Natural History Of Patients Research Articles

782 - Natural History of Patients Diagnosed with Asymptomatic Gallstones

782 - Natural History of Patients Diagnosed with Asymptomatic Gallstones

Biochemical Recurrence after Radical Prostatectomy

After receiving local treatment, many patients will develop a biochemical recurrence (BCR) in the absence of detectable distant disease (cM0) and comprise a significant proportion (20.1%) of prostate cancer disease states. The natural history of patients with BCR ranges from those with indolent, nonprogressive, slow prostate-specific antigen (PSA)-only progression to those ultimately destined to develop metastases and progress to a cancer-specific death. Pathologic predictors of BCR, clinical progression, and cancer-specific mortality are well established in the literature, although multiple novel predictors are emerging, which are highlighted. Traditional imaging cannot reliably distinguish local versus distant microscopic metastasis at the PSA levels that have been shown to confer survival advantage for salvage radiation therapy. We review past and present imaging standards and discuss novel imaging modalities, which may improve staging and offer opportunity for novel salvage therapies, including salvage lymph node dissection and stereotactic beam radiation therapy. With an emphasis on BCR after radical prostatectomy, both curative and palliative treatments are reviewed. This review contains 7 figures, 6 tables and 73 references Key words: biochemical recurrence, clinically undetectable metastases, molecular imaging, monitoring treatment response, prostate cancer, radical prostatectomy, rising prostate-specific antigen, salvage lymph node dissection, salvage radiation

Impact of age at diagnosis on natural history of patients with elderly-onset ulcerative colitis: A French population-based study

BackgroundRecent population-based study of elderly-onset Crohn’s disease patients reported age-related differences in disease phenotype and outcome. AimsThe aim was to assess the impact of age at diagnosis on natural history of elderly-onset ulcerative colitis patients with emphasis on disease presentation, phenotype and treatment. MethodsElderly-onset patients with ulcerative colitis (≥60 years at diagnosis) registered in a French population-based Registry EPIMAD (1988–2006) were included. Demographic and clinical data at diagnosis and at maximal follow-up were collected using predefined questionnaire. ResultsFour-hundred and sixty-five elderly-onset ulcerative colitis patients were included (median follow-up 6.2 years); 276 (59%) were <70 and 189 (41%) ≥70 years at diagnosis. Patients aged <70 years presented with more rectal bleeding (86% vs. 79%, p = .06) and abdominal pain (44% vs. 34%, p = .04) while those ≥70 years had higher rate of left-sided colitis (62% vs. 49%; p = .02). Cumulative exposure to 5-ASA, corticosteroids and immunosuppressants was similar between the groups as well as surgery rate. However, patients <70 years were significantly more steroid-resistant than older individuals (12% vs. 3%, p < .05) while no significant difference in steroid-dependency was observed. ConclusionPatients with elderly-onset ulcerative colitis differed in presentation, disease phenotype and response to medication with respect to age at diagnosis.

SAT-242 - Natural history of patients with compensated cirrhosis and a hepatic venous pressure gradient >20 mmHg: A prospective longitudinal cohort study

SAT-242 - Natural history of patients with compensated cirrhosis and a hepatic venous pressure gradient >20 mmHg: A prospective longitudinal cohort study

