Primary systemic amyloidosis

Gertz, Morie A.; Rajkumar, S. Vincent

doi:10.1007/s11864-002-0016-1

Cited by 50 publications

(32 citation statements)

References 47 publications

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“…The mean age was 56 years; 36% of patients were #50 years of age and only 22% were elderly (.64 years of age). In comparison, the reported mean age at diagnosis was 66 years for MCN (11) and 62 years for light chain amyloidosis (12). Therefore, in patients with dysproteinemia, the chance of finding pure MIDD and not amyloid or MCN is higher in middle-aged adults compared with the elderly population.…”

Section: Discussionmentioning

confidence: 96%

Renal Monoclonal Immunoglobulin Deposition Disease

Nasr¹,

Valeri²,

Cornell³

et al. 2012

Clinical Journal of the American Society of Nephrology

250

214

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SummaryBackground and objectives To better define the clinical-pathologic spectrum and prognosis of monoclonal immunoglobulin deposition disease (MIDD), this study reports the largest series.Design, setting, participants, & measurements Characteristics of 64 MIDD patients who were seen at Mayo Clinic are provided.Results Of 64 patients with MIDD, 51 had light chain deposition disease, 7 had heavy chain deposition disease, and 6 had light and heavy chain deposition disease. The mean age at diagnosis was 56 years, and 23 patients (36%) were #50 years of age. Clinical evidence of dysproteinemia was present in 62 patients (97%), including multiple myeloma in 38 (59%). M-spike was detected on serum protein electrophoresis in 47 (73%). Serum free light chain ratio was abnormal in all 51 patients tested. Presentation included renal insufficiency, proteinuria, hematuria, and hypertension. Nodular mesangial sclerosis was seen in 39 patients (61%). During a median of 25 months of follow-up (range, 1-140) in 56 patients, 32 (57%) had stable/improved renal function, 2 (4%) had worsening renal function, and 22 (39%) progressed to ESRD. The mean renal and patient survivals were 64 and 90 months, respectively. The disease recurred in three of four patients who received a kidney transplant.Conclusions Patients with MIDD generally present at a younger age than those with light chain amyloidosis or light chain cast nephropathy. Serum free light chain ratio is abnormal in all MIDD patients, whereas only threequarters have abnormal serum protein electrophoresis. The prognosis for MIDD is improving compared with historical controls, likely reflecting earlier detection and improved therapies.

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Section: Discussionmentioning

confidence: 96%

Renal Monoclonal Immunoglobulin Deposition Disease

Nasr¹,

Valeri²,

Cornell³

et al. 2012

Clinical Journal of the American Society of Nephrology

250

214

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“…27 Two prospective randomized clinical trials have shown that treatment with oral melphalan and prednisone results in objective responses and improved survival 16,17 in a minority of patients. However, because the median overall survival remains only 18 months with standard therapy, new strategies are required.…”

Section: Discussionmentioning

confidence: 99%

A multicenter phase 2 trial of stem cell transplantation for immunoglobulin light-chain amyloidosis (E4A97): an Eastern Cooperative Oncology Group Study

Gertz

Blood

Vesole

et al. 2004

Bone Marrow Transplant

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Summary:Stem cell transplantation was introduced as a new therapeutic modality for amyloidosis. The purpose of the current study was to determine the feasibility and toxicity of stem cell transplantation for amyloidosis in a cooperative group setting in which most participating institutions would have limited experience in managing the disorder. A total of 30 patients with biopsy-proven amyloidosis shown to be immunoglobulin light-chain type were enrolled on this trial. The protocol required mobilization of a minimum of 6 Â 10 8 mononuclear cells/ kg or 5 Â 10 6 CD34 þ cells/kg ideal body weight. These targets had to be achieved within seven collections. Patients with advanced hepatic, renal, or cardiac failure were excluded. End points included objective response rate and overall survival. The secondary end point of the protocol was nonhematologic toxicity. Accrual to the study was faster than expected. The overall response rate (hematologic and organ) was 64%, with three treatmentrelated deaths. Another patient died before day 30 of sudden cardiac death not treatment related. The median follow-up of surviving patients is 30.3 months. Median survival has not been reached. Stem cell transplantation for selected patients with amyloidosis is feasible in a cooperative group setting. A multicenter phase 3 trial of high-dose therapy is indicated.

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“…5 Effective treatments for AL amyloidosis exist, but treatment options are limited once cardiac disease becomes clinically apparent. Recently, the use of high-dose melphalan with rescue autologous peripheral blood stem cell transplantation has resulted in reversal of the clinical manifestations of AL amyloidosis in a significant proportion of patients who survived the procedure.…”

Section: See P 186mentioning

confidence: 99%