IgM-related amyloid light-chain (AL) amyloidosis is a rare disease, with patients presenting with more renal and neurologic involvement and less cardiac involvement compared with those with non-IgM-related disease. We retrospectively reviewed 38 patients receiving autologous stem cell transplant (ASCT) for IgM-related AL amyloidosis at the Mayo Clinic between May 1999 and June 2018. Median age was 61years, and 71% were men. The most common organs involved were renal (63%), neurologic (32%), and cardiac (26%). The median difference between involved and uninvolved free light chains was 6.2mg/dL, and most patients had early Mayo stage disease (87% Mayo stage I 2004 and 74% Mayo stage I 2012). The overall response rate was 92%, with 76% of patients achieving at least a very good partial response. Renal response was seen in 65% of patients (15/23; median time, 18 months post-ASCT; range 3 to 52) and cardiac response in 60% of patients (6/10; median time, 12 months post-ASCT; range 10 to 35). Median progression-free survival (PFS) and overall survival (OS) was 48 and 106 months, respectively. Organ response predicted better PFS and OS (median PFS, 93 months for organ response versus 16 months for no organ response [P = .0006]; and median OS, 123 months for organ response versus 41 months for no organ response [P = .02]). Two patients died within 100days of transplant, representing a 5% 100-day mortality. ASCT is an effective therapy that can be safely delivered to carefully selected patients with IgM-related AL amyloidosis.
Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6445701 | PMC |
| http://dx.doi.org/10.1016/j.bbmt.2018.12.003 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Screening and prognostic roles of renal volumetry and scintigraphy in the assessment of living kidney transplant donors, considering the early recovery of the residual renal function.
BMC Nephrol
January 2025
Department of Urology, Nara Medical University, 840 Shijo-cho, Kashihara, Nara, 634-8522, Japan.
Background: The existing criteria for living kidney donors (LKDs)in Japan are controversial. We evaluated the roles of computed tomography volumetry (CTV) and 99 m Tc-diethylenetriamine penta-acetic acid (DTPA) scintigraphy in assessing preoperative and postoperative renal function and predicting early recovery of residual renal function.
Methods: We retrospectively reviewed the medical charts of 175 consecutive LKDs who underwent donor nephrectomy (DN) at our institution between 2006 and 2022.
Zellweger spectrum disorder presenting with opsoclonus-myoclonus-ataxia syndrome: a case report on immunotherapy.
Acta Neurol Belg
January 2025
Department of Pediatrics, Neurology Unit, University of Health Sciences, Ankara Etlik City Hospital, Ankara, Turkey.
Introduction: Zellweger spectrum disorder (ZSD) refers to a group of autosomal recessive genetic disorders that affect multiple organ systems and are predominantly caused by pathogenic variants in PEX genes. ZSD present a wide clinical spectrum, ranging from the most severe form, Zellweger syndrome, to the mildest form, Heimler syndrome.
Case Report: A 14-month-old male patient was brought to our clinic with recent-onset ocular tremors and unsteady gait.
A Phase II Trial of Onapristone and Fulvestrant for Patients With ER+ and HER2- Metastatic Breast Cancer.
Clin Breast Cancer
December 2024
Medical Oncology and Palliative Care, Department of Medicine, Breast Cancer Disease Oriented Team, University of Wisconsin Carbone Cancer Center, University of Wisconsin School of Medicine and Public Health, Madison, WI 53792-3252.
Background: The SMILE study is a multi-institutional phase II clinical trial to determine the efficacy and safety of an antiprogestin, onapristone, in combination with fulvestrant as second-line therapy for patients with ER+, PgR+/-, HER2- metastatic breast cancer. This study was terminated early and herein, we report patient characteristics, and outcomes.
Methods: Eligibility criteria included disease progression on ≥2 lines of prior therapy, ECOG performance status ≤ 2, measurable disease per RECIST 1.
Safety and efficacy of PD-1 inhibitor (sintilimab) combined with transarterial chemoembolization as the initial treatment in patients with intermediate-stage hepatocellular carcinoma beyond up-to-seven criteria.
J Immunother Cancer
January 2025
Department of Liver Surgery and Transplantation, Liver Cancer Institute, Zhongshan Hospital, Fudan University, Shanghai, China
Background: Numerous studies have demonstrated limited survival benefits of transarterial chemoembolization (TACE) alone in the treatment of intermediate-stage hepatocellular carcinoma (HCC) beyond up-to-seven criteria. The advent of immunotherapy, particularly immune checkpoint inhibitors (ICIs), has opened new avenues for HCC treatment. However, TACE combined with ICIs has not been investigated for patients with intermediate-stage HCC beyond the up-to-seven criteria.
View Article and Find Full Text PDFImpact of Obesity on GVHD in Patients Undergoing Allogeneic Hematopoietic Cell Transplant for Hematologic Malignancies.
Transplant Cell Ther
January 2025
Blood and Marrow Transplant Program, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH, USA. Electronic address:
Background: The relationship between obesity and graft-versus-host disease (GVHD) has been studied in both pre-clinical and clinical studies with varying results.
Objectives: We aimed to investigate the impact of obesity, as measured by body mass index (BMI), on the incidence, severity, and response to therapy of GVHD in a contemporary cohort.
Study Design: We conducted a retrospective study of patients undergoing allogeneic hematopoietic cell transplant (HCT) for acute myelogenous leukemia and myelodysplastic syndrome between January 2010 and December 2021 at the Cleveland Clinic.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!