High Prognostic Value of a 3-Genes Molecular Score in Non-High Risk Acute Promyelocytic Leukemia Treated with AIDA 2000 Protocol: A Retrospective Study from a Regional Register

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 2601-2601
Author(s):  
Guardo Daniela ◽  
Fabio Guolo ◽  
Paola Minetto ◽  
Marino Clavio ◽  
Livia Giannoni ◽  
...  

Abstract BACKGROUND AND AIMS Non high risk acute promyelocytic leukemia (APL) patients, as defined by WBC count at diagnosis, have nowadays a very good prognosis when treated with ATRA plus chemotherapy based protocols, with high rate of complete molecular remission after consolidation therapy. However, a small proportion of patients (roughly 10-15%) will eventually relapse, despite the achievement of molecular CR. In the past years, some groups showed that relapse risk could be predicted by testing for some gene alterations, such as FLT3-ITD, or by break point cluster region (BCR) analysis. However, those results were not confirmed in prospective trials. We retrospectively applied a simple 3 gene based molecular panel to low-intermediate risk acute promyelocytic patients, alongside with BCR analysis, in order to develop a risk score. MATERIALS AND METHODS Fifty-nine low/intermediate risk APL patients, treated with the Italian age-adapted AIDA protocol from January 1st 2004 to December 31st 2014 in our center were retrospectively included in this study. Median age was 47 years (range 19-88), 16 patients were older than 60 years (27%). BCR analysis was available in all patients, 41 patients showed BCR1/2, whereas 18 patients had BCR3 (30%). Molecular profile included determination of FLT3-ITD mutation, WT1 and BAALC gene expression levels and was available in 38/59 patients (64%); bone marrow sample of diagnosis is available for all other 21 patients for further analysis. Cut-off values for WT1 (20000/abl x 104) and BAALC (450/abl x 104) were arbitrarily chosen after pre-analysis and comparison with our published data. Five patients had FLT3-ITD mutation (13%), 18 (45%) patients had higher WT1 expression levels and BAALC was overexpressed in 9 (24%) patients. Pre-analysis showed that FLT3-ITD, low WT1 levels, high BAALC levels but not the presence of BCR3 were associated with higher relapse risk. A molecular score including those 3 genes was built: 16 patients had no risk factors (42%), 17 patients had one risk factor (45%), 5 had two risk factors (13%). RESULTS Fifty-four patients survived induction, all of them achieved CR, in 52 PML/RARα transcript was undetectable after last consolidation therapy. The two patients with molecular persistence of disease received further therapy and then achieved molecular CR. Nine patients (15%) relapsed after a median follow-up of 56 months, 45 patients are alive at the time of analysis (76%). Relapse risk was influenced only by the presence of at least one molecular risk factor (p<0.05), whereas age at diagnosis older than 60 years had only a borderline impact (p=0.105); none of the molecular alterations reached statistical significance if taken alone. Disease free survival (DFS) duration was significantly higher in patients who had none of the molecular risk factor, when compared to patients with at least one (3-years DFS of 100% and 70.8%, respectively, p<0.05, Figure 1). Younger patients had also a longer DFS, with a 3-years DFS of 90.7% and 72% for patients younger or older than 60 years, respectively (p<0.03). Overall survival (OS) analysis led to similar results: patients with no molecular risk factors had a very good outcome when compared to patients with at least one alteration (3-years OS of 100% and 71.5%, respectively, p<0.03). Survival was also influenced by age at diagnosis, as younger patients did significantly better, with 3-years OS of 90% (p<0.05). Again, none of the molecular alterations, if taken alone, had a significant impact on DFS or OS. CONCLUSIONS Even in our small cohort, molecular profile could identify a subset of low-intermediate APL patients at higher risk of relapse, who may take benefit from an intensified consolidation therapy, as it is currently applied to high risk patients defined by WBC at diagnosis. Figure 1. DFS according to molecular risk score Figure 1. DFS according to molecular risk score Disclosures No relevant conflicts of interest to declare.

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 2992-2992 ◽  
Author(s):  
Pau Montesinos ◽  
Edo Vellenga ◽  
Aleksandra Holowiecka ◽  
Chelo Rayon ◽  
Gustavo Milone ◽  
...  

Abstract Background: Pseudotumor cerebri associated with all-trans-retinoic acid (ATRA) treatment in acute promyelocytic leukemia (APL) have been frequently described in pediatric patients. However, the incidence, outcome and risk factors of pseudotumor cerebri in APL are not well-known. We analyze the incidence and risk factors of this complication in a large series of patients with newly diagnosed APL enrolled in three consecutive trials of the PETHEMA Group (LPA96, LPA99 and LPA2005). Mehods: AIDA regimen (ATRA 45 mg/m2/d [25mg/m2/d in patients younger than 20] until CR and idarubicin 12 mg/m2/d on days 2, 4, 6 and 8) was given as induction therapy. Patients in CR received 3 monthly courses of risk-adapted consolidation therapy: idarubicin 5 mg/m2/d × 4 (course #1), mitoxantrone 10 mg/m2/d × 5 (course #2), and idarubicin 12 mg/m2/d × 1 (course #3). Since November 1999 (LPA99 trial), for patients with intermediate or high risk of relapse (Sanz et al, Blood 2000), consolidation was slightly intensified by increasing idarubicin doses in courses #1 and #3, and by simultaneously administering 25 mg/m2 ATRA together with chemotherapy in all three courses. Since July 2005, consolidation therapy in the ongoing LPA 2005 trial included the following modifications: the administration of ATRA for all patients; for low- and intermediate-risk patients, mitoxantrone has been reduced from five to three days in the second course; and for high-risk patients, cytarabine has been added to idarubicin in the first and third course. Maintenance therapy consisted of 50 mg/m2/d mercaptopurine orally, 15 mg/m2/week methotrexate intramuscularly, and 25 mg/m2/d ATRA for 15 days every three months. Diagnosis of pseudotumor cerebri was made in the presence of signs and symptoms of intracranial hypertension without clinical or radiological evidence of infective or space occupying lesions. Results: Of 1034 patients enrolled between November 1996 and July 2008, 32 (3%) experienced pseudotumor cerebri. Headaches without pseudotumor were present in 252 patients (25%). Thirty cases of pseudotumor occurred during induction therapy and 2 cases manifested only during consolidation. In all, 9 of 32 patients (28%) had recurrent pseudotumor cerebri after reinitiating ATRA. All these side effects were transient, reversible, and never a cause of death. CR rates were 96% and 90% in patients with and without pseudotumor cerebri, respectively (p=0.32). The incidence of pseudotumor cerebri among patients younger than 18 years, 18–25 years, 25–50 years and older than 50 years was 13%, 7%, 2% and 0.3%, respectively (p&lt;0.0001). There was a trend toward a correlation between fibrinogen &lt;170 mg/dL and worse general state (ECOG&gt;1) at presentation and development of pseudotumor cerebri (p=0.08 and p=0.06, respectively). We did not found any significant association between pseudotumor cerebri and WBC, platelets, relapse risk-score, hemoglobin, creatinine, PETHEMA trial, gender, morphological subtype, PML/RARA isoform, FLT3 mutations, and surface antigens (CD2, CD11b, CD13, CD15, CD34, CD56, and CD117). Conclusion: This study shows an overall incidence of pseudotumor cerebri of 3% among APL patients treated with ATRA and anthracycline-based regimens, with higher incidences in children and young adults (13% and 7%, respectively). No other prognostic factors could be demonstrated. The development of pseudotumor cerebri was not associated with a worse induction outcome. Side effects were reversible and transient, but roughly a third of patients had recurrent pseudotumor cerebri after reinitiating ATRA


Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 3450-3450 ◽  
Author(s):  
Boglarka Gyurkocza ◽  
Rainer Storb ◽  
Barry E. Storer ◽  
Thomas Chauncey ◽  
Thoralf Lange ◽  
...  

Abstract Abstract 3450 Objective: Allogeneic hematopoietic cell transplantation (HCT) following a variety of reduced-intensity conditioning regimens has been reported to produce encouraging results in patients with AML. In these studies disease relapse was the main cause of treatment failure, with 2–4 year relapse rates ranging between 32–61% resulting in overall survivals of 28–45% at 2–4 years. Our goal here was to examine whether pre-HCT variables could identify patients at high risk for relapse following nonmyeloablative allogeneic HCT, who thus would become candidates for additional interventions to reduce the risk of AML relapse. Methods: The data were derived from 274 consecutive Seattle Consortium patients (median age: 60 years) with de novo or secondary AML who underwent allogeneic HCT from related or unrelated donors after conditioning with 2 Gy total body irradiation (TBI) with or without fludarabine (90 mg/m2) as recently reported (Gyurkocza et al., JCO 2010 Jun 10;28(17):2859–2867). Cox regression was used to perform multivariate analysis of risk factors for relapse in a subset of 231 patients in morphologic leukemia-free state (defined as less than 5% marrow blasts) with (n=134) or without (n=97) peripheral blood cell count recovery (defined as platelets > 100,000/ml and neutrophils > 1,000/ml) at the time of HCT. In this multivariate model, AML beyond 1st complete remission (CR1), unfavorable cytogenetics (according to SWOG criteria), incomplete peripheral blood cell count recovery before HCT, and shorter time between diagnosis and HCT were associated with statistically significantly higher risk of relapse (Table 1). From this multivariate model we developed a relapse risk score that summarizes the contribution of multiple risk factors by assigning weights based on the relative magnitude of the log hazard ratios associated with the principal risk factors. From a starting score of 0, points were added or subtracted based on the following factors: 2nd CR: +1 point; 3rd or later CR: +2 points; unfavorable cytogenetics: +1 point; absence of pre-HCT peripheral blood cell count recovery: +0.5 points; time from diagnosis to HCT > 18 months, -2 points (Table 2). Patients were then stratified into 2 relapse risk groups according to whether the total risk score was ≤0 (low-risk) or >0 (high-risk). Results: Stratification of patients according to the proposed Relapse Risk Score resulted in a clear separation of the two risk groups, with 5-year relapse rates of 50% and 17% in the high- and low-risk groups, respectively (Figure 1A). Five-year overall survival rates were 26% and 50% in the high- and low-risk groups, respectively (Figure 1B). Conclusion: Our Relapse Risk Score may be a useful tool to identify patients with AML at high risk for relapse, who could potentially benefit from additional interventions to reduce the risk of relapse following allogeneic HCT. We are currently attempting validation in an independent cohort of patients with AML to make this Risk Score more generalizable. Relapse/Progression (A) and Overall Survival (B) rates stratified by Relapse Risk Score. Disclosures: Off Label Use: Off label usage of fludarabine, cyclosporine, tacrolimus and mycophenolate mofetil is discussed.


2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Sandra Chamat-Hedemand ◽  
Niels Eske Bruun ◽  
Lauge Østergaard ◽  
Magnus Arpi ◽  
Emil Fosbøl ◽  
...  

Abstract Background Infective endocarditis (IE) is diagnosed in 7–8% of streptococcal bloodstream infections (BSIs), yet it is unclear when to perform transthoracic (TTE) and transoesophageal echocardiography (TOE) according to different streptococcal species. The aim of this sub-study was to propose a flowchart for the use of echocardiography in streptococcal BSIs. Methods In a population-based setup, we investigated all patients admitted with streptococcal BSIs and crosslinked data with nationwide registries to identify comorbidities and concomitant hospitalization with IE. Streptococcal species were divided in four groups based on the crude risk of being diagnosed with IE (low-risk < 3%, moderate-risk 3–10%, high-risk 10–30% and very high-risk > 30%). Based on number of positive blood culture (BC) bottles and IE risk factors (prosthetic valve, previous IE, native valve disease, and cardiac device), we further stratified cases according to probability of concomitant IE diagnosis to create a flowchart suggesting TTE plus TOE (IE > 10%), TTE (IE 3–10%), or “wait & see” (IE < 3%). Results We included 6393 cases with streptococcal BSIs (mean age 68.1 years [SD 16.2], 52.8% men). BSIs with low-risk streptococci (S. pneumoniae, S. pyogenes, S. intermedius) are not initially recommended echocardiography, unless they have ≥3 positive BC bottles and an IE risk factor. Moderate-risk streptococci (S. agalactiae, S. anginosus, S. constellatus, S. dysgalactiae, S. salivarius, S. thermophilus) are guided to “wait & see” strategy if they neither have a risk factor nor ≥3 positive BC bottles, while a TTE is recommended if they have either ≥3 positive BC bottles or a risk factor. Further, a TTE and TOE are recommended if they present with both. High-risk streptococci (S. mitis/oralis, S. parasanguinis, G. adiacens) are directed to a TTE if they neither have a risk factor nor ≥3 positive BC bottles, but to TTE and TOE if they have either ≥3 positive BC bottles or a risk factor. Very high-risk streptococci (S. gordonii, S. gallolyticus, S. mutans, S. sanguinis) are guided directly to TTE and TOE due to a high baseline IE prevalence. Conclusion In addition to the clinical picture, this flowchart based on streptococcal species, number of positive blood culture bottles, and risk factors, can help guide the use of echocardiography in streptococcal bloodstream infections. Since echocardiography results are not available the findings should be confirmed prospectively with the use of systematic echocardiography.


