Best Predictor Of Long-term Outcome Research Articles

DOP17 Evaluating segmental healing with the modified Mayo endoscopic score (MMES) has a clear additional value in predicting long-term outcome in patients with Ulcerative Colitis: Results from a prospective cohort study

Abstract Background Most endoscopic scoring systems (including the Mayo endoscopic subscore, MES) do not consider the extent of inflammation and can therefore not pick up segmental endoscopic improvement. The modified Mayo endoscopic score (MMES) was developed to overcome this limitation. We examined the predictive value of the MMES in addition to the MES on long-term clinical outcome in ulcerative colitis (UC). Methods Between 2014 and 2017, patients initiating biologic therapy for active UC (baseline MES ≥2) were recruited. Patients without assessment of the upper margin of inflammation or with a clinical follow-up <12 months were excluded. We conducted a clinical and endoscopic assessment at baseline and week 8 (adalimumab, ADM) or 14 (golimumab, GOL; infliximab, IFX; vedolizumab, VDZ). Clinical response was defined as a decrease in the adapted Mayo score (excluding physician global assessment) with ≥2 points and ≥30%, plus a decrease in rectal bleeding score ≥1 or an absolute rectal bleeding score ≤1. We classified patients by evolution of endoscopic activity at week 8/14 in group A (endoscopic healing, MES ≤1), group B (segmental healing, MES >1 but decrease in MMES ≥30%) and group C (MES >1 and no drop in MMES ≥30%). Clinical relapse-free, discontinuation-free and colectomy-free survival was estimated by Kaplan-Meier analysis with log-rank test. Results A total of 150 UC patients were included (52% male, median age 42 years, median disease duration 7 years) with a median (IQR) follow-up of 61 (48–68) months. An anti-TNF was initiated in 74 (35 IFX, 23 ADM, 16 GOL), and VDZ in 76 patients. A significant reduction in MES and MMES was observed at week 8/14 (cf. table). In group A 67/69 (97%) patients achieved clinical response compared to 15/27 (55%) in group B and 11/54 (20%) in group C. During follow-up 60/93 (65%) patients maintained clinical response, 83/150 (55%) patients discontinued treatment due to primary non-response or loss of response and 33/150 (22%) patients underwent colectomy. The ΔMMES demonstrated to be of additional predictive value: there was a significant difference between groups B and C regarding clinical relapse, drug persistence and need for colectomy (cf. figure). Conclusion Although MES ≤1 remains the best predictor of long-term outcome, the MMES - identifying a subgroup with a segmental endoscopic response - demonstrated a clear additional value predicting long-term outcome in UC. These promising results merit inclusion of the MMES in future clinical trials. Table The MMES is calculated by multiplying the Mayo endoscopic subscore for the different colon segments with the maximal extent of inflammation (in decimeters) divided by the number of segments with active inflammation (Lobatón T, et al. JCC 2015; 9:846–52.) Figure

Predicting long-term trends in inflammatory neuropathy outcome measures using latent class modelling.

Immunoglobulin (Ig) is used to treat chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) and multifocal motor neuropathy with conduction block (MMNCB). Regular infusions may be used for symptom control. Disease activity is monitored with clinical outcome measurements. We examined outcome measure variation during clinically stable periods in Ig-treated CIDP and MMNCB patients. We explored utility of serial outcome measurement in long-term outcome prediction. Retrospective longitudinal analysis of a single neuroscience centre's Ig-treated CIDP and MMNCB patients, 2009-2020, was performed. Mean and percentage change for grip strength, Rasch-built overall disability scales (RODS) and MRC sum scores (MRC-SS) during periods of clinical stability were compared to score-specific minimal clinically important differences (MCID). Latent class mixed modelling (LCMM) was used to identify longitudinal trends and factors influencing long-term outcome. We identified 85 CIDP and 23 MMNCB patients (1423 datapoints; 5635 treatment-months). Group-averaged outcome measures varied little over time. Intra-individual variation exceeded MCID for RODS in 44.2% CIDP and 16.7% MMNCB datapoints, grip strength in 10.6% (CIDP) and 8.8%/27.2% (MMNCB right/left hand) and MRC-SS in 43.5% (CIDP) and 20% (MMNCB). Multivariate LCMM identified subclinical trends towards improvement (32 patients) and deterioration (73 patients) in both cohorts. At baseline, CIDP 'deteriorators' were older than 'improvers' (66.2 vs 57 years, P= .025). No other individual factors predicted categorisation. The best model for 'deteriorator' identification was contiguous sub-MCID decline in more than one outcome measure (CIDP: sensitivity 74%, specificity 59%; MMNCB: sensitivity 73%, specificity 88%). Outcome measure interpretation determines therapeutic decision-making in Ig-dependent neuropathy patients, but intra-individual variation is common, often exceeding MCID. Here we show sub-MCID contiguous changes in more than one outcome measurement are a better predictor of long-term outcome.

