scholarly journals A risk model for relapsed/refractory aggressive NHL integrating clinical risk factors and pretransplant Deauville score

2020 ◽  
Vol 4 (22) ◽  
pp. 5762-5771
Author(s):  
Ho-Young Yhim ◽  
Yael Eshet ◽  
Ur Metser ◽  
Chae-Hong Lim ◽  
Katherine Lajkosz ◽  
...  
Keyword(s):  
Risk Factors ◽  
High Risk ◽  
International Prognostic Index ◽  
Prognostic Index ◽  
Salvage Chemotherapy ◽  
Low Risk ◽  
Clinical Risk Factors ◽  
Intermediate Risk ◽  
Clinical Risk ◽  
Deauville Score

Abstract There are limited data regarding the combined value of the pretransplant Deauville score (DS) from a positron emission tomography scan and clinical risk factors in patients with relapsed/refractory aggressive non-Hodgkin lymphoma (NHL). We performed a retrospective analysis to assess the prognostic role of pretransplant DS in patients with relapsed/refractory aggressive NHL who underwent salvage chemotherapy and autologous stem cell transplantation (ASCT). We identified 174 eligible patients between January 2013 and March 2019. In multivariable analysis, pretransplant DS, B symptoms, and secondary International Prognostic Index (sIPI) were independent risk factors for event-free survival (EFS). These variables were used to derive an integrated risk score that categorized 166 patients with available information for all risk factors into 3 groups: low (n = 92; 55.4%), intermediate (n = 48; 28.9%), and high (n = 26; 15.7%). The new prognostic index showed a strong association with EFS (low-risk vs intermediate-risk hazard ratio [HR], 3.94; 95% confidence interval [CI], 2.16-7.17; P < .001; low-risk vs high-risk HR, 10.83; 95% CI, 5.81-20.19; P < .001) and outperformed models based on clinical risk factors or DS alone. These results were validated in 60 patients from an independent external cohort (low-risk vs intermediate-risk HR, 4.04; 95% CI, 1.51-10.82; P = .005; low-risk vs high-risk HR, 10.49; 95% CI, 4.11-26.73; P < .001). We propose and validate a new prognostic index that risk-stratifies patients undergoing salvage chemotherapy followed by ASCT, thereby identifying patients at high risk for posttransplant treatment failure.

scholarly journals Serbian lymphoma study group: Demografic characteristics of 257 patients with follicular lymphoma

2015 ◽  
Vol 72 (6) ◽  
pp. 483-488
Author(s):  
Olivera Simonovic ◽  
Lana Macukanovic-Golubovic ◽  
Bosko Andjelic ◽  
Darko Antic ◽  
Biljana Mihaljevic
Keyword(s):  
High Risk ◽  
Follicular Lymphoma ◽  
International Prognostic Index ◽  
Prognostic Index ◽  
Treatment Decision ◽  
Risk Groups ◽  
Low Risk ◽  
Intermediate Risk ◽  
Prognostic Groups

Background/Aim. Follicular lymphoma (FL) is a B-cell tumor usually with indolent clinical course, yet in some cases the course of the disease can be very aggressive. The aim of the re-search was to determine distribution of patients into prognostic groups based on the International Prognostic Index (IPI) and Folicular Lymphoma International Prognostic Index (FLIPI) criteria, as well as to determine the importance of classifying patients into the prognostic groups, since this could potentially have the influence on selection of the treatment modality. Methods. The retrospective study was performed on 257 patients with follicular lymphoma diagnosed between January 2000 and April 2011. Results. Based on the IPI score, 153 (59.53%) patients had low risk, 57 (22.18%) low intermediate risk, 15 (5.84%) high intermediate risk, 9 (3.50%) high risk, whereas the classification of 23 patients diagnosed with FL remained with unknown risk according to the IPI. Based on the FLIPI prognostic index, 113 (43.97%) patients had low risk, 70 (27.24%) intermediate risk and 51 (19.84%) high risk, whereas the classification of 23 (8.95%) patients remained unknown. On the basis of the FLIPI 2 prognostic index, 48 (18.68%) patients had low risk, 145 (56.42%) intermediate risk and 41 (15.95%) high risk. The classification into prognostic groups for 23 (8.95%) patients remained unknown. According to the IPI, FLIPI and FLIPI 2 there were the patients that required treatment in all the risk groups. Conclusion. The FLIPI and FLIPI 2 effectively identify patients at high risk, thus helping in treatment decision for each single patient.

