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The Social Life of Data

Sun, 04/23/2017 - 10:04pm


So, besides this amusing parallel between data and children, what have we learned? We are reminded to get identifiers for our data, encourage our data to socialize with other data, and plan in advance for the long-term archival of our data. If we can do this in a well-controlled environment, the benefits, individually and as a community, in terms credit, value and reproducibility, are substantial.

In short, if you love your data, set it free, and watch what happens! Its social life may surprise you.

Integrating Permanent Supportive Housing and Co-Occurring Disorders Treatment for Individuals Who Are Homeless

Sun, 04/23/2017 - 10:04pm

OBJECTIVES: While permanent housing, addictions, and mental health treatment are often critical needs to achieve housing stability and community reintegration, few studies have systematically integrated them into a single comprehensive approach for people experiencing chronic homelessness. This pilot study examined the feasibility and preliminary outcomes of systematically integrating permanent supportive housing and an evidence-based co-occurring disorders intervention called Maintaining Independence and Sobriety Through Systems Integration, Outreach, and Networking (MISSION).

METHODS: This single-group open pilot enrolled 107 people with co-occurring disorders experiencing chronic homelessness from two Massachusetts inner-city and rural areas. Enrolled subjects were interested in receiving permanent supportive housing along with 1 year of MISSION services. Data were collected through baseline and 6- and 12-month follow-up assessments.

RESULTS: Participants (Mage = 49.52 years, SD = 10.61) were mostly male (76.6%), Caucasian (52.3%), and unemployed (86.0%), with an average of 8.34 years (SD = 8.01) of homelessness. Self-reported lifetime problems with anxiety (75.7%) and depression (76.6%) were common, as was use of alcohol (30.8%), cannabis (31.8%), and cocaine (15.9%). Almost all participants (95.3%) were placed into permanent housing, which took on average 42.6 days from enrollment (SD = 50.09). Among those placed, nearly 80% of the clients were able to retain housing through the end of the study. Overall retention was high, with 86.0% remaining in MISSION treatment until the end of the study. While there were no significant changes in rehospitalization, service utilization, or substance use, there were modest significant mental health symptom improvements from baseline to program completion.

CONCLUSIONS: This pilot study suggests that co-occurring disorder interventions like MISSION are feasible to integrate with permanent supportive housing despite the somewhat differing philosophies, and preliminary data suggested substantial improvements in housing and modest improvements in mental health symptoms. While caution is warranted given the lack of a comparison group, these findings are consistent with other rigorous studies using MISSION among homeless individuals who did not receive permanent supportive housing.

Whole-genome sequencing identifies a novel ABCB7 gene mutation for X-linked congenital cerebellar ataxia in a large family of Mongolian ancestry

Sun, 04/23/2017 - 10:04pm

X-linked congenital cerebellar ataxia is a heterogeneous nonprogressive neurodevelopmental disorder with onset in early childhood. We searched for a genetic cause of this condition, previously reported in a Buryat pedigree of Mongolian ancestry from southeastern Russia. Using whole-genome sequencing on Illumina HiSeq 2000 platform, we found a missense mutation in the ABCB7 (ABC-binding cassette transporter B7) gene, encoding a mitochondrial transporter, involved in heme synthesis and previously associated with sideroblastic anemia and ataxia. The mutation resulting in a substitution of a highly conserved glycine to serine in position 682 is apparently a major causative factor of the cerebellar hypoplasia/atrophy found in affected individuals of a Buryat family who had no evidence of sideroblastic anemia. Moreover, in these affected men we also found the genetic defects in two other genes closely linked to ABCB7 on chromosome X: a deletion of a genomic region harboring the second exon of copper-transporter gene (ATP7A) and a complete deletion of PGAM4 (phosphoglycerate mutase family member 4) retrogene located in the intronic region of the ATP7A gene. Despite the deletion, eliminating the first of six metal-binding domains in ATP7A, no signs for Menkes disease or occipital horn syndrome associated with ATP7A mutations were found in male carriers. The role of the PGAM4 gene has been previously implicated in human reproduction, but our data indicate that its complete loss does not disrupt male fertility. Our finding links cerebellar pathology to the genetic defect in ABCB7 and ATP7A structural variant inherited as X-linked trait, and further reveals the genetic heterogeneity of X-linked cerebellar disorders.