2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 1232.1-1232
Author(s):  
M. Di Battista ◽  
S. Barsotti ◽  
A. Della Rossa ◽  
M. Mosca

Background:Cardiovascular (CV) diseases, namely myocardial infarction and stroke, are not among the most known and frequent complications of systemic sclerosis (SSc), but there is growing evidence that SSc patients have a higher prevalence of CV diseases than the general population [1].Objectives:To compare two algorithms for CV risk estimation in a cohort of patients with SSc, finding any correlation with clinical characteristics of the disease.Methods:SSc patients without previous myocardial infarction or stroke were enrolled. Traditional CV risk factors, SSc-specific characteristics and ongoing therapies were assessed. Framingham and QRISK3 algorithms were then used to estimate the risk of develop a CV disease over the next 10 years.Results:Fifty-six SSc patients were enrolled. Framingham reported a median risk score of 9.6% (IQR 8.5), classifying 24 (42.9%) subjects at high risk, with a two-fold increase of the mean relative risk in comparison to general population. QRISK3 showed a median risk score of 15.8% (IQR 19.4), with 36 (64.3%) patients considered at high-risk. Both algorithms revealed a significant role of some traditional risk factors and a noteworthy potential protective role of endothelin receptor antagonists (p=0.003). QRISK3 was also significantly influenced by some SSc-specific characteristics, as limited cutaneous subset (p=0.01), interstitial lung disease (p=0.04) and non-ischemic heart involvement (p=0.03), with the first two that maintain statistically significance in the multivariate analysis (p=0.02 for both).Conclusion:QRISK3 classifies more SSc patients at high-risk to develop CV diseases than Framingham, and it seems to be influenced by some SSc-specific characteristics. If its predictive accuracy were prospectively verified, the use of QRISK3 as a tool in the early detection of SSc patients at high CV risk should be recommended.References:[1]Ngian GS, Sahhar J, Proudman SM, Stevens W, Wicks IP, Van Doornum S. Prevalence of coronary heart disease and cardiovascular risk factors in a national cross-sectional cohort study of systemic sclerosis. Ann Rheum Dis. 2012;71:1980-3.Disclosure of Interests:None declared


2016 ◽  
Vol 34 (2) ◽  
pp. 117-122 ◽  
Author(s):  
Brenda J. Weigel ◽  
Elizabeth Lyden ◽  
James R. Anderson ◽  
William H. Meyer ◽  
David M. Parham ◽  
...  