Validation of Mantle Cell International Prognostic Index (MIPI) in Mantle Cell Lymphoma (MCL) in a Retrospective Single Institution Series

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 2828-2828
Author(s):  
Annalisa Chiappella ◽  
Barbara Botto ◽  
Filippo Marmont ◽  
Ernesta Audisio ◽  
Ileana Baldi ◽  
...  
Keyword(s):  
High Risk ◽  
Clinical Course ◽  
Prognostic Score ◽  
International Prognostic Index ◽  
Prognostic Index ◽  
Low Risk ◽  
Intermediate Risk ◽  
Single Institution ◽  
Mantle Cell

Abstract Introduction: The clinical course of MCL is characterized by a continuous pattern of relapse and a poor long term outcome with a median Overall Survival (OS) of four years and a 15% of long term survivors. Recently a new clinical prognostic score (MIPI), including performance status, age, LDH level and leukocyte count has been reported. This score allows a more reliable estimation of individual clinical course. We retrospectively applied the MIPI score to patients with MCL. Patients and methods: Between 1999 and 2007, 40 patients with MCL diagnosed and treated in a single institution entered into the study. Clinical characteristics were as follows: median age 56 years (range 37–81), 80% male; 82% stage IV; 78% bone marrow involvement and 15% MCL with blastoid variant. First line treatments were: high dose chemoimmunotherapy including Rituximab (R) with autologous stem cell transplantation (R-HDC) in 26 patients and Rituximab-CHOP like chemotherapy (R-CHOP) in 14. Crude Kaplan-Meier OS and progression-free survival (PFS) curves were estimated both overall and stratified by MIPI and International Prognostic Index (IPI) score. Differences between curves were tested using the 2-tailed log-rank test. In order to quantify the predictive discrimination of MIPI and IPI scores, univariate logistic models (with death and progression event as binary outcomes) were fitted and the area under the receiver operating characteristic (ROC) curves (c index) was estimated. Results: According to MIPI score 17 patients (43%) were at low risk (LR, score 0–3), 13 patients (32%) at intermediate risk (IR, score 4–5) and 10 patients (25%) at high risk (HR, score >5). According to IPI score 14 patients (35%) were at low risk (LR), 16 patients (40%) at low-intermediate risk (LIR) and 10 patients (25%) at intermediate-high and high risk (IH-HR). At the end of the treatment, 30 patients achieved a CR, five a PR and five did not respond. Relapses occurred in 17 patients and seven of them died of lymphoma. With a median follow-up (FU) of 29 months, OS was 85% (95% CI: 66%–93%); with a median FU of 21 months, PFS was 70% (95% CI: 51%–83%). Twenty-nine months OS rates for MIPI score were: LR 100%, IR 81%, HR 66% respectively (p=.07) and for IPI score were: LR 92%, LIR 94%, IH-HR 65% respectively (p=.09). Twenty-one months PFS rates for MIPI score were: LR 92%, IR 59%, HR 45% respectively (p=.006) and for IPI score were: LR 73%, LIR 87%, IH-HR 44% respectively (p=.09). MIPI score was more predictive than IPI score for the death event and for the progression event: the c index was 74% and 73% for MIPI compared to 72% and 69% for IPI respectively. In a subgroup analysis performed on 26 R-HDC patients, OS and PFS rates stratified for MIPI were: for OS, LR 100% vs IR 80% vs HR 69% (p=.4) and for PFS, LR 91% vs IR 80% vs HR 57% (p=.04) respectively. Discussion: in our retrospective series of patients, MIPI prognostic score discriminates among patients with different PFS. Relapses remain the most important issue for all patients affected by MCL, namely in HR group according to MIPI. New therapeutic strategies are warranted to improve the prognosis of MCL.

