Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease

Deborah Elstein, Atul Mehta, Derralynn A. Hughes, Pilar Giraldo, Joel Charrow, Laurie Smith, Suma Shankar, Thomas N. Hangartner, Yune Kunes, Nan Wang, Eric Crombez, Ari Zimran

Research output: Contribution to journalArticle

9 Citations (Scopus)

Abstract

Gaucher disease (GD) is a lysosomal storage disorder; symptomatic patients with type 1 GD need long-term disease-specific therapy of which the standard of care has been enzyme replacement therapy (ERT). Thirty-eight of 40 patients (aged 9-71 years) clinically stable on ERT with imiglucerase, safely switched to a comparable dose of velaglucerase alfa (units/kg) during TKT034, a 12-month, open-label clinical study, and for 10-50 months in an extension study. The most common adverse events (AEs) judged to be drug-related in the extension were fatigue and bone pain. No drug-related serious AEs were reported. No AEs led to study withdrawal. At 24 months from baseline (baseline being TKT034 week 0), patients had generally stable hemoglobin, platelet, spleen, liver, and bone density parameters. Nevertheless, dose adjustment based on the achievement of therapeutic goals was permitted, and 10 patients, including seven patients who had platelet counts <100 × 109/L at baseline, were given at least one 15 U/kg-dose increase during the extension. Trends indicative of improvement in platelet count and spleen volume, and decreasing levels of GD biomarkers, chitotriosidase and CCL18, were observed. Immunogenicity was seen in one patient positive for anti-imiglucerase antibodies at baseline. This patient tested positive for anti-velaglucerase alfa antibodies in TKT034, with low antibody concentrations, and throughout the extension study; however, the patient continued to receive velaglucerase alfa without clinical deterioration. In conclusion, clinically stable patients can be switched from imiglucerase to velaglucerase alfa ERT and maintain or achieve good therapeutic outcomes.

Original languageEnglish (US)
Pages (from-to)592-597
Number of pages6
JournalAmerican Journal of Hematology
Volume90
Issue number7
DOIs
StatePublished - Jan 1 2015
Externally publishedYes

Fingerprint

Gaucher Disease
Safety
Enzyme Replacement Therapy
Therapeutics
Platelet Count
Spleen
Social Adjustment
imiglucerase
Antibodies
Standard of Care
Pharmaceutical Preparations
Bone Density
Fatigue
Anti-Idiotypic Antibodies
Hemoglobins
Blood Platelets
Biomarkers
Bone and Bones
Pain

ASJC Scopus subject areas

  • Hematology

Cite this

Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease. / Elstein, Deborah; Mehta, Atul; Hughes, Derralynn A.; Giraldo, Pilar; Charrow, Joel; Smith, Laurie; Shankar, Suma; Hangartner, Thomas N.; Kunes, Yune; Wang, Nan; Crombez, Eric; Zimran, Ari.

In: American Journal of Hematology, Vol. 90, No. 7, 01.01.2015, p. 592-597.

Research output: Contribution to journalArticle

Elstein, D, Mehta, A, Hughes, DA, Giraldo, P, Charrow, J, Smith, L, Shankar, S, Hangartner, TN, Kunes, Y, Wang, N, Crombez, E & Zimran, A 2015, 'Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease', American Journal of Hematology, vol. 90, no. 7, pp. 592-597. https://doi.org/10.1002/ajh.24007
Elstein, Deborah ; Mehta, Atul ; Hughes, Derralynn A. ; Giraldo, Pilar ; Charrow, Joel ; Smith, Laurie ; Shankar, Suma ; Hangartner, Thomas N. ; Kunes, Yune ; Wang, Nan ; Crombez, Eric ; Zimran, Ari. / Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease. In: American Journal of Hematology. 2015 ; Vol. 90, No. 7. pp. 592-597.
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AU - Smith, Laurie

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AU - Crombez, Eric

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