Matched Pair Analysis of Cryopreserved Versus Fresh Allogeneic Peripheral Blood Stem Cell Transplantation

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4529-4529
Author(s):  
Arne Trummer ◽  
Juergen Krauter ◽  
Michael Stadler ◽  
Arnold Ganser ◽  
Stefanie Buchholz

Abstract Abstract 4529 Published data about outcome after cryopreserved allogeneic peripheral blood stem cell transplantation are scarce and, so far, have only been compared to historic cohorts of fresh graft recipients. We have performed a matched pair analysis of 66 patients receiving either cryopreserved or fresh grafts from matched related donors at our institution between January 2005 and June 2011 with a median follow-up time of 576 days (range: 18–2080 days). For matching patients we calculated a propensity score including patient age, sex, diagnosis, performance status, remission status before transplantation (first remission vs. other), conditioning therapy (standard vs. reduced intensity), GvHD prophylaxis (with or without methotrexate) and CD34 cell count in the graft. Consequently, there were no significant differences between both groups for these parameters: median patient and donor age were 53 years (range: 18–68 and 23–76 years, respectively) for 34 male and 32 female patients with a performance status of ECOG 0 (n=60) or ECOG 1 (n=6). Diagnoses were AML (n=47), ALL (8), lymphoma (10) and CMPN (1). 31 patients were in first remission before transplantation. Reduced intensity conditioning therapy (n=52) and GvHD prophylaxis without methotrexate (n=41) were more frequent than standard conditioning (n=14) and prophylaxis with methotrexate (n=25), respectively. Median cell counts were also almost equal in fresh and cryopreserved grafts for CD34 cells (5.7×106/kg (range: 3.1–11.4×106/kg) vs. 5.1×106/kg (2.6–12.3×106/kg), respectively), total nuclear cells (10.0×108/kg (4.9–21.4×108/kg) vs. 9.6×108/kg (5.0–19.1×108/kg) and CD3 lymphocytes (3.8×108/kg (2.1–6.8×108/kg vs. 3.4×108/kg (0.6–4.5×108/kg)). All patients engrafted. Median neutrophil engraftment with an ANC >0.5×109/l was reached after 16 days (range: 10–21) vs. 15 days (10–31) (p=.15 by paired t-test) and platelet engraftment >20×109/l (for 3 consecutive days without requiring transfusion) occurred after 13 days (8–33) vs. 14 days (9–45) (p=.27). Median follow-up time was similar between both groups (566 vs. 586 days, p=.894) and mean overall survival time, as calculated by Kaplan-Meyer analysis, was 1113 days for patients receiving fresh compared to 1258 days for patients receiving cryopreserved grafts (p=.582 by log rank test). Relapse or progression occurred in 13 vs. 14 patients, giving a mean disease/progression-free survival time of 922 vs. 1114 days (p=.467). In summary, we did not observe any relevant outcome differences between patients receiving fresh or cryopreserved peripheral stem cell grafts of matched related donors. Therefore, the use of cryopreserved grafts can be considered safe and may even allow a more flexible transplant scheduling compared to fresh allografts. Disclosures: No relevant conflicts of interest to declare.

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 2403-2403 ◽  
Author(s):  
Régis Peffault de Latour ◽  
Hubert Schrezenmeier ◽  
Andrea Bacigalupo ◽  
Didier Blaise ◽  
Carmino Antonio De Souza ◽  
...  