Any BCR-ABL Reduction Below 10% At 6 Months Of Therapy Significantly Improves Outcome For CML Patients With a Poor Response At 3 Months

▪ BackgroundThe molecular response at 3 months (mo) after commencement of tyrosine kinase inhibitor (TKI) therapy for CML patients (pts) has prognostic significance and has been confirmed by many groups. Analyses by Marin, JCO 2012 and Neelakantan, Blood 2013 went further and suggest that measuring BCR-ABL at 3 mo is the only requirement to predict outcome, with BCR-ABL at later timepoints adding little useful information. However, Nazha (Haematologica 2013) using cytogenetic measurement suggest that assessing the 6 mo response for pts with a poor response at 3 mo may provide a better predictor of long-term outcome. AimIn this study, we aimed to dissect the prognostic importance of assessing both the 3 and 6 mo molecular response in our large cohort and determine whether a poor response at 3 mo identifies a group with high ongoing risk of treatment failure regardless of subsequent molecular response. MethodWe evaluated 528 pts enrolled in consecutive trials of first line imatinib from 2000-2009. Many pts were treated before alternative TKIs were available, but 89 switched. The utility of BCR-ABL as a predictor of death, progression (AP/BC/death: PFS) and treatment failure (according to ELN 2013: FFS) was assessed by ROC analysis, Kaplan-Meier survival with log rank test, and cumulative incidence with Fine & Gray regression for MMR. Pts were divided according to the 2013 ELN 3 and 6 mo molecular response definition: 3 mo, Optimal (Opt) ≤10% or Warning (Warn) >10%; 6 mo, Opt <1%, Warn 1-10% or Failure (Fail) >10%. The Warn category requires more frequent monitoring and Fail mandates a therapy change. ResultsOptimal BCR-ABL threshold values at 3 mo for outcome prediction were concordant with Marin, JCO 2012: death 16.1%; progression 9.5%; failure 8.4%; MMR 1.4%, demonstrating the commutability of the international reporting scale. Consistent with other studies, responses after 4 years were significantly superior for pts ≤10% (Opt) compared with >10% (Warn) at 3 mo: survival 97% v 90%, P = .003; PFS 96% v 84%, P < .0001; FFS 83% v 42%, P < .0001; and MMR 89% v 42%, P< .0001. 100 pts met the BCR-ABL Warn criterion at 3 mo: 14 of these progressed; 4 before 6 mo and a further 7 before 12 mo.When modeling BCR-ABL measurements at 0, 1, 2, 3, 6 and 12 mo as a time-dependent continuous covariate in Cox regression, a BCR-ABL decrease at any time significantly reduced the risk of death (P = .0002), progression (P < .0001) and treatment failure (P < .0001) indicating that response after 3 mo also has prognostic significance. Of the 528 pts, 45 had missing molecular analysis at 3 or 6 mo, including 13 who ceased before 6 mo. The remaining 483 pts were categorized according to the ELN response definition at 3 and 6 mo and included 89 pts >10% at 3 mo. The Table and Figure detail outcomes at 4 years. Any reduction below 10% at 6 mo for pts in the 3 mo Warn category significantly reduced the subsequent risk of death, progression and failure, and increased MMR rates. Notably, there was no significant difference in outcome for pts >10% at 3 mo and <1% at 6 mo (Warn / Opt) compared to pts with an optimal response at both timepoints. ConclusionOur data confirms the importance of achieving BCR-ABL values of 10% or below at 3 mo after starting therapy. Based on the 3 mo assessment, the prognosis was significantly superior for these pts. Progression before the 6 mo timepoint occurred in 4% of pts with BCR-ABL >10% at 3 mo. However, the vast majority of pts with >10% at 3 mo continued therapy and the 6 mo BCR-ABL assessment provided important long-term prognostic information. Any reduction below 10% at 6 mo led to significantly superior outcomes, approximating those with optimal response at 3 mo. The impact and optimal timing of therapeutic intervention for pts in the poor risk category at 3 mo still needs to be determined in prospective studies and may require large patient cohorts. However, pts who are >10% at both 3 and 6 mo have inferior outcomes and are undoubtedly in need of a therapy change. [Display omitted] Disclosures:Branford:Novartis: Honoraria, Research Funding; BMS: Honoraria, Research Funding; Ariad: Honoraria, Research Funding. Yeung:Novartis: Honoraria, Research Funding; BMS: Honoraria. Hughes:Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; BMS: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Ariad: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding.

