scholarly journals BEAM Vs Cyclophosphamide-Based Conditioning Regimen in Aggressive Multiple Sclerosis: A Retrospective Analysis of European Blood and Marrow Transplantation Society

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3313-3313
Author(s):  
Riccardo Saccardi ◽  
Manuela Badoglio ◽  
Joachim Burman ◽  
Grzegorz Helbig ◽  
Majid A. Kazmi ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Retrospective Analysis ◽  
Conditioning Regimen ◽  
Working Party ◽  
High Rate ◽  
Hematological Toxicity ◽  
Significant Difference ◽  
Conditioning Regimens ◽  
The Impact ◽  
Autologous Hsct

Background Multiple Sclerosis (MS) is a chronic, immuno-mediated disease of Central Nervous System (CNS), mostly affecting young adults and frequently resulting in a progressive, irreversible disability despite the administration of approved Disease Modifying Treatments (DMTs). Autologous HSCT was shown to induce a high rate of sustained, treatment-free remissions in cases of aggressive MS, seldom associated to a partial reversal of disability. Toxicity of Conditioning Regimen is still a major concern. We retrospectively analyzed the outcome of 926 MS patients reported to the EBMT Registry who underwent autologous HSCT following the two most frequent CRs for this indication in the last 20 years. Patients and Methods Patient data were extracted from both the EBMT database and a disease-specific database developed by the EBMT Autoimmune Diseases Working Party (ADWP). Patients were selected for having received either BEAM + ATG (BEAM) or HD-Cyclophosphamide + ATG (CYC) as conditioning regimen. Hematological toxicity was assessed through Neutrophil (PMN) engraftment and 100-days (early) mortality (eTRM). MS forms at HSCT were reported as Relapsing-Remitting (RR), Secondary Progressive (SP), Primary Progressive (PP) and Progressive-Relapsing (PR). The impact of variables related to both patients (age, gender, year of HSCT, EDSS at HSCT) and disease characteristics (MS form, interval diagnosis-HSCT) at HSCT in the two groups were also evaluated. Results The utilization of conditioning regimens along the observed time period (1998-2018) was variable, with an increase of the HSCT activity in general after 2010 (230 vs 697 procedures) and a prevalence of BEAM before 2010 (205 BEAM vs 25 CYC) and of CYC thereafter (205 BEAM vs 492 CYC, p=0.001). Also, RR forms of MS prevailed over Progressive forms after 2010 (p=0.001) which is reflected in the different distribution across the two regimens, with RR significantly more frequently treated with CYC-based regimen (p<0.001). Gender distribution and age at HSCT was similar in the two groups (p=ns), whilst the interval between diagnosis and HSCT was longer in BEAM group than CYC (8.47 years ± 5.9 vs 7.57 ±5.5, mean ± SD, p=0.012). PMN engraftment in BEAM/ATG- and CYC/ATG-treated patients occurred at +11.0 (8-42) and +10.9 (8-95) days, respectively (median and range, p=ns). Overall eTRM was low (1.4%), but slightly higher in BEAM over CYC (8/402, 2% vs 5/517, 1%, p=ns). Discussion Although autologous HSCT is increasingly used as a treatment in highly active MS, toxicity remains a principal concern in the neurological community despite a marked decrease of TRM over time. The intensity of conditioning regimens has varied in the literature, but the best toxicity/efficacy ratio remains unclear. The non-myeloablative regimen CYC-ATG has become the most common conditioning regimen despite a lack of comparative data with more intense regimens. In our large retrospective analysis of the two most frequent conditioning regimens in the EBMT Registry, there was no significant difference in major toxicity indicators despite differences in chronological period and patients characteristics in the two groups. Comparative analysis of neurological efficacy is currently ongoing and will inform the toxicity/efficacy ratio and clinical choice of conditioning regimen in autologous HSCT in MS. Disclosures Mielke: Bellicum: Consultancy, Honoraria, Other: Travel (via institution); Jazz Pharma: Honoraria, Other: Travel support, Speakers Bureau; IACH: Other: Travel support; Kiadis Pharma: Consultancy, Honoraria, Other: Travel support (via institution), Speakers Bureau; Miltenyi: Consultancy, Honoraria, Other: Travel and speakers fee (via institution), Speakers Bureau; DGHO: Other: Travel support; GILEAD: Consultancy, Honoraria, Other: travel (via institution), Speakers Bureau; Celgene: Honoraria, Other: Travel support (via institution), Speakers Bureau; ISCT: Other: Travel support; EBMT/EHA: Other: Travel support.