scholarly journals Prediction of Venous Thromboembolism Based on Clinical and Genetic Factors

2020 ◽  
Author(s):  
David A. Kolin ◽  
Scott Kulm ◽  
Olivier Elemento
Keyword(s):  
Risk Factors ◽  
Venous Thromboembolism ◽  
High Risk ◽  
Genetic Risk ◽  
Genetic Factors ◽  
Hazard Ratio ◽  
Low Risk ◽  
Clinical Risk Factors ◽  
Clinical Risk ◽  
Increased Risk

BACKGROUNDBoth clinical and genetic factors drive the risk of venous thromboembolism. However, whether clinically recorded risk factors and genetic variants can be combined into a clinically applicable predictive score remains unknown.METHODSUsing Cox proportional-hazard models, we analyzed the association of risk factors with the likelihood of venous thromboembolism in U.K. Biobank, a large prospective cohort. We created a novel ten point clinical score using seven established clinical risk factors for venous thromboembolism. We also generated a polygenic risk score of 21 single nucleotide polymorphisms to quantify genetic risk. The genetic score was categorized into high risk (top two deciles of scores), intermediate risk (deciles three to eight), and low risk (lowest two deciles). The discrete clinical score led to the following approximate decile categorizations: high risk (5 to 10 points), intermediate risk (3 to 4 points), and low risk (0 to 2 points).RESULTSAmongst the 502,536 participants in the U.K. Biobank, there were 4,843 events of venous thromboembolism. Analyses of established clinical risk factors and the most commonly used medications revealed that participants were at decreased risk of venous thromboembolism if they had ever used oral contraceptive pills (hazard ratio, 0.88; 95% confidence interval [CI], 0.79 to 0.99) or if they currently used bendroflumethiazide (hazard ratio, 0.84; 95% CI, 0.74 to 0.95), cod liver oil capsules (hazard ratio, 0.87; 95% CI, 0.77 to 0.99), or atenolol (hazard ratio, 0.79; 95% CI, 0.68 to 0.91). Participants were at significantly increased risk of venous thromboembolism if they were at high clinical risk (hazard ratio, 5.98; 95% CI, 5.43 to 6.59) or high genetic risk (hazard ratio, 2.28; 95% CI, 2.07 to 2.51) relative to participants at low clinical or genetic risk, respectively. Combining clinical risk factors with genetic risk factors produced a model that better predicted risk of venous thromboembolism than either model alone (P<0.001). Participants at high clinical and genetic risk in the combined score had over an eightfold increased risk of venous thromboembolism relative to participants at low risk (hazard ratio, 8.27; 95% CI 7.59 to 9.00).CONCLUSIONSBy assessing venous thromboembolic events in over 500,000 participants, we identified several known and novel associations between risk factors and venous thromboembolism. Participants in the high risk group of a combined score, consisting of clinical and genetic factors, were over eight times more likely to experience venous thromboembolism than participants in the low risk group.

Predicting Early Mortality in Diffuse Large B-Cell Non-Hodgkin Lymphoma

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 5411-5411
Author(s):  
Jamie M Maddox
Keyword(s):  
Risk Factors ◽  
High Risk ◽  
International Prognostic Index ◽  
Prognostic Index ◽  
Early Mortality ◽  
Intermediate Risk ◽  
High Risk Patients ◽  
Non Hodgkin Lymphoma ◽  
Risk Patients ◽  
Large B Cell

