Transplant: Understanding Its Role in Myeloma Today and Tomorrow

What is the role of stem cell transplant in multiple myeloma? And what will it be in the near and distant future?
Of course, answers to what role the stem cell transplant will play in the future can only be guessed or predicted. Still, it's an important question, especially since efficacious and less invasive myeloma treatments are more inviting than the complicated and temporarily debilitating stem cell (bone marrow) transplant and the chemotherapy surrounding it.
Dr. Cristina Gasperetto, a multiple myeloma specialist at Duke, shared the current role of stem cell transplant within myeloma therapy with the audience at the Raleigh Myeloma Round Table just last month.
Today's Myeloma (and how we got there)
While the first case of multiple myeloma was officially documented in 1844, the stem cell (or bone marrow transplant) was not used regularly for myeloma patients until the 1990s. Previously to this time, although some treatments had been found to treat myeloma, the chemotherapy was intensive and did not result in a significant increase in overall survival.
Dr. Bart Barlogie, a ground-breaking myeloma specialist, began treating myeloma patients with stem cell transplants in the late '80s. By the '90s he published disruptive clinic trial results that would lead to a new standard of care, the stem cell transplant for almost all myeloma patients. After 40 years with no significant increase in overall survival, the myeloma life expectancy rate climbed to an average of 5 years (within this study population).
Since that time, researchers and specialists have worked to improve the stem cell transplant procedure (as well as the surrounding induction and maintenance therapies) to maximize results and eliminate complications. These improvements have led to longer, higher-quality survival for many patients, with several patients living a decade after their diagnosis.
What have these investigators learned about improving the stem cell transplant process?
- When it comes to patients with high-risk disease or high tumor burden, eliminating the disease quickly and deeply is incredibly important for controlling the myeloma.
- Maintenance therapy is important in achieving and sustaining these deep responses.
The IMF 2009 study compared an arm of patients with lenalidomide, bortezomib, and dexamethasone continued therapy to another arm with the same induction therapy, followed by a stem cell transplant, a couple of rounds of consolidation therapy, and maintenance therapy of lenalidomide.
The progression-free survival was superior within the transplant arm of this study at both 44 and 90 months. Now, there isn't a significant difference when comparing the overall survival between the two arms.
But what the study doesn't tell you is that 75% of those who were in the continued therapy arm later proceeded to have a stem cell transplant outside of the trial, meaning that the majority of patients in this trial received a stem cell transplant (whether it be upfront or delayed) and therefore comparing their overall survival statistics becomes insignificant.
Other more recent studies trying to determine the depth of response after a stem cell transplant using minimal residual disease markers. For more information about these trials, please watch the video above.
For now, the stem cell transplant remains a widely successful procedure for myeloma patients who want to eliminate their disease burden and go without significant medications (or perhaps no medication at all) for a long period of time.
The Future of Stem Cell Transplant in Myeloma
Today, the rise of immunotherapy treatment in myeloma calls into question the role and timing of stem cell transplants in myeloma treatment.
This is a heavily debated topic within the myeloma world, and investigators have already begun to develop clinical trials directly comparing CAR-T with stem cell transplant.
When asked in a recent myeloma educational webinar what he believed the future of the stem cell transplant would be, Dr. David Coffey shared,
I think that we'll always have the opporutnity to do a stem cell transplant for patients whom fail CAR-T or bispecific therapy... it's not that we are going to replace it, but it will be placed further down the line so that we focus first on the less-intense, less-side effect type therapy... hopefully, patients will undergo CAR-T Cell maybe in their first relapse... or at diagnosis, as opposed to waiting to their fifth relapse... I would love to give the CAR as early in therapy as possible when the T-cells are the healthiest.
The stem cell transplant works for most patients, so it wouldn't make sense to eliminate it completely. Where will it land in the sequencing of treatment for myeloma? Only time will tell.
Looking for more education on the subject?
For an extensive compilation of stem cell transplant resources, including advice on what to pack, financial help, hair loss, and more, visit https://healthtree.org/myeloma/community/asct.
See our Stem Cell Transplant Chapter to view past educational webinars on a variety of stem cell transplant-related subjects.
See our Immunotherapy Treatment Chapter to view past educational webinars on a variety of immunotherapy treatment-related subjects.
What is the role of stem cell transplant in multiple myeloma? And what will it be in the near and distant future?