Antithymocyte Globuline Given as Part of the Conditioning Regimen Has No Impact On Lung Function at 1 Year After Transplantation.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 3306-3306
Author(s):  
Filippo Milano ◽  
Margaret A. Au ◽  
H. J. Deeg ◽  
Jason Chien
Keyword(s):  
Stem Cell ◽  
Lung Function ◽  
Pulmonary Function ◽  
Retrospective Analysis ◽  
Conditioning Regimen ◽  
Hematopoietic Stem ◽  
Lung Dysfunction ◽  
Significant Difference ◽  
The Mean ◽  
The Impact

Abstract Abstract 3306 Poster Board III-194 Background Pulmonary dysfunction is common following allogeneic hematopoietic stem cell transplantation (HSCT) and is associated with significant morbidity and mortality. The use of Antithymocyte globuline (ATG) has reduced the incidence of graft versus host disease (GVHD), particularly in its chronic form, but the impact of this approach on the prevention of lung dysfunction is not well characterized. Methods We performed a retrospective analysis of pulmonary function in all patients transplanted from January 2001 through December 2005 following conditioning with oral busulfan (BU) followed by either cyclophosphamide (CY) or fludarabine (FLU) with or without the addition of ATG. Pulmonary function tests (PFTs) were performed per transplant protocol before and around day 80 and 1 year after transplantation. Results Of 406 patients, 13 (3%) were excluded due to lack of pre-transplant PFTs. Seventy five patients received ATG (thymoglobulin, given on days -3 to -1 for total doses of 4.5 – 6 mg/kg), and 318 did not. The median patient age was 49 years for both the ATG (range 9-65) and non-ATG (range 7-66) groups; the median follow-up was 956 (range 19-2071) days and 903 (range 8-2697) days, respectively. The ATG group had proportionally more patients with high risk diseases (62.7% vs 45.7%, p= .03), whereas the use of bone marrow as stem cell source was higher in the non-ATG group (14.6% vs 5.3%, p=.03). The proportion of patients with PFTs at 1 year was similar for both groups (49.3% vs 54.6%, p=.55). No significant statistical differences between the 2 groups were seen in the mean percentage of the predicted values for FEV1, FVC, TLC and DLco at 80 days or 1 year after transplantation. The mean value of FEV1/FVC ratio at 1 year was higher for ATG patients (0.81±0.05 vs 0.77±0.09, p=.003). The mean change in PFT parameters from baseline to 1 year after HSCT also did not show a statistically significant difference between the 2 groups for any PFT parameters except for the FEV1/FVC ratio, which decline was lower in the ATG group (0.4±3.9 vs -2.8±7.3 p=.0002). This difference remained significant in multivariate analysis. The lung function score at 1 year after transplantation was similar in the 2 groups; they also had similar rates of lung function decline (expressed as the annualized rate of FEV1 decline) from baseline to 1 year after HSCT. The risk of developing severe airflow obstruction (AFO) or a restrictive pattern (RP), as well as the cumulative incidence of AFO and RP were not statistically significantly different between the 2 treatment groups at 1 year after HSCT. Conclusions In this retrospective analysis, incorporation of ATG into the HSCT conditioning regimen did not appear to be associated with superior outcome in terms of post transplant pulmonary function. However, further evaluations are needed to better clarify the role that ATG might have in the development of late-term pulmonary complications. Disclosures No relevant conflicts of interest to declare.

Evaluation of Enteral Versus Parenteral Feeding As Nutritional Support In Allogeneic Hematopoietic Stem Cell Transplantation

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4576-4576
Author(s):  
Richard Lemal ◽  
Romain Guièze ◽  
Cécile Moluçon-Chabrot ◽  
Eric Hermet ◽  
Aurélie Ravinet ◽  
...  
Keyword(s):  
Stem Cell ◽  
Median Duration ◽  
Nutritional Support ◽  
Conflicts Of Interest ◽  
Conditioning Regimen ◽  
Hematopoietic Stem ◽  
Early Outcome ◽  
Transfusion Requirements ◽  
Significant Difference ◽  
The Impact

