Adrenoleukodystrophy

Treatment

Why do you wait for early signs before offering a boy with X-linked adrenoleukodystrophy a stem cell transplant?

Stem cell transplants (HSCT) are not offered before a boy exhibits early signs of X-linked adrenoleukodystrophy because stem cell transplants are not without their own risks. The stem cell transplant itself can have a mortality rate of 20-40%. Because of the variation in the degree of severity of symptoms it is recommended that boys do not undergo bone marrow transplant until cerebral (brain) findings are identified. Even at this point deciding to pursue a transplant is still a personal family choice.

Treating ALD. The ALD Foundation. http://www.stopald.org/treating-ald/

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