Clinical Prognostic Scoring Systems Research Articles

Validation of imatinib therapy failure score (IMTF) in chronic phase chronic myeloid leukemia in real life practice

The outcome of chronic myeloid leukemia (CML) patients improved in the last decade. Clinical prognostic scoring systems aim to provide information about survival in the long-term, without determining from baseline the subset of patients who require a strictly monitoring because at increased risk of failure. Imatinib, the first-generation tyrosine kinase inhibitor (TKI), is still widely used as frontline treatment: recently, the imatinib therapy failure (IMTF) score was proposed to identify the failure free survival. Aim of our study was to validate this index in a large cohort of patients treated with imatinib.

Prediction of lymphoma response to CAR T cells by deep learning-based image analysis.

Clinical prognostic scoring systems have limited utility for predicting treatment outcomes in lymphomas. We therefore tested the feasibility of a deep-learning (DL)-based image analysis methodology on pre-treatment diagnostic computed tomography (dCT), low-dose CT (lCT), and 18F-fluorodeoxyglucose positron emission tomography (FDG-PET) images and rule-based reasoning to predict treatment response to chimeric antigen receptor (CAR) T-cell therapy in B-cell lymphomas. Pre-treatment images of 770 lymph node lesions from 39 adult patients with B-cell lymphomas treated with CD19-directed CAR T-cells were analyzed. Transfer learning using a pre-trained neural network model, then retrained for a specific task, was used to predict lesion-level treatment responses from separate dCT, lCT, and FDG-PET images. Patient-level response analysis was performed by applying rule-based reasoning to lesion-level prediction results. Patient-level response prediction was also compared to prediction based on the international prognostic index (IPI) for diffuse large B-cell lymphoma. The average accuracy of lesion-level response prediction based on single whole dCT slice-based input was 0.82+0.05 with sensitivity 0.87+0.07, specificity 0.77+0.12, and AUC 0.91+0.03. Patient-level response prediction from dCT, using the "Majority 60%" rule, had accuracy 0.81, sensitivity 0.75, and specificity 0.88 using 12-month post-treatment patient response as the reference standard and outperformed response prediction based on IPI risk factors (accuracy 0.54, sensitivity 0.38, and specificity 0.61 (p = 0.046)). Prediction of treatment outcome in B-cell lymphomas from pre-treatment medical images using DL-based image analysis and rule-based reasoning is feasible. This approach can potentially provide clinically useful prognostic information for decision-making in advance of initiating CAR T-cell therapy.

Analysis on echocardiographic data of fetal heart defects at high altitude in Yunnan province and surrounding high altitude areas

