A cost and resource utilization analysis of micafungin bridging for hemato-oncological high-risk patients undergoing allogeneic stem cell transplantation

2015 ◽  
Vol 94 (6) ◽  
pp. 526-531 ◽  
Author(s):  
Sebastian M. Heimann ◽  
Maria J.G.T. Vehreschild ◽  
Oliver A. Cornely ◽  
Bernd Franke ◽  
Michael von Bergwelt-Baildon ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Resource Utilization ◽  
Allogeneic Stem Cell Transplantation ◽  
High Risk Patients ◽  
Risk Patients

Rapid Risk-Profiling Including Fast Donor Search within a Multi-Center Trial Allows for Early Allogeneic Stem Cell Transplantation during Aplasia in Patients with High-Risk Acute Myeloid Leukemia.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 2767-2767 ◽  
Author(s):  
Markus Andreas Schaich ◽  
Monika Fuessel ◽  
Martin Bornhaeuser ◽  
Christian Thiede ◽  
Brigitte Mohr ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Induction Therapy ◽  
Allogeneic Stem Cell Transplantation ◽  
Unrelated Donor ◽  
High Risk Patients ◽  
Risk Profiling ◽  
Risk Patients

Abstract Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment option for most high-risk patients with acute myeloid leukemia (AML). However, many high-risk patients regenerate with blasts or relapse early after induction therapy. Thus, consolidation with allogeneic HSCT in first CR is often not possible. Performing allogeneic HSCT within induction therapy can circumvent these problems and may furthermore reduce cumulative toxicity in high risk patients. Therefore, the prospective randomized treatment trial AML2003 for patients <= 60 years was set up, to investigate the feasibility and value of an intensified treatment strategy, i.e. early allogeneic stem cell transplantation in aplasia after induction therapy, for high risk AML patients in a multi-center setting. To achieve this goal, rapid analysis of cytogenetics, FLT3 status and HLA-types of the patient and possible family donors is of utmost importance. This fast search diagnostics together with routine analyses of morphology and immunophenotyping is accomplished centrally in all enclosed patients. Furthermore, in all patients the likelihood to find an unrelated donor is checked by an internet search in the BMDW database. Within the first 8 months 107 AML patients with a median age of 48 (17–60) years were included in the study. Fast search diagnostics was complete within a median of 15 (range 5–31) days after arrival of the bone marrow samples for all patients. 57/107 patients were randomized into the intensified treatment arms. Out of these 25 (44%) patients with high risk characteristics have been identified. A suitable related or unrelated donor was found for 22 (88%) of those high-risk patients. Nine of those high risk patients with a donor (41%) received early allogeneic stem cell transplantation in aplasia after the first (n=4) or the second (n=5) induction therapy course on an intend to treat basis within the protocol. Three were transplanted with stem cells of related and six of unrelated donors. The preparative regimen consisted of melphalan 150mg/m2 and fludarabine 150mg/m2. So far no treatment associated death had to be recognized. These encouraging preliminary results show that fast risk-profiling and early donor-search is feasible in a large multi-center study. This leads to a significant proportion of early allogeneic stem cell transplants in aplasia after induction therapy within the group of high risk AML patients, which may improve the disastrous prognosis of this group of patients in the future.

Upfront Allogeneic Stem Cell Transplantation for Remission Induction in High-Risk Acute Myeloid Leukemia Patients within the Randomized Multi- Center Trial AML2003.

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 978-978 ◽  
Author(s):  
Markus Schaich ◽  
Thomas Illmer ◽  
Walter E. Aulitzky ◽  
Martin Bornhaeuser ◽  
Martin Griesshammer ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Induction Therapy ◽  
Allogeneic Stem Cell Transplantation ◽  
Allogeneic Transplantation ◽  
Remission Induction ◽  
High Risk Patients ◽  
Risk Patients

