Table 33Treatment benefits and harms for adrenoleukodystrophy

DiseaseTreatmentSource, Evidence TypeIndicationsBenefitsHarms
AdrenoleukodystrophyAllogeneic HSCTPeters 2004,270
literature review

recommended as soon as diagnosis for child onset of cerebral adrenoleukodystrophy is confirmed


18 months post HSCT, behavioral and cognitive functions improveda


MRI showed complete disappearance of lesions in brain if demyelination moderatea,b,c


MRI showed deterioration stabilized if demyelination more extensiveb,c


cognitive function stabilized or improved in 7 of 12 ptsb


8 of 12 functioning normally in school with no additional supportb


5 yr survival: 70% with 0 neurologic deficits, 67% with 1 neurological deficit, 35% with 2 or more neurological deficitsd


31 of 58 had no further neurological progression of diseased


treatment-related mortality at 3 yrs: 10% with related donor, 18% with unrelated donord


severe acute GVHD: 17% with related donor, 8% with unrelated donord

Krivit et al. 1999,337
literature review

Aubourg et al. 1990,340 case report, 18 months post HSCT


Shapiro et al. 2000,341 case series of 12 pts, 5-10 yrs post HSCT


Loes et al. 1994,342 case series of 7 pts, 1-2 yrs post HSCT


Peters et al. 2004,270 case series of 94 pts, 0.4-11.2 yrs post HSCT

From: Narrative Reviews

Cover of Hematopoietic Stem-Cell Transplantation in the Pediatric Population
Hematopoietic Stem-Cell Transplantation in the Pediatric Population [Internet].
Comparative Effectiveness Reviews, No. 48.
Ratko TA, Belinson SE, Brown HM, et al.

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