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Psychiatric Times. Vol. 23 No. 11
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Better Tools Needed to Measure Treatment Outcome

By Kenneth J. Bender, PharmD, MA | October 1, 2006

Biomarkers in clinical research

John Kane, MD, of the Zucker Hillside Hospital, Glen Oaks, NY, and Albert Einstein College of Medicine of Yeshiva University, Bronx, NY, noted the increasing use of biomarkers as another emerging trend in clinical research but acknowledged that many have been found too inconsistent to serve as correlates of psychiatric illness. "The integration of biomarkers with valid measures of symptom severity may herald a new model for future clinical trials," Kane projected.

The renewed interest in using biomarkers in clinical studies was welcomed by Mark Opler, PhD, MPH, of Columbia University and the PANSS Institute in New York City. Opler noted that clinical studies of psychiatric disorders have been complicated "by the almost universal absence of reliable physical or biochemical pathologies."

While noting many unsuccessful efforts to establish valid biologic markers for a range of conditions--including depression, schizophrenia, and Alzheimer disease--Opler pointed to some recent successes in studies employing "hybrid" measures that combine traditional data collection approaches with tests of biochemical and systemic function.

"There is renewed interest in searching for biological markers that can be used to diagnose, subtype, or quantify disease severity," Opler indicated.

John Sweeney, PhD, of the University of Illinois at Chicago, pointed out that eye-movement studies have provided a quantitative biomarker for studying cognitive and motor systems for decades. As an example, he mentioned that eye-movement studies have tracked the ability to suppress context-inappropriate responses after antipsychotic treatment, which emerges more gradually than the reduction in psychotic symptoms.

"While studies of eye tracking in psychiatry began with interest in their use as an endophenotype for family/ genetic research, more recent work has shown that studies of eye movements hold great promise as a translational biomarker in testing the neurocognitive efficacy of treatments," Sweeney observed.

Progress in adopting technologies for clinical research, as Frank and others have advocated, was the theme of a workshop on telepsychiatry and biomarkers conducted by Kane. He observed that telepsychiatry--a form of telemedicine involving videoconferencing, telephones, secure e-mail, and other modalities--has been considered by some to be too complex and impractical to employ in research settings.

According to Kane, however, this technology is enjoying growing popularity. "Telepsychiatry is emerging as a viable alternative to in-person assessments," he noted, "with particular advantage demonstrated in obtaining standardized, reliable assessments at fewer sites and with fewer raters."

Don Hilty, MD, from the University of California, Davis, agreed that telepsychiatry--which he characterized as a component of "e-health"--is developing into a useful technology for research, in addition to its wider application in clinical care and education. Hilty noted its successful use in several research populations, its particular utility for outreach to rural areas, and its application as a novel tool in subject recruitment.

"It's well received by participants, it's affordable, and it's versatile," Hilty concluded.

Engelhardt reported using telepsychiatry for conducting psychiatric assessments in clinical trials, as well as in evaluating and training the raters. Her group has used both 2-way audio conferencing and videoconferencing for those purposes, remotely assessing patients from a centralized location in real time in multicenter studies of treatments for schizophrenia and depression.

Searching for optimal scales

The putative benefit of the use of second-generation antipsychotics to treat the negative symptoms of schizophrenia has not been supported by sufficient evidence for these agents to be approved for that indication. In the introduction to a workshop on the methodologic hurdles in proving treatment effectiveness for negative symptoms, Nina R. Schooler, PhD, of the Washington DC Veterans Affairs Medical Center, and Stephen R. Marder, MD, of the University of California, Los Angeles, attributed this partly to a lack of consensus on an effective means to measure these symptoms.

"Currently available measurement tools for negative symptoms vary widely in their face validity, psychometric properties, and user friendliness to the clinical trials investigator," they wrote in the workshop overview.

Brian W. Kirkpatrick, MD, of the Medical College of Georgia, described recent work on a new rating scale appropriate for clinical trials based on recommendations from the NIMH-sponsored Consensus Development Conference on Negative Symptoms held in January 2005. The instrument contains subscales for 5 domains that were identified at the conference: alogia, blunted affect, avolition, anhedonia, and asociality. This new scale is close to being field tested, although Kirkpatrick anticipates that it could undergo modifications through that process.

Fabien Tremeau, MD, and colleagues from the Nathan S. Kline Institute for Psychiatric Research, Orangeburg, NY, described another new scale for negative symptoms--the Motor-Affective-Social Scale (MASS). This instrument is based on the determination that negative symptoms can be measured as expressiveness during an interview or by certain social behaviors. The MASS is applied during a 5-minute structured interview with ratings of coverbal hand gestures and spontaneous/voluntary smiling, as well as answers to interview questions.

Delusions are another component of schizophrenia for which there have been few quantitative measures. Barnett S. Meyers, MD, of the Weill Medical College of Cornell University in New York City, described the development of the Delusional Assessment Scale (DAS). The DAS, which measures the intensity of beliefs across multiple delusional domains, was recently applied to determine whether delusions among older patients differ from those in young adults. Meyers reported that delusions appeared to have a greater impact in older patients and that men demonstrated greater conviction than women, regardless of age.

With the amount of interest in new scales facilitating clinical research, it was probably inevitable that a scale would be developed to rate how well the evidence from research is adopted into clinical practice. Jessica L. Garno, PhD, reported on the validation of a rating scale to assess adherence to evidence-based psychopharmacotherapy practices. Despite recent efforts to expand research based on effectiveness, Garno asserted that practitioners underutilize the findings and have insufficient guidance to extrapolate from efficacy studies to the treatments they provide.

"The rapid growth of new pharmacotherapies for mood disorders, coupled with the promulgation of numerous practice guidelines, has prompted the need for more systematic approaches to choosing longitudinal treatment strategies," Garno explained.

Garno and colleagues developed a 20-item, 20-point scale to rate fidelity to pharmacotherapeutic practices that have been commonly accepted in consensus meetings and expert reviews. The scale was found to have face validity, internal consistency, and item-total, as well as overall inter-rater reliability in validity testing with psychiatrists who referred patients to an inpatient facility for treatment of unipolar or bipolar mood disorder and comorbid substance abuse.

Garno and colleagues suggested that the application of their scale "may hold value in tracking performance improvement among clinicians or in examining relationships between evidence-based practices and patient service utilization or outcome."

This is part 1 of the 2-part coverage of the 2006 NCDEU meeting; the second report, on treatment investigations, will appear in a future issue of Psychiatric Times.

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