Table 2.
Clinical characteristics of the 34 Taiwanese patients with MPS II who died between 1995 and 2012
| Clinical characteristics | Number (%) |
|---|---|
| Age at onset of symptoms (years) (n = 32) | 2.5 ± 2.1 |
| Age at diagnosis (years) (n = 32) | 4.8 ± 3.1 |
| Gestational age (weeks) (n = 24) | 39.2 ± 1.8 |
| Birth weight (gram) (n = 24) | 3522 ± 581 |
| Birth weight (SDS) (n = 24) | 0.56 ± 1.30 |
| Primary cause of death, n (%) | |
| Respiratory failure | 19 (56 %) |
| Cardiac failure | 6 (18 %) |
| Post-traumatic organ failure | 1 (3 %) |
| Infection (sepsis) | 1 (3 %) |
| Unknown | 7 (21 %) |
| Age at death (years) | 14.2 ± 4.2 |
| Age at death, n (%) | |
| <5 years | 0 |
| ≧5 to <10 years | 5 (15 %) |
| ≧10 to <15 years | 16 (47 %) |
| ≧15 to <20 years | 11 (32 %) |
| ≧20 years | 2 (6 %) |
| Year of death, n (%) | |
| 1995–2000 | 4 (12 %) |
| 2001–2004 | 10 (29 %) |
| 2005–2008 | 10 (29 %) |
| 2009–2012 | 10 (29 %) |
| Phenotype, n (%) | |
| Severe | 31 (91 %) |
| Intermediate | 1 (3 %) |
| Mild | 2 (6 %) |
| ERT, n (%) | |
| Yes | 2 (6 %) |
| No | 32 (94 %) |
| HSCT, n (%) | |
| Yes | 2 (6 %) |
| No | 32 (94 %) |
MPS mucopolysaccharidosis, SDS standard deviation score, ERT enzyme replacement therapy, HSCT hematopoietic stem cell transplantation