My fingers in PSP pies

If, like me, you like hearing about ideas in progress, here are the PSP-related projects at various points in my own pipeline right now:

  • I’ve written a long paper with statistician Ronald Thomas, PhD on clinical trial design issues in PSP that has been accepted for publication.  It’s a review of previously published work but includes our original calculation of a “minimal clinically important difference (MCID)” for PSP.
  • What’s an MCID?  It’s the smallest quantity of improvement that perceptibly changes a person’s quality of life.  This can be measured in multiple ways and how to do that in PSP is the topic of another project in which I’m collaborating.  Its leader is Anne-Marie Wills, MD of Massachusetts General Hospital and the Parkinson Study Group’s Atypical Parkinsonism Working Group.
  • I’m a sort of senior advisor – not really a collaborator – in a project led by Deepak Gupta, MD of the University of Vermont to create AI-assisted diagnostic software for the major Parkinsonian disorders, including PSP.  It’s based on the current, validated, published criteria, but is a lot easier to use.  It does require skill in the neurological exam, so it’s not for home use.  I hope it will soon be submitted for publication.
  • CurePSP has undertaken a project to determine the impact of PSP, CBS, and MSA on a family’s finances.  I’m working with a young staffer, Saira Mehra, on sending a detailed questionnaire to as many people with those disorders as we can.  We now have enough responses to start the statistical analysis.
  • I’m a moderately minor collaborator in a review of swallowing abnormalities in PSP, CBD and MSA.  It’s another project of the same working group and its primary author is Federico Rodriguez-Porcel, MD of the University of South Carolina.  It’s been submitted, so fingers are crossed.
  • I’m a more minor collaborator in another review, this one of imaging and fluid biomarkers in PSP, CBD and MSA.  Its primary author is Guillaume Lamotte, MD of the University of Utah.  All of the authors are affiliated with the Diagnosis and Treatment Working Group of CurePSP’s Centers of Care network.  It’s almost ready to submit for publication.
  • I’m an even more minor collaborator in an analysis of causal risk factors in PSP using medical records from 240 patients registered with the UK Biobank.  It’s been submitted and its primary author is Charlie Weige Zhao, MD of Mass General.  There were a couple of interesting findings, but I can’t discuss them until the paper is accepted (or maybe until it’s published, depending on the journal’s rules).
  • I’m advising several drug companies on how to design their PSP trials and to implement the PSP Rating Scale as the primary outcome measure. (I created the PSPRS in the mid-1990s and published it in 2007 along with statistician Pam Ohman-Strickland, PhD, of Rutgers.)

Five years ago, when I told my wife I was retiring, she actually believed me just because the paychecks stopped.

One thought on “My fingers in PSP pies

  1. Dr. Golbe:
    These are extremely interesting and important research projects. I’d be especially interested in the first on minimally clinically important differences for PSP. There is some debate as to whether a trial is designed to have high power for the MCID. Clinical trialists generally say no. (High power, they say should be reserved for differences sufficiently impressive that we would not like to miss them (e.g., Stephen Senn). But there’s ambiguity in how “clinically relevant” is used, and adding “minimally” might confuse things further. This leads to confusion in interpreting clinical results. I’m interested in your view.

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