Objective: To explore the current situation of fetal heart defects in Yunnan Province and surrounding high altitude areas and the social factors affecting pregnancy outcome. Methods: This is a retrospective study. Pregnant woman who underwent fetal echocardiography and diagnosed as fetal cardiac defects in Yunnan Fuwai Cardiovascular Hospital from June 2017 to January 2021 were included. According to the clinical prognostic risk scoring system and grading criteria of fetal cardiac birth defects, the cases were divided into grade Ⅰ to Ⅳ. The disease distribution and proportion of each prognostic grade, pregnancy outcomes were analyzed and compared. The cases were divided into continued pregnancy group and terminated pregnancy group according to pregnancy outcome. The social factors that may affect the selection of pregnancy outcomes were analyzed by multivariate logistic regression analysis. Results: A total of 4 929 fetal echocardiography examination data were collected, and 4 464 cases (90.57%) were from Yunnan Province and surrounding high altitude areas. 2 166 cases of heart defects were finally analyzed, including 998 cases of congenital heart disease (CHD), 93 cases of cardiac tumors, cardiomyopathy and arrhythmia, 1 075 cases of foramen ovale, ductus arteriosus abnormalities and normal variations. The pregnant women were (29.2±5.0) years old with (25.6±3.8) gestational weeks. The number of cases with prognostic grade from Ⅰ to Ⅳ was 1 037 (47.88%), 620 (28.62%), 314 (14.50%), and 44 (2.03%), respectively. And 151 cases (6.97%) were not classified. The cases of normal variation and thin aortic arch development accounted for 42.66% (924/2 166), 5.22% (113/2 166), respectively. The top 3 diseases of grade Ⅱ were ventricular septal defect, coarctation of aorta and mild-moderate pulmonary stenosis, respectively, and their distribution was 11.63% (252/2 166), 3.92% (85/2 166) and 2.35% (51/2 166) respectively in all cases of heart defects, and 25.25% (252/998), 8.52% (85/998) and 5.11% (51/998) respectively in cases of CHD. Among the cases rated as grade Ⅲ and Ⅳ, most of them were complicated congenital heart disease, and the disease types are scattered. The more common cases in grade Ⅲ were complete transposition of great arteries (accounting for 2.40% (52/2 166) of all cases with heart defects, 5.21% (52/998) of all cases with CHD) and pulmonary artery occlusion (type Ⅰ to Ⅲ) with ventricular septal defect (accounting for 2.17% (47/2 166) of all cases with heart defects, and 4.71% (47/998) of all cases with CHD). In grade Ⅳ, single ventricle (0.74% (16/2 166) of all cases with heart defects, 1.60% (16/998) of all cases with CHD) and left ventricular dysplasia syndrome (0.65% (14/2 166) of all cases with heart defects, 1.40% (14/998) of all cases with CHD) are more common. A total of 1 084 cases were successfully followed up, and 675 cases were born, 392 cases were terminated, spontaneous abortion occurred in 17 cases. The proportion of terminated pregnancy cases was significantly increased from grade Ⅰ to Ⅳ, accounting for 5.24% (21/401), 27.78% (70/252), 89.54% (214/239) and 95.56% (43/45), respectively. Among the terminated pregnancy cases, those with grade Ⅲ accounted for the highest proportion (54.59% (214/392)). The distribution of terminated pregnancy cases was mainly complex congenital malformations or diseases with very poor prognosis (pregnancy outcome grade Ⅲ and Ⅳ), and proportion of terminated pregnancy with pregnancy outcome grade Ⅰ and Ⅱ cases (normal variation or good prognosis) accounted for 5.36% (21/392) and 17.86% (70/392), respectively. The results of multivariate logistic regression analysis showed that pregnant women with low education (high school and below: OR=2.73, 95%CI 1.26-5.93, P<0.001; illiteracy: OR=3.27, 95%CI 1.29-7.10, P<0.001) and low family income (Annual income<100 000 yuan: OR=2.47, 95%CI 1.69-5.12, P<0.001) were more likely to choose termination of pregnancy in case of fetal heart defect. Conclusion: In Yunnan province and the surrounding high altitude areas, the disease distribution of fetal heart defect is mainly simple or low-risk disease, but the complex malformation, especially the disease with poor pregnancy outcome, accounts for a relative high proportion. Pregnancy termination also occurs in some cases with good pregnancy outcome. The education level and family income of pregnant women may affect their choice of pregnancy outcome in case of fetal heart defect.

Brain re-expansion predict the recurrence of unilateral CSDH: A clinical grading system.

ObjectiveAssessing the risk of postoperative recurrence of chronic subdural hematoma (CSDH) is a clinical focus. To screen the main factors associated with the perioperative hematoma recurrence. The brain re-expansion is the core factor of recurrence. A clinical prognostic scoring system was also proposed.MethodsWe included 295 patients with unilateral CSDH as the training group for modeling. Factors predicting postoperative recurrence requiring reoperation (RrR) were determined using univariate and multivariate regression analyses, and bivariate Pearson correlation coefficient analysis was used to exclude related factors. Receiver operating characteristic curve analysis evaluates the ability of main factors to predict RrR and determines the cut-off value of brain re-expansion rate. We developed a prognostic scoring system and conducted preliminary verification. A verification group including 119 patients with unilateral CSDH was used to verify the grading systems.ResultsThe key factors for predicting unilateral CSDH recurrence were cerebral re-expansion rate (≤ 40%) at postoperative days 7–9 (OR 25.91, p < 0.001) and the preoperative CT density classification (isodense or hyperdense, or separated or laminar types) (OR 8.19, p = 0.007). Cerebral atrophy played a key role in brain re-expansion (OR 2.36, p = 0.002). The CSDH prognostic grading system ranged from 0 to 3. An increased score was associated with a more accurate progressive increase in the RrR rate (AUC = 0.856).ConclusionsOur prognostic grading system could screen clinically high-risk RrR patients with unilateral CSDH. However, increased attention should be paid to brain re-expansion rate after surgery in patients with CSDH.