Liver toxicity associated with tuberculosis chemotherapy in the REMoxTB study

BackgroundDrug-induced liver injury (DILI) is a common complication of tuberculosis treatment. We utilised data from the REMoxTB clinical trial to describe the incidence of predisposing factors and the natural history in patients with liver enzyme levels elevated in response to tuberculosis treatment.MethodsPatients received either standard tuberculosis treatment (2EHRZ/4HR), or a 4-month regimen in which moxifloxacin replaced either ethambutol (isoniazid arm, 2MHRZ/2MHR) or isoniazid (ethambutol arm, 2EMRZ/2MR). Hepatic enzymes were measured at 0, 2, 4, 8, 12 and 17 weeks and as clinically indicated during reported adverse events. Patients included were those receiving at least one dose of drug and with two or more hepatic enzyme measurements.ResultsA total of 1928 patients were included (639 2EHRZ/4HR, 654 2MHRZ/2MHR and 635 2EMRZ/2MR). DILI was defined as peak alanine aminotransferase (ALT) ≥ 5 times the upper limit of normal (5 × ULN) or ALT ≥ 3 × ULN with total bilirubin > 2 × ULN. DILI was identified in 58 of the 1928 (3.0%) patients at a median time of 28 days (interquartile range IQR 14–56). Of 639 (6.4%) patients taking standard tuberculosis therapy, 41 experienced clinically significant enzyme elevations (peak ALT ≥ 3 × ULN). On standard therapy, 21.1% of patients aged >55 years developed a peak ALT/aspartate aminotransferase (AST) ≥ 3 × ULN (p = 0.01) and 15% of HIV-positive patients experienced a peak ALT/AST ≥ 3 × ULN compared to 9% of HIV-negative patients (p = 0.160). The median peak ALT/AST was higher in isoniazid-containing regimens vs no-isoniazid regimens (p < 0.05), and lower in moxifloxacin-containing arms vs no-moxifloxacin arms (p < 0.05). Patients receiving isoniazid reached a peak ALT ≥ 3 × ULN 9.5 days earlier than those on the ethambutol arm (median time of 28 days vs 18.5 days). Of the 67 Asian patients with a peak ALT/AST ≥ 3 × ULN, 57 (85.1%) were on an isoniazid-containing regimen (p = 0.008).ConclusionsOur results provide evidence of the risk of DILI in tuberculosis patients on standard treatment. Older patients on standard therapy, HIV-positive patients, Asian patients and those receiving isoniazid were at higher risk of elevated enzyme levels. Monitoring hepatic enzymes during the first 2 months of standard therapy detected approximately 75% of patients with a peak enzyme elevation ≥3 × ULN, suggesting this should be a standard of care. These results provide evidence for the potential of moxifloxacin in hepatic sparing.

Prevalence and clinical challenges among adults with primary immunodeficiency and recombination-activating gene deficiency

Prevalence and clinical challenges among adults with primary immunodeficiency and recombination-activating gene deficiency

Hepatic neuroendocrine tumors without an identifiable extrahepatic primary

Background: Hepatic neuroendocrine tumors without an identifiable primary represent either primary tumors or metastases from occult primaries. Primary hepatic neuroendocrine tumors (NET) are very rare. The natural history of patients with liver only disease is poorly defined. Methods: Patients with resected hepatic neuroendocrine tumors were identified for the years 1998–2017. Clinicopathologic variables were abstracted by chart review and immunohistochemistry reviewed for all specimens. Results: Thirteen patients with hepatic NET, without any evidence of non-hepatic primary disease were identified. Extrahepatic primary disease was not identified during a median follow-up of 38 months (1–164 months). The mean age at diagnosis was 54.7 years, 62% female. Pre-operative evaluation to rule out a primary source included CT enterography (n = 1), EUS/ERCP (n = 8), and octreotide scan (n = 11). Six patients had a solitary lesion, while 7 had multifocal disease. Operations performed were right hepatectomy (n = 4), left hepatectomy (n = 4), central hepatectomy (n = 1) and others had a combination of wedge resections and radiofrequency ablation. Six patients had Grade I disease, 6 were Grade 2 and one patient was classified as Grade 3. Two patients died 20 and 33 months after surgery secondary to disease progression (progressive liver disease and solitary brain metastases), both of which were grade 2 with a Ki-67 of 5-10%. The overall survival was 54.1 months (1–164 months) and recurrence free survival was 25.8 months (1–82 months). Conclusion: Primary hepatic NETs are rare, as are liver metastases without an identifiable primary. The natural history of these tumors is in keeping with other neuroendocrine tumors and is generally favorable, especially for low grade disease.

