Prognostic mutations identified by whole-exome sequencing and validation of the Molecular International Prognostic Scoring System in myelodysplastic syndromes after allogeneic haematopoietic stem cell transplantation.

In this study, we used the whole-exome sequencing (WES) approach to obtain genomic profiles from 92 marrow samples of myelodysplastic syndrome (MDS) patients before haematopoietic stem cell transplantation. We identified 129 mutations in 45 driver genes. Fifty-five patients (59.8%) carried at least 1 driver mutation. The splicing factor U2AF1 was the most frequently mutated in the cohort (21 cases, 23%), followed by BCOR (9 cases, 10%), ASXL1 (8 cases, 9%), TET2 (6 cases, 7%), NPM1 (5 cases, 5%), RUNX1 (5 cases, 5%), and SETBP1 (5 cases, 5%). WES also identified 49 possible oncogenic variants in six genes (PIEZO1, LOXHD1, MYH13, DNAH5, DPH1, and USH2A) that were associated with overall survival (OS) or relapse-free survival (RFS) in MDS after transplantation. Multivariate analysis showed mutations in DNAH5 and USH2A to be independent risk factors for OS. Mutations in DNAH5 and LOXHD1 were risk factors for worse RFS. The Molecular International Prognostic Scoring System retained its independent prognostic significance for RFS after multivariate analysis.

The indication of palliative whole-brain radiotherapy for patients with brain metastases: a simple prognostic scoring system in the era of stereotactic radiosurgery.

BACKGROUND: Stereotactic irradiation has become the mainstay treatment for brain metastases (BM), and whole-brain radiotherapy (WBRT) is often used for symptom palliation. However, the survival time of patients with BM undergoing palliative WBRT (pWBRT) is limited, making it difficult to select patients who should receive treatment. METHODS: We collected patient data from 2016 to 2022 at the Shizuoka Cancer Center and retrospectively analyzed the factors related to survival time. Overall survival (OS) was defined as the survival time after WBRT. RESULTS: A total of 301 patients (median age, 66 years) who underwent pWBRT were included. The primary cancers were lung, breast, gastrointestinal tract, and other cancers in 203 (67%), 38 (13%), 33 (11%), and 27 (9%) patients, respectively. Median OS of all patients was 4.1 months. In the multivariate analysis, male sex (hazard ratio [HR]:1.4), Karnofsky Performance Status (KPS) ≤ 60 (HR:1.7), presence of extracranial metastasis (ECM) (HR:1.6), neutrophil-lymphocyte ratio (NLR) ≥ 5 (HR:1.6), and lactate dehydrogenase (LDH) ≥ upper limit of normal (ULN) (HR:1.3) were significantly associated with shorter OS (all P < 0.05). To predict the OS, we created a prognostic scoring system (PSS). We gave one point to each independent prognostic factor. Median OS for patients with scores of 0-2, 3, and 4-5 were 9.0, 3.5 and 1.7 months, respectively (P < 0.001). CONCLUSIONS: Male sex, KPS ≤ 60, presence of ECM, NLR ≥ 5, and LDH ≥ ULN were poor prognostic factors for patients with BM undergoing pWBRT. By PSS combining these factors, it may be possible to select patients who should undergo pWBRT.

Prognostic scoring system for pediatric Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis based on baseline characteristics: A multicenter retrospective study.

