Impact of velaglucerase alfa on bone marrow burden score in adult patients with type 1 Gaucher disease: 7-Year follow-up

Deborah Elstein, Andrew H. Haims, David Zahrieh, Gabriel M. Cohn, Ari Zimran

Research output: Contribution to journalArticle

10 Citations (Scopus)

Abstract

Background: Bone marrow infiltration by substrate-engorged "Gaucher" cells manifests early in Gaucher disease (GD). The impact of velaglucerase alfa on bone marrow burden (BMB) was evaluated as an exploratory assessment. Methods: BMB scores were assessed using T1- and T2-weighted magnetic resonance images of the lumbar spine (LS) and femora among symptomatic GD patients who participated in the 9-month Phase I/II trial and long-term extension study for velaglucerase alfa. A post-hoc assessment of marrow involvement was performed. BMB scores per site are 0-8 (0/1 = normal; 8 = severe infiltration). Results: The median LS-BMB score at baseline was 6 (n = 12; range 3-8); at 9. months, compared with baseline, there was a median change of - 2 (n = 11; two-sided p-value = 0.0078). LS-BMB scores continued to decrease through 5. years (n = 8; median change from baseline - 5 [p = 0.0078], median score 1 [range 1-4]) and were subsequently sustained through 7. years (n = 8). LS-BMB decreases of ≥ 2 points occurred in 6/11 patients at 9. months, and in all assessable patients (8/8) by 5. years. Long-term femoral BMB (F-BMB) assessment was possible for three patients; all experienced reductions of ≥ 2 points at 5. years with a total score (LS-BMB. +. F-BMB) decrease ≥ 4. Conclusions: This post hoc analysis suggests improvement in BMB scores through 5. years that was sustained through 7. years, despite dose reduction from 15. months. Prospective studies in a large cohort are needed to validate these findings.

Original languageEnglish (US)
Pages (from-to)56-60
Number of pages5
JournalBlood Cells, Molecules, and Diseases
Volume53
Issue number1-2
DOIs
StatePublished - Jan 1 2014
Externally publishedYes

Fingerprint

Gaucher Disease
Bone Marrow
Spine
Thigh
Femur
Magnetic Resonance Spectroscopy
Prospective Studies

Keywords

  • Bone marrow burden
  • Bone mineral density
  • Enzyme replacement therapy
  • Gaucher disease
  • MRI
  • Velaglucerase alfa

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Hematology
  • Cell Biology

Cite this

Impact of velaglucerase alfa on bone marrow burden score in adult patients with type 1 Gaucher disease : 7-Year follow-up. / Elstein, Deborah; Haims, Andrew H.; Zahrieh, David; Cohn, Gabriel M.; Zimran, Ari.

In: Blood Cells, Molecules, and Diseases, Vol. 53, No. 1-2, 01.01.2014, p. 56-60.

Research output: Contribution to journalArticle

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T1 - Impact of velaglucerase alfa on bone marrow burden score in adult patients with type 1 Gaucher disease

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AU - Haims, Andrew H.

AU - Zahrieh, David

AU - Cohn, Gabriel M.

AU - Zimran, Ari

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AB - Background: Bone marrow infiltration by substrate-engorged "Gaucher" cells manifests early in Gaucher disease (GD). The impact of velaglucerase alfa on bone marrow burden (BMB) was evaluated as an exploratory assessment. Methods: BMB scores were assessed using T1- and T2-weighted magnetic resonance images of the lumbar spine (LS) and femora among symptomatic GD patients who participated in the 9-month Phase I/II trial and long-term extension study for velaglucerase alfa. A post-hoc assessment of marrow involvement was performed. BMB scores per site are 0-8 (0/1 = normal; 8 = severe infiltration). Results: The median LS-BMB score at baseline was 6 (n = 12; range 3-8); at 9. months, compared with baseline, there was a median change of - 2 (n = 11; two-sided p-value = 0.0078). LS-BMB scores continued to decrease through 5. years (n = 8; median change from baseline - 5 [p = 0.0078], median score 1 [range 1-4]) and were subsequently sustained through 7. years (n = 8). LS-BMB decreases of ≥ 2 points occurred in 6/11 patients at 9. months, and in all assessable patients (8/8) by 5. years. Long-term femoral BMB (F-BMB) assessment was possible for three patients; all experienced reductions of ≥ 2 points at 5. years with a total score (LS-BMB. +. F-BMB) decrease ≥ 4. Conclusions: This post hoc analysis suggests improvement in BMB scores through 5. years that was sustained through 7. years, despite dose reduction from 15. months. Prospective studies in a large cohort are needed to validate these findings.

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