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1.  Amyloid Deposits in the Bone Marrow of Patients with AL Amyloidosis Do Not Impact Stem Cell Mobilization or Engraftment 
Amyloid deposits are often found in the bone marrow in patients with AL amyloidosis; we sought to determine whether this affects stem cell collection or engraftment following high dose melphalan and autologous stem cell transplantation (HDM/SCT). Data on 361 patients with AL amyloidosis who had Congo red staining of the pre-treatment bone marrow biopsy and underwent HDM/SCT from July 1994 to December 2011 were reviewed. Data were analyzed for stem cell yield, number of days of stem cell collection, neutrophil and platelet engraftment post SCT. Sixty-five % of patients (n=233) had bone marrow amyloid deposits. There were no significant differences in median number of stem cells collected, days to neutrophil or platelet engraftment between patients with and without bone marrow amyloid deposits. Thus, while amyloid involvement of the bone marrow is common, it does not negatively impact stem cell mobilization or neutrophil and platelet engraftment after HDM/SCT.
PMCID: PMC4161277  PMID: 22842332
Amyloidosis; light-chain; melphalan; stem cell transplantation; stem cell mobilization; stem cell collection; engraftment; bone marrow
2.  Amyloidotic Cardiomyopathy: Multidisciplinary Approach to Diagnosis and Treatment 
Heart failure clinics  2011;7(3):385-393.
PMCID: PMC3135875  PMID: 21749890
amyloidosis; cardiomyopathy; transthyretin; immunoglobulin light chain; matrix metalloproteinase; autologous stem cell transplantation; biomarkers
3.  Kidney dysfunction during lenalidomide treatment for AL amyloidosis 
Background. Lenalidomide is an immunomodulatory agent used to treat plasma cell dyscrasias. We previously observed worsening of kidney function in a high proportion of patients with AL amyloidosis during lenalidomide treatment. The objective of this study is to characterize alterations in kidney function among patients with AL amyloidosis undergoing treatment with lenalidomide.
Methods. This is a secondary analysis of an ongoing clinical trial at a single referral centre. Forty-one patients with AL amyloidosis received lenalidomide with or without dexamethasone in monthly cycles. Kidney dysfunction was defined as ≥ 50% increase in serum creatinine. Severe kidney dysfunction was defined as ≥ 100% increase in serum creatinine. Recovery of renal function was defined as a return of serum creatinine to within 25% of the pre-treatment value or discontinuation of dialysis.
Results. Twenty-seven of 41 patients (66%) developed kidney dysfunction during lenalidomide treatment. The kidney dysfunction was severe in 13 of these patients (32%); four of whom required initiation of dialysis (10%). The median time to kidney dysfunction after starting lenalidomide was 44 days (interquartile range 15–108 days). Four of eight patients without underlying renal amyloidosis developed kidney dysfunction. Patients with severe kidney dysfunction were older and had a higher frequency of underlying renal amyloidosis, greater urinary protein excretion, and lower serum albumin. Recovery of renal function occurred in 12 patients (44%).
Conclusions. Among patients with AL amyloidosis, worsening of kidney function occurs frequently during lenalidomide treatment. While a causal role of the drug has not been established, our findings suggest that kidney function should be monitored closely during treatment with this drug.
PMCID: PMC3108346  PMID: 20693160
acute kidney injury; AL amyloidosis; lenalidomide; nephrotic syndrome; treatment
4.  Role of high-dose melphalan and autologous peripheral blood stem cell transplantation in AL amyloidosis 
AL amyloidosis is the most common form of systemic amyloidosis and is associated with an underlying plasma cell dyscrasia. The disease is often difficult to recognize because of its broad range of manifestations and, what are often, vague symptoms. Recent diagnostic and prognostic advances include the serum free light chain assay, cardiac magnetic resonance imaging, and serologic cardiac biomarkers. Treatment strategies that have evolved during the past decade are prolonging survival and preserving organ function in patients with this disease. This review outlines the role of high dose melphalan and stem cell transplantation in the treatment of AL amyloidosis.
PMCID: PMC3301435  PMID: 22432083
AL amyloidosis; stem cell transplantation; melphalan
5.  Cardiac Transplantation Followed by Dose-Intensive Melphalan and Autologous Stem Cell Transplantation for AL Amyloidosis and Heart Failure 
Transplantation  2010;90(8):905-911.
Patients with AL amyloidosis who present with severe heart failure due to cardiac involvement rarely survive more than six months. Survival after cardiac transplantation is markedly reduced due to the progression of amyloidosis. Autologous stem cell transplantation (ASCT) has become a common therapy for AL amyloidosis, but there is an exceedingly high treatment-related mortality in patients with heart failure.
We developed a treatment strategy of cardiac transplant followed by ASCT. 26 patients were evaluated, and of 18 eligible patients, nine patients underwent cardiac transplantation. Eight of these patients subsequently received an ASCT.
Six of seven evaluable patients achieved a complete hematologic remission, and one achieved a partial remission. At a median follow-up of 56 months from cardiac transplant, five of seven patients are alive without recurrent amyloidosis. Their survival is comparable to 17,389 patients who received heart transplants for non-amyloid heart disease: 64% in non-amyloid vs. 60% in amyloid patients at seven years (p= 0.83). Seven of eight transplanted patients have had no evidence of amyloid in their cardiac allograft.
This demonstrates that cardiac transplantation followed by ASCT is feasible in selected patients with AL amyloidosis and heart failure, and that such a strategy may lead to improved overall survival. (, NCT00456040)
PMCID: PMC2964067  PMID: 20733534
Amyloid; Cardiac Amyloidosis; Stem Cell Transplantation
6.  Spontaneous rupture of the liver in a patient with systemic AL amyloidosis undergoing treatment with high-dose melphalan and autologous stem cell transplantation: A case report with literature review 
A 55-year-old woman with primary Immunoglobulin light chain (AL) systemic amyloidosis died due to spontaneous rupture of her liver following treatment with high-dose melphalan and autologous stem cell transplant (HDM/SCT). She was first diagnosed after developing nephrotic-range proteinuria. Spontaneous rupture of her liver occurred 10 days after treatment with HDM/SCT and was complicated by septic shock. She was not eligible for surgical intervention and died shortly after. Amyloid fibrils were extracted from the autopsied liver sample (05-135L) and the biochemical nature of the fibrils was analyzed using electrophoretic and immunohistochemical techniques. Our testing showed that the fibrils were composed of immunoglobulin lambda light chains that were not glycosylated.
While the liver is often involved in AL amyloidosis, this is the first documented case of a spontaneous hepatic rupture in a patient during treatment with HDM/SCT. A literature review of spontaneous liver rupture in patients with amyloidosis is presented.
PMCID: PMC2911629  PMID: 20536404
AL amyloidosis; liver rupture; stem cell transplantation

Results 1-6 (6)