Neurological level at birth predicts survival to the mid-40s and urological deaths in open spina bifida: a complete prospective cohort study.

AimTo conduct a 50-year complete, community-based, prospective cohort study to investigate long-term survival, causes of death, and influence of level of the lesion in treated open spina bifida.MethodThe cohort comprised 117 consecutive cases whose backs were closed non-selectively at birth between 1963 and 1971 in Cambridge, UK. In 2013 we surveyed the survivors (n=39, 18 males, 21 females; mean age 46y, range 43–49y) by postal questionnaire and telephone interview. We compared outcomes in those born with a neurological deficit in terms of sensory and motor levels of L1 and above versus L2 and below.ResultsTwo-thirds of the cohort (78/117) had died. Causes of death were cardiorespiratory (n=26), neurological (n=24), urological (n=22), or other (n=6). Only the urological deaths were related to level of the lesion: there were none in those with a sensory level of L2 or below (p<0.001). Birth findings also predicted survival: of the 57 infants with a neurological level of L1 or above, only 12% (n=7) survived compared with 55% (30/55) of the remainder (p<0.001).InterpretationThe increased mortality in those born with an extensive neurological deficit was mainly due to urological deaths. Neurological level, particularly the sensory level, is the best predictor of long-term outcome and should be assessed routinely at birth.What this paper addsIn open spina bifida, sensory level predicts urological life expectancy.Death due to urological causes only occurred in those with a sensory level of T6–L1.Neurological level predicts outcome better than cutaneous or radiological level.Sensory level should be assessed in all infants with open spina bifida.This article is commented on by Worley on pages 594–595 of this issue.

Predictors for good therapeutic outcome and drop-out in technology assisted guided self-help in the treatment of bulimia nervosa and bulimia like phenotype.

Technology assisted guided self-help has been proven to be effective in the treatment of bulimia nervosa (BN). The aim of this study was to determine predictors of good long-term outcome as well as drop-out, in order to identify patients for whom these interventions are most suitable. One hundred and fifty six patients with BN were assigned to either 7 months internet-based guided self-help (INT-GSH) or to conventional guided bibliotherapy (BIB-GSH), both guided by e-mail support. Evaluations were taken at baseline, after 4, 7, and 18 months. As potential predictors, psychiatric comorbidity, personality features, and eating disorder psychopathology were considered. Higher motivation, lower frequency of binge eating, and lower body dissatisfaction at baseline predicted good outcome after the end of treatment. Lower frequency of binge eating predicted good outcome at long-term follow-up. Factors prediciting drop-out were higher depression and lower self-directedness at baseline. Technology assisted self-help can be recommended for patients with a high motivation to change, lower binge-eating frequency and lower depression scores.

