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Table 1 Characteristics of 9 GD patients who developed a GD associated malignancy

From: Modelling Gaucher disease progression: long-term enzyme replacement therapy reduces the incidence of splenectomy and bone complications

Complication

Treatment status at diagnosis

Disease phase at diagnosis

Genotype

Amyloidosis/MM

ERT <2 years

Multiple complications

N370S/L444P

HCC

ERT <2 years

Multiple complications

N370S/I260T

Amyloidosis/MM

untreated

Complication: Sx

N370S/L444P

HCC

ERT 6 years

Complication: Sx

R463C/?

Amyloidosis/MM

ERT 2 years

Multiple complications

N370S/R257Q

Amyloidosis/MM

ERT 15 years

Multiple complications

N370S/L444P

HCC

ERT >17 years

Multiple complications

N370S/L324P

Amyloidosis/MM

ERT 19 years

Multiple complications

N370S/G84GG

HCC

ERT 18 years

Multiple complications

N370S/N370S