July 20, 2017
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Autologous stem cell transplantation improves outcomes in refractory myeloma

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High-dose therapy with autologous hematopoietic stem cell transplantation appeared safe and effective among patients with refractory multiple myeloma, according to a retrospective analysis published in Cancer.

Patients with multiple myeloma who fail to respond to induction therapy or who become refractory after initial response demonstrate poor outcomes. This especially includes patients who are double refractory, or refractory to both proteasome inhibitors and immunomodulatory agents.

“These constitute a higher risk population for which there are scant data on the role of autologous HSCT,” Qaiser Bashir, MD, assistant professor in the department of stem cell transplantation at The University of Texas MD Anderson Cancer Center, and colleagues wrote.

The researchers retrospectively analyzed 223 patients with refractory multiple myeloma (double refractory, n = 105; nondouble-refractory, n = 128) who underwent autologous HSCT between March 2000 and October 2015 at MD Anderson Cancer Center.

Median follow-up was 42 months.

Researchers reported an overall response rate of 80%, including 22% of patients with complete responses, 18% with very good partial response and 40% with partial response.

Patients with double-refractory multiple myeloma demonstrated an ORR of 79%, including 25 patients with complete response. Patients with nondouble-refractory multiple myeloma had an ORR of 82%, including 27 patients with complete response.

Seventy-five percent of patients experienced disease progression and 60% died.

All refractory patients demonstrated:

  • a median PFS of 17.6 months (double-refractory group, 14.4 months; nondouble-refractory group, 18.2 months);
  • 2-year PFS rate of 38% (double-refractory group, 35%; nondouble-refractory group, 40%);
  • median OS of 48 months (double-refractory group, 38.9 months; nondouble-refractory group, 56.6 months); and
  • a 2-year OS rate of 74% (double-refractory group, 71%; nondouble-refractory group, 76%).

Researchers observed worse PFS among patients with relapsed and refractory multiple myeloma (HR = 1.9; P < .001), hemoglobin levels less than 10 g/dL (HR = 1.6; P = .004), high-risk cytogenetics (HR = 2.2; P < .001), and those who received more lines of prior treatment (HR = 1.2; P = .004) or had progressive disease before autologous HSCT (HR = 2.1; P < .001).

Similarly, an increased risk for death occurred among patients who had relapsed and refractory multiple myeloma (HR = 2; P < .001), hemoglobin levels less than 10 g/dL (HR = 1.8; P < .001), high-risk cytogenetics (HR = 2.3; P < .001), received more lines of prior treatment (HR = 1.2; P = .013), progressive disease before autologous HSCT (HR = 2.4; P < .001), and received triplet-induction treatment (HR = 1.6; P = .014).

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“Considering the current treatment options, combined high-dose therapy with autologous HSCT stands as an effective tool to implement response in patients who have refractory myeloma; however, further studies should be performed to evaluate the role of autologous HSCT in combination with newer agents,” Bashir and colleagues wrote. – by Kristie L. Kahl

Disclosure: Bashir reports he has no relevant financial disclosures. Please see the full study for a list of all other researchers’ relevant financial disclosures.