The first step — Your doctor.
The road to stem cell transplantation starts in the hematologist’s office with a patient’s personal physician. As a gatekeeper to the long and difficult events that will transform a patient’s myeloproliferative neoplasm, the physician is required to answer critical, often unasked, questions.
To guide us through this first step we turned to the MPNclinic’s newest member, the much acclaimed and deeply experienced Dr. David Steensma, Associate Professor of Medicine, Harvard Medical School, physician, Dana Farber Cancer Institute, specializing in research and patient care in the leukemias, myelodysplasic syndrome and myeloproliferative neoplasm.
He is joined by Professor Claire Harrison, a founding member of MPNclinic, Consultant haematologist and Deputy Director, Guy’s and St. Thomas’ NHS. This introduction will be added to the full transplant specialist roundtable discussion in the MPN Quarterly Journal, June 20, 2014.
Will you continue to follow me post-transplantation?
Steensma: This varies by institution. At some institutions the physician caring for the patient for myelofibrosis is the same as the physician who will take the patient through transplant. At other centers (including mine), the transplant team is separate so there is a transfer of care as transplant approaches. If problems arise post-transplant that are related to the MPN rather than a transplant complication like GVH, failed engraftment, or infection, then the MPN physician usually becomes involved again.
Harrison: Usually the doctor who follows a patient after transplant is the transplant doctor since at this point the patient will not have the original disease, e.g., myelofibrosis. Sometimes, however, they are one and the same person.
How can I improve my chances of a successful SCT?
Steensma: Following the recommendations of the treating team can be helpful (these protocols about diet, permitted and prohibited activities, and logistics such as follow-up schedule differ from center to center), but to a large extent success or failure of SCT is outside of the control of the patient. This loss of control and uncertainty can be quite difficult.
Harrison: Keep as fit as possible and have a positive mental attitude.
On what factors would my estimated survival outcome be based?
Steensma: There are two types of factors that are the major drivers determining patient outcome with SCT: 1) patient factors such as age and the presence or absence of other medical problems (“comorbid conditions”), and 2) disease factors. Patients who are younger, lack other medical problems besides the MPN, and are able to be active (“good performance score”) do best with stem cell transplant. Those who have high WBC, high circulating blasts, high risk chromosome profile, and other negative disease factors tend to do more poorly.
Harrison: Many, and this is complex. Your general fitness will be assessed, for example your lungs, heart and kidneys but also any other illnesses such as diabetes for example. What stage your disease is at, how aggressive it is, how old you are (we can never escape this one!), how well matched your donor is, whether your donor is male or female, whether you or your donor have had a common virus called CMV (cytomegalovirus), etc.
When is it too late to consider transplantation?
Steensma: Usually the factors that prevent transplant arise if the patient is too old or too sick or the disease is too poorly controlled (e.g., progression to leukemia and refractory to treatments attempted to try to reduce the leukemic blast cells).
How do I choose the right transplant team or institution?
Steensma: Many factors are involved. The patient needs to have a comfort level with the treating team and institution. In general outcomes are better at larger transplant centers, but geography needs to be taken into account and sometimes insurance (in the US) restricts where patients can have a transplant.
Harrison: A few factors are important: Choose a site that is close to home as you will be travelling to and from this centre for up to a year and your family needs to be able to visit you! Choose a centre where you feel comfortable with the environment and the team.
What are some of the emotional, social issues post-SCT I should expect?
Steensma: Isolation due to risk of infection, uncertainty about whether the MPN will relapse, feeling unwell and loss of control can take a heavy psychological toll on patients. Having supportive family, friends, and treatment team can help but it is a long and difficult road.
Am I likely to require long-term drug therapy post-SCT?
Steensma: Yes. Almost all patients require extended treatment with immunosuppressive medications to prevent GVH and prevent infection. Whether other drugs such as ruxolitinib have a role post-transplant is currently being studied.
How do you measure outcome, “success’ or cure?
Steensma: Periodic monitoring with physical exam, blood tests and bone marrow biopsies allow serial assessment of response to transplant and monitoring for complications or relapse. Long-term survival with full donor cell engraftment/chimerism and freedom from GVH enough to maintain a good quality of life is the measure of success. “Cure” means living a long time and then dying of some unrelated cause with no evidence of MPN. Remission is necessary for cure, but not the same as cure.
Harrison: There are multiple factors. Primarily: the patient being alive, being free of disease, being free of complications, in particular GVHD (graft versus host disease).
Is there anything a patient can do to help avoid serious GVHD?
Steensma: Adherence to the GVHD medications and monitoring program is about the only thing that can be done.
Harrison: Other than complying with treatments to prevent GVHD, not as far as I know.
How much of a factor is age?
Steensma: Age is a large factor in outcomes in transplant; in general, older patients do poorly, both due to decreased tolerance of treatment and the presence of comorbid conditions (e.g., kidney dysfunction, lung or heart impairment). At my institution the upper age limit is 75 years old but whether an individual patient is eligible for transplant is not just a factor of chronological age, it is also a factor of ‘biological age’. Some 50 year olds are too unhealthy to proceed to transplant whereas I have sent a number of 65-75 year olds to transplant with good outcomes.
What restrictions will an SCT place on my life
Steensma: For the first year after transplant the patient’s immune system is not fully recovered and numerous precautions about infection need to be followed. This is often very difficult for patients since they spend much of the time at home or shuttling between doctors’ appointments and can easily get ‘cabin fever.’ In the short term after transplant quality of life is usually quite poor; the tradeoff is the hoped-for improvement later on.
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