Long-term Outcome Of Adult Patients Research Articles

The Long-Term Outcome Of Adult Patients With Acute Myeloid Leukemia NPM+ and FLT3/ITD- Is Not Significantly Improved By The Application Of An Allogeneic Stem Cell Transplantation

▪ IntroductionThe discovery of the NPM mutation in acute myeloid leukemia (AML) allowed to identify a distinct entity with intermediate-good prognosis particularly when the FLT3/ITD mutation is absent. The most appropriate consolidation treatment of these patients upon the achievement of the first complete remission has not been established yet and the role of allogeneic stem cell transplantation (SCT) is still debated. Aimto assess long-term outcome of adult patients with NPM positive acute myeloid leukemia according to type and intensity of consolidation therapy. Patients and methodsBetween May 2000 and February 2012, 1155 patients were enrolled into two consecutive, prospective Northern Italy Leukemia Group (NILG) trials (00/01 and 02/06). Six-hundred sixty nine were studied for NPM mutation and 218 (33%) proved positive (by immunohistochemistry or by molecular analysis) (Falini et al, Haematologica. 2007 Apr;92(4):519-32). Median age of NPM+ patients was 50 years (range 16-72) and 134 (61%) were female. Median WBC was 33.3 x 10^9/L (range 0.9-313.9), 71 (33%) had myelomonocytic leukemia (FAB M4), and 211 (96%) had de novo AML. According to the European Leukemia Net (ELN) classification, cytogenetic risk groups were: normal 178 (82%), intermediate 26 (12%), unfavorable 2 (1%) and unknown 12 (5%). Eighty-two (38%) patients had a concurrent FLT3/ITD mutation and 31 (14%) a FLT3/TKD mutation. According to cytogenetics and additional risk factors (late response, WBC count >50x10^9/L, FAB class M0/6/7, hepato/splenomegaly, MDS-related/secondary AML, FLT3/ITD mutation), patients were stratified in standard (SR) and high (HR) risk groups.In both studies the remission induction was based on combination of cytarabine with idarubicin. Post-remission therapy was allogeneic SCT in HR while high-dose cytarabine or busulfan/cyclophosphamide with autologous SCT was given to SR patients. The molecular evaluation of minimal residual disease (MRD) was planned after induction, before the post-remission consolidation and the follow-up. ResultsComplete remission (CR) was achieved in 196/218 (90%) NPM+ patients. One hundred sixty-eight out of 196 remitters (86%) received post-remission consolidation therapy: allogeneic SCT 72 (37%), high dose Ara-C 74 (38%), autologous SCT 14 (7%), other therapy 8 (4%); 28 patients did not receive consolidation due to early relapse (n=24), CR death (n=3), and loss to follow-up (n=1). With a median follow-up of 1.8 years (range 0.008-12.66), 99 CR patients (50.5%) were alive in 1st CR, 13 (6.5%) died of complications, and 84 (43%) had recurrent AML. In a cumulative analysis, 5-year overall and disease-free survival were 46% (OS) and 43% (DFS), respectively. In univariate analysis FLT3/ITD mutation (n=82) affected negatively 5-year OS (29% vs. 49%, P < .0001) and DFS (27% vs. 46%, P < .0001), whereas FLT3/TDK mutation did not. In patients with FLT3/ITD mutation able to receive consolidation therapy (n=49), the application of allogeneic SCT improved DFS significantly (55% vs. 18%, P = .03) and reduced the cumulative incidence of relapse (CIR) (39% vs. 81%, P = .026). In patients with NPM+ and FLT3/ITD- AML, the risk of relapse after high dose cytarabine or autologous SCT (n=68) was more than doubled compared to that observed after allogeneic SCT (n=42) (50% vs. 23%, P= .08) (Figure). However, DFS (48% vs. 69.5%, p= .17), and OS (60% vs. 72%, p= .80) were not significantly different since allogeneic SCT was associated with higher treatment related mortality, albeit effective in the salvage of some relapsed patients. In multivariate analysis, FLT3/ITD mutation was the most powerful factor that unfavorably affected OS, DFS and CIR, while age > 55 years negatively affected OS and DFS. No other clinical factor was predictive for relapse in FLT3- patients. The relationship between MRD and clinical outcome is currently being studied and will be presented. ConclusionsOur data indicate that allogeneic SCT is the most active post-remission treatment for NPM+ AML, but its benefit over chemotherapy may be limited in patients without FLT3/ITD. For this reason the evaluation of MRD could help identify the patients for whom an allogeneic SCT should be preferable. [Display omitted] Disclosures:No relevant conflicts of interest to declare.

