Do all patients respond to an autologous stem cell transplant (ASCT)?
Only a minority of patients do not achieve some level of response from an ASCT; but only about 30% to 40% of patients will achieve a complete response (CR). It is important to know that even if patients do not achieve a CR after ASCT (or even if patients do not achieve minimal residual disease negativity), the majority of patients will still have years of stable, low-level disease. Furthermore, receiving maintenance therapy can deepen a patient’s response over time. Patients who achieve a very good partial response (VGPR) after ASCT may do quite well on maintenance for many years. The key is to have regular follow-up with your doctor to ensure that the disease remains stable.
If maintenance therapy has to be discontinued because of tolerability issues, what other options are available?
Revlimid is commonly used as maintenance therapy and is associated with rash or gastrointestinal problems, such as constipation and diarrhea. These side effects may become intolerable over time (since patients have to be on maintenance therapy indefinitely). If tolerability to Revlimid is an issue, the doctor may first want to try lowering the dose of Revlimid before even considering discontinuing Revlimid altogether. Also, medications to help manage symptoms (such as Imodium to help with diarrhea) or to target underlying causes (such as colestipol to bind up bile acids), as well as changes in diet, may be helpful in addressing or preventing some of the side effects. However, if a patient needs to discontinue Revlimid, the doctor may switch to another maintenance agent that is in a different class than Revlimid such as a proteasome inhibitor (such as Kyprolis, Velcade, Ninlaro) or a monoclonal antibody (such as Darzalex). Ultimately, it is important for patients to receive and stay on maintenance therapy as long as it is tolerated since studies have shown that patients are at greater risk of relapse if maintenance therapy is discontinued.
What is the status of CAR T-cell availability?
Access to CAR T-cell therapy for patients with relapsed/refractory myeloma—either the two approved therapies (Abecma and Carvykti) or various clinical trials—has been getting better. To receive this type of therapy, patients need to remember two main items: (1) CAR T-cell administration occurs at large, academic medical centers; and (2) the actual procedure for making CAR T cells is complex. The process involves collecting a patient’s T cells and then waiting 4 to 6 weeks after T cell collection for the manufacturing of the CAR T cells. While a patient waits, their doctor needs to ensure that the myeloma remains stable. In very rare instances, a problem may arise with the manufacturing of the CAR T cells (for example, contamination may occur); if this happens, a patient may undergo another T cell harvest or they may have enough T cells frozen from another harvest to use.
Many clinical studies are under way investigating the use of CAR T-cell therapy in patients earlier in their disease stage (for example, after their first relapse). Talk to your doctor about the availability of these types of trials in your area.