scholarly journals Survival Comparability Between Thalassemia Major Versus Thalassemia Intermedia

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 2141-2141
Author(s):  
Angela Vitrano ◽  
Giuseppina Calvaruso ◽  
Eliana Lai ◽  
Grazia Colletta ◽  
Alessandra Quota ◽  
...  

Abstract Introduction. In the last few decades, the life expectancy of Thalassemia Major (TM) patients has progressively been increasing. The improvement can be due to several factors, including introduction of chelation treatment (Deferoxamine 1965, Deferiprone 1987, Deferasirox 2006), screening of blood for the most common viral agents, aggressive treatment of infection and improved treatment of cardiac complications. However, no comparative survival curves between TM versus Thalassemia Intermedia (TI) have been so far reported. Moreover, no data on life expectancy, after introduction of chelation treatment have been described. Methods. Data coming from several randomized clinical trials, carried ahead by Campus of Hematology Franco and Piera Cutino-A.O.O.R Villa Sofia-V. Cervello, Palermo (Italy), were retrospectively considered for this study. Primary goal of the study was to provide evidence of possible differences in survival curves between TM versus TI. Survival curves in TM versus TI patients were compared using Kaplan-Meier method and the log-rank test before and after the introduction of Deferoxamine (DFO) (1965). Moreover, Cox regression model was even used to explore risk of death between the two diagnoses. Each dead patient was observed from its birth to its death, and each alive patient was observed from its birth to June 30, 2015. Results. Three hundred seventy-nine patients with TM (n=284, dead 40) and TI (n=95, dead 13) entered into the study. Males were 50.7% of this cohort of patients. Among the cohort of dead patients, 15% (6/40) TM and 76.9% (10/13) TI patients were born before introduction of DFO (1965) . The mean age survival was 50.6 (SE 0.9) and 70.6 (SE 1.7) for TM and TI, respectively. Table 1 shows the main causes of death. In TM patients the most common causes of death were heart damage (16 cases, 40%, Tab. 1), followed by cancer (3 cases, 7.5%, Tab. 1), liver cirrhosis (3 cases, 7.5%, Tab. 1) and infections (3 cases, 7.5%, Tab. 1). In TI patients the most common causes of death were cancer (2 cases, 38.5%, Tab. 1), followed by infections (3 cases, 23.1% , Tab. 1), heart damage (2 case, 15.4%, Tab. 1). Kaplan-Meir curves showed statistically significant difference in TM versus TI survival (log-rank test, p- value<0.0001; Figure 1A). Survival was higher for TI subjects (median age was 73.6 years). Cox regression models of TM versus TI suggested that risk of death for TM patients was 6.8 times higher than TI patients (HR 6.8 (3.3), p- value<0.0001). However, the introduction of chelation treatment (DFO, 1965), changed the Kaplan-Meier curves showing that there was not statistically significant difference between TM versus TI patients in life expectancy ( log-rank test, p- value=0.086; Fig. 1B). Conclusion. These results suggest as TM survival, after the introduction of chelation treatment, improved so much that nowadays it is not different in comparison with TI one's. Moreover, the TM risk of death has been decreased from 6.8 to 2.8 (Cox Model HR 2.8 (1.7), p- value=0.099). These findings, if further confirmed, suggest as, in Western countries, our approach for genetic counselling of "at risk couples" for TM should be reconsidered. Table 1. Causes of death in Thalassemia Major and Thalassemia Intermedia patients. Diagnosis Causes of Death TM n (%) TI n (%) Cancer 3 (7,5) 5 (38,5) Heart Damage 16 (40,0) 2 (15,4) Infection 3 (7,5) 3 (23,1) Multi Organ Failure 1 (2,5) 0 (0,0) Stroke 1 (2,5) 0 (0,0) Liver Failure 3 (7,5) 1 (7,7) Not Available 11 (27,5) 1 (7,7) Other complications not related to Thalassemia 2 (5,0) 1 (7,7) Total 13 40 Figure 1. Kaplan-Meier Survival curves of Thalassemia Major versus Thalassemia Intermedia patients before and after the introduction of chelation treatment (DFO, 1965). Figure 1. Kaplan-Meier Survival curves of Thalassemia Major versus Thalassemia Intermedia patients before and after the introduction of chelation treatment (DFO, 1965). Disclosures Pepe: Chiesi: Speakers Bureau; ApoPharma Inc: Speakers Bureau; Novartis: Speakers Bureau.

