Promising Tool to Measure Patient Outcomes Worldwide, approximately 2 million people suffer from some form of inherited retinal degeneration (IRD). These slowly progressing diseases, caused by varying genetic alterations, can lead to permanent vision loss. The effects of retinal dystrophies are unique to each patient and type of disease. All can significantly
Until recently, treatments for IRDs were
unavailable. However, new therapeutic advances such as gene therapy are now options for IRD treatment. As noted in last year’s report, Kellogg offers the first FDA approved gene therapy, Luxterna. Clinical trials are under way at Kellogg to evaluate the efficacy of additional gene therapies. These and future regenerative medicine trials would benefit from standardized outcome
“
THIS IS A TOOL TO HELP BRIDGE
THE COMMUNICATION BETWEEN THE
PROVIDER AND THE PATIENT. IT GIVES
THEIR CONCERNS AND TO MAKE SURE
Gabrielle D. Lacy, M.S., a University of Michigan
THEIR NEEDS ARE BEING ADDRESSED.
medical student and first
— Gabrielle D. Lacy, M.S.
author of the MRDQ paper,
the benefits to patients. That’s why doctors and researchers
at the Kellogg Eye Center have developed an instrument to capture standardized patient-reported
between the provider and the patient. It
Retinal Degeneration Questionnaire (MRDQ) has been validated in a subset of the IRD population after extensive
stresses the importance of patient-centered care. “This is a
tool to help bridge the communication
outcomes (PROs) for use in IRD trials. The Michigan
gives patients an opportunity to express their concerns and to make sure their needs are being addressed.”
patient interviews, initial field testing, and data analysis.
K. Thiran Jayasundera, M.D., M.S.
Gabrielle D. Lacy, M.S.
PATIENTS AN OPPORTUNITY TO EXPRESS
measures and testing protocols to assess
“
impact a patient’s quality of life.
The current version of the MRDQ will undergo further
K. Thiran Jayasundera, M.D., M.S.,
assessment. “This process will require administration of the
of the Kellogg Eye Center and lead
questionnaire to a larger sample of participants with varying
investigator of the the National
IRDs,” says MRDQ study co-investigator David C. Musch,
Eye Institute funded MRDQ study,
Ph.D., M.P.H. These additional studies will measure on a
notes that, as new treatments are
larger scale how well the MRDQ captures changes in visual
developed, it is important to evalu-
function and allow the MRDQ to become a standardized
ate whether or not the responses
measurement tool for future IRD clinical trials.
to treatment are meaningful to the
patient. For example, a patient may
on daily tasks that will effectively capture patient experiences
have 20/20 visual acuity, but be un-
as well as bolster research efforts in this field,” says Michigan
able to walk through an unfamiliar
Medicine statistician Chris Andrews, Ph.D.
“This novel PRO measure addresses the impact of IRDs
room due to a severely constrained visual field or background illumination.
“We might see improvements in the testing of patients,
but what we don’t know is how the treatment is enhancing the
4
Other contributors to the MRDQ study include Kellogg Eye Center researcher Rebhi Abuzaitoun, M.D., and faculty
patient’s everyday life,” says Dr. Jayasundera. “The MRDQ
members Maria Fernanda Abalem, M.D. Ph.D., M.Sc.;
addresses this.”
Julie Rosenthal, M.D. M.S.; and Joshua Ehrlich, M.D., M.P.H.
