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Treating Cerebral ALD

Allogeneic hematopoietic stem cell transplantation (allo-HSCT)

Currently, the only treatment for cerebral adrenoleukodystrophy (ALD) that can arrest disease progression is allogeneic hematopoietic stem cell transplantation (allo-HSCT)—and it can be lifesaving.1 Early diagnosis during the first signs of cerebral involvement is critical because outcomes with allo-HSCT are more favorable with earlier initiation of treatment.2

In a study of patients with both early and advanced cerebral ALD, treatment early in the course of cerebral disease demonstrated greater major function disability (MFD)-free survival and overall survival.3

  MFD-FREE SURVIVAL OVERALL
SURVIVAL*
2 YEARS2 YRS 5 YEARS5 YRS
Early Disease (n=27) 91% 76% 94%
Advanced Disease (n=10) 20% 10% 90%
*Overall survival was assessed at both 2 and 5 years from cerebral ALD diagnosis.

In addition to measuring overall survival, it is also important to measure MFD-free survival in cerebral ALD. MFDs are six severe disabilities commonly attributed to cerebral ALD—they are of particular clinical importance because they can significantly compromise a patient’s ability to function independently. These MFDs are:3

  • Loss of communication
  • Cortical blindness
  • Tube feeding dependence
  • Wheelchair dependence
  • Complete loss of voluntary movement
  • Death related to neurologic deterioration

Outcomes of allo-HSCT are more favorable when hematopoietic stem cells from a matched sibling donor are used compared to cells from a mismatched or unrelated donor.3 Less than 30% of boys with cerebral ALD receive cells from a matched sibling donor.1

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