Research Article

Skeletal Manifestations, Bone Pain, and BMD Changes in Albanian Type 1 Gaucher Patients Treated with Taliglucerase Alfa

Table 1

General data of our patients.

Number of patients at baseline24

Number of patients at the end of study20

Gender (F-M)7/13 (end of the study)

Genotype19/p.Asn409Ser:p.His294Gln/pAsp448His
2/p.Asn409Ser:pArg 502His
1/p.Asn409Ser:pSer146Leu
1/p.Asn409Ser:p/Arg86
1/p. Asn409Ser: p/LeuProfs4: Arg87Trp

Age at baseline28.7 ± 16.5 (min age: 7 y, max age: 72 y)

Children (under 18 y)/adults8/16

Naïve patients8 patients

Switched patients12 patients switched at baseline (imiglucerase to taliglucerase)
2 patients switched during FU (taliglucerase to velaglucerase)

Splenectomized2