Abstract It is difficult to find figures for the expected rate of early mortality in diffuse large B-cell non-Hodgkin lymphoma (DLBCL) as many of the patients who are destined to die early do not enter clinical trials. Our own rate of early mortality (death within 100 days of the diagnostic test being performed) was higher than we had anticipated (23%). We undertook a study to look at risk factors for early mortality, and to see if there were any factors which could be improved by altering our investigation and initial management. Our haematology database has baseline demographic and prognostic information on all cases of haematological malignancy diagnosed in our centre. A two year period was chosen retrospectively from 1st January 2013 until 31st December 2014. This gave 97 registered patients with DLBCL. Early mortality was significantly related to the patient age, Eastern Cooperative Oncology Group (ECOG) performance status, lactase dehydrogenase value, presence of 2 or more sites of extranodal disease and the presence of B-symptoms. We did not see a significant relationship to the presence of marrow disease or the presence of disease bulk. As the majority of the relevant factors are already part of validated prognostic scoring systems, we evaluated the (International Prognostic Index) IPI, the R-IPI (revised International Prognostic Index) and the NCCN-IPI (enhanced International Prognostic Index) to see which was the best predictor of early mortality. The IPI gave the chance of 100 day mortality as 4% for low or low-intermediate risk patients, 16% for high-intermediate risk patients and 53% for high risk patients. The R-IPI gave the chance of 100 day mortality as 0% for low risk patients, 5% for intermediate risk patients and 48% for high risk patients. The NCCN-IPI gave the chance of 100 day mortality as 0% for low or low-intermediate risk patients, 13% for high-intermediate risk patients and 57% for high risk patients (figure 1). By six months, the mortality rate in high risk NCCN-IPI patients had reached 71% while the low and low-intermediate groups remained at 0%. Some patients were included who were not considered for potentially curative chemotherapy. Even with these patients excluded, the risk of 100 day mortality was still 50% in the high risk NCCN-IPI group. Some of the risk factors for mortality will likely worsen if the diagnosis or initial treatment are delayed. We therefore looked at the overall pathway from referral until first treatment. We found that those with early mortality tended to have a shorter time course until receiving their first treatment, likely reflecting the fact that they were more unwell when they first presented. Disclosures Maddox: Janssen: Other: Funding to attend ASH 2016 (travel, accommodation, registration); Boehringer-Ingelheim: Other: Funding to attend ASH 2014 (travel, accommodation, registration).

scholarly journals Is the International Prognostic INDEX for CLL (CLL-IPI) Useful to Predict Time to First Treatment of Patients with EARLY Disease? Results of a Prospective Multicenter Analysis

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 498-498 ◽  
Author(s):  
Stefano Molica ◽  
Diana Giannarelli ◽  
Luciano Levato ◽  
Rosanna Mirabelli ◽  
Massimo Gentile ◽  
...  
Keyword(s):  
High Risk ◽  
Conflicts Of Interest ◽  
Risk Group ◽  
International Prognostic Index ◽  
Prognostic Index ◽  
Low Risk ◽  
Intermediate Risk ◽  
Early Disease ◽  
Binet Stage ◽  
Time To First Treatment