Purpose Patients with metastatic rhabdomyosarcoma (RMS), except those younger than 10 years with embryonal RMS, have an estimated long-term event-free survival (EFS) of less than 20%. The main goal of this study was to improve outcome of patients with metastatic RMS by dose intensification with interval compression, use of the most active agents determined in phase II window studies, and use of irinotecan as a radiation sensitizer. Patients and Methods Patients with metastatic RMS received 54 weeks of therapy: blocks of therapy with vincristine/irinotecan (weeks 1 to 6, 20 to 25, and 47 to 52), interval compression with vincristine/doxorubicin/cyclophosphamide alternating with etoposide/ifosfamide (weeks 7 to 19 and 26 to 34), and vincristine/dactinomycin/cyclophosphamide (weeks 38 to 46). Radiation therapy occurred at weeks 20 to 25 (primary) but was also permitted at weeks 1 to 6 (for intracranial or paraspinal extension) and weeks 47 to 52 (for extensive metastatic sites). Results One hundred nine eligible patients were enrolled, with a median follow-up of surviving patients of 3.8 years (3-year EFS for all patients, 38% [95% CI, 29% to 48%]; survival, 56% [95% CI, 46% to 66%]). Patients with one or no Oberlin risk factor (age > 10 years or < 1 year, unfavorable primary site of disease, ≥ three metastatic sites, and bone or bone marrow involvement) had a 3-year EFS of 69% (95% CI, 52% to 82%); high-risk patients with two or more risk factors had a 3-year EFS of 20% (95% CI, 11% to 30%). Toxicity was similar to that on prior RMS studies. Conclusion Patients with metastatic RMS with one or no Oberlin risk factor had an improved 3-year EFS of 69% on ARST0431 compared with an historical cohort from pooled European and US studies; those with two or more risk factors have a dismal prognosis, and new approaches are needed for this very-high-risk group.


Circulation ◽  
2015 ◽  
Vol 132 (suppl_3) ◽  
Author(s):  
Norrina B Allen ◽  
Lihui Zhao ◽  
Lei Liu ◽  
Martha Daviglus ◽  
Kiang Liu ◽  
...  

Introduction: We sought to determine the association of CV health at younger ages with the proportion of life lived free of morbidity, the cumulative burden of morbidity, and average healthcare costs at older ages. Methods: The Chicago Heart Association (CHA) study is a longitudinal cohort of employed men and women aged 18-59 years at baseline exam in 1967-1973. Baseline risk factor levels included blood pressure, cholesterol, diabetes, BMI and smoking. Individuals were classified into one of four strata: favorable levels of all factors, 0 factors high but 1+ elevated, 1 high, and ≥2 high risk factors. Linked CMS/NDI data from 1984-2010 were used to determine morbidity in older age providing up to 40 years of follow-up. We included participants who were age 65+ between 1984 and 2010 and enrolled in Medicare FFS. All-cause morbidity was defined using the Gagne score. A CV morbidity score was defined as the sum of 4 CVDs including CHD (includes MI), PVD, cerebrovascular disease and CHF. Results: We included 25,390 participants (43% female, 90% White, mean age 44 at baseline); 6% had favorable levels, 19% had 1+ risk factors at elevated levels, 40% had 1 high risk factor and 35% had 2+ high risk factors. As compared to those with 2+ high risk factors, favorable CV health had lower levels of all-cause and CV morbidity from age 65-90 years, and a lower cumulative morbidity burden (p<0.001) translating to lower average annual healthcare costs ($15,905 vs $20,791 per year, p<0.001). Favorable CV health postponed the onset of all-cause morbidity by 4.5 years, the onset of CV morbidity by almost 7 years and extended life by almost 4 years resulting in a compression of morbidity on both the absolute and relative scale (see figure). Conclusion: Individuals in favorable CV health live a longer, healthier life and a greater proportion of life free of morbidity. These findings provide support for prevention efforts aimed at preserving cardiovascular health and reducing the burden of disease in older ages.


2018 ◽  
Vol 58 (6) ◽  
pp. 1125 ◽  
Author(s):  
B. J. Horton ◽  
R. Corkrey ◽  
G. N. Hinch

In eight closely recorded Australian Merino and crossbred sheep flocks, all lamb deaths were examined and the cause of deaths identified if possible. Dystocia was identified as one of the major causes of lamb death and this study examined factors that could be used to identify ewes at high risk of dystocia, either to avoid dystocia or to assist with early intervention where possible. Dystocia was least common in lambs of ~4.8 kg, but there was a higher risk at both lower and higher birthweights. Dystocia with both low and high birthweight was more common in older ewes, ranging from negligible low birthweight dystocia in ewes less than 3 years old at lambing, to 5% in older ewes. Low birthweight dystocia increased with increasing litter size, with 40% dystocia in ewes at least 4 years of age with triplets. In contrast, high birthweight dystocia was not affected by litter size. A previous record of low birthweight dystocia was a risk factor for future low birthweight dystocia, but the same relationship was not observed for high birthweight dystocia. A high lambing ease score (difficult birth) with high birthweight was a risk factor for future high birthweight dystocia, but this was not the case for low birthweight dystocia. These differences between the risk factors for low and high birthweight dystocia suggest that they have different causes. High ewe liveweight and condition score during pregnancy may be additional indicators of the risk of dystocia, particularly for ewes with high liveweight in the first 60 days of pregnancy. For most ewes dystocia was difficult to predict, but there was a small proportion of ewes with a very high risk of dystocia and if these could be identified in advance they could be monitored much more closely than the rest of the flock.


