Table 1.

Disease and transplant characteristics of Patient 1 and 2

Pre-transplant characteristics						Conditioning regimen
	Age at MLD Diagnosis	Age at Treatment	ARSA gene Mutation analysis	ARSA Activity (n.v. 22–103)	MLD type	Bu AUC	Infused cell dose	Engraftment
								N > 0.5 × 109/L	PLT > 50 × 109/L
Pt 1	3 months	8 months	465 + 1 G > A c.240dup	1.4 nmol/h/mg	LI pre-symptomatic	84.9 mg h/L	18.2 × 106 CD34 + /kg	day +39	day +109
Pt 2	3 months	9 months	465 + 1 G > A c.240dup	1.4 nmol/h/mg	LI pre-symptomatic	63.4 mg h/L	14.1 × 106 CD34 + /kg	day +43	day +81

Pt patient, ARSA arylsulfatase A, MLD metachromatic leukodystrophy, LI late-infantile, Bu busulfan, AUC area under the curve, N neutrophil, PLT platelet.