Abstract BACKGROUND: The CLL-IPI score is a large cooperative effort in which clinical data collected from 8 randomized trials were used to develop an internationally applicable prognostic index for CLL patients. The model includes 5 independent parameters that predict for overall survival such as age, clinical stage, del(17p) and/or TP53 mutation, IGHV mutation status and β2-microglobulin (B2M) level. A potential limitation for an extensive use of CLL-IPI is represented, however, by the fact that only 20% of patients included in the full analysis set had early disease. PATIENTS: The present analysis based on an observational multicenter CLL database including 337 Binet stage A patients (O-CLL1 protocol, clinicaltrial.gov identifier NCT00917540) was designed to assess the utility of the CLL-IPI score to predict time to first treatment (TTFT) in patients with early disease. RESULTS: Patients were followed up for a total of 2038 person-years (median, 42 months; range, 1-82 months), during which 91 (26.9%) experienced disease-progression requiring therapy according to 1996 IWCLL guidelines. The CLL-IPI score enabled Binet stage A patients to be divided into three subgroups [i.e., score 0-1, low-risk (n=229); score 2-3, intermediate-risk(n=99); score 4 or higher, high-risk (n=9)] that differed with respect to TTFT (P<0.0001). A comparative performance analysis between CLL-IPI and 2007 MD Anderson Cancer Center (MDACC) score, barely based on traditional clinical parameters (i.e., age, gender, Rai substage, absolute lymphocyte count, number of involved nodal groups and B2M), revealed that prediction of the TTFT was more accurate with the former. The c-statistic of the MDACC model was 0.62 (95% CI: 0.49-0.75) a level below than that of the CLL-IPI (c=0.70; 95% CI:0.58-0.81) and below the accepted 0.7 threshold necessary to have value at the individual patient level. These results are in keeping with the change in area under the receiver operating characteristic (ROC) curve (AUC) which increased from 0.646 (95% CI: 0.578-0.714) to 0.720 (95%CI:0.658-0.783) when moving from MDACC model to CLL-IPI score. Since the CLL-IPI score was originally derived from patients with active CLL enrolled in phase 3 trials we sought for different cut-off scores that better predict for TTFT in our patient cohort of early CLL. According to the recursive partitioning (RPART) analysis, a classification tree was built that identified three subsets of patients who scored 0 (low- risk,n=139), 1(intermediate-risk, n=90) and >1 (high-risk, n=108), respectively. The probability of remaining free from therapy at 5 years was 85% in the low-risk group, 68% in the intermediate-risk group and 47% in the high-risk group (P<0.0001)(Fig 1). Our revised IPI score remained a predictor of TTFT also when analysis was limited to 262 Rai stage 0 (P<0.0001) and 99 clinical monoclonal B-cell lymphocytosis (cMBL) cases (P=0.006). CONCLUSIONS: The results of this study confirm the utility of CLL-IPI score for predicting TTFT in a prospective cohort of community-based patients with early CLL at presentation. Our effort to adapt CLL-IPI score to patients with early disease meets the need to separate Binet stage A patients into different prognostic groups suitable for individualized follow-up programmes and possibly for early therapeutic interventions. Figure 1. Figure 1. Disclosures No relevant conflicts of interest to declare.

Prediction of Preeclampsia and Delivery of Small for Gestational Age Babies Based on a Combination of Clinical Risk Factors in High-Risk Women

2010 ◽  
Vol 30 (1) ◽  
pp. 58-73 ◽  
Author(s):  
Paul T. Seed ◽  
Lucy C. Chappell ◽  
Michael A. Black ◽  
Katrina K. Poppe ◽  
Yuan-Chun Hwang ◽  
...  
Keyword(s):  
Risk Factors ◽  
High Risk ◽  
Gestational Age ◽  
Small For Gestational Age ◽  
Clinical Risk Factors ◽  
High Risk Women ◽  
Clinical Risk

scholarly journals Simple electrocardiographic measures improve sudden arrhythmic death prediction in coronary disease

2020 ◽  
Vol 41 (21) ◽  
pp. 1988-1999 ◽  
Author(s):  
Neal A Chatterjee ◽  
Jani T Tikkanen ◽  
Gopi K Panicker ◽  
Dhiraj Narula ◽  
Daniel C Lee ◽  
...  
Keyword(s):  
Risk Factors ◽  
High Risk ◽  
Risk Stratification ◽  
Validation Cohort ◽  
Clinical Risk Factors ◽  
Left Ventricular Ejection ◽  
Derivation Cohort ◽  
Arrhythmic Death ◽  
Clinical Risk ◽  
Ecg Score