Cavernous Malformation of Brainstem, Thalamus, and Basal Ganglia

Cavernous malformations (CMs) in deep locations account for 9% to 35% of brain malformations and are surgically challenging. To study the clinical features and outcomes following surgery for deep CMs and the complication of hypertrophic olivary degeneration (HOD). Clinical records, radiological findings, operative details, and complications of 176 patients with deep CMs were reviewed retrospectively. Of 176 patients with 179 CMs, 136 CMs were in the brainstem, 27 in the basal ganglia, and 16 in the thalamus. Cranial nerve deficits (51.1%), hemiparesis (40.9%), numbness (34.7%), and cerebellar symptoms (38.6%) presented most commonly. Hemorrhage presented in 172 patients (70 single, 102 multiple). The annual retrospective hemorrhage rate was 5.1% (assuming CMs are congenital with uniform hemorrhage risk throughout life); the rebleed rate was 31.5%/patient per year. Surgical approach depended on the proximity of the CM to the pial or ependymal surface. Postoperatively, 121 patients (68.8%) had no new neurological deficits. Follow-up occurred in 170 patients. Delayed postoperative HOD developed in 9/134 (6.7%) patients with brainstem CMs. HOD occurred predominantly following surgery for pontine CMs (9/10 patients). Three patients with HOD had palatal myoclonus, nystagmus, and oscillopsia, whereas 1 patient each had limb tremor and hemiballismus. At follow-up, 105 patients (61.8%) improved, 44 (25.9%) were unchanged, and 19 (11.2%) worsened neurologically. Good preoperative modified Rankin Score (98.2% vs 54.5%, P = .001) and single hemorrhage (89% vs 77.3%, P < .05) were predictive of good long-term outcome. Symptomatic deep CMs can be resected with acceptable morbidity and outcomes. Good preoperative modified Rankin Score and single hemorrhage are predictors of good long-term outcome.

Microvascular decompression for hemifacial spasm: long-term outcome and prognostic factors, with emphasis on delayed cure

The postoperative course of microvascular decompression (MVD) for hemifacial spasm (HFS) is variable, and the optimal time for assessing the results is unclear. From April 1997 to October 2007, MVD for HFS was performed in 801 patients. Patients were divided into two groups (cured or failed) according to subjective patient assessments over a 3-year period. We analyzed patient characteristics and surgical findings to determine prognostic factors. Medical records were analyzed retrospectively over the 3-year follow-up period. Of the 801 patients who underwent surgery, 743 (92.8%) appeared to be cured, 70 (8.7%) had residual or recurrent spasms more than 1year after surgery, 11 (1.3%) had gradual improvement over 3years, and 1 (0.1%) had delayed improvement more than 3years after surgery. Fifty-eight patients (7.2%) had residual or recurrent spasms more than 3years after surgery, of which 19 (2.4%) had recurrence after initial relief. The mean time to spasm recurrence was 18.9months. Intraoperative resolution of the lateral spread response (LSR) after decompression (p = 0.048) and severe indentation (p = 0.038) were significant predictors of good long-term outcome after MVD for HFS. In our series, 70 patients (8.7%) had residual or recurrent spasms more than 1year after surgery, of which 12 (17.1%) improved gradually after 1year. If the surgeon can confirm intraoperative resolution of the LSR and severe indentation, reoperation can be delayed until 3years after MVD.

