OBJECTIVETo 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).

METHODSThirty-eight patients diagnosed from September 2004 to September 2012 were analyzed retrospectively, including 15 cases received auto-PBSCT, 23 cases exposed with VD.

RESULTSThe 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).

CONCLUSIONSThe 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.

Amyloidosis ; therapy ; Bortezomib ; therapeutic use ; Dexamethasone ; therapeutic use ; Humans ; Immunoglobulin Light-chain Amyloidosis ; Melphalan ; therapeutic use ; Myeloablative Agonists ; therapeutic use ; Peripheral Blood Stem Cell Transplantation ; Retrospective Studies

An 8-year-old male Austrian Pinscher and a 14-year-old male Golden Retriever were presented for evaluation due to unexplainable high fructosamine values despite euglycemia and epistaxis in combination with polydipsia/polyuria, respectively. Blood analysis revealed severe hyperglobulinemia, hypoalbuminemia and markedly elevated fructosamine concentrations in both dogs. Multiple myeloma with IgA-monoclonal gammopathy was diagnosed by serum and urine electrophoresis including immunodetection with an anti-dog IgA antibody and bone marrow aspirations. Diabetes mellitus was excluded by repeated plasma and urine glucose measurements. Fructosamine values were positively correlated with globulin, but negatively correlated with albumin concentrations. These cases suggest that, as in human patients, monoclonal IgA gammopathy should be considered as a possible differential diagnosis for dogs with high fructosamine concentrations.
Animals ; Blood Proteins/analysis ; Dog Diseases/*blood/drug therapy ; Dogs ; Fructosamine/*blood ; Immunoglobulin A/*metabolism ; Male ; Melphalan/therapeutic use ; Multiple Myeloma/complications/drug therapy/*veterinary ; Myeloablative Agonists/therapeutic use ; Paraproteinemias/blood/complications/drug therapy/*veterinary

Autologous stem cell transplantation (ASCT) for the treatment of high-risk neuroblastoma (NBL) is an accepted method for restoring bone marrow depression after high dose chemotherapy. We retrospectively analyzed eighty eight cases of NBL that underwent ASCT following marrow ablative therapy at 12 transplant centers of the Korean Society of Pediatric Hematology-Oncology between January 1996 and September 2000. Seventy nine children were of stage IV NBL and 9 were of stage III with N-myc amplification. Various cytoreductive regimens were used. However, the main regimen was 'CEM' consisting of carboplatin, etoposide and melphalan, and this was used in 66 patients. Total body irradiation was also added in 36 patients for myeloablation. To reduce tumor cell contamination, stem cell infusions after CD34+ cell selection were performed in 16 patients. Post-transplantation therapies included the second transplantation in 18 patients, interleukin2 therapy in 45, 13-cis retinoic acid in 40, 131-meta-iodobenzylguanidine in 4, conventional chemotherapy in 11, and local radiotherapy in 8. Twenty two patients died, sixty six patients are surviving 1 to 46 months after ASCT (median followup duration, 14.5 months). Although the follow-up period was short and the number of patients small, we believe that ASCT might improve the survival rate in high-risk NBL.
Adolescent ; Child ; Child, Preschool ; Combined Modality Therapy ; Female ; Human ; Korea ; Male ; Myeloablative Agonists/therapeutic use ; Neuroblastoma/mortality ; Neuroblastoma/pathology ; Neuroblastoma/therapy* ; Retrospective Studies ; Stem Cell Transplantation* ; Survival Rate ; Transplantation Conditioning ; Transplantation, Autologous ; Treatment Outcome

The aim of this study was to evaluate the efficacy of palifermin, an N-terminal truncated version of endogenous keratinocyte growth factor, in the control of oral mucositis during antiblastic therapy. Twenty patients undergoing allogeneic stem-cell transplantation for acute lymphoblastic leukaemia were treated with palifermin, and compared to a control group with the same number of subjects and similar inclusion criteria. Statistical analysis were performed to compare the outcomes in the treatment vs. control groups. In the treatment group, we found a statistically significant reduction in the duration of parenteral nutrition (P=0.002), duration of mucositis (P=0.003) and the average grade of mucositis (P=0.03). The statistical analysis showed that the drug was able to decrease the severity of mucositis. These data, although preliminary, suggest that palifermin could be a valid therapeutic adjuvant to improve the quality of life of patients suffering from leukaemia.
Adolescent ; Allografts ; transplantation ; Anti-Inflammatory Agents ; therapeutic use ; Case-Control Studies ; Child ; Cohort Studies ; Cyclophosphamide ; therapeutic use ; Female ; Fibroblast Growth Factor 7 ; therapeutic use ; Hematopoietic Stem Cell Transplantation ; methods ; Humans ; Male ; Myeloablative Agonists ; therapeutic use ; Parenteral Nutrition ; Precursor Cell Lymphoblastic Leukemia-Lymphoma ; therapy ; Radiotherapy Dosage ; Stomatitis ; classification ; drug therapy ; prevention & control ; Time Factors ; Transplantation Conditioning ; methods ; Treatment Outcome ; Whole-Body Irradiation

BACKGROUND/AIMS: This retrospective study evaluated the transplantation outcomes of patients with adult lymphoid malignancies who received chemotherapy-based conditioning with busulfan and fludarabine (BuFlu) and busulfan and cyclophosphamide (BuCy2). METHODS: Thirty-eight patients (34 with acute lymphoblastic leukemia and 4 with lymphoblastic lymphoma) were included in the current study. The conditioning regimen was BuCy2 for 14 patients and BuFlu for the remaining 24 patients. Eight and 13 patients were high risk disease in the BuCy2 and BuFlu groups, respectively. RESULTS: The cumulative incidence of grade II-IV acute graft-versus-host disease (GVHD) was 56.5% and 55.2% and that of extensive chronic GVHD 17.0% and 55.6% (p = 0.018) for the BuFlu and BuCy2 groups, respectively. The 3-year relapse rate was 27.8% and 31.4% and 3-year overall survival 34.3% and 46.8% for the BuFlu and BuCy2 groups, respectively. Treatment-related mortality (TRM) was significantly lower in the BuFlu group (16.9%) than in the BuCy2 group (57.1%, p = 0.010). In multivariate analyses, the BuFlu regimen was identified as an independent favorable risk factor for TRM (hazard ratio [HR], 0.036; p = 0.017) and extensive chronic GVHD (HR, 0.168; p = 0.034). CONCLUSIONS: Our BuFlu regimen would appear to be an acceptable conditioning option for lymphoid malignancies, including high-risk diseases. It was safely administered with a lower TRM rate than BuCy2 conditioning.
Adolescent ; Adult ; Busulfan/adverse effects/*therapeutic use ; Chi-Square Distribution ; Disease-Free Survival ; Drug Therapy, Combination ; Feasibility Studies ; Female ; Graft vs Host Disease/etiology ; Humans ; Kaplan-Meier Estimate ; Male ; Middle Aged ; Multivariate Analysis ; Myeloablative Agonists/adverse effects/*therapeutic use ; Precursor Cell Lymphoblastic Leukemia-Lymphoma/drug therapy/mortality/surgery/*therapy ; Proportional Hazards Models ; Republic of Korea ; Retrospective Studies ; Risk Assessment ; Risk Factors ; *Stem Cell Transplantation/adverse effects/mortality ; Time Factors ; Transplantation Conditioning/adverse effects/*methods/mortality ; Transplantation, Homologous ; Treatment Outcome ; Vidarabine/adverse effects/*analogs & derivatives/therapeutic use ; Young Adult