Comparison analysis of outcomes in primary light chain amyloidosis patients treated by auto peripheral blood stem cell transplantation or bortezomib plus dexamethasone / 中华血液学杂志

<p><b>OBJECTIVE</b>To explore the feature of primary light chain amyloidosis patients treated with high-dose melphalan with auto peripheral blood stem cell transplantation (auto-PBSCT) and bortezomib plus dexamethasone (VD).</p><p><b>METHODS</b>Thirty-eight patients diagnosed from September 2004 to September 2012 were analyzed retrospectively, including 15 cases received auto-PBSCT, 23 cases exposed with VD.</p><p><b>RESULTS</b>The median follow-up duration for the patients was 34 months (range, 1-112 months), including auto-PBSCT group of 38 months (range, 5-112 months) and VD group of 31 months (range, 1-108 months). The organ response rate in all the patients was 39.5% (15/38), and the organ response rate between these two groups has no significant difference [33.3% (5/15) vs 43.5% (10/23), P=0.532]. However, the median time of organ response was significant difference [6 (3-10) months vs 3 (1-6) months, respectively (P=0.032)]. The 3-year overall survival (OS) rates in the two groups were 72.0% and 66.9%, and their average survival were 84.7 months and 75.9 months, respectively (P=0.683). In the patients with auto-PBSCT, the occurrence of III-IV grade of bone marrow suppression (P<0.001), fever (P<0.001), nausea and infection (P=0.006) were obviously higher than those with VD, but there was no statistically significant difference in pulmonary infection (P=0.069) and bloodstream infection (P=0.059).</p><p><b>CONCLUSIONS</b>The preliminary results have presented that primary light chain amyloidosis patients treated with auto-PBSCT or VD had similar organ response rate and survival. However, more adverse events occurred in the group of auto-PBSCT.</p>

Autologous peripheral blood CD34+ stem cells transplanted into 100 patients with advanced cirrhosis / 中华肝脏病杂志

<p><b>OBJECTIVE</b>To investigate whether transplantation of autologous peripheral blood CD34+ stem cells is a viable approach for treating patients with advanced cirrhosis,which is currently hindered by a shortage in liver donors.</p><p><b>METHODS</b>A total of 100 patients with advanced cirrhosis and who had failed to respond to conservative therapy were recruited for transplantation of autologous peripheral blood CD34+ stem cells.The success of transplantation was investigated 6-and 12-months later by measuring markers of liver biosynthesis function (coagulation,albumin level,indocyanine green clearance,Child-Pugh score) and assessing pathological changes (Knodell score) and morphologic changes in the liver tissue.Complications were also recorded during follow-up.</p><p><b>RESULTS</b>The 1-year cumulative survival rate was 100%. Fifty-two patients with massive ascites showed gradual reduction and disappearance of the ascites.Four patients experienced upper gastrointestinal bleeding and three patients developed with hepatic encephalopathy (I-II degree) at 3 months post-transplantation.All patients showed significantly improved liver biosynthesis function,liver elasticity and Knodell score after transplantation (P less than 0.05).</p><p><b>CONCLUSION</b>Autologous peripheral blood CD34+ stem cell transplantation is a safe and effective treatment for advanced cirrhosis,and has high cost-benefit since it improves liver function,liver histology,and quality of life.</p>

The clinical significance of serum free light chain in primary systemic amyloidosis / 中华内科杂志

Objective To evaluate the diagnostic and therapeutic significance of serum free light chain (sFLC) in primary systemic(AL) amyloidosis. Methods Twenty-five patients with AL amyloidosis,including 18 men and 7 women with a mean age of 54(47-77) years old, were enrolled from October, 2005to May, 2010. sFLC was measured by immunoturbidimetric assay. The type of monoclonal light chain was judged upon sFLC κ/λ and its sensibility was compared with serum immunofixation and immunohistochemical analysis. Four patients were treated with M (T)D (melphalan/thalidomideand, dexamethasone), one with VD (velcade and dexamethasone) and four with high-dose melphalan followed by autologous stem cell support. The changes of sFLC were serially determined before and after treatment. Results Among the 25 patients with AL amyloidosis, two were κ light chains of precursor protein and 23 were λ light chains. Mean plasma cell in bone marrow was 3.5% (0-15%). Nineteen (76%) patients had abnormal elevated sFLC and abnormal κ/λ ratios, and 17(68% ) patients with immunofixation positive. The sFLC test had similar sensitivity as serum immunofixation (P = 0. 727 ). Twenty-one (84%) patients were shown to have either κor λ immunoreactive amyloid deposits on biopsied tissues. The sFLC test combined with serum immunofixation allowed the M protein to be detected in 22 (88%) patients. The positive rates of immunohistochemical analysis combined with sFLC test and/or serum immunofixation were 96%. Four patients with hematologic response showed obvious improvement in visceral organ involvement, but illness of 5 patients without hematologic response kept stable or progressed. Conclusions sFLC test is a sensitive qualitative and quantitative method to detect M protein. Preliminary data show the patients with obvious sFLC level decrease and/or κ/λ recovery to normal may have a high percentage of improved organs function. sFLC is critical index in diagnosing AL amyloidosis, which might help efficacy assessment.