Haploidentical stem cell transplantation in two children with mucopolysaccharidosis VI: clinical and biochemical outcome

Table 1 Details of the stem cell transplantations (SCT)

	Pat1 (male)	Pat2 (female)
Age at diagnosis	10 months	0 months
Age at umbilical-SCT	20 months	15 months
Conditioning regimen umbilical-SCT ¹	Bu/Cy/Thy	Bu/Cy/Thy
HLA match	6/6	4/6
TNC (×10 ⁶ /kg)	75	44
CD34+ (×10 ⁶ /kg)	1.2	0.26
GvHD prophylaxis	Ciclosporin and methylprednisolon
Outcome	Graft rejection day +39	Non engraftment with autologous reconstitution day +42
Time from umbilical- to haplo-SCT	54 days	59 days
Age at haplo-SCT	22 months	17 months
Cell source	Peripheral blood stem cells from father
CD34+ (10 ⁶ /kg)	27	15
CD3+ (10 ⁶ /kg)	0.047	0.0024
T-cell depletion	CliniMACS selection of CD34+ cells [20]
B-cell depletion	Rituximab in vivo day +1
Conditioning regimen haplo-SCT ¹	Flu/Eto/OKT3	Flu/TT/Mel/OKT3
GvHD prophylaxis	Mycophenolate mofetil day -1 to +28
Engraftment ANC > 500/ml	Day +10	Day +16
Complications	None	Encephalitis of unknown origin - recovered

TNC total nucleated cells, GvHD graft versus host disease, ANC absolute neutrophil count, Bu busulfan, Cy cyclophosphamide, Thy thymoglobuline, Flu fludarabine, Eto etoposide, OKT3 muromonab, TT thiotepa, Mel melphalan. ¹ for doses, see Methods.

ISSN: 1750-1172