Hilar Cholangiocarcinoma

The American Joint Committee on Cancer staging system for hilar cholangiocarcinoma may be inaccurate because the bile duct lacks discrete tissue boundaries. To examine the accuracy of the American Joint Committee on Cancer staging schemes and to determine the prognostic implications of tumor depth. From January 1, 1987, through December 31, 2009, there were 106 patients who underwent resection of hilar cholangiocarcinoma who had pathologic slides available for re-review. Tumor depth and overall survival. Overall median survival was 19.9 months. The 6th and 7th editions of the T-classification criteria were unable to discriminate among T1, T2, and T3 lesions (P > .05 for all). Median survival was associated with the invasion depth of the tumor (≥5 mm vs <5 mm): 18 months vs 30 months (P = .01). On multivariate analysis, tumor depth remained predictive of disease-specific death (hazard ratio, 1.70; P = .03). The American Joint Committee on Cancer T-classification criteria did not stratify patients with regard to prognosis. Depth of tumor invasion is a better predictor of long-term outcome.

Comparison of C‐reactive Protein and Albumin Excretion as Prognostic Markers for 10‐Year Mortality After Myocardial Infarction

C-reactive protein (CRP) is an established prognostic marker in the setting of acute coronary syndromes. Recently, albumin excretion rate also has been found to be associated with adverse outcomes in this clinical setting. Our aim was to compare the prognostic power of CRP and albumin excretion rate for long-term mortality following acute myocardial infarction (AMI). To determine whether albumin excretion rate is a better predictor of long-term outcome than CRP in post-AMI patients. We prospectively studied 220 unselected patients with definite AMI (median [interquartile] age 67 [60-74] y, female 26%, heart failure 39%). CRP and albumin-to-creatinine ratio (ACR) were measured on day 1, day 3, and day 7 after admission in 24-hour urine samples. Follow-up duration was 10 years for all patients. At survival analysis, both CRP and ACR were associated with increased risk of 10-year all-cause mortality, also after adjusting for age, hypertension, diabetes mellitus, prehospital time delay, creatine kinase-MB isoenzyme peak, heart failure, and creatinine clearance. CRP and ACR were associated with nonsudden cardiovascular (non-SCV) mortality but not with sudden death (SD) or noncardiovascular (non-CV) death. CRP was not associated with long-term mortality, while ACR was independently associated with outcome both in short- and long-term analyses. At C-statistic analysis, CRP did not improve the baseline prediction model for all-cause mortality, while it did for short-term non-SCV mortality. ACR improved all-cause and non-SCV mortality prediction, both in the short and long term. ACR was a better predictor of long-term mortality after AMI than CRP.

Long-term outcome of combined valve repair and maze procedure for nonrheumatic mitral regurgitation

The long-term outcomes of combined mitral repair and maze procedure for patients with nonrheumatic mitral regurgitation and chronic atrial fibrillation were evaluated. Between June 1992 and December 2008, 187 patients underwent a combined mitral repair and maze procedure. The mean follow-up period was 7.4 ± 4.3 years. Chordal reconstruction was performed in 69 patients, leaflet resection in 91, edge-to-edge leaflet suture in 30, and ring annuloplasty in 156. In addition, a cryo-maze procedure was applied in 110, and a Cox-Kosakai maze and radiofrequency maze were applied in the others. There were 2 operative deaths and the 15-year survival was 71%. The 15-year freedom from greater than grade 3 mitral regurgitation was 61%; rates of freedom from heart failure (New York Heart Association class ≥ III) and reoperations were 79% and 91%, respectively. Cardiac function was improved and left ventricular size was decreased significantly postoperatively. Multivariate analysis showed that a large left ventricular diastolic diameter (≥65 mm) was an independent risk factor for recurrent mitral regurgitation. Eleven thromboembolic episodes (0.79%/patient-year) were detected during follow-up examinations, of which 7 occurred in patients with recurrent atrial fibrillation. Sinus rhythm was regained in 86% after 6 months and in 63% after 15 years. Multivariate analysis showed that a small-voltage f wave was an independent risk factor for AF recurrence. A combined mitral valve repair and maze procedure provided low rates of morbidity and mortality and led to well-preserved cardiac function. Left ventricular diastolic diameter and f-wave voltage can be accurate predictors of good long-term outcome.