2009 ◽  
Vol 46 (5) ◽  
pp. 928-933 ◽  
Author(s):  
K. D. McSporran

Local recurrence of marginally excised subcutaneous soft tissue sarcomas is variable and difficult to predict. This study aimed to identify predictors of local recurrence after excisional biopsy. Medical records of 236 dogs from which tumors had been received between 2004 and 2007 were analyzed. Medium- to large-breed dogs, median age 10 years, were most commonly affected. A total of 139 tumors were graded histologically: 71 were grade 1 (51%); 59, grade 2 (42%); and 9, grade 3 (7%). Of these, 34 tumors (25%) were completely excised, and 104 (75%) were marginally excised. None of 30 completely excised tumors with follow-up information recurred. Three of 41 grade 1 tumors (7%), 14 of 41 grade 2 tumors (34%), and 3 out of 4 grade 3 tumors recurred after marginal excision. Kaplan-Meier survival curves were generated to evaluate survival and the tumor-free interval. The log-rank test and log-rank test for trend were used for comparisons. Tumor recurrence-free intervals for dogs with grade 1 and 2 tumors and for those with grade 1 and 3 tumors differed significantly ( P = .0027 and .0001, respectively) and overall were inversely related to tumor grade ( P = .0007). Kaplan-Meier survival curves, regardless of recurrence, for patients with grade 1, 2, or 3 tumors treated by marginal excision did not differ significantly, and none differed from the survival curves of patients treated by complete excision. In conclusion, histologic grade is a strong predictor for recurrence of marginally excised subcutaneous soft tissue sarcomas. Clean margins predict nonrecurrence. Tumor recurrence did not significantly reduce survival time.


2021 ◽  
Vol 8 (Supplement_1) ◽  
pp. S378-S379
Author(s):  
Guillermo Rodriguez-Nava ◽  
Goar Egoryan ◽  
Daniela Patricia Trelles-Garcia ◽  
Maria Adriana Yanez-Bello ◽  
Qishuo Zhang ◽  
...  

Abstract Background Growing evidence supports the use of remdesivir and tocilizumab for the treatment of hospitalized patients with severe COVID-19. The purpose of this study was to evaluate the use of remdesivir and tocilizumab for the treatment of severe COVID-19 in a community hospital setting. Methods We used a de-identified dataset of hospitalized adults with severe COVID-19 according to the National Institutes of Health definition (SpO2 &lt; 94% on room air, a PaO2/FiO2 &lt; 300 mm Hg, respiratory frequency &gt; 30/min, or lung infiltrates &gt; 50%) admitted to our community hospital located in Evanston Illinois, between March 1, 2020, and March 1, 2021. We performed a Cox proportional hazards regression model to examine the relationship between the use of remdesivir and tocilizumab and inpatient mortality. To minimize confounders, we adjusted for age, qSOFA score, noninvasive positive-pressure ventilation, invasive mechanical ventilation, and steroids, forcing these variables into the model. We implemented a sensitivity analysis calculating the E-value (with the lower confidence limit) for the obtained point estimates to assess the potential effect of unmeasured confounding. Figure 1. Kaplan–Meier survival curves for in-hospital death among patients treated with and without steroids The hazard ratio was derived from a bivariable Cox regression model. The survival curves were compared with a log-rank test, where a two-sided P value of less than 0.05 was considered statistically significant. Figure 2. Kaplan–Meier survival curves for in-hospital death among patients treated with and without remdesivir The hazard ratio was derived from a bivariable Cox regression model. The survival curves were compared with a log-rank test, where a two-sided P value of less than 0.05 was considered statistically significant. Results A total of 549 patients were included. The median age was 69 years (interquartile range, 59 – 80 years), 333 (59.6%) were male, 231 were White (41.3%), and 235 (42%) were admitted from long-term care facilities. 394 (70.5%) received steroids, 192 (34.3%) received remdesivir, and 49 (8.8%) received tocilizumab. By the cutoff date for data analysis, 389 (69.6%) patients survived, and 170 (30.4%) had died. The bivariable Cox regression models showed decreased hazard of in-hospital death associated with the administration of steroids (Figure 1), remdesivir (Figure 2), and tocilizumab (Figure 3). This association persisted in the multivariable Cox regression controlling for other predictors (Figure 4). The E value for the multivariable Cox regression point estimates and the lower confidence intervals are shown in Table 1. Figure 3. Kaplan–Meier survival curves for in-hospital death among patients treated with and without tocilizumab The hazard ratio was derived from a bivariable Cox regression model. The survival curves were compared with a log-rank test, where a two-sided P value of less than 0.05 was considered statistically significant. Figure 4. Forest plot on effect estimates and confidence intervals for treatments The hazard ratios were derived from a multivariable Cox regression model adjusting for age as a continuous variable, qSOFA score, noninvasive positive-pressure ventilation, and invasive mechanical ventilation. Table 1. Sensitivity analysis of unmeasured confounding using E-values CI, confidence interval. Point estimate from multivariable Cox regression model. The E value is defined as the minimum strength of association on the risk ratio scale that an unmeasured confounder would need to have with both the exposure and the outcome, conditional on the measured covariates, to explain away a specific exposure-outcome association fully: i.e., a confounder not included in the multivariable Cox regression model associated with remdesivir or tocilizumab use and in-hospital death in patients with severe COVID-19 by a hazard ratio of 1.64-fold or 1.54-fold each, respectively, could explain away the lower confidence limit, but weaker confounding could not. Conclusion For patients with severe COVID-19 admitted to our community hospital, the use of steroids, remdesivir, and tocilizumab were significantly associated with a slower progression to in-hospital death while controlling for other predictors included in the models. Disclosures All Authors: No reported disclosures


Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 4484-4484
Author(s):  
Smith Giri ◽  
Nunnery Sara ◽  
Syed S. Nasir ◽  
Michael G Martin

Abstract Background: Limited data exists regarding the characteristics and outcomes of adolescents and young adults (AYAs) with acute myeloid leukemia (AML) which are largely under-represented in both pediatric and adult trials. We sought to compare the characteristics and outcomes of AYAs with AML using a large population based registry in the United States. Methods: We utilized Surveillance Epidemiology and End Results (SEER)-18 registry to identify all pediatric (0-18 years) and AYA (age 19-30 years) patients diagnosed with AML using appropriate histology codes based on the International Classification of Diseases for Oncology, 3rd version. Patients with acute promyelocytic leukemia (APL) were excluded from all analysis. Survival statistics were computed for each group using actuarial (Kaplan-Meier method) and compared using Z test for comparison of population proportions. Early mortality, defined as mortality within 1 month of diagnosis, was used as a surrogate for treatment related mortality. Kaplan Meier survival curves were plotted and compared using log-rank test. Multivariate analysis was done using logistic regression and Cox proportional hazard regression model. All p values were two sided and the level of significance was chosen at 0.05. Results: A total of 6343 eligible patients were identified, which comprised 2836 (44.7%) AYAs. A total of 52% (n=3346) were males, whereas 76%(n=4825) were whites. Histologically, majority of patients (56%; n=3545) were categorized as AML, not otherwise specified, followed by acute monocytic leukemia (9.9%, n=630). Majority (55%; n-3509) of the patients were diagnosed between 2001-2012. The early mortality rate was lower in the pediatric AML patients (pAML) as compared to AYAs (6.2% vs 9.2%; p<0.01). Similarly the 1 year (70.3% versus 62.1%; p <0.01) and 5 year (48.2% vs 36.4%; p<0.01) was higher in pediatric patients as compared to AYAs. Kaplan Meier plot showed worse overall survival of AYAs compared to pAMLs (Figure 1; p value of log rank <0.01). Multivariate logistic regression showed higher early mortality among AYAs as compared to pAML patients (OR 1.48; 95% CI 1.23-1.79; p<0.01). Similarly Cox regression showed worse overall survival among AYAs as compared to pAML (HR 1.34; 95% CI 1.26-1.44; p <0.01) Conclusions: Our population based analysis shows worse overall survival among AYAs as compared to pAML patients. Future clinical trials specifically focused on this age group are warranted to establish appropriate treatment regimens in this population. Figure 1. Kaplan Meier Survival curve showing cumulative survival among pediatric patients with AML as compared to AYAs. Log rank test showed statistically significant difference between the two curves (p value <0.01) Figure 1. Kaplan Meier Survival curve showing cumulative survival among pediatric patients with AML as compared to AYAs. Log rank test showed statistically significant difference between the two curves (p value <0.01) Disclosures No relevant conflicts of interest to declare.


