scholarly journals Impact of CD133 positive stem cell proportion on survival in patients with glioblastoma multiforme

2013 ◽  
Vol 47 (4) ◽  
pp. 405-410 ◽  
Author(s):  
Marju Kase ◽  
Ave Minajeva ◽  
Kristi Niinepuu ◽  
Sandra Kase ◽  
Markus Vardja ◽  
...  
Keyword(s):  
Stem Cells ◽  
Overall Survival ◽  
Stem Cell ◽  
Glioblastoma Multiforme ◽  
Survival Time ◽  
Median Survival ◽  
Postoperative Radiotherapy ◽  
Study Group ◽  
Survival Times ◽  
Cell Proportion

Abstract Background. The aim of the study was to assess the impact of CD133-positive (CD133+) cancer stem cell proportions on treatment results of glioblastoma multiforme (GBM) patients. Patients and methods. Patients with GBM (n = 42) received postoperative radiotherapy (± chemotherapy). Surgically excised GBM tissue sections were immunohistochemically examined for CD133 expression. The proportions of CD133+ GBM cells were determined (%). The proportion of CD133+ GBM stem cells was established by 2 independent researchers whose results were in good accordance (R = 0.8, p < 0.01). Additionally, CD133 expression levels were correlated with patients overall survival. Results. The proportion of CD133+ cells varied between patients, being from 0.5% to 82%. Mean and median proportions of CD133+ cells of the entire study group were 33% ± 24% (mean ± SD) and 28%, respectively. Clinical data do not support the association between higher proportion of stem cells and the aggressiveness of GBM. Median survival time of the study group was 10.0 months (95% CI 9.0-11.0). The survival time clearly depended on the proportion of CD133+ cells (log rank test, p = 0.02). Median survival times for patients with low (< median) and high (≥ median) proportion of CD133+ cells were 9.0 months (95% CI 7.6-10.5) and 12.0 months (95% CI 9.3-14.7), respectively. In multivariate analysis, the proportion of CD133+ cells emerged as a significant independent predictor for longer overall survival (HR 2.0, 95% CI 1.0-3.8, p = 0.04). Conclusions. In patients with higher stem cell proportion, significantly longer survival times after postoperative radiotherapy were achieved. Underlying reasons and possible higher sensitivity of GBM stem cells to fractionated radiotherapy should be clarified in further studies.

Statistical analysis of clinicopathological features, radiotherapy, and survival in 170 cases of oligodendroglioma

1987 ◽  
Vol 67 (2) ◽  
pp. 224-230 ◽  
Author(s):  
Karl-Fredrik Lindegaard ◽  
Sverre J. Mørk ◽  
Geir E. Eide ◽  
Tore B. Halvorsen ◽  
Reidulv Hatlevoll ◽  
...  
Keyword(s):  
Survival Time ◽  
Median Survival ◽  
Postoperative Radiotherapy ◽  
Survival Rates ◽  
Tumor Resection ◽  
Survival Times ◽  
Term Survival ◽  
Content Type ◽  
Postoperative Irradiation

✓ The postoperative survival time of 170 nonrandomized patients treated for cerebral oligodendrogliomas in Norway from 1953 to 1977 was studied. Survival times were significantly prolonged if postoperative irradiation was performed in addition to surgery (median survival time 26.5 vs. 38 months, p = 0.039). In the group without postoperative radiotherapy, the 5-year rate of survival was 27% compared with 36% in the irradiated patients. The respective survival rates after 8 years were 14% versus 17%; thus, there was little effect on long-term survival. Irradiation appears not to be of benefit after “total” removal. Patients with partly resected lesions appeared to benefit from postoperative radiotherapy; the median survival period after subtotal tumor resection was 37 months with and 26 months without radiotherapy (p = 0.0089). The findings also indicate that irradiation doses between 40 and 50 Gy were as effective as doses between 50 and 60 Gy in increasing the patients' probability of surviving 5 years after subtotal tumor resection. Since the risk of radiation necrosis is proportional to the dose applied, the lower dose is recommended. These conclusions were also valid when adjustments were made for prognostically significant histological and clinical features.

