Patient-Reported Outcomes With Stem Cell Transplant Signal Progress in Myeloma

Noa Biran, MD

Preliminary pilot data presented at the 2018 ASCO Annual Meeting suggest that autologous stem cell transplant (ASCT) does not impair short-term cognition, function, or symptoms of patients with newly diagnosed multiple myeloma, explained lead study author Noa Biran, MD.

Patient-reported outcomes focused on cognition, symptom extremity, and financial toxicity following ASCT. Patients were examined prior to and 6 months following treatment.

Twenty-two patients (79%) underwent assessment before and after ASCT; the median age was 66. Symptoms improved over the course prior to and following transplant, explained Biran. Patients experienced less anxiety (mean, 53.8; SD, 11.5-44.2; SD, 5), depression (mean, 50.0; SD, 10.5-42.5; SD, 3.9), fatigue (mean, 55.2; SD, 10.6-46.7; SD, 7.3), and pain interference (mean, 58.8; SD, 12.2-44.3; SD, 4.6).

Findings also reported a higher incidence of satisfaction with participation in social roles (mean, 48.2; SD, 10.3-57; SD, 9.2) and physical function (mean, 40.3; SD, 7.4-48.2; SD, 6.5). Additionally, there was less financial toxicity (mean, 30.1; SD, 5.6-23.4; SD, 9.4) and improvements in cognitive function (mean, 51.2; SD, 11.4-59.4; SD, 6.7).

Outcomes were measured on a scale of 0 to 100 with the Patient Reported Outcomes Measurement Information System (PROMIS). The PROMIS cognitive function v2 6a short-form accounted for cognitive decline. The COmprehensive Score for financial Toxicity (COST) was used to asses financial toxicity.

The question of whether ASCT improves quality of life (QOL) in these patients is an important one, said Biran, as larger randomized studies may one day direct FDA approvals and tailor treatment recommendations.

OncLive: What do these patient-reported outcomes tell us about ASCT in myeloma?

In an interview with OncLive, Biran, a physician in the Multiple Myeloma Division at John Theurer Cancer Center, discussed patient-reported outcomes following ASCT in patients with myeloma.Biran: We did a study that looked at patient-reported outcomes, financial toxicity, and cognitive function in patients who undergo ASCT. This is a pilot study with the purpose of obtaining information on feasibility, so that we can obtain funding for a larger grant and investigate this in a larger group of patients. We have enrolled about 75 patients. The data from a preliminary analysis showed that there was an improvement in many patient-reported symptoms, such as financial stress, neuropathy, mobility, and overall cognitive function based on the Common Terminology Criteria for Adverse Events.

What has the impact of the study been?

Some of the limitations of the study are that it is a small study. Most of the patients enrolled undergo ASCT. We'll need to look at more patients and randomize them to determine whether there is truly a difference.What patients don't understand is that the alternative to ASCT is chronic therapy. [Patients are] faced with a decision of continuing on chemotherapy for the rest of their lives, and that in itself has its own toxicity and can worsen over time.

Will patient-reported outcome studies become a focus across all types of therapy in multiple myeloma?

The hypothesis is that [chemotherapy] causes a continued decline in cognitive function in QOL compared with stem cell transplant. [With transplant] you have an acute decline in those phenomena, but eventually they will come back to baseline. It offers an opportunity to be monitored with little to no therapy afterwards for a number of years. Absolutely. The FDA will use these outcomes in terms of approving drugs. The real-world data are very different compared with what's published in the studies. For example, one triplet combination published a median PFS of 26.5 months, but the real-world data show that the median time on therapy is 8 months; that's a big difference.

Can you elaborate on other trials looking at patient-reported outcomes?

Is financial toxicity a big part of the equation?

We have to understand why our patients are not staying on therapy. We are doing active trials looking at patient-reported outcomes in order to answer that question. Patients often feel disease burden and treatment burden, so they're not staying on therapy. In multiple myeloma, it's very important to find regimens that are well tolerated because it [requires] continuous therapy; it's a chronic disease. We know that the longer people stay on therapy, the longer they live. The only way to find out how to do that is to look at the QOL. We're looking at using an app that allows frequent reporting of symptoms compared with people who just report symptoms at visits [to determine] if that will help patients stay on therapy. With the increasing burden of drugs on our society, our insurance, and the overall cost of care, patients suffer an exorbitant amount of financial toxicity. Most of the time, oral medications are not fully approved. They have very high copays plus physician visits. It's a very high burden of cost. Oftentimes, patients have to decide if they are going to get treatment or not. A lot of patients say, “I'd rather not get treatment. It's too much money. I just want my family to have that money.” [Treatment] is very important, but it's often limiting. Our resources are not unlimited.

Biran N, Jensen R, Potosky A, et al. Patient-reported outcomes following autologous stem cell transplantation for patients with multiple myeloma. J Clin Oncol. 2018;36 (suppl; abstr e22069).