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Improvement in bone marrow infiltration in patients with type I Gaucher disease treated with taliglucerase alfa
Author(s) -
Zimran Ari,
Dinur Tama,
RevelVilk Shoshana,
Akkerman Eric M.,
Dussen Laura,
Hollak Carla E. M.,
Maayan Hannah,
Altarescu Gheona,
Chertkoff Raul,
Maas Mario
Publication year - 2018
Publication title -
journal of inherited metabolic disease
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.462
H-Index - 102
eISSN - 1573-2665
pISSN - 0141-8955
DOI - 10.1007/s10545-018-0195-y
Subject(s) - medicine , enzyme replacement therapy , bone marrow , gastroenterology , nuclear medicine , surgery , disease
Preliminary data suggest a positive effect of taliglucerase alfa on the bone marrow infiltration of Gaucher cells. In this investigator‐initiated study, we report the impact of taliglucerase alfa on the bone marrow fat fraction (FF) in 26 patients assessed by quantitative chemical shift imaging (QCSI). Of 15 treatment‐naïve patients (median age 48 [range 24–68] years), eight had baseline FF ≤ 0.3, six of those with a FF ≤ 0.23 (‘bone at risk’). All significantly improved from a median baseline FF of 0.24 (0.15–0.32) to 1st year FF of 0.37 (0.25–0.54) and 2nd year FF of 0.42 (0.27–0.59) ( p = 0.01). Among the 11 ‘switch‐over’ patients (median age 42 [range 33–69] years; median imiglucerase exposure 8 [range 1–17] years), eight had baseline FF ≤ 0.3, five of those with FF < 0.23. All, but one, significantly improved from a median baseline FF of 0.17 (0.08–0.28) to 1st year FF of 0.3 (0.05–0.34) and 2nd year FF of 0.34 (0.08–0.44) ( p = 0.03). Two elderly female patients (age 43 and 58 years, with 17 years imiglucerase exposure) who remained at the same enzyme replacement therapy dose, increased from baseline FF of 0.13 and 0.19 to 0.26 at 1 year. Although the number of observations is small, we hypothesize that switching to taliglucerase may result in an improved bone marrow response. A larger study is needed to assess the early benefit of taliglucerase alfa in adult patients with type 1 Gaucher disease on the bone marrow compartment.