scholarly journals 3217 Catatonia, Delirium and Coma: Implications for Mortality

2019 ◽  
Vol 3 (s1) ◽  
pp. 37-37
Author(s):  
Jo Ellen Wilson ◽  
Sarasota Mihalko ◽  
Stephan Heckers ◽  
Pratik P. Pandharipande ◽  
Timothy D. Girard ◽  
...  
Keyword(s):  
Survival Time ◽  
Critically Ill ◽  
Median Survival ◽  
Critically Ill Patients ◽  
Median Survival Time ◽  
Rating Scale ◽  
Brain Dysfunction ◽  
Survival Times ◽  
Dsm 5 ◽  
Acute Brain Dysfunction

OBJECTIVES/SPECIFIC AIMS: Delirium, a form of acute brain dysfunction, characterized by changes in attention and alertness, is a known independent predictor of mortality in the Intensive Care Unit (ICU). We sought to understand whether catatonia, a more recently recognized form of acute brain dysfunction, is associated with increased 30-day mortality in critically ill older adults. METHODS/STUDY POPULATION: We prospectively enrolled critically ill patients at a single institution who were on a ventilator or in shock and evaluated them daily for delirium using the Confusion Assessment for the ICU and for catatonia using the Bush Francis Catatonia Rating Scale. Coma, was defined as a Richmond Agitation Scale score of −4 or −5. We used the Cox Proportional Hazards model predicting 30-day mortality after adjusting for delirium, coma and catatonia status. RESULTS/ANTICIPATED RESULTS: We enrolled 335 medical, surgical or trauma critically ill patients with 1103 matched delirium and catatonia assessments. Median age was 58 years (IQR: 48 - 67). Main indications for admission to the ICU included: airway disease or protection (32%; N=100) or sepsis and/or shock (25%; N=79. In the unadjusted analysis, regardless of the presence of catatonia, non-delirious individuals have the highest median survival times, while delirious patients have the lowest median survival time. Comparing the absence and presence of catatonia, the presence of catatonia worsens survival (Figure 1). In a time-dependent Cox model, comparing non-delirious individuals, holding catatonia status constant, delirious individuals have 1.72 times the hazards of death (IQR: 1.321, 2.231) while those with coma have 5.48 times the hazards of death (IQR: 4.298, 6.984). For DSM-5 catatonia scores, a 1-unit increase in the score is associated with 1.18 times the hazards of in-hospital mortality. Comparing two individuals with the same delirium status, an individual with a DSM-5 catatonia score of 0 (no catatonia) will have 1.178 times the hazard of death (IQR: 1.086, 1.278), while an individual with a score of 3 catatonia items (catatonia) present will have 1.63 times the hazard of death. DISCUSSION/SIGNIFICANCE OF IMPACT: Non-delirious individuals have the highest median survival times, while those who are comatose have the lowest median survival times after a critical illness, holding catatonia status constant. Comparing the absence and presence of catatonia, the presence of catatonia seems to worsen survival. Those individual who are both comatose and catatonic have the lowest median survival time.

scholarly journals A Clinicopathological Analysis on Diffuse Midline Glioma in Adults

2021 ◽  
Author(s):  
Xiao Mu Hu ◽  
Xiao Yu Nie ◽  
Kai Lun Xu ◽  
Yin Wang ◽  
Feng Tang ◽  
...  
Keyword(s):  
Overall Survival ◽  
Survival Time ◽  
Median Survival ◽  
Median Survival Time ◽  
Age Groups ◽  
Young Patients ◽  
Imaging Data ◽  
Wild Type ◽  
Old Patients ◽  
Diffuse Midline Glioma

Abstract Purpose: Diffuse midline glioma (DMG), H3K27M mutant is a new entity that has become widely recognized. However, studies concerning DMG in adult patients remains rare. We did a retrospective study covering the largest amount of patients to date to analyze the clinicopathological characteristics of DMG in adult. Methods: We reviewed 117 cases of adult DMG, collected their clinical and imaging data along with pathological results including H3K27M. Summarized their features and the connection with overall survival in different age groups.Results: Among 117 cases, most tumors were located at the thalamus, 39 patients had H3K27M mutation, of whom 38 demonstrated down regulation of H3K27me3. The average overall survival of H3K27M-mutant gliomas was 13 months, while that of 78 H3K27M wild-type gliomas were 11.8 months. For young patients (age＜35), The median survival time of the H3K27M-mutant was 20.1 months, while that of the H3K27M wild-type was 39.5 months. For older patients (age≥35), the median survival time of the H3K27M-mutant was 22.3 months, while that of the H3K27M wild-type was 17.1 months. The OS of patients who received biopsies, subtotal resections, and total resections were 15.8, 17.6, and 11.6 months respectively. Conclusion: The DMG in adults mainly occurred in the thalamus. H3K27M mutations tend to happen more frequently in young adults, and this genetic alteration results in a worse outcome only in young patients. For old patients, age and the approach of surgery are independent prognostic factors. Patients received biopsy instead of total resection had a better prognosis.