ECHO-DOPPLER ASSESSMENT OF VENTRICULAR FILLING PRESSURES IN ADULT FONTAN PATIENTS

Diastolic dysfunction is felt to be part of the natural history of patients with prior Fontan operation. Despite that, data on noninvasive assessment of diastolic function and ventricular filling pressures in Fontan patients are limited. We included 48 adult Fontan patients who underwent right

Improving Revised International Prognostic Scoring System Pre-Allogeneic Stem Cell Transplantation Does Not Translate Into Better Post-Transplantation Outcomes for Patients with Myelodysplastic Syndromes: A Single-Center Experience

The natural history of patients with myelodysplastic syndromes (MDS) is variable. The Revised International Prognostic Score (IPSS-R) is commonly used in practice to predict outcomes in patients with MDS at both diagnosis and before hematopoietic stem cell transplantation (HSCT). However, the effect of change in the IPSS-R before allogeneic HSCT with chemotherapy or hypomethylating agents on post-transplantation outcomes is currently unknown. We assessed whether improvement in IPSS-R prognostic score pre-HSCT would result in improvement in clinical outcomes post-HSCT. Secondary goals included studying the effect of prognostic factors on post-transplantation survival. All patients with MDS who underwent allogeneic HSCT at the Leukemia/BMT Program of British Columbia between February 1997 and April 2013 were included. Pertinent information was reviewed from the program database. IPSS-R was calculated based on data from the time of MDS diagnosis and before HSCT. Outcomes of patients who had improved IPSS-R pre-HSCT were compared with those with stable or worse IPSS-R. Overall survival (OS) and event-free survival (EFS) were estimated using the Kaplan-Meier method, with P values determined using the log-rank test. Hazard ratios were calculated using multivariable Cox proportional hazards regression models to study the effects of the prognostic variables on OS and EFS. A total of 138 consecutive patients were included. IPSS-R improved in 62 of these patients (45%), worsened in 23 (17%), remained stable in 41 (30%), and was unknown in 12 (9%). OS was not statistically different across the improved, worsened, and stable groups (30% versus 22% versus 40%, respectively; P = .63). The cumulative incidences of relapse and nonrelapse mortality at 5 years were 28.4% (95% confidence interval [CI], 21.1 to 36.1) and 31.6% (95% CI, 23.8 to 39.7), respectively. The rate of relapse was 23% in patients with <5% blasts at the time of HSCT, 69% in those with 5% to 20% blasts, and 66% in those with >20% blasts (P = .0004). In the entire cohort OS was 34% and EFS was 33%. There was no significant difference in outcomes between patients who received myeloablative conditioning and those who received nonmyeloablative conditioning before HSCT (OS, 34% and 39%, respectively; P = .63 and EFS, 34% and 32%, respectively; P = .86). OS was not statistically different among patients with improved, worsened, or stable IPSS-R. On multivariate analysis, only 3 factors were associated with OS: cytogenetic risk group at diagnosis, blast count at transplantation, and the presence or absence of chronic graft-versus-host disease. Improving IPSS-R before HSCT does not translate into better survival outcomes. Blast count pretransplantation was highly predictive of post-transplantation outcomes.

Abstract WP12: Mechanical Thrombectomy for Basilar Artery Occlusion

Background and Purpose: Basilar artery occlusion (BAO) is an infrequent cause of stroke, accounting for 1.1% of acute ischemic stroke cases. The natural history of patients with BAO is devastating, with morbidity rates of up to 80%. No previous randomized controlled studies on the efficacy of recanalization therapy have been reported. Methods: In the present single-center study, consecutive BAO patients were treated with mechanical thrombectomy (MT) using the stent retriever and the Penumbra system. Computed tomography perfusion was used for patient evaluation. Clinical outcomes were correlated with demographic, clinical, and radiographic findings. Results: Between October 2011 and March 2017, MT was performed in 37 patients with BAO (mean age 70.1±10.9). Mean baseline National Institutes of Health Stroke Scale was 23±11. Recanalization rate (≧thrombolysis in cerebral infarction 2b) was 100%. Mean onset to recanalization time (OTR) was 226.3±117.8minutes. Favorable outcome at 90 days (modified Rankin scale≦2) was 60.6%. Mortality rate at 90 days was 10.8%. In an univariate analysis, IV rt-PA use, and OTR were significantly associated with favorable outcomes. In a multivariate logistic regression model, IV rt-PA use and lower NIHSS score were significantly related to favorable outcomes. Conclusions: Multimodal endovascular therapy using the Penumbra system and/or stent retriever demonstrated high recanalization rates and improved outcomes in BAO. Both devices were feasible and effective in the treatment of BAO. An approach combining MT with IV thrombolysis provided better recanalization rates and improved clinical outcomes.