Abstract Abstract 4576 Introduction Allo-HSCT procedure is associated with a frequent and potentially severe malnutrition which could highly participate to the transplant-related morbidity (TRM). Optimal nutritional management is still poorly known while both enteral nutrition (EN) and parenteral nutrition (PN) are effective. We proposed to retrospectively evaluate the impact of EN versus PN as nutritional support on early outcome of allo-HSCT. Patients and methods We retrospectively analyzed all the successive patients who needed a nutritional support during their first allo-HSCT in our center from January 2009 to October 2010, excepting whose who had a progressive disease at time of transplant. Datas were compared in an intent to treat analysis according the EN or PN initial nutritional support strategy. Results We analysed early outcome of 56 successive patients. Twenty of them received a myeloablative conditioning regimen and 36 a reduced intensity one. A total of 28 agreed to receive EN via a nasogastric feeding tube and the remaining 28 received PN. No significant difference in terms of age, diagnosis, disease status at transplant, conditioning regimens, stem cell source, GVHD nor antifungal secondary prophylaxis could be observed between the EN and PN groups. We found a lower median duration of fever in EN (2[0–8] vs. 5[0–17] (days); p=0.0026) and a lower need for antifungal therapy in EN group (7/28 vs. 17/28; p=0.0069), with a lower median duration of intravenous antifungal use (0 day [0–99] in EN vs. 7 days [0–93] in PN; p=0.00034) while incidence of bacteriemiae was not different. We observed a lower rate of replacement of central veinous catheter in EN group (3/28 in EN vs. 9/28 in PN; p=0.05) and a lower rate of transfer to ICU in the EN group (2/28 in EN vs. 8/28 in PN, p=0.036) but early death rate (<100 days) was the same in each group (4/28 vs. 4/28, p=NS). Median neutropenia and thrombopenia duration and median transfusion requirements were not significantly different. Fourteen patients in EN group and 18 in PN group presented a grade 3–4 oral mucositis (p=NS). Grade III-IV GVHD incidence was comparable in both groups (4/28 vs. 8/28; p=0.19). Conclusion Compared with PN, EN directly decreases the infectious risk, particularly the fungal risk, and its complications in allo-HSCT, without influencing hematopoietic toxicity nor GVHD incidence. Based on these encouraging results, we are now conducting a prospective, multicentric and randomized trial to confirm EN benefice in allo-HSCT. Disclosures: No relevant conflicts of interest to declare.

Alkylator-Based Conditioning for Allogeneic Hematopoietic Stem Cell Transplantation (alloHSCT) in Lymphoma: A Registry Study Comparing Thiotepa-, Busulfan-, Melphalan- and Treosulfan-Containing Regimens On Behalf of the EBMT Lymphoma Working Party

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 2033-2033
Author(s):  
Peter Dreger ◽  
Herve Finel ◽  
Renato Fanin ◽  
Paolo Corradini ◽  
Michele Falda ◽  
...  
Keyword(s):  
Cell Lymphoma ◽  
Performance Status ◽  
Conditioning Regimen ◽  
Working Party ◽  
B Cell Lymphoma ◽  
Poor Performance Status ◽  
Unrelated Donor ◽  
Hematopoietic Stem ◽  
Remission Status ◽  
Significant Difference

Abstract Abstract 2033 Background: Thiotepa (TT) is an alkylating agent approved for conditioning for alloHSCT. TT-based alloHSCT has been pioneered in a variety of lymphoma subtypes with promising results, but the available information about the value of thiotepa in this indication compared to other alkylator-based regimens is still limited. Primary objective was to compare the outcome of TT-based alloHSCT with that of alloHSCT conditioned with other alkylator regimens (non-TT) separately for diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL), and peripheral T Cell lymphoma (PTCL). Primary endpoint was event-free survival (EFS); secondary endpoints were overall survival, non-relapse mortality (NRM), and relapse incidence. Eligible were patients >18 years who had received TT-, busulfan (BU)-, melphalan- (MEL), or treosulfan- (TREO) based conditioning for T-replete alloHSCT between 2003–2010 for FL, DLBCL, or PTCL. Statistical analysis was based on multivariable comparisons using stratified Cox and Fine & Gray regression models. Results: 201 patients with TT fulfilled the inclusion criteria and were compared with 578 non-TT patients (BU 55%, MEL 35%, TREO 10%). The most frequently used specific regimens were TT-cyclophosphamide combinations (75%) in the TT group and BU-fludarabine combinations (52%) in the non-TT group. Of the total 779 patients, 43%% had FL, 39% DLBCL, and 18% PTCL. TT and non-TT patients were comparable for age, sex, time from diagnosis, remission status at HSCT, and proportion of unrelated donor transplants. However, the TT group contained significantly more patients with PTCL (24% vs 15%), with poor performance status (PS; 12.5% vs 3%), and with BM as HSCT source (14% vs 9%). By multivariate comparisons considering conditioning, age, sex, remission status, PS, and time from diagnosis, EFS was significantly affected by active disease at HSCT and poor PS in all three lymphoma subtypes. In contrast, conditioning with TT had no significant impact (Hazard ratio (HR) 1.06 (0.67–1.67) for FL; 1.01 (0.67–1.51) for DLBCL; 1.33 (0.75–2.36 for PTCL) on EFS or any other endpoint. Similar results were seen when the analysis was broken down to specific conditioning regimens (TT-CY vs BU-based). MEL- and TREO-based regimens did not show a significant difference compared to BU for any endpoint. Conclusions: This study failed to identify significant outcome differences between the four conditioning regimen types tested. However, the limitations inherent to registry analyses have to be considered. In particular, conclusions on differential regimen toxicity apart from NRM will require additional, ideally prospective studies. Disclosures: Dreger: Riemser G, Greifswald, Germany: Consultancy, Honoraria, Research Funding. Off Label Use: Treosulfan for conditioning for allogeneic HSCT. Schmitz:Riemser AG, Greifswald, Germany: Honoraria.