Telephone Smoking-Cessation Counseling for Smokers in Mental Health Clinics: A Patient-Randomized Controlled Trial

Sun, 04/23/2017 - 10:04pm

INTRODUCTION: People with a mental health diagnosis have high rates of tobacco use and encounter limited availability of tobacco treatment targeted to their needs. This study compared the effectiveness of a specialized telephone smoking-cessation intervention developed for mental health patients with standard state quit-line counseling. DESIGN: RCT. SETTING/PARTICIPANTS: The study was conducted at six Veterans Health Administration facilities in the Northeast U.S. Participants were 577 mental health clinic patients referred by their providers for smoking-cessation treatment. INTERVENTION: From 2010 to 2012, the study implemented a telephone program that included patient referral from a mental health provider, mailed cessation medications, and telephone counseling. Participants were randomized to receive a specialized multisession telephone counseling protocol (n=270) or transfer to their state's quit-line for counseling (n=307). MAIN OUTCOME MEASURES: Participants completed telephone surveys at baseline, 2 months, and 6 months. The study's primary outcome was self-reported 30-day abstinence at 6 months. Secondary outcomes were self-reported 30-day abstinence, counseling satisfaction and counseling content at 2 months, and self-reported use of cessation treatment and quit attempts at 6 months. Logistic regression was used to compare treatment groups on outcomes, controlling for baseline cigarettes per day and site. Inverse probability weighting and multiple imputation were used to handle missing abstinence outcomes. Data were analyzed in 2014-2015. RESULTS: At 6 months, participants in the specialized counseling arm were more likely to report 30-day abstinence (26% vs 18%, OR=1.62, 95% CI=1.24, 2.11). There was no significant group difference in abstinence at 2 months (18% vs 14%, OR=1.31, 95% CI=0.49, 3.49). Participants in the specialized arm were more likely to be assisted with developing a quit plan; receive follow-up calls after quitting; and receive counseling on several domains, including motivation, confidence, smoking triggers, coping with urges, and mental health symptoms (all p<0.05). Specialized counseling participants were more satisfied with treatment and more likely to find the counseling useful (p<0.05). CONCLUSIONS: The specialized counseling intervention was more effective at helping patients quit than transfer to a state quit-line. Patients were more satisfied with the specialized counseling program. TRIAL REGISTRATION: This study is registered at NCT00724308.

Women's Behavioral Responses to the Threat of a Hypothetical Date Rape Stimulus: A Qualitative Analysis

Sun, 04/23/2017 - 10:03pm

One in four college women experience sexual assault on campus; yet, campuses rarely provide the in-depth self-defense programs needed to reduce sexual assault risk. Further, little is known about the range of possible behaviors elicited by sexual assault threat stimuli besides assertion. To fill this gap, the aim of the current study was to explore qualitative themes in women's intended behavioral responses to a hypothetical sexual assault threat, date rape, by using a laboratory-controlled threat. College women (N = 139) were randomly assigned to one of four different levels of sexual assault threat presented via an audio-recorded vignette. Participants articulated how they would hypothetically respond to the experimentally assigned threat. Responses were blinded and analyzed using Consensual Qualitative Research methodology. Six major themes emerged: assertion, compliance/acceptance, conditional decision making, avoidance, expressions of discomfort, and allusion to future contact. Although almost all participants described assertion, a number of non-assertive responses were described that are not currently recognized in the literature. These non-assertive responses, including compliance/acceptance, conditional decision making, and avoidance, may represent unique behavioral response styles and likely reflect the complex psychological process of behavioral response to threat. The variety of themes found illustrates the great range of behavioral responses to threat. This broad range is not currently well represented or measured in the literature and better understanding of these responses can inform future interventions, advocacy efforts, and policies focused on sexual assault.

Trajectories of drinking urges and the initiation of abstinence during cognitive-behavioral alcohol treatment

Sun, 04/23/2017 - 10:03pm

BACKGROUND AND AIMS: Drinking urges during treatment for alcohol use disorders (AUDs) are common, can cause distress and predict relapse. Clients may have little awareness of how their drinking urges might be expected to change during AUD treatment in general and in response to initiating abstinence. The aim of the present study was to test whether drinking urges change on a daily level during treatment and after initiating abstinence.

DESIGN: Secondary data analysis was performed using daily drinking urge ratings from two randomized clinical trials.

SETTING AND PARTICIPANTS: Women (n = 98) and men (n = 79) with AUDs in separate clinical trials of out-patient AUD-focused cognitive-behavioral therapy.

MEASUREMENTS: Daily dichotomous indicators of any drinking urges or acute escalations in urges (i.e. at least two more urges compared with the previous day) were examined using generalized linear mixed growth-curve modeling.