BACKGROUND: The prognosis of pediatric Epstein-Barr virus (EBV)-associated hemophagocytic lymphohistiocytosis (EBV-HLH) varies. This study aimed to identify high-risk children early. PROCEDURE: Data from 264 children (0-14 years of age), diagnosed with EBV-HLH at six centers in China between January 2016 and December 2021, were analyzed. Patients were randomly divided into derivation (n = 185) and verification (n = 79) cohorts. A Cox regression model was used to explore risk predictors and establish a prognostic scoring system for death events that occurred during the follow-up period. RESULTS: Chronic active EBV infection (CAEBV) history (hazard ratio [HR] 1.82 [95% confidence interval, CI: 1.02-3.26]; p = .0441), plasma EBV-DNA more than 104  copies/mL (HR 2.89 [95% CI: 1.62-5.16]; p = .0003), pulmonary infection (HR 2.24 [95% CI: 1.06-4.75]; p = .0353), digestive tract hemorrhage (HR 2.55 [95% CI: 1.35-4.82]; p = .0041), and hypoxemia (HR 3.95 [95% CI: 2.15-7.26]; p < .0001) were independent risk factors. Accordingly, the CAEBV history, plasma EBV-DNA copy number, pulmonary infection hemorrhage of digestive tract, hypoxemia prognostic scoring system (CEPHO-PSS) were developed, which separated patients into low- (0-1 points), middle- (2-3 points), and high- (4-8 points) risk groups. Survival curves for the three groups exhibited statistically significant differences (p < .0001). Internal and external verification of CEPHO-PSS was performed using receiver operating characteristic (ROC) and calibration curves in the derivation and verification cohorts, respectively, confirming good accuracy and applicability. CONCLUSIONS: The CEPHO-PSS identified three risk groups with statistically significant differences in survival curves. It was based on the baseline characteristics, and can give clinicians a convenient check for risk prediction.

Development and validation of a prognostic scoring system for 1-year mortality in older patients with hip fractures.

BACKGROUND: Hip fractures in older people result in increased mortality. OBJECTIVE: We developed and validated an accurate and simple prognostic scoring system for hip fractures that can be used preoperatively. DESIGN: Retrospective study. SETTING: Multicenter. PARTICIPANTS: Patients aged ≥65 years with hip fractures who underwent surgery between 2011 and 2021 were enrolled. METHODS: The significant factors were determined with logistic regression analysis, and a scoring system was developed. The patients were classified into three groups, and a log-rank test was performed to evaluate 1-year survival rates. The model was internally and externally validated using the 5-fold cross-validation and data from another hospital, respectively. RESULTS: We included 1026 patients. The analysis revealed eight significant prognostic factors: sex, body mass index, history of chronic heart failure and malignancy, activities of daily living (ADLs) before injury, hemoglobin and the prognostic nutritional index (PNI) at injury, and the American Society of Anesthesiologists Physical Status. The area under the receiver operating characteristic curve (AUC) after internal validation was 0.853. The external validation data consisted of 110 patients. The AUC of the model for the validation data was 0.905, showing outstanding discrimination. Sensitivity and specificity were 88.7% vs. 100% and 93.3% vs. 95.2% for the development and validation data, respectively. CONCLUSIONS: We developed and validated an accurate and simple prognostic scoring system for hip fractures using only preoperative factors. Our findings highlight PNI as an important predictor of prognosis in hip fracture patients.

Assessment of survival prediction after surgery in spinal metastases patients using the Global Spine Study Tumor Group (GSTSG) risk calculator; an external validation from a tertiary cancer hospital.

PURPOSE: We aim to validate the Global Spine Tumor Study Group (GSTSG) score compared to previous prognostic scoring systems in spinal metastasis. METHODS: We conducted a retrospective study from January 2013 to December 2022. The survival prediction was compared between the GSTSG, Tomita Score, Revised Tokuhashi Score, and Skeletal Oncology Research Group (SORG) Nomogram. Single-variable factors associated with survival rate were analyzed using univariate Cox regression and multivariable Cox proportional hazard model. Receiver operating characteristic was used for external validity analysis at 3, 6, 12, and 24 months. The overall survival rate was reported using the Kaplan-Meier survival curve. RESULTS: 248 spinal metastasis patients were included. The mean age was 59.23 ± 12.55 years. The mean duration of follow-up time was 470.29 ± 441.98 days. The external validity of GSTSG was the highest at all follow-up times (sufficiently accurate AUC > 0.7), which was about the same as SORG at 3 months (both AUC of GSTSG and SORG = 0.76) and higher than modified Tokuhashi and Tomita score at 12 months (AUC of GSTSG = 0.78, SORG = 0.71, Tomita = 0.64, and modified Tokuhashi = 0.61, respectively). CONCLUSION: From our study, the Multivariate Cox regression analysis indicates that the significant factors related to survival rate are regular analgesic use of weak opioids, lung metastasis, and previous chemotherapy. Compared to other traditional spinal metastases prognostic scoring systems, GSTSG shows the highest AUC for external validity in all follow-up times up to 24 months.