Predictors of long-term success after microvascular decompression for trigeminal neuralgia

Microvascular decompression (MVD) is an effective treatment for trigeminal neuralgia (TN). However, many patients do not experience complete pain relief, and relapse can occur even after an initial excellent result. This study was designed to identify characteristics associated with improved long-term outcome after MVD. One hundred seventy-nine consecutive patients who had undergone MVD for TN at the authors' institution were contacted, and 95 were enrolled in the study. Patients provided information about preoperative pain characteristics including preponderance of shock-like (Type 1 TN) or constant (Type 2 TN) pain, preoperative duration, trigger points, anticonvulsant therapy response, memorable onset, and pain-free intervals. Three groups were defined based on outcome: 1) excellent, pain relief without medication; 2) good, mild or intermittent pain controlled with low-dose medication; and 3) poor, severe persistent pain or need for additional surgical treatment. Results Type of TN pain (Type 1 TN vs Type 2 TN) was the only significant predictor of outcome after MVD. were excellent, good, and poor for Type 1 TN versus Type 2 TN patients in 60 versus 25%, 24 versus 39%, and 16 versus 36%, respectively. Among patients with each TN type, there was a significant trend toward better outcome with greater proportional contribution of Type 1 TN (lancinating) symptoms (p < 0.05). Pain relief after MVD is strongly correlated with the lancinating pain component, and therefore type of TN pain is the best predictor of long-term outcome after MVD. Application of this information should be helpful in the selection of TN patients likely to benefit from MVD.

Elevated C-reactive protein in depression: a predictor of good long-term outcome with antidepressants and poor outcome with psychotherapy

Elevated C-reactive protein in depression: a predictor of good long-term outcome with antidepressants and poor outcome with psychotherapy

Preoperative or postoperative self-efficacy: Which is a better predictor of outcome after total hip or knee arthroplasty?

Objective Self-efficacy is considered an important determinant of outcome after total hip or knee arthroplasty. Aim of this study is to evaluate the contributions of preoperative and short-term postoperative self-efficacy in predicting long-term outcome. Methods Self-efficacy was determined in 103 total hip and knee arthroplasty patients preoperatively and 6 weeks postoperatively with the Self-Efficacy for Rehabilitation Outcome Scale (SER). The 6-month outcome was assessed with a disease-specific and a generic self-report questionnaire and an objective measure of function (walking speed). Multiple linear regression analyses were used to examine the value of preoperative and short-term postoperative self-efficacy in predicting 6-month outcomes. Results Preoperative self-efficacy was only a significant predictor of long-term postoperative walking speed, with higher self-efficacy resulting in faster walking speed at the long-term measurement ( R 2 = 0.47). Short-term postoperative self-efficacy was a significant predictor of the long-term postoperative generic outcome measure (physical functioning: R 2 = 0.30; mental health: R 2 = 0.53) and of walking speed ( R 2 = 0.66), with higher self-efficacy resulting in a better long-term outcome. Conclusion Short-term postoperative self-efficacy seems a better predictor of long-term outcome after total hip or knee arthroplasty than preoperative self-efficacy. Practice implications Interventions should focus on enhancing short-term postoperative rather than preoperative self-efficacy.

The impact of mitral valve morphology on the long‐term outcome of mitral balloon valvuloplasty