Abstract Aims To determine whether the combination of standard electrocardiographic (ECG) markers reflecting domains of arrhythmic risk improves sudden and/or arrhythmic death (SAD) risk stratification in patients with coronary heart disease (CHD). Methods and results The association between ECG markers and SAD was examined in a derivation cohort (PREDETERMINE; N = 5462) with adjustment for clinical risk factors, left ventricular ejection fraction (LVEF), and competing risk. Competing outcome models assessed the differential association of ECG markers with SAD and competing mortality. The predictive value of a derived ECG score was then validated (ARTEMIS; N = 1900). In the derivation cohort, the 5-year cumulative incidence of SAD was 1.5% [95% confidence interval (CI) 1.1–1.9] and 6.2% (95% CI 4.5–8.3) in those with a low- and high-risk ECG score, respectively (P for Δ &lt; 0.001). A high-risk ECG score was more strongly associated with SAD than non-SAD mortality (adjusted hazard ratios = 2.87 vs. 1.38 respectively; P for Δ = 0.003) and the proportion of deaths due to SAD was greater in the high vs. low risk groups (24.9% vs. 16.5%, P for Δ = 0.03). Similar findings were observed in the validation cohort. The addition of ECG markers to a clinical risk factor model inclusive of LVEF improved indices of discrimination and reclassification in both derivation and validation cohorts, including correct reclassification of 28% of patients in the validation cohort [net reclassification improvement 28 (7–49%), P = 0.009]. Conclusion For patients with CHD, an externally validated ECG score enriched for both absolute and proportional SAD risk and significantly improved risk stratification compared to standard clinical risk factors including LVEF. Clinical Trial Registration https://clinicaltrials.gov/ct2/show/NCT01114269. ClinicalTrials.gov ID NCT01114269.

The Follicular Lymphoma International Prognostic Index (FLIPI) Predicts Treatment Outcome in Patients with Advanced Stage Follicular Lymphoma Treated Front-Line with Rituximab and the Combination of Cyclophosphamide, Doxorubicin, Vincristine and Prednisone (R-CHOP).

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 925-925 ◽  
Author(s):  
Christian Buske ◽  
Eva Hoster ◽  
Martin Dreyling ◽  
Joerg Hasford ◽  
Michael Unterhalt ◽  
...  
Keyword(s):  
Treatment Outcome ◽  
Relative Risk ◽  
High Risk ◽  
Follicular Lymphoma ◽  
Risk Group ◽  
International Prognostic Index ◽  
Prognostic Index ◽  
Intermediate Risk ◽  
Advanced Stage ◽  
Front Line

Abstract Background: The Follicular Lymphoma International Prognostic Index (FLIPI) has been developed to predict prognosis and to allow risk adapted treatment decisions in patients with follicular lymphoma (FL) before the widely use of Rituximab. However, the addition of Rituximab to standard induction chemotherapy with its long-term beneficial effects has profoundly changed the treatment outcome in patients with advanced stage FL and has become the new standard in the first line therapy of this disease. Therefore, we addressed the question, whether the prognostic value of the FLIPI could be reconfirmed in patients with advanced stage FL treated initially with a rituximab/chemotherapy combination. Methods: The FLIPI index was tested in patients treated with Rituximab and CHOP (Cyclophosphamide, Doxorubicin, Vincristine and Prednisone, R-CHOP) in a prospective multicenter phase 3 trial of the GLSG using the time to treatment failure (TTF) as target parameter. Results: 362 Patients treated with R-CHOP were evaluable for TTF. Of the 338 patients evaluable for all FLIPI risk factors, 14% of patients were classified as low, 41% as intermediate and 45% as high risk. After a median follow-up time of 20 months, patients with low risk and intermediate risk FLIPI had almost identical TTF (2-years TTF 92% vs 90%, 95% C.I., 83% to 100% and 84% to 96%, respectively). In contrast, the TTF was significantly shorter in the high risk FLIPI group (2-years TTF 67%, 95% C.I., 58% to 76%) as compared to the combined low/intermediate risk FLIPI group (relative risk 3.0, 95% C.I., 1.7 to 5.1; p &lt; 0.0001). In addition, responding patients with high risk FLIPI had a significantly shorter progression free survival as compared to the low/intermediate risk group (relative risk 3.3, 95% C.I., 1.8 to 6.0; p &lt; 0.0001). When postremission treatment was taken into account, the FLIPI separated the high risk group from the low/intermediate risk group in 65 patients treated with autologous stem cell transplantation (relative risk 6.0, 95% C.I., 1.4 to 25.2) as well as in 242 patients who had received IFN-α maintenance or no postremission therapy (relative risk 3.2, 95% C.I., 1.8 to 5.8). As the FLIPI was able to separate the high from the low/intermediate risk group in patients with advanced follicular lymphoma treated initially with Rituximab and CHOP we next performed a multivariate analysis to determine the impact of the individual parameters incorporated in the FLIPI on the TTF. The serum LDH level greater than the upper normal limit (relative risk 2.6, 95% C.I., 1.5 to 4.5) and the hemoglobin level below 12 g/dl (relative risk 2.5, 95% C.I., 1.4 to 4.3) were independently associated with a shorter TTF in these patients, whereas the age and the number of nodal areas were not discriminant. Conclusion: Taken together, these data indicate that the FLIPI is a valid prognostic index for identifying high-risk patients in FL, also after front-line combined immuno-chemotherapy. The index will remain an important tool to adjust treatment decisions in individual patients according their risk profile and to design clinical trials for the different risk groups in the era of antibody-based therapy.