Anticoagulant therapy in patients with liver cirrhosis and portal vein thrombosis: insights for the clinician.

Portal vein thrombosis (PVT) is a frequent complication in the natural history of patients with liver cirrhosis (LC). The prevalence of PVT in LC is highly variable, ranging from 0.6% to 25% according to different reports. The impact of PVT on the natural history of LC is unclear, but it seems to negatively affect the prognosis of patients undergoing liver transplantation (LT) by increasing post-LT mortality and delaying waiting time. The antithrombotic treatment of PVT is still challenging as PVT may often remain asymptomatic and incidentally diagnosed, and a spontaneous partial/total regression of PVT is observed in an important proportion of patients, even in the absence of anticoagulation. Recent evidence suggested that the anticoagulant treatment for PVT may favorably affect both ischemic and bleeding outcomes in LC patients. Anticoagulant therapies so far available include unfractioned heparin, low molecular weight heparins (LMWHs) and fondaparinux for acute treatment, and LMWHs and vitamin K antagonists (VKAs) for long-term treatment. No robust data currently support the use of direct oral anticoagulants (DOACs) in patients with LC and PVT, as the safety and efficacy of DOACs in this setting is still unclear. This review summarizes current evidence for the evaluation and management of patients with LC and PVT.

Major Abdominal and Perianal Surgery in Crohn's Disease: Long-term Follow-up of Australian Patients With Crohn's Disease.

Most patients with Crohn's disease still require surgery despite significant advances in medical therapy, surveillance, and management strategies. The purpose of this study was to assess surgical strategies and outcomes in Crohn's disease, including surgical recurrence and emergency surgery. This was a multicenter, retrospective review of a prospectively collected database. A specialist-referred cohort of patients with Crohn's disease between 1970 and 2009 was studied. Included were 972 patients with Crohn's disease who were referred to the Sydney Inflammatory Bowel Disease cohort database. Main outcomes of interest were the rates of major abdominal and perianal surgery between decades (1970-1979, 1980-1989, 1990-1999, and 2000-2009), indications for surgery, types of procedure performed, rate of elective and emergency surgery, risk of surgical recurrence, and predictive factors for surgery. Between 1970 and 2009, the overall risks of surgery within 5, 10, and 15 years of diagnosis were 31.7%, 43.3%, and 48.4%. The median time to first surgery from time of diagnosis was 2 years (range, 0-31 years). A total of 6.7% of patients required emergency surgery within 5 years of diagnosis. In total, 8.8% of patients required emergency surgery within 15 years. The overall risk of surgical recurrence was 35.9%. The risk of major abdominal surgery significantly decreased between 2000 and 2009 when compared with the 1970 to 1979 period (OR = 0.49 (95% CI, 0.34-0.70). However, the rate of perianal surgery significantly increased (OR = 5.76 (95% CI, 2.54-13.06)). The main indications for surgery were enteric stricture or obstruction, perianal disease, and intra-abdominal fistulas/abscess. Of the 972 patients over 4 decades, only 11 patients (1.1%) were diagnosed with colorectal cancer. This was a specialist-referred cohort, not a population-based study. The rate of major abdominal surgery has decreased, with surgery reserved for more severe and complicated disease. The natural history of patients with more complicated Crohn's disease and severe phenotypes puts them at higher risk of surgical recurrence and emergency surgery. There has been no reduction in emergency surgery rates and there has been an increase in surgical recurrence despite the reduction in surgical rate morbidity. See Video Abstract at http://links.lww.com/DCR/A483.