A three-gene leukaemic stem cell signature score is robustly prognostic in chronic myelomonocytic leukaemia.

Leukaemic stem cell (LSC) gene expression has recently been linked to prognosis in patients with acute myeloid leukaemia (17-gene LSC score, LSC-17) and myelodysplastic syndromes. Although chronic myelomonocytic leukaemia (CMML) is regarded as a stem cell disorder, the clinical and biological impact of LSCs on CMML patients remains elusive. Making use of multiple independent validation cohorts, we here describe a concise three-gene expression signature (LSC-3, derived from the LSC-17 score) as an independent and robust prognostic factor for leukaemia-free and overall survival in CMML. We propose that LSC-3 could be used to supplement existing risk stratification systems, to improve prognostic performance and guide management decisions.

Current Therapeutic Landscape in Lower Risk Myelodysplastic Syndromes.

OPINION STATEMENT: Lower risk myelodysplastic syndromes are typically characterized by an indolent disease course with a relatively low risk of transformation into acute myeloid leukemia. These patients are classically identified using the revised International Prognostic Scoring System and most likely its molecular version in the near future which may change the paradigm of treatment. The overall goals of care are symptomatic control to reduce transfusion requirements and improve quality of life. Symptomatic anemia is the most common indication to initiate disease-specific therapies after the optimization of supportive measures. Currently, erythropoiesis-stimulating agents remain the standard upfront therapy for anemia, and patients with del(5q) cytogenetic changes can benefit from lenalidomide monotherapy. Other therapeutic options after failure of upfront treatment include luspatercept, hypomethylating agents, and immunosuppressive therapies after taking into account of individualized disease features. Allogeneic hematopoietic stem cell transplant is the only potentially curative option and is usually reserved for medically fit patients with severe symptomatic cytopenias who failed all standard options and/or the disease is progressing toward higher risk categories. Fortunately, novel investigational therapies are rapidly emerging by targeting different biological processes contributing to MDS pathogenesis, and eligible patients should be managed in clinical trials if available.

Telomere integrated scoring system of myelodysplastic syndrome.

INTRODUCTION: Recently, multigene target sequencing is widely performed for the purpose of prognostic prediction and application of targeted therapy. Here, we proposed a new scoring system that encompasses gene variations, telomere length, and Revised International Prognostic Scoring System (IPSS-R) together in Asian myelodysplastic syndrome. METHODS: We developed a new scoring model of these variables: age ≥ 65 years + IPSS-R score + ASXL1 mutation + TP53 mutation + Telomere length (<5.37). According to this new scoring system, patients were divided into four groups: very good score cutoff (≤3.0), good (3.0-4.5), poor (4.5-7.0), and very poor (>7.0). RESULTS: The median OS was 170.1, 100.4, 46.0, and 12.0 months for very good, good, poor, and very poor, retrospectively (p < 0.001). Meanwhile, according to the conventional IPSS-R scoring system, the median OS was 141.3, 50.2, 93.0, 36.0, and 16.2 months for very low, low, intermediate, high, and very high, retrospectively (p < 0.001). CONCLUSIONS: The newly developed model incorporating molecular variations and TL yielded more clear separations of the survival curves. By adding the presence of gene mutation and telomere length to the existing IPSS-R, its predictive ability can be further improved in myelodysplastic syndrome.

The SLANT Score Predicts Poor Neurologic Outcome in Comatose Survivors of Cardiac Arrest: An External Validation Using a Retrospective Cohort.

