Table 1. Nonmyeloablative allo-HCT vs... | American Society of Hematology

Table 1.

Nonmyeloablative allo-HCT vs gene therapy/editing

Variable	BMT CTN²⁷	Lovo-cel²⁸	Exa-cel²⁹
Number of patients	42	35	44
Follow-up, mo (range)	37.2 (20.4-56.4)	17.3 (3.7-37.6)	19.3 (0.8-48.1)
Median age, y (range)	22.8 (15.5-43.2)	24 (12-38)	21.2 (12-35)
Pretransplant conditioning	Reduced intensity	Myeloablative	Myeloablative
Neutrophil engraftment (median day)	25.5	20	27
Plt recovery (median day)	34.5	36	34.5
Hb (g/dL, mean)	13.5	11	11
GVHD	5%	None	None
Graft failure	5%	None	None
OS	95%	96%	98%
EFS	88%	85%	90%
Durability	Likely lifelong	?	?
Hemolysis	None	Yes, but clinically insignificant	Yes, but clinically insignificant
Infertility	? (Probably low in patients aged <30 years)	? (Probably near 100%)	? (Probably near 100%)
Leukemia	Rare with full chimerism	?	?
Cost ($)	<300 000	3.3 million	2.2 million

Variable	BMT CTN²⁷	Lovo-cel²⁸	Exa-cel²⁹
Number of patients	42	35	44
Follow-up, mo (range)	37.2 (20.4-56.4)	17.3 (3.7-37.6)	19.3 (0.8-48.1)
Median age, y (range)	22.8 (15.5-43.2)	24 (12-38)	21.2 (12-35)
Pretransplant conditioning	Reduced intensity	Myeloablative	Myeloablative
Neutrophil engraftment (median day)	25.5	20	27
Plt recovery (median day)	34.5	36	34.5
Hb (g/dL, mean)	13.5	11	11
GVHD	5%	None	None
Graft failure	5%	None	None
OS	95%	96%	98%
EFS	88%	85%	90%
Durability	Likely lifelong	?	?
Hemolysis	None	Yes, but clinically insignificant	Yes, but clinically insignificant
Infertility	? (Probably low in patients aged <30 years)	? (Probably near 100%)	? (Probably near 100%)
Leukemia	Rare with full chimerism	?	?
Cost ($)	<300 000	3.3 million	2.2 million

BMT CTN, Blood and Marrow Transplant Clinical Trials Network 1507; plt, platelet; ?, unknown.