scholarly journals OR25-07 A Multi-Center, Open-Label, Pivotal Phase 2 Study of Azedra® (HSA I-131-MIBG) in Patients with Unresectable, Locally Advanced or Metastatic Pheochromocytoma or Paraganglioma: Updated Long-Term Survival and Safety

2020 ◽  
Vol 4 (Supplement_1) ◽  
Author(s):  
Camilo Jimenez ◽  
Bennett B Chin ◽  
Richard B Noto ◽  
Joseph Stephen Dillon ◽  
Lilja B Solnes ◽  
...  
Keyword(s):  
Overall Survival ◽  
Survival Time ◽  
Median Survival ◽  
Median Survival Time ◽  
Locally Advanced ◽  
Phase 2 ◽  
Pivotal Phase ◽  
Term Survival ◽  
Phase 2 Study ◽  
Therapeutic Doses

Abstract Background: Pheochromocytoma/Paraganglioma (PPGL) are rare neuroendocrine tumors with a 5-yr survival rate as low as 12%. There is a high unmet medical need for effective treatment options for patients with advanced disease. AZEDRA®, a high-specific-activity iodine-131 meta-iodobenzylguanidine (HSA I-131-MIBG), is the first and only FDA-approved therapeutic radiopharmaceutical agent indicated for the treatment of adult and pediatric patients with iobenguane scan positive, unresectable, locally advanced or metastatic PPGL who require systemic anticancer therapy. Methods: Patients with advanced PPGL who were heavily pre-treated and were ineligible for curative surgery or chemotherapy received a dosimetric dose followed by up to two therapeutic doses (each at 296 MBq/kg to a max of 18.5 GBq). The primary endpoint, defined as the proportion of patients with at least 50% reduction of all antihypertensive medication(s) lasting ≥6 months, was met and previously reported. Updated secondary endpoints including overall survival (OS) and safety are reported. Results: A dosimetric dose of HSA I-131-MIBG was administered to 74 patients. Of those, 68 patients received one therapeutic dose and 50 received two doses of HSA I-131-MIBG. Clinical benefit rates (objective tumor responses defined by RECIST 1.0 and stable disease) were observed in 71.4% and 98.0% of patients receiving one and two therapeutic doses, respectively. As of October 10, 2019, median survival time for all patients was 43.2 months (95% CI 31.4, &gt;60). Median survival time was 19.3 months (95% CI 4.5, 32.4) and 49.1 months (95% CI 36.9, &gt;60) in patients receiving one and two doses, respectively. The overall survival was 73.8% at 2 yrs, 47.5% at 4 yrs and 41.5% at 5 yrs. The most common (≥50%) adverse events were nausea, fatigue, and myelosuppression. Myelosuppressive events resolved within 4-8 wks without requiring stem cell transplantation. Late radiation toxicity included 7 patients with secondary malignancies (myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), colon cancer, and lung carcinoma) of which MDS, ALL and AML were considered related to I-131 radiotherapy. Conclusions: Results from this pivotal phase 2 study suggest that HSA I-131-MIBG is an efficacious and safe treatment for advanced PPGL.

Effect of Cytokine Therapy on Survival for Patients With Advanced Renal Cell Carcinoma

2000 ◽  
Vol 18 (9) ◽  
pp. 1928-1935 ◽  
Author(s):  
Robert J. Motzer ◽  
Madhu Mazumdar ◽  
Jennifer Bacik ◽  
Paul Russo ◽  
William J. Berg ◽  
...  
Keyword(s):  
Renal Cell Carcinoma ◽  
Survival Time ◽  
Median Survival ◽  
Renal Cell ◽  
Interleukin 2 ◽  
Interferon Alfa ◽  
Cytokine Therapy ◽  
Survival Times ◽  
Long Term Survivors

