scholarly journals Results of the Blood and Marrow Transplant Clinical Trials Network Study BMT CTN 0601: Scurt - a Multicenter Phase II Trial of Unrelated Donor Reduced Intensity Bone Marrow Transplantation (BMT) for Children with Severe Sickle Cell Disease

2016 ◽  
Vol 22 (3) ◽  
pp. S104 ◽  
Author(s):  
Shalini Shenoy ◽  
Mary Eapen ◽  
Juan (Maggie) Wu ◽  
Mark C. Walters ◽  
John E. Levine ◽  
...  
2008 ◽  
Vol 88 (3) ◽  
pp. 324-330 ◽  
Author(s):  
Sung-Won Kim ◽  
Keitaro Matsuo ◽  
Takahiro Fukuda ◽  
Masamichi Hara ◽  
Kosei Matsue ◽  
...  

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 2009-2009
Author(s):  
Benedetto Bruno ◽  
Roberto Passera ◽  
Francesca Patriarca ◽  
Francesca Bonifazi ◽  
Vittorio Montefusco ◽  
...  

Abstract Abstract 2009 To evaluate the role of allografting from unrelated donors in the treatment of myeloma we conducted a retrospective study through the Italian Bone Marrow Transplantation Donor Registry. Overall, from 2000 through 2009, 196 myeloma patients, median age 51 years (32–67), for a total of 199 allografts, were transplanted from an unrelated donor at 34 Centers in Italy. Fifty-two (28.1%%), 69 (37.3%), and 64 (34.6%) patients were prepared for transplant with a myeloablative, a reduced-intensity and a non-myeloablative conditioning respectively. Patient characteristics of the 3 cohorts are reported in Table 1.ConditioningMyeloablativeReduced-intensityNon-myeloablativePatient number (%)52/185 (28)69/185 (37)64/185 (35)Median Age455355Previous therapy lines < 2 (%)23 (27)33 (38)30 (35)Previous therapy lines ≥ 2 (%)29 (29)36 (37)34 (34)Stem Cell Source BM (%)24 (57)18 (43)0 (0)Stem Cell Source PBSC (%)28 (19)51 (36)64 (45) Cumulative incidence of acute grade II-IV graft-versus-host-disease (GVHD) was 46.4% whereas chronic GVHD was 45.1%. There was no difference in GVHD incidence among the 3 cohorts defined by type of conditioning. Complete and partial remissions in patients who survived at least 3 months post-transplant were 27% and 28% for an overall response rate of 55%. At a median follow up of 32 (0–118) months post-transplant, in the entire study population, median OS from diagnosis was 70.6 months while OS and EFS from the allograft were 18.9 and 14.9 months. Overall, the cumulative incidence of transplant related mortality (TRM) was 29.6% at 1 year and 32.4% at 5 years post-transplant. OS from diagnosis and EFS from transplant were 70.6 and 28.2 months; 66.8 and 9.1 months; and 111.9 and 22.4 months in patients who respectively underwent a myeloablative, a reduced-intensity and a non-myeloablative transplant. One-year and 5-year TRM was 33.3% and 35.7%, 32.2% and 34.4%, and 22.1% and 26.5% respectively. Univariate and multivariate analyses, assessed by multivariate Cox proportional hazards models, were performed for the following variables: number of previous chemotherapy lines (1,2 vs. ≥3), disease status at transplant, HLA- matched antigens (10/10 vs. 9/10 vs. ≤8/10), recipient/donor gender combinations, hematopoietic cell source (peripheral blood vs. bone marrow), conditioning (myeloablative vs. reduced-intensity vs. non-myeloablative), use of anti-thymoglobulin in the conditioning, acute GVHD, chronic GVHD, best response post-transplant, year of transplant (2000–02 vs. 2003–05vs. 2006–09). By univariate analysis, lower number of chemotherapy lines before the allograft, disease status at transplant, a fully HLA-identical donor, the use of peripheral hematopoietic cells rather than bone marrow were statistically significant variables for better OS whereas disease status at transplant, a fully HLA-identical donor, chronic GVHD (either limited or extensive) were statistically significant for better EFS. However, by multivariate analysis, only the development of chronic GVHD (HR 0.50; p<0.001) and a better response post-transplant (HR 2.11; p<0.03) were significantly associated with longer OS whereas chronic GVHD was the only variable associated with better EFS (HR 0.32; p<0.001). Acute GVHD was associated with both poorer OS (HR 2.35; p<0.001)) and EFS (HR 3.19; p<0.001). In conclusion, with a degree of caution given the retrospective nature of this study, there appears to be a strong association between both limited and extensive chronic GVHD and graft-vs.-myeloma effects. However, long term disease control remains an issue independent of the conditioning employed. Prospective trials may allow to define which patient category may most benefit from an unrelated donor allograft. Disclosures: Patriarca: Roche: Honoraria; Celgene: Honoraria; Schering-Plough: Honoraria; Janssen: Honoraria. Boccadoro:Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Janssen-Cilag: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding.


Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 6006-6006
Author(s):  
Mark J. Fesler ◽  
Crystal Weaver ◽  
Kimberly McCormick ◽  
Andrew Dwiggins

Abstract Introduction: According to the Commission on Cancer's 2012 program standards, patients diagnosed with cancer may experience psychological issues that can interfere with patient treatment plans and adversely affect outcomes. To address these issues, the Commission developed the following guidelines to accurately determine patient distress levels: 1) patients with cancer are offered screening for distress at least 1 time during a pivotal medical visit, 2) the mode of administration for the distress screening is to be determined by the program, and 3) facilities select the tool to be administered to screen for distress with preference being given to standardized, validated instruments. To meet this standard and plan future work in distress reduction for stem cell transplant recipients, the St. Louis University Blood and Marrow Transplant Program began implementing The State Trait Anxiety Inventory (STAI) with patients during pivotal medical visits. The STAI is a psychological inventory based on a 4-point Likert scale and consists of 40 questions on a self-report basis. The STAI differentiates between the temporary condition of "state anxiety" and the long-standing quality of "trait anxiety." The essential qualities evaluated by the STAI scale are feelings of apprehension, tension, nervousness, stress, and worry. Scores on the STAI scale increase in response to physical danger and psychological stress and decrease as a result of relaxation training. Average scores for working, male adults are 35.72 (state) and 34.89 (trait). Average scores for working, female adults are 35.20 (state) and 34.79 (trait). After implementing the STAI, it was realized that these screenings could be de-identified and analyzed in groups to determine if patterns emerged regarding patients' perceived anxiety levels throughout the bone marrow transplantation process. Method: The study team received Institutional Review Board approval to perform a retrospective examination of STAIs completed by patients throughout the bone marrow transplantation process at the St. Louis University Blood and Marrow Transplant Program from 03/11/2104 through 06/24/2014. A total of 30 inventories were collected, de-identified, and categorized by the following medical visits: arrival visit (the patient's first visit to the Blood and Marrow Transplant clinic), data review visit (the visit to review transplant related testing and sign consents), start of preparative regimen visit, day 0 visit, day +30 bone marrow biopsy visit for allogenic transplantation, day +30 bone marrow biopsy result visit for allogenic transplantation, and day +100 visit for auto transplantation. Averages for each medical visit category were determined by finding the mean score. Category averages were then compared to determine if a particular pivotal medical visit caused patients to experience an overall increase in anxiety level. Results: Results from the study indicate that patients experience the highest levels of anxiety during the early medical visits of the bone marrow transplantation process. Average state anxiety scores were 46 during the arrival visits, 41 during the data review visits, and 44 during the start of preparative regimen visits. Average trait anxiety scores were 38 during the arrival visits, 45 during the data review visits, and 39 during the start of preparative regimen visits. During the day 0 visits, patients' state anxiety scores decreased to an average of 36 and trait anxiety scores decreased to an average of 35. Day +30 and day +100 visits demonstrated even further decreases in state and trait anxiety scores. Conclusion: The surprising finding of this study was that patients demonstrated a higher level of distress in the period leading up to the transplant which gradually decreased once the preparative regimen was administered. The sample size for this study was small and could possibly skew results. However, this study does provide a starting basis for future study in bone marrow transplant recipient distress, and larger, multi-site studies are being planned to ensure the accuracy of the patterns, which emerged from this study. If patterns could be accurately identified and predicted, the study team may also be able to develop future studies to primitively lower patients' anxiety levels early in the bone marrow transplantation process and thus improve outcomes. Abstract 6006 Figure 1 Abstract 6006 Figure 1. Disclosures No relevant conflicts of interest to declare.


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