FINDINGS: Participants who initiated abstinence reported reductions in urges immediately thereafter (log odds ratios: women B = -0.701, P < 0.001; men B = -0.628, P = 0.018), followed by additional, gradual reductions over time (women B = -0.118, P < 0.001; men B = -0.141, P < 0.001). Participants who entered treatment abstaining from alcohol also reported significant reductions in urges over time (women B = -0.147, P < 0.001; men B = -0.142, P < 0.001). Participants who drank throughout treatment had smaller (women B = -0.042, P = 0.012) or no reductions in urges (men B = 0.015, P = 0.545). There was no evidence that urges increased systematically in response to initiating abstinence.

CONCLUSIONS: Drinking urges during out-patient behavioral treatment for alcohol use disorders may be maintained in part by alcohol consumption. Initiating abstinence is associated with reductions in drinking urges immediately and then more gradually over time.

rAAV Gene Therapy in a Canavan's Disease Mouse Model Reveals Immune Impairments and an Extended Pathology Beyond the Central Nervous System

Sun, 04/23/2017 - 10:03pm

Aspartoacylase (AspA) gene mutations cause the pediatric lethal neurodegenerative Canavan disease (CD). There is emerging promise of successful gene therapy for CD using recombinant adeno-associated viruses (rAAVs). Here, we report an intracerebroventricularly delivered AspA gene therapy regime using three serotypes of rAAVs at a 20-fold reduced dose than previously described in AspA(-/-) mice, a bona-fide mouse model of CD. Interestingly, central nervous system (CNS)-restricted therapy prolonged survival over systemic therapy in CD mice but failed to sustain motor functions seen in systemically treated mice. Importantly, we reveal through histological and functional examination of untreated CD mice that AspA deficiency in peripheral tissues causes morphological and functional abnormalities in this heretofore CNS-defined disorder. We demonstrate for the first time that AspA deficiency, possibly through excessive N-acetyl aspartic acid accumulation, elicits both a peripheral and CNS immune response in CD mice. Our data establish a role for peripheral tissues in CD pathology and serve to aid the development of more efficacious and sustained gene therapy for this disease.

Does psychosis increase the risk of suicide in patients with major depression? A systematic review

Sun, 04/23/2017 - 10:03pm

OBJECTIVE: Over the years studies have shown conflicting results about the risk of suicide in psychotic depression (MD-psych). To understand this association, we undertook a comprehensive review of the literature to ascertain whether individuals with MD-psych have higher rates of completed suicides, suicide attempts or suicidal ideation compared to those with non-psychotic depression (MD-nonpsych).

METHODS: We searched Pubmed, PsycINFO and Ovid in English language, from 1946-October 2015. Studies were included if suicidal ideation, attempts or completed suicides were assessed.

RESULTS: During the acute episode of depression, patients with MD-psych have higher rates of suicide, suicide attempts, and suicidal ideation than patients with MD-nonpsych, especially when the patient is hospitalized on an inpatient psychiatric unit. Studies done after the acute episode has resolved are less likely to show this difference, likely due to patients having received treatment.

LIMITATIONS: Diagnostic interviews were not conducted in all studies. Many studies did not report whether psychotic symptoms in MD-psych patients were mood-congruent or mood-incongruent; hence it is unclear whether the type of delusion increases suicide risk. Studies did not describe whether MD-psych patients experienced command hallucinations encouraging them to engage in suicidal behavior. Only 24 studies met inclusion criteria; several of them had small sample size and a quality score of zero, hence impacting validity.

CONCLUSIONS: This review indicates that the seemingly conflicting data in suicide risk between MD-psych and MD-nonpsych in previous studies appears to be related to whether one looks at differences during the acute episode or over the long-term.

Habenula cholinergic neurons regulate anxiety during nicotine withdrawal via nicotinic acetylcholine receptors