Of course, answers to what role the stem cell transplant will play in the future can only be guessed or predicted. Still, it's an important question, especially since efficacious and less invasive myeloma treatments are more inviting than the complicated and temporarily debilitating stem cell (bone marrow) transplant and the chemotherapy surrounding it.
Dr. Cristina Gasperetto, a multiple myeloma specialist at Duke, shared the current role of stem cell transplant within myeloma therapy with the audience at the Raleigh Myeloma Round Table just last month.
Today's Myeloma (and how we got there)
While the first case of multiple myeloma was officially documented in 1844, the stem cell (or bone marrow transplant) was not used regularly for myeloma patients until the 1990s. Previously to this time, although some treatments had been found to treat myeloma, the chemotherapy was intensive and did not result in a significant increase in overall survival.
Dr. Bart Barlogie, a ground-breaking myeloma specialist, began treating myeloma patients with stem cell transplants in the late '80s. By the '90s he published disruptive clinic trial results that would lead to a new standard of care, the stem cell transplant for almost all myeloma patients. After 40 years with no significant increase in overall survival, the myeloma life expectancy rate climbed to an average of 5 years (within this study population).
Since that time, researchers and specialists have worked to improve the stem cell transplant procedure (as well as the surrounding induction and maintenance therapies) to maximize results and eliminate complications. These improvements have led to longer, higher-quality survival for many patients, with several patients living a decade after their diagnosis.
What have these investigators learned about improving the stem cell transplant process?
- When it comes to patients with high-risk disease or high tumor burden, eliminating the disease quickly and deeply is incredibly important for controlling the myeloma.
- Maintenance therapy is important in achieving and sustaining these deep responses.
The IMF 2009 study compared an arm of patients with lenalidomide, bortezomib, and dexamethasone continued therapy to another arm with the same induction therapy, followed by a stem cell transplant, a couple of rounds of consolidation therapy, and maintenance therapy of lenalidomide.
The progression-free survival was superior within the transplant arm of this study at both 44 and 90 months. Now, there isn't a significant difference when comparing the overall survival between the two arms.
But what the study doesn't tell you is that 75% of those who were in the continued therapy arm later proceeded to have a stem cell transplant outside of the trial, meaning that the majority of patients in this trial received a stem cell transplant (whether it be upfront or delayed) and therefore comparing their overall survival statistics becomes insignificant.
Other more recent studies trying to determine the depth of response after a stem cell transplant using minimal residual disease markers. For more information about these trials, please watch the video above.
For now, the stem cell transplant remains a widely successful procedure for myeloma patients who want to eliminate their disease burden and go without significant medications (or perhaps no medication at all) for a long period of time.
The Future of Stem Cell Transplant in Myeloma
Today, the rise of immunotherapy treatment in myeloma calls into question the role and timing of stem cell transplants in myeloma treatment.
This is a heavily debated topic within the myeloma world, and investigators have already begun to develop clinical trials directly comparing CAR-T with stem cell transplant.
When asked in a recent myeloma educational webinar what he believed the future of the stem cell transplant would be, Dr. David Coffey shared,
I think that we'll always have the opporutnity to do a stem cell transplant for patients whom fail CAR-T or bispecific therapy... it's not that we are going to replace it, but it will be placed further down the line so that we focus first on the less-intense, less-side effect type therapy... hopefully, patients will undergo CAR-T Cell maybe in their first relapse... or at diagnosis, as opposed to waiting to their fifth relapse... I would love to give the CAR as early in therapy as possible when the T-cells are the healthiest.
The stem cell transplant works for most patients, so it wouldn't make sense to eliminate it completely. Where will it land in the sequencing of treatment for myeloma? Only time will tell.
Looking for more education on the subject?
For an extensive compilation of stem cell transplant resources, including advice on what to pack, financial help, hair loss, and more, visit https://healthtree.org/myeloma/community/asct.
See our Stem Cell Transplant Chapter to view past educational webinars on a variety of stem cell transplant-related subjects.
See our Immunotherapy Treatment Chapter to view past educational webinars on a variety of immunotherapy treatment-related subjects.

about the author
Audrey Burton-Bethke
Audrey is a content writer and editor for the HealthTree Foundation. She originally joined the HealthTree Foundation in 2020. Audrey loves spending time with her supportive husband, energetic four-year-old, and new baby.
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