PURPOSE: To evaluate the relationship between treatment with cytokine therapy and survival, investigate the effect of nephrectomy on survival, and identify long-term survivors among a cohort of 670 patients with advanced renal cell carcinoma (RCC). PATIENTS AND METHODS: A total of 670 patients with advanced RCC treated on 24 clinical trials of systemic chemotherapy or cytokine therapy were the subjects of this retrospective analysis. Treatment was categorized as cytokine (containing interferon alfa and/or interleukin-2) in 396 patients (59%) and as chemotherapy (cytotoxic or hormonal therapy) in 274 (41%). Among the 670 patients, those with survival times of greater than 5 years were identified as long-term survivors. RESULTS: Patients treated with cytokine therapy had a longer survival time than did those treated with chemotherapy, regardless of the year of treatment or risk category based on pretreatment features. The median survival times for favorable-, intermediate-, and poor-risk patients were 27, 12, and 6 months for those treated with cytokines and 15, 7, and 3 months for those treated with chemotherapy, respectively. The magnitude of difference in median survival was greater in the favorable- and intermediate-risk groups. The median survival time was less than 6 months in the poor-risk group for both treatment programs. Median survival time was 14 months among patients with prior nephrectomy plus time from diagnosis to treatment greater than 1 year versus 8 months among those with time from diagnosis to treatment less than 1 year, regardless of pretreatment nephrectomy status. Thirty patients (4.5%) among the 670 patients were identified as long-term survivors; 12 were free of disease after nephrectomy and treatment with interferon alfa, interleukin-2, or surgical resection of metastasis. CONCLUSION: The low proportion of patients with advanced RCC who achieve long-term survival emphasizes the need for clinical investigation to identify more effective therapy.

scholarly journals Controlling Nutritional Status (CONUT) Score is Associated with Overall Survival in Patients with Unresectable Hepatocellular Carcinoma Treated with Lenvatinib: A Multicenter Cohort Study

Nutrients ◽  
2020 ◽  
Vol 12 (4) ◽  
pp. 1076 ◽  
Author(s):  
Shigeo Shimose ◽  
Takumi Kawaguchi ◽  
Hideki Iwamoto ◽  
Masatoshi Tanaka ◽  
Ken Miyazaki ◽  
...  
Keyword(s):  
Hepatocellular Carcinoma ◽  
Overall Survival ◽  
Nutritional Status ◽  
Survival Time ◽  
Median Survival ◽  
Median Survival Time ◽  
Rank Test ◽  
Multicenter Cohort Study ◽  
The Impact ◽  
Conut Score

We aimed to investigate the impact of the controlling nutritional status (CONUT) score, an immuno-nutritional biomarker, on the prognosis of patients with hepatocellular carcinoma (HCC) treated with lenvatinib (LEN). This retrospective study enrolled 164 patients with HCC and treated with LEN (median age 73 years, Barcelona Clinic Liver Cancer (BCLC) stage B/C 93/71). Factors associated with overall survival (OS) were evaluated using multivariate and decision tree analyses. OS was calculated using the Kaplan–Meier method and analyzed using the log–rank test. Independent factors for OS were albumin–bilirubin grade 1, BCLC stage B, and CONUT score <5 (hazard ratio (HR) 2.9, 95% confidence interval (CI) 1.58–5.31, p < 0.001). The CONUT score was the most important variable for OS, with OS rates of 70.0% and 29.0% in the low and high CONUT groups, respectively. Additionally, the median survival time was longer in the low CONUT group than in the high CONUT group (median survival time not reached vs. 11.3 months, p < 0.001). The CONUT score was the most important prognostic variable, rather than albumin–bilirubin grade and BCLC stage, in patients with HCC treated with LEN. Accordingly, immuno-nutritional status may be an important factor in the management of patients with HCC treated with LEN.

Plasmablastic Morphology Is an Independent Predictor of Poor Survival After Autologous Stem-Cell Transplantation for Multiple Myeloma

1999 ◽  
Vol 17 (5) ◽  
pp. 1551-1551 ◽  
Author(s):  
S. Vincent Rajkumar ◽  
Rafael Fonseca ◽  
Martha Q. Lacy ◽  
Thomas E. Witzig ◽  
Terry M. Therneau ◽  
...  
Keyword(s):  
Overall Survival ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Survival Time ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
Independent Predictor ◽  
Progression Free Survival ◽  
Poor Survival ◽  
Free Survival

PURPOSE: To study the prognostic value of plasmablastic morphology after autologous stem-cell transplantation for relapsed or primary refractory myeloma. PATIENTS AND METHODS: Seventy-five patients were studied. Investigators blinded to the clinical details of the individual cases reviewed bone marrow aspirate slides to determine plasmablastic classification. Plasmablasts were defined using strict, well-described criteria. Plasmablastic morphology was considered to be present (plasmablastic myeloma) when 2% or more plasmablasts were present in the plasma-cell population. RESULTS: Patients underwent transplantation 5 to 88 months (median, 20 months) after the initial diagnosis of myeloma. Twenty-eight percent of patients had plasmablastic morphology. A significantly greater proportion of patients with plasmablastic morphology had abnormal cytogenetics compared with those with nonplasmablastic classification (73% v 31%, respectively; P = .003). The overall survival rate measured from the time of transplantation was significantly worse in patients with plasmablastic morphology compared with those without (median survival time, 5 months v 24 months, respectively; P < .001). Progression-free survival time was shortened also, with a median time of 4 months compared with 12 months, respectively (P < .001). In the multivariate analysis, plasmablastic classification was the most powerful prognostic factor after transplantation for both overall (P = .001) and progression-free survival rates (P < .001). We also identified three risk groups based on plasmablastic morphology: plasma-cell labeling index, lactate dehydrogenase, and cytogenetics. The median overall survival time was 38 months when none of these factors was abnormal, 17 months with one abnormal factor, and 8 months with two or more abnormal factors (P < .001). CONCLUSION: Plasmablastic morphology is a powerful independent predictor of poor survival rate after autologous stem-cell transplantation for relapsed or primary refractory myeloma.

Sirolimus and Thrombotic Microangiopathy after Allogeneic Stem Cell Transplantation.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 1834-1834 ◽  
Author(s):  
N. Lynn Henry ◽  
Shuli Li ◽  
Haesook T. Kim ◽  
Colm Magee ◽  
Edwin Alyea ◽  
...  
Keyword(s):  
Overall Survival ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Allogeneic Stem Cell Transplantation ◽  
Thrombotic Microangiopathy ◽  
Simultaneous Occurrence ◽  
Control Group ◽  
Study Group ◽  
Gvhd Prophylaxis

Abstract Sirolimus (SRL) is a novel immunosuppressive agent that has been demonstrated to reduce GVHD and minimize morbidity after allogeneic stem cell transplantation (alloSCT). We have described a syndrome of thrombotic microangiopathy (TMA), characterized by microangiopathic hemolysis, thrombocytopenia, and renal dysfunction, whose incidence is increased when SRL is used in association with calcineurin inhibitors (CIs) but not with SRL monotherapy. To determine if SRL use potentiates the effects of CIs on TMA incidence and risk factors, we performed a retrospective cohort analysis of subjects who underwent alloSCT between 1997 and 2003. Methods: Subjects who received a SRL-containing GVHD prophylaxis after a myeloablative preparative regimen were compared with a cohort who received a non-SRL regimen. All subjects received CIs. Diagnosis of TMA required the simultaneous occurrence of: (1) creatinine elevation >2 mg/dL or >50% above baseline, (2) schistocytosis, (3) elevated LDH, and (4) no laboratory evidence of disseminated intravascular coagulopathy. Results: 111 patients who received SRL were compared with 216 patients who received no SRL during the first 100 days after alloSCT. The two groups of patients were balanced for demographic parameters; however, more patients in the SRL group received peripheral blood stem cells (50.5 vs. 18.1%, p<0.01) and had unrelated donors (58.6 vs. 42.6%, p<0.01). The incidence of TMA in the SRL group was 10.8% in comparison with an incidence of 4.2% in the non-SRL group (OR 2.57, p=0.03). Patients who received SRL developed TMA earlier than those who did not receive SRL (median 25 vs. 58 days, p=0.04). At the time of TMA diagnosis, median blood levels of immunosuppressive medications were in their respective therapeutic ranges: SRL (study group) 6.1 ng/ml, tacrolimus (study group) 9.9 ng/ml, tacrolimus (control group) 9.1 ng/ml; cyclosporine (control group) 418 ng/ml. In a multivariable logistic regression model, only the use of SRL (Adjusted Exact OR 3.49, p =0.02) and grade II-IV acute GVHD (Adjusted Exact OR 6.60, p = 0.0002) predicted the occurrence of TMA. Treatment of TMA consisted of discontinuation or dose adjustment of CIs. SRL was discontinued or dosed according to serum level. Two subjects in each group required temporary hemodialysis, and 3 subjects (1 SRL, 2 non-SRL) underwent plasmapheresis. 78% of surviving SRL-treated subjects regained normal renal function. No subject had a TMA recurrence if SRL was reintroduced. Overall survival after TMA diagnosis was better for SRL patients than non-SRL patients (58.3 vs. 11.1%, log rank p=0.02). Conclusion: SRL use is associated with an increased risk of TMA after alloSCT and may act by potentiating the effects of CIs. TMA associated with SRL appears reversible and does not affect overall survival after alloSCT. A careful monitoring strategy for TMA should be employed as part of a SRL-containing GVHD prophylaxis regimen.