Characteristics of 26 patients with type 3 Gaucher disease: A descriptive analysis from the Gaucher Outcome Survey

The Gaucher Outcome Survey (GOS) is an international disease-specific registry established in 2010 for patients with a confirmed diagnosis of Gaucher disease (GD), regardless of GD type or treatment status. Historically, there has been a limited understanding of type 3 GD (GD3) and its natural history in patients irrespective of their treatment status. Here, we describe the disease characteristics of patients with GD3 enrolled in GOS. As of October 2015, 1002 patients had been enrolled, 26 of whom were reported as GD3. The majority of patients with GD3 were from the US (13; 50.0%), seven (26.9%) were from the UK, three (11.5%) from Israel, and three (11.5%) from Brazil. No patients were of Ashkenazi Jewish origin. Median age of symptom onset was 1.4 (interquartile range: 0.5–2.0) years. The most common GBA1 mutation genotype was L444P/L444P, occurring in 16 (69.6%) of 23 patients who had genotyping information available. Nine patients reported a family history of GD (any type). Of 21 patients with treatment status information, 20 (95.2%) had received GD-specific treatment at any time, primarily imiglucerase (14 patients) and/or velaglucerase alfa (13 patients). Hemoglobin concentrations and platelet counts at GOS entry were within normal ranges for most patients, and there were no reports of severe hepatomegaly or of splenomegaly in non-splenectomized patients, most likely indicative of the effects of treatment received prior to GOS entry. This analysis provides information on the characteristics of patients with GD3 that could be used as the baseline for longitudinal follow-up of these patients.

Risk of peritoneal metastases in patients who had negative peritoneal staging and received therapy for localized gastric adenocarcinoma.

Positive peritoneal cytology (+PCyt) or gross carcinomatosis (GPC) carries a poor prognosis. Laparoscopic staging to detect +PCyt/GPC is recommended for all ≥T1b gastric adenocarcinoma (GAC). The natural history of patients with GAC who have baseline -PCyt and then undergo multimodality therapy is not well documented, particularly for the risk of subsequent GPC. We identified 238 GAC patients with baseline -PCyt who were followed for the development of peritoneal carcinomatosis (PC). Standard statistical methods were employed. Of 238 patients, 192 had attempted surgery after preoperative therapy. Of these, 13 patients (6.8%) had GPC and one had liver metastases, thus surgery was aborted. We followed 164 patients who had an R0 resection. The median follow-up duration was 3.4 (range, 0.6-18) years. The rate of PC was 13.4%, (22/164 patients) and the median time to PC was 15.6 months. Female gender was associated with PC on multivariate analysis. The 5-year OS rate for patients without subsequent PC was 75%. Conclusion Even with baseline -Cyt, ∼25% of patients develop PC following multimodality therapy. Patients who do not develop PC have an excellent OS rate. Further research is warranted to detect PC at baseline by the use of biomarkers.

Survival of localized NSCLC patients without active treatment or treated with SBRT

Background: Little information on the natural history of patients with localized NSCLC is available since many of the studies covering the subject lack information on pathological confirmation, staging procedures and comorbidity. No randomized studies have compared SBRT with no treatment for patients with localized NSCLC. The purpose of this study was to evaluate whether SBRT has influence on overall survival for patients with localized NSCLC and investigate the effect of baseline ventilatory lung function on overall survival.Material and methods: From 2007 to 2013, 136 patients treated with SBRT at Odense University Hospital were prospectively recorded. The thoracic SBRT consisted of three fractions of 15–22 Gy delivered in 9 days. For comparison, a national group of 73 untreated patients in the same time period was extracted from the Danish Lung Cancer Registry. All patients had histologically/cytologically proven NSCLC T1-2N0M0 with a tumour diameter ≤5 cm.Results: The 5-year relative survival was 44% versus 7% for the SBRT and untreated groups, respectively. In a propensity score matched comparison the median overall survival was 47 months versus 11 months for the SBRT and untreated groups, respectively (p < .05). On multivariate analysis, SBRT was significantly associated with improved prognosis while ECOG performance status 2+ and tumour diameter ≥3 cm significantly predicted poorer prognosis. Severe to very severe reduction of forced expiratory volume in one second (FEV1) did not predict poorer survival for the SBRT treated patients with localized NSCLC.Conclusions: SBRT offers more favourable survival than no treatment for patients with localized NSCLC. Performance status of 0–1, tumour diameter less than 3 cm and SBRT predicted improved survival. SBRT should not be withheld for patients with localized NSCLC based on poor ventilatory lung function.