2009 ◽  
Vol 98 (3) ◽  
pp. 164-168 ◽  
Author(s):  
J. Virkkunen ◽  
M. Venermo ◽  
J. Saarinen ◽  
J. Salenius

Background and Aims: The ability to predict post-operative mortality reliably will be of assistance in making decisions concerning the treatment of an individual patient. The aim of this study was to test the GAS score as a predictor of post-operative mortality in vascular surgical patients. Material and Methods: A total of 157 consecutive patients who underwent an elective vascular surgical procedure were included in the study. The Cox proportional hazards model was used in analyzing the importance of various preoperative risk factors for the postoperative outcome. ASA and GAS were tested in predicting the short and long-term outcome. On the basis of the GAS cut-off value 77, patients were selected into low-risk (GAS low: GAS < 77) and high-risk (GAS high: GAS > = 77) groups, and the examined risk factors were analyzed to determine which of them had predictive value for the prognosis. Results: None of the patients in the GAS low group died, and mortality in the GAS high group was 4.8% (p = 0.03) at 30 days' follow-up. The 12-month survival rates were 98.6% and 78.6% (p = 0.0001), respectively, with the respective 5-year survival rates of 76.7% and 44.0% (p = 0.0001). The only independent risk factor for 30-day mortality was the renal risk factor (OR 20.2). The combination of all three GAS variables(chronic renal failure, cardiac disease and cerebrovascular disease), excluding age, was associated with a 100% two-year mortality. Conclusions: Mortality is low for patients with GAS<77. For the high-risk patients (GAS> = 77), due to its low predictive value for death, GAS yields limited value in clinical practice. In cases of patients with all three risk factors (renal, cardiac and cerebrovascular), vascular surgery should be considered very carefully.


Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4501-4501
Author(s):  
Xiaoyu Zhu ◽  
Jiang Zhu ◽  
Baolin Tang ◽  
Kaidi Song ◽  
Linlin Jin ◽  
...  

