A Family Guide to Non-Oncologic Blood Disorders
How is MLD treated? There is no curative treatment for MLD. Stem cell transplant (SCT) has been proven as an effective method in slowing down the disease in people who do not show significant symptoms. Your medical team will determine the best treatment plan for your child. How successful is the treatment? For children with certain forms of MLD, an SCT may stabilize neurocognitive function in patients, but the loss of motor function often continues to progress. Some children may have normal or near-normal mental and physical development.
Stem Cell Transplant Indications • Patients who are mildly symptomatic or have asymp- tomatic late juvenile or adult onset forms of the disease • An identified matched and unaffected relative donor whose cells manufacture adequate levels of Arylsulfa- tase A • Unrelated and cord blood donors also have been utilized
Notes from Your Child’s Medical Team
Metachromatic Leukodystrophy
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