Abstract Introduction: Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment option for most high-risk patients with acute myeloid leukemia (AML). However, many high-risk patients regenerate with blasts or relapse early after induction therapy. Thus, consolidation with allogeneic HSCT in first CR is often not possible. Performing upfront allogeneic HSCT for remission induction as part of induction therapy has the potential to circumvent these problems and might furthermore reduce cumulative toxicity in high-risk patients. Therefore, in 2003 we started a prospective multicenter randomized trial that investigates both the feasibility and efficacy of upfront allogeneic stem cell transplantation for remission induction in high-risk AML patients. Methods: The AML2003 study compares in a randomized fashion an intensified treatment approach using upfront allogeneic transplantation in high risk patients as part of the induction therapy (IT) during marrow aplasia achieved by DA (daunorubicin 60 mg/ m2 – day 3–5; cytarabine 100 mg/m2 – day1–7) to a “conventional” treatment strategy, which allows for allogeneic transplantation only in patients achieving remission after two induction courses (DA). To do this, rapid analysis of cytogenetics, FLT3 status and HLA-DNA-typing of the patient and possible family donors is of utmost importance. This “fast search diagnostics” together with routine analyses of morphology and immunophenotyping is accomplished centrally in all enclosed patients. The dose-reduced preparative regimen for upfront allogeneic stem cell transplantation within induction therapy consisted of melphalan 150mg/m2 and fludarabine 150mg/m2. Results: Until the last update we recruited 679 patients <= 60 years with de novo (n=570) or secondary (n=109) AML. Out of 340 patients randomized for an intensified treatment approach we identified 139 patients (41%) with high-risk defined by cytogenetic criteria (n=87), FLT3 status (n=15) or day 15 blast count (n=37). Fast search strategy revealed HLA identical donors (related or unrelated) for 106 patients. Consequently, 78 high-risk AML patients assigned to the intensified treatment strategy received allogeneic transplantation. Upfront allogeneic stem cell transplantation for remission induction was feasible in 28 high-risk AML patients during marrow aplasia after IT1 (n=10) or IT2 (n=18), respectively. Fifteen of these patients received unrelated grafts. Conclusions: These preliminary results show that rapid risk profiling and fast donor-search is feasible in a large multi-center study. This leads to a significant proportion of upfront allogeneic stem cell transplants as part of the induction therapy within the group of high-risk AML patients, which may improve the disastrous prognosis of this group of patients in the future.

scholarly journals RVD induction and autologous stem cell transplantation followed by lenalidomide maintenance in newly diagnosed multiple myeloma: a phase 2 study of the Finnish Myeloma Group

2019 ◽  
Vol 98 (12) ◽  
pp. 2781-2792 ◽  
Author(s):  
Sini Luoma ◽  
Pekka Anttila ◽  
Marjaana Säily ◽  
Tuija Lundan ◽  
Jouni Heiskanen ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
Maintenance Phase ◽  
Molecular Response ◽  
Newly Diagnosed ◽  
High Risk Patients ◽  
Risk Patients

Abstract Autologous stem cell transplantation (ASCT) combined with novel agents is the standard treatment for transplant-eligible, newly diagnosed myeloma (NDMM) patients. Lenalidomide is approved for maintenance after ASCT until progression, although the optimal duration of maintenance is unknown. In this trial, 80 patients with NDMM received three cycles of lenalidomide, bortezomib, and dexamethasone followed by ASCT and lenalidomide maintenance until progression or toxicity. The primary endpoint was the proportion of flow-negative patients. Molecular response was assessed if patients were flow-negative or in stringent complete response (sCR). By intention to treat, the overall response rate was 89%. Neither median progression-free survival nor overall survival (OS) has been reached. The OS at 3 years was 83%. Flow-negativity was reached in 53% and PCR-negativity in 28% of the patients. With a median follow-up of 27 months, 29 (36%) patients are still on lenalidomide and 66% of them have sustained flow-negativity. Lenalidomide maintenance phase was reached in 8/16 high-risk patients but seven of them have progressed after a median of only 6 months. In low- or standard-risk patients, the outcome was promising, but high-risk patients need more effective treatment approach. Flow-negativity with the conventional flow was an independent predictor for longer PFS.

scholarly journals Survival of Allogeneic Stem Cell Transplantation Vs Conventional Therapies per DIPSS Stratification in Patients with Primary Myelofibrosis Younger Than 65 Years: A Retrospective Analysis on 673 Patients

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 633-633
Author(s):  
Nicolaus Kröger ◽  
Toni Giorgino ◽  
Bart L Scott ◽  
Marcus Ditschkowski ◽  
Haefaa Alchalby ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Patient Selection ◽  
Allogeneic Stem Cell Transplantation ◽  
Primary Myelofibrosis ◽  
Low Risk ◽  
Time Dependent ◽  
Risk Patients