Long-term outcome of correction of tetralogy of Fallot in 56 adult patients

Background Although most patients with tetralogy of Fallot undergo radical repair during infancy and childhood, patients that remain undiagnosed and untreated until adulthood can still be treated. This study aimed to evaluate long-term outcomes of adult patients with tetralogy of Fallot who were treated surgically, and to determine the predictors of postoperative pulmonary regurgitation. Methods Fifty-six adult patients underwent complete surgical repair. Forty-three patients (76.8%) required a transannular patch. Systolic, diastolic, and mean pressure in the main pulmonary artery were measured after repair. Results The early mortality rate was 3.6%. The 16-year survival rate was (84.4±11.5)%. Late echocardiography revealed 41 patients with transannular patch who had pulmonary regurgitation, consisting of mild pulmonary regurgitation in 28 patients, moderate in eight, and severe regurgitation in five patients. In addition, there was right ventricular outflow tract stenosis in nine patients, moderate/severe tricuspid valve regurgitation in seven, and residual ventricular septal defect in five. Logistic regression analysis demonstrated that the mean pulmonary pressure measured just after repair predicted late pulmonary regurgitation. Conclusions The long-term survival of surgically treated adult patients with tetralogy of Fallot is acceptable. The mean pressure &gt;20 mmHg in the main pulmonary artery measured right after surgical repair may be a feasible reference to time the reconstruction of the pulmonary valve.

SAT0456 Long-Term Outcome in Adult Patients with Juvenile Idiopathic Arthritis: Preliminary Results from a Greek Population

BackgroundDuring the last decade there is a growing evidence on the long-term outcome of patients with juvenile idiopathic arthritis (JIA).ObjectivesTo describe the disease characteristics, course and long-term outcome of JIA...

Substance Use, Dental Hygiene, and Physical Activity in Adult Patients with Single Ventricle Physiology

The study aims to describe substance use, dental hygiene, and physical activity in adult survivors with single ventricle physiology (SVP) and to compare the behaviors with matched controls, while the patients are particularly at risk for general health problems. The present study is part of a larger research project on long-term outcomes in adult patients with SVP. A cross-sectional, case-control study including 59 patients out of 83 eligible patients participated in the study (response rate 71%). The patients were matched to 172 healthy controls. A questionnaire was mailed to the patients. Nonresponders did not differ significantly from the included participants on age, gender, or physical function. The study was conducted at the Heart Center of Copenhagen University Hospital or the Department of Cardiology, Aarhus University Hospital. In these patients, 85% report alcohol consumption (92% in controls; odds ratio [OR] = 0.91; P = 0.575); 26% admit "binge drinking" (41% in controls; OR = 0.56; P = 0.041); 20% are cigarette smokers (36% in controls; OR = 0.59; P = 0.100); 12% have used cannabis over the past year (15% in controls; OR = 0.80; P = 0.596); 20% have had no dental visits during the last year (25% in controls; OR = 1.07; P = 0.684); 46% are not flossing their teeth (32% in controls; OR = 1.32; P = 0.239); and 39% are not physically active (24% in controls; OR = 1.63; P = 0.069). While in general there was no significant differences in overall health behaviors between SVP patients and controls, SVP patients are less physically active and are less likely to binge drink.

Abstract TP443: Clinical Features, Surgical Treatment, And Long-term Outcome In Adult Patients With Moyamoya Disease In China

Background: Moyamoya disease develops mostly in Asian countries including Japan, Korea, mainland China and Taiwan. However, there was few detailed demographic and clinical data about Chinese patients with moyamoya disease. Here we describe the clinical features, surgical treatment, and long-term outcome of adults with moyamoya disease treated at a single institution in China. Methods: Our cohort included 470 adult patients with moyamoya disease. The demographic and clinical characteristics were obtained by retrospective chart review and long-term outcome was evaluated using the stroke status. Univariate and multivariate logistic regression analyses were performed to determine risk factors for postoperative morbidity and functional outcome. Risk of subsequent stroke was determined using the Kaplan-Meier method and Cox regression was used to determine risk factors for postoperative or subsequent strokes. Results: The median (range) age for the onset of symptoms was 36.8 (18 to 59) years. The ratio of female to male patients was 1:1(231/239). Familial occurrence of moyamoya disease was 2.3%. The most common initial symptom was a cerebral ischemic event. The incidence of postoperative ischemic events or hemorrhage was 5.9% (9.8% of patients). Older age of symptom onset, PCA involvement and present of TIA were identified as predictors of adverse postoperative events. The Kaplan-Meier estimate stroke risk was 10.1% in the first 2 years, and the 5-year-Kaplan-Meier risk of stroke was 13% after surgery for all patients treated with surgical revascularization. Older age of symptom onset, PCA involvement, and present of TIA were identified as predictors of postoperative or subsequent strokes. Overall, 73.2% of patients had an independent life with no significant disability and the strongest predictor being the preoperative mRS score. Conclusion: Clinical characteristics of adult moyamoya disease in China are different from that in other Asian countries. EDAS in adult patients with moyamoya disease carries a low risk, is effective at preventing future ischemic events, and improves quality of life.

Clinical Features, Surgical Treatment and Long-Term Outcome in Adult Patients with Moyamoya Disease in China

Background: Moyamoya disease (MMD) develops mostly in Asian countries including Japan, Korea, mainland China and Taiwan. However, there are few detailed demographic and clinical data about Chinese patients with MMD. Currently, the most effective treatment in adult patients with MMD is unknown. There have only been a few small case series reporting on encephaloduroarteriosynangiosis (EDAS) in an adult population. Here we describe the clinical features, surgical treatment and long-term outcome of adults with MMD treated at a single institution in China. Methods: Our cohort included 470 adult patients with MMD. The demographic and clinical characteristics were obtained by retrospective chart review and long-term outcome was evaluated using the stroke status. The modified Rankin Scale (mRS) was used to determine the neurological functional outcome. Univariate and multivariate logistic regression analyses were performed to determine risk factors for postoperative morbidity and functional outcome. The risk of subsequent stroke was determined using the Kaplan-Meier method and Cox regression was used to determine risk factors for postoperative or subsequent strokes. Results: The median age for the onset of symptoms was 36.8 (range, 18–59) years. The ratio of female to male patients was 1:1 (231/239). Familial occurrence of MMD was 2.3%. The most common initial symptom was a cerebral ischemic event. The incidence of postoperative ischemic events or hemorrhage was 5.9% (9.8% of patients). Older age at symptom onset, posterior cerebral artery (PCA) involvement and the presence of transient ischemic attack (TIA) were identified as predictors of adverse postoperative events. The Kaplan-Meier estimate stroke risk was 10.1% in the first 2 years, and the 5-year Kaplan-Meier risk of stroke was 13% after surgery for all patients treated with surgical revascularization. Older age at symptom onset, PCA involvement and the presence of TIA were identified as predictors of postoperative or subsequent strokes. Overall, 73.2% of patients had an independent life with no significant disability, with the strongest predictor being the preoperative mRS score. Conclusion: Clinical characteristics of adult MMD in China are different from those in other Asian countries. EDAS in adult patients with MMD carries a low risk, is effective at preventing future ischemic events and improves quality of life.

