A Real-World Investigation of MRI Changes in Bone in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: The EIROS Study

Author:

Bengherbia Monia1,Berger Marc2,Hivert Bénédicte3ORCID,Rigaudier Florian4,Bracoud Luc5ORCID,Vaeterlein Ole6,Yousfi Karima1,Maric Michele7,Malcles Marie7,Belmatoug Nadia1ORCID

Affiliation:

1. Department of Internal Medicine, Referral Center for Lysosomal Diseases, Beaujon Hospital, AP-HP, Université Paris Cité, 92110 Clichy, France

2. Department of Biological and Clinical Hematology, Estaing Hospital, CHU Clermont-Ferrand, 63000 Clermont-Ferrand, France

3. Department of Hematology, Saint Vincent de Paul Hospital, GHICL, 59000 Lille, France

4. CEN Biotech, 21000 Dijon, France

5. Clario Inc. (Formerly Bioclinica, Inc.), 69006 Lyon, France

6. Clario Inc. (Formerly Bioclinica, Inc.), 20355 Hamburg, Germany

7. Takeda France SAS, 75116 Paris, France

Abstract

Background/Objectives: Gaucher disease type 1 (GD1) is characterized by hepatosplenomegaly, thrombocytopenia, and disabling bone manifestations requiring regular MRI monitoring. The EIROS study assessed the real-world impact of velaglucerase alfa on GD1 bone disease, using MRI data collected in French clinical practice. Methods: MRIs collected retrospectively from treatment initiation and prospectively during follow-up (12-months) were analyzed centrally by a blinded expert radiologist to evaluate bone infiltration using the Bone Marrow Burden (BMB) score and a qualitative method (stable, improved or worsened for the spine and femur). Abdominal MRIs were also centrally analyzed to assess hepatosplenomegaly. Bone manifestations, hepatosplenomegaly, and hematologic parameters were analyzed from medical records. Results: MRI data were available for 20 patients: 6 treatment-naive patients and 14 patients who switched to velaglucerase alfa from another GD treatment. Interpretable MRIs for BMB scoring were available for seven patients for the spine and one patient for the femur. Qualitative assessments (n = 18) revealed stability in spine and femur infiltration in 100.0% and 84.6% of treatment-switched patients (n = 13), respectively, and improvements in 80.0% and 60.0% of treatment-naive patients (n = 5), respectively; no worsening of bone infiltration was observed. Liver, spleen, and hematologic parameters improved in treatment-naive patients and remained stable in treatment-switched patients. Conclusions: The qualitative real-world data support findings from clinical trials suggesting the long-term effectiveness of velaglucerase alfa on GD1 bone manifestations. When MRI assessment by radiologists with experience of GD is not possible, a simplified qualitative assessment may be sufficient in clinical practice for monitoring bone disease progression and treatment response.

Funder

Takeda France SAS

Publisher

MDPI AG

Reference57 articles.

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2. Gaucher disease epidemiology and natural history: A comprehensive review of the literature;Nalysnyk;Hematology,2017

3. Gaucher Disease in Bone: From Pathophysiology to Practice;Hughes;J. Bone Miner. Res.,2019

4. EMA (2022, November 01). European Medicines Agency Summary of Product Characterisics for Imiglucerase. Available online: https://www.ema.europa.eu/en/medicines/human/EPAR/cerezyme.

5. EMA (2022, November 01). European Medicines Agency Summary of Product Characterisics for Velaglucerase Alfa. Available online: https://www.ema.europa.eu/en/medicines/human/EPAR/vpriv.

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