Risk Stratification for Survival and Leukemic Transformation in Essential Thrombocythemia: A Single Institutional Study of 605 Patients.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 3599-3599
Author(s):  
Naseema Gangat ◽  
Alexandra Wolanskyj ◽  
Rebecca F. McClure ◽  
Chin Y. Li ◽  
Susan M. Schwager ◽  
...  
Keyword(s):  
Risk Factors ◽  
High Risk ◽  
Univariate Analysis ◽  
Low Risk ◽  
Prognostic Models ◽  
Intermediate Risk ◽  
Prognostic Variables ◽  
Leukemic Transformation ◽  
Male Sex

Abstract Background It is widely recognized that advanced age and prior thrombosis predict recurrent thrombosis in essential thrombocythemia (ET) and are used to risk-stratify patients. However, the paucity of large sample size and long-term follow-up has limited the development of similar prognostic models for survival and leukemic transformation (LT). Methods Data was abstracted from the medical records of a consecutive cohort of patients with WHO-defined ET seen at the Mayo Clinic. Cox proportional hazards was used to determine the impact of clinical and laboratory variables on survival and LT. Overall survival and leukemia-free survival was estimated by Kaplan-Meier plots. Results i. Patient characteristics and outcome The study cohort included 605 patients of which 399 (66%) were females (median age, 57 years; range 5–91). Median follow-up was 84 months (range; 0–424). During this period, 155 patients (26%) have died and LT was documented in 20 patients (3.3%) occurring at a median of 138 months (range; 23–422) from ET diagnosis. ii. Prognostic variables for overall survival Univariate analysis of parameters at diagnosis identified age ≥ 60 years, hemoglobin less than normal (defined as < 12 g/dL in females and < 13.5 g/dL in males), leukocyte count ≥ 15 x 109/L, tobacco use, diabetes mellitus, thrombosis, male sex, and the absence of microvascular symptoms as independent predictors of inferior survival. All of the above except the last two (i.e. male sex and the absence of microvascular symptoms) sustained their prognostic significance on multivariate analysis. Based on the first three prognostic variables: age, hemoglobin level, and leukocyte count, we constructed a prognostic model for survival: low-risk (none of the risk factors), intermediate-risk (1of 3 risk factors), and high-risk (≥ 2 risk factors). The respective median survivals were 278, 200, and 111 months (p<0.0001; Figure 1) iii. Prognostic variables for leukemic transformation On univariate analysis of parameters at ET diagnosis, LT was significantly associated with platelet count ≥ 1000 x 109/L, hemoglobin less than normal, and exposure to P-32. However, on multivariate analysis, only hemoglobin less than normal and platelet count ≥ 1000 x 109/L maintained independent prognostic value. Accordingly, we utilized these two variables, to construct a prognostic model for LT: low-risk (none of the risk factors), intermediate-risk (1 risk factor), and high-risk (both risk factors). Only 1 of the 239 patients (0.4%) in the low-risk group vs. 14 of the 289 (4.8%) in the intermediate-risk and 5 of the 77 (6.5%) in the high-risk group underwent LT (p=0.0009; Figure 2). Conclusion The current study provides clinician-friendly prognostic models for both survival and LT in ET. Figure 1 Figure 1. Figure 2 Figure 2.

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