How low risk are low risk myelodysplastic syndromes?

ABSTRACT Introduction Risk stratification is crucial to the appropriate management of many diseases, but in patients with myelodysplastic syndromes (MDS), for whom expected survival can vary greatly, accurate disease prognostication is especially important. This is further supported by a relative lack of therapies in MDS, and thus we must prognosticate carefully and accurately. Currently, patients with MDS are often grouped into higher-risk (HR) versus lower-risk (LR) disease using clinical prognostic scoring systems, but these systems have limitations. Areas covered The authors reviewed the literature on diagnostics, prognostics, therapeutics and outcomes in MDS. Factors such as disease etiology, specific clinical characteristics, or molecular genetic information not captured in the international prognostic scoring system revised IPSS-R can alter risk stratification, and identify a subset of LR-MDS patients who actually behave more like HR-MDS. Expert opinion This review will describe the current identification and management of patients with LR MDS disease whose condition is likely to behave in a less favorable manner than predicted by the IPSS-R. The authors comment on clinical and molecular features which are believe to upstage a patient from lower to higher risk disease.

Lower risk but high risk

Abstract Risk stratification is crucial to the appropriate management of most cancers, but in patients with myelodysplastic syndromes (MDS), for whom expected survival can vary from a few months to more than a decade, accurate disease prognostication is especially important. Currently, patients with MDS are often grouped into higher-risk (HR) vs lower-risk (LR) disease using clinical prognostic scoring systems, but these systems have limitations. Factors such as molecular genetic information or disease characteristics not captured in the International Prognostic Scoring System–Revised (IPSS-R) can alter risk stratification and identify a subset of patients with LR-MDS who actually behave more like those with HR-MDS. This review describes the current identification and management of patients with LR-MDS whose condition is likely to behave in a less favorable manner than predicted by the IPSS-R.

Diagnostic and prognostic scoring systems for autoimmune hepatitis: a review.

Auto-immune hepatitis (AIH) is a rare condition which primarily affects young women. Several diagnostic scoring systems exist based on clinical, biochemical, immunologic and histologic characteristics of AIH. Additionally, prognostic parameters can be identified. The purpose of this literature review is to compare the clinical value, strengths and limitations of these diagnostic and prognostic scoring systems. A literature search was performed in two databases and selected based on diagnostic and prognostic criteria. Only studies concerning AIH in adults were included. The backbone of scoring systems remains the revised AIH criteria published in 1999 and the simplified from 2008. The revised system shows a higher sensitivity, lower specificity and lower diagnostic accuracy compared to the simplified. Limitations to these scoring systems include limited diagnostic accuracy in acute or fulminant liver failure, insufficient inclusion of atypical auto-antibodies and lacking diagnostic power in presence of overlap syndromes. Concerning these overlap syndromes, the Paris criteria show a higher diagnostic accuracy compared to the scoring systems for AIH. Presently, no clinical prognostic scoring systems are available. However, a first system based on response to treatment accurately predicts long-term survival in AIH. Diagnostic scoring systems are useful in diagnosing AIH and have complementary value. However, they are no substitute for the gold standard of appropriate clinical assessment and are mostly useful in defining cohorts for research purposes. An evolution towards a more dynamic scoring system, using prognostic parameters and the progression of typical features, seems more valuable than the current diagnostic systems.

