ICCBH2013 Poster Presentations (1) (201 abstracts)
Linear growth over 2 years of velaglucerase alfa therapy in children with type 1 Gaucher disease previously treated with imiglucerase
Ari Zimran 1 , Derralynn Hughes 2 , Deborah Elstein 1 , Laurie Smith 3 , Paul Harmatz 4 , William Rhead 5 , Pilar Giraldo 6 , Nancy Mendelsohn 7 , Chan-Hoo Park 8 , David Zahrieh 9 & Eric Crombez 9
1Gaucher Clinic, Hadassah Medical School, Shaare Zedek Medical Center and Hebrew University, Jerusalem, Israel; 2University College London, London, UK; 3Children’s Mercy Hospital, Kansas City, Missouri, USA; 4Children’s Hospital Oakland, California, USA; 5Children’s Hospital of Wisconsin, Milwaukee, Wisconsin, USA; 6Centro de Investigación Biomédica en Red de Enfermedades Raras and Hospital Universitario Miguel Servet, Zaragoza, Spain; 7Children’s Hospitals and Clinics of Minnesota and University of Minnesota, Minneapolis, Minnesota, USA; 8Gyeongsang National University Hospital, Jinju, Republic of Korea; 9Shire Human Genetic Therapies, Inc., Lexington, Massachusetts, USA.
Objectives: As children with confirmed type 1 Gaucher disease (inherited metabolic disorder) may have linear growth retardation, we evaluated linear growth over 2 years in children enrolled in the interventional study TKT034, in which patients receiving imiglucerase enzyme replacement therapy were switched to velaglucerase alfa.
Methods: Trial TKT034 enrolled patients who were ≥2 years of age with type 1 Gaucher disease and stable clinical parameters on imiglucerase therapy. They were allocated to velaglucerase alfa at the same dose as their previous imiglucerase dose. Eligible patients who had completed TKT034 (1 year) could enrol in an extension study (HGT-GCB-044). The height percentiles and height Z-scores of children were determined by gender and age (months) using WHO 2007 growth reference data (exploratory analysis).
Results: Nine children, who had previously received imiglucerase for ≥4 years, received velaglucerase alfa in both TKT034 and HGT-GCB-044 for a total of 2 years. One child was at or below the 50th percentile at baseline of TKT034 (Table). At baseline and 2 years respectively, the median (range) Z-scores were 0.65 (−1.11, 1.31) and 0.79 (−1.33, 1.38), corresponding to percentiles 74.2 and 78.5. Comparing Baseline with 2-year values, no child crossed a major percentile (3, 15, 50, 85, or 97) downward; the Z-score change in each child was no >±0.51 (half a S.D.); and the mean Z-score change was not significant (0.06 (95% CI −0.17, 0.30); P>0.05).
| Prior imiglucerase exposure | Tanner stage | Height (cm) | Percentile | Z-score | |||||||
| Baseline age (years) | Gender | Units/kg per 2 weeks | Duration (months) | Baseline | 2 years | Baseline | 2 years | Baseline | 2 years | Baseline | 2 years |
| 9 | Female | 35 | 69 | 1 | Not donea | 139.9 | 153.0 | 78.2 | 78.5 | 0.78 | 0.79 |
| 10 | Female | 51 | 65 | 1 | 3 | 147.0 | 161.5 | 70.9 | 81.6 | 0.55 | 0.90 |
| 12 | Female | 34 | 92 | 3 | 4 | 145.0 | 151.0 | 13.3 | 9.2 | −1.11 | −1.33 |
| 13 | Female | 56 | 54 | 5 | 5 | 165.6 | 171.5 | 82.6 | 91.1 | 0.94 | 1.35 |
| 13 | Male | 44 | 49 | 2 | 4 | 160.0 | 175.4 | 54.8 | 73.6 | 0.12 | 0.63 |
| 14 | Female | 57 | 95 | 4 | 4 | 170.6 | 170.2 | 90.5 | 86.4 | 1.31 | 1.10 |
| 14 | Female | 60 | 108 | 4 | 5 | 164.0 | 165.0 | 65.9 | 63.3 | 0.41 | 0.34 |
| 14 | Male | 47 | 93 | 4 | 5 | 169.8 | 175.8 | 74.2 | 62.2 | 0.65 | 0.31 |
| 16 | Female | 62 | 124 | 4 | 5 | 170.9 | 172.2 | 89.3 | 91.6 | 1.24 | 1.38 |
| a1 at 1 year. | |||||||||||
Conclusion: In nine children with type 1 Gaucher disease switched from long-term imiglucerase therapy, height relative to age- and gender-matched reference populations was stable on average over 2 years of velaglucerase alfa therapy.
Declaration of interest: A Zimran: consulting fees (Protalix Biotherapeutics); options in Protalix Biotherapeutics and Scientific Advisory Board membership; honoraria (Shire HGT, Actelion, Pfizer). A Zimran’s institution: support (Genzyme (ICGG Gaucher Registry), Shire HGT (GOS), Medison). D Hughes: consulting fees, travel and research grants, and honoraria for speaking (Shire HGT, Genzyme, Protalix, Amicus). D Elstein: reimbursement for travel expenses to meetings (Shire HGT). L Smith: research grants (Genzyme, Shire HGT, BioMarin); consultant fees (Shire HGT, BioMarin). P Harmatz: consulting support (Shire HGT); speakers’ honorarium and travel support (Shire HGT); advisory boards (Shire (HOS)); consulting support (BioMarin); advisory boards, research support, and travel and speakers’ honoraria (BioMarin); speakers’ honorarium (Genzyme). W Rhead: clinical trial support (TKT, Genzyme, Shire, Hyperion); speaker fees (Ucyclyd). P Giraldo: consulting fees (Shire HGT, Genzyme, Actelion). N Mendelsohn: travel funding (Genzyme, BioMarin, Shire); grant funding (Genzyme, BioMarin, Shire); seat on North American Advisory Board and publications committee for Shire. C-H Park: research grants (Genzyme, Shire HGT). D Zahrieh and E Crombez: Shire HGT employees.
Funding: TKT034 and HGT-GCB-044 were sponsored by Shire Human Genetic Therapies, Inc.
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