"Autologous Stem Cell Transplantation Proven Beneficial for Older Patients with Multiple Myeloma"... 4-Year Survival Rate Up 12.8 Percentage Points

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[Sportschosun Reporter Jong-Ho Jang] A study has found that autologous hematopoietic stem cell transplantation improves survival and is cost-effective even for older patients aged 65 to 69 with multiple myeloma.

The study is considered significant because it provides evidence from Korean data that eligibility for transplantation should not be determined solely by age.

Multiple myeloma is an intractable blood cancer in which plasma cells in the bone marrow proliferate malignantly. The number of cases in Korea rose nearly fourfold from 492 in 2000 to 1,961 in 2022. Patients aged 60 and older account for about 90% of all cases, making it an important challenge to establish treatment strategies for older patients as Korea’s population rapidly ages.

Autologous hematopoietic stem cell transplantation is considered a cornerstone of first-line treatment for multiple myeloma. However, debate has continued over whether to perform it in patients aged 65 and older because of treatment toxicity and financial burdens. As a result, coverage for transplantation in patients aged 65 and older was not introduced in Korea until 2019. The problem was that the evidence supporting such decisions was based largely on Western studies. Until now, no large-scale study had analyzed the cost-effectiveness of transplantation in Asian patients under a single-payer system with drug prices and medical cost structures different from those in the West.

Against this backdrop, a research team led by Chang-Ki Min of the Hematology Hospital at The Catholic University of Korea Seoul St. Mary’s Hospital, with Su-In Choi of the Department of Pharmacology at the Catholic University College of Medicine as co-corresponding author and Jeong-Yeon Lee of the Hematology Hospital as first author, analyzed claims data from the Health Insurance Review & Assessment Service (HIRA) covering 2009 to 2024. The team identified 735 patients aged 65 to 69 who had been newly diagnosed with multiple myeloma. Of these, 458 received autologous hematopoietic stem cell transplantation after induction therapy with bortezomib, thalidomide, and Dexamethasone (VTd), while 277 received drug treatment alone without transplantation. Because the two groups initially differed in factors such as age and health status, the researchers applied inverse probability of treatment weighting (IPTW) to make the groups as comparable as possible. Survival and costs were estimated using a semi-Markov model. This statistical method uses computer simulations of patients’ transitions through stages such as treatment, relapse, and death to predict survival and medical costs over a future period longer than the actual observation period.

The analysis showed that the transplantation group had a 4-year survival rate of 67.8%, 12.8 percentage points higher than the 55.0% recorded in the non-transplantation group. The risk of death was 38.0% lower among patients who received transplantation. The time until the start of subsequent treatment was also longer in the transplantation group, at 29.8 months compared with 19.7 months in the non-transplantation group—a difference of 10.1 months.

Cost-effectiveness also pointed in the same direction as the clinical outcomes. Although total medical costs were approximately 29.9 million won higher in the transplantation group, its quality-adjusted life expectancy was 0.71 years longer. The incremental cost-effectiveness ratio—the additional cost required for each additional quality-adjusted life-year—was approximately 42.4 million won, below the Korean threshold of about 52.4 million won, equivalent to the per-capita gross domestic product (GDP).

A 1,000-run simulation to assess uncertainty found a 70.3% probability that transplantation was cost-effective. When the time until subsequent treatment was used as the basis, the additional cost per additional quality-adjusted life-year fell to approximately 23.6 million won, and the probability of cost-effectiveness rose further to 98.9%. In other words, patients who received transplantation incurred higher costs but lived longer and in better health, while the additional expense remained within the range that Korea’s healthcare system could afford.

The structure of medical costs in Korea helps explain these results. While the average cost of inpatient transplantation in the United States is approximately 232.7 million won, the cost in Korea is only about 29.7 million to 32.0 million won. The researchers explained that because the upfront burden of transplantation is relatively low, the long-term benefits of delaying relapse can more easily offset the cost.

The research team said the findings are consistent with the direction of international clinical guidelines, which recommend determining transplant eligibility based on physical fitness rather than age. Although Korea has expanded transplant coverage eligibility to patients up to age 70, only 62.3% of eligible patients were found to actually receive transplantation.

Chang-Ki Min, who led the study, said, "Although transplantation is still not even considered in some cases simply because a patient is older, this analysis used Korean data to show that transplantation can extend survival even in patients aged 65 to 69 and that its cost remains within the range our healthcare system can afford." He added, "The decision on whether to perform transplantation should be based on the patient’s overall physical condition, not age."

Su-In Choi, the co-corresponding author, stated, "Overseas cost-effectiveness studies are difficult to apply directly to Korea because drug prices and medical cost structures differ. Since this study established evidence suited to Korea’s circumstances using claims data from the entire population, we expect it to contribute to future discussions on coverage policy."

The study was conducted as a multicenter study, CAREMM-2110, by the Catholic Multiple Myeloma Research Network (CAREMM). Sung-Soo Park and Seong-Gyu Byun of the Hematology Hospital at Seoul St. Mary’s Hospital also participated as co-authors. The findings were published online ahead of print in *Value in Health*, the official journal of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), and are scheduled for formal publication in October.

Jong-Ho Jang bellho@sportschosun.com

(From left) Chang-Ki Min, Sung-Soo Park, and Jeong-Yeon Lee of the Hematology Hospital at Seoul St. Mary’s Hospital, and Su-In Choi of the Department
(From left) Chang-Ki Min, Sung-Soo Park, and Jeong-Yeon Lee of the Hematology Hospital at Seoul St. Mary’s Hospital, and Su-In Choi of the Department of Pharmacology at the Catholic University College of Medicine

This article has been translated by GripLabs Mingo AI.

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