The effect of mitral valve morphology (MVM) on the long-term results of mitral balloon valvuloplasty (MBV) is not well established. The aim of the study was to evaluate the impact of MVM on long-term outcome of MBV. Five hundred and eighteen consecutive patients (mean age, 31+/-11 years) who underwent successful MBV were followed up for 0.5-16.5 (mean, 6+/-4.5) years. Patients were divided into two groups according to their mitral echo score (MES) before MBV: group A (n=340; MES<or=8) and group B (n=178; MES>8). We report the immediate and long-term clinical and echocardiographic results of the above-mentioned 518 consecutive patients. The mitral valve area was significantly larger in group A than in group B, both immediately after MBV (2.0+/-0.3 vs. 1.82+/-0.3 cm2, respectively; P<0.0001) and also at the last follow-up (1.8+/-0.33 vs. 1.5+/-0.33 cm2, respectively; P<0.0001). Restenosis occurred in 38/340 (11%) in group A vs. 73/178 (41%) in group B (P<0.0001). Actuarial freedom from restenosis at 5, 10, 15 years were 92+/-2%, 85+/-3%, 65+/-6% for group A vs. 72+/-4%, 44+/-5%, 9+/-6% for group B (P<0.001). Event-free survival rates at 5, 10, 15 years for group A were 93+/-1%, 88+/-2%, 66+/-6% vs. 82+/-3%, 59+/-6%, 8+/-7% for group B (P<0.0001). Stepwise Cox multivariate regression analysis identified MES, preprocedure functional class, and postprocedure mitral valve area<or=1.8 cm2, as predictors of restenosis (P<0.0001, P=0.014, P=0.0015, respectively); MES and age as predictors of event-free survival (P<0.0001, P<0.0001, respectively). MBV has excellent long-term results in patients with favorable MVM. Favourable MVM is a strong predictor of good long-term outcome.

Surgical management of brainstem hemangioblastomas in patients with von Hippel—Lindau disease

Hemangioblastomas of the brainstem constitute 5 to 10% of central nervous system (CNS) tumors in patients with von Hippel-Lindau (VHL) disease. At present, optimal management of brainstem hemangioblastomas associated with VHL disease is incompletely defined. In an attempt to clarify some of the uncertainty about the operative treatment of these lesions and its outcome, the authors reviewed all cases of VHL disease in which resection of brainstem hemangioblastomas was performed at the National Institutes of Health during a 10-year period. Twelve consecutive patients with VHL disease (six male and six female patients [mean age 31.7 +/- 9 years; range 15-46 years]) who underwent 13 operations to remove 17 brainstem hemangioblastomas were included in this study (mean follow-up period, 88.4 +/- 37.4 months; range 37-144 months). Serial examinations, hospital charts, magnetic resonance images, and operative records were reviewed. To evaluate clinical course, clinical grades were assigned to each patient before and after surgery. Preoperative neurological function was the best predictor of long-term outcome. In addition, patients who underwent CNS surgeries for hemangioblastomas were more likely to improve or to remain neurologically stable. Tumor or cyst size, the presence of a cyst, or the location of the tumor (intramedullary, extramedullary, or mixed; posterior medullary, obex, or lateral) did not affect outcome. No patient was neurologically worse after brainstem surgery. At long-term follow-up review (mean 88.4 months), only one patient had declined neurologically and this was due to the cumulative neurological effects caused by eight additional hemangioblastomas of the spinal cord and their surgical treatment. Brainstem hemangioblastomas in patients with VHL disease can be removed safely; they generally should be resected when they become symptomatic or when the tumor has reached a size such that further growth will increase the risks associated with surgery, or in the presence of an enlarging cyst. Magnetic resonance imaging is usually sufficient for preoperative evaluation and presurgical embolization is unnecessary. The goal of surgery is complete resection of the lesion before the patient experiences a disabling neurological deficit.

Neurodevelopmental outcome at 3.5 years of age in children treated with extracorporeal life support: Relationship to primary diagnosis

Objective: Recent studies suggest that for neonates treated with extracorporeal membrane oxygenation (ECMO), children with congenital diaphragmatic hernia (CDH) have poorer neurodevelopmental outcome than children with other diagnoses. We therefore analyzed the neurodevelopmental outcome at 3½ years of age in 130 neonatal ECMO survivors with 6 different primary diagnoses. Study design: Children were assessed with the McCarthy Scales of Children’s Abilities, Peabody Picture Vocabulary Test, Vineland Adaptive Behavior Scales, and a neurologic/physical examination; 12 factors related to infant characteristics and ECMO/hospital course including primary diagnosis were identified as independent variables. Dependent variables included test scores and 2 outcome categories: functional status (normal, risk, abnormal) and major neurologic sequelae (presence or absence). Statistical tools included chi-squared analysis, t test, analysis of variance, and discriminant and regression analysis. Results: No significant differences were found between diagnostic groups in functional status or neurologic sequelae. Hospital days was the only variable consistently expressed in all analyses as having significant influence on the outcome measures. This was not a factor of the longer hospital days experienced by children with CDH. Conclusion: Neurodevelopmental outcome in neonatal ECMO is multifactorial. Although hospital days has the greatest association with outcome at age 3½ years, these days likely reflect degree of illness and various complications that are independent of diagnostic group. Further study is required to determine which factors influencing the length of hospital stay may be the best predictor of long-term outcome. (J Pediatr 2000;136:338-44)

