Table 2.

Selected studies of novel agents as secondary or first line therapy in acute GVHD

Novel Therapy	Type of trial	Type of treatment	Sample size	Overall Response at 4 weeks	Survival at 3–6 months	Study	Reference
Denileukin diftitox	Phase I	steroid refractory	30	71%	33%	Ho et al.	[148]
Denileukin diftitox	Phase II	steroid refractory	22	41%	ND	Shaughnessy et al.	[149]
Denileukin diftitox	Phase II randomized	Initial therapy	60	60%	49%	Alousi et al.	[122]
Etanercept	Phase II	Initial therapy	61	69%	69%	Levine et al.	[150]
Etanercept	Phase II randomized	Initial therapy	60	48%	56%	Alousi et al.	[122]
Pentostatin	Phase I	steroid refractory	23	78%	ND	Bolanos-Meade et al.	[151]
Pentostatin	Phase II randomized	Initial therapy	60	62%	52%	Alousi et al.	[122]
MMF	Phase I/II	steroid refractory	48	72%	78%	Basara et al.	[152]
MMF	Phase I/II	steroid refractory	10	60%	70%	Krejci et al.	[153]
MMF	Phase I/II	steroid refractory	6	67%	64%	Takami et al.	[154]
MMF	Phase II randomized	Initial therapy	60	78%	64%	Alousi et al.	[122]
Sirolimus	Phase I/II	steroid refractory	21	57%	28%	Benito et al.	[155]
BDP	Phase II randomized	Initial therapy	129	71%	87%	Hockenbery et al.	[119]
ECP	Phase II	steroid refractory	59	68%	ND	Greinix et al.	[125]
ECP	Phase II	steroid refractory	23	52%	48%	Perfetti et al.	[126]
ECP	Phase II	steroid refractory	41 (children)	73%	100%	Calore et al.	[127]
ECP	Phase II	steroid refractory	15 (children)	0% for grade IV GVHD	ND	Berger et al.	[128]
MSC	Phase II	steroid refractory	55	71%	ND	Le Blanc et al.	[130]
MSC	Phase II	steroid refractory	13	15%	31%	von Bonin et al.	[156]
Alefacept	Phase I	steroid refractory	16	75%	ND	Shapira et al.	[157]

GVHD indicates graft versus host disease, MMF, mycophenolate mofetil, BDP, beclomethasone diproprionate, ECP, extracorporeal photopheresis, MSC, mesenchymal stromal cell