Defining Natural History: Assessment of the Ability of College Students to Aid in Characterizing Clinical Progression of Niemann-Pick Disease, Type C
2011

Assessing Clinical Progression of Niemann-Pick Disease by College Students

Sample size: 64 publication Evidence: moderate

Author Information

Author(s): Shin Jenny, Epperson Katrina, Yanjanin Nicole M., Albus Jennifer, Borgenheimer Laura, Bott Natalie, Brennan Erin, Castellanos Daniel, Cheng Melissa, Clark Michael, Devany Margaret, Ensslin Courtney, Farivari Nina, Fernando Shanik, Gabriel Lauren, Gallardo Rani, Castleman Moriah, Gutierrez Olimpia, Herschel Allison, Hodge Sarah, Horst Anne, Howard Mary, James Evan, Jones Lindsey, Kearns Mary, Kelly Mary, Kim Christine, Kiser Kinzie, Klazura Gregory, Knoedler Chris, Kolbus Emily, Lange Lauren, Lee Joan, Li Eileena, Lu Wei, Luttrell Andrew, Ly Emily, McKeough Katherine, McSorley Brianna, Miller Catherine, Mitchell Sean, Moon Abbey, Moser Kevin, O'Brien Shane, Olivieri Paula, Patzwahl Aaron, Pereira Marie, Pymento Craig, Ramelb Erin, Ramos Bryce, Raya Teresa, Riney Stephen, Roberts Geoff, Robertshaw Mark, Rudolf Frannie, Rund Samuel, Sansone Stephanie, Schwartz Lindsay, Shay Ryan, Siu Edwin, Spear Timothy, Tan Catherine, Truong Marisa, Uddin Mairaj, VanTrieste Jennifer, Veloz Omar, White Elizabeth, Porter Forbes D., Haldar Kasturi

Primary Institution: University of Notre Dame

Hypothesis

Can trained college students accurately assess clinical records to characterize disease progression in Niemann-Pick Disease?

Conclusion

The study shows that college students can be trained to assess patient records and provide insights into the natural history of Niemann-Pick Disease.

Supporting Evidence

  • Students were trained to recognize major and minor symptoms of Niemann-Pick Disease.
  • Assessment of clinical records showed expected characteristics of linear disease progression.
  • Two new records revealed accelerated disease progression compared to existing severity scales.
  • Students created clinical summary sheets to assist physicians treating NPC patients.
  • Training included didactic lectures and discussions with clinical practitioners.

Takeaway

College students can learn to look at medical records and help doctors understand how a disease gets worse over time.

Methodology

Students were trained to assess clinical records and quantify disease progression using a defined clinical severity scale.

Potential Biases

Variability in scoring among students could lead to inconsistencies in disease severity assessments.

Limitations

The study relied on the quality of existing medical records, which varied in detail and completeness.

Participant Demographics

Participants were 64 undergraduate students from the University of Notre Dame, with no prior experience in clinical research.

Digital Object Identifier (DOI)

10.1371/journal.pone.0023666

Want to read the original?

Access the complete publication on the publisher's website

View Original Publication