An MDS Specific Frailty Index Based on Cumulative Deficits Adds Independent Prognostic Value to Established Clinical Prognostic Scoring Systems

An MDS Specific Frailty Index Based on Cumulative Deficits Adds Independent Prognostic Value to Established Clinical Prognostic Scoring Systems

Novel Multiparametric Nomogram for Overall Survival Prediction in Complicated Intra-Abdominal Infection: A Multicenter Study in China.

Background: Complicated intra-abdominal infections (cIAIs) in the abdominal cavity or within an abdominal organ are numerous and frequent dangerous entities in the treatment of critically ill patients. Early clinical evaluation is necessary.Methods: This retrospective multicenter study included patients from 10 intensive care units (ICUs). Risk factors for the overall survival (OS) of patients with cIAI were selected using least absolute shrinkage and selection operator regression, and a nomogram was constructed subsequently. Calibration curve and receiver operating characteristic (ROC) curve were used to evaluate the calibration and discriminative ability.Results: In total, 544 patients diagnosed with cIAI were enrolled and divided into the study (n = 276) and validation (n = 268) sets. Sex, acute gastrointestinal injury, acute kidney injury, rare bacterium infection, Charlson score, and APACHE II score were identified as independent risk factors and were constructed for the nomogram. The nomogram showed marked calibration capability with a concordance index (C-index) of 0.909 and 0.831 in the study and validation set, respectively. Compared with the common clinical prognostic scoring system, the nomogram achieved the highest discrimination ability with an area under the curve (AUC) value of 0.91 and 0.83 in the study set and validation set, respectively.Conclusions: Our newly constructed nomogram provides a useful tool for risk stratification and prognosis evaluation of cIAI.

A Clinical Prognostic Scoring System for Neurocysticercosis.

Objectives In patients with neurocysticercosis (NCC), an accurate risk prediction would allow a better therapeutic approach; however, there are currently no tools that can enhance the accuracy of risk prediction. We designed a prognostic scoring system to be used by neurologists and other physicians managing patients with NCC. Materials and Methods Using data from clinical records of patients from a third-level national reference center for neurological diseases, we assessed demographic, clinical, and tomographic variables among 293 patients diagnosed with NCC. Multivariable logistic regression analyses were used to develop a clinical prognostic scoring instrument. Patients with NCC were assessed for neurological impairment at 3 months after diagnosis. Statistical Analysis Score accuracy was assessed by receiver operating characteristic (ROC) curve analysis. The primary outcome was the presence of neurological impairment, resulting in disability according to self-report or caregiver reports; this outcome was assessed during follow-up visits at 3 ± 1 months after discharge. Results The most common symptoms at presentation were headache (67%) and seizure (63%). A six-item (total score from –4 to + 2) prognostic instrument was constructed on the basis of the presence of seizures/headache at presentation, a leukocyte count above 12x 109/dL, the presence of six to ten parasites, subarachnoid localization, and the use of anthelmintic drugs. Among 113 patients with negative scores, 79.6% developed neurological deficits. Among patients with scores of 1 to 2, 64.6% recovered completely, with an overall accuracy of prediction of 74.7% and area under the ROC curve = 0.722 (95% CI, 0.664–0.780, p < 0.0001). Conclusions The clinical prognostic scoring system for NCC described in this study is a new instrument for use in daily clinical practice. It is simple to administer, and it has a prognostic accuracy of 75%. Its use has the potential to improve the quality of care by guiding appropriate decision-making and early management of patients with NCC.