BACKGROUND: Hypoxic brain injury is the leading cause of death in comatose patients following resuscitation from cardiac arrest. Neurological outcome can be difficult to prognosticate following resuscitation, and goals of care discussions are often informed by multiple prognostic tools. One tool that has shown promise is the SLANT score, which encompasses five metrics including initial nonshockable rhythm, leukocyte count after targeted temperature management, total adrenaline dose during resuscitation, lack of bystander cardiopulmonary resuscitation, and time to return of spontaneous circulation. This cohort study aimed to provide an external validation of this score by using a database of comatose cardiac arrest survivors from our institution. METHODS: We retrospectively queried our database of cardiac arrest survivors, selecting for patients with coma, sustained return of spontaneous circulation, and use of targeted temperature management to have a comparable sample to the index study. We calculated SLANT scores for each patient and separated them into risk levels, both according to the original study and according to a Youden index analysis. The primary outcome was poor neurologic outcome (defined by a cerebral performance category score of 3 or greater at discharge), and the secondary outcome was in-hospital mortality. Univariable and multivariable analyses, as well as a receiver operator characteristic curve, were used to assess the SLANT score for independent predictability and diagnostic accuracy for poor outcomes. RESULTS: We demonstrate significant association between a SLANT group with increased risk and poor neurologic outcome on univariable (p = 0.005) and multivariable analysis (odds ratio 1.162, 95% confidence interval 1.003-1.346, p = 0.046). A receiver operating characteristic analysis indicates that SLANT scoring is a fair prognostic test for poor neurologic outcome (area under the curve 0.708, 95% confidence interval 0.536-0.879, p = 0.024). Among this cohort, the most frequent SLANT elements were initial nonshockable rhythm (84.5%) and total adrenaline dose ≥ 5 mg (63.9%). There was no significant association between SLANT score and in-hospital mortality (p = 0.064). CONCLUSIONS: The SLANT score may independently predict poor neurologic outcome but not in-hospital mortality. Including the SLANT score as part of a multimodal approach may improve our ability to accurately prognosticate comatose survivors of cardiac arrest.

Development of a prognostic scoring system for hepatic vena cava Budd-Chiari syndrome with hepatocellular carcinoma.

BACKGROUND: Hepatocellular carcinoma (HCC) is a serious complication of hepatic vena cava Budd-Chiari syndrome (HVC-BCS) that significantly reduces the survival time of patients. Our study aimed to analyze the prognostic factors influencing the survival of HVC-BCS patients with HCC and to develop a prognostic scoring system. METHODS: The clinical and follow-up data of 64 HVC-BCS patients with HCC who received invasive treatment at the First Affiliated Hospital of Zhengzhou University between January 2015 and December 2019 were retrospectively analyzed. Kaplan-Meier curves and log-rank tests were used to analyze the survival curve of patients and the difference in prognoses between the groups. Univariate and multivariate Cox regression analyses were performed to analyze the influence of biochemical, tumor, and etiological characteristics on the total survival time of patients, and a new prognostic scoring system was developed according to the regression coefficients of the independent predictors in the statistical model. The prediction efficiency was evaluated using the time-dependent receiver operating characteristics curve and concordance index. RESULTS: Multivariate analysis showed that serum albumin level < 34 g/L [hazard ratio (HR) = 4.207, 95% confidence interval (CI): 1.816-8.932, P = 0.001], maximum tumor diameter > 7 cm (HR = 3.612, 95% CI: 1.646-7.928, P = 0.001), and inferior vena cava stenosis (HR = 8.623, 95% CI: 3.771-19.715, P < 0.001) were independent predictors of survival. A prognostic scoring system was developed according to the above-mentioned independent predictors, and patients were classified into grades A, B, C and D. Significant differences in survival were found among the four groups. CONCLUSIONS: This study successfully developed a prognostic scoring system for HVC-BCS patients with HCC, which is helpful for clinical evaluation of patient prognosis.

Inflammation-based prognostic scoring system for predicting the prognosis of advanced small cell lung cancer patients receiving anlotinib monotherapy.

