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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]).