Abstract Abstract 2403 Background: Hematopoietic Stem Cell Transplantation (HSCT) is the only curative treatment for paroxysmal nocturnal hemoglobinuria (PNH). However, the risk of treatment-related mortality (TRM) usually leads to postponing HSCT in PNH until disease progression (especially in case of life-threatening thromboembolism (TE) or severe aplastic anemia (SAA)). Recent studies demonstrated that eculizumab, a C5-inhibitor, is highly effective in reducing intravascular hemolysis with protective effect on the risk of TE. Today, identifying PNH patients (pts) who may benefit from HSCT represents a particularly difficult challenge. We thus conducted an overall survival (OS) comparison between a cohort of transplanted PNH pts and a matched cohort of non-transplanted PNH pts before the eculizumab era. Patients and methods: This retrospective multi-center study was conducted through the SAA WP of the EBMT and the SFH. Between 1978 and 2008, 211 pts with PNH who were transplanted have been reported to the EBMT registry. Data concerning another 401 non-transplanted PNH pts came from the SFH (Peffault de Latour Blood et al., 2008). The OS comparison between the 2 cohorts was performed from the time of life-threatening complication occurrence among pts who experienced the same type of complication, being either TE or SAA. Among pts who experienced one of these complications, two matching procedures were applied in order to select non-transplanted pts comparable to transplanted pts. First, an individual matching procedure was completed using the following complication occurrence criteria: severity, patient age (per decade), year (per decade), and the delay between diagnosis of PNH and complication occurrence (relatively to 3 or 6 months). In addition, the survival time from complication for a non-transplanted patient should be longer than the delay from the complication to HSCT for the corresponding transplanted patient. In a second step, a global matching procedure was performed using the same qualitative prognostic factors. For each factor related to OS within one cohort, a category was discarded from the analysis in the absence or too few numbers of pts in one cohort. After these selections, OS-related factors allowed us to define strata through their combination. OS comparisons between cohorts were performed by using proportional hazards models stratified on each individual matched pair, or adjusted on strata to allow interaction test between cohort and stratum. Results: After a median follow-up time of 5 yrs, the 5-yr OS rate (SE) was 68% (3%) in the transplanted pts group (75 received HSCT from unrelated donors). The only factor associated with OS was the indication for HSCT with worse outcome when the indication was TE (p=0.03). In the non-transplanted cohort, the 5-yr OS rate was 83% (2%) after a median follow-up time of 7 yrs. From the 122 pts diagnosed with TE in the SFH cohort, 92 were eligible for the matched-pair analysis, while from the 47 pts who were transplanted for TE in the EBMT population, 42 were eligible. We then identified 24 pairs of transplanted and non-transplanted pts eligible for the matched-pair comparison. Worst OS was observed for transplanted pts (p=0.007, HR=10.0 [95%CI, 1.3 – 78.1]). In the global matching analysis, we derived 2 prognostic strata: stratum A (age <30 yrs and delay from diagnosis of PNH to TE ≥ 3 months, or delay < 3 months) and stratum B (age ≥30 yrs and delay from diagnosis of PNH to TE ≥ 3 months). Adjusted analysis on prognostic strata is presented in Figure 1. From the 141 pts experiencing SAA in the SFH cohort, 99 were eligible for the matched-pair analysis, while from the 119 pts who were transplanted for SAA in the EBMT population, 100 were eligible. We then identified 30 pairs of transplanted and non-transplanted pts eligible for the matched-pair comparison. Worst OS was observed for transplanted pts, but the difference was not statistically significant (p=0.06, HR=4.0 [0.9–18.9]). The global matching was not feasible for pts experiencing SAA because the procedure led to too many strata. Conclusion: Our analysis indicates that HSCT is probably not a good treatment option for life-threatening TE in PNH pts. We can make no conclusions regarding PNH pts experiencing SAA due to the impossibility to perform the global matching procedure. These results are of particular importance in the eculizumab era to help physicians in the management of PNH pts, especially pts with TE. Disclosures: Peffault de Latour: Alexion: Consultancy, Research Funding.


2016 ◽  
Vol 97 (2) ◽  
pp. 142-147 ◽  
Author(s):  
Sebastian Nestler ◽  
Patrick Levien ◽  
Andreas Neisius ◽  
Christian Thomas ◽  
Mohammed M. Kamal ◽  
...  

2013 ◽  
Vol 31 (15_suppl) ◽  
pp. 1120-1120 ◽  
Author(s):  
Elena Sperk ◽  
Daniela Astor ◽  
Grit Welzel ◽  
Axel Gerhardt ◽  
Marc Suetterlin ◽  
...  

1120 Background: After breast conserving surgery, radiotherapy leads to a better overall survival. In addition to whole breast radiotherapy (WBRT) a boost to the tumor bed leads to a better local control. The tumor bed boost is usually added after WBRT or can be done intraoperative (IORT). Belletti et al. (Clin Cancer Res., 2008) described positive effects, an antitumoral effect and modulation of microenvironment after IORT with 50kV x-rays. A matched pair analysis was performed to investigate the impact of IORT boost on overall survival compared to standard external beam boost. Methods: Between 2002 – 2009, 370 patients were treated for breast cancer with WBRT + boost (external beam (EBRT) boost n = 146, IORT boost n =224). A matched pair analysis (1:1 propensity score matching for age, TNM, grading, hormonal treatment and chemotherapy) for overall survival and local recurrence free survival could be done for 53 pairs. All patients underwent breast conserving surgery and WBRT with 46-50Gy. 53 patients received an EBRT boost with 16Gy (2Gy/fraction, dedicated linear accelerator) and 53 patients received an IORT boost with 20Gy (INTRABEAM system, 50kV x-rays). Median follow-up was 6 months (range, 1-77 months) for the EBRT boost patients and 56 months (range, 2-97 months) for IORT boost patients. Kaplan Meier estimates were performed for overall survival and local recurrence free survival. Results: IORT boost patients had a longer follow-up than EBRT boost patients. Despite the difference in follow-up times, there was a strong trend towards better overall survival after IORT boost (90.2% vs. 62.3%, p = 0.375). One local recurrence was present in each group (EBRT boost after 15 months, local recurrence free survival 95%; IORT boost after 12 months, local recurrence free survival 98.1%). Conclusions: IORT given as a boost seems to have a positive impact on overall survival in breast cancer patients after breast conserving surgery. To identify such an effect a prospective randomized trial should be conducted.


2001 ◽  
Vol 37 ◽  
pp. S185
Author(s):  
H. Sommer ◽  
W. Janni ◽  
B. Strobl ◽  
D. Rjosk ◽  
C. Schindlbeck ◽  
...  

Sign in / Sign up

Export Citation Format

Share Document