2016 ◽  
Vol 34 (2_suppl) ◽  
pp. 268-268
Author(s):  
Beerinder S. Karir ◽  
Panagiotis J. Vlachostergios ◽  
Paul J. Christos ◽  
Victor RA Febles ◽  
Kavya Pinto-Chengot ◽  
...  

268 Background: Various clinical and pathologic criteria have been proposed to identify neuroendocrine (NE) or aggressive variant (AV) prostate cancer (PC). We assessed the prognostic value of clinical parameters in a single-institution cohort. Methods: An IRB-approved database was screened for clinical and/or pathologic criteria (Table 1) for NE/AV PC. Patients with advanced CRPC not meeting any of the criteria served as contemporary controls. Overall survival (OS) for each group was assessed using Kaplan-Meier method and comparisons with log-rank test. Results: 249 men were identified, median age 71.5 (45.1-90.8 years). 145 patients met at least 1 criterion suggestive of NE/AV PC, whereas 104 were CRPC only. Median OS for each subgroup, the combined NE/AV PC group, and the CRPC cohort are provided in Table 1. OS for NE/AV PC vs. CRPC cohort was 25.4 vs 33 months (p = 0.26). Patients with parenchymal brain metastasis had the worst survival of 5.2 mo [95%CI 2.1, 8.3]. On multivariate analysis, bulky high-grade disease in prostate/pelvis carried the highest risk of death (HR 1.71 [1.07, 2.74; p = 0.02]). Conclusions: A number of clinical and pathologic criteria have been used to define NE/AV PC for clinical practice or trial enrollment. Some criteria are associated with a shorter survival than others. Additional studies are warranted to further define both prognostic and molecularly defined subgroups. [Table: see text]


2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e15124-e15124 ◽  
Author(s):  
Srividya Srinivasamaharaj ◽  
Dhruv Ranchhodbhai Chaudhary ◽  
Xiaoyong Wu ◽  
Shesh Rai ◽  
Mary Ann Sanders ◽  
...  

e15124 Background: Metastatic ovarian involvement in primary gastrointestinal (GI) carcinomas (CA) is associated with a poor prognosis. We performed a survival analysis in patients with ovarian metastases, based on site of primary GI CA (appendiceal, colorectal (CRC), gastric, and pancreatic). We also examined the association between hyperthermic intraperitoneal chemotherapy (HIPEC) and death in these patients. Methods: A search was conducted in a single institution pathology database for patients with primary GI CA and ovarian metastases diagnosed from 2010 to 2017. The search yielded 39 patients, and data pertaining to tumor characteristics and treatment were obtained by chart review. Chi-square (log rank) test was used to test for associations between both site of primary GI CA and HIPEC, and death, and Kaplan-Meier analysis was done. P-value < 0.05 was deemed statistically significant. Results: CRC accounted for the majority of patients (51.29%) with appendiceal CA accounting for 23.08% and gastric and pancreatic cancer making up the remainder. Primary site of malignancy was associated with survival (p = 0.036), favoring those with appendiceal and colorectal primaries. A total of 30 patients (76.92%) received HIPEC. Having undergone HIPEC was significantly associated with survival (p = 0.017). Conclusions: Ovarian metastases secondary to gastric and pancreatic cancer were associated with inferior survival as compared to those with appendiceal or colorectal primaries. A significant association was demonstrated between HIPEC and survival. Further investigation to define the role of HIPEC in the treatment of carcinomas of gastrointestinal primaries is warranted.


Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 533-533 ◽  
Author(s):  
Smith Giri ◽  
Valerie Shostrom ◽  
Krishna Gundabolu ◽  
KM Monirul Islam ◽  
Ranjan Pathak ◽  
...  

Abstract Introduction: Prior studies in cancer have suggested better OS of patients treated at AC as compared to NAC. This may be related to the availability of physicians with expertise in specific malignancies, better multidisciplinary care and access to more resources and clinical trials. Whether academic status of the facility affects OS of AML is unknown. Methods: We utilized the National Cancer Database Participant User File (NCDB PUF) to extract patient-level data of patients with AML reported between 1998 to 2011. Hospital facilities were classified as either AC (academic/research program) or NAC (community cancer program, comprehensive community cancer program, and other, per NCDB classification).We included only those patients, who had all of the first course treatment or a decision not to treat made at the reporting facility. Subjects with complete and known data for the variables sex, age, race, education, income, distance traveled for health care, hospital type, facility location, urban/rural location, insurance, Charlson co-morbidity score, chemotherapy use, time from diagnosis to treatment initiation, use of hematopoietic stem cell transplant, 30-day mortality, last contact, and vital status were included. These variables were analyzed in a univariate analysis. Kaplan Meier curves were drawn and compared using log rank test. Multivariate analysis was done using logistic regression for 30-day mortality and Cox regression with backward elimination approach for OS. Statistical analysis was done using PC SAS version 9.4. Results: A total of 7823 AML patients were studied, of which 4681 (60%) patients received treatment at AC. Patients treated at AC differed from NAC in the median age (62 vs. 67years; p <0.001), race (p <0.001), education (p=0.005), income (p <0.001), co-morbidity score (p=0.019), insurance (p<0.001), receipt of chemotherapy (p<0.001), transplant (p<0.001) and facility location (p<0.001). The median OS (12.6 vs. 7.0 months; p value <0.001) and 1-year OS (51% vs. 39%; p value <0.001) was better in AC as compared to NAC. In a multivariate analysis, the 30-day mortality was significantly worse in NAC as compared to AC (odds ratio, OR 1.52; 95% confidence interval, CI 1.33-1.74; p <0.001) (Table 1). Similarly, Cox regression showed that the OS was significantly worse in NAC as compared to AC (hazard ratio, HR 1.13; 95% CI 1.07-1.19; p <0.001) after adjusting for age, sex, Charlson co-morbidity score, receipt of chemotherapy, transplant, insurance and income status and facility location. Conclusion: Our population-based study suggests that the receipt of initial therapy at AC versus NAC is associated with lower 30-day mortality and higher 1-year OS. This may presumably be related to the provision of dedicated multidisciplinary leukemia teams, access to more resources and clinical trials in AC. The reasons behind such differences should be investigated in future studies, and necessary steps be taken to minimize this gap. Table 1. Multivariate logistic regression of 30-day mortality Variable Odds ratio 95% confidence interval P value Academic (ref) Non-Academic 1 1.52 1.33-1..74 <0.001 Age - <60 years (ref) - > 60 years 1 2.32 1.92-2.80 <0.001 Charlsonco-morbidity score -0 (ref) -1 - 2 or more 1 1.45 2.14 1.23-1.69 1.74-2.63 <0.001 <0.001 Chemotherapy - Yes (ref) - No 1 2.55 1.93-3.38 <0.001 Days until first treatment 0.87 0.86-0.89 <0.001 Income status - $ 46,000 + (ref) - < $ 30,000 - 30,000-34,999 - 35,000-45,999 1 1.31 1.31 1.21 1.06-1.62 1.09-1.58 1.03-1.43 0.011 0.004 0.023 Insurance Status - Private insurance/managed care (ref) - Not insured - Medicaid - Medicare - Other government 1 2.32 0.81 1.56 1.13 1.66-3.24 0.59-1.10 1.30-1.87 0.63-2.02 <0.001 0.178 <0.001 0.686 Figure 1. Kaplan Meier curve showing cumulative survival among AML patients treated at academic versus non-academic centers (p value of log rank test <0.001) Figure 1. Kaplan Meier curve showing cumulative survival among AML patients treated at academic versus non-academic centers (p value of log rank test <0.001) Disclosures No relevant conflicts of interest to declare.


