UPDATED with comments December 9, 2019 // ORLANDO, Florida — A global survey has found an increase in the number of autologous hematopoietic stem cell transplants (HSCT) in patients with multiple myeloma.
"We have reported, for the first time, data on stem cell transplantation for multiple myeloma," said lead author Andrew J. Cowan, MD, from the Department of Medicine, University of Washington, and the Fred Hutchinson Cancer Research Center, Seattle. "Transplantation for myeloma remains highly utilized globally, and we consider it to be part of the standard of care."
"We also report declines in the use of allogeneic transplantation, which is likely driven by conflicting clinical trial data," he added. "And we think more work is needed to improve access."
The study results were presented here at the American Society of Hematology (ASH) 2019 annual meeting.
During the period from 2006 to 2015, the global number of autologous HSCT procedures increased by 107% for patients with multiple myeloma. Every region showed an increase during that period, although utilization of autologous HSCT was highest in North America and Europe, with an increase from 13% to 24% in North America and from 15% to 22% in Europe.
Activity in Latin American countries also increased markedly, by 46%, and utilization reached more than 10% for the first time.
Conversely, utilization was much lower in the Africa/Mediterranean and Asian/Pacific region, and there was only a marginal increase from 1.8% in 2006 to 4% in 2015.
"We see a totally different story with allogeneic stem cell transplants, where globally, the numbers have remained largely stable," he said. "Some regions, notably North America, have seen about a 50% decline."
Overall, there was a global 3% decline in the number of first allogeneic HSCT that were performed for patients with multiple myeloma, after peaking in 2012, and rates remain highest in Europe with an increase of 8%.
The findings come from a new analysis of a retrospective global survey from the Worldwide Network for Blood and Marrow Transplantation that was published a few years ago (Bone Marrow Transplant. 2016;51:778-785). This included data on 68,146 HSCTs (53% autologous and 47% allogeneic) that was obtained from 77 countries. The Global Burden of Disease study was used to obtain incidence data estimates. South Asia and East Asia regions were combined for this analysis.
HSCT continues to remain the standard of care for eligible patients and is typically more affordable and accessible on a global level as compared with novel therapies, Cowan said.
Access to effective care (including proteasome inhibitors, immunomodulatory agents, and HSCT) has been largely limited to patients residing in high-income sociodemographic index nations. "There is a marked disparity in access and utilization of effective myeloma therapies," Cowan said. "Some countries in Africa and Central Asia lack access to transplant entirely."
In 2016, there were 138,509 incident cases of multiple myeloma, and a global increase of 126% from 1990 to 2016, explained Cowan. "This is largely attributable to an aging population," he said.
Approached for comment, Joshua Richter, MD, an assistant professor of medicine in The Tisch Cancer Institute at Mount Sinai Hospital, New York City, noted that "it's an ongoing discussion, at least in the US, about what the role is for autologous transplant in the setting of all of these new therapies."
"It may be that as we become better and better with our therapies, there becomes less and less of a push to use it," he told Medscape Medical News. "However, there are few therapies where you can give a half-hour infusion and people can be in remission for years, if not longer."
About 5%-10% of patients who receive a transplant "are going to have these amazing durable remissions that are going to last years and years, so I think there's still a very big role for it," Richter explained. "It's still a standard of care and underutilized in a variety of patients, both in the US and abroad. It's underutilized in older patients and there are clear racial disparities, and efforts need to be enhanced to make sure that everyone has the same opportunities."
He pointed out that a transplant can work out to be more economical in the long run. "A year's worth of triplet therapy is $300,000 to $500,000 (and is continued indefinitely) while a transplant is a one-off cost of about $50,000 to 100,000, depending on where it's done," he said. "So it's a very efficient means of preventing high healthcare costs utilization."
Richter said that the disparities observed in low- to middle-income countries is not surprising, as many countries lack the infrastructure and resources to treat complex diseases. The drugs are expensive, as are the upfront costs of HSCT. "There are clear disparities, but their resources are limited and have other pressing needs that require high healthcare utilization," he said.
Richter added that the decline in allogeneic transplant in the US is not surprising. "In the US, they are not 'standard of care' and only a handful of centers offer them, and they are reserved for very young and very high-risk patients," he explained. "At the moment we are on the cusp of approval for CAR-T cell therapy [for myeloma] and many of these therapies are currently in clinical trials. One of the exclusion criteria is previous allogeneic transplant, so I think that for some patients who might have gotten one a decade ago, they are now being directed toward some type of CAR-T."
Another expert noted that even though worldwide, the number of autologous hematopoietic stem cell transplants increased more than 100% from 2006 to 2015, it is still underutilized in Africa and Asia.
"Some of this may be due to socioeconomic circumstances, but that would need to be examined by country," commented Philip L. McCarthy MD, director of the Transplant and Cellular Therapy Center and professor of oncology and internal medicine at the Roswell Park Comprehensive Cancer Center, Buffalo, New York.
"Of note, transplant may be less economically burdensome than some of the new drugs, so it remains to be determined if the use of transplant will increase. Note, not all patients are transplant eligible, so it would be important to factor in eligibility based on comorbidities and performance status which affect suitability for AHSCT."
Richter has disclosed the following relationships with industry: Speaker's bureau: Celgene, Janssen; Advisory board/consulting: Celgene, Janssen, Karyopharm, Oncopeptides, Adaptive Biotechnologies, Antengene.
American Society of Hematology (ASH) 2019 Annual Meeting: Abstract 412. Presented December 8, 2019.
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Cite this: Increase in Autologous HSCT for Myeloma, Decline in Allogeneic - Medscape - Dec 08, 2019.