BACKGROUND: According to the randomized multicenter phase II trial (ALTER1202), anlotinib has been approved as a third-line therapy for advanced small-cell lung cancer (SCLC). Some studies showed the predictive function of inflammatory markers, including neutrophil-to-lymphocyte ratio (NLR), platelet-to-lymphocyte ratio (PLR), and lymphocyte-to-monocyte ratio (LMR) in the different cancers treated with anti-vascular targeting drugs. However, none of the studies showed the roles of NLR, PLR, and LMR in SCLC patients receiving anlotinib. Thus, our objective was to establish a scoring system based on inflammation to individuate patient stratification and selection based on NLR, PLR, and LMR. METHODS: NLR, PLR, and LMR and their variations were calculated in 53 advanced SCLC patients receiving anlotinib as a third- or further-line treatment at Ningbo Medical Center Lihuili Hospital between January 2019 and December 2021. Kaplan-Meier curves were plotted. Both univariate and multivariate Cox regressions were used to identify predictors of survival. RESULTS: Disease control rate was related to pre-NLR, pre-PLR, pre-LMR, post-NLR elevation, post-PLR elevation, and post-LMR elevation. The multivariate analysis determined post-NLR elevation, pre-PLR > 240.56, and pre-LMR ≤1.61 to be independently associated with progression-free survival, not overall survival. The inflammation-based prognostic scoring system demonstrated favorable predictive ability from the receiver operating characteristic curve (AUC: 0.791, 95% CI: 0.645-0.938). CONCLUSIONS: Post-NLR variation, pre-PLR, and pre-LMR were independent prognostic factors for PFS in advanced SCLC receiving anlotinib monotherapy. The inflammation-based prognostic scoring system can accurately predict effectiveness and survival.

[Allogeneic hematopoietic cell transplantation for myelofibrosis: current status and future challenges].

Remarkable progress has been made in the field of myelofibrosis recently. Along with the use of driver gene mutations for diagnosis of myelofibrosis, non-driver gene mutations that affect its prognosis have also been identified, and new prognostic models based on them have been proposed. Furthermore, several important findings have been reported across diverse research fields, such as determining the appropriate modality for reducing splenomegaly before transplantation either by splenectomy or drug therapy, pre-transplant conditioning and donor selection, and long-term follow-up after transplantation. However, due to the relative rarity of myelofibrosis, it is difficult to keep up with the latest findings and develop the best clinical treatment regimens for patients. The purpose of this study is to summarize the current status and recent findings in transplantation therapy for myelofibrosis and to identify the challenges faced during treatment.

Overall survival of myelodysplastic syndrome patients after azacitidine discontinuation and applicability of the North American MDS Consortium scoring system in clinical practice.

BACKGROUND: Azacitidine (AZA) is the standard treatment for myelodysplastic syndromes (MDS); however, many patients prematurely stop therapy and have a dismal outcome. METHODS: The authors analyzed outcomes after AZA treatment for 402 MDS patients consecutively enrolled in the Italian MDS Registry of the Fondazione Italiana Sindromi Mielodisplastiche, and they evaluated the North American MDS Consortium scoring system in a clinical practice setting. RESULTS: At treatment discontinuation, 20.3% of the patients were still responding to AZA, 35.4% of the cases had primary resistance, and 44.3% developed adaptive resistance. Overall survival (OS) was better for patients who discontinued treatment while in response because of planned allogeneic hematopoietic stem cell transplantation (HSCT; median OS, not reached) in comparison with patients with primary resistance (median OS, 4 months) or adaptive resistance (median OS, 5 months) or patients responsive but noncompliant/intolerant to AZA (median OS, 4 months; P = .004). After AZA discontinuation, 309 patients (77%) received best supportive care (BSC), 60 (15%) received active treatments, and 33 (8%) received HSCT. HSCT was associated with a significant survival advantage, regardless of the response to AZA. The North American MDS Consortium scoring system was evaluable in 278 of the 402 cases: patients at high risk had worse OS than patients at low risk (3 and 7 months, respectively; P < .001). The score was predictive of survival both in patients receiving BSC (median OS, 2 months for high-risk patients vs 5 months for low-risk patients) and in patients being actively treated (median OS, 8 months for high-risk patients vs 16 months for low-risk patients; P < .001), including transplant patients. CONCLUSIONS: Real-life data confirm that this prognostic scoring system for MDS patients failing a hypomethylating agent seems to be a useful tool for optimal prognostic stratification and for choosing a second-line treatment after AZA discontinuation.

