A patient with primary (AL) systemic amyloidosis developed mononeuropathy multiplex complicated by diaphragmatic failure. High dose melphalan and autologous stem cell transplantation did not ...ameliorate neuropathy or diaphragm dysfunction. Nocturnal non-invasive ventilation lowered arterial carbon dioxide levels and improved daytime dyspnea. This is the first case associating AL amyloid-induced neuropathy with diaphragm dysfunction.
Introduction Previously, specific criteria for recognizing organ involvement and defining response in amyloid (AL) had minimal utility. However, new therapies directed at the plasma cell, including ...conventional dose chemotherapy, myeloablative chemotherapy, and agents directed at disruption of fibril structure are regularly being used to treat patients. Currently criteria for response and organ involvement differ from institution to institution, making it difficult to directly compare outcomes. Outcomes are directly related to the number of organs involved with amyloid and accurate definition as to what constitutes organ involvement are more than an academic exercise. Thirteen leaders in the field were invited to submit institutional criteria from which the current guidelines were developed.
Results The panel drew up consensus guidelines for the following questions: What is required for a diagnosis of amyloidosis?, differentiating systemic from localized amyloidosis, How is amyloidosis characterized as AL type?, Definitions of organ involvement, Criteria for organ response (heart, kidney, liver, nerve and soft tissue) and organ progression, hematologic response criteria (including utilization of the newly developed immunoglobulin nephelometric free light chain assay) and Definitions for progressive disease both hematologic and organ based.
Conclusion Defining organ involvement and response criteria, both hematologic and organ-based for amyloidosis AL has always been challenging. The thirteen members of the consensus panel have defined criteria proposed to be used worldwide by physicians who treat patients with this disease and to permit uniform reporting criteria of treatment-related outcomes. In the future integration of new imaging techniques, as well as serum biomarkers such as serum troponin and brain naturetic peptide will further define the definitions of organ involvement and response.
We undertook this study to develop uniformly accepted criteria for the definition of organ involvement and response for patients on treatment protocols for immunoglobulin light-chain amyloidosis ...(AL). A consensus panel was convened comprising 13 specialists actively involved in the treatment of patients with amyloidosis. Institutional criteria were submitted from each, and a consensus was developed defining each organ involved and the criteria for response. Specific criteria have been developed with agreed on definitions of organ and hematologic response as a result of discussions at the 10th International Symposium on Amyloid and Amyloidosis held in Tours, France, April 2004. These criteria now form the working definition of involvement and response for the purposes of future data collection and reporting. We report criteria that centers can now use to define organ involvement and uniform response criteria for reporting outcomes in patients with light-chain AL.
Amyloid deposits are often found in the bone marrow in patients with Immunoglobulin light chain (AL) amyloidosis. We sought to determine whether this affects stem cell collection or engraftment after ...high-dose melphalan and autologous stem cell transplantation (HDM-SCT). We reviewed data on 361 patients with AL amyloidosis who had Congo red staining of pretreatment bone marrow biopsy specimens and underwent HDM-SCT between July 1994 and December 2011. We analyzed data on stem cell yield, days of stem cell collection, and days to neutrophil and platelet engraftment posttransplantation. Bone marrow amyloid deposits were found in 65% of patients (n = 233). There were no significant differences in median number of stem cells collected and days to neutrophil or platelet engraftment between patients with bone marrow amyloid deposits and those without these deposits. Thus, our data indicate that although amyloid involvement of the bone marrow is common, it does not negatively affect stem cell mobilization or neutrophil and platelet engraftment after HDM-SCT.
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.
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