Seizure outcome after temporal lobectomy for temporal lobe epilepsy: a Kaplan-Meier survival analysis.

To determine seizure outcome and its predictors in patients with medically refractory temporal lobe epilepsy (TLE) after temporal lobectomy (TL). TL is the most common surgical procedure performed in adolescents and adults for the treatment of medically refractory TLE. Seizure outcome has been reported extensively during the first few postoperative years, but little is known beyond that time. The authors analyzed seizure outcome in 79 patients who underwent TL for epilepsy at the Duke University Medical Center from 1962 through 1984. Patients with less than 2 years of follow-up and degenerative disorders were excluded. Predictors of seizure outcome were analyzed using Kaplan-Meier survival analyses. The mean follow-up was 14 years (range, 2.1 to 33.6 years). Using Engel's classification, 65% of patients were class I, 15% were class II, 11% were class III, and 9% were class IV. At least one postoperative seizure occurred in 55% of subjects. The majority of recurrences (86%) took place within 2 years of surgery. Later recurrences tended not to lead to medical intractability. Higher monthly preoperative seizure frequency was associated with poor seizure outcome. A seizure-free state at 2 years was found to be a better predictor of long-term outcome than the 6-, 12-, and 18-month landmarks. TL provides sustained, long-term benefit in patients with medically refractory TLE. Seizure-free status at 2 years from the time of surgery is predictive of long-term remission.

Early onset of lithium prophylaxis as a predictor of good long-term outcome.

The recurrence rates during lithium preventive treatment were investigated in a sample of 270 Mood Disorder subjects subdivided according to their onset time for lithium prophylaxis as very early (within 5 years from the onset of illness), early (6-10 years), late (11-20 years) and very late (more than 21 years). 131 subjects of the sample followed for 4 years prolonged the observation for a further period of 8 years. Results indicated that beginning lithium therapy within the first ten years of illness predicts better preventive outcomes than beginning prophylaxis later, both in major depression, recurrent and bipolar patients.

What Can We Learn from Provoking Ischemia?

What Can We Learn from Provoking Ischemia?

Characterisation of complete responders to combination chemotherapy for advanced breast cancer: a retrospective EORTC Breast Group study.

This retrospective study was undertaken to characterise the natural history of women achieving complete response (CR) following standard dose combination chemotherapy for metastatic breast cancer (MBC), and to analyse the significance of various patient, disease and treatment characteristics in determining survival and time to disease progression. 75 patients achieving a CR following standard dose combination chemotherapy or combined chemoendocrine therapy for MBC have been studied. At a median follow-up of 6 years, 28% of patients are still alive, with 18 of 21 patients showing no evidence of disease. 15 (20%) patients, with median follow-up of 61 months from start of chemotherapy, have never experienced relapse. Median overall survival is 32.5 months. Multivariate analysis for survival identified inclusion of anthracyclines and WHO performance status as significant predictors of good long-term outcome. Concomitant hormonotherapy almost reached statistical significance in our multivariate analysis. Neither dominant site of disease nor disease-free interval were significant determinants of complete remission. With conventional dose combination chemotherapy, approximately 20% of women with MBC who have achieved a clinical CR have been shown to be expected to remain alive and free of disease at 5 years. Inclusion of an anthracycline appears to be an important determinant of durability of CR and patient survival.