Encouraging the outcomes of children with beta-thalassaemia major who underwent fresh cord blood transplantation from an HLA-matched sibling donor

Figures & data

Table 1. Patient characteristics of MSD-F-CBT for β-TM.

Characteristics	Number of patients (%) or median (range)
Number of patients	68
Median age in years (range)	4.5(1.6–8.5)
Male:female	42:26
Median weight(range)	16.0(9.0–26.0)
Group of TM patients
Class Ⅰ	17(25.0%)
Class Ⅱ	51(75.0%)
Class Ⅲ	0(0.0%)
Conditioning regimens
Thiotepa containing regimen	40(58.8%)
Thiotepa free regimen	28(41.2%)
GVHD prophylaxis
Scheme including MTX	56(82.4%)
Scheme without MTX	12(17.6%)
Matching at Ag level for HLA-A and -B and allele level for HLA-DRB1
Fully matched	67(98.5%)
Mismatched	1(1.5%)
TNC dose, median (range)(×107/kg)	8.51(3.0–20.3)
CD34 + cell dose, median (range) (×105/kg)	3.16(0.78–15.6)

Table 2. NF-08-TM conditioning regimens.

Conditioning	Cy		IV Bu (mg/kg/d)*		TT		Flu
	mg/kg/d	total days	mg/kg/d	total days	mg/kg/d	total days	mg/m^2/d	total days
Regimen for group I	60	2(−8 to −7)		3(−6 to −4)	5	1(−3)	40	5(−6 to −2)
1–2 year			4.4
>2 year			4.0
Regimen for group II	55	2(−8 to −7)		3(−6 to −4)	5	1(−3)	40	5(−6 to −2)
<6 years			3.6
>6 years			3.2
Regimen for group III	50	2(−8 to −7)		3(−6 to −4)	5	1(−3)	40	5(−6 to −2)
<8 years			3.0
>8 years			2.8

* The average plasma Css of busulfan was evaluated on day –8 and was targeted between 300 and 600 µg/L. The dose of busulfan was increased or decreased by 1.2 mg/kg when the Css was <300 µg/L or >600 µg/L and was given in divided doses on day –6.

Figure 1. Outcomes of patients given F-CBT.

Table 3. Clinical outcomes of patients who were given F-CBT.

Characteristics	Number of patients (%) or median (range)
Engraftment (median, range), days
Neutrophil >0.5 × 10^9/L	27(14–47)
Platelet >20 × 10^9/L	31(9–132)
Acute GVHD, n (%)
Grade Ⅰ-Ⅱ	5(7.8%)
Grade Ⅲ–IV	0(0.0%)
Chronic GVHD, n (%)	0(0.0%)
Transplantation-related complications, n (%)
CMV reactivation	9(13.2%)
Bacteraemia	17(25.0%)
Invasive fungal disease	6(8.8%)
Mucositis	31(45.6%)
Haemorrhagic cystitis (HC)	15(23.5%)
VOD	1(1.5%)
Autoimmune haemolytic anaemia (AHA)	3(4.4%)
Cytopenia	3(4.4%)
Follow-up (median, range), months	61(18–114)
Results of transplantation (at 5 years), n (%)
OS	67(98.5%)
DFS	60(87.9%)
GR	3(4.5%)
TRM	1(1.5%)
Chimerism status, n (%)
Completed chimerism	56(87.5%)
Mixed chimerism	8(12.5%)

Figure 2. Incidence of grade I to II acute graft-versus host disease.

Figure 3. Probabilities of overall survival and disease-free survival.