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Real-world effectiveness of eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry.

Publication ,  Journal Article
Mistry, PK; Balwani, M; Charrow, J; Kishnani, P; Niederau, C; Underhill, LH; McClain, MR
Published in: Am J Hematol
September 2020

Eliglustat is a first-line oral therapy for adults with Gaucher disease type 1 (GD1) with extensive, intermediate, or poor CYP2D6-metabolizer phenotypes (90% of patients). We report real-world outcomes after 2 years of eliglustat therapy in the International Collaborative Gaucher Group Gaucher Registry (NCT00358943). As of January 2019, baseline and 2-year data (±1 year) were available for 231 eliglustat-treated GD1 patients: 19 treatment-naïve (zero splenectomized) and 212 ERT patients who switched to eliglustat (36 splenectomized). Most patients (89%) were from the United States, where eliglustat was first approved. In treatment-naïve patients, mean hemoglobin increased from 12.4 to 13.4 g/dL (P = .004, n = 18), mean platelet count increased from 113 to 156 × 109 /L (P < .001, n = 17); mean spleen volume decreased from 7.4 to 3.5 multiples of normal (MN) (P = .02, n = 7); mean liver volume remained normal (n = 7), and median spine Z-score was unchanged (-1.3 to -1.2, n = 6). In non-splenectomized switch patients, mean hemoglobin remained stable/non-anemic (n = 167); mean platelet count remained stable/normal (n = 165); mean spleen volume decreased from 3.3 to 2.8 MN (P < .001, n = 64); mean liver volume remained normal (n = 63), and median lumbar spine Z-score improved from -0.7 to -0.4 (P = .014, n = 68). In splenectomized switch patients, mean hemoglobin remained stable/non-anemic (n = 31); mean platelet count increased from 297 to 324 × 109 /L (non-significant, n = 29); mean liver volume remained normal (n = 13); median spine Z-score improved from -0.8 to -0.6 (non-significant, n = 11). Median chitotriosidase decreased in all groups (P < .01 for all). These real-world results are consistent with eliglustat clinical trial results demonstrating long-term benefit in treatment-naïve patients and stability in ERT switch patients.

Duke Scholars

Published In

Am J Hematol

DOI

EISSN

1096-8652

Publication Date

September 2020

Volume

95

Issue

9

Start / End Page

1038 / 1046

Location

United States

Related Subject Headings

  • Splenectomy
  • Spleen
  • Registries
  • Pyrrolidines
  • Platelet Count
  • Organ Size
  • Middle Aged
  • Male
  • Longitudinal Studies
  • Liver
 

Citation

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Mistry, P. K., Balwani, M., Charrow, J., Kishnani, P., Niederau, C., Underhill, L. H., & McClain, M. R. (2020). Real-world effectiveness of eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry. Am J Hematol, 95(9), 1038–1046. https://doi.org/10.1002/ajh.25875
Mistry, Pramod K., Manisha Balwani, Joel Charrow, Priya Kishnani, Claus Niederau, Lisa H. Underhill, and Monica R. McClain. “Real-world effectiveness of eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry.Am J Hematol 95, no. 9 (September 2020): 1038–46. https://doi.org/10.1002/ajh.25875.
Mistry PK, Balwani M, Charrow J, Kishnani P, Niederau C, Underhill LH, et al. Real-world effectiveness of eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry. Am J Hematol. 2020 Sep;95(9):1038–46.
Mistry, Pramod K., et al. “Real-world effectiveness of eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry.Am J Hematol, vol. 95, no. 9, Sept. 2020, pp. 1038–46. Pubmed, doi:10.1002/ajh.25875.
Mistry PK, Balwani M, Charrow J, Kishnani P, Niederau C, Underhill LH, McClain MR. Real-world effectiveness of eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry. Am J Hematol. 2020 Sep;95(9):1038–1046.
Journal cover image

Published In

Am J Hematol

DOI

EISSN

1096-8652

Publication Date

September 2020

Volume

95

Issue

9

Start / End Page

1038 / 1046

Location

United States

Related Subject Headings

  • Splenectomy
  • Spleen
  • Registries
  • Pyrrolidines
  • Platelet Count
  • Organ Size
  • Middle Aged
  • Male
  • Longitudinal Studies
  • Liver