Childhood Philadelphia-chromosome-positive B-lymphoblastic leukaemia

The role of tyrosine-kinase inhibitors of BCR-ABL1 for treatment of de novo Philadelphia-chromosome-positive B-lymphoblastic leukaemia has changed since the inclusion of imatinib in front-line chemotherapy regimens. Results of several phase 2 studies have shown improved outcomes for adults, with long-term survival averaging 50–75% in patients aged 60 years or younger. 1 Thomas DA Faderl S Cortes J et al. Treatment of Philadelphia chromosome-positive acute lymphocytic leukemia with hyper-CVAD and imatinib mesylate. Blood. 2004; 103: 4396-4407 Crossref PubMed Scopus (453) Google Scholar , 2 Yanada M Takeuchi J Sugiura I et al. High complete remission rate and promising outcome by combination of imatinib and chemotherapy for newly diagnosed BCR-ABL-positive acute lymphoblastic leukemia: a phase II study by the Japan Adult Leukemia Study Group. J Clin Oncol. 2006; 24: 460-466 Crossref PubMed Scopus (359) Google Scholar , 3 Wassmann B Pfeifer H Goekbuget N et al. Alternating versus concurrent schedules of imatinib and chemotherapy as front-line therapy for Philadelphia-positive acute lymphoblastic leukemia (Ph+ ALL). Blood. 2006; 108: 1469-1477 Crossref PubMed Scopus (263) Google Scholar , 4 de Labarthe A Rousselot P Huguet-Rigal F et al. Imatinib combined with induction or consolidation chemotherapy in patients with de novo Philadelphia chromosome-positive acute lymphoblastic leukemia: results of the GRAAPH-2003 study. Blood. 2007; 109: 1408-1413 Crossref PubMed Scopus (270) Google Scholar , 5 Ribera JM Oriol A Gonzalez M et al. Concurrent intensive chemotherapy and imatinib before and after stem cell transplantation in newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia. Final results of the CSTIBES02 trial. Haematologica. 2010; 95: 87-95 Crossref PubMed Scopus (136) Google Scholar , 6 Bassan R Rossi G Pogliani EM et al. Chemotherapy-phased imatinib pulses improve long-term outcome of adult patients with Philadelphia chromosome-positive acute lymphoblastic leukemia: Northern Italy Leukemia Group protocol 09/00. J Clin Oncol. 2010; 28: 3644-3652 Crossref PubMed Scopus (205) Google Scholar Randomised trials that include a treatment group not taking tyrosine-kinase inhibitors during any phase of treatment have not been done in adults because of the poor prognosis of chemotherapy only; expected 2-year survival is 20–25% in the absence of allogeneic stem-cell transplantation in first complete remission. For childhood Philadelphia-chromosome-positive B-lymphoblastic leukaemia—which accounts for only 3–5% of cases of paediatric acute lymphoblastic leukaemia (ALL)—survival is 45–50% with chemotherapy, which is better than survival in adults but worse than in paediatric patients with Philadelphia-chromosome-negative ALL, for whom survival is more than 85%. 7 Arico M Schrappe M Hunger SP et al. Clinical outcome of children with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia treated between 1995 and 2005. J Clin Oncol. 2010; 28: 4755-4761 Crossref PubMed Scopus (163) Google Scholar Imatinib after induction for treatment of children and adolescents with Philadelphia-chromosome-positive acute lymphoblastic leukaemia (EsPhALL): a randomised, open-label, intergroup studyOur results suggests that imatinib in conjunction with intensive chemotherapy is well tolerated and might be beneficial for treatment of children with Philadelphia-chromosome-positive ALL. Full-Text PDF Open Access

Long-term outcomes for adult craniopharyngioma following radiation therapy

Background. We report long-term outcomes in adult patients with craniopharyngioma following surgery and radiation therapy (RT). Material and methods. Fifty-three patients treated with RT (median, 50 Gy in 25 fractions) between 1980 and 2009 with pathologically confirmed craniopharyngioma were reviewed (53% solid and 47% cystic/solid). The median age was 53 years (range, 22–76), 53% were female, 83% were sub-totally resected, 6% were gross totally resected and 11% had a biopsy and/or cyst aspiration alone. RT was delivered adjuvantly in 53% of patients as opposed to salvage intent upon progression. Results. Median follow-up was seven years (86 months, range, 8–259). The 5- and 10-year progression-free survival (PFS) rates were 85% and 69%, overall survival (OS) rates were 76% and 70%, and cause-specific survival (CSS) rates were both 88%, respectively. Both univariable and multivariable analysis identified age (<53 or ≥53) as a prognostic factor for OS (p =0.0003) and CSS (p =0.05). PFS was observed to be worse in patients with >2 surgeries prior to RT (p =0.01). Neither the intent of radiation or tumor type (cystic vs. solid/cystic) were prognostic or predictive. New endocrinopathies and visual dysfunction were observed in 53% and 17% of patients post-surgery, and in 11% and 6% post-RT, respectively. Conclusion. We report long-term favorable PFS, CSS and OS for craniopharyngioma post-RT. We observe age as a significant prognostic factor, however, timing of radiation was not.

Acute and Long-Term Outcomes of Catheter Ablation Using Remote Magnetic Navigation in Patients With Congenital Heart Disease

The aim of the present study was to assess the feasibility, safety, and long-term results of remote magnetic navigation in arrhythmias associated with complex congenital heart disease (CHD). The improved outcomes for CHD resulted in an increased number of complex arrhythmias requiring distinctive ablation techniques. Thirty-six patients with CHD (age 35 ± 19 years, 21 male) were divided into 3 complexity groups and underwent 43 radiofrequency catheter ablation procedures using the magnetic navigation system (including 7 redo ablations) in combination with the CARTO RMT system. A total of 59 tachyarrhythmias were identified. Most patients had surgical scar-related tachycardia (25 focal, including 4 microreentrant atrial tachycardia, and 27 macroreentrant atrial tachycardia). Four accessory pathways and three ventricular tachycardias were diagnosed and treated. In 31 patients, ablation was successful, with an end point of noninducibility (86%). The success rate for CHD complexity of type I, II, and III was 50%, 88%, and 89%, respectively. The mean procedure and fluoroscopy time was 216 ± 101 minutes and 40 ± 34 minutes, respectively. The number of radiofrequency applications was 42 ± 47. No major complications related to the procedures occurred. Of the patients, 67% remained free of recurrence during a mean follow-up of 26 ± 4 months. Recurrence developed in 0%, 16%, and 45% of patients with CHD type I, II, and III, respectively. In conclusion, the magnetic navigation system is feasible to treat arrhythmias with reasonable success rates and good long-term outcomes in adult patients with CHD. The use of the magnetic navigation system offers advantages in complex anatomic situations.

Chemotherapy-Phased Imatinib Pulses Improve Long-Term Outcome of Adult Patients With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia: Northern Italy Leukemia Group Protocol 09/00

Short imatinib pulses were added to chemotherapy to improve the long-term survival of adult patients with Philadelphia chromosome (Ph) -positive acute lymphoblastic leukemia (ALL), to optimize complete remission (CR) and stem-cell transplantation (SCT) rates. Of 94 total patients (age range, 19 to 66 years), 35 represented the control cohort (ie, imatinib-negative [IM-negative] group), and 59 received imatinib 600 mg/d orally for 7 consecutive days (ie, imatinib-positive [IM-positive] group), starting from day 15 of chemotherapy course 1 and from 3 days before chemotherapy during courses 2 to 8. Patients in CR were eligible for allogeneic SCT or, alternatively, for high-dose therapy with autologous SCT followed by long-term maintenance with intermittent imatinib. CR and SCT rates were greater in the IM-positive group (CR: 92% v 80.5%; P = .08; allogeneic SCT: 63% v 39%; P = .041). At a median observation time of 5 years (range, 0.6 to 9.2 years), 22 patients in the IM-positive group versus five patients in the IM-negative group were alive in first CR (P = .037). Patients in the IM-positive group had significantly greater overall and disease-free survival probabilities (overall: 0.38 v 0.23; P = .009; disease free: 0.39 v 0.25; P = .044) and a lower incidence of relapse (P = .005). SCT-related mortality was 28% (ie, 15 of 54 patients), and postgraft survival probability was 0.46 overall. This imatinib-based protocol improved long-term outcome of adult patients with Ph-positive ALL. With SCT, post-transplantation mortality and relapse remain the major hindrance to additional therapeutic improvement. Additional intensification of imatinib therapy should warrant a better molecular response and clinical outcome, both in patients selected for SCT and in those unable to undergo this procedure.

Long-term outcomes of a 5-year follow up of patients with immune thrombocytopenic purpura after splenectomy

BackgroundThe long-term outcomes of adult patients with immune thrombocytopenic purpura (ITP) after splenectomy are not clear.MethodsWe retrospectively analyzed 31 patients who underwent splenectomy after diagnosis of ITP at our institution between 1990 and 2009. Long-term follow-up was defined as a follow-up that lasted 1 year or more from splenectomy to the last follow-up.ResultsThe overall response rate to splenectomy was 84%. However, the response rate at 6 and 12 months decreased to 77% and 68%, respectively. During the 6 years of median follow-up after splenectomy, 11 patients (35%) relapsed. The long-term response rate was 55%. The long-term follow-up of 26 patients after responding to splenectomy showed that the median time from splenectomy to relapse was 19 months in the partial response (PR) group; however, there was no relapse after 9 months in the complete response (CR) group. Variables, including age, were not predictive of the long-term response after splenectomy. Additional treatment in patients who did not respond or relapsed after splenectomy was mostly effective. After a median follow-up of 7 years (range: 1-25 years) from the diagnosis, there were 2 deaths, including one due to spontaneous bleeding after repair of duodenal ulcer perforation.ConclusionAlthough splenectomy is safe and effective, the response rate after splenectomy continuously decreases over time. The duration of response is different between the patients that achieved CR and those that achieved PR. Factors, including age, were not predictors of a response to splenectomy.