Abstract Introduction Primary myelofibrosis (PMF) is a clonal myeloproliferative neoplasm with a high risk of clonal evolution and mortality. Allogeneic stem cell transplantation (ASCT) is currently the only curative treatment approach for myelofibrosis. Due to the inherent complications, careful patient selection is mandatory. Patients and Methods With the goal of refining patient selection for ASCT we compared results of ASCT with non-transplant approaches in 673 patients with PMF stratified according the Dynamic International Prognostic Scoring System (DIPSS), which assigns a time-dependent risk on a scale of low, intermediate (int)-1, int-2, and high. Considering a commonly used age-threshold for indication of ASCT, we restricted the analysis to patients aged 65 years or younger. This included 190 PMF patients (median 50 years at diagnosis) who received ASCT (HLA-matched related, n= 88 [46%] HLA-matched unrelated and mismatched related, n= 102 [54%] donors) after reduced intensity (n=93), or high intensity conditioning (n=97), and 248 patients, median age 55 years, from the DIPSS database (PMF patients not receiving any experimental drug at data cut-off and censored at the time of ASCT). DIPSS scores at ASCT, or, for non-transplanted patients, at diagnosis, were low in 22 (11%) and 125 (49%), int-1 in 38 (20%) and 75 (29%), int-2 in 85 (45%) and 52 (20%), and high in 45 (24%) and 3 (1%) patients, respectively. The median time from diagnosis to ASCT was 1.2 years. In line with the dynamic nature of the DIPSS, risk scores in the non-transplanted cohort change with time and, accordingly, were accounted for as time-dependent covariates. The date of diagnosis was considered as origin of the time scale, and patients entered the analysis when receiving ASCT (ASCT cohort) or at the time of acquisition of a specific DIPSS category of a non-transplant cohort. Therefore, left truncated, right censored data were used for both cohorts. Left truncation (begin of observation) for the ASCT cohort was the time to ASCT after diagnosis, while for the non-transplant cohort it was the time of achieving the respective DIPSS risk category. Cox proportional hazard models were built separately for the four DIPSS stages, considering the cohort as a discrete covariate; the corresponding relative risks (RR) between the two cohorts were computed under the proportional-hazards approximation, and Wald tests were used to report significance. Age at diagnosis was not significant in any model and, therefore, was not included in the regressions. The present study could not address the potentially confounding factor of selection bias regarding ASCT. Results The relative risk (RR) of dying among patients receiving ASCT relative to those receiving conventional therapies was 5.6 (95% CI: 1.7-19, p=0.0051) for low risk DIPSS, 1.6 (95% CI: 0.79-3.2, p=0.19) for int-1 risk DIPSS, 0.55 (95%CI: 0.36-0.83, p=0.005) for int-2 , and 0.37 (95%CI: 0.21-0.66, p=0.0007) for high risk DIPSS patients. The proportions of patients with low risk DPSS surviving at 1, 5 and 10 years were 100%, 69%, and 60%, respectively, for the ASCT cohort, and 98%, 95%, and 92% for the non-transplant cohort. The corresponding figures for patients with int-1 risk were 78%, 52%, and 41%, respectively, after ASCT, and 97%, 77%, and 63% for non-transplanted patients. Among patients with int-2 risk survival after ASCT was 82%, 50%, and 32%, respectively, and 77%, 41% and 11% among non-transplanted patients. The corresponding figures for high risk patients were 65%, 37% and 27% for the ASCT cohort, and 67%, 11% and 1%, respectively, for the non-transplant cohort. It should be noted that hazard ratios are not constant over time, and summarizing the risk in a single “average” RR ignores the specifics of the survival trends. Survival differences became pronounced beyond 5 years following diagnosis for int-2 and high risk patients. Conclusion Patients with PMF who, at any age≤ 65 years, have int-2 or high risk disease by DIPSS have superior survival if receiving ASCT during their disease history. Conversely, conventional therapy appears to result in superior outcome in low risk patients, while intermediate-1 risk patients represent a group in whom individual counseling may be particularly important. Disclosures Vannucchi: Novartis Pharmaceuticals Corporation: Consultancy, Honoraria, Research Funding.

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