scholarly journals Treatment-Related Mortality in Patients with AL Amyloidosis Undergoing High-Dose Melphalan and Stem Cell Transplantation: Trend Over The Past 14 Years at a Single Institution

2009 ◽  
Vol 15 (2) ◽  
pp. 11 ◽  
Author(s):  
V. Sanchorawala ◽  
M. Skinner ◽  
K.T. Finn ◽  
K. Quillen ◽  
D.C. Seldin
Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 2873-2873
Author(s):  
Saulius Girnius ◽  
David C. Seldin ◽  
Martha Skinner ◽  
Kathleen T. Finn ◽  
Vaishali Sanchorawala

Abstract Immunoglobulin light chain (AL) amyloidosis is a plasma cell dyscrasia with extracellular protein deposition in various organ systems, including the liver. The natural history of AL amyloidosis with liver involvement has a poor prognosis, with median survival of only 8.5 to 9 months. High-dose melphalan chemotherapy and autologous peripheral blood stem cell transplantation (HDM/SCT) has been shown to result in durable hematologic response and prolonged overall survival in systemic AL amyloidosis. Patients were included who had hepatic involvement and received HDM/SCT from 1998 to 2006. Data was collected with the approval of the Institutional Review Board. Patients receiving HDM/SCT had to meet eligibility criteria to qualify for this aggressive treatment. Stem cells were mobilized using G-CSF alone. Intravenous melphalan was administered at 100–200 mg/m2 over 2 days in divided doses and stem cells were re-infused 24–72 hours after HDM. Liver response was determined by criteria set up by the Consensus Opinion from the 10th International Symposium on Amyloid and Amyloidosis. Sixty-nine patients qualified for this study, including 47 males and 22 females. The median age was 56 years (range, 37–75), median bilirubin was 0.5 mg/dL (range, 0.1 to 5.7), median alkaline phosphatase was 193 U/L (range, 59 to 1243) and the median liver size was 3 cm (range, 0–20cm) below the costal margin. Nine patients (13%) died from treatment-related mortality. The hematologic CR one year after treatment was achieved by 53% (31/58) of evaluable patients. The overall survival was 84% at 1 year and 49% at 5 years, by Kaplan-Meier estimates. Hepatic response occurred in 21% (10/47) at 1 year after HDM/SCT and 40% (12/30) at 2 years after HDM/SCT. In summary, hepatic involvement with AL amyloidosis does not increase treatment-related mortality from HDM/SCT. Hepatic response follows a hematological response and can occur as far as 2 years from HDM/SCT. Forty % of surviving evaluable patients showed remission of their hepatic disease from AL amyloidosis by 2 years.


Cancers ◽  
2020 ◽  
Vol 12 (6) ◽  
pp. 1678
Author(s):  
Panagiotis Karagiannis ◽  
Lena Sänger ◽  
Winfried Alsdorf ◽  
Katja Weisel ◽  
Walter Fiedler ◽  
...  

High dose chemotherapy (HDT) followed by autologous peripheral blood stem cell transplantation (ASCT) is standard of care including a curative treatment option for several cancers. While much is known about the management of patients with allogenic SCT at the intensive care unit (ICU), data regarding incidence, clinical impact, and outcome of critical illness following ASCT are less reported. This study included 256 patients with different cancer entities. Median age was 56 years (interquartile ranges (IQR): 45–64), and 67% were male. One-year survival was 89%; 15 patients (6%) required treatment at the ICU following HDT. The main reason for ICU admission was septic shock (80%) with the predominant focus being the respiratory tract (53%). Three patients died, twelve recovered, and six (40%) were alive at one-year, resulting in an immediate treatment-related mortality of 1.2%. Independent risk factors for ICU admission were age (odds ratio (OR) 1.05; 95% confidence interval (CI) 1.00–1.09; p = 0.043), duration of aplasia (OR: 1.37; CI: 1.07–1.75; p = 0.013), and Charlson comorbidity score (OR: 1.64; CI: 1.20–2.23; p = 0.002). HDT followed by ASCT performed at an experienced centre is generally associated with a low risk for treatment related mortality. ICU treatment is warranted mainly due to infectious complications and has a strong positive impact on intermediate-term survival.


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