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X linked- ALD: Should Loes score be the only benchmark for HSCT eligibility?
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  • Pavit Singh,
  • Parvathy KN,
  • Aditya Gupta,
  • Atin Kumar,
  • Gargi Das,
  • Sayoni Roy Chowdhury,
  • Jagdish Meena,
  • Biswaroop Chakrabarty,
  • Prashant Jauhari,
  • Rachna Seth,
  • Sheffali Gulati
Pavit Singh
All India Institute of Medical Sciences
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Parvathy KN
All India Institute of Medical Sciences
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Aditya Gupta
All India Institute of Medical Sciences Department of Pediatrics

Corresponding Author:[email protected]

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Atin Kumar
All India Institute of Medical Sciences Department of Radiodiagnosis
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Gargi Das
All India Institute of Medical Sciences Department of Pediatrics
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Sayoni Roy Chowdhury
All India Institute of Medical Sciences Department of Pediatrics
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Jagdish Meena
All India Institute of Medical Sciences Department of Pediatrics
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Biswaroop Chakrabarty
All India Institute of Medical Sciences Department of Pediatrics
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Prashant Jauhari
All India Institute of Medical Sciences Department of Pediatrics
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Rachna Seth
All India Institute of Medical Sciences Department of Pediatrics
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Sheffali Gulati
All India Institute of Medical Sciences Department of Pediatrics
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Abstract

Hematopoietic stem cell transplant(HSCT) is the only modality that, if performed in the early- stages of cerebral X-linked adrenoleukodystrophy(X-ALD) can lead to good outcomes. Loes- score, calculated based on the findings of the magnetic resonance imaging(MRI) of the brain, helps in the selection of patients for HSCT. As per recommendations, the Loes-score should be <10 for a patient to be eligible for HSCT. Here we report a child of cerebral X-ALD with Loes of 12, but a good neurological function score(NFS), who was treated with HSCT, and had good outcomes. It is therefore important to utilize other parameters along with the Loes score, in selecting patients for HSCT in X-ALD.