TY - JOUR
T1 - Successful cord blood transplantation with reduced-intensity conditioning for childhood cerebral X-linked adrenoleukodystrophy at advanced and early stages
AU - Niizuma, Hidetaka
AU - Uematsu, Mitsugu
AU - Sakamoto, Osamu
AU - Uchiyama, Toru
AU - Horino, Satoshi
AU - Onuma, Masaei
AU - Matsuhashi, Tetsuro
AU - Rikiishi, Takeshi
AU - Sasahara, Yoji
AU - Minegishi, Masayoshi
AU - Tsuchiya, Shigeru
PY - 2012/3
Y1 - 2012/3
N2 - 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.
AB - 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.
KW - adrenoleukodystrophy
KW - cord blood transplantation
KW - fludarabine
KW - melphalan
KW - reduced-intensity conditioning
UR - http://www.scopus.com/inward/record.url?scp=84857655767&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84857655767&partnerID=8YFLogxK
U2 - 10.1111/j.1399-3046.2011.01539.x
DO - 10.1111/j.1399-3046.2011.01539.x
M3 - Article
C2 - 21834803
AN - SCOPUS:84857655767
VL - 16
SP - E63-E70
JO - Pediatric Transplantation
JF - Pediatric Transplantation
SN - 1397-3142
IS - 2
ER -