Sun, 04/23/2017 - 10:03pm

Cholinergic neurons in the medial habenula (MHb) modulate anxiety during nicotine withdrawal although the molecular neuroadaptation(s) within the MHb that induce affective behaviors during nicotine cessation is largely unknown. MHb cholinergic neurons are unique in that they robustly express neuronal nicotinic acetylcholine receptors (nAChRs), although their behavioral role as autoreceptors in these neurons has not been described. To test the hypothesis that nAChR signaling in MHb cholinergic neurons could modulate anxiety, we expressed novel "gain of function" nAChR subunits selectively in MHb cholinergic neurons of adult mice. Mice expressing these mutant nAChRs exhibited increased anxiety-like behavior that was alleviated by blockade with a nAChR antagonist. To test the hypothesis that anxiety induced by nicotine withdrawal may be mediated by increased MHb nicotinic receptor signaling, we infused nAChR subtype selective antagonists into the MHb of nicotine naive and withdrawn mice. While antagonists had little effect on nicotine naive mice, blocking alpha4beta2 or alpha6beta2, but not alpha3beta4 nAChRs in the MHb alleviated anxiety in mice undergoing nicotine withdrawal. Consistent with behavioral results, there was increased functional expression of nAChRs containing the alpha6 subunit in MHb neurons that also expressed the alpha4 subunit. Together, these data indicate that MHb cholinergic neurons regulate nicotine withdrawal-induced anxiety via increased signaling through nicotinic receptors containing the alpha6 subunit and point toward nAChRs in MHb cholinergic neurons as molecular targets for smoking cessation therapeutics.

Impairments of Social Motor Synchrony Evident in Autism Spectrum Disorder

Sun, 04/23/2017 - 10:03pm

Social interactions typically involve movements of the body that become synchronized over time and both intentional and spontaneous interactional synchrony have been found to be an essential part of successful human interaction. However, our understanding of the importance of temporal dimensions of social motor synchrony in social dysfunction is limited. Here, we used a pendulum coordination paradigm to assess dynamic, process-oriented measures of social motor synchrony in adolescents with and without autism spectrum disorder (ASD). Our data indicate that adolescents with ASD demonstrate less synchronization in both spontaneous and intentional interpersonal coordination. Coupled oscillator modeling suggests that ASD participants assembled a synchronization dynamic with a weaker coupling strength, which corresponds to a lower sensitivity and decreased attention to the movements of the other person, but do not demonstrate evidence of a delay in information transmission. The implication of these findings for isolating an ASD-specific social synchronization deficit that could serve as an objective, bio-behavioral marker is discussed.

A Systematic Review of Evidence for the Clubhouse Model of Psychosocial Rehabilitation

Sun, 04/23/2017 - 10:03pm

The Clubhouse Model has been in existence for over sixty-five years; however, a review that synthesizes the literature on the model is needed. The current study makes use of the existing research to conduct a systematic review of articles providing a comprehensive understanding of what is known about the Clubhouse Model, to identify the best evidence available, as well as areas that would benefit from further study. Findings are summarized and evidence is classified by outcome domains. Fifty-two articles met the selection criteria of Randomized Clinical Trials (RCT's), quasi-experimental studies, or observational studies for domains of employment (N = 29); quality of life/satisfaction (N = 10); reductions in psychiatric hospitalization(s) (N = 10); social relationships (N = 10); education (N = 3); and health promotion activities (N = 2). RCT results support the efficacy of the Clubhouse Model in promoting employment, reducing hospitalization(s), and improving quality of life. Quasi-experimental and observational studies offer support in education and social domains. The findings from this review indicate that Clubhouses are a promising practice but additional studies using rigorous methods that report the strength of the outcomes are needed to evaluate Clubhouse programs with fidelity to the Clubhouse Model.

Clusters of alpha satellite on human chromosome 21 are dispersed far onto the short arm and lack ancient layers

Sun, 04/23/2017 - 10:03pm

Human alpha satellite (AS) sequence domains that currently function as centromeres are typically flanked by layers of evolutionarily older AS that presumably represent the remnants of earlier primate centromeres. Studies on several human chromosomes reveal that these older AS arrays are arranged in an age gradient, with the oldest arrays farthest from the functional centromere and arrays progressively closer to the centromere being progressively younger. The organization of AS on human chromosome 21 (HC21) has not been well-characterized. We have used newly available HC21 sequence data and an HC21p YAC map to determine the size, organization, and location of the AS arrays, and compared them to AS arrays found on other chromosomes. We find that the majority of the HC21 AS sequences are present on the p-arm of the chromosome and are organized into at least five distinct isolated clusters which are distributed over a larger distance from the functional centromere than that typically seen for AS on other chromosomes. Using both phylogenetic and L1 element age estimations, we found that all of the HC21 AS clusters outside the functional centromere are of a similar relatively recent evolutionary origin. HC21 contains none of the ancient AS layers associated with early primate evolution which is present on other chromosomes, possibly due to the fact that the p-arm of HC21 and the other acrocentric chromosomes underwent substantial reorganization about 20 million years ago.