P2669Prognostication in out-of-hospital cardiac arrest

Abstract Background Out-of-hospital cardiac arrest (OOHCA) remains an area where little progress has been made in outcomes despite significant advances in cardiac care. The OOHCA cohort requires a large amount of resources, including primary coronary intervention and intensive care, often with poor eventual outcomes. A clinical prognostic scoring system would allow resources to be focused on OOHCA patients who are likely to survive; rather than expensive and futile treatments in those who are not. Several different scoring systems have been published but have not been compared in a real world cohort of OOHCA patients. Method The Cardiac Arrest Hospital Prognosis (CAHP) score that utilises 7 variables and a simpler three-factor prognostic score derived by Hirt et al were applied retrospectively to a cohort of 157 consecutive OOHCA patients presenting to a regional Centre in our city, UK between 2016 and 2018. For each patient the CAHP score was calculated where a score of &gt;200 predicts a Cerebral Performance Category (CPC) outcome of 3–5 (severe disability, vegetative coma or death). They were also categorised using the Hirt algorithm into “Futile”, “Approaching Futility” and “Non-Futile” groups. CPC outcomes were then compared using both scores and diagnostic accuracy assessed. Results A CAHP score of &gt;200 predicted a CPC outcome of 3–5 with 97% specificity and a positive predictive value of 92%. A CAHP score &gt;200 predicted 30-day mortality with 96% specificity, and a positive predictive value of 89% (Fig. 1A). The same cohort categorised by the Hirt algorithm was less accurate; in those patients sorted into the “futile” category 15% of patients survived, and in the “approaching futility” group survival was 100% (Fig. 1B). The specificity and PPV of predicting futility was 94% and 85% respectively. Conclusion Although a simple three-factor prognostic score as suggested by Hirt et al is attractive in this cohort of OOHCA patients it did not discriminate those patients who were likely to survive and, the CAHP score was more accurate in predicting outcome. Further validation is required in prospective, multi-centre studies to ensure that application of these scores are reproducible in real-world OOHCA populations in decision making for emergency coronary angiography. Acknowledgement/Funding None

Predicting Outcomes in Diffuse Large B-Cell Lymphoma

Currently available clinical prognostic scoring systems and correlative biomarkers are insufficient for predicting outcomes in diffuse large B-cell

Research Advances of Molecular Genetic Abnormality in Myelodysplastic Syndrome--Review

The myelodysplastic syndromes (MDS) are a heterogeneous group of clonal myeloid disorders characterized by ineffective hematopoiesis and increased risk of transformation to acute myelogenous leukemia (AML). The treatment of MDS is highly dependent on the reliability of the prognostic evaluation model. Current clinical prognostic scoring systems are comprised of morphology, pivotal clinical trials and cytogenetic findings. However, none of the available prognostic systems incorporates disease-related molecular abnormalities, such as somatic mutations. Cumulative evidence suggests that genomic data can also be used clinically to assist the diagnosis, prognosis, prediction of response to specific therapies, and the development of novel and accurate targeted therapies. Therefore, it is not possible to predict the response of patients to molecular targeted drugs, such as demethylation drugs. With the recent advance in whole- genome sequencing technologies, cumulative evidence suggests that genomic data can also be associated with the genesis, prognosis, prediction of response to specific therapies, and the development of novel accuvate targeted therapies, the issue of having some mechanism to dissect this heterogeneity and precision treatment is coming to the fore. However, there are still several hindrances to its clinical application. If these problems can be solved, molecular genetics will further provide a theoretical basis for the application of precision medicine in MDS.

Pulmonary embolism severity assessment and prognostication

For patients who have acute symptomatic pulmonary embolism (PE), risk of short-term death and adverse outcomes should drive the initial treatment decisions. Practice guidelines recommend that patients who have a high-risk of PE-related death and adverse outcomes, determined by the presence of haemodynamic instability (i.e., shock or hypotension), should receive systemically administered thrombolytic therapy. Intermediate-high risk patients might benefit from close observation, and some should undergo escalation of therapy beyond standard anticoagulation, particularly if haemodynamic deterioration occurs. Low-risk for adverse outcomes should lead to early hospital discharge or full treatment at home. Validated prognostic tools (i.e., clinical prognostic scoring systems, imaging studies, and cardiac laboratory biomarkers) assist with risk classification of patients who have acute symptomatic PE.

Risk Stratification for Proven Acute Pulmonary Embolism: What Information Is Needed?

Classification of risk drives treatment decisions for patients with acute symptomatic pulmonary embolism (PE). High-risk patients with acute symptomatic PE have hemodynamic instability (i.e., shock or hypotension present), and treatment guidelines suggest systemically administered thrombolytic therapy in this setting. Normotensive PE patients at low risk for early complications (low-risk PE) might benefit from treatment at home or early discharge, while normotensive patients with preserved systemic arterial pressure deemed as having a high risk for PE-related adverse clinical events (intermediate-high-risk PE) might benefit from close observation and consideration of escalation of therapy. Prognostic tools (e.g., clinical prognostic scoring systems, imaging testing, and cardiac laboratory biomarkers) assist with the classification of patients into these categories.

