Table 4 Key studies of carfilzomib in... | American Society of Hematology

Table 4

Key studies of carfilzomib in relapsed/refractory and frontline MM

Study	Regimen	N	Response rates, %	Outcomes, %	Grade 3 or 4 AEs, %
Studies in relapsed/refractory MM
PX-171-004 phase 2	Cfz 20	59 btz-naive⁸⁵	ORR: 42	DOR: 13.1 mo	Thrombocytopenia 15; lymphopenia 14; pneumonia 14; anemia 12; neutropenia 12; fatigue 12; leukopenia 7; hyperglycemia 7; serum sodium decreased 7; dyspnea 5
			≥ VGPR: 17	TTP: 8.3 mo	PN 2
				PFS: 8.2 mo
	Cfz 20/27	70 btz-naive⁸⁵	ORR: 52	DOR: not reached	Lymphopenia 19; anemia 17; neutropenia 14; thrombocytopenia 11; pneumonia 11; serum phosphorous decrease 7; hypophosphatemia 7; hyponatremia 7
			≥ VGPR: 28	TTP: not reached	PN: 0
				PFS: not reached
	Cfz 20	35 btz-treated⁸⁶	ORR: 18	DOR: 9.0 mo	Anemia, thrombocytopenia, neutropenia 10 each; combined analysis with btz-naive group
			≥ VGPR: 6	TTP: 5.3 mo
PX-171-003-A0 phase 2¹⁰⁰	Cfz 20	46	≥ MR: 26	DOR: 7.4 mo	NR
			ORR: 13	TTP: 6.2 mo
PX-171-003-A1 phase 2^88,89	Cfz 20/27	266	≥ MR: 36	DOR: 7.4 mo	Thrombocytopenia 22; anemia 20; lymphopenia 10; pneumonia 8; neutropenia 8; fatigue 7; hyponatremia 5; hypercalcemia 5
			ORR: 24
			≥ VGPR: 5
Phase 1b⁹⁰	CRd	32	ORR: 55	NR	Thrombocytopenia 15; anemia 15; neutropenia 8
			≥ VGPR: 30
Studies in frontline MM
Phase 2 EMN study⁹¹	CARTHADEX (CTd)	34	ORR: 85	NR	Cardiovascular including DVT 26; metabolic disorders 21; reversible renal failure 16; tumor lysis syndrome 11; gastrointestinal 11; skin rash 11
			≥ VGPR: 68
Phase 1/2 study⁹²	CRd	53	After 4 cycles:	NR	Hyperglycemia 24; anemia 18; neutropenia 12; thrombocytopenia 10; DVT/PE while on aspirin prophylaxis 10; infection 6
			ORR: 100
			≥ VGPR: 89
			CR/nCR: 71

Study	Regimen	N	Response rates, %	Outcomes, %	Grade 3 or 4 AEs, %
Studies in relapsed/refractory MM
PX-171-004 phase 2	Cfz 20	59 btz-naive⁸⁵	ORR: 42	DOR: 13.1 mo	Thrombocytopenia 15; lymphopenia 14; pneumonia 14; anemia 12; neutropenia 12; fatigue 12; leukopenia 7; hyperglycemia 7; serum sodium decreased 7; dyspnea 5
			≥ VGPR: 17	TTP: 8.3 mo	PN 2
				PFS: 8.2 mo
	Cfz 20/27	70 btz-naive⁸⁵	ORR: 52	DOR: not reached	Lymphopenia 19; anemia 17; neutropenia 14; thrombocytopenia 11; pneumonia 11; serum phosphorous decrease 7; hypophosphatemia 7; hyponatremia 7
			≥ VGPR: 28	TTP: not reached	PN: 0
				PFS: not reached
	Cfz 20	35 btz-treated⁸⁶	ORR: 18	DOR: 9.0 mo	Anemia, thrombocytopenia, neutropenia 10 each; combined analysis with btz-naive group
			≥ VGPR: 6	TTP: 5.3 mo
PX-171-003-A0 phase 2¹⁰⁰	Cfz 20	46	≥ MR: 26	DOR: 7.4 mo	NR
			ORR: 13	TTP: 6.2 mo
PX-171-003-A1 phase 2^88,89	Cfz 20/27	266	≥ MR: 36	DOR: 7.4 mo	Thrombocytopenia 22; anemia 20; lymphopenia 10; pneumonia 8; neutropenia 8; fatigue 7; hyponatremia 5; hypercalcemia 5
			ORR: 24
			≥ VGPR: 5
Phase 1b⁹⁰	CRd	32	ORR: 55	NR	Thrombocytopenia 15; anemia 15; neutropenia 8
			≥ VGPR: 30
Studies in frontline MM
Phase 2 EMN study⁹¹	CARTHADEX (CTd)	34	ORR: 85	NR	Cardiovascular including DVT 26; metabolic disorders 21; reversible renal failure 16; tumor lysis syndrome 11; gastrointestinal 11; skin rash 11
			≥ VGPR: 68
Phase 1/2 study⁹²	CRd	53	After 4 cycles:	NR	Hyperglycemia 24; anemia 18; neutropenia 12; thrombocytopenia 10; DVT/PE while on aspirin prophylaxis 10; infection 6
			ORR: 100
			≥ VGPR: 89
			CR/nCR: 71

Single-agent carfilzomib administered at either 20 or 27 mg/m² on days 1, 2, 8, 9, 15, and 16, in 28-day cycles. The 20/27 regimen was composed of one cycle at 20 mg/m² followed by 11 cycles at 27 mg/m².

Cfz indicates carfilzomib; DOR, duration of response; CRd, carfilzomib, lenalidomide, and dexamethasone; CTd, carfilzomib, thalidomide, and dexamethasone; MR, minimal response; NR, not reported; EMN, European Myeloma Network; DVT, deep vein thrombosis; and PE, pulmonary embolism.

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