The natural history of the patients with Duchenne muscular dystrophy in Taiwan: A medical center experience

Duchenne muscular dystrophy (DMD) is the most common hereditary muscular dystrophy and caused by DMD gene mutation. In addition to progressive proximal muscle weakness, respiratory, orthopedic, and gastrointestinal complications are often observed in DMD. The natural history of patients with DMD in Taiwan has not been reported thus far. Medical records of 39 patients who received a diagnosis of DMD between 1999 and 2016 at Kaohsiung Medical University Hospital were reviewed. The diagnosis of DMD was confirmed through muscle biopsy or DMD genetic analysis. The mean onset age and mean follow-up period were 2.75 years and 6.76 years, respectively. Seventeen patients (43.5%) had a family history of DMD. The mean full intelligence quotient of the patients was 71.08, and the mean age of walking ability loss was 9.7 years (25 patients). The mean onset age of respiratory insufficiency was 10.64 years with a decline rate of 5.18% per year (25 patients). The mean onset age of cardiomyopathy was 14.69 years (seven patients). The mean onset age of scoliosis was 13.29 years with a progression rate of 11.48° per year (14 patients). Eleven (28.2%) and eight (20.5%) patients had deletions and duplications of DMD, respectively. Fourteen patients (35.9%) had point mutations or small deletions or insertions. Five patients received only multiplex ligation-dependent probe amplification (MLPA) analysis and exhibited neither deletion nor duplication. No mutation was identified in one patient through both MLPA and exon sequencing. The clinical phenotypes and disease course in our cohort were consistent with that reported in previous studies. However, the proportion of point mutations or small deletions or insertions in our study was considerably higher than that in reports from other populations. Cardiac ejection fraction was found not a reliable biomarker for identifying cardiac problems, discovering a better parameter is necessary.

Low-Flow Severe Aortic Stenosis: Evolving Role of Transcatheter Aortic Valve Replacement.

The definition of severe aortic stenosis has classically and retrospectively been based on the natural history of patients with medically managed aortic stenosis and preserved left ventricular function in an era where surgical aortic valve replacement was the sole therapy. We now recognize that this disease is more heterogeneous and includes important subsets of patients with low stroke volume index (low flow) and low-gradient with reduced (classical) or preserved (paradoxical) ejection fraction. These patients pose diagnostic and treatment dilemmas, requiring a comprehensive assessment with integration of multimodality imaging, testing, and clinical assessment. Surgery in these patients has been associated with higher operative mortality and lower long-term survival. Transcatheter aortic valve replacement (TAVR), because of its less-invasive nature, avoidance of the detrimental effects of cardiopulmonary bypass, and larger effective orifice area, offers several potential advantages. Studies of TAVR in low-flow severe aortic stenosis patients have demonstrated that TAVR has a significant mortality benefit compared with medical therapy and a similar benefit compared with surgery. Both low flow and low ejection fraction have emerged as important factors in predicting mortality post-TAVR, with particularly poor survival when flow or ejection fraction fail to improve. The recognition, diagnosis, and treatment of patients with low-flow severe aortic stenosis remains challenging. It is likely that TAVR will play an increasingly important role in the management of these patients.

Malignant pheochromocytoma-paraganglioma: pathogenesis, TNM staging, and current clinical trials.