Keeping Clubhouses Open: Toward a Roadmap for Sustainability

Sun, 04/23/2017 - 10:03pm

Clubhouses are recovery centers that help persons with serious mental illness obtain and maintain community-based employment, education, housing, social integration, and other services. Key informants from U.S. clubhouses were interviewed to create a conceptual framework for clubhouse sustainability. Survival analyses tested this model for 261 clubhouses. Clubhouses stayed open significantly longer if they had received full accreditation, had more administrative autonomy, and received funding from multiple rather than sole sources. Cox regression analyses showed that freestanding clubhouses which were accredited endured the longest. Budget size, clubhouse size, and access to managed care did not contribute significantly to sustainability.

Awake whole-brain functional connectivity alterations in the adolescent spontaneously hypertensive rat feature visual streams and striatal networks

Sun, 04/23/2017 - 10:02pm

Brain mechanisms underpinning attention deficit/hyperactivity disorder (ADHD) are incompletely understood. The adolescent spontaneously hypertensive rat (SHR) is a widely studied preclinical model that expresses several of the key behavioral features associated with ADHD. Yet, little is known about large-scale functional connectivity patterns in the SHR, and their potential similarity to those of humans with ADHD. Using an approach comparable to human studies, magnetic resonance imaging in the awake animal was performed to identify whole-brain intrinsic neural connectivity patterns. An independent components analysis of resting-state functional connectivity demonstrated many common components between the SHR and both Wistar Kyoto and Sprague-Dawley control strains, but there was a divergence in other networks. In the SHR, three functional networks involving the striatum had only weak correlations with networks in the two control strains. Conversely, networks involving the visual cortex that was present in both control strains had only weak correlations with networks in the SHR. The implication is that the patterns of brain activity differ between the SHR and the other strains, suggesting that brain connectivity patterns in this animal model of ADHD may provide insights into the neural basis of ADHD. Brain connectivity patterns might also serve to identify brain circuits that could be targeted for the manipulation and evaluation of potential therapeutic options.

Development and Validation of a Computerized-Adaptive Test for PTSD (P-CAT)

Sun, 04/23/2017 - 10:02pm

OBJECTIVE: The primary purpose was to develop, field test, and validate a computerized-adaptive test (CAT) for posttraumatic stress disorder (PTSD) to enhance PTSD assessment and decrease the burden of symptom monitoring.

METHODS: Data sources included self-report and interviewer-administered diagnostic interviews. The sample included 1,288 veterans. In phase 1, 89 items from a previously developed PTSD item pool were administered to a national sample of 1,085 veterans. A multidimensional graded-response item response theory model was used to calibrate items for incorporation into a CAT for PTSD (P-CAT). In phase 2, in a separate sample of 203 veterans, the P-CAT was validated against three other self-report measures (PTSD Checklist, Civilian Version; Mississippi Scale for Combat-Related PTSD; and Primary Care PTSD Screen) and the PTSD module of the Structured Clinical Interview for DSM-IV.

RESULTS: A bifactor model with one general PTSD factor and four subfactors consistent with DSM-5 (reexperiencing, avoidance, negative mood-cognitions, and arousal), yielded good fit. The P-CAT discriminated veterans with PTSD from those with other mental health conditions and those with no mental health conditions (Cohen's d effect sizes > .90). The P-CAT also discriminated those with and without a PTSD diagnosis and those who screened positive versus negative for PTSD. Concurrent validity was supported by high correlations (r=.85-.89) with the validation measures.

CONCLUSIONS: The P-CAT appears to be a promising tool for efficient and accurate assessment of PTSD symptomatology. Further testing is needed to evaluate its responsiveness to change. With increasing availability of computers and other technologies, CAT may be a viable and efficient assessment method.

Developing a multi-institutional PACS archive and designing processes to manage the shift from a film to a digital-based archive

Fri, 04/21/2017 - 2:43pm

The Quality Assurance Review Center (QARC) works to improve the standards of care in treating cancer by improving the quality of clinical trials medicine. QARC operates as a data management and review center providing quality assurance services for multiple external groups including cooperative groups and pharmaceutical companies. As the medical world migrates from analog film to digital files, QARC has developed an innovative and unique digital imaging management system to accommodate this trend. As QARC acquires electronic data from institutions across six continents, the system is continually developed to accommodate Digital Imaging and Communications in Medicine (DICOM) imaging originating from a wide variety of Picture Archival and Communications System (PACS) manufacturers, thus creating one of the largest and most diverse multi-institutional imaging archives in the cancer research community.

