Table 2. List of immunosuppressive... | American Society of Hematology

Table 2.

List of immunosuppressive agents

		Published experience in hemophilia A
Agent	Primary mechanism of action	Human patients	Murine models	Reference
B- and T-cell lineage
CTX^*	Nonspecific cytotoxicity	Clinical trials w/ or w/o IVIG; partial response	No report	9, 10
MMF^*	IMPDH inhibition - primarily inhibits B + T proliferation	No report	No report	49
B-cell lineage
Anti-CD20 (rituximab)	Peripheral B-cell depletion	Case report	No report	7
Anti-CD40L, eg, MR^*	Blockade of CD40-dependent immunoglobulin class switch, T-helper-dependent B-cell activation	No report	Efficient blockade of inhibitor formation, transient duration	12, 13, 50
T-cell lineage
Anti-CD3	T-cell depletion or partial activation leading to anergy	No report	No report	51-53
CSA^*†	Inhibition of TCR-dependent signals	Case reports	No report	6, 54
CTLA4-lg^*	Inhibition of T-cell costimulation	No report	Efficient blockade of inhibitor formation, transient	55
RAP^*†	mTOR inhibition - blocks TCR signaling	No report	No report	56, 57

		Published experience in hemophilia A
Agent	Primary mechanism of action	Human patients	Murine models	Reference
B- and T-cell lineage
CTX^*	Nonspecific cytotoxicity	Clinical trials w/ or w/o IVIG; partial response	No report	9, 10
MMF^*	IMPDH inhibition - primarily inhibits B + T proliferation	No report	No report	49
B-cell lineage
Anti-CD20 (rituximab)	Peripheral B-cell depletion	Case report	No report	7
Anti-CD40L, eg, MR^*	Blockade of CD40-dependent immunoglobulin class switch, T-helper-dependent B-cell activation	No report	Efficient blockade of inhibitor formation, transient duration	12, 13, 50
T-cell lineage
Anti-CD3	T-cell depletion or partial activation leading to anergy	No report	No report	51-53
CSA^*†	Inhibition of TCR-dependent signals	Case reports	No report	6, 54
CTLA4-lg^*	Inhibition of T-cell costimulation	No report	Efficient blockade of inhibitor formation, transient	55
RAP^*†	mTOR inhibition - blocks TCR signaling	No report	No report	56, 57

IVIG indicates intravenous immunoglobulin; IMPDH, inosine monophosphate dehydrogenase; TCR, T-cell receptor; and mTOR, mammalian target of rapamycin.

Used in this study.

Also likely alter B-cell activation.