Medicare Breakthrough for Patients Seeking a Transplant for Myeloma

Persons over age 65 who have multiple myeloma will no longer have to dig into their personal savings to pay for an autologous stem cell transplant. Effective October 1, 2000 Medicare will cover autologous stem cell transplants for myeloma patients, provided they meet certain criteria.

The Health Care Financing Administration (HCFA) - the agency that determines which medical procedures are eligible for coverage under Medicare - approved payment for the procedure for patients under age 78 who have Durie-Salmon Stage II or III disease, provided they are newly diagnosed or have responded to prior therapy. Patients must also have adequate heart, kidney, lung and liver function.

The ruling, which reversed Medicare's 10-year policy of not covering stem cell transplants for patients with myeloma, was heralded by patients and health care providers alike. Kathy Giusti, President of the Multiple Myeloma Research Foundation, called the decision "a major breakthrough."

"Multiple myeloma is still considered an incurable blood cancer," said Giusti, "but stem cell transplantation is one of the few treatments that can extend patients' lives. It should be available to all patients, including those covered by Medicare."

Approximately 12,000 new cases of multiple myeloma are diagnosed in the US each year. The majority of patients are over age 60, and for those who have retired from employment, their primary health insurance is Medicare. Until now, these patients have had to rely on personal funds or fundraising efforts to cover the cost of their transplant.

The ruling came one year after HCFA heard two days of testimony from patients and doctors about the benefits of treating myeloma patients with high-dose chemotherapy and an autologous stem cell transplant.

Kenneth Anderson MD, a transplant physician at the Dana Farber Cancer Institute in Boston, told the panel that an autologous stem cell transplant is the current standard of care for myeloma patients, both in the US and Europe. He described four studies that found high-dose chemotherapy and autologous stem cell transplantation safe and effective for myeloma patients.

The first was a French randomized trial¹, involving 200 patients, that compared high-dose chemotherapy and a stem cell transplant to several cycles of conventional-dose chemotherapy. The study found a significant survival advantage for patients on the transplant arm.

The second study², also from France, randomized 200 patients to receive either a transplant immediately after induction chemotherapy, or a transplant later after they relapsed. Both groups of patients had equivalent survival rates, but those who were transplanted earlier in the course of their disease enjoyed, on average, 39 months of disease-free survival, while those transplanted later were disease-free an average of only 13 months.

A third study³ demonstrated that myeloma patients' blood and immune systems recovered rapidly when transplanted with peripheral blood stem cells-more rapidly than had been observed after autologous bone marrow transplants-thereby shortening the time that they were susceptible to life-threatening infections.

The final study, a US trial that randomized 800 patients to receive either high-dose chemotherapy and an autologous stem cell transplant or several cycles of conventional dose chemotherapy, is still ongoing. Although the results will not be available for several years, Anderson noted that the mortality rate from treatment was less than 1 percent, indicating that the therapy is safe.

A major stumbling block to approving coverage of autologous transplants for myeloma patients was HCFA's concern that older patients could not tolerate the rigors of a transplant. But Anderson, as well as Bart Barlogie MD, Director of the Arkansas Cancer Research Center, and Anne Traynor MD, from Northwestern Memorial Hospital in Chicago, presented evidence that older patients were as capable of handling the therapy as younger patients.

Several transplant survivors testified at the proceedings offering living proof that older myeloma patients can withstand the rigors of an autologous stem cell transplant.

Seventy-one-year-old Ray Stevenson, a six-month survivor of a stem cell transplant for myeloma, told the panel "I'm happy to say that I progressed through the high-dose chemotherapy and stem cell transplant procedure without the many side effects experienced by others, most of whom are much younger [than me]…My cancer is currently in remission, and I'm feeling better now than I have for two years."

Although pleased with Medicare's new standards, Bart Barlogie MD believes coverage should be expanded to include patients with Stage I disease, patients older than 77, and tandem (double) transplants. "From our studies at the Arkansas Cancer Research Center, we are convinced that tandem transplants offer a superior outcome for patients, regardless of age," says Barlogie. "Nonetheless, the coverage which will be available Oct. 1, 2000 is an exciting beginning for providing care to a segment of patients that had been previously denied transplant options."

¹ Attal M, Harousseau J, Stoppa A et al. A prospective randomized trial of autologous bone marrow transplantation and chemotherapy in multiple myeloma. N Engl J Med 1996; 3355; 91-97

² Fermande J, Ravaud P, Chevret S et al. High dose therapy (HDT) and autologous blood stem cell transplantation versus convetional chemotherapy with HDT rescue in multiple myeloma: Results of a prospective randomized trial. Blood 1995; 86; 205a (abstract)

³ Vescio R, Schiller G, Stewart K, Ballester O et al. Multicenter phase III trial to evaluate CD34+ selected versus unselected autologous peripheral blood projenitor cell transplantation in multiple myeloma. Blood 1999; 93: 1-13