Sirolimus and Tacrolimus as Graft-vs.-Host Disease Prophylaxis in Allogeneic Stem Cell Transplantation: The Dana-Farber Cancer Institute Experience.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 1227-1227
Author(s):  
Corey Cutler ◽  
Shuli Li ◽  
Haesook T. Kim ◽  
Edwin Alyea ◽  
Vincent Ho ◽  
...  
Keyword(s):  
Stem Cells ◽  
Overall Survival ◽  
Stem Cell ◽  
Serum Level ◽  
Acute Gvhd ◽  
Cell Function ◽  
Peripheral Blood Stem Cells ◽  
Gvhd Prophylaxis ◽  
Blood Stem Cells

Abstract Sirolimus is a novel immunosuppressant similar to tacrolimus, however, sirolimus inhibits T cell function uniquely via FKBP12/mTOR and can impair dendritic cell function. Sirolimus acts synergistically with tacrolimus and has a non-overlapping toxicity profile, making their use in combination attractive. We began employing sirolimus as GVHD prophylaxis in 2000, and herein describe our experience with this compound in the myeloablative transplant setting. Methods: Two clinical trials were performed using sirolimus (target serum level 3–12 ng/ml) and tacrolimus (target serum level 5–10 ng/ml) as primary GVHD prophylaxis. Cytoxan (1800 mg/m2/d x 2) and TBI (14 Gy, 7 fractions) was the conditioning regimen used in all patients. Trial 1 enrolled 76 patients who received HLA-matched, unrelated or 1-Ag mismatched related or unrelated bone marrow or peripheral blood stem cells. Abbreviated methotrexate (20 mg/m2 total) was routinely given post-transplant. Trial 2 enrolled 53 patients who received HLA-matched, related peripheral blood stem cells. No methotrexate was given post-transplant. Results: The median times to neutrophil engraftment (&gt;500/ml) after transplantation were 17 (range 11–32) and 14 (range 9–17) days for trials 1 and 2 respectively. The median times to platelet engraftment (&gt;20,000/ml) after transplantation were 29 (range 11–98) and 12 (range 10–47) days for trials 1 and 2. Grade II–IV acute GVHD occurred in 35% and 19% of patients in the two trials. Grade III–IV acute GVHD occurred in 21% and 4% of patients in the two trials. The median time to first hospital discharge was 33 and 19 days from transplantation for trials 1 and 2. Seven patients in trial 1 (9%) and 3 patients in trial 2 (6%) did not survive to first hospital discharge. The non-actuarial incidence of chronic GVHD in the two trials was 49 and 44%, respectively. Treatment-related mortality at 100 days was 13% and 6% in the trials. At two years, the relapse-free and overall survival estimates for trial 1 are 46 and 48% (Median follow-up of survivors: 27 months). One year relapse-free and overall survival estimates for trial 2 are 71% and 75% (Median follow-up of survivors: 15 months). Overall survival at 2 years is 72%. Toxicity related to the addition of sirolimus was modest. Among all patients (n=129), VOD occurred in 16 patients (12%), idiopathic pneumonia syndrome occurred in 9 patients (7%) and thrombotic microangiopathy occurred in 13 patients (10%). Conclusions: Sirolimus, when added to tacrolimus after allogeneic stem cell transplantation is effective for GVHD prophylaxis. Engraftment is prompt, the incidence and severity of acute GVHD are reduced and transplant-related morbidity and mortality is reduced, regardless of stem cell source and methotrexate use. Early survival estimates are excellent due to reduced GVHD and transplant-related toxicity. Sirolimus is worthy of broader study in allogeneic transplantation.

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