AL amyloidosis is a disease in which immunoglobulin L chain is deposited in multiple organs, and the prognosis of cardiac amyloidosis is extremely poor. Although several treatments based on that for multiple myeloma, have been performed, there is no clear evidence that cardiac function is improved. We report a case of AL cardiac amyloidosis with moderate cardiac dysfunction for which we performed autologous peripheral blood stem cell transplantation (auto-PBSCT) in combination with high-dose melphalan therapy. This treatment resulted in significant improvement in cardiac function and good prognosis for about 3.5 years after the diagnosis. Therefore, auto-PBSCT is a possible option as up-front therapy for AL cardiac amyloidosis.
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http://dx.doi.org/10.2169/internalmedicine.46.0142 | DOI Listing |
Int J Hematol
December 2024
Department of Hematology and Oncology, Nagoya City University Institute of Medical and Pharmaceutical Sciences, Kawasaki 1, Mizuno-cho, Mizuno-ku, Nagoya, Aichi, 467-8601, Japan.
This post-marketing surveillance (PMS) assessed the safety and effectiveness of isatuximab plus pomalidomide and dexamethasone (Isa-Pd) for relapsed or refractory multiple myeloma (RRMM) in frail individuals during real-world use in Japan. Data from all individuals with RRMM treated with Isa-Pd in Japan between October 2020 and October 2021 were collected, with follow-up continued up to 12 months after starting Isa-Pd or until discontinuation. In the overall PMS population, 40 participants were classified as frail (33.
View Article and Find Full Text PDFOchsner J
January 2024
Division of Cardiology, Baylor Scott & White Medical Center, Temple, TX.
Waldenström macroglobulinemia is a rare cancer of plasma cells characterized by the excessive production of immunoglobulin M (IgM). IgM-associated systemic amyloid light chain (AL) amyloidosis is a rare complication of Waldenström macroglobulinemia, characterized by the misfolding of lambda light chains that deposit in various organs, including the heart. We describe a case of progressive nonischemic cardiomyopathy secondary to Waldenström macroglobulinemia and IgM-associated AL amyloidosis that was refractory to medical therapy and highlight the challenges in diagnosis and management.
View Article and Find Full Text PDFBackground: For the time being, tafamidis is the only approved treatment for wild-type transthyretin cardiac amyloidosis. However, benefits on all-cause death only emerge after ≈18 months. The current available staging systems are unable to specifically discriminate patients at high risk of death within 18 months from diagnosis, and the selection of patients who are expected to benefit from tafamidis is left to the clinical judgment of treating physicians, being often based primarily (and sometimes only) on age.
View Article and Find Full Text PDFVirchows Arch
December 2024
Department of Cardiology, Hospital Álvaro Cunqueiro, Vigo, Spain.
Amyloidosis is a rare disease that can affect genitourinary organs but the involvement of the prostate has been documented in a limited number of cases. We have reviewed morphologic and immunohistochemical features of prostate biopsies or surgical specimens in which an initial diagnosis of amyloidosis was made. Prostatic amyloidosis was diagnosed in 25 patients, 21 of them were needle biopsies (1.
View Article and Find Full Text PDFPharmacotherapy
December 2024
Department of Pharmacy Practice, University of Illinois Chicago, Chicago, Illinois, USA.
Transthyretin amyloidosis (ATTR) is a rare disease that results in amyloid fibril misfolding and deposition in multiple organs, including the heart, leading to the development of ATTR cardiomyopathy (ATTR-CM), which is associated with poor outcomes. In the last decade, several disease-modifying medications are in advanced stages of clinical development or have been approved to treat ATTR-CM. The purpose of this review is to critically evaluate clinical trial data investigating the use of approved and investigational medications for the treatment of ATTR-CM.
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