2015 ◽  
Vol 33 (7_suppl) ◽  
pp. 191-191 ◽  
Author(s):  
Christoph A. J. von Klot ◽  
Alena Boeker ◽  
Thomas R. W. Herrmann ◽  
Mario W. Kramer ◽  
Markus A. Kuczyk ◽  
...  

191 Background: It is common practice to continue anti-androgen therapy in terms of androgendeprivation when performing chemotherapy or androgendeprivation with new second generation therapeutic agents such as enzalutamide or abiraterone acetate. Clinical Studies aiming at the question whether continuation of conventional ADT is necessary in this setting are currently recruiting. In this study we analyzed androgen deprivation in patients with mCRPC under chemotherapy and second generation androgen suppression. Methods: Out of 620 screened patients a total of 36 patients with continuous testosterone monitoring and mCRPC underwent therapy with docetaxel, abiraterone acetate, enzalutamide, carboplatin, carbozantinib or cabazitaxel and were evaluated. Data were gathered from our center over a median follow up period of 27.8 (0.6 - 65.1) month. A cutoff of 0.5 ng/dL was used to discriminate patients according to testosterone castration levels. Statistical evaluation was performed applying Kaplan Meier survival estimates, Cox regression and log rank test. Results: Median follow up was 26.2 month (range 1.4 - 64.8 month). Mean patient age was 70.9 years (range 51 - 86 years). The mean testosterone concentration in our cohort was 0.5 ng/dL. Serum testosterone levels varied greatly: ranging from 0 to 16 ng/dL. A total of 18 patients died during follow up. Median survival over all patients according to Kaplan-Meier survival estimation was 38.7 month (95% CI: 31 - NA month). Median survival for patients with testosterone levels below and above 0.5 ng/dL were 48.67 and 18.13 month respectively (log rank test: p = 0.0029). In Cox regression analysis, the hazard ratio for risk of death for patients with testosterone concentrations > 0.5ng/dL was 6.03 (95% CI: 1.5 - 25, p - 0.0132). For the covariates PSA velocity, patient age and primary Gleason score there was no significant effect on risk of death (p = 0.0597, 0.5006, 0.7354). Conclusions: In patients with mCRPC i.e rising PSA or progression under androgen deprivation, conventional suppression of testosterone levels still represents a vital factor for overall survival even at the mCRPC stage and under therapy with second line anti hormonal therapeutic medication and chemotherapy.


2020 ◽  
Vol 3 (3) ◽  
pp. 82-86
Author(s):  
Gede Wirya Kusuma Duarsa ◽  
Oka Udrayana ◽  
Yeni Kandarini ◽  
Raka Widiana ◽  
Marleen

Background. To determine risk factors that influence peritonitis event on patients with Continuous Ambulatory Peritoneal Dialysis (CAPD) in Sanglah Hospital, thus, we can prevent the occurrence of peritonitis in CRF patients with CAPD. Methods. This is a retrospective cohort study, which was conducted at the Sanglah Hospital in Denpasar from August to September 2016. All data are processed using SPSS 17.0 for Windows, data analysis by using the Kaplan Meier (K-M) curves, hypothesis using the log-rank test, while for the survival is by using the median or mean survival. The significance is determined by the value of p < 0.05 with 95% CI. Results. A total of 78 people (46 men and 32 women) who meet the inclusion criteria of this study. Thirteen people (16.7%) are experiencing peritonitis. K-M Survival Curves between in CRF patients with CAPD, with Age ≥ 50 years (51.36 months survival rates, 95% CI 44.79 until 57.93) with < 50 years (56.1 months Survival rates, 95% CI 51.41 until 60.78) with RR 2.54 log-rank p 0.084. K-M Survival Curves between in CRF patients with CAPD, with DM (mean 52.63 months survival rates, 95% CI 47.21 until 58.06) with No DM (56.88 months survival rates, 95% CI 52.89 until 60.88) with RR 4.16 and 0.037 log-rank p. Conclusion. There is a correlation between DM and the incidence of peritonitis in CRF patients with CAPD at Sanglah Hospital. However, age and education level are not related.


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