An updated overall survival analysis with correction for protocol-planned crossover of the international, phase III, randomized, placebo-controlled trial of regorafenib in advanced gastrointestinal stromal tumors after failure of imatinib and sunitinib (GRID).

2015 ◽  
Vol 33 (3_suppl) ◽  
pp. 110-110 ◽  
Author(s):  
George D. Demetri ◽  
Peter Reichardt ◽  
Yoon-Koo Kang ◽  
Jean-Yves Blay ◽  
Heikki Joensuu ◽  
...  
Keyword(s):  
Overall Survival ◽  
Failure Time ◽  
Cox Model ◽  
Controlled Trial ◽  
Progression Free Survival ◽  
High Rate ◽  
Phase Iii ◽  
Primary Analysis ◽  
Significant Difference ◽  
The Impact

110 Background: The GRID study showed that regorafenib improves progression-free survival compared with placebo in patients with advanced GIST after failure of at least imatinib and sunitinib (HR 0.27; 1-sided p<0.0001; Demetri 2013). At the time of the primary analysis, no significant difference in the secondary endpoint of overall survival (OS) was observed (HR 0.77; p=0.199), but this result may have been confounded by the high rate of crossover to regorafenib (85%) of placebo patients at progression. We conducted exploratory analyses of updated OS data to assess the effect of correcting for this protocol-planned crossover. Methods: The data cut-off for this updated OS analysis was 31 January 2014 (2 years after the primary analysis). OS was corrected using two randomization-based methods: rank preserving structural failure time (RPSFT) and iterative parameter estimation (IPE); both methods are considered as best choice among all correction analytics. Hazard ratios and 95% CI were derived using the Cox model. Results: A total of 139 deaths had occurred at the time of data cut-off: 91 events (68.4% of patients) in the regorafenib group and 48 (72.7%) in the placebo group. A total of 22 patients remained on regorafenib treatment (median duration 2.1 years, range 0.9–2.4). The updated hazard ratio for OS favored regorafenib (0.85, 95% CI: 0.60 - 1.21; p=0.18). Median OS was estimated as 17.4 months in both groups, with crossover from placebo. The corrected HRs for OS are less than the uncorrected HR (Table). Conclusions: The updated analysis of OS in the GRID trial is consistent with the primary analysis. An exploratory analysis correcting for the impact of cross-over on OS suggests a survival benefit for regorafenib in GIST. Clinical trial information: NCT01271712. [Table: see text]

scholarly journals Immunophenotypic Response After Allografting In Multiple Myeloma

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 3371-3371 ◽  
Author(s):  
Luisa Giaccone ◽  
Lucia Brunello ◽  
Roberto Passera ◽  
Moreno Festuccia ◽  
Milena Gilestro ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Bone Marrow ◽  
Flow Cytometry ◽  
Conflicts Of Interest ◽  
Univariate Analysis ◽  
Conditioning Regimen ◽  
First Line ◽  
Significant Difference ◽  
The Impact