Short Chemotherapy-Phased Imatinib (IM) Pulses Improve Long-Term Outcome of Adult Patients with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ ALL).

Abstract 2029Poster Board II-6In a prospective NILG (Northern Italy Leukemia Group) study, short IM pulses were added to chemotherapy in order to 1) reduce incidence of early failures, 2) obtain higher transfer rates to stem cell transplantation (SCT), and 3) improve survival in comparison with a prior patient cohort treated with the same chemotherapy program without IM. IM 600 mg/d was given orally for 7 consecutive dd. with each chemotherapy block, starting from day 15 of induction (IDR/VCR/PDN±ASP) and day –3 of the following consolidation courses (5x IDR/VCR/CY/DEXA; 2x HD-MTX/ARA-C). All pts. received CNS chemoradioprophylaxis and were eligible to allogeneic SCT, or alternatively to HD therapy with autologous SCT and long-term maintenance with 6MP/MTX and intermittent IM. Between April '00 and November '08, 100 out of 404 pts. registered in NILG study 09/00 had Ph+ ALL (Ph chromosome and/or BCR-ABL rearrangement). M/F ratio was 1.17 and median age 46 years (range 19-66). 35 pts. constituted the control cohort (IM-) while, starting December '02, 59 pts. were included in the modified protocol (IM+), and 6 were excluded from analysis because treated on a continuous IM schedule. Of 59 IM+ pts., 53 received IM during induction/consolidation as planned and 6 during consolidation only (included in IM- group for remission induction analysis). Outcome to induction therapy of IM+ vs. IM- group was: CR 49/53 (92%) vs. 33/41 (80%), NR 2 (3.7%) vs. 5 (12%), ED 2 (3.7%) vs. 3 (7%) (P=NS). With a median observation interval from diagnosis of 5 years (range 0.6-9.2 years), 21 IM+ vs. 6 IM- pts. are alive in remission (CR1 pts.: 43% vs. 18%, P=0.02), and both OS (Figure) and DFS (0.39 vs. 0.21, P=0.044) rates at 5 years are significantly improved in IM+ group, especially when IM was administered from the induction cycle. The ability to perform a SCT increased from 53%(n=15: 11 allogeneic, 4 autologous) to 68%(n=37: 22 allogeneic, 5 autologous), partly owing to lower incidence of early relapse (26% vs. 56% at 1 year, P=0.005). SCT-related mortality was not different (P=0.58) and postgraft survival probability at 5 years was 0.45 overall, again with no difference related to SCT source or IM. Use of IM was not associated with greater reduction of BCR-ABL transcripts: by PCR analysis of BM samples taken at weeks 10, 16 and 22 in pretransplantation pts., a major response (absent/<10-4 PCR signals) was documented in 13/27 (48%) determinations from 13 evaluable IM- pts. vs. 16/49 (33%) from 25 IM+ pts.(P=NS). The short IM schedule used in this multicentric study demonstrated synergy with chemotherapy, resulting for the first time in NILG studies in improved long-term survival for this adverse subset. Problems of SCT-related toxicity and postgraft relapse need to be addressed further, to confirm curability of Ph+ ALL in a greater proportion of cases. [Display omitted] Disclosures:No relevant conflicts of interest to declare.

Long-term outcomes in adult patients below the age of 55 years with acute lymphoblastic leukemia treated with chemotherapy or allogeneic BM transplant in first CR

Long-term outcomes in adult patients below the age of 55 years with acute lymphoblastic leukemia treated with chemotherapy or allogeneic BM transplant in first CR

Clinical features, surgical treatment, and long-term outcome in adult patients with moyamoya disease

