Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry.

Deegan, Patrick; Lau, Heather; Elstein, Deborah; Fernandez-Sasso, Diego; Giraldo, Pilar; Hughes, Derralynn; Zimran, Ari; Istaiti, Majdolen; Gadir, Noga; Botha, Jaco; Revel-Vilk, Shoshana

Deegan, Patrick; Lau, Heather; Elstein, Deborah; Fernandez-Sasso, Diego; Giraldo, Pilar; Hughes, Derralynn; Zimran, Ari; Istaiti, Majdolen; Gadir, Noga; Botha, Jaco; Revel-Vilk, Shoshana.

Afiliación

Deegan P; Lysosomal Disorders Unit, Cambridge University Hospitals, Hills Road, Cambridge CB2 0QQ, UK.
Lau H; Langone Medical Center, New York University, 333 E 33rd St, New York, NY 10016, USA.
Elstein D; Takeda Pharmaceuticals International AG, Thurgauerstrasse 130, 8152 Zurich, Switzerland.
Fernandez-Sasso D; Instituto William Osler, Paraguay 2935, Buenos Aires C1425 BRI, Argentina.
Giraldo P; En el Centro de Investigación Biomédica en Red (CIBER) de Enfermedades Raras, IIS Aragon, C. de San Juan Bosco 13, 50009 Zaragoza, Spain.
Hughes D; Translational Research Unit, IIS Aragon, Paseo de Isabel la Católica 1-3, 50009 Zaragoza, Spain.
Zimran A; Lysosomal Storage Disorders Unit, Department of Haematology, Royal Free Hospital, UCL Medical School, Pond Street, London NW3 2QG, UK.
Istaiti M; Gaucher Unit, Shaare Zedek Medical Center, Shmuel (Hans) Beyth St 12, Jerusalem 9103102, Israel.
Gadir N; The Faculty of Medicine, Hebrew University, Campus Ein Kerem, Jerusalem 9112102, Israel.
Botha J; Gaucher Unit, Shaare Zedek Medical Center, Shmuel (Hans) Beyth St 12, Jerusalem 9103102, Israel.
Revel-Vilk S; Takeda Pharmaceuticals International AG, Thurgauerstrasse 130, 8152 Zurich, Switzerland.

J Clin Med ; 13(10)2024 May 09.

Article en En | MEDLINE | ID: mdl-38792324

ABSTRACT

ABSTRACT

Background:

Gaucher disease (GD) is a rare, autosomal, recessive condition characterized by hepatosplenomegaly, thrombocytopenia, anemia, and bone abnormalities, often requiring life-long treatment. Velaglucerase alfa has improved hematologic and visceral parameters in clinical trials; however, limited long-term efficacy and safety data are available.

Methods:

The Gaucher Outcome Survey (GOS), a structured and validated international registry for patients with confirmed GD, provides an opportunity to evaluate long-term data from patients receiving velaglucerase alfa.

Results:

This analysis included 376 treatment-naïve children and adults with GD enrolled in GOS, including 20 with type 3 GD, who initiated velaglucerase alfa through participation in clinical trials or as part of their clinical management and continued treatment for a mean (range) time of 6.6 (0.003-18.6) years. Initial improvements in hematologic and visceral parameters and the biomarkers glucosylsphingosine (lyso-GL1) and chitotriosidase were observed after one year of treatment and were maintained throughout the follow-up period. Of 129 (34.3%) patients who developed adverse events during the follow-up period, events were considered related to treatment in 33 (8.8%). None led to treatment discontinuation. There were 21 deaths overall, none of which were considered related to treatment.

Conclusions:

This analysis of data from the GOS registry supports the safety and efficacy of velaglucerase alfa in patients with GD.

Palabras clave

ERT; GOS; Gaucher disease; enzyme replacement therapy; long term; registry; velaglucerase alfa

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: J Clin Med Año: 2024 Tipo del documento: Article Pais de publicación: Suiza