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Table 7 Attainment of therapeutic goals: South Florida patients with GD1 and the ICCG benchmark cohort[18]

From: Patients with type 1 Gaucher disease in South Florida, USA: demographics, genotypes, disease severity and treatment outcomes

   Therapeutic goal

South Florida GD1 patients (Median treatment:12 years minimum treatment: 3 years)

ICGG cohort (N-195) (evaluated after 4 years of ERT)

Bone pain (N = 61)

45.3%#

70.2%#

Hemoglobin (N = 61)

90.6%*

91.6%

§Platelets (N = 46)

85.4%

79.6%

§Spleen volume (N = 46)

93.3%

78.0%

Liver volume (N = 61)

93.4%

90.6%

Bone crises (N = 61)

100%

99.9%

§Attained 6/6 goals (N = 46)

30.4%

41.4%

§§Attained 5/5 goals (N = 15)

40.0%

Not applicable

  1. §Intact spleen patients only.
  2. §§Splenectomy patients.
  3. #63% of the ICGG patient cohort had no reported bone pain prior to initiation of treatment. 73.4% of the South Florida patients reported bone pain prior to initiation of treatment; bone pain was scaled as moderate or worse in 59.6%.
  4. *In 5/6 patients, Hb was depressed because of concurrent illnesses at evaluation point (auto-immune hemolytic anemia, acute myeloid leukemia, myelodysplasia, chronic kidney disease[2]).
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172