Showing papers by "Martha Skinner published in 2011"
TL;DR: Treatment of selected AL patients with HDM/SCT resulted in a high organ response rate and long OS, even for those patients who did not achieve CR.
251 citations
TL;DR: It is concluded that patients with SSA are primarily men who present with dominant cardiac involvement at an older age than patients with familial TTR cardiomyopathy.
Abstract: Senile systemic amyloidosis (SSA) features cardiomyopathy resulting from amyloid deposits of wild-type transthyretin (TTR). From 1994 to 2009, 82 patients with SSA were diagnosed at our center; 79 were men (96%) and median age at diagnosis was 73.8 years (range, 59.1–86.0). Most patients (77/78) presented with abnormal echocardiography; median values for interventricular septal thickness and left ventricular ejection fraction were 16 mm (range, 9–24) and 50% (range, 20–70), respectively. Fat aspirates were positive for amyloid in 27% of patients. Mean levels of brain natriuretic peptide (n = 41) and troponin I (n = 19) were 422 ± 279 pg/ml and 0.151 ± 0.107 pg/ml. Median survival was 4.3 years (95% CI, 3.7–5.0). SSA and familial TTR cardiomyopathy were compared; survival distribution was significantly different across groups (log-rank test = 11.97, p-value = 0.0075). We conclude that patients with SSA are primarily men who present with dominant cardiac involvement at an older age than patients with familial TTR cardiomyopathy.
45 citations
TL;DR: None of these seven patients had evidence of a systemic plasma cell dyscrasia or amyloidosis in other organs, nor did systemic disease develop with a median follow-up of 6 years, so other than excisional biopsy to exclude malignancy, no systemic therapy is indicated for this disorder.
Abstract: We report on the clinical presentation and histopathology of a series of seven patients with localized amyloidosis of the breast. These patients were diagnosed by biopsy performed to rule out malignancy because of calcifications seen by mammography, and represented 0.5% of patients referred to the Amyloid Treatment and Research Program at Boston University Medical Campus in an 18-year period. The patients ranged in age from 35 to 75, median 63 years. None of these seven patients had evidence of a systemic plasma cell dyscrasia or amyloidosis in other organs, nor did systemic disease develop with a median follow-up of 6 years. Thus, other than excisional biopsy to exclude malignancy, no systemic therapy is indicated for this disorder.
44 citations
TL;DR: In this analysis, echocardiographic regression of WT was observed in a substantial fraction of subjects with ALC and CR after HDM/SCT, and the between group difference in change in WT was statistically significant.
Abstract: Increased cardiac wall thickness (WT) is a hallmark of amyloid cardiomyopathy. Sporadic reports have suggested that there may be a regression of cardiac WT after successful therapy of pri...
33 citations
TL;DR: Familial amyloidotic polyneuropathy is a lethal genetic disorder that affects the peripheral and autonomic nervous systems, heart, gastro-intestinal (GI) tract, and soft tissues.
Abstract: Familial amyloidotic polyneuropathy (FAP) is a lethal genetic disorder that affects the peripheral and autonomic nervous systems, heart, gastro-intestinal (GI) tract, and soft tissues. Di ...
24 citations
TL;DR: Survival comparisons showed that the group with traditional causes of AA had a nonstatistically significant trend toward shorter survival, which is similar to that of other atypical causes of inflammation.
Abstract: With improved antimicrobial and immunosuppressive therapies and diagnostic techniques for AA, cases are being identified with atypical or unknown causes of inflammation. We performed a re...
23 citations
TL;DR: Two cases of systemic amyloidosis with macroglossia that defied routine clinical diagnosis are reported, in which the deposits were typed as ATTR in one case and AL in the other using immunoelectron microscopy, highlighting the difficulty of typing amyloidsosis on clinical criteria alone.
Abstract: AL amyloidosis and transthyretin (ATTR) amyloidosis are the most frequent forms of systemic amyloidosis diagnosed in the United States. Macroglossia is considered to be a pathognomonic feature of AL amyloidosis. We report on two cases of systemic amyloidosis with macroglossia that defied routine clinical diagnosis, in which the deposits were typed as ATTR in one case and AL in the other using immunoelectron microscopy. These cases highlight: (1) the difficulty of typing amyloidosis on clinical criteria alone; (2) the utility of immunoelectron microscopy and (3) that macroglossia, while occurring much more frequently in AL, can also accompany ATTR amyloidosis.
17 citations
TL;DR: There was a steady reduction in rates of treatment-related mortality and improvement in 1-year survival, now approximately 5% and 90%, respectively, based upon an intention-to-treat analysis, over the past 10 years.
Abstract: Treatment with high-dose melphalan chemotherapy supported by hematopoietic rescue with autologous stem cells produces high rates of hematologic responses and improvement in survival and organ function for patients with AL amyloidosis. Ongoing clinical trials explore pre-transplant induction regimens, post-transplant consolidation or maintenance approaches, and compare transplant to non-transplant regimens. To put these studies into context, we reviewed our recent experience with transplant for AL amyloidosis in the Amyloid Treatment and Research Program at Boston Medical Center and Boston University School of Medicine. Over the past 10 years, there was a steady reduction in rates of treatment-related mortality and improvement in 1-year survival, now approximately 5% and 90%, respectively, based upon an intention-to-treat analysis. Median overall survival of patients treated with this approach at our center exceeds 7.5 years.
8 citations
2 citations
21 Aug 2011
TL;DR: In this article, the authors determined plasma levels of B-natriuretic peptide (BNP) in patients with light-chain-associated amyloidosis and correlate them with the presence or absence of heart failure (HF) and the presence of echocardiographic abnormalities.
Abstract: This study sought to determine plasma levels of B-natriuretic peptide (BNP) in patients with light-chain-associated amyloidosis and correlate them with the presence or absence of heart failure (HF) and the presence or absence of echocardiographic abnormalities. Patients with normal echocardiographic results had significantly lower BNP levels than those with echocardiographic features of cardiac amyloidosis, whereas BNP levels in the group with HF did not differ from those in patients with asymptomatic cardiac amyloidosis. This observation supports previous observations, suggesting that the elevation of BNP in cardiac amyloidosis may be due not only to elevated ventricular filling pressure but also to direct myocyte damage due to extracellular deposits of amyloid.
1 citations