Idecabtagene Vicleucel in Relapsed and Refractory Multiple Myeloma


Por: Munshi, NC, Anderson, LD, Shah, N, Madduri, D, Berdeja, J, Lonial, S, Raje, N, Lin, Y, Siegel, D, Oriol, A, Moreau, P, Yakoub-Agha, I, Delforge, M, Cavo, M, Einsele, H, Goldschmidt, H, Weisel, K, Rambaldi, A, Reece, D, Petrocca, F, Massaro, M, Connarn, JN, Kaiser, S, Patel, P, Huang, LP, Campbell, TB, Hege, K and San-Miguel, J

Publicada: 25 feb 2021
Resumen:
BACKGROUND Idecabtagene vicleucel (ide-cel, also called bb2121), a B-cell maturation antigendirected chimeric antigen receptor (CAR) T-cell therapy, has shown clinical activity with expected CAR T-cell toxic effects in patients with relapsed and refractory multiple myeloma. METHODS In this phase 2 study, we sought to confirm the efficacy and safety of ide-cel in patients with relapsed and refractory myeloma. Patients with disease after at least three previous regimens including a proteasome inhibitor, an immunomodulating agent, and an anti-CD38 antibody were enrolled. Patients received ide-cel target doses of 150 x 10(6) to 450 x 10(6) CAR-positive (CAR+) T cells. The primary end point was an overall response (partial response or better); a key secondary end point was a complete response or better (comprising complete and stringent complete responses). RESULTS Of 140 patients enrolled, 128 received ide-cel. At a median follow-up of 13.3 months, 94 of 128 patients (73%) had a response, and 42 of 128 (33%) had a complete response or better. Minimal residual disease (MRD)-negative status (<10(-5) nucleated cells) was confirmed in 33 patients, representing 26% of all 128 patients who were treated and 79% of the 42 patients who had a complete response or better. The median progression-free survival was 8.8 months (95% confidence interval, 5.6 to 11.6). Common toxic effects among the 128 treated patients included neutropenia in 117 patients (91%), anemia in 89 (70%), and thrombocytopenia in 81 (63%). Cytokine release syndrome was reported in 107 patients (84%), including 7 (5%) who had events of grade 3 or higher. Neurotoxic effects developed in 23 patients (18%) and were of grade 3 in 4 patients (3%); no neurotoxic effects higher than grade 3 occurred. Cellular kinetic analysis confirmed CAR+ T cells in 29 of 49 patients (59%) at 6 months and 4 of 11 patients (36%) at 12 months after infusion. CONCLUSIONS Ide-cel induced responses in a majority of heavily pretreated patients with refractory and relapsed myeloma; MRD-negative status was achieved in 26% of treated patients. Almost all patients had grade 3 or 4 toxic effects, most commonly hematologic toxic effects and cytokine release syndrome.

Filiaciones:
Munshi, NC:
 Dana Farber Canc Inst, Jerome Lipper Multiple Myeloma Ctr, Boston, MA USA

 Harvard Med Sch, Vet Affairs Boston Healthcare Syst, Boston, MA 02215 USA

Anderson, LD:
 Univ Texas Southwestern Med Ctr Dallas, Simmons Comprehens Canc Ctr, Dallas, TX 75390 USA

Shah, N:
 Univ Calif San Francisco, San Francisco, CA 94143 USA

Madduri, D:
 Icahn Sch Med Mt Sinai, New York, NY 10029 USA

Berdeja, J:
 Sarah Cannon Res Inst, Nashville, TN USA

 Tennessee Oncol, Nashville, TN USA

Lonial, S:
 Emory Sch Med, Atlanta, GA USA

Raje, N:
 Massachusetts Gen Hosp, Boston, MA USA

Lin, Y:
 Mayo Clin, Rochester, MN USA

Siegel, D:
 Hackensack Univ, Med Ctr, Hackensack, NJ USA

:
 Hosp Badalona Germans Trias & Pujol, Inst Josep Carreras, Badalona, Spain

 Hosp Badalona Germans Trias & Pujol, Inst Catala Oncol, Badalona, Spain

Moreau, P:
 Ctr Hosp Univ CHU Nantes, Nantes, France

Yakoub-Agha, I:
 Univ Lille, Inst Translat Res Inflammat, INSERM Unite 1286, CHU Lille, Lille, France

Delforge, M:
 Univ Hosp Leuven, Leuven, Belgium

Cavo, M:
 Bologna Univ, Seragnoli Inst Hematol, Sch Med, Bologna, Italy

Einsele, H:
 Univ Hosp Wurzburg, Wurzburg, Germany

Goldschmidt, H:
 Univ Hosp Heidelberg, Heidelberg, Germany

 Natl Ctr Tumor Dis, Heidelberg, Germany

Weisel, K:
 Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany

 Univ Klinikum Tubingen, Tubingen, Germany

Rambaldi, A:
 Univ Milan, Dept Oncol & Hematol, Milan, Italy

 Azienda Socio Sanitaria Terr Papa Giovanni XXIII, Bergamo, Italy

Reece, D:
 Princess Margaret Canc Ctr, Toronto, ON, Canada

Petrocca, F:
 Bluebird bio, Cambridge, MA USA

Massaro, M:
 Bluebird bio, Cambridge, MA USA

Connarn, JN:
 Bristol Myers Squibb, Princeton, NJ USA

Kaiser, S:
 Bristol Myers Squibb, Princeton, NJ USA

Patel, P:
 Bristol Myers Squibb, Princeton, NJ USA

Huang, LP:
 Bristol Myers Squibb, Princeton, NJ USA

Campbell, TB:
 Bristol Myers Squibb, Princeton, NJ USA

Hege, K:
 Bristol Myers Squibb, Princeton, NJ USA

San-Miguel, J:
 Ctr Invest Biomed Red Canc, Inst Invest Sanitaria Navarra, Ctr Invest Med Aplicada, Clin Univ Navarra, Pamplona, Spain
ISSN: 00284793





New England Journal of Medicine
Editorial
Massachusetts Medical Society, WALTHAM WOODS CENTER, 860 WINTER ST,, WALTHAM, MA 02451-1413 USA, Estados Unidos America
Tipo de documento: Article
Volumen: 384 Número: 8
Páginas: 705-716
WOS Id: 000623807600010
ID de PubMed: 33626253
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