The object of this study was to report the clinical features, surgical treatment, and long-term outcomes in adults with moyamoya phenomenon treated at a single institution in the US. Forty-three adult patients with moyamoya disease (mean age 40 +/- 11 years [SD], range 18-69 years) were treated with encephaloduroarteriosynangiosis (EDAS). Neurologists examined patients pre- and postoperatively. Follow-up was obtained in person or by structured telephone interview (median 41 months, range 4-126 months). The following outcomes were collected: transient ischemic attack (TIA), infarction, graft collateralization, change in cerebral perfusion, and functional level according to the modified Rankin scale (mRS). Kaplan-Meier estimates of infarction risk were calculated for comparison of surgically treated and contralateral hemispheres. The majority of patients were women (65%), were Caucasian (65%), presented with ischemic symptoms (98%), and had bilateral disease (86%). Nineteen patients underwent unilateral and 24 patients bilateral EDAS (67 treated hemispheres). Collateral vessels developed in 50 (98%) of 52 hemispheres for which imaging was available and there was evidence of increased perfusion on SPECT scans in 41 (82%) of the 50 hemispheres evaluated. Periprocedural infarction (< 48 hours) occurred in 3% of the hemispheres treated. In the follow-up period patients experienced 10 TIAs, 6 infarctions, and 1 intracranial hemorrhage. Although the hemisphere selected for surgery was based upon patients' symptoms and severity of pathology, the 5-year infarction-free survival rate was 94% in the surgically treated hemispheres versus < 36% in the untreated hemispheres (p = 0.007). After controlling for age and sex, infarction was 89% less likely to occur in the surgically treated hemispheres than in the contralateral hemispheres (hazard ratio 0.11, 95% CI 0.02-0.56). Thirty-eight (88%) of 43 patients had preserved or improved mRS scores, relative to baseline status. In this mixed-race population of North American patients, indirect bypass promoted adequate pial collateral development and increased perfusion in the majority of adult patients with moyamoya disease. Patients had low rates of postoperative TIAs, infarction, and hemorrhage, and the majority of patients had preserved or improved functional status.

A comparison of segmental vs subtotal/total colectomy for colonic Crohn's disease: a meta‐analysis

Using meta-analytical techniques the present study evaluated differences in short-term and long-term outcomes of adult patients with colonic Crohn's disease who underwent either colectomy with ileorectal anastomosis (IRA) or segmental colectomy (SC). Comparative studies published between 1988 and 2002, of subtotal/total colectomy and ileorectal anastomosis vs segmental colectomy, were used. The study end points included were surgical and overall recurrence, time to recurrence, postoperative morbidity and incidence of permanent stoma. Random and fixed-effect meta-analytical models were used to evaluate the study outcomes. Sensitivity analysis, funnel plot and meta-regressive techniques were carried out to explain the heterogeneity and selection bias between the studies. Six studies, consisting of a total of 488 patients (223 IRA and 265 SC) were included. Analysis of the data suggested that there was no significant difference between IRA and SC in recurrence of Crohn's disease. Time to recurrence was longer in the IRA group by 4.4 years (95% CI: 3.1-5.8), P < 0.001. There was no difference between the incidence of postoperative complications (OR = 1.4., 95% CI 0.16-12.74) or the need for a permanent stoma between the two groups (OR = 2.75, 95% CI 0.78-9.71). Patients with two or more colonic segments involved were associated with lower re-operation rate in the IRA group, a difference which did not reach statistical significance (P = 0.177). Both procedures were equally effective as treatment options for colonic Crohn's disease however, patients in the SC group exhibited recurrence earlier than those in the IRA group. The choice of operation is dependent on the extent of colonic disease, with a trend towards better outcomes with IRA for two or more colonic segments involved. Since no prospective randomised study has been undertaken, a clear view about which approach is more suitable for localised colonic Crohn's disease cannot be obtained.

A Rare Tricuspid Anomaly

54-year-old Mexican American woman was admitted to the Erie County Medical Center, Buffalo, NY, with the general symptoms of tiredness, weight loss with loss of appetite, leg weakness, and low blood pressure (80/60 mm Hg). The patient denied any paroxysmal nocturnal dyspnea, orthopnea, or leg edema. She did have some facial swelling, a skin rash on both hands, and joint swelling, which was consistent with a previous diagnosis of polymyositis and dermatomyositis. The patient also had evidence of septic arthritis of the left elbow. Physical examination revealed extensive wheezing and a grade 2/3 systolic ejection murmur, heard predominantly over the tricuspid valve area. Laboratory workup revealed pancytopenia and an elevation in liver function tests. All the markers for autoimmune disease were negative. Biopsies and computerized tomographic scans performed to search for visceral malignancy were negative. Blood cultures grew methicillin-sensitive Staphylococcus aureus. Electrocardiography revealed normal sinus rhythm with evidence of right atrial enlarge

Patients' recollections of stressful experiences while receiving prolonged mechanical ventilation in an intensive care unit.

