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Table 1 Baseline patient and treatment characteristics

From: Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome

Patient characteristics: N % Median Range
All MPS I 61    
  Gender (male/female) 38/23 62/38   
  Phenotype (Hurler/Attenuated) 44/17 69/31   
  Age @ Start of treatment (mths)    18 3-364
  Age @ final assessment (mths)    82 0.3-420
HSCT treated Hurler patient characteristics    
  Gender (male/female) 30/14    
  Age @ Start of treatment (mths)    14 3-30
  Age @ final assessment (mths)    66 11-203
  Interventions    
  Therapeutic airway intervention post-HSCT 3 7% Adenotonsillar surgery: 2 pts (1 had revision). Longterm O2: 1
  Treatments and metabolic characteristics    
  No HSCT (1/2/3) 32/8/1 78/20/2   
  Source (CB/BM/PBSC/Unknown) 17/15/7/2 41/37/17/5   
  Donor (Related/MUD) 15/26 37/63   
  IDUA @ 1 year post HSCT 36   30.0 6.3-87.0
  Heterozygote Donors 12   19.7 6.7-49.4
  Matched Unrelated Donors 24   34.6 17.7-87.0
  Pre HSCT DS:CS ratio 20   1.6 0.7-3.4
  DS:CS ratio @ 1 year 32   0.5 0.2-0.8
ERT treated Hurler Patients   
  Gender (male/female) 2/1    
  Age @ Start of treatment (mths)    85 74-144
  Age @ final assessment (mths)    123 98-148
ERT Treated attenuated patients   
  Gender (male/female) 9/8    
  Age @ Start of treatment (mths)    60 24-364
  Age @ final assessment (mths)    131 72-420
  Interventions     
  Therapeutic airway intervention on ERT 7 41% CPAP: 5 pts
Adenotonsillar Surgery: 2 pts (1 had revision)
  Metabolic characteristics    
  Pre ERT DS:CS ratio 5   1.9 1.1-2.7
  DS:CS ratio @ 1 year 13   0.8 0.4-2.2
  1. All characteristics concern the last HSCT. BM indicates bone marrow; CB, cord blood; IDUA, alpha-L-iduronidase enzyme level; mths, months; MUD, matched unrelated donor; N, number; PBSC, peripheral blood stem cells.