We previously reported that in pts with CVA aggressively transfused for 4 yrs, reduction of intensity of CTX prevents recurrent CVA and results in a 31% reduction in transfusion requirements. While we still have not seen confirmed recurrent CVA in any of our original 14 pts, 1 pt died with status epilepticus and 6 others have died (ages 19-24 yrs after 9-16 yrs of CTX) of other causes(liver/heart failure, infection, acute chest syndrome); all had hemochromatosis. In an attempt to stop CTX in some pts, conventional (CA) and/or magnetic resonance (MRA) angiography were done in 16 pts after 1-188 mos (median 115 mos) of CTX. Six (38%) had normal studies (2 CA, 3 MRA, 1 both) and discontinued CTX after 2-13.8 yrs. The pt who discontinued CTX after only 2 yrs of CTX had recurrent CVA and markedly abnormal MRA 8 mos after stopping CTX. One pt died CVA-free with liver failure and suspected sepsis 17 mos after stopping, and the remainder (67%) are CVA-free 9+, 22+, 27+, and 57+ mos off CTX. In conclusion, CTX modification alone is not sufficient to prevent mortality due largely to hemochromatosis; a significant subgroup of CVA pts may have normal vessels and be at low risk for CVA recurrence; and some pts may have progressive vascular disease if not transfused.

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