Pheochromocytomas and paragangliomas (PPGs) are rare neuroendocrine tumors. Over the last 15 years, substantial progress has been made toward understanding the clinical aspects and molecular origins of this disease. Nevertheless, predicting and managing malignancy remains the biggest challenge in clinical practice. The natural history of patients with malignant PPGs has not yet been described, and their prognosis varies. Currently, the diagnosis of malignant PPGs relies on the presence of metastases, by which time the disease is usually advanced. Better understanding of the clinical and molecular characteristics of patients with malignant PPGs has spurred several prospective clinical trials. Several molecular targeted therapies, a novel radiopharmaceutical medication that targets the catecholamine transporter, and immunotherapy are under evaluation for the treatment of patients with malignant PPGs. Furthermore, the identification of clinical predictors of malignancy and survival has led to the first TNM staging classification for PPGs. Prospective clinical trials are providing patients with therapeutic options beyond systemic chemotherapy. The knowledge derived from these trials and from the evaluation of the TNM staging in clinical practice will help to clarify how to most effectively treat malignant PPGs.

Natural History of Patients Followed Radiographically with Mucinous Cysts of the Pancreas

ObjectiveThe aim of this study was to evaluate the outcome of patients presumed to have mucinous cysts of the pancreas who were initially selected for radiographic surveillance. MethodsPatients with a pancreatic cyst and a measured cyst fluid carcinoembryonic antigen (CEA) ≥192 ng/mL were included. Patients were stratified by those who underwent initial resection and those who were recommended for radiographic surveillance. The natural history of these two groups was examined. ResultsFrom 1999 to 2014, 227 patients were identified who had a cyst fluid CEA ≥192 ng/mL (median 961, range 192–300,000 ng/mL). Immediate resection was performed on 63 patients (28%). Initial radiographic surveillance was recommended for 164 patients; 87% did not have main pancreatic duct dilation, and 87% met consensus criteria for radiographic surveillance. After a median follow-up of 56 months, 48 of the 164 patients (29%) had undergone resection. Ultimately, there were three cases (2%) of high-grade dysplasia and two cases of invasive carcinoma (1%) within these 164 patients selected for observation. Three of the five cases of either high-grade dysplasia or invasive carcinoma were among the 22 patients followed outside of consensus guidelines. ConclusionsAppropriately selected patients with mucinous pancreatic cysts can be safely followed with serial surveillance with a low risk of malignant progression.

Natural history of patients with tuberous sclerosis complex related renal angiomyolipoma

Objective: To examine temporal relationships between tuberous sclerosis complex (TSC) and renal angiomyolipoma diagnosis and outcomes, treatment, and healthcare utilization.Methods: Administrative data from the MarketScan Commercial Database was used to select TSC-related renal angiomyolipoma patients during 1 January 2000–31 March 2013. Patients were followed until the earliest of inpatient death or end of enrollment or study. Occurrence of kidney-related outcomes, kidney-related procedures, and all-cause healthcare utilization and time to occurrence were reported. Kaplan–Meier curves were used to display the unadjusted distribution of time to outcome.Results: A total of 605 patients were selected (<18 years N = 225; ≥18 years N = 380). Mean time from TSC to renal angiomyolipoma diagnosis was 25.7 months in younger and 16.9 months in older patients. Patients ≥18 years had higher rates of chronic kidney disease (CKD), hematuria, kidney failure, embolization (EMB), and partial and complete nephrectomy compared to patients <18 years (all p < .05). Mean time from TSC-related renal angiomyolipoma diagnosis to CKD, hematuria, kidney failure, EMB, first emergency room and inpatient visits was shorter in older compared to younger patients (all p < .05). Probability of developing CKD was approximately 0.8 and 0.95 within 3 years in younger and older patients, respectively.Conclusions: Patients with TSC-related renal angiomyolipoma had high rates of kidney-related outcomes and procedures. These events sometimes preceded the angiomyolipoma diagnosis. A key study limitation was that due to the small sample size, results may have been biased by outliers. Research is needed to determine whether earlier angiomyolipoma diagnosis can impact occurrence of events and reduce healthcare utilization.