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Table 1 Patient characteristics showing age of onset, baseline characertistics at HSCT, conditioning regimen, and early outcome classification

From: Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy

ID Age at onset
(in years)
Age at HSCT + year GMFC.MLD at HSCT GMFM at HSCT (%) FSIQ at HSCT MRI-score at HSCT NCV at HSCT (m/s) Conditioning regimen Early HSCT outcome
1 13.1
(late juvenile)
13.9 2014 1 99.4 82 20 47 Treosulfan, Fludarabin Rapidly progressive
2 4.3
(early juvenile)
6.0 2009 1 93.1 98 17 15 Treosulfan, Fludarabin Stable
3 7.3
(late juvenile)
11.8 2015 1 99.5 79 18 22 Treosulfan, Fludarabin Rapidly progressive
4 9.8
(late juvenile)
11.6 2014 0 100 77 16 41 Treosulfan, Fludarabin Stable
5 4.8
(early juvenile)
4.9 2001 1 99 100 14 31 Busulfan, Cyclophosphamid Stable
6 4.3
(early juvenile)
5.2 2015 1 89.6 84 19 29 Treosulfan, Fludarabin Rapidly progressive
7 Pre-symptomatic 13.7 2008 0 100 112 0 34 Busulfan, Cyclophosphamid Stable
8 Pre-symptomatic 15.2 2006 0 100 114 11 42 Busulfan, Cyclophosphamid Stable
9 9.5
(late juvenile)
18.2 2010 0 98 68 21 50 Treosulfan, Fludarabin Stable
10 Pre-symptomatic (sibling onset 9.5) 14.8 2012 0 97 91 7 53 Treosulfan, Fludarabin Stable
11 11.4
(late juvenile)
13.6 2015 1 99.4 103 18 30 Treosulfan, Fludarabin Rapidly progressive
12 8.1
(late juvenile)
10.1 2009 1 98.6 66 20 17 Treosulfan, Fludarabin Rapidly progressive