To describe stressful experiences of adult patients who received mechanical ventilation for > or =48 hrs in an intensive care unit. Prospective cohort study. Four intensive care units within an East Coast tertiary-care university medical center. Patients were 150 adult intensive care unit patients receiving mechanical ventilation for > or =48 hrs. None. As part of a study of the long-term outcomes of adult patients requiring prolonged mechanical ventilation, we used a 32-item questionnaire to collect data on patients' stressful experiences, both psychological (e.g., fearfulness, anxiety) and physical (e.g., pain, difficulty breathing), associated with the mechanical ventilation endotracheal tube and with being in an intensive care unit. Of 554 patients who met study criteria and survived prolonged mechanical ventilation, 150 consented and were oriented to person, place, and situation. Two thirds of these patients remembered the endotracheal tube and/or being in an intensive care unit. The median numbers of endotracheal tube and intensive care unit experiences remembered were 3 (of 7) and 9 (of 22), respectively. If a patient remembered an experience in the questionnaire, it was likely to be moderately to extremely bothersome. Some of the items that many patients found to be moderately to extremely bothersome were pain, fear, anxiety, lack of sleep, feeling tense, inability to speak/communicate, lack of control, nightmares, and loneliness. Stressful experiences associated with the endotracheal tube were strongly associated with subjects' experiencing spells of terror, feeling nervous when left alone, and poor sleeping patterns. Subjects were more likely to remember experiences that were moderately to extremely bothersome. This might be because the more bothersome experiences were easier to recall or because most of these experiences are common and significant stressors to many of these patients. In either case, these data indicate that these patients are subject to numerous stressful experiences, which many find quite bothersome. This suggests the potential for improved symptom management, which could contribute to a less stressful intensive care unit stay and improved patient outcomes.

Long-Term Outcome in Adult Patients With Early-Treated Phenylketonuria (PKU).73

Background: Due to late onset neurological symptoms, MRI abnormalities, and neuropsychological deficits the long-term outcome of treated PKU is still unclear.

Balloon aortic valvuloplasty in adults: Failure of procedure to improve long-term survival

This study sought to determine the long-term outcome of adult patients undergoing percutaneous balloon aortic valvuloplasty. Percutaneous balloon aortic valvuloplasty has been offered as an alternative to aortic valve replacement for selected patients with valvular aortic stenosis. Although balloon aortic valvuloplasty produces an immediate reduction in the transvalvular aortic gradient, a high incidence of restenosis frequently leads to recurrent symptoms. Therefore, it is unclear whether balloon aortic valvuloplasty impacts on the long-term outcome of these patients. Clinical, hemodynamic and echocardiographic data were collected at baseline in 165 patients undergoing balloon aortic valvuloplasty and examined for their ability to predict long-term outcome. The median duration follow-up was 3.9 years (range 1 to 6). Ninety-nine percent follow-up was achieved. During this 6-year period, 152 patients (93%) died or underwent aortic valve replacement, and 99 (60%) died of cardiac-related causes. The probability of event-free survival (freedom from death, aortic valve replacement or repeat balloon aortic valvuloplasty) 1, 2 and 3 years after valvuloplasty was 40%, 19% and 6%, respectively. In contrast, the probability of survival 3 years after balloon aortic valvuloplasty in a subset of 42 patients who underwent subsequent aortic valve replacement was 84%. Survival after aortic valvuloplasty was poor regardless of the presenting symptom, but patients with New York Heart Association functional class IV congestive heart failure had events earliest. Univariable predictors of decreased event-free survival were younger age, advanced congestive heart failure symptoms, lower ejection fraction, elevated left ventricular end-diastolic pressure, presence of coronary artery disease and increased left ventricular internal diastolic diameter. Stepwise multivariable logistic regression analysis found that only younger age and a lower left ventricular ejection fraction contributed independent adverse prognostic information (chi-square 14.89, p = 0.0006). Long-term event-free and actuarial survival after balloon aortic valvuloplasty is dismal and resembles the natural history of untreated aortic stenosis. Aortic valve replacement may be performed in selected subjects with good results. However, the prognosis for the remainder of patients who are not candidates for aortic valve replacement is particularly poor.