Abstract Background Minimal residual disease (MRD) by multiparameter flow-cytometry recently showed a promising role in predicting outcomes in patients with multiple myeloma. However, data on immunophenotypic response (IR) after allografting are lacking. Aim To evaluate the impact of IR and compare it to conventional complete remission (CR) following allografting in myeloma patients. Methods Sixty-six consecutive patients, median age 54 years (35-66), who underwent an allograft between January 2000 and December 2011 with a follow-up of at least 3 months were included. Disease response was evaluated by serum and urine electrophoresis, and bone marrow aspirate at baseline, 3, 6, 12, 18, 24 months after transplant and yearly thereafter. Skeletal survey or MRI were performed yearly or as clinically indicated (overt relapse or complaints of bone pain). Bone marrow aspirates had to contain at least 13000 cells/µL for flow-cytometry studies and IR was defined as absence of monoclonal plasma-cells detected by 4 or 6-colour staining with the following antibodies: CD38, CD138, CD56, CD19, CD45, cyKappa, cyLambda. CR was defined according to standard criteria (Durie et al, Leukemia 2006; 20:1467-73). Results Conditioning regimen was non-myeloablative 2Gy TBI-based in 55 patients, reduced intensity (fludarabine-melphalan-based) in 10 and myeloablative in 1 patient. Post-grafting immunosuppression consisted of cyclosporine with mycophenolate mofetil or methotrexate. Donors were HLA identical siblings in 58 patients and unrelated in 8. Only 1 patient received bone marrow as source of stem cells. Thirty-five/66 (53%) received the allograft as part of the first line treatment, whereas the remaining 31/66, (47%) were transplanted at relapse. At the time of transplant, 5/66 were both in IR and CR, 16 were only in IR and 4 patients were only in clinical CR. All 21 patients in IR at the time of transplant maintained it, while 26/45 (58%) entered IR after the allograft. Among patients surviving at least 3 months, overall treatment related mortality was 10.6% at 3 years. After a median follow-up of 69 months (range 19-147), the incidence of acute and chronic graft-versus-host disease was 45.6% and 49.3% without significant difference between responsive and non-responsive patients. At follow-up, overall, 24 patients achieved CR and IR (CR/IR group), 21 achieved IR but not CR because of persistence of urine/serum M-component (noCR/IR group), and 21 did not achieve either CR or IR (noCR/noIR group). Interestingly, none achieved CR without IR. Median overall survival (OS) and event-free survival (EFS) in patients who achieved IR were 96 and 55 months versus 36 and 7 months in those who did not (p<0.001). Median OS and EFS were not reached and 59 months in the CR/IR group, 77 and 15 months in the noCR/IR, and 30 and 5 months in the noCR/noIR respectively (p<0.001 for both EFS and OS-fig.1). In univariate analysis, being in the CR/IR group was the only significant predictor for prolonged OS and EFS (p<0.001). Of note, cumulative incidence of extra-medullary disease at first relapse after the allograft was 4% in the CR/IR, 32% in the noCR/IR and 15% in the noCR/noIR groups respectively (p<0.001). Receiving the allograft as first line therapy or later during the disease course did not significantly impact on OS and EFS. Conclusion The achievement of IR confers a favorable impact on OS and EFS after allografting. A higher incidence of extra-medullary in the noCR/IR group (some 30% of our patient cohort) may suggest that myeloma cells escape immune control outside the bone marrow. In this group, imaging studies such as positron emission tomography may clinically be indicated during follow-up to detect early relapse. Disclosures: No relevant conflicts of interest to declare.

Myeloablative Versus Nonmyeloablative Hemopoietic Cell Transplantation (HCT) for Patients with Myelodysplasia (MDS) or AML with Multilineage Dysplasia Following MDS (tAML).

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 2320-2320
Author(s):  
Bart L. Scott ◽  
Michael Maris ◽  
Brenda Sandmaier ◽  
Barry Storer ◽  
Thomas Chauncey ◽  
...  
Keyword(s):  
High Risk ◽  
Retrospective Review ◽  
Conditioning Regimen ◽  
Refractory Anemia ◽  
High Risk Disease ◽  
Significant Difference ◽  
Unrelated Donors ◽  
Conditioning Regimens ◽  
Conditioning Intensity ◽  
Cmv Status

