International MPN News, Science & Opinion

The Empowered Patient – Stem Cell Transplant

Michael G. Goldstein

Should I Consider Hematopoietic Stem Cell Transplant (hSCT)?

Since SCT has become an accepted treatment for advanced myelofibrosis (MF), this is a question that many members of our MPN community are asking. As you may know, Zhen and other members of the MPNforum have asked me to moderate a discussion on this topic for our MPNforum Facebook site. To help frame the discussion, I will share some thoughts about my understanding of the process of making tough medical treatment decisions.

Because my PV has responded well to interferon, with no signs of myelofibrosis, I haven’t had to wrestle with this daunting question. Yet, my understanding from reading the current MPN research literature is that there is a reasonable chance, probably about 1 in 5, that I will develop post-PV myelofibrosis. When and if I develop MF, I will have to consider whether to undergo a transplant.

For me, the question, “Should I consider SCT,” raises broader questions about medical decision making. The first is, “When should clinicians involve patients and caregivers in treatment decisions?”

As a physician and medical educator, I feel strongly that it is a physician’s responsibility to involve patients in treatment decisions (or involve caregivers if the patient is unable to participate in decision making) to the extent that they wish to be involved. Of course, there are exceptions to this position, such as in emergency situations when timely treatment is lifesaving. Research on people’s desire to participate in decision indicates that most people want to have some role in treatment decisions.

Participating in treatment decisions is especially important when there is not a clear best choice among treatment options or when there are significant trade-offs between treatment gains and the side effects or risks of treatment.

At the present time, because there is limited experience with SCT for MF, the decision about whether to undergo SCT is a complicated one that requires weighing of benefits and risks of SCT for each individual. This position was expressed by MPN experts Vikas Gupta, Parameswarian Hari and Ronald Hoffman in a review article published in Blood in August, 2012.

In the article Dr. Gupta and colleagues  share what is currently known about the benefits and risks of SCT for MF, which vary based on an individual’s experience with MF, response to other treatments and a number of other factors, including age, the presence of other medical conditions and the presence of biological characteristics (e.g., cytogenenetics).

Moreover, changes in the procedure itself (e.g., use of reduced intensity conditioning) and the emergence of other new therapies for MF (e.g., JAK2 inhibitors) have impacted SCT outcome and complication rates, making it even harder to be certain about benefits and risks.

Gupta and colleagues provide the following “snapshot” of the benefits of SCT for MF:

“Progression-free survival is observed in approximately 45% to 50% at 3 years in patients undergoing HCT using modern conditioning regimens.”

In addition, the authors provide the following data on the incidence of risks and complications of SCT for MF:

    Graft failure occurs in 5-25% of patients
    Severe acute graft versus host disease occurs in 12-21% undergoing reduced intensity conditioning;
    Liver toxicity is common (up to 36 – 44% in one series).

Chronic graft versus host disease (GVHD) is even more common. Authors of a July, 2010 review article in Blood reported a 53% incidence of chronic GVHD. (Note: GVHD is a complication that occurs when transplanted cells “attack” the recipient cells because of they are viewed as “foreign.” A variety of symptoms and problems result, including gastrointestinal problems, skin rashes, breathing problems and increased risk of infections.)

Information about risks and benefits of SCT, as well as alternative treatments, is changing rapidly. So, consultation with physicians who can share the latest information about the risks and benefits of SCT and other treatments for MF is extremely valuable.

A second broad question about decision making is, “How can we, as patients and caregivers, insure that the decisions being made about our treatment are the right decisions for us?” When there is uncertainty about the best choice among treatment options, the process of decision making involves more than just understanding the risks and benefits of specific treatment options.

Because of differences in how we weigh the importance of achieving a specific treatment outcome, exploration of our values, goals and preferences is key to a quality decision. For some, the most important consideration in choosing a treatment that increases our chances of becoming disease-free, while for others, it is more important to maintain current functioning (e.g., one’s ability to care for our family). Some are more comfortable accepting the risks associated with a potentially life-prolonging therapy, while others want to avoid taking risks that might impair current functioning.

The Informed Medical Decisions Foundation (IMDF) has developed resources and tools for patients, caregivers and clinicians to help them make better quality decisions. Their approach to shared decision making includes insuring that patients (and/or caregivers) and clinicians have the right facts to weigh the pros and cons of therapy AND a shared understanding of the patient’s goals, values and preferences.

One of the key strategies for getting in touch with one’s own goals, values and experiences is learning how others have weighed the pros and cons for choosing among treatment options. As a member of this MPN community, I have learned from many of you about the challenges of living with myelofibrosis. Recently, many of these stories have been about your experiences with SCT.

The editors of MPNforum and I would like to encourage you to share your stories about making a decision about undergoing SCT. We would like to hear not only from those who decided to have SCT, but also from those who decided against it. Sharing what was important in your decision and your experience with the decision will undoubtedly help those of us currently grappling with this difficult decision and also those of us who may be faced with this decision in the future.

Soon we will announce an opportunity to share your thoughts and experiences on this topic on MPNforum’s Facebook page.I hope you will join us in this discussion.

(Note:MPNforum Facebook is open to all in the MPN community. It is a private page to permit discussion among participants without general Internet sharing of posts. To join go to Facebook and type MPNforum is the search box.)

Take me back to the Contents

© Michael Goldstein and MPNforum.com, 2013. Unauthorized use and/or duplication of this material without express and written permission from this blog’s author and/or owner is strictly prohibited.Excerpts and links may be used, provided that full and clear credit is given to Michael Goldstein and MPNforum.com with appropriate and specific direction to the original content.

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out / Change )

Twitter picture

You are commenting using your Twitter account. Log Out / Change )

Facebook photo

You are commenting using your Facebook account. Log Out / Change )

Google+ photo

You are commenting using your Google+ account. Log Out / Change )

Connecting to %s

%d bloggers like this: