020,143(5):500-503.
[30] Gertz MA,Landau H,Comenzo RL,et al.First-in-Human Phase I/II Study of NEOD001 in Patients With Light Chain Amyloidosis and Persistent Organ Dysfunction[J].J Clin Oncol,2016,34(10):1097-1103.
[31] Gertz MA,Cohen AD,Comenzo RL,et al.Results of the Phase3 VITAL Study of NEOD001(Birtamimab)Plus Standard of Care in Patients with Light Chain(AL)Amyloidosis Suggest Survival Benefit for Mayo Stage IV Patients[J].Blood,2019,134(Supplement_1):3166-3166.
[32] Khouri J,Anwer F,Samaras CJ,et al.Safety,Tolerability and Efficacy of Cael-101 in AL Amyloidosis Patients Treated on a Phase 2,Open-Label,Dose Selection Study to Evaluate the Safety and Tolerability of Cael-101 in Patients with AL Amyloidosis[J].Blood,2020,136(Supplement1):21-21.
[33] Richards DB,Cookson LM,Berges AC,et al.Therapeutic Clearance of Amyloidby Antibodies to Serum Amyloid P Component[J].N Engl J Med,2015,373(12):1106-1114.
[34] Wechalekar AD,Whelan C.Encouraging impact of doxycycline on early mortality in cardiac light chain(AL) amyloidosis[J].Blood Cancer J,2017,7(3):e546.
[35] Shen K,Dong Y,Fu W,et al.Doxycycline Combined with Bortezomib-Cyclophosphamide-Dexamethasone Chemotherapy for Newly Diagnosed Patients with Mayo2004 Stage II-III Light-Chain Amyloidosis:A Randomized Controlled Multicenter Study[J].Blood,2019,134(Supplement_1):1870-1870.
[36] Mereles D,Buss SJ,Hardt SE,et al.Effects of the main green teapolyphenol epigallocatechin-3-gallateon cardiac involvement in patients with AL amyloidosis[J].Clin Res Cardiol,2010,99(8):483-490.
[37] Meshitsuka S,Shingaki S,Hotta M,et al.Phase 2 trial of daily,oral epigallocatechin gallate in patients with light-chain amyloidosis[J].Int J Hematol,2017,105(3):295-308.
[38] Lentzsch S,Lagos GG,Comenzo RL,et al.Bendamustine With Dexamethasone in Relapsed/Refractory Systemic Light-Chain Amyloidosis:Results of a Phase II Study[J].J Clin Oncol,2020,38(13):1455-1462.
[39] Rosenzweig M,Urak R,Walter M,et al.Preclinical data support lever aging CS1 chimeric antigen receptor T-cell therapy for systemic light chain amyloidosis[J].Cytotherapy,2017,19(7):861-866.
[40] Palladini G,Schönland S,Merlini G,et al.First Glimpse on Real-World Efficacy Outcomes for 2000 Patients with Systemic Light Chain Amyloidosis in Europe:A Retrospective Observational Multicenter Study By the European Myeloma Network[J].Blood,2020,136(Supplement1):50-51.
计量
- 文章访问数: 392
- PDF下载数: 659
- 施引文献: 0