Treatment outcomes of different prognostic groups of patients on cancer and leukemia group B trial 39801: induction chemotherapy followed by chemoradiotherapy compared with chemoradiotherapy alone for unresectable stage III non-small cell lung cancer

Fri, 04/21/2017 - 2:43pm

BACKGROUND: In Cancer and Leukemia Group B 39801, we evaluated whether induction chemotherapy before concurrent chemoradiotherapy would result in improved survival and demonstrated no significant benefit from the addition of induction chemotherapy. The primary objective of this analysis was to dichotomize patients into prognostic groups using factors predictive of survival and to investigate whether induction chemotherapy was beneficial in either prognostic group.

PATIENTS AND METHODS: A Cox proportional hazard model was used to assess the impact on survival of the following factors: (>or=70 versus <70 years), gender, race, stage (IIIB versus IIIA), hemoglobin (hgb) (<13 versus >or=13 g/dl), performance status (PS) (1 versus 0), weight loss (>or=5% versus <5%), treatment arm, and the interaction between weight loss and hgb.

RESULTS: Factors predictive of decreased survival were weight loss >or=5%, age >or=70 years, PS of 1, and hgb <13 g/dl (p < 0.05). Patients were classified as having >or=2 poor prognostic factors (n = 165) or or=2 versus patients with or=2 factors (HR = 0.86, 95% CI, 0.63-1.17; p = 0.34) or

CONCLUSIONS: There is no evidence that induction chemotherapy is beneficial in either prognostic group.

Improving cancer outcomes through international collaboration in academic cancer treatment trials

Fri, 04/21/2017 - 2:43pm

PURPOSE: The need for international collaboration in cancer clinical trials has grown stronger as we have made progress both in cancer treatment and screening. We sought to identify those efforts already underway which facilitate such collaboration, as well as barriers to greater collaboration.

METHODS: We reviewed the collective experiences of many cooperative groups, governmental organizations, nongovernmental organizations, and academic investigators in their work to build international collaboration in cancer clinical trials across multiple disease sites.

RESULTS: More than a decade of work has led to effective global harmonization for many of the elements critical to cancer clinical trials. Many barriers remain, but effective international collaboration in academic cancer treatment trials should become the norm, rather than the exception.

CONCLUSION: Our ability to strengthen international collaborations will result in maximization of our resources and patients, permitting us to change practice by establishing more effective therapeutic strategies. Regulatory, logistical, and financial hurdles, however, often hamper the conduct of joint trials. We must work together as a global community to overcome these barriers so that we may continue to improve cancer treatment for patients around the world.

Treatment results for patients with localized, completely resected (Group I) alveolar rhabdomyosarcoma on Intergroup Rhabdomyosarcoma Study Group (IRSG) protocols III and IV, 1984-1997: a report from the Children's Oncology Group

Fri, 04/21/2017 - 2:43pm

PURPOSE: To assess local control, event-free survival (EFS), and overall survival (OS) rates in 71 patients with localized, completely resected (Group I) alveolar rhabdomyosarcoma (ALV RMS) and their relation to radiation therapy (RT) on IRSG Protocols III and IV, 1984-1997.

METHODS: Chart review and standard statistical procedures.

PATIENTS AND TUMORS: Patients were 1-18 years at diagnosis (median, 6 years). Primary tumor sites were extremity/trunk (N = 54), head/neck (N = 9), genitourinary tract (N = 7), and perineum (N = 1). Thirty patients received VA +/- C with RT; 41 received VA +/- C alone. RT was assigned, not randomized.

RESULTS: Fifty-four patients had Stage 1 (favorable site, any size) or Stage 2 (unfavorable site, < or = 5 cm) tumors. Eight-year EFS was 90%, with 100% local control for 17 patients given RT. Eight-year EFS was 88%, with 92% local control for 37 patients without RT; P = 0.52 for EFS comparisons, 0.3 for local control comparisons. In 17 Stage 3 patients (unfavorable site, tumors > 5 cm, N0), 8-year EFS was 84% with 100% local control in 13 patients given RT; 8-year EFS was only 25% and local control 50% in 4 patients without RT. Local recurrence was the most common site of first failure in non-irradiated patients.

CONCLUSION: Patients with Stage 1-2 ALV RMS had slightly but statistically insignificantly improved local control, EFS, and OS rates when local RT was given. The need for local RT in Stage 1-2 patients deserves evaluation in a randomized study. Local control, EFS, and OS rates were significantly improved in Stage 3 patients receiving local RT.