Abstract The median age at diagnosis of MDS is 72 years (yrs), and conventional HCT is not an option for many patients (pts). The primary benefit of nonmyeloablative conditioning regimens is a reduction in acute post-HCT morbidity and mortality. However, this benefit may be offset by an increase in post-HCT relapse. To address these issues, we performed a retrospective review of data from 172 pts over age 40 yrs with a diagnosis of MDS or tAML as determined by WHO criteria who underwent HCT between January 1998 and December 2003. Data were analyzed as of July 28, 2004. One hundred thirty two pts were conditioned with a myeloablative regimen of targeted (800–900 ng/ml) busulfan (starting dose 1mg/kg every 6 hours for 16 doses) and cyclophosphamide (120mg/kg) with or without thymoglobulin. Forty pts selected for a nonmyeloablative regimen because of age or comorbid illnesses were conditioned with 2 Gy TBI with or without fludarabine, 30mg/m2/day for three days. The median age was 52 (40–65) yrs for the myeloablative group, and 62 (40–73) yrs for the nonmyeloablative group. The majority of pts in the nonmyeloablative group had progressed to tAML (55%) and had high risk disease by IPSS (55%). The WHO distribution (highest at any time) was as follows: myeloablative/nonmyeloablative- 34%/55% with tAML, 29%/22% with refractory anemia with excess blasts (RAEB-1/2), and 37%/22% with refractory anemia with or without ringed sideroblasts (RA/RARS). The IPSS distribution (highest at any time) was as follows: myeloablative/nonmyeloablative-26%/55% high, 23%/30% int-2, 41%/15% int-1, and 11%/0% low risk. All pts received HCT from HLA-matched related (50% of myeloablative, 65% of nonmyeloablative) or unrelated donors. There were no differences between the two groups with regards to gender distribution, donor CMV status, recipient CMV status, duration of disease prior to HCT, primary or secondary etiology of MDS, or source of stem cells. Overall survival (OS) [46%/31%], relapse free survival (RFS) [40%/28%], and non-relapse mortality [37%/37%] did not differ significantly between myeloablative/nonmyeloablative pts. There was a trend towards improved RFS in pts with RA/RARS (p=0.16) who received myeloablative HCT, and a trend towards improved RFS in pts with tAML who received nonmyeloablative HCT (p=0.14). Among pts with more advanced disease (RAEB-1/2 or tAML) given myeloablative/nonmyeloablative HCT there were 39%/87% who received induction chemotherapy (IC) pre-HCT and 59%/85% given IC achieved a CR. Post-HCT OS and RFS were similar for pts who achieved CR following IC regardless of conditioning regimen used. Thus, while there was a trend towards superior survival with myeloablative HCT in pts with RA/RARS and with nonmyeloablative HCT in pts with tAML, in this retrospective review there were no significant differences between results obtained with myeloablative and nonmyeloablative conditioning regimens. Furthermore, even though most nonmyeloablative pts had high risk disease, there was no significant difference in RFS between the two groups suggesting that a graft vs. tumor effect was more important than conditioning intensity in preventing relapse in pts with high risk MDS or tAML in CR after IC. This raises the question of whether nonmyeloablative HCT should be considered in all pts with high risk MDS or tAML who achieve a CR with IC.

Combination of Rituximab with Initial Chemotherapy Improves Outcome of Primary Mediastinal B-Cell Lymphoma: A Retrospective Analysis of a Single Institution Cohort

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 1283-1283 ◽  
Author(s):  
Abraham Avigdor ◽  
Tsvi Sirotkin ◽  
Noga Shemtov ◽  
Miriam Berkowicz ◽  
Yaron Davidovitz ◽  
...  
Keyword(s):  
B Cell ◽  
Retrospective Analysis ◽  
Predictive Value ◽  
Cell Lymphoma ◽  
Univariate Analysis ◽  
B Cell Lymphoma ◽  
Historical Comparison ◽  
Pet Ct ◽  
Significant Difference ◽  
The Impact

Abstract Primary mediastinal B-cell lymphoma (PMBCL) is a relatively rare clinico-pathologic subtype of diffuse large B-cell lymohoma. The optimal management, the prognostic factors and the role of PET/CT scan in this entity remain a matter of debate. While several retrospective studies suggested that dose-dense regimens are more effective than standard CHOP, the impact of adding rituximab (R) on the outcome of patients (pts) with PMBCL has not been fully evaluated. In this retrospective analysis we reviewed the clinical and radiological records of 81 consecutive pts with PMBCL treated in Sheba Medical Center between August 1985 and October 2006. Chemotherapy in the pre-rituximab era (−R cohort) included VACOPB (n=47) or 6 courses of standard CHOP (n=5). Since October 2002, 6 cycles of R were added concurrent with the treatment in another 29 pts (+R cohort): R-VACOPB (n=21) and R-CHOP (n=8). Radiotherapy was not administered following initial chemotherapy to any of the pts. Median age at diagnosis was 31 years (yrs) (range 17–61). Stage I/II and bulky mediastinum (≥ 10 cm in diameter) were present in 88% and 33%, respectively, and extranodal involvement was evident in 44% of all pts. After a median follow-up of 85 months (range 9–240), the overall (OS) and progression-free (PFS) survival at 5 yrs for the entire cohort were 89% and 66%, respectively, with a plateau 1–2 yrs following treatment. PFS at 5 years was significantly better with +R (81%) than with -R cohort (58%, P=0.03). Five-year PFS in pts treated with R-VACOPB, R-CHOP, VACOP-B and CHOP were 84%, 74%, 62%, 20%, respectively (P=0.025). Yet, there was no significant difference in OS between +R and -R cohorts (96% vs. 88% at 5 yrs, p=0.29). Direct survival comparisons demonstrated that 5-yr OS and PFS were significantly better in VACOPB than in CHOP (P=0.04 and 0.05, respectively) and that R-VACOPB was significantly superior to VACOPB in terms of PFS (p=0.05). In contrast, there was no difference in 5-yr PFS between R-VACOPB and R-CHOP (p=0.44). Univariate analysis revealed that aaIPI was not predictive of OS (p=0.51). Age above 31 yrs (p=0.02) and pericardial effusion (p=0.04) were the only predictors of reduced OS. Furthermore, beginning in 2003, 16 consecutive pts in the +R cohort, who were scanned by PET/CT-FDG before starting and after completion of therapy, were also evaluated in the middle (mid-PET) of treatment. The estimated 3-year PFS rate for mid-PET negative pts (n=8) and for mid-PET positive pts (n=8) was 86% and 75%, respectively (P=0.48). In terms of treatment failure, the negative predictive value of mid-PET was 100%, while the positive predictive value was only 25%. In conclusion, our population-based historical comparison demonstrates that the addition of R to anthracycline-based therapy significantly improved PFS in pts with PMBCL. We observed superior PFS with VACOPB compared with CHOP, but this superiority was abrogated by the introduction of R as part of initial therapy. These findings merit further study in randomized prospective studies.