Prognostic scoring systems and comorbidities in chronic myelomonocytic leukaemia: a nationwide population-based study.

Outcomes in chronic myelomonocytic leukaemia (CMML) are highly variable and may be affected by comorbidity. Therefore, prognostic models and comorbidity indices are important tools to estimate survival and to guide clinicians in individualising treatment. In this nationwide population-based study, we assess comorbidities and for the first time validate comorbidity indices in CMML. We also compare the prognostic power of: the revised International Prognostic Scoring System (IPSS-R), CMML-specific prognostic scoring system (CPSS), MD Anderson Prognostic Scoring System (MDAPS) and Mayo score. In this cohort of 337 patients with CMML, diagnosed between 2009 and 2015, the median overall survival was 21·3 months. Autoimmune conditions were present in 25% of the patients, with polymyalgia rheumatica and Hashimoto's thyroiditis being most common. Of the tested comorbidity indices: the Charlson Comorbidity Index (CCI), Haematopoietic cell transplantation-specific Comorbidity Index (HCT-CI) and Myelodysplastic Syndrome-Specific Comorbidity Index (MDS-CI), CCI had the highest C-index (0·62) and was the only comorbidity index independently associated with survival in multivariable analyses. When comparing the prognostic power of the scoring systems, the CPSS had the highest C-index (0·69). In conclusion, using 'real-world' data we found that the CCI and CPSS have the best prognostic power and that autoimmune conditions are overrepresented in CMML.

Comparing the safety and efficacy of ruxolitinib in patients with Dynamic International Prognostic Scoring System low-, intermediate-1-, intermediate-2-, and high-risk myelofibrosis in JUMP, a Phase 3b, expanded-access study.

Ruxolitinib, a potent Janus kinase 1/2 inhibitor, has demonstrated durable improvements in patients with myelofibrosis. In this analysis of the Phase 3b JUMP study, which included patients aged ≥18 years with a diagnosis of primary or secondary myelofibrosis, we assessed the safety and efficacy of ruxolitinib in patients stratified by Dynamic International Prognostic Scoring System (DIPSS) risk categories. Baseline characteristic data were available to assess DIPSS status for 1844 of the 2233 enrolled patients; 60, 835, 755, and 194 in the low-, intermediate (Int)-1-, Int-2-, and high-risk groups, respectively. Ruxolitinib was generally well tolerated across all risk groups, with an adverse-event (AE) profile consistent with previous reports. The most common hematologic AEs were thrombocytopenia and anemia, with highest rates of Grade ≥3 events in high-risk patients. Approximately, 73% of patients experienced ≥50% reductions in palpable spleen length at any point in the ≤24-month treatment period, with highest rates in lower-risk categories (low, 82.1%; Int-1, 79.3%; Int-2, 67.1%; high risk, 61.6%). Median time to spleen length reduction was 5.1 weeks and was shortest in lower-risk patients. Across measures, 40%-57% of patients showed clinically meaningful symptom improvements, which were observed from 4 weeks after treatment initiation and maintained throughout the study. Overall survival (OS) was 92% at Week 72 and 75% at Week 240 (4.6 years). Median OS was longer for Int-2-risk than high-risk patients (253.6 vs. 147.3 weeks), but not evaluable in low-/Int-1-risk patients. By Week 240, progression-free survival (PFS) and leukemia-free survival (LFS) rates were higher in lower-risk patients (PFS: low, 90%; Int-1, 82%; Int-2, 46%; high risk, 15%; LFS: low, 92%; Int-1, 86%; Int-2, 58%; high risk, 19%). Clinical benefit was seen across risk groups, with more rapid improvements in lower risk patients. Overall, this analysis indicates that ruxolitinib benefits lower-risk DIPSS patients in addition to higher risk.

