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Blood Cells Mol Dis. 2016 Jul 18. pii: S1079-9796(16)30087-0. doi: 10.1016/j.bcmd.2016.07.002. [Epub ahead of print]

Long-term efficacy and safety results of taliglucerase alfa through 5years in adult treatment-naïve patients with Gaucher disease.

Author information

1
Gaucher Clinic, Shaare Zedek Medical Center, Hebrew University and Hadassah Medical School, Jerusalem, Israel. Electronic address: azimran@gmail.com.
2
Pontificia Universidad Catolica de Chile, Santiago, Chile.
3
CIBERER, Hospital Universitario Miguel Servet, IIS Aragón, Zaragoza, Spain.
4
Rambam Medical Center, Haifa, Israel.
5
Sapienza University, Rome, Italy.
6
Clinical Center of Serbia, Clinic of Endocrinology, Diabetes and Metabolic Disease, Belgrade University Medical School, Belgrade, Serbia.
7
Centro Médico Nacional Siglo XXI, Servicio de Hematologia, Mexico City, Mexico.
8
Hospital de Especialidades No1, Leon, Mexico.
9
Faculty of Health Sciences, University of the Witwatersrand, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, South Africa.
10
Protalix BioTherapeutics, Carmiel, Israel.

Abstract

Taliglucerase alfa, the first available plant cell-expressed recombinant therapeutic protein, is an enzyme replacement therapy approved for Gaucher disease (GD). PB-06-001, a pivotal phase 3, multicenter, randomized, double-blind, parallel-dose study investigated taliglucerase alfa 30 or 60U/kg every other week through 9months in treatment-naïve adults with GD; 30-month extension study PB-06-003 followed. Patients completing PB-06-001 and PB-06-003 could continue treatment in PB-06-007. Nineteen patients enrolled in PB-06-007 (30U/kg, n=8; 60U/kg, n=9; dose adjusted, n=2); 17 completed 5 total years of treatment. In these 3 groups, respectively, taliglucerase alfa resulted in mean decreases in spleen volume (-8.7, -6.9, -12.4 multiples of normal), liver volume (-0.6, -0.4, -0.5 multiples of normal), chitotriosidase activity (-83.1%, -93.4%, -87.9%), and chemokine (CC motif) ligand 18 concentration (-66.7%, -83.3%, -78.9%), as well as mean increases in hemoglobin concentration (+2.1, +2.1, +1.8mg/dL) and platelet count (+31,871, +106,800, +34,000/mm3). The most common adverse events were nasopharyngitis and arthralgia. Most adverse events were mild/moderate; no serious adverse events were considered treatment-related. These results demonstrate continued improvement of disease parameters during 5years of taliglucerase alfa therapy in 17 treatment-naive patients with no new safety concerns, extending the taliglucerase alfa clinical efficacy and safety dataset. This study was registered at www.clinicaltrials.gov as NCT01422187.

KEYWORDS:

Anemia; Chemokine (CC motif) ligand 18; Chitotriosidase; Enzyme replacement therapy; Gaucher disease; Hepatomegaly; Splenomegaly; Taliglucerase alfa; Thrombocytopenia

PMID:
27499018
DOI:
10.1016/j.bcmd.2016.07.002
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