Ronald L. Paquette, MD, discusses the choice of using bone marrow and stem cell transplants as treatment for patients with myelodysplastic syndrome.
Ronald L. Paquette, MD, clinical director of the Stem Cell and Bone Marrow Transplant Program at Cedars-Sinai Medical Center and Samuel Oschin Cancer Center, discusses the choice of using bone marrow and stem cell transplants as treatment for patients with myelodysplastic syndrome (MDS).
Stem cell transplant was primarily considered as an option for patients with higher-risk MDS due to the associated toxicities, while lower-risk patients would receive a hypomethylating agent, which is the standard of care. However, the risks associated with transplant have been reduced due to use of prophylaxis for chronic graft-vs-host disease, according to Paquette.
Even for older patients, 1-year survival rates with transplant are at approximately 90%, and transplant has advantages compared with the standard of care since it offers a chance of curing MDS. Patients who do not receive transplant must receive long-term therapy until it no longer offers benefit.
There are increased morbidities and risk of mortality with transplant recipients that could be avoided with hypomethylating agent therapy or chemotherapy, which is a concern in older patients, according to Paquette. In addition, it takes longer for older patients to recover from the transplant procedure than younger patients. More research into the effects of transplant on older patients could improve the availability of curative therapy to these patients.
0:08 | There have been retrospective analyses in the past that suggested that transplant was suitable for patients only with higher-risk MDS. What's changed over time is that the risks associated with transplant have been progressively reduced. With 1-year survivors, even in older patients, [there were survival rates of] approximately 90%; that looks very attractive when you consider that the condition that you're treating is not curable. And since we only tend to transplant patients with high-risk disease, it looks like a very attractive option in comparison to receiving lifelong therapy until there's no longer a therapy available that offers any chance for continued benefit.
What’s the downside? The downside is that patients who go through transplant may have morbidity or mortality that they would be having with the transplant procedure that would be worse than if they're just getting standard-of-care therapy with a hypomethylating agent or hypomethylating agent combination. In elderly patients, I think that's an area where we still need to do more study. Although survival is quite remarkable, it takes older people longer to recover from the toxicities that are associated with the transplant procedure.