A radiomics prognostic scoring system for predicting progression-free survival in patients with stage IV non-small cell lung cancer treated with platinum-based chemotherapy.

OBJECTIVE: To develop and validate a radiomics prognostic scoring system (RPSS) for prediction of progression-free survival (PFS) in patients with stage IV non-small cell lung cancer (NSCLC) treated with platinum-based chemotherapy. METHODS: In this retrospective study, four independent cohorts of stage IV NSCLC patients treated with platinum-based chemotherapy were included for model construction and validation (Discovery: n=159; Internal validation: n=156; External validation: n=81, Mutation validation: n=64). First, a total of 1,182 three-dimensional radiomics features were extracted from pre-treatment computed tomography (CT) images of each patient. Then, a radiomics signature was constructed using the least absolute shrinkage and selection operator method (LASSO) penalized Cox regression analysis. Finally, an individualized prognostic scoring system incorporating radiomics signature and clinicopathologic risk factors was proposed for PFS prediction. RESULTS: The established radiomics signature consisting of 16 features showed good discrimination for classifying patients with high-risk and low-risk progression to chemotherapy in all cohorts (All P<0.05). On the multivariable analysis, independent factors for PFS were radiomics signature, performance status (PS), and N stage, which were all selected into construction of RPSS. The RPSS showed significant prognostic performance for predicting PFS in discovery [C-index: 0.772, 95% confidence interval (95% CI): 0.765-0.779], internal validation (C-index: 0.738, 95% CI: 0.730-0.746), external validation (C-index: 0.750, 95% CI: 0.734-0.765), and mutation validation (C-index: 0.739, 95% CI: 0.720-0.758). Decision curve analysis revealed that RPSS significantly outperformed the clinicopathologic-based model in terms of clinical usefulness (All P<0.05). CONCLUSIONS: This study established a radiomics prognostic scoring system as RPSS that can be conveniently used to achieve individualized prediction of PFS probability for stage IV NSCLC patients treated with platinum-based chemotherapy, which holds promise for guiding personalized pre-therapy of stage IV NSCLC.

Novel prognostic scoring system for autologous hematopoietic cell transplantation in multiple myeloma.

We studied 2,528 patients with upfront autologous haematopoietic cell transplantation (AHCT) for multiple myeloma (MM) from 2008-2017 to develop a prognostic model to predict outcomes. High-risk cytogenetics included t(4;14), t(14;16), t(14;20), del13q on karyotype, del17p, +1q or 1pdel. A Cox model identified factors prognostic of progression/relapse in a training subset (n = 1,246). A weighted score using these factors was assigned to a validation cohort (n = 774). Presence of high-risk cytogenetics [hazard ratio, (HR) 1·68 (1·3-2·17)] and pre-AHCT bone marrow plasma cells (BMPCs) ≥10% [1·68 (1·33-2·12)] were assigned 4 points each; albumin at diagnosis <3·5 g/dl [1·31 (1·07-1·61)] 2; standard risk cytogenetics 1, and no cytogenetics abnormality, BMPCs <10% at AHCT and albumin ≥3·5 g/dl at diagnosis 0 points each. A three-category system with low risk (0-3), intermediate risk (4-8) and high risk (9-10) showed 3-year progression-free survival in the low vs. intermediate vs. high risk of 58% (95% CI: 52-63) vs. 49% (95% CI: 43-56) vs. 31% (95% CI: 12-51), P < 0.001 respectively, and 3-year OS in low vs. intermediate vs. high risk of 88% (95% CI: 84-91) vs. 81% (95% CI: 76-86) vs. 64% (95% CI: 39-80); P < 0·001. Our prognostic scoring system can identify MM patients at risk for early relapse after AHCT.

