Ciltacabtagene autoleucel

A prospective randomized trial (NCT04181827) included 419 patients with relapsed myeloma after one to three prior lines of treatment who also had lenalidomide-refractory disease. Patients received either CAR T-cell therapy with cilta-cel or the standard of care at the discretion of physicians.[43]Based on this trial, the FDA approved cilta-cel for patients with (1) relapsed or refractory myeloma after at least one line of therapy including a proteosome inhibitor (such as bortezomib) and an immunomodulatory agent (such as lenalidomide) AND (2) lenalidomide-refractory disease.

A prospective randomized trial (NCT04181827) included 419 patients with relapsed myeloma after one to three prior lines of treatment who also had lenalidomide-refractory disease. Patients received either CAR T-cell therapy with cilta-cel or the standard of care at the discretion of physicians.[43]Based on this trial, the FDA approved cilta-cel for patients with (1) relapsed or refractory myeloma after at least one line of therapy including a proteosome inhibitor (such as bortezomib) and an immunomodulatory agent (such as lenalidomide) AND (2) lenalidomide-refractory disease.

With a median follow-up of 15.9 months, the 12-month PFS rate was 75.9% (95% CI, 69.4%–81.1%) in the cilta-cel group and 48.6% (95% CI, 41.5%–55.3%) in the standard-of-care group (HR, 0.26; 95% CI, 0.18–0.38; P < .001).[43][Level of evidence B1]

A prospective randomized trial (NCT04181827) included 419 patients with relapsed myeloma after one to three prior lines of treatment who also had lenalidomide-refractory disease. Patients received either CAR T-cell therapy with cilta-cel or the standard of care at the discretion of physicians.[43]Based on this trial, the FDA approved cilta-cel for patients with (1) relapsed or refractory myeloma after at least one line of therapy including a proteosome inhibitor (such as bortezomib) and an immunomodulatory agent (such as lenalidomide) AND (2) lenalidomide-refractory disease.

No difference in OS was reported. Grade 3 or higher cytokine-release syndrome occurred in 1.1% of patients in the CAR T-cell therapy group, and no patients had grade 3 or higher neurotoxicity.

A prospective randomized trial (NCT04181827) included 419 patients with relapsed myeloma after one to three prior lines of treatment who also had lenalidomide-refractory disease. Patients received either CAR T-cell therapy with cilta-cel or the standard of care at the discretion of physicians.[43]Based on this trial, the FDA approved cilta-cel for patients with (1) relapsed or refractory myeloma after at least one line of therapy including a proteosome inhibitor (such as bortezomib) and an immunomodulatory agent (such as lenalidomide) AND (2) lenalidomide-refractory disease.

Two patients developed a T-cell lymphoma on the skin at 5 and 16 months after cilta-cell infusion, and the T cells had detectable CAR transgenic expression and integration.[44] Both patients attained a complete response with lymphoma-directed therapy.

A prospective randomized trial (NCT04181827) included 419 patients with relapsed myeloma after one to three prior lines of treatment who also had lenalidomide-refractory disease. Patients received either CAR T-cell therapy with cilta-cel or the standard of care at the discretion of physicians.[43]Based on this trial, the FDA approved cilta-cel for patients with (1) relapsed or refractory myeloma after at least one line of therapy including a proteosome inhibitor (such as bortezomib) and an immunomodulatory agent (such as lenalidomide) AND (2) lenalidomide-refractory disease.

A quality-of-life evaluation showed significant improvement in global health status and visual analogue scale responses for patients who received cilta-cel.[45][Level of evidence A3]