Successful cord blood transplantation with reduced-intensity conditioning for childhood cerebral X-linked adrenoleukodystrophy at advanced and early stages

Hidetaka Niizuma, Mitsugu Uematsu, Osamu Sakamoto, Toru Uchiyama, Satoshi Horino, Masaei Onuma, Tetsuro Matsuhashi, Takeshi Rikiishi, Yoji Sasahara, Masayoshi Minegishi, Shigeru Tsuchiya

Research output: Contribution to journalArticlepeer-review

4 Citations (Scopus)

Abstract

Childhood cerebral ALD is a rapidly progressive and neurodegenerative disorder for which HSCT is the curative therapy if carried out at early stages. We successfully treated two patients of childhood cerebral ALD by CBT with RIC. The proband was a seven-yr-old boy whose brain MRI severity score (Loes score) was 14.5. Unrelated CBT was performed in five wk. To minimize conditioning regimen-related neurotoxicity, the combination of fludarabine (125 mg/m 2), melphalan (140 mg/m2), and 4 Gy of brain-sparing TBI was used. The second patient was a six-yr-old brother of the proband. Four wk after the detection of a single small lesion (Loes score 1), he received unrelated CBT with the same RIC as the proband. In both patients, the engraftment was fast and stable, and severe complications were not observed. Furthermore, gadolinium-enhanced inflammation on brain MRI rapidly disappeared after CBT. Now, 20 and 13 months have passed after CBT, respectively, and both patients are neurologically stable. The RIC we used was sufficient for stable engraftment of cord blood and also tolerable even to the patient with advanced ALD. RIC-CBT should be considered for the patients with cerebral ALD at advanced stages, as well as those at early stages.

Original languageEnglish
Pages (from-to)E63-E70
JournalPediatric Transplantation
Volume16
Issue number2
DOIs
Publication statusPublished - 2012 Mar

Keywords

  • adrenoleukodystrophy
  • cord blood transplantation
  • fludarabine
  • melphalan
  • reduced-intensity conditioning

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Transplantation

Fingerprint Dive into the research topics of 'Successful cord blood transplantation with reduced-intensity conditioning for childhood cerebral X-linked adrenoleukodystrophy at advanced and early stages'. Together they form a unique fingerprint.

Cite this