Impact of marrow blasts percentage on high-grade myelodysplastic syndrome assessed using revised international prognostic scoring system.

Clinical trials and treatment guidelines for myelodysplastic syndrome depend on several prognostic scoring systems to stratify patients by risk. These include different variables: the degree of cytopenia, percentage of bone marrow blasts, and cytogenetics. Little is known about the impact of bone marrow blasts in patients with adverse cytogenetics. In this retrospective study, we analyzed 536 patients with high-grade myelodysplastic syndrome to examine the differences in survival for patients with different percentages of bone marrow blasts. The median overall survival in patients with ≥ 5% marrow blasts was not statistically different from that for patients with < 5% marrow blasts; however, the former group had a higher risk of progression to acute myeloid leukemia (p < 0.001). Therefore, cytogenetics is the most important factor in our prognostic tools to determine survival outcomes for patients with myelodysplastic syndrome, and patients with high-risk disease have poor prognosis irrespective of their marrow blasts percentage.

Development and validation of a prognostic nomogram for the overall survival of patients living with spinal metastases.

INTRODUCTION: The primary goal of treatment in spinal metastasis is typically to extend patients' lifespan as much as possible, and optimally to relieve the symptoms and so improve quality of life. It is crucial to avoid over- or under-treatment, according to each patient's individual situation. Thus, this study aimed to identify significant prognostic factors for patients living with metastatic spine disease, and create a new nomogram for the prediction of survival rates. METHODS: Data from patients who had undergone operations for spinal metastasis between 2005 and 2016 were retrieved retrospectively, and randomized into training (70%) and validation groups (30%). A selection of pre-operative factors was analyzed using univariable and multivariable COX model for the training group. A nomogram was then developed using significant predictors in multivariable analysis. Accuracy was validated using a concordance index (C-index) and calibration curve for the training and validation groups, respectively. RESULTS: A total of 244 participants were enrolled, including 171 in the training group and 73 in the validation group. Primary tumor, Frankel Grade, Karnofsky Performance Score (KPS) and adjuvant therapy were found to be significant for predicting survival rates. A nomogram was developed by utilizing these predictors. The C-indexes for the two groups were 0.711 and 0.703 respectively. Moreover, a favorable consistency between the predicted and actual survival probabilities was demonstrated using calibration curves. CONCLUSIONS: A user-friendly nomogram model for facilitating medical procedures during clinical encounters was established to aid clinical decision making for individual patients.

Hematopoietic Cell Transplantation for Myelofibrosis: the Dynamic International Prognostic Scoring System Plus Risk Predicts Post-Transplant Outcomes.

Hematopoietic cell transplantation (HCT) provides potentially curative treatment for patients with myelofibrosis (MF). HCT outcomes are associated with the Dynamic International Prognostic Scoring System (DIPSS) risk scores. In the present study we analyzed results in 233 patients to determine if the DIPSS plus classification, which adds cytogenetics, thrombocytopenia, and RBC transfusion dependence as risk factors, would better predict post-HCT outcomes than the original DIPSS. Multivariate analysis showed that each risk parameter incorporated into the DIPPS plus model contributed to its predictive power of overall mortality, relapse-free survival, and nonrelapse mortality. The 5-year overall survival (OS), relapse, and treatment-related mortality (TRM) rates for patients with low/intermediate-1 risk MF were 78%, 5%, and 20%, respectively. The 5-year OS, relapse, and TRM rates for patients with high-risk MF were 35%, 28%, and 40%, respectively. The HCT-specific comorbidity index of 3 or greater was associated with higher nonrelapse and overall mortality and reduced relapse-free survival. The relapse incidence was significantly increased in older patients (HR, 3.02; P = .0007). With a median follow-up of 8 years 124 patients (53%) were surviving. The components of the DIPSS plus classification still have prognostic relevance after adjustment by the DIPSS classification. This information should enhance our ability to advise patients when making decisions regarding timing of transplant.