Table 3.

Key clinical trials that led to the FDA approvals for acute and chronic GVHD

GVHD	Agent	Study	Number of subjects	Main eligibility Criteria	ORR at 28 days(CR)	Best ORR (CR)
Acute GVHD	Ruxolitinib	REACH1; Single arm Phase II	71	Age ≥ 12, any donor source for HCT, Grade II-IV steroid-refractory GVHD*, no more than 1 prior systemic treatment in addition to corticosteroids, myeloid engraftment	55% (27%)	73.2
		REACH 2; Randomized Phase III	309		62% (34%)	Not reported
Chronic GVHD	Ibrutinib	PYC-1129; Open-label, Phase Ib/II	42	Age ≥ 18, steroid-dependent or steroid-refractory GVHD, received no more than 2 previous regimens for GVHD, Erythematous rash >25% BSA or total National Institutes of Health (NIH) mouth score > 4	Not reported	67% (21%)
	Belumosudil	ROCKSTAR; Randomized Phase II	132	Age ≥ 12, persistent GVHD manifestations after 2-5 lines of systemic therapy, stable dose of corticosteroids for 2 weeks prior to enrollment	Not reported	76% (5%)
	Ruxolitinib	REACH 3; Randomized Phase III	329	Age ≥ 12, moderate-to-severe steroid-refractory or steroid dependent GVHD**, no more than 1 prior systemic treatment in addition to corticosteroids	50%*** (7%)	76% (12%)

Abbreviations: FDA, US Food and Drug Administation; GVHD, graft-versus-host disease; ORR, objective reponse rate; CR, complete response; HCT, hematopoietic cell transplantation; BSA, body surface area.

*Per Mount Sinai Acute GVHD International Consortium (MAGIC) criteria.

**According to NIH consensus criteria.