Many multiple sclerosis (MS) patients consider autologous hematopoietic stem cell transplant (aHSCT) potentially effective in treating their disease, but most report needing more — and more reliable — information to make a reasoned decision regarding its benefits and risks, a survey found.
Those who are dissatisfied with their current therapies, have shorter disease duration, or greater disability were also the most likely to consider aHSCT as an immediate or future treatment.
The study “Patients’ expectations of autologous hematopoietic stem cell transplantation as a treatment for MS” was published in the journal Multiple Sclerosis and Related Disorders.
For an aHSCT, a patient’s own (i.e., autologous) healthy hematopoietic (blood cell-producing) stem cells are collected from bone marrow. Then the person undergoes a partial or complete ablation of their immune system, using a combination of chemotherapy and other approaches. After immunoablation, the collected stem cells are re-infused to reconstitute the immune system.
This strategy is intended to build a new immune system, one that does not attack myelin, the protective layer surrounding nerve fibers.
aHSCT use has shown promising results, but it has not been directly compared to other treatments in controlled and randomized clinical trials in MS patients. As a result, what’s known about this experimental therapy is not sufficient for clinicians to recommend it as a treatment option for MS, despite an apparent growing interest.
“In daily practice, neurologists, including the authors, not only notice an increasing number of questions regarding aHSC, but also unrealistic expectations in many patients,” the researchers wrote.
A research team in the Netherlands conducted a survey to objectively evaluate patients’ expectations regarding aHSCT, and to better understand the sources of information they rely on.
The survey consisted of 44 questions focused on disease history, knowledge about aHSCT, expectations of aHSCT, information sources, and the role patients assign to their neurologists.
In total, 137 MS patients (median age, 41) being treated at Amsterdam University Medical Center MS clinic completed the questionnaire. The majority (92%) were diagnosed with MS, and 5% with clinically isolated syndrome or MS on a second opinion. Information was lacking on the remaining 3%.
Sixty-six percent were using a disease-modifying therapy (DMT).
Results showed that a majority of these patients (83%) had heard about aHSCT, but only 25% believed they had sufficient information about the therapy. None of the patients classified their knowledge of aHSCT as excellent.
About 79% expected that aHSCT would stop disability progression, and 46% expected this therapy would lessen disability. Half of these people, 50%, think aHSCT would be a more effective treatment than current DMTs like Lemtrada (alemtuzumab) or Tysabri (natalizumab).
However, 52% of the respondents also thought aHSCT could carry more severe side effects than a DMT. A third reported that too little is known about aHSCT side effects, and less than half (45%) were able to identify at least one possible side effect of aHSCT — namely, increased sensitivity to infections and a risk of death.
Asked if they would like to undergo aHSCT now, 19% responded positively, while 54% said they would consider such a transplant as a possible future treatment.
Patients who felt their current therapy regimen was not of much help, were diagnosed within 10 or fewer years, or had greater disability (an Expanded Disability Status Scale, EDSS, score higher than 3.5) were more likely to favor aHSC for current or future use, the survey showed. Overall, 18% of patients said they were dissatisfied with their current treatment.
The internet and television were the most common sources of information on aHSCT, but only 15% consider information available online to be reliable. More than 80% named their neurologist as the information source they most trusted, followed by an experienced expert (58%), and scientific literature (49%).
Overall, these responses indicate that MS patients are considerably positive in their assumptions about aHSCT, but urgently need access to reliable information so they might make an informed decision, the study noted.
“We have found that expectations of aHSCT in MS patients are high, even though many patients indicate not to have sufficient knowledge about aHSCT,” the researchers wrote.
“We think that neurologists would do well to pro-actively inform their patients about the potential benefits and risks of aHSCT, as compared to currently approved treatments, to prevent injudicious decisions in MS patients,” the team concluded. “In our opinion, it would not be prudent to leave the initiative to others, including those with commercial interests.”