Relationship between somatic mutations and the response to the treatment of de novo patients with myelodysplastic syndromes: reports from the 57th American Society of Hematology annual meeting

Research progress of somatic mutations and the response to the treatment of de novo myelodysplastic syndromes (MDS) patients in the 57th American Society of Hematology (ASH) annual meetings was reviewed. The optimal methods and therapy time for patients with high-risk de novo MDS remained an area of ongoing investigation. The clinical prognostic scoring system does not include the molecular genetic abnormalities and DNA metlylation of histone/nuclear chromatin modifications, which may predict the effect of hypomethylation (HMA). Treatment of HMA may change the expression of genes related with prognosis, and the response rate to the HMA treatment was significantly increased for TET2-mutated patients with high-variant allele frequencies. The overall grade of recommendation for choosing HMA therapy over induction chemotherapy in high-risk MDS based on molecular genetic mutations was 2C, according to less-associated toxicity and increased responses primarily in TET2-mutated disease. Further prospective studies are needed to evaluate the long-term effects of HMA therapy, particularly in TET2-mutated patients. Key words: Myelodysplastic syndromes; Somatic mutations; American Society of Hematology annual meeting

Predicting survival in malignant pleural effusion: development and validation of the LENT prognostic score

BackgroundMalignant pleural effusion (MPE) causes debilitating breathlessness and predicting survival is challenging. This study aimed to obtain contemporary data on survival by underlying tumour type in patients with MPE, identify...

A New Clinical Prognostic Scoring System for Pituitary Adenoma

Objective: Clinical manifestations of pituitary adenomas are the most changeful and directly associated with prognosis. In the past three years, we have established a new clinical prognosis score systerm for preoperative pituitary adenomas. We try to use this score systerm to help the neurosurgeon choose therapy strategy and evaluate the risk correctly. Materials and methods: New clinical prognosis score systerm for preoperative pituitary adenomas were based on MRI image, Visual manifestations and Endocrinological dysfunction. 756 cases were admitted to prove the reliability and validity of the new score systerm. The clinical information of each case were cllected carefully, such as MRI image, result of visal test, endocrinological dysfunction,surgical approach,amount of bleeding,hours of operationa,expense and so on. All the data were statistically. Results: The reliability and validity of the new clinical prognosis score systerm for preoperative pituitary adenomas are very good. The highest score is set to 10. There are 666 cases(88%) with low scores(< 6) got satisfied result, especially those cases got score less than 3. 90 cases(12%) got scores over 6 suffer diffcult treatment and high cost. Conclusion: The new clinical prognosis score systerm for preoperative pituitary adenomas are accuracy and easy to use. The higher the score the worse prognosis.Those cases which got high score faced with greater risks and more cost.

A Clinical Prognostic Scoring System for Neurocysticercosis (P04.236)

A Clinical Prognostic Scoring System for Neurocysticercosis (P04.236)

Discovery Science and Integrating Molecular Diagnosis into Clinical Practice

Abstract SCI-31Recent discoveries have yielded profound insights into the genetic basis of myeloid malignancies. A central challenge is now to translate these discoveries into clinical practice. Genetic lesions are powerfully associated with specific aspects of clinical phenotype, including hematologic parameters, particular morphologic abnormalities, response to therapy, and overall survival. Moreover, gene expression profiling, evaluation of microRNA expression, and epigenetic analyses add further biological, prognostic, and predictive information. Current research studies aim to integrate existing clinical prognostic scoring systems with molecular data to refine the taxonomy of myeloid malignancies, to improve the prediction of prognosis and response to specific therapies, and to develop novel therapeutic strategies based on insights into disease biology. Disclosures:Ebert:Celgene: Consultancy; Genoptix: Consultancy.