Jada Bascom Foundation
All conditions

Inherited metabolic disorders

Adult-onset metachromatic leukodystrophy

Also called: Adult MLD · AO-MLD · ARSA deficiency · metachromatic leukodystrophy · MLD · Adult arylsulfatase A deficiency · Adult metachromatic leukodystrophy

What a donor has to do with this

A transplant is not a standard part of treating this condition. It is used rarely, in particular situations, and most people diagnosed with it will not have one.

This is our reading of published transplant guidelines for this condition, not a measurement of how many people need a donor. Where a source actually counted donors, the figure and the people it counted are shown further down. Where none did, we say so rather than estimate.

What the evidence says

Who it affects
Typical onset/diagnosis: adult MLD begins after age 16, sometimes in the fourth or fifth decade, in both sexes and represents about 10–20% of MLD. Evidence: U.S. GeneReviews (revised 2024) and a German four-patient series diagnosed at ages 17–37; no markedly enriched population was identified.
Treatments other than a transplant
Supportive neurologic, psychiatric, rehabilitative, and palliative care — standard noncurative management — multiple regions — No approved gene-therapy indication for adult-onset MLD.
If a transplant is used, the cells come from
bone marrow: used in 2 of 4 adults in the 2024 German single-center case series; mobilized peripheral blood stem cells: used in 2 of 4 adults in the 2024 German single-center case series; umbilical cord blood: not reported in the 2024 German adult case series; dominance: bone marrow and peripheral blood were tied at 2 grafts each in the 2024 German four-adult series
How often the donor was unrelated
Not reported. No source we could read states this for this condition, so we do not give a number. An estimate here would be a guess dressed as evidence.

Where this gets complicated

Evidence is limited to the 2024 German single-center series of four adults with approximately one year of neurologic follow-up; this supports a rare, selected role.; In the 2024 German four-adult series, three achieved full donor chimerism and remained neurologically stable at one year; the fourth deteriorated.

We are not asking you to register on this page

An unrelated donor is not a usual part of treating this condition, so it would be dishonest to use this page to ask you to register. Other conditions in the library are a different story.

Related conditions

Others in inherited metabolic disorders. They are genuinely different diseases with different treatments — the group name is not a diagnosis.

Where this came from