Introduction Pre-engraftment syndrome (PES) is a common immune reaction prior to neutrophil engraftment after unrelated cord blood transplantation (UCBT), with a unique clinical manifestation of non-infectious fever and skin rash. The reported incidence of PES ranges from 20% to 78%. Although many researchers believe that PES is associated with a high incidence of acute graft-versus-host disease (GVHD) but not with transplant-related mortality (TRM) , relapse, or overall survival (OS), they did not stratify the risk factors of PES, and how to carry out different doses of methylprednisolone (MP) stratified intervention therapy still remains unknown. Method s First, 136 hematological malignancy patients treated with UCBT from April 2000 to February 2012 in our transplantation center were retrospectively analysis. Among them, 92 patients occurred PES. High-risk factors for 180-day TRM in PES patients were established by univariate and multivariate analysis. Then, from January 2013 to August 2016, 221 PES patients were scored according to the risk scoring system and stratified treated with different doses of MP. Finally, in order to validate the efficacy of MP stratification treatment, we conducted a prospective, open label and non-randomized clinical trial including 240 PES patients who underwent UCBT from September 2016 to December 2018. This trial is registered at www.chictr.org.cn as ChiCTR-ONC-16009013. Results The cumulative incidence of neutrophil and platelet engraftment was significantly higher in PES group than non-PES group (97.8% vs 70.5%, P<0.001; 75.0% vs 54.5%, P=0.05). In 92 PES patients, multivariate analysis showed that failed MP treatment, multiple clinical symptoms and early onset of PES were independent high risk factors affecting180-day TRM. One high risk factor was scored as 1. The 92 PES patients were divided into PES-0, PES-1,PES-2 and PES-3, and the higher the score, the higher the TRM (17.7% vs 21.9% vs 62.5% vs 100%,respectively; P<0.001), and the lower the OS (68.3% vs 56.2% vs 25.0% vs 0%, respectively; P<0.001). Then, from January 2013 to August 2016, 221 PES patients were scored as PES-0, PES-1 and PES-2 according to the following two high risk factors (multiple clinical symptoms and early onset of PES) and stratified treated with different doses of MP (0.5mg/kg/d for PES-0, 1mg/kg/d for PES-1 and 2mg/kg/d for PES-2). Compared to the previous PES patients with the same risk score, the 180-day TRM of PES-1 and PES-2 patients was significantly reduced and the OS, disease free survival (DFS), and GVHD-free and Relapse-free survival (GRFS) were significantly increased after stratified treatment. The results in the prospective trial were similar to the retrospective study. In addition, although stratified therapy could significantly improve the prognosis of PES-2 patients cohort, the cumulative incidence of acute GVHD and GRFS are still the worst compared with other risk score patients. Therefore, how to improve the outcomes of PES-2 patients remains to be further studied. Conclusion s PES after UCBT is benefit for engraftment, but should be graded according the risk scoring system. Different doses of MP stratified intervention therapy can significantly improve the prognosis of severe PES patients. The risk scoring system of PES after UCBT and MP stratification treatment are worthy of clinical application. But the cumulative incidence of acute GVHD and GRFS in severe PES patients still need to be ameliorated in the further study. Disclosures No relevant conflicts of interest to declare.


2019 ◽  
Vol 10 (1) ◽  
pp. 40-47
Author(s):  
Nazma Akter

Background: Diabetes mellitus (DM) is considered as one of the major health problems worldwide. The rising prevalence of type 2 diabetes mellitus (T2DM) in Bangladesh is primarily attributed to rapid urbanization and associated changes in lifestyle, such as sedentary lifestyle, higher calorie food intake and stressful life. Studies support the utilization of riskassessment scoring systems in quantifying individual’s risk for developing T2DM. Thus, a simple risk-assessment scoring system for early screening of T2DM among Bangladeshi adults will be beneficial to identify the high-risk adults and thus taking adequate preventive measures in combating DM.The purpose of the study was to calculate the risk assessment score of developing T2DM within 10 years among Bangladeshi adults. Methods: The cross-sectional observational study was carried out in the outpatient department (OPD) of Medicine, MARKS Medical College & Hospital, a tertiary care hospital in Dhaka, Bangladesh from February 2018 to July 2018 among randomly sampled 205 adult subjects. Subjects undiagnosed with diabetes mellitus and had previous history of high blood glucose during pregnancy or other health examination (i.e. impaired fasting glucose, impaired glucose tolerance or gestational diabetes mellitus) were included. From a review of literature regarding risk factors of developing DM in Bangladesh, the Finnish Diabetes Risk Score (FINDRISC) system was found to be more useful for the Bangladeshi adults. The Finnish Diabetes Risk Score (FINDRISC) questionnaire was used to collect the data including demographic characteristics and different risk factors and to calculate total risk score for predicting the risk of developing T2DM within 10 years. Results: Among 205 subjects, male and female were 57.1% and 42.9% respectively. The Mean (±SD) age of the study subjects was 37.64±1.07 years. In this study, both non-modifiable and modifiable risk factors showed statistically significant association with the FINDRISC among Bangladeshi adults (p<0.05). There was a significant association among FINDRISC with history of previous high blood glucose, and treated hypertensive Bangladeshi adults.33.65% of the Bangladeshi adults had slightly elevated diabetes risk score (DRS). This study predicts that 17.55% of the Bangladeshi adults may have moderate to high risk to develop T2DM within the consecutive 10 years. Conclusion: This study provides a simple, feasible, non-invasive and convenient screening FINDRISC tool that identifies individuals at risk of having T2DM. People with high risk of DM should be referred for early intervention and changes to a healthy lifestyle and primary prevention to prevent or delay the onset of T2DM. Birdem Med J 2020; 10(1): 40-47


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