MS over Time: The Sonya Slifka Longitudinal Multiple Sclerosis Study
Despite decades of scientific research, new technology, and the cumulative experience of specialists, the long-term effects of MS on employment, family relations, and personal well-being are understood in tiny patches only. Predicting the course of MS is still more art than science. And now, there are new MS drugs to watch. Only a few people the earliest clinical trial volunteers–have been taking one of the drugs now called Avonex, Betaseron, or Copaxone for longer than 10 years.
Accurate data on how MS behaves over time would be a powerful shaper of programs, research directions, and advocacy priorities and so the National MS Society is making a start. The Society has launched the Sonya Slifka Longitudinal Multiple Sclerosis Study to gather in-depth information from a representative sample of people with MS across the U.S. over an extended period of time. This is the first such study on MS ever attempted and it represents a major commitment of time and money The resulting database should dramatically expand scientific knowledge.
Richard Slifka, chairman of the National Multiple Sclerosis Society, is leading with a start-up gift of $1 million. The study is named in honor of his mother.
Behind the name
Young Sonya Slifka and part of her family immigrated to the United States from Latvia in the 1920s, part of a great wave fleeing war and revolution in Europe. Shortly before the stock market crash of 1929, she married, began a family, and was soon working with her husband to build up a small business delivering home-heating oil in Boston. In 1948, when the difficult years seemed to be in the past, she was diagnosed with MS. She’d experienced several years of unexplained trembling, muscle weakness, slurred speech, and numbness. As her MS progressed, tremors in her hands made writing almost impossible, and she developed difficulty walking.
Richard, her teenage son, was the family member most at home, and he often acted as his mother’s personal caregiver. MS did not diminish Sonya Slifka’s positive attitude toward life. She had a special grace that allowed her to accept help without shame or reluctance. And she also had an awe-inspiring stubbornness. Despite her wildly trembling hands, she continued to cook and clean house and to be, Richard Slifka remembers, “first in line if there was something new and exciting to see.” While she was never bitter about her MS, she hated it and the limitations it imposed on her life.
“If this breadth of information had been available when my mother was alive, who knows how much sooner researchers would have developed breakthrough therapies such as the current ABC drugs,” Richard Slifka said recently. In 1999, after 2 years of review and deliberation, the Society’s Research Programs Advisory Committee approved the study.
The right representatives
Abt Associates Inc. of Cambridge, Massachusetts, with lead researcher Sarah Minden, MD, was chosen to implement the study. During the 3-year start-up phase, Abt will select a broadly representative group of participants, hone the information-gathering process, and design the database.
The first step is finding participants who represent, as accurately as possible, the MS population in this country. This is more difficult than one might think. Putting out a call for volunteers would bias the data, since the people who respond select themselves. Relying on data from clinics leaves out people who don’t go to clinics. Instead, Abt will identify a random sample of physicians around the country, and with the help of Society chapter staff, recruit them to participate. The physicians, in turn, will identify, in a random fashion, people with MS, and ask them to enroll. Again, using random sampling techniques, Abt will recruit people with MS who don’t go to doctors on a regular basis and people with MS who live in nursing homes.
The goal is to have more than 1,800 persons in the database by the end of 2002. The sample will be diverse in age, sex, ethnicity, and socioeconomic status. It will include people with all forms of the disease–and range from newly diagnosed people to those who’ve had MS for many years.
The right questions
The second challenge is making sure that the most useful facts are collected. Statisticians understand all too well the “garbage in, garbage out” principle. Questions must be free of hidden bias. And they must be clear. For example, “When did you have your last exacerbation?” might seem straightforward, but even physicians can understand the term “exacerbation” to mean different things. Unless the term is defined exactly, 1,800 answers to this question could end up being scientifically useless. So, in the first phase of the study, Abt will be pilot-testing everything.
The database will also include clinical and laboratory data derived from physician reports, blood samples for genetic and immunologic studies, and MRI studies on selected patients. Data on disability status and on use and cost of health services will be collected every 3 months.
Answers over time
Recently, Dr. Minden took a moment to look ahead at the immense usefulness of all this information for the future.
“What happens to people with MS over time?” she asked. “Do married people with MS really have an unusually high divorce rate? What types of health services do people with MS use, with what outcomes, and at what cost? How does unemployment come about? This database will give us a chance to answer questions like these with fact, not speculation.”
Collecting grains of sand
Every person with MS eventually accumulates a store of information about how the disease runs its course and how it has affected day-to-day living. These stores of fact are scattered like sand all over the landscape. The ambition of the Sonya Slifka Longitudinal MS Study is to gather these grains into a meaningful overall picture. It’s a grand ambition, and will ultimately provide the MS community with precious information and insight from which specialists can create better ways to manage this devastating disease.
COPYRIGHT 2000 National Multiple Sclerosis Society
COPYRIGHT 2001 Gale Group