The Roel of High Dose Chemotherapy and Autologous Stem Cell Transplantation (ASCT) In PATIENTS with POEMS SYNDROME: A Retrospective study of the MM Subcommittee of the Chronic Leukemia Working Party of the EBMT,

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4115-4115 ◽  
Author(s):  
Gordon Cook ◽  
Laurent Garderet ◽  
Anja van Biezen ◽  
Anja Henseler ◽  
Véronique Leblond ◽  
...  
Keyword(s):  
Plasma Cell ◽  
Conflicts Of Interest ◽  
Conditioning Regimen ◽  
Working Party ◽  
Poems Syndrome ◽  
Plasma Cell Dyscrasia ◽  
Bone Lesions ◽  
High Dose ◽  
Engraftment Syndrome ◽  
The Impact

Abstract Abstract 4115 Introduction: Polyneuropathy, organomegaly, endocrinopathy, skin changes associated with a paraproteinaemia (POEMS syndrome) is a rare paraneoplastic syndrome secondary to a plasma cell dyscrasia. Effective treatment, including ASCT, of the underlying plasma cell dyscrasia can control the disease and often dramatically control symptoms. Limited data is available for ASCT in POEMS. Specific Aim: The aim of this study was to describe the clinical outcome of ASCT for patients with POEMS syndrome, determining the impact of patient and disease-specific factors on prognosis. The incidence of engraftment syndrome and the presentation of relapse were examined. Methodology: Patient-, disease-, and transplant-related variables were collected according to the data entries in the EBMT database, including tracking incomplete data entries from participating centers. Results: 116 patients underwent an ASCT between 1997–2009 and satisfied the entry criteria. The median age was 50 yrs (range 26–69) with 56.8% of patients '50 year of age. 58.6% had peripheral neuropathy, 66.2% volume overload, 48.3% had organomegaly, 19.8% had papiloedema, 46.6% had dermopathies and 34.5% had sclerotic bone lesions at presentation. The median time from diagnosis to ASCT was 7.8 mns (range 1–346) with 34.5% of patients receiving an ASCT >12 months from diagnosis. The graft source was PBSC in 100% of patients. Disease status at ASCT was: 32% CR/PR, 30% SD/MR/untreated and 5 in PD. Missing information on stage in 33% of the cases. The conditioning regimen was Melphalan ≥200mg/m2 in 52.5%, Melphalan <200mg/m2 in 9.3% (38.1 of data on dose is missing) and TBI-containing only 1 patient. Engraftment was seen in 112 (96.6%) patients with failed engraftment reported in 3 patients (2.5%). Details of the occurrence of engraftment syndrome are currently under collection and analysis though peri-engraftment fever was reported in 23.4% and pulmonary infiltrates in 4.8%. Haematological response was characterized as CR in 31%, PR in 20.7%, <PR in 20.7% and currently unknown in 27.6%. Best disease response, in terms of end-organ response is under evaluation. With a median follow-up of 30.1 mns (range 0.1–161), 90.5 % of patients are alive and only 8.6% of patients have relapsed. The non-relapse mortality was 6.9%. Causes of death: 5 died of infection, 2 from graft failure, 1 from cardiac toxicity. The 3-year probabilities of PFS and OS are 82% and 94%, respectively. The 5-year probabilities of PFS and OS are 80% and 92%, respectively. The data analyzed in this study, to-date, demonstrates that ASCT can be an effective and safe therapeutic modality for patients with POEMS syndrome. The role of high dose therapy compared with more conventional dose therapies warrants further investigation. Disclosures: No relevant conflicts of interest to declare.

Artifact in Pediatric Oculomotor Findings during Videonystagmography: A Retrospective Analysis

2017 ◽  
Vol 28 (04) ◽  
pp. 314-324 ◽  
Author(s):  
Steven M. Doettl ◽  
Patrick N. Plyler ◽  
Devin L. McCaslin
Keyword(s):  
Eye Movements ◽  
Retrospective Analysis ◽  
Smooth Pursuit ◽  
Repeated Measures ◽  
Pediatric Population ◽  
Age Group ◽  
Repeated Measures Design ◽  
Significant Difference ◽  
Artifact Rejection ◽  
The Impact

Background: Accurate measurement of oculomotor function using videonystagmography (VNG) is imperative for diagnosis and management of patients with reported dizziness. The oculomotor evaluation during VNG utilizes video-oculography providing valuable information regarding the central structures and pathways that control eye movements. Artifact may have an effect on the overall validity and reliability of VNG oculomotor tracings and can result from patient and/or recording errors. It is postulated that artifact could occur more frequently in the pediatric population due to both patient and equipment factors. Purpose: The purpose of this study was to systematically evaluate the occurrence and impact of artifact on saccades, smooth pursuit, and optokinetic (OPK) testing in normal pediatric and adult subjects using commercially available clinical VNG equipment and standard clinical protocols for oculomotor testing. Research Design: The present study utilized a retrospective analysis of a repeated measures design. Study Sample: Oculomotor results from a total of 62 participants were analyzed. Portions of these data have been presented in a previous research study. Group 1 consisted of twenty-nine 4- to 6-yr-olds with an average age of 4.86 (SD = 0.88) yr. Group 2 consisted of thirty-three 22- to 44-yr-olds with an average age of 25.2 (SD = 5.34) yr. Data Collection and Analysis: Raw oculomotor recordings were analyzed “offline” by a single masked, trained investigator. Each tracing was evaluated for instances of artifact including eye blinks, eye closure, eyes moving in opposite direction of the target, eye tracking software problems, and overall poor morphology. The number of instances of artifact were noted and recorded for each participant in both groups. Individual eye movements not affected by artifact were included for final analysis. Artifact rejection techniques were also compared. Results: The results indicated increased artifact for the pediatric group for saccade and smooth pursuit testing. Additionally, a significant decrease in instances of artifact was noted with an increase in age in months for both saccade and smooth pursuit findings. OPK results did not indicate any significant difference in instances of artifact between the pediatric and adult groups or any decrease in instances of artifact with increasing age in the pediatric group. Artifact rejection technique did not have a significant effect on oculomotor measures for either age group. Conclusions: Pediatric patients exhibit increased instances of artifact during VNG oculomotor testing, specifically during saccade and smooth pursuit testing, at least for the 4- to 6-yr-old population. A general age effect was also noted in this age group, with decreased artifact noted with increasing age. Artifact rejection technique was not a significant factor suggesting standard compared based strategies may be sufficient for use in the pediatric population. Additional study into the effect of artifact on oculomotor results for infants to age 3 yr and ages 7- to 18-yr-old, in the disordered population, and with additional equipment manufacturers is needed to confirm these results and further describe the impact of artifact on oculomotor findings in the pediatric population.

Fall prevention education for people with multiple sclerosis: a randomized clinical trial

2021 ◽  
Vol 33 (1) ◽  
Author(s):  
Daniella Cristina Chanes ◽  
Felipe Maia de Toledo Piza ◽  
Gustavo San Martin ◽  
Eliseth Ribeiro Leão ◽  
Oscar Fernando Pavão Dos Santos
Keyword(s):  
Multiple Sclerosis ◽  
Patient Education ◽  
Behavior Change ◽  
Fall Prevention ◽  
Intervention Group ◽  
Knowledge Retention ◽  
Fall Rate ◽  
Significant Difference ◽  
Spaced Education ◽  
The Impact

Abstract Background Online spaced education (OSE) is a method recognized for promoting long-term knowledge retention, changing behaviors and improving outcomes for students and healthcare professionals. However, there is little evidence about its impacts on patient education. Objectives The aim of this research was to compare knowledge retention using educational brochure and OSE on individuals with multiple sclerosis (MS) and to verify the impact of educational methods on fall outcome. Methods Individuals with MS (n = 230) were randomly assigned to two types of patient education—educational brochure (control) and OSE (intervention). During 12 weeks, the intervention group received multiple-choice tests on fall prevention. Knowledge retention, behavior change and fall incidence were assessed before intervention and after 3 and 6 months. The participants’ satisfaction with the education method was also evaluated. Results Knowledge retention was similar between groups, and behavior change was observed in both groups. There was a significant reduction in fall rate in the intervention group, from 0.60 to 0.27 at 6 months (P &lt; 0.001). Participants’ satisfaction achieved an average of 8.75, with no differences between groups. Conclusion Individuals demonstrated significant improvement in fall rate outcome